Page last updated: 2024-09-27

mycophenolic acid

Description

Mycophenolic Acid: Compound derived from Penicillium stoloniferum and related species. It blocks de novo biosynthesis of purine nucleotides by inhibition of the enzyme inosine monophosphate dehydrogenase (IMP DEHYDROGENASE). Mycophenolic acid exerts selective effects on the immune system in which it prevents the proliferation of T-CELLS, LYMPHOCYTES, and the formation of antibodies from B-CELLS. It may also inhibit recruitment of LEUKOCYTES to sites of INFLAMMATION. [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

mycophenolate : A monocarboxylic acid anion resulting from the removal of a proton from the carboxy group of mycophenolic acid. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

mycophenolic acid : A member of the class of 2-benzofurans that is 2-benzofuran-1(3H)-one which is substituted at positions 4, 5, 6, and 7 by methyl, methoxy, (2E)-5-carboxy-3-methylpent-2-en-1-yl, and hydroxy groups, respectively. It is an antibiotic produced by Penicillium brevi-compactum, P. stoloniferum, P. echinulatum and related species. An immunosuppressant, it is widely used (partiularly as its sodium salt and as the 2-(morpholin-4-yl)ethyl ester prodrug, mycophenolate mofetil) to prevent tissue rejection following organ transplants and for the treatment of certain autoimmune diseases. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Cross-References

ID SourceID
PubMed CID446541
CHEMBL ID866
CHEBI ID168396
CHEBI ID92545
SCHEMBL ID4549
SCHEMBL ID2514376
MeSH IDM0014304

Synonyms (208)

Synonym
MLS001074701
smr000471887
BIDD:GT0456
AC-4491
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydro-2-benzofuran-5-yl)-4-methylhex-4-enoic acid
a-249 ,
AB00052466-14
AB00052466-13
BRD-K63750851-001-06-6
4-hexenoic acid,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5- isobenzofuranyl)-4-methyl-, (e)-
lilly 68618
4-hexenoic acid, (e)-
nsc129185
24280-93-1
mycophenolic acid
nsc-129185
melbex
SDCCGMLS-0066618.P001
PRESTWICK2_000556
BPBIO1_000695
acido micofenolico [inn-spanish]
micofenolico acido [spanish]
acide mycophenolique [inn-french]
einecs 246-119-3
acidum mycophenolicum [inn-latin]
-hexenoic acid, 6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-, (e)-
PRESTWICK_817
NCGC00016786-02
tocris-1505
tnp00198 ,
NCGC00025190-01
NCGC00016786-01
cas-24280-93-1
IDI1_000146
ACON1_000496
PRESTWICK3_000556
(e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1h-isobenzofuran-5-yl)-4-methyl-hex-4-enoic acid
lilly-68618
6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-4-hexenoic acid
m 5255
mycophenolic acid (usan/inn)
mycophenolic acid (tn)
D05096
MEGXM0_000120
BSPBIO_000631
ACON0_000960
4-hexenoic acid, 6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-, (e)- (8ci)
nsc 129185
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoic acid
ly 68618
(e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoic acid
4-hexenoic acid, 6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-, (e)-
6-(1,3-dihydro-7-hydroxy-5-methoxy-4-methyl-1-oxoisobenzofuran-6-yl)-4-methyl-4-hexanoic acid
(e)-6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-4-hexenoic acid
ccris 5565
4-methyl-5-methoxy-7-hydroxy-6-(5-carboxy-3-methylpent-2-en-1-yl)-phthalide (e)-
MOA ,
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthanlanyl)-4-methyl-4-hexanoic acid
4-hexenoic acid, 6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-, (4e)- (9ci)
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoic acid (e)-
mycophenolsaeure
4-hexenoic acid, 6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-, (e)-
UPCMLD-DP028:001
mycophenolic acid, powder, bioreagent, suitable for cell culture, >=98%
mycophenolic acid, >=98%
1JR1
mycophenolate
UPCMLD-DP028
DB01024
SPECTRUM5_001654
NCGC00025190-03
NCGC00025190-02
NCGC00025190-07
NCGC00025190-08
NCGC00025190-04
SPECTRUM1500674
BSPBIO_002534
NCGC00025190-05
NCGC00016786-03
NCGC00025190-09
mycophenolic acid (mpa)
bdbm19264
(4e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydro-2-benzofuran-5-yl)-4-methylhex-4-enoic acid
chembl866 ,
(e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1h-2-benzofuran-5-yl)-4-methylhex-4-enoic acid
HMS2089A17
HMS2092G22
acidum mycophenolicum
micofenolico acido
CHEBI:168396 ,
acide mycophenolique
acido micofenolico
MLS002695945
rs-61443 [as mofetil]
mycophenolate mofetil impurity, mycophenolic acid-
MLS002222265
HMS500H08
HMS1921A18
HMS1569P13
483-60-3
NCGC00025190-10
HMS2096P13
(4e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxohydroisobenzofuran-5-yl)-4-methylhex -4-enoic acid
M2216
1162256-90-7
C20380
(e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydroisobenzofuran-5-yl)-4-methylhex-4-enoic acid
A817192
nsc757424
nsc-757424
pharmakon1600-01500674
dtxsid4041070 ,
dtxcid2021070
tox21_110610
HMS2268H22
CCG-39914
NCGC00016786-11
NCGC00016786-06
NCGC00016786-04
NCGC00016786-09
NCGC00016786-12
NCGC00016786-07
NCGC00016786-10
NCGC00016786-08
NCGC00016786-05
ec 246-119-3
unii-hu9dx48n0t
mycophenolic acid [usan:inn:ban]
hu9dx48n0t ,
BCP9000970
mycophenoic acid
6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methylhex-4-enoic acid
einecs 207-595-8
mycophenolate mofetil impurity f [ep impurity]
4-hexenoic acid, 6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-, (4e)-
mycophenolic acid [who-dd]
mycophenolate mofetil impurity, mycophenolic acid- [usp impurity]
mycophenolic acid [inn]
mycophenolic acid [vandf]
mycophenolic acid [usan]
mycophenolate [vandf]
mycophenolic acid [orange book]
mycophenolic acid [mi]
mycophenolic acid [mart.]
AKOS015888214
S2487
myfortic (mycophenolate sodium)
gtpl6832
HY-B0421
SCHEMBL4549
tox21_110610_1
NCGC00016786-15
(e)-6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxoisobenzofuran-5-yl)-4-methyl-4-hexenoic acid
(e)-6-(1,3-dihydro-4-hydroxy-6- methoxy-7-methyl-3-oxoisobenzofuran-5-yl)-4-methyl-4-hexenoic acid
e-6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-4-hexenoic acid
SCHEMBL2514376
STL419986
BBL034696
DS-1638
HB3987
HMS3403P09
AB00052466_15
AB00052466_16
mfcd00036814
mycophenolic acid, analytical standard
EX-A975
SR-01000597602-1
SR-01000597602-4
sr-01000597602
SR-01000597602-3
CHEBI:92545
(4e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydroisobenzofuran-5-yl)-4-methylhex-4-enoic acid; mycophenolate mofetil imp. f (ep); mycophenolic acid; mycophenolate mofetil impurity f
SBI-0051945.P003
SW196951-2
'6-(1,3-dihydro-7-hydroxy-5-methoxy-4-methyl-1-oxoisobenzofuran-6-yl)-4-methyl-4-hexanoic acid'
mycophenolate;rs-61443
mycophenolate; myfortic; melbex
BCP05321
Q420553
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1h-isobenzofuran-5-yl)-4-methyl-4-hexenoic acid
mycophenolic acid - cas 24280-93-1
mycophenolic (mycophenolate) ,
HMS3676F09
HMS3412F09
BRD-K63750851-001-13-2
EN300-122327
4-hexenoic acid,6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-, (4e)-
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydroisobenzofuran-5-yl)-4-methylhex-4-enoic acid
mycophenolic-acid
AMY40494
M02087
(4e)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydroisobenzofuran-5-yl)-4-methylhex-4-enoic acid (mycophenolic acid)
mycophenolic acid 100 microg/ml in acetonitrile
BM164624
EN300-1273238
STARBLD0040186
6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoic acid;nsc 129185
mpa - mycophenolate
Z2315575694
mycophenolate mofetil impurity, mycophenolic acid-(usp impurity)
mycophenilic acid
mycophenolate mofetil impurity f (ep impurity)
l04aa06
acide mycophenolique (inn-french)
acido micofenolico (inn-spanish)
acidum mycophenolicum (inn-latin)
mycophenolsaure
mycophenolic acid (mart.)

Research Excerpts

Overview

ExcerptReference
"Mycophenolic acid (MPA) is a potent inhibitor of IMPDHs."( Iwamori, K; Mitsuhashi, S; Nakajima, N; Takenaka, J; Ubukata, M, 2010)
"Mycophenolic acid (MPA) is an immunosuppressive agent commonly used after organ transplantation. "( Akhlaghi, F; Court, MH; Dostalek, M; Hazarika, S, 2011)
"Mycophenolic acid (MPA) is a common immunosuppressive drug for kidney transplantation patients, and is characterized by a narrow therapeutic index and significant individual variability. UGTs are the main enzymes responsible for the metabolism of MPA. "( Hu, N; Jiang, Z, 2021)
"Mycophenolic acid (MPA) is a widely used immunosuppressant in transplantation and autoimmune disease. "( Kiang, TKL; Patel, V; Rong, Y, 2021)
"Mycophenolic acid (MPA) is an occurring antibiotic produced through Penicillium brevicompactum. "( Bilal, M; Li, M; Li, X; Wu, Q; Yang, Y; Zhang, J, 2022)
"• Mycophenolic acid is an inhibitor of inosine-5'-monophosphate dehydrogenase, expressed in lymphocytes; therefore, MMF could impair the immune system function."( Azzari, C; Barbati, F; Canessa, C; Ferrara, G; Lodi, L; Maccora, I; Marrani, E; Mastrolia, MV; Ricci, S; Simonini, G; Volpi, B, 2022)
"Mycophenolic acid (MP) is an active metabolite of mycophenolate mofetil, a widely used immunosuppressive drug. "( Kawai, A; Kuyama, K; Nishi, K; Otagiri, M; Teshima, H; Tsukigawa, K; Yamasaki, K; Yukizawa, R, 2023)
"Mycophenolic acid (MPA) is an immunosuppressant that is used as an adjunct therapy in renal, liver, and heart transplantation. "( Frazee, C; Garg, U; Munar, A, 2023)
"Mycophenolic acid (MPA) is an immunosuppressant commonly prescribed in pediatric kidney transplantation to prevent graft rejection. "( Hamiwka, L; Kiang, TKL; Rong, Y; Wichart, J, 2023)
"Mycophenolic acid (MPA) is a potent natural product inhibitor of fungal and other eukaryotic inosine 5'-monophosphate dehydrogenases (IMPDHs) originally isolated from spoiled corn silage. "( Freedman, R; Hedstrom, L; Sarkis, AW; Yu, R, 2020)
"Mycophenolic acid (MPA) is an immunosuppressive agent commonly prescribed during posttransplant periods for the prevention of acute and chronic rejection following organ transplantation. "( Benjanuwattra, J; Koonrungsesomboon, N; Pruksakorn, D, 2020)
"Mycophenolic acid (MPA) is a noncompetitive inhibitor of inosine monophosphate dehydrogenase (IMPDH) and approved for prophylaxis of acute rejection after kidney, heart, and liver transplantation, where it has become a part of the standard therapy."( Böhnisch, S; Gotthardt, D; Mehrabi, A; Metzendorf, N; Neuberger, M; Rupp, C; Sommerer, C; Stremmel, W; Weiss, KH; Zeier, M, 2020)
"Mycophenolic acid (MPA) is an immunosuppressant commonly used to prevent renal transplant rejection and treat glomerulonephritis. "( Cheng, YH; Collins, KS; Dagher, PC; Dollins, MD; Eadon, MT; Ferreira, RM; Gao, H; Janosevic, D; Khan, NA; White, C, 2020)
"Mycophenolic acid (MPA) is a commonly used immunosuppressant in this setting."( Antonucci, M; Benagli, C; Cantù, M; D'Avolio, A; De Nicolò, A; Della Bruna, R; Ianniello, A; Keller, F, 2020)
"Mycophenolic acid (MPA) is a standard immunosuppressant in organ transplantation. "( Bachmann, F; Budde, K; Eckardt, KU; Friedersdorff, F; Gaedeke, J; Glander, P; Hambach, P; Kron, S; Liefeldt, L; Lorkowski, C; Mai, M; Neumayer, HH; Peters, R; Rudolph, B; Schmidt, D; Waiser, J; Wu, K, 2021)
"Mycophenolic acid (MPA) is an effective oral immunosuppressive drug used to treat lupus nephritis (LN), which exhibits large pharmacokinetic variability. "( Abud-Mendoza, C; Martínez-Martínez, MU; Medellín-Garibay, SE; Milán-Segovia, RDC; Reséndiz-Galván, JE; Romano-Aguilar, M; Romano-Moreno, S, 2020)
"Mycophenolic acid (MPA) is an immunosuppressor commonly used in transplantation, and its effect on DCs has not been fully investigated."( Bestard, O; Cerezo, G; Cruzado, JM; Fontova, P; Grinyó, JM; Llaudó, I; Lloberas, N; Manzano, A; Rama, I; Torras, J; Vidal-Alabró, A, 2021)
"Mycophenolic acid (MPA) is an immunomodulating agent commonly used in human medicine for the treatment of immune-mediated diseases. "( Anderson, WH; Klotsman, M; Sathyan, G, 2021)
"Mycophenolic acid (MPA) is an immunosuppressive drug commonly used for prophylaxis of graft rejection in solid organ transplant recipients. "( Hanprasertpong, N; Hirunsatitpron, P; Koonrungsesomboon, N; Noppakun, K; Pruksakorn, D; Teekachunhatean, S, 2022)
"Mycophenolic acid (MPA) is an immunosuppressant drug commonly used to prevent organ rejection in transplanted patients. "( Arola, HO; Benito-Peña, E; Iljin, K; Luque-Uría, Á; Moreno-Bondi, MC; Nevanen, TK; Peltomaa, R, 2021)
"Mycophenolic Acid (MPA) is an immunosuppressive drug widely used in the treatment of organ transplantation and autoimmune diseases. "( Gu, M; Guo, M; Meng, L; Tan, RY; Wang, ZJ; Wei, JF; Yang, HW, 2018)
"Mycophenolic acid is a drug widely used for HXGPRT positive selection of Toxoplasma mutant strains along with xanthine incubation in the culture medium; under such conditions, formation of tissue cysts has not been reported."( Castro-Elizalde, KN; Gómez de León, CT; González-Pozos, S; Hernández-Contreras, P; Mondragón-Castelán, M; Mondragón-Flores, R; Ramírez-Flores, CJ, 2018)
"Mycophenolic acid (MPA) is an approved immunosuppressive agent widely prescribed to prevent rejection after kidney transplantation. "( Ahmed, MA; Brundage, RC; Israni, AK; Jacobson, PA; Okour, M, 2018)
"Mycophenolic acid (MPA) is an immunosuppressant that is used in renal, liver, and heart transplantation. "( Frazee, C; Garg, U; Munar, A, 2018)
"Mycophenolic acid (MPA) is a potent inosine-5′-monophosphate dehydrogenase (IMPDH) inhibitor for immunosuppressive chemotherapy. "( Hu, Z; Li, F; Lin, S; Liu, M; Sun, W; Wang, J; Xue, Y; Yang, B; Zhang, Q; Zhang, Y; Zhu, H, 2018)
"Mycophenolic acid (MPA) is a currently widely used immunosuppressant."( Boor, PPC; Cao, W; Chen, K; Felczak, K; Hernanda, PY; Kwekkeboom, J; Liu, J; Ma, B; Ma, Z; Metselaar, HJ; Pan, Q; Pankiewicz, KW; Peppelenbosch, MP; Sheng, J; Sprengers, D; Tjon, ASW, 2019)
"Mycophenolic acid (MPA) is a group of metabolite derived from several species of "( Lan, B; Liu, J; Luo, M; Luo, X; Mei, X; Song, X; Tu, R; Wu, S; Zhang, L, 2020)
"Mycophenolic acid (MPA) is an immunosuppressive agent widely used in the treatment of solid organ transplant rejection. "( da Silva Cunha, A; de Oliveira Fulgêncio, G; Martins Duarte Byrro, R; Pianetti, GA; Rocha Chellini, P, 2013)
"Mycophenolic acid (MPA) is an immunosuppressant widely used to prevent organ rejection after organ transplantation. "( Capron, A; Mourad, M; Nguyen Thi, MT; Wallemacq, P, 2013)
"Mycophenolic acid (MPA) is an antirejection drug used in various types of solid organ transplants. "( Ensom, MH; Kiang, TK; Ng, K, 2013)
"Mycophenolic acid (MPA) is a commonly used immunosuppressive drug for human islet transplantation. "( Mahato, RI; Miller, D; Pagadala, J; Wu, H; Yates, CR, 2013)
"Mycophenolic acid (MPA) is a potent immunosuppressant agent, which is increasingly being used in the treatment of patients with various autoimmune diseases. "( Abd Rahman, AN; Staatz, CE; Tett, SE, 2014)
"Mycophenolic acid (MPA) is an immunosuppressant used in transplant rejection, often in combination with cyclosporine (CsA) and tacrolimus (Tac). "( El-Sheikh, AA; Koenderink, JB; Masereeuw, R; Russel, FG; van den Broek, PH; Verweij, VG; Wouterse, AC, 2014)
"Mycophenolic acid (MPA) is a key immunosuppressive drug that acts through inhibition of inosine monophosphate dehydrogenase (IMPDH). "( Cox, S; Fukuda, T; Goebel, J; Sherbotie, JR; Smits, TA; Vinks, AA; Ward, RM, 2014)
"Mycophenolic acid (MPA) is a promising drug owing to its immunosuppressive and biological activities. "( Ahmed, AS; El-Sayed, el-SR; Ismaiel, AA, 2014)
"Mycophenolic acid (MPA, 1) is a clinically important immunosuppressant. "( Bai, F; Cao, S; Huang, S; Li, S; Li, Z; Liu, C; Qi, F; Qiu, L; Wan, X; Yang, Y; Zhang, W, 2015)
"Mycophenolic acid (MPA) is an active metabolite of mycophenolate mofetil, a new immunosuppressive drug effective in the treatment of canine autoimmune diseases. "( Archer, J; Cywińska, A; Guzera, M; Szulc-Dąbrowska, L; Winnicka, A, 2016)
"Mycophenolic acid (MPA) is an important immunosuppressant broadly used in renal transplantation. "( Cooper, BR; Decker, BS; Favus, MJ; Liu, W; Lu, Y; Overholser, BR; Peng, Z; Shen, B; Sun, X; Tang, H; Wang, H; Wang, X; Zhong, L; Zhou, C, 2016)
"Mycophenolic acid (MPA) is an immunosuppressive agent which is commonly used in a fixed dose regime in solid organ transplantation. "( Bauer, S; Budde, K; Glander, P; Hambach, P; Mai, M; Rissling, O; Shipkova, M, 2016)
"Mycophenolic acid (MPA) is a secondary metabolite produced by various Penicillium species including Penicillium roqueforti. "( Coton, E; Coton, M; Debaets, S; Dominguez-Santos, R; Gillot, G; Hidalgo, PI; Jany, JL; Poirier, E; Ullán, RV, 2017)
"Mycophenolic acid (MPA) is a potentially useful immunosuppressant drug in cats and dogs, because of its well-documented efficacy in controlling autoimmune disease in humans."( Court, MH; Hwang, JK; Rivera, SM; Slovak, JE; Villarino, NF, 2017)
"Mycophenolic acid (MPA) is an effective immunosuppressive treatment for renal transplant recipients, but its effective use and best practice are not established in cardiac transplantation. "( Dengler, T; Hetzer, R; Hummel, M; Kobashigawa, J; Ladenburger, S; Lehmkuhl, H; Rothenburger, M; Sack, F, 2008)
"Mycophenolic acid (MPA) is an inhibitor of cellular inosine monophosphate dehydrogenase (IMPDH), an enzyme that catalyzes an essential step in the biosynthesis of GTP."( Estepa, A; Marroquí, L; Perez, L, 2008)
"Mycophenolic acid (MPA) is a potent inhibitor of the inosine monophosphate dehydrogenase and used as an immunosuppressive drug in transplantation. "( Goulimari, P; Grosse, R; Hahn, M; Krautkramer, E; Lichter, P; Morath, C; Muranyi, W; Reuter, H; Schwenger, V; Simon, V; Zeier, M, 2008)
"Mycophenolic acid (MPA) is an immunosuppressant drug commonly used in patients undergoing solid organ transplant. "( Montgomery, E; Nguyen, T; Park, JY; Scudiere, JR, 2009)
"Mycophenolic acid (MPA) is a selective inhibitor of inosine 5'-monophosphate dehydrogenase (IMPDH), the rate-limiting enzyme of de novo synthesis of guanine nucleotides. "( Bajari, T; Herkner, H; Sengoelge, G; Sunder-Plassmann, G; Weigel, G; Winnicki, W; Winter, B, 2010)
"Mycophenolic acid (MPA) is a potent, selective, noncompetitive and reversible inhibitor of inosine-5'-monophosphate deshydrogenase (IMPDH). "( Hidalgo, M; Jimeno, A; Villarroel, MC, 2009)
"* Mycophenolic acid (MPA) is a potent, selective and reversible inhibitor of inosine 5'-monophosphate dehydrogenase (IMPDH), the rate-limiting enzyme for de novo guanosine triphosphate biosynthesis. "( Chiarelli, LR; Cosmai, L; Dal Canton, A; Libetta, C; Molinaro, M; Regazzi, M; Tinelli, C; Valentini, G, 2010)
"Mycophenolic acid (MPA) is an immunosuppressant drug which powerfully inhibits lymphocyte proliferation. "( Götting, C; Kleesiek, K; Kuhn, J, 2010)
"Mycophenolic acid (MPA) is a cornerstone immunosuppressant therapy in solid organ transplantation. "( Barraclough, KA; Lee, KJ; Staatz, CE, 2010)
"Mycophenolic acid (MPA) is an immunosuppressive drug which induces resistance to several maturation signals in human dendritic cells (DC) by unknown mechanisms. "( Baron, C; Faugaret, D; Lebranchu, Y; Lemoine, R; Velge-Roussel, F, 2010)
"Mycophenolic acid is a commonly used immunosuppressant after organ transplantation and in autoimmune diseases; however, myelosuppression is a major complication despite its largely favorable side-effect profile. "( Ley, K; Ouyang, H; von Vietinghoff, S, 2010)
"Mycophenolic acid (MPA) is a basis for the immunosuppressive drugs used in clinic against rejection in solid organs transplantations. "( Cholewinski, G; Dzierzbicka, K; Malachowska-Ugarte, M, 2010)
"Mycophenolic acid (MPA) is an effective treatment for active lupus nephritis despite its variable efficacy in different ethnic groups. "( Avihingsanon, Y; Kittanamongkolchai, W; Lertdumrongluk, P; Somparn, P; Traitanon, O; Vadcharavivad, S, 2010)
"Mycophenolic acid (MPA) is an inosine monophosphate dehydrogenase inhibitor used for glomerulonephritis treatment. "( Joy, MS; Sam, WJ, 2010)
"Mycophenolic acid (MPA) therapy is a fundamental component of most post-transplant immunosuppressive regimens. "( Budde, K; Dürr, M; Glander, P; Liefeldt, L; Neumayer, HH, 2010)
"Mycophenolic acid (MPA) is an immunosuppressive drug widely used in the prevention of acute rejection in pediatric renal transplant recipients and is characterized by a wide inter-individual variability in its pharmacokinetics. "( Armstrong, VW; Marquet, P; Oellerich, M; Prémaud, A; Rousseau, A; Tönshoff, B; Urien, S; Weber, LT, 2011)
"Mycophenolic acid (MPA) is an immunosuppressant for which therapeutic drug monitoring (TDM) is performed for optimal prophylaxis and avoidance of toxicity in transplant patients. "( Decavele, AS; Favoreel, N; Heyden, FV; Verstraete, AG, 2011)
"MPA (mycophenolic acid) is an immunosuppressive drug produced by several fungi in Penicillium subgenus Penicillium. "( Frisvad, JC; Genee, HJ; Hansen, BG; Hedstrom, L; Kaas, CS; Mortensen, UH; Nielsen, JB; Sun, XE, 2012)
"Mycophenolic acid (MPA) is a highly effective immunosuppressant that has broad antiviral activity against different viruses and can act in synergy with interferon-α (IFN-α) on hepatitis C virus (HCV) replication. "( de Jonge, J; de Ruiter, PE; Janssen, HL; Kwekkeboom, J; Metselaar, HJ; Pan, Q; Tilanus, HW; van der Laan, LJ, 2012)
"Mycophenolic acid is a prodrug which is rapidly de-esterified in the gut wall, blood, liver and tissue to the active moiety, mycophenolic acid (MPA)."( Dupuis, R; Innocenti, F; Yuen, A, 2012)
"Mycophenolic acid (MPA) is an immunosuppressant."( Agarwal, SK; Guleria, S; Gupta, YK; Kaleekal, T; Reeta, KH; Sarangi, SC, 2012)
"Mycophenolic acid (MPA) is a fungal secondary metabolite and the active component in several immunosuppressive pharmaceuticals. "( Hansen, BG; Larsen, TO; Mnich, E; Mortensen, UH; Nielsen, JB; Nielsen, KF; Nielsen, MT; Patil, KR, 2012)
"Mycophenolic acid (MPA) is an immunosuppressive agent that is widely used in clinical therapy, including pancreas and islet transplantation. "( Cho, Y; Do, JH; Huh, KH; Joo, DJ; Kim, MS; Kim, YS; Park, YJ, 2012)
"Mycophenolic acid (MPA) is an immunosuppressive agent that acts as a selective, non-reversible inhibitor of the enzyme inosine-5'-monophosphate dehydrogenase (IMPDH). "( Eckardt, K; Glander, P; Schmidt, F; Shakibaei, M; Stahlmann, R, 2013)
"Mycophenolic acid (MPA) is an inosine monophosphate dehydrogenase inhibitor whose antiviral mechanism of action is supposed to interfere with NAD(+)/NADH conversion. "( Luvisi, A; Panattoni, A; Rinaldelli, E; Triolo, E, 2012)
"Mycophenolic acid (MPA) is an immunosuppression agent for the prophylaxis of organ rejection in patients receiving allogeneic transplants. "( Carrasco García, C; García Pinteño, S; Joumady, I; Sáez-Benito Godino, A; Vara Gil, F; Vergara Chozas, JM; Zopeque García, N, 2012)
"Mycophenolic acid (MPA) is a fungally-derived inhibitor of inosine 5'-monophosphate dehydrogenase (IMPDH). "( Jayaram, HN; Pankiewicz, K; Patterson, SE; Yalowitz, JA, 2002)
"Mycophenolic acid (MPA) is an inhibitor of inosine monophosphate dehydrogenase (mainly used clinically for immunosuppression) that inhibits the replication of several viruses."( Coombs, KM; Hermann, LL; Robertson, CM, 2004)
"Mycophenolic acid (MPA) is an immunosuppressive drug partly metabolized to MPA-glucuronide (MPAG), which is pharmacologically inactive. "( Lachâtre, G; Le Meur, Y; Marquet, P; Prémaud, A; Rousseau, A, 2004)
"Mycophenolic acid (MPA) is a mycotoxin commonly found as Penicillium genus secondary metabolite in feedstuffs and silages. "( Bauer, J; Dzidic, A; Meyer, HH; Meyer, K; Mohr, A; Pfaffl, MW; Prgomet, C, 2006)
"Mycophenolic acid (MPA) is a drug that has found widespread use as an immunosuppressive agent which limits rejection of transplanted organs. "( Hanson, GR; Jones, CE; McEwan, AG; Taylor, PJ, 2006)
"Mycophenolic acid (MPA) is a highly selective, non-competitive and reversible inhibitor of the inosine monophosphate dehydrogenase (IMPDH), the rate-limiting enzyme in the de novo biosynthesis of guanosine nucleotides. "( Beimler, J; Mehrabi, A; Morath, C; Muranyi, W; Schmidt, J; Schwenger, V; Zeier, M, 2006)
"Mycophenolic acid (MPA) is an inhibitor of lymphocyte proliferation and is well established as an immunosuppressive agent in solid organ transplantation. "( He, X; Holt, DW; Johnston, A, 2006)
"Mycophenolic acid (MPA) is an immunosuppressive metabolite of various fungi, especially of Penicillium roqueforti, and can be found in considerable amounts in mouldy silage. "( Bauer, J; Baum, B; Dzidić, A; Hewicker-Trautwein, M; Lorenz, I; Meyer, HH; Meyer, K; Mohr, AI; Pfaffl, M, 2007)
"Mycophenolic acid (MPA) is a reversible inhibitor of inosine monophosphate dehydrogenase (IMPDH) and, in combination with other immunosuppressive drugs, effectively inhibits rejection in solid organ transplant recipients. "( Alloway, RR; Derotte, M; Vinks, AA; Weimert, NA; Woodle, ES, 2007)
"Mycophenolic acid (MPA) is a relatively new adjuvant drug that selectively inhibits T and B lymphocyte proliferation by suppressing de novo purine synthesis."( Caputo, R; Dassoni, F; Marzano, AV, 2006)
"Mycophenolic acid mofetil (MMF) is an agent which has recently gained a lot of attention. "( Azuma, H; Heemann, U; Philipp, T; Schmid, C; Tilney, N, 1996)
"Mycophenolic acid (MPA) is a potent and specific inhibitor of mammalian inosine-monophosphate dehydrogenases (IMPDH); most microbial IMPDHs are not sensitive to MPA. "( Cahoon, M; Farazi, T; Harris, T; Hedstrom, L; Leichman, J, 1997)
"1. Mycophenolic acid (MPA) is an inhibitor of inosine-5'-monophosphate dehydrogenase and therefore interferes with cellular GTP biosynthesis. "( Goppelt-Strübe, M; Hauser, IA; Johnson, DR; Thévenod, F, 1997)
"Mycophenolic acid is an immunosuppressant administered as a bioavailable ester, mycophenolate mofetil. "( Beal, JL; Jones, CE; Taylor, PJ; Tett, SE, 1998)
"Mycophenolic acid (MPA) is a potent inhibitor of mammalian IMPDHs but a poor inhibitor of microbial IMPDHs."( Digits, JA; Hedstrom, L, 1999)
"Mycophenolic acid (MPA) is a species-specific inhibitor of IMPDH; mammalian IMPDHs are very sensitive to MPA, while the microbial enzymes are resistant to the inhibitor."( Digits, JA; Hedstrom, L, 2000)
"Mycophenolic acid (MPA) is an immunosuppressive drug given as the prodrug of mycophenolate mofetil (MMF). "( Hosotsubo, H; Kitamura, M; Kokado, Y; Matsumiya, K; Okuyama, A; Permpongkosol, S; Sugimoto, H; Takahara, S; Tanaka, T; Wang, JD, 2001)
"Mycophenolic acid is an antimetabolite used experimentally for the treatment of psoriasis. "( Lynch, WS; Roenigk, HH, 1977)
"Mycophenolic acid (MPA) is a fermentation product of a penicillium mould which has shown antitumour acitivity in certain animal models. "( McDonald, CJ; Rudnicka, A; Spatz, S, 1978)

Effects

ExcerptReference
"Mycophenolic acid (MPA) has a low therapeutic index and large inter-individual pharmacokinetic variability necessitating therapeutic drug monitoring to individualise dosing after transplantation. "( Coller, JK; Hesselink, DA; Md Dom, ZI; Noll, BD; Russ, GR; Sallustio, BC; Somogyi, AA; van Gelder, T, 2014)
"Mycophenolic acid (MPA) has an anti-inflammatory effect."( Han, NJ; Kim, YJ; Koo, TY; Lee, JM; Park, JS; Park, SK; Yang, WS, 2013)
"Mycophenolic acid (MPA) has promising anticancer properties; however, it has limited clinical applications in vivo due to hydrophobic nature, high first-pass metabolism, lack of targeting, etc. "( Banerjee, UC; Jain, S; Kai, G; Kushwah, V; Nile, SH; Patel, G; Patil, MD; Thakur, NS, 2020)
"Mycophenolic acid (MPA) has become a cornerstone of immunosuppressive therapy, in particular for transplant patients. "( Arnion, H; Benmameri, M; Berthier, J; Chantemargue, B; Fabre, G; Marquet, P; Picard, N; Saint-Marcoux, F; Sauvage, FL; Trouillas, P, 2021)
"Mycophenolic acid (MPA) has been shown to be promising for the treatment of autoimmune diseases in dogs and cats. "( Court, MH; Morassi, A; Rivera-Vélez, SM; Slovak, JE; Villarino, NF, 2018)
"Mycophenolic acid has played an important role in treating immunosuppression and autoimmune diseases. "( Gong, T; Lin, Y; Sun, X; Wang, X; Zeng, Y; Zhang, Z, 2013)
"Mycophenolic acid (MPA) has a low therapeutic index and large inter-individual pharmacokinetic variability necessitating therapeutic drug monitoring to individualise dosing after transplantation. "( Coller, JK; Hesselink, DA; Md Dom, ZI; Noll, BD; Russ, GR; Sallustio, BC; Somogyi, AA; van Gelder, T, 2014)
"Mycophenolic acid (MPA) has become the cornerstone in the treatment of lupus nephritis. "( Berden, JH; Berger, SP; van Gelder, T, 2015)
"Mycophenolic acid (MPA) has been identified as a new river contaminant according to its wide use and high predicted concentration. "( Boleda, MR; Franquet-Griell, H; Lacorte, S; Ventura, F, 2016)
"Mycophenolic acid (MPA) has been known for decades to be an anticancer and immunosuppressive agent and has significant anticancer properties, but its underlying molecular mechanisms are poorly characterized. "( He, XB; Jiang, Q; Liu, C; Lu, XH; Shui, XX; Si, SY; Yang, Y; Zhang, H; Zhao, BH; Zheng, ZH, 2011)
"Mycophenolic acid (MPA) has become the first-line drug therapy for proliferative lupus nephritis, a common and serious complication of systemic lupus erythematosus. "( Avihingsanon, Y; Kittanamongkolchai, W; Lertdumrongluk, P; Punyawudho, B; Somparn, P; Traitanon, O; Vadcharavivad, S, 2012)
"Mycophenolic acid (MPA) has been shown to reduce panel reactive antibody (PRA) formation in kidney transplant recipients."( Cicciarelli, J; Corrales-Tellez, E; Hutchinson, I; Min, DI; Naraghi, R; Shah, T; Vu, D, 2013)
"Mycophenolic acid (MPA) has an anti-inflammatory effect."( Han, NJ; Kim, YJ; Koo, TY; Lee, JM; Park, JS; Park, SK; Yang, WS, 2013)
"Mycophenolic acid has proved useful for long-term management of psoriasis."( Hood, KA; Zarembski, DG, 1997)
"Oral mycophenolic acid (MPA) therapy has been investigated in the treatment of moderate to severe psoriasis since the early 1970s and has been found to be both safe and effective. "( Kitchin, JE; Pak, G; Pomeranz, MK; Shupack, JL; Washenik, K, 1997)
"Mycophenolic acid kinetics have been reported to vary after renal transplantation, and mycophenolic acid area under the concentration-time curve (AUC) is the best predictor of suppression of graft rejection."( Allen, J; Falk, MC; Franzen, K; Johnson, AG; Jones, CE; Nicol, D; Rigby, RJ; Taylor, PJ, 1999)
"Mycophenolic acid has been shown to be effective for the prevention and treatment of renal allograft rejection. "( Baer, PC; Gauer, S; Geiger, H; Hauser, IA; Scherberich, JE, 2000)
"Mycophenolic acid has been shown to be a potent inhibitor of vaccinia virus growth. "( Boyle, DB; Coupar, BE, 1988)

Actions

ExcerptReference
"Mycophenolic acid (MPA) may cause gastrointestinal adverse effects by damaging the intestinal epithelial barrier, the underlying mechanisms remain elusive. "( Deng, Y; Feng, L; Su, Y; Wang, J; Xu, D; Yang, H; Zhang, Z, 2022)
"Mycophenolic acid (MPA) displays a complex pharmacokinetic profile susceptible to potential significant interactions with fenofibrate."( Alvarez, PA; Di Girolamo, G; Egozcue, J; Keller, GA; Moretti, L; Sleiman, J, 2012)
"Mycophenolic acid (MPA) did inhibit agonist-induced OPN expression in cultured cardiomyocytes."( Hirose, S; Hongo, M; Ikeda, U; Kamiyoshi, Y; Kinoshita, O; Morimoto, H; Takahashi, M; Watanabe, N; Yazaki, Y; Yokoseki, O, 2005)

Treatment

ExcerptReference
"Mycophenolic acid-treated cells were anergic, but not suppressive, and at the same time proved hyperblastoid with high metabolic activity."( Boots, AM; He, X; Joosten, I; Koenen, HJ; Smeets, RL; Vink, PM; Wagenaars, J, 2011)
"Mycophenolic acid-treated long-term bone marrow cultures were charged with stromally depleted bone marrow cells along with 10 to 500 U/ml recombinant human TNF-alpha, and assayed each week for 5 wk for the presence of primitive (CFU-S and high proliferative potential colony-forming cell) and mature (CFU-C) colony-forming units."( Berman, JW; Rogers, JA, 1994)
"Mycophenolic acid treatment of mouse long-term bone marrow cultures depletes them of all assayable hematopoietic precursors."( Berman, JW; Rogers, JA, 1993)
"Mycophenolic acid treatment also appears to cause breakdown of high-molecular-weight DNA."( Brox, L; Henderson, JF; Lowe, JK, 1977)
"Treatment with mycophenolic acid (MPA) resulted in a 99% inhibition of viral RNA production at the clinically relevant concentration (10 μg/ml) in Caco2 cells."( Bijvelds, M; Dang, W; de Jonge, H; Felczak, K; Koopmans, MP; Metselaar, HJ; Pan, Q; Pankiewicz, KW; Peppelenbosch, MP; Sprengers, D; Su, J; van der Eijk, AA; van der Laan, LJ; Wang, W; Wang, Y; Xu, L; Yin, Y; Zhou, X, 2016)
"DC treated with mycophenolic acid during their maturation (MPA-DC) have a regulatory phenotype and may therefore provide a new approach to induce allograft tolerance."( Baron, C; Lagaraine, C; Lebranchu, Y; Lemoine, R; Nivet, H; Velge-Roussel, F, 2008)
"Treatment with mycophenolic acid led to significantly increased survival times in a nematode model, validating de novo GTP biosynthesis as an antifungal target in Cryptococcus."( Chow, EW; Djordjevic, JT; Fraser, JA; Hill, JM; Kappler, U; Kobe, B; Lee, IR; Morrow, CA; Stamp, A; Valkov, E; Williams, SJ; Wronski, A, 2012)

Roles (9)

RoleDescription
antineoplastic agentA substance that inhibits or prevents the proliferation of neoplasms.
antimicrobial agentA substance that kills or slows the growth of microorganisms, including bacteria, viruses, fungi and protozoans.
EC 1.1.1.205 (IMP dehydrogenase) inhibitorAn EC 1.1.1.* (oxidoreductase acting on donor CH-OH group, NAD(+) or NADP(+) acceptor) inhibitor that interferes with the action of IMP dehydrogenase (EC 1.1.1.205), so blocking de novo biosynthesis of purine nucleotides.
immunosuppressive agentAn agent that suppresses immune function by one of several mechanisms of action. Classical cytotoxic immunosuppressants act by inhibiting DNA synthesis. Others may act through activation of T-cells or by inhibiting the activation of helper cells. In addition, an immunosuppressive agent is a role played by a compound which is exhibited by a capability to diminish the extent and/or voracity of an immune response.
mycotoxinPoisonous substance produced by fungi.
Penicillium metaboliteAny fungal metabolite produced during a metabolic reaction in Penicillium.
environmental contaminantAny minor or unwanted substance introduced into the environment that can have undesired effects.
xenobioticA xenobiotic (Greek, xenos "foreign"; bios "life") is a compound that is foreign to a living organism. Principal xenobiotics include: drugs, carcinogens and various compounds that have been introduced into the environment by artificial means.
anticoronaviral agentAny antiviral agent which inhibits the activity of coronaviruses.
[role information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Drug Classes (4)

ClassDescription
2-benzofurans
gamma-lactoneA lactone having a five-membered lactone ring.
phenolsOrganic aromatic compounds having one or more hydroxy groups attached to a benzene or other arene ring.
monocarboxylic acidAn oxoacid containing a single carboxy group.
[compound class information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Pathways (3)

mycophenolic acid is involved in 3 pathway(s), involving a total of 99 unique proteins and 132 unique compounds

PathwayProteinsCompounds
Mycophenolic Acid Metabolism Pathway1618
The impact of Nsp14 on metabolism (COVID-19 Disease Map)084
Nsp9 interactions (COVID-19 Disease Map)8330

Protein Targets (79)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Chain A, HADH2 proteinHomo sapiens (human)Potency25.83960.025120.237639.8107AID886; AID893
Chain B, HADH2 proteinHomo sapiens (human)Potency25.83960.025120.237639.8107AID886; AID893
Chain A, 2-oxoglutarate OxygenaseHomo sapiens (human)Potency27.01470.177814.390939.8107AID2147
acetylcholinesteraseHomo sapiens (human)Potency43.64860.002541.796015,848.9004AID1347398
glp-1 receptor, partialHomo sapiens (human)Potency0.89130.01846.806014.1254AID624417
RAR-related orphan receptor gammaMus musculus (house mouse)Potency0.13860.006038.004119,952.5996AID1159521; AID1159523
SMAD family member 2Homo sapiens (human)Potency6.74000.173734.304761.8120AID1346859
SMAD family member 3Homo sapiens (human)Potency6.74000.173734.304761.8120AID1346859
TDP1 proteinHomo sapiens (human)Potency0.24610.000811.382244.6684AID686978; AID686979
AR proteinHomo sapiens (human)Potency0.53470.000221.22318,912.5098AID743035; AID743036; AID743063
aldehyde dehydrogenase 1 family, member A1Homo sapiens (human)Potency39.81070.011212.4002100.0000AID1030
estrogen receptor 2 (ER beta)Homo sapiens (human)Potency18.83360.000657.913322,387.1992AID1259378
nuclear receptor subfamily 1, group I, member 3Homo sapiens (human)Potency0.55200.001022.650876.6163AID1224838; AID1224893
progesterone receptorHomo sapiens (human)Potency0.13330.000417.946075.1148AID1346795
glucocorticoid receptor [Homo sapiens]Homo sapiens (human)Potency0.12850.000214.376460.0339AID720692
retinoid X nuclear receptor alphaHomo sapiens (human)Potency2.04560.000817.505159.3239AID1159527; AID1159531
estrogen-related nuclear receptor alphaHomo sapiens (human)Potency1.06980.001530.607315,848.9004AID1224841; AID1224842; AID1224848; AID1224849; AID1259403
farnesoid X nuclear receptorHomo sapiens (human)Potency0.74970.375827.485161.6524AID743217
estrogen nuclear receptor alphaHomo sapiens (human)Potency0.74050.000229.305416,493.5996AID1259244; AID1259248; AID743069; AID743075; AID743078; AID743080; AID743091
67.9K proteinVaccinia virusPotency5.04510.00018.4406100.0000AID720579
peroxisome proliferator-activated receptor deltaHomo sapiens (human)Potency14.95890.001024.504861.6448AID743215
peroxisome proliferator activated receptor gammaHomo sapiens (human)Potency0.47300.001019.414170.9645AID743191
vitamin D (1,25- dihydroxyvitamin D3) receptorHomo sapiens (human)Potency0.42530.023723.228263.5986AID743223
euchromatic histone-lysine N-methyltransferase 2Homo sapiens (human)Potency10.00000.035520.977089.1251AID504332
aryl hydrocarbon receptorHomo sapiens (human)Potency12.74480.000723.06741,258.9301AID743122
cytochrome P450, family 19, subfamily A, polypeptide 1, isoform CRA_aHomo sapiens (human)Potency0.22660.001723.839378.1014AID743083
activating transcription factor 6Homo sapiens (human)Potency26.83250.143427.612159.8106AID1159516; AID1159519
nuclear factor of kappa light polypeptide gene enhancer in B-cells 1 (p105), isoform CRA_aHomo sapiens (human)Potency10.682219.739145.978464.9432AID1159509
Histone H2A.xCricetulus griseus (Chinese hamster)Potency1.02070.039147.5451146.8240AID1224845; AID1224896
15-hydroxyprostaglandin dehydrogenase [NAD(+)] isoform 1Homo sapiens (human)Potency29.40030.001815.663839.8107AID894
nuclear factor erythroid 2-related factor 2 isoform 2Homo sapiens (human)Potency2.31090.00419.984825.9290AID504444
thyroid hormone receptor beta isoform 2Rattus norvegicus (Norway rat)Potency0.50740.000323.4451159.6830AID743065; AID743067
nuclear factor erythroid 2-related factor 2 isoform 1Homo sapiens (human)Potency0.56220.000627.21521,122.0200AID743219
urokinase-type plasminogen activator precursorMus musculus (house mouse)Potency0.31620.15855.287912.5893AID540303
plasminogen precursorMus musculus (house mouse)Potency0.31620.15855.287912.5893AID540303
urokinase plasminogen activator surface receptor precursorMus musculus (house mouse)Potency0.31620.15855.287912.5893AID540303
gemininHomo sapiens (human)Potency0.36630.004611.374133.4983AID624296
peripheral myelin protein 22Rattus norvegicus (Norway rat)Potency3.48870.005612.367736.1254AID624032; AID624044
survival motor neuron protein isoform dHomo sapiens (human)Potency0.31520.125912.234435.4813AID1458; AID1740
cytochrome P450 3A4 isoform 1Homo sapiens (human)Potency14.97630.031610.279239.8107AID884; AID885
lethal factor (plasmid)Bacillus anthracis str. A2012Potency1.25890.020010.786931.6228AID912
lamin isoform A-delta10Homo sapiens (human)Potency0.38610.891312.067628.1838AID1487; AID1498
Gamma-aminobutyric acid receptor subunit piRattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Voltage-dependent calcium channel gamma-2 subunitMus musculus (house mouse)Potency1.33330.001557.789015,848.9004AID1259244
Cellular tumor antigen p53Homo sapiens (human)Potency0.06970.002319.595674.0614AID651631; AID720552
Gamma-aminobutyric acid receptor subunit beta-1Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit deltaRattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit gamma-2Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Glutamate receptor 2Rattus norvegicus (Norway rat)Potency1.33330.001551.739315,848.9004AID1259244
Gamma-aminobutyric acid receptor subunit alpha-5Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-3Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit gamma-1Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-2Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-4Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit gamma-3Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-6Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Alpha-synucleinHomo sapiens (human)Potency7.07950.56239.398525.1189AID652106
Gamma-aminobutyric acid receptor subunit alpha-1Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit beta-3Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit beta-2Rattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
TAR DNA-binding protein 43Homo sapiens (human)Potency35.48131.778316.208135.4813AID652104
GABA theta subunitRattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
ATPase family AAA domain-containing protein 5Homo sapiens (human)Potency9.02260.011917.942071.5630AID651632
Ataxin-2Homo sapiens (human)Potency9.02260.011912.222168.7989AID651632
Gamma-aminobutyric acid receptor subunit epsilonRattus norvegicus (Norway rat)Potency14.97631.000012.224831.6228AID885
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Inhibition Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Chain A, Inosine-5'-Monophosphate Dehydrogenase 2Cricetulus griseus (Chinese hamster)Ki0.00600.00600.00600.0060AID977610
Inosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)IC50 (µMol)1.14210.00700.18071.5000AID1408889; AID1422682; AID1797842; AID1803439; AID267726; AID292040; AID538354; AID637676; AID657090; AID91401; AID91402; AID92616; AID92617; AID92622; AID92624; AID92740; AID92741; AID92742; AID92743; AID92745
Inosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)Ki0.00840.00600.12620.2600AID1797841; AID303352; AID304372; AID342523; AID348819; AID580126; AID92748; AID92749
Glutamate receptor 1Rattus norvegicus (Norway rat)IC50 (µMol)0.01400.00011.617910.0000AID92624
Glutamate receptor 2Rattus norvegicus (Norway rat)IC50 (µMol)0.01400.00011.700010.0000AID92624
Glutamate receptor 3Rattus norvegicus (Norway rat)IC50 (µMol)0.01400.00011.700010.0000AID92624
Glutamate receptor 4Rattus norvegicus (Norway rat)IC50 (µMol)0.01400.00011.700010.0000AID92624
Inosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)IC50 (µMol)3.39770.00700.06550.2600AID1422681; AID267725; AID538353; AID91401; AID92486; AID92606
Inosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)Ki0.03650.03300.16540.3300AID1797840; AID303351; AID304371; AID342522; AID348818; AID580128; AID92607; AID92608
Sodium-dependent serotonin transporterRattus norvegicus (Norway rat)IC50 (µMol)0.01100.00030.81978.4900AID267726
Inosine-5'-monophosphate dehydrogenaseCryptococcus neoformans var. neoformans JEC21IC50 (µMol)0.12000.12000.12000.1200AID1422680
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Activation Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Peroxisome proliferator-activated receptor gammaHomo sapiens (human)Kd110.00000.00120.95314.9800AID300652
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Other Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
UDP-glucuronosyltransferase 1A9Homo sapiens (human)Km225.50005.00006.830010.0000AID1210129; AID624637
UDP-glucuronosyltransferase 1A1 Homo sapiens (human)Km185.00004.49006.51339.0000AID624630
UDP-glucuronosyltransferase 1A7Homo sapiens (human)Km30.00005.00007.20009.4000AID624635
UDP-glucuronosyltransferase 1A10Homo sapiens (human)Km76.50002.74004.21005.6800AID624631
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (313)

Processvia Protein(s)Taxonomy
xenobiotic metabolic processUDP-glucuronosyltransferase 1A9Homo sapiens (human)
retinoic acid metabolic processUDP-glucuronosyltransferase 1A9Homo sapiens (human)
flavone metabolic processUDP-glucuronosyltransferase 1A9Homo sapiens (human)
cellular glucuronidationUDP-glucuronosyltransferase 1A9Homo sapiens (human)
flavonoid glucuronidationUDP-glucuronosyltransferase 1A9Homo sapiens (human)
xenobiotic glucuronidationUDP-glucuronosyltransferase 1A9Homo sapiens (human)
liver developmentUDP-glucuronosyltransferase 1A9Homo sapiens (human)
negative regulation of cell population proliferationCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycleCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycle G2/M phase transitionCellular tumor antigen p53Homo sapiens (human)
DNA damage responseCellular tumor antigen p53Homo sapiens (human)
ER overload responseCellular tumor antigen p53Homo sapiens (human)
cellular response to glucose starvationCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
positive regulation of miRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
negative regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
mitophagyCellular tumor antigen p53Homo sapiens (human)
in utero embryonic developmentCellular tumor antigen p53Homo sapiens (human)
somitogenesisCellular tumor antigen p53Homo sapiens (human)
release of cytochrome c from mitochondriaCellular tumor antigen p53Homo sapiens (human)
hematopoietic progenitor cell differentiationCellular tumor antigen p53Homo sapiens (human)
T cell proliferation involved in immune responseCellular tumor antigen p53Homo sapiens (human)
B cell lineage commitmentCellular tumor antigen p53Homo sapiens (human)
T cell lineage commitmentCellular tumor antigen p53Homo sapiens (human)
response to ischemiaCellular tumor antigen p53Homo sapiens (human)
nucleotide-excision repairCellular tumor antigen p53Homo sapiens (human)
double-strand break repairCellular tumor antigen p53Homo sapiens (human)
regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
protein import into nucleusCellular tumor antigen p53Homo sapiens (human)
autophagyCellular tumor antigen p53Homo sapiens (human)
DNA damage responseCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediator resulting in cell cycle arrestCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediator resulting in transcription of p21 class mediatorCellular tumor antigen p53Homo sapiens (human)
transforming growth factor beta receptor signaling pathwayCellular tumor antigen p53Homo sapiens (human)
Ras protein signal transductionCellular tumor antigen p53Homo sapiens (human)
gastrulationCellular tumor antigen p53Homo sapiens (human)
neuroblast proliferationCellular tumor antigen p53Homo sapiens (human)
negative regulation of neuroblast proliferationCellular tumor antigen p53Homo sapiens (human)
protein localizationCellular tumor antigen p53Homo sapiens (human)
negative regulation of DNA replicationCellular tumor antigen p53Homo sapiens (human)
negative regulation of cell population proliferationCellular tumor antigen p53Homo sapiens (human)
determination of adult lifespanCellular tumor antigen p53Homo sapiens (human)
mRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
rRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
response to salt stressCellular tumor antigen p53Homo sapiens (human)
response to inorganic substanceCellular tumor antigen p53Homo sapiens (human)
response to X-rayCellular tumor antigen p53Homo sapiens (human)
response to gamma radiationCellular tumor antigen p53Homo sapiens (human)
positive regulation of gene expressionCellular tumor antigen p53Homo sapiens (human)
cardiac muscle cell apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of cardiac muscle cell apoptotic processCellular tumor antigen p53Homo sapiens (human)
glial cell proliferationCellular tumor antigen p53Homo sapiens (human)
viral processCellular tumor antigen p53Homo sapiens (human)
glucose catabolic process to lactate via pyruvateCellular tumor antigen p53Homo sapiens (human)
cerebellum developmentCellular tumor antigen p53Homo sapiens (human)
negative regulation of cell growthCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
negative regulation of transforming growth factor beta receptor signaling pathwayCellular tumor antigen p53Homo sapiens (human)
mitotic G1 DNA damage checkpoint signalingCellular tumor antigen p53Homo sapiens (human)
negative regulation of telomere maintenance via telomeraseCellular tumor antigen p53Homo sapiens (human)
T cell differentiation in thymusCellular tumor antigen p53Homo sapiens (human)
tumor necrosis factor-mediated signaling pathwayCellular tumor antigen p53Homo sapiens (human)
regulation of tissue remodelingCellular tumor antigen p53Homo sapiens (human)
cellular response to UVCellular tumor antigen p53Homo sapiens (human)
multicellular organism growthCellular tumor antigen p53Homo sapiens (human)
positive regulation of mitochondrial membrane permeabilityCellular tumor antigen p53Homo sapiens (human)
cellular response to glucose starvationCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
entrainment of circadian clock by photoperiodCellular tumor antigen p53Homo sapiens (human)
mitochondrial DNA repairCellular tumor antigen p53Homo sapiens (human)
regulation of DNA damage response, signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of neuron apoptotic processCellular tumor antigen p53Homo sapiens (human)
transcription initiation-coupled chromatin remodelingCellular tumor antigen p53Homo sapiens (human)
negative regulation of proteolysisCellular tumor antigen p53Homo sapiens (human)
negative regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
positive regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
positive regulation of RNA polymerase II transcription preinitiation complex assemblyCellular tumor antigen p53Homo sapiens (human)
positive regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
response to antibioticCellular tumor antigen p53Homo sapiens (human)
fibroblast proliferationCellular tumor antigen p53Homo sapiens (human)
negative regulation of fibroblast proliferationCellular tumor antigen p53Homo sapiens (human)
circadian behaviorCellular tumor antigen p53Homo sapiens (human)
bone marrow developmentCellular tumor antigen p53Homo sapiens (human)
embryonic organ developmentCellular tumor antigen p53Homo sapiens (human)
positive regulation of peptidyl-tyrosine phosphorylationCellular tumor antigen p53Homo sapiens (human)
protein stabilizationCellular tumor antigen p53Homo sapiens (human)
negative regulation of helicase activityCellular tumor antigen p53Homo sapiens (human)
protein tetramerizationCellular tumor antigen p53Homo sapiens (human)
chromosome organizationCellular tumor antigen p53Homo sapiens (human)
neuron apoptotic processCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycleCellular tumor antigen p53Homo sapiens (human)
hematopoietic stem cell differentiationCellular tumor antigen p53Homo sapiens (human)
negative regulation of glial cell proliferationCellular tumor antigen p53Homo sapiens (human)
type II interferon-mediated signaling pathwayCellular tumor antigen p53Homo sapiens (human)
cardiac septum morphogenesisCellular tumor antigen p53Homo sapiens (human)
positive regulation of programmed necrotic cell deathCellular tumor antigen p53Homo sapiens (human)
protein-containing complex assemblyCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to endoplasmic reticulum stressCellular tumor antigen p53Homo sapiens (human)
thymocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of thymocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
necroptotic processCellular tumor antigen p53Homo sapiens (human)
cellular response to hypoxiaCellular tumor antigen p53Homo sapiens (human)
cellular response to xenobiotic stimulusCellular tumor antigen p53Homo sapiens (human)
cellular response to ionizing radiationCellular tumor antigen p53Homo sapiens (human)
cellular response to gamma radiationCellular tumor antigen p53Homo sapiens (human)
cellular response to UV-CCellular tumor antigen p53Homo sapiens (human)
stem cell proliferationCellular tumor antigen p53Homo sapiens (human)
signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
cellular response to actinomycin DCellular tumor antigen p53Homo sapiens (human)
positive regulation of release of cytochrome c from mitochondriaCellular tumor antigen p53Homo sapiens (human)
cellular senescenceCellular tumor antigen p53Homo sapiens (human)
replicative senescenceCellular tumor antigen p53Homo sapiens (human)
oxidative stress-induced premature senescenceCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathwayCellular tumor antigen p53Homo sapiens (human)
oligodendrocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of execution phase of apoptosisCellular tumor antigen p53Homo sapiens (human)
negative regulation of mitophagyCellular tumor antigen p53Homo sapiens (human)
regulation of mitochondrial membrane permeability involved in apoptotic processCellular tumor antigen p53Homo sapiens (human)
regulation of intrinsic apoptotic signaling pathway by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of miRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
negative regulation of G1 to G0 transitionCellular tumor antigen p53Homo sapiens (human)
negative regulation of miRNA processingCellular tumor antigen p53Homo sapiens (human)
negative regulation of glucose catabolic process to lactate via pyruvateCellular tumor antigen p53Homo sapiens (human)
negative regulation of pentose-phosphate shuntCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to hypoxiaCellular tumor antigen p53Homo sapiens (human)
regulation of fibroblast apoptotic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of stem cell proliferationCellular tumor antigen p53Homo sapiens (human)
positive regulation of cellular senescenceCellular tumor antigen p53Homo sapiens (human)
positive regulation of intrinsic apoptotic signaling pathwayCellular tumor antigen p53Homo sapiens (human)
GMP biosynthetic processInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
GTP biosynthetic processInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
circadian rhythmInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
lymphocyte proliferationInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
cellular response to interleukin-4Inosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
'de novo' XMP biosynthetic processInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
GMP biosynthetic processInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
GTP biosynthetic processInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
liver developmentUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
bilirubin conjugationUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
xenobiotic metabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
acute-phase responseUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
response to nutrientUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
steroid metabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
estrogen metabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
animal organ regenerationUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
response to lipopolysaccharideUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
retinoic acid metabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
response to starvationUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
negative regulation of steroid metabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
flavone metabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
cellular glucuronidationUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
flavonoid glucuronidationUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
xenobiotic glucuronidationUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
biphenyl catabolic processUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
cellular response to ethanolUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
cellular response to glucocorticoid stimulusUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
cellular response to estradiol stimulusUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
negative regulation of gene expressionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of cholesterol effluxPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
long-chain fatty acid transportPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of osteoblast differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of smooth muscle cell proliferationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of receptor signaling pathway via STATPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of low-density lipoprotein receptor activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of signaling receptor activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of gene expressionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of transforming growth factor beta receptor signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of BMP signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of MAP kinase activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of adiponectin secretionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of miRNA transcriptionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of cardiac muscle hypertrophy in response to stressPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of connective tissue replacement involved in inflammatory response wound healingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of transcription by RNA polymerase IIPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
placenta developmentPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
regulation of transcription by RNA polymerase IIPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
lipid metabolic processPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
activation of cysteine-type endopeptidase activity involved in apoptotic processPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
signal transductionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
G protein-coupled receptor signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
response to nutrientPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
regulation of blood pressurePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of gene expressionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of gene expressionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
macrophage derived foam cell differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of macrophage derived foam cell differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of cholesterol storagePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of lipid storagePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of sequestering of triglyceridePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of angiogenesisPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
monocyte differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
BMP signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of transforming growth factor beta receptor signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
epithelial cell differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cellular response to insulin stimulusPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
response to lipidPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
peroxisome proliferator activated receptor signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
glucose homeostasisPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
regulation of circadian rhythmPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
mRNA transcription by RNA polymerase IIPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
lipoprotein transportPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of blood vessel endothelial cell migrationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
innate immune responsePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cell fate commitmentPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of fat cell differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of DNA-templated transcriptionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of DNA-templated transcriptionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of transcription by RNA polymerase IIPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
retinoic acid receptor signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cell maturationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
rhythmic processPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
white fat cell differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of DNA-binding transcription factor activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
lipid homeostasisPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of type II interferon-mediated signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of SMAD protein signal transductionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
regulation of cholesterol transporter activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cellular response to low-density lipoprotein particle stimulusPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cellular response to hypoxiaPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of mitochondrial fissionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
regulation of cellular response to insulin stimulusPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of extracellular matrix assemblyPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of miRNA transcriptionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of miRNA transcriptionPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of cellular response to transforming growth factor beta stimulusPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of adipose tissue developmentPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of vascular associated smooth muscle cell proliferationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of vascular associated smooth muscle cell apoptotic processPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of vascular endothelial cell proliferationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
positive regulation of fatty acid metabolic processPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
fatty acid metabolic processPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
negative regulation of inflammatory responsePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cell differentiationPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
hormone-mediated signaling pathwayPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
calcium ion homeostasisAlpha-synucleinHomo sapiens (human)
negative regulation of transcription by RNA polymerase IIAlpha-synucleinHomo sapiens (human)
microglial cell activationAlpha-synucleinHomo sapiens (human)
positive regulation of receptor recyclingAlpha-synucleinHomo sapiens (human)
positive regulation of neurotransmitter secretionAlpha-synucleinHomo sapiens (human)
negative regulation of protein kinase activityAlpha-synucleinHomo sapiens (human)
fatty acid metabolic processAlpha-synucleinHomo sapiens (human)
neutral lipid metabolic processAlpha-synucleinHomo sapiens (human)
phospholipid metabolic processAlpha-synucleinHomo sapiens (human)
activation of cysteine-type endopeptidase activity involved in apoptotic processAlpha-synucleinHomo sapiens (human)
mitochondrial membrane organizationAlpha-synucleinHomo sapiens (human)
adult locomotory behaviorAlpha-synucleinHomo sapiens (human)
response to xenobiotic stimulusAlpha-synucleinHomo sapiens (human)
response to iron(II) ionAlpha-synucleinHomo sapiens (human)
regulation of phospholipase activityAlpha-synucleinHomo sapiens (human)
negative regulation of platelet-derived growth factor receptor signaling pathwayAlpha-synucleinHomo sapiens (human)
regulation of glutamate secretionAlpha-synucleinHomo sapiens (human)
regulation of dopamine secretionAlpha-synucleinHomo sapiens (human)
synaptic vesicle exocytosisAlpha-synucleinHomo sapiens (human)
synaptic vesicle primingAlpha-synucleinHomo sapiens (human)
regulation of transmembrane transporter activityAlpha-synucleinHomo sapiens (human)
negative regulation of microtubule polymerizationAlpha-synucleinHomo sapiens (human)
receptor internalizationAlpha-synucleinHomo sapiens (human)
protein destabilizationAlpha-synucleinHomo sapiens (human)
response to magnesium ionAlpha-synucleinHomo sapiens (human)
negative regulation of transporter activityAlpha-synucleinHomo sapiens (human)
response to lipopolysaccharideAlpha-synucleinHomo sapiens (human)
negative regulation of monooxygenase activityAlpha-synucleinHomo sapiens (human)
positive regulation of peptidyl-serine phosphorylationAlpha-synucleinHomo sapiens (human)
response to type II interferonAlpha-synucleinHomo sapiens (human)
cellular response to oxidative stressAlpha-synucleinHomo sapiens (human)
SNARE complex assemblyAlpha-synucleinHomo sapiens (human)
positive regulation of SNARE complex assemblyAlpha-synucleinHomo sapiens (human)
regulation of locomotionAlpha-synucleinHomo sapiens (human)
dopamine biosynthetic processAlpha-synucleinHomo sapiens (human)
mitochondrial ATP synthesis coupled electron transportAlpha-synucleinHomo sapiens (human)
regulation of macrophage activationAlpha-synucleinHomo sapiens (human)
positive regulation of apoptotic processAlpha-synucleinHomo sapiens (human)
negative regulation of apoptotic processAlpha-synucleinHomo sapiens (human)
negative regulation of cysteine-type endopeptidase activity involved in apoptotic processAlpha-synucleinHomo sapiens (human)
negative regulation of neuron apoptotic processAlpha-synucleinHomo sapiens (human)
positive regulation of endocytosisAlpha-synucleinHomo sapiens (human)
negative regulation of exocytosisAlpha-synucleinHomo sapiens (human)
positive regulation of exocytosisAlpha-synucleinHomo sapiens (human)
regulation of long-term neuronal synaptic plasticityAlpha-synucleinHomo sapiens (human)
synaptic vesicle endocytosisAlpha-synucleinHomo sapiens (human)
synaptic vesicle transportAlpha-synucleinHomo sapiens (human)
positive regulation of inflammatory responseAlpha-synucleinHomo sapiens (human)
regulation of acyl-CoA biosynthetic processAlpha-synucleinHomo sapiens (human)
protein tetramerizationAlpha-synucleinHomo sapiens (human)
positive regulation of release of sequestered calcium ion into cytosolAlpha-synucleinHomo sapiens (human)
neuron apoptotic processAlpha-synucleinHomo sapiens (human)
dopamine uptake involved in synaptic transmissionAlpha-synucleinHomo sapiens (human)
negative regulation of dopamine uptake involved in synaptic transmissionAlpha-synucleinHomo sapiens (human)
negative regulation of serotonin uptakeAlpha-synucleinHomo sapiens (human)
regulation of norepinephrine uptakeAlpha-synucleinHomo sapiens (human)
negative regulation of norepinephrine uptakeAlpha-synucleinHomo sapiens (human)
excitatory postsynaptic potentialAlpha-synucleinHomo sapiens (human)
long-term synaptic potentiationAlpha-synucleinHomo sapiens (human)
positive regulation of inositol phosphate biosynthetic processAlpha-synucleinHomo sapiens (human)
negative regulation of thrombin-activated receptor signaling pathwayAlpha-synucleinHomo sapiens (human)
response to interleukin-1Alpha-synucleinHomo sapiens (human)
cellular response to copper ionAlpha-synucleinHomo sapiens (human)
cellular response to epinephrine stimulusAlpha-synucleinHomo sapiens (human)
positive regulation of protein serine/threonine kinase activityAlpha-synucleinHomo sapiens (human)
supramolecular fiber organizationAlpha-synucleinHomo sapiens (human)
negative regulation of mitochondrial electron transport, NADH to ubiquinoneAlpha-synucleinHomo sapiens (human)
positive regulation of glutathione peroxidase activityAlpha-synucleinHomo sapiens (human)
positive regulation of hydrogen peroxide catabolic processAlpha-synucleinHomo sapiens (human)
regulation of synaptic vesicle recyclingAlpha-synucleinHomo sapiens (human)
regulation of reactive oxygen species biosynthetic processAlpha-synucleinHomo sapiens (human)
positive regulation of protein localization to cell peripheryAlpha-synucleinHomo sapiens (human)
negative regulation of chaperone-mediated autophagyAlpha-synucleinHomo sapiens (human)
regulation of presynapse assemblyAlpha-synucleinHomo sapiens (human)
amyloid fibril formationAlpha-synucleinHomo sapiens (human)
synapse organizationAlpha-synucleinHomo sapiens (human)
chemical synaptic transmissionAlpha-synucleinHomo sapiens (human)
negative regulation of protein phosphorylationTAR DNA-binding protein 43Homo sapiens (human)
mRNA processingTAR DNA-binding protein 43Homo sapiens (human)
RNA splicingTAR DNA-binding protein 43Homo sapiens (human)
negative regulation of gene expressionTAR DNA-binding protein 43Homo sapiens (human)
regulation of protein stabilityTAR DNA-binding protein 43Homo sapiens (human)
positive regulation of insulin secretionTAR DNA-binding protein 43Homo sapiens (human)
response to endoplasmic reticulum stressTAR DNA-binding protein 43Homo sapiens (human)
positive regulation of protein import into nucleusTAR DNA-binding protein 43Homo sapiens (human)
regulation of circadian rhythmTAR DNA-binding protein 43Homo sapiens (human)
regulation of apoptotic processTAR DNA-binding protein 43Homo sapiens (human)
negative regulation by host of viral transcriptionTAR DNA-binding protein 43Homo sapiens (human)
rhythmic processTAR DNA-binding protein 43Homo sapiens (human)
regulation of cell cycleTAR DNA-binding protein 43Homo sapiens (human)
3'-UTR-mediated mRNA destabilizationTAR DNA-binding protein 43Homo sapiens (human)
3'-UTR-mediated mRNA stabilizationTAR DNA-binding protein 43Homo sapiens (human)
nuclear inner membrane organizationTAR DNA-binding protein 43Homo sapiens (human)
amyloid fibril formationTAR DNA-binding protein 43Homo sapiens (human)
regulation of gene expressionTAR DNA-binding protein 43Homo sapiens (human)
cell population proliferationATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of B cell proliferationATPase family AAA domain-containing protein 5Homo sapiens (human)
nuclear DNA replicationATPase family AAA domain-containing protein 5Homo sapiens (human)
signal transduction in response to DNA damageATPase family AAA domain-containing protein 5Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorATPase family AAA domain-containing protein 5Homo sapiens (human)
isotype switchingATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of DNA replicationATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of isotype switching to IgG isotypesATPase family AAA domain-containing protein 5Homo sapiens (human)
DNA clamp unloadingATPase family AAA domain-containing protein 5Homo sapiens (human)
regulation of mitotic cell cycle phase transitionATPase family AAA domain-containing protein 5Homo sapiens (human)
negative regulation of intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of cell cycle G2/M phase transitionATPase family AAA domain-containing protein 5Homo sapiens (human)
negative regulation of receptor internalizationAtaxin-2Homo sapiens (human)
regulation of translationAtaxin-2Homo sapiens (human)
RNA metabolic processAtaxin-2Homo sapiens (human)
P-body assemblyAtaxin-2Homo sapiens (human)
stress granule assemblyAtaxin-2Homo sapiens (human)
RNA transportAtaxin-2Homo sapiens (human)
xenobiotic metabolic processUDP-glucuronosyltransferase 1A7Homo sapiens (human)
estrogen metabolic processUDP-glucuronosyltransferase 1A7Homo sapiens (human)
coumarin metabolic processUDP-glucuronosyltransferase 1A7Homo sapiens (human)
retinoic acid metabolic processUDP-glucuronosyltransferase 1A7Homo sapiens (human)
flavone metabolic processUDP-glucuronosyltransferase 1A7Homo sapiens (human)
cellular glucuronidationUDP-glucuronosyltransferase 1A7Homo sapiens (human)
flavonoid glucuronidationUDP-glucuronosyltransferase 1A7Homo sapiens (human)
xenobiotic glucuronidationUDP-glucuronosyltransferase 1A7Homo sapiens (human)
liver developmentUDP-glucuronosyltransferase 1A7Homo sapiens (human)
lipid metabolic processUDP-glucuronosyltransferase 1A10Homo sapiens (human)
xenobiotic metabolic processUDP-glucuronosyltransferase 1A10Homo sapiens (human)
flavone metabolic processUDP-glucuronosyltransferase 1A10Homo sapiens (human)
cellular glucuronidationUDP-glucuronosyltransferase 1A10Homo sapiens (human)
liver developmentUDP-glucuronosyltransferase 1A10Homo sapiens (human)
fatty acid metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
steroid metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
coumarin metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
retinoic acid metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
negative regulation of fatty acid metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
negative regulation of steroid metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
flavone metabolic processUDP-glucuronosyltransferase 1A8Homo sapiens (human)
flavonoid glucuronidationUDP-glucuronosyltransferase 1A8Homo sapiens (human)
xenobiotic glucuronidationUDP-glucuronosyltransferase 1A8Homo sapiens (human)
liver developmentUDP-glucuronosyltransferase 1A8Homo sapiens (human)
cellular glucuronidationUDP-glucuronosyltransferase 1A8Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (88)

Processvia Protein(s)Taxonomy
retinoic acid bindingUDP-glucuronosyltransferase 1A9Homo sapiens (human)
glucuronosyltransferase activityUDP-glucuronosyltransferase 1A9Homo sapiens (human)
enzyme bindingUDP-glucuronosyltransferase 1A9Homo sapiens (human)
protein homodimerization activityUDP-glucuronosyltransferase 1A9Homo sapiens (human)
protein heterodimerization activityUDP-glucuronosyltransferase 1A9Homo sapiens (human)
transcription cis-regulatory region bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription factor activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
cis-regulatory region sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
core promoter sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
TFIID-class transcription factor complex bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription repressor activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription activator activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
protease bindingCellular tumor antigen p53Homo sapiens (human)
p53 bindingCellular tumor antigen p53Homo sapiens (human)
DNA bindingCellular tumor antigen p53Homo sapiens (human)
chromatin bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription factor activityCellular tumor antigen p53Homo sapiens (human)
mRNA 3'-UTR bindingCellular tumor antigen p53Homo sapiens (human)
copper ion bindingCellular tumor antigen p53Homo sapiens (human)
protein bindingCellular tumor antigen p53Homo sapiens (human)
zinc ion bindingCellular tumor antigen p53Homo sapiens (human)
enzyme bindingCellular tumor antigen p53Homo sapiens (human)
receptor tyrosine kinase bindingCellular tumor antigen p53Homo sapiens (human)
ubiquitin protein ligase bindingCellular tumor antigen p53Homo sapiens (human)
histone deacetylase regulator activityCellular tumor antigen p53Homo sapiens (human)
ATP-dependent DNA/DNA annealing activityCellular tumor antigen p53Homo sapiens (human)
identical protein bindingCellular tumor antigen p53Homo sapiens (human)
histone deacetylase bindingCellular tumor antigen p53Homo sapiens (human)
protein heterodimerization activityCellular tumor antigen p53Homo sapiens (human)
protein-folding chaperone bindingCellular tumor antigen p53Homo sapiens (human)
protein phosphatase 2A bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II-specific DNA-binding transcription factor bindingCellular tumor antigen p53Homo sapiens (human)
14-3-3 protein bindingCellular tumor antigen p53Homo sapiens (human)
MDM2/MDM4 family protein bindingCellular tumor antigen p53Homo sapiens (human)
disordered domain specific bindingCellular tumor antigen p53Homo sapiens (human)
general transcription initiation factor bindingCellular tumor antigen p53Homo sapiens (human)
molecular function activator activityCellular tumor antigen p53Homo sapiens (human)
promoter-specific chromatin bindingCellular tumor antigen p53Homo sapiens (human)
nucleotide bindingInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
DNA bindingInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
RNA bindingInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
IMP dehydrogenase activityInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
protein bindingInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
metal ion bindingInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
nucleotide bindingInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
nucleic acid bindingInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
DNA bindingInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
RNA bindingInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
metal ion bindingInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
IMP dehydrogenase activityInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
retinoic acid bindingUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
enzyme inhibitor activityUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
steroid bindingUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
glucuronosyltransferase activityUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
enzyme bindingUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
protein homodimerization activityUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
protein heterodimerization activityUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
transcription cis-regulatory region bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA-binding transcription factor activity, RNA polymerase II-specificPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
transcription coregulator bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA-binding transcription activator activity, RNA polymerase II-specificPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
nucleic acid bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
chromatin bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
double-stranded DNA bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA-binding transcription factor activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
nuclear receptor activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
prostaglandin receptor activityPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
protein bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
zinc ion bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
enzyme bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
peptide bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
identical protein bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
sequence-specific DNA bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
nuclear retinoid X receptor bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
arachidonic acid bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA binding domain bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
LBD domain bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
alpha-actinin bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
R-SMAD bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
E-box bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
STAT family protein bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA-binding transcription factor bindingPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
DNA-binding transcription repressor activity, RNA polymerase II-specificPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
fatty acid bindingAlpha-synucleinHomo sapiens (human)
phospholipase D inhibitor activityAlpha-synucleinHomo sapiens (human)
SNARE bindingAlpha-synucleinHomo sapiens (human)
magnesium ion bindingAlpha-synucleinHomo sapiens (human)
transcription cis-regulatory region bindingAlpha-synucleinHomo sapiens (human)
actin bindingAlpha-synucleinHomo sapiens (human)
protein kinase inhibitor activityAlpha-synucleinHomo sapiens (human)
copper ion bindingAlpha-synucleinHomo sapiens (human)
calcium ion bindingAlpha-synucleinHomo sapiens (human)
protein bindingAlpha-synucleinHomo sapiens (human)
phospholipid bindingAlpha-synucleinHomo sapiens (human)
ferrous iron bindingAlpha-synucleinHomo sapiens (human)
zinc ion bindingAlpha-synucleinHomo sapiens (human)
lipid bindingAlpha-synucleinHomo sapiens (human)
oxidoreductase activityAlpha-synucleinHomo sapiens (human)
kinesin bindingAlpha-synucleinHomo sapiens (human)
Hsp70 protein bindingAlpha-synucleinHomo sapiens (human)
histone bindingAlpha-synucleinHomo sapiens (human)
identical protein bindingAlpha-synucleinHomo sapiens (human)
alpha-tubulin bindingAlpha-synucleinHomo sapiens (human)
cysteine-type endopeptidase inhibitor activity involved in apoptotic processAlpha-synucleinHomo sapiens (human)
tau protein bindingAlpha-synucleinHomo sapiens (human)
phosphoprotein bindingAlpha-synucleinHomo sapiens (human)
molecular adaptor activityAlpha-synucleinHomo sapiens (human)
dynein complex bindingAlpha-synucleinHomo sapiens (human)
cuprous ion bindingAlpha-synucleinHomo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
double-stranded DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
RNA bindingTAR DNA-binding protein 43Homo sapiens (human)
mRNA 3'-UTR bindingTAR DNA-binding protein 43Homo sapiens (human)
protein bindingTAR DNA-binding protein 43Homo sapiens (human)
lipid bindingTAR DNA-binding protein 43Homo sapiens (human)
identical protein bindingTAR DNA-binding protein 43Homo sapiens (human)
pre-mRNA intronic bindingTAR DNA-binding protein 43Homo sapiens (human)
molecular condensate scaffold activityTAR DNA-binding protein 43Homo sapiens (human)
protein bindingATPase family AAA domain-containing protein 5Homo sapiens (human)
ATP bindingATPase family AAA domain-containing protein 5Homo sapiens (human)
ATP hydrolysis activityATPase family AAA domain-containing protein 5Homo sapiens (human)
DNA clamp unloader activityATPase family AAA domain-containing protein 5Homo sapiens (human)
DNA bindingATPase family AAA domain-containing protein 5Homo sapiens (human)
RNA bindingAtaxin-2Homo sapiens (human)
epidermal growth factor receptor bindingAtaxin-2Homo sapiens (human)
protein bindingAtaxin-2Homo sapiens (human)
mRNA bindingAtaxin-2Homo sapiens (human)
retinoic acid bindingUDP-glucuronosyltransferase 1A7Homo sapiens (human)
enzyme inhibitor activityUDP-glucuronosyltransferase 1A7Homo sapiens (human)
protein kinase C bindingUDP-glucuronosyltransferase 1A7Homo sapiens (human)
glucuronosyltransferase activityUDP-glucuronosyltransferase 1A7Homo sapiens (human)
enzyme bindingUDP-glucuronosyltransferase 1A7Homo sapiens (human)
protein homodimerization activityUDP-glucuronosyltransferase 1A7Homo sapiens (human)
protein heterodimerization activityUDP-glucuronosyltransferase 1A7Homo sapiens (human)
retinoic acid bindingUDP-glucuronosyltransferase 1A10Homo sapiens (human)
protein kinase C bindingUDP-glucuronosyltransferase 1A10Homo sapiens (human)
glucuronosyltransferase activityUDP-glucuronosyltransferase 1A10Homo sapiens (human)
enzyme bindingUDP-glucuronosyltransferase 1A10Homo sapiens (human)
protein homodimerization activityUDP-glucuronosyltransferase 1A10Homo sapiens (human)
protein heterodimerization activityUDP-glucuronosyltransferase 1A10Homo sapiens (human)
retinoic acid bindingUDP-glucuronosyltransferase 1A8Homo sapiens (human)
enzyme inhibitor activityUDP-glucuronosyltransferase 1A8Homo sapiens (human)
steroid bindingUDP-glucuronosyltransferase 1A8Homo sapiens (human)
fatty acid bindingUDP-glucuronosyltransferase 1A8Homo sapiens (human)
glucuronosyltransferase activityUDP-glucuronosyltransferase 1A8Homo sapiens (human)
enzyme bindingUDP-glucuronosyltransferase 1A8Homo sapiens (human)
protein homodimerization activityUDP-glucuronosyltransferase 1A8Homo sapiens (human)
protein heterodimerization activityUDP-glucuronosyltransferase 1A8Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (56)

Processvia Protein(s)Taxonomy
endoplasmic reticulumUDP-glucuronosyltransferase 1A9Homo sapiens (human)
endoplasmic reticulum membraneUDP-glucuronosyltransferase 1A9Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A9Homo sapiens (human)
nuclear bodyCellular tumor antigen p53Homo sapiens (human)
nucleusCellular tumor antigen p53Homo sapiens (human)
nucleoplasmCellular tumor antigen p53Homo sapiens (human)
replication forkCellular tumor antigen p53Homo sapiens (human)
nucleolusCellular tumor antigen p53Homo sapiens (human)
cytoplasmCellular tumor antigen p53Homo sapiens (human)
mitochondrionCellular tumor antigen p53Homo sapiens (human)
mitochondrial matrixCellular tumor antigen p53Homo sapiens (human)
endoplasmic reticulumCellular tumor antigen p53Homo sapiens (human)
centrosomeCellular tumor antigen p53Homo sapiens (human)
cytosolCellular tumor antigen p53Homo sapiens (human)
nuclear matrixCellular tumor antigen p53Homo sapiens (human)
PML bodyCellular tumor antigen p53Homo sapiens (human)
transcription repressor complexCellular tumor antigen p53Homo sapiens (human)
site of double-strand breakCellular tumor antigen p53Homo sapiens (human)
germ cell nucleusCellular tumor antigen p53Homo sapiens (human)
chromatinCellular tumor antigen p53Homo sapiens (human)
transcription regulator complexCellular tumor antigen p53Homo sapiens (human)
protein-containing complexCellular tumor antigen p53Homo sapiens (human)
extracellular regionInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
nucleusInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
cytoplasmInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
peroxisomal membraneInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
cytosolInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
membraneInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
secretory granule lumenInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
extracellular exosomeInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
ficolin-1-rich granule lumenInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
cytoplasmInosine-5'-monophosphate dehydrogenase 2Homo sapiens (human)
plasma membraneGamma-aminobutyric acid receptor subunit gamma-2Rattus norvegicus (Norway rat)
plasma membraneGlutamate receptor 1Rattus norvegicus (Norway rat)
plasma membraneGlutamate receptor 2Rattus norvegicus (Norway rat)
extracellular regionInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
nucleusInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
cytoplasmInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
cytosolInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
secretory granule lumenInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
azurophil granule lumenInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
ficolin-1-rich granule lumenInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
cytoplasmInosine-5'-monophosphate dehydrogenase 1 Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
endoplasmic reticulum membraneUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
plasma membraneUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
perinuclear region of cytoplasmUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
endoplasmic reticulum chaperone complexUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
cytochrome complexUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A1 Homo sapiens (human)
nucleusPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
nucleusPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
nucleoplasmPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
cytosolPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
intracellular membrane-bounded organellePeroxisome proliferator-activated receptor gammaHomo sapiens (human)
RNA polymerase II transcription regulator complexPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
chromatinPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
receptor complexPeroxisome proliferator-activated receptor gammaHomo sapiens (human)
platelet alpha granule membraneAlpha-synucleinHomo sapiens (human)
extracellular regionAlpha-synucleinHomo sapiens (human)
extracellular spaceAlpha-synucleinHomo sapiens (human)
nucleusAlpha-synucleinHomo sapiens (human)
cytoplasmAlpha-synucleinHomo sapiens (human)
mitochondrionAlpha-synucleinHomo sapiens (human)
lysosomeAlpha-synucleinHomo sapiens (human)
cytosolAlpha-synucleinHomo sapiens (human)
plasma membraneAlpha-synucleinHomo sapiens (human)
cell cortexAlpha-synucleinHomo sapiens (human)
actin cytoskeletonAlpha-synucleinHomo sapiens (human)
membraneAlpha-synucleinHomo sapiens (human)
inclusion bodyAlpha-synucleinHomo sapiens (human)
axonAlpha-synucleinHomo sapiens (human)
growth coneAlpha-synucleinHomo sapiens (human)
synaptic vesicle membraneAlpha-synucleinHomo sapiens (human)
perinuclear region of cytoplasmAlpha-synucleinHomo sapiens (human)
postsynapseAlpha-synucleinHomo sapiens (human)
supramolecular fiberAlpha-synucleinHomo sapiens (human)
protein-containing complexAlpha-synucleinHomo sapiens (human)
cytoplasmAlpha-synucleinHomo sapiens (human)
axon terminusAlpha-synucleinHomo sapiens (human)
neuronal cell bodyAlpha-synucleinHomo sapiens (human)
plasma membraneGamma-aminobutyric acid receptor subunit alpha-1Rattus norvegicus (Norway rat)
plasma membraneGamma-aminobutyric acid receptor subunit beta-2Rattus norvegicus (Norway rat)
intracellular non-membrane-bounded organelleTAR DNA-binding protein 43Homo sapiens (human)
nucleusTAR DNA-binding protein 43Homo sapiens (human)
nucleoplasmTAR DNA-binding protein 43Homo sapiens (human)
perichromatin fibrilsTAR DNA-binding protein 43Homo sapiens (human)
mitochondrionTAR DNA-binding protein 43Homo sapiens (human)
cytoplasmic stress granuleTAR DNA-binding protein 43Homo sapiens (human)
nuclear speckTAR DNA-binding protein 43Homo sapiens (human)
interchromatin granuleTAR DNA-binding protein 43Homo sapiens (human)
nucleoplasmTAR DNA-binding protein 43Homo sapiens (human)
chromatinTAR DNA-binding protein 43Homo sapiens (human)
Elg1 RFC-like complexATPase family AAA domain-containing protein 5Homo sapiens (human)
nucleusATPase family AAA domain-containing protein 5Homo sapiens (human)
cytoplasmAtaxin-2Homo sapiens (human)
Golgi apparatusAtaxin-2Homo sapiens (human)
trans-Golgi networkAtaxin-2Homo sapiens (human)
cytosolAtaxin-2Homo sapiens (human)
cytoplasmic stress granuleAtaxin-2Homo sapiens (human)
membraneAtaxin-2Homo sapiens (human)
perinuclear region of cytoplasmAtaxin-2Homo sapiens (human)
ribonucleoprotein complexAtaxin-2Homo sapiens (human)
cytoplasmic stress granuleAtaxin-2Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A7Homo sapiens (human)
endoplasmic reticulum membraneUDP-glucuronosyltransferase 1A7Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A7Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A10Homo sapiens (human)
endoplasmic reticulum membraneUDP-glucuronosyltransferase 1A10Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A10Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A8Homo sapiens (human)
endoplasmic reticulum membraneUDP-glucuronosyltransferase 1A8Homo sapiens (human)
endoplasmic reticulumUDP-glucuronosyltransferase 1A8Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (591)

Assay IDTitleYearJournalArticle
AID504749qHTS profiling for inhibitors of Plasmodium falciparum proliferation2011Science (New York, N.Y.), Aug-05, Volume: 333, Issue:6043
Chemical genomic profiling for antimalarial therapies, response signatures, and molecular targets.
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
AID1745845Primary qHTS for Inhibitors of ATXN expression
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID540299A screen for compounds that inhibit the MenB enzyme of Mycobacterium tuberculosis2010Bioorganic & medicinal chemistry letters, Nov-01, Volume: 20, Issue:21
Synthesis and SAR studies of 1,4-benzoxazine MenB inhibitors: novel antibacterial agents against Mycobacterium tuberculosis.
AID588519A screen for compounds that inhibit viral RNA polymerase binding and polymerization activities2011Antiviral research, Sep, Volume: 91, Issue:3
High-throughput screening identification of poliovirus RNA-dependent RNA polymerase inhibitors.
AID1296008Cytotoxic Profiling of Annotated Libraries Using Quantitative High-Throughput Screening2020SLAS discovery : advancing life sciences R & D, 01, Volume: 25, Issue:1
Cytotoxic Profiling of Annotated and Diverse Chemical Libraries Using Quantitative High-Throughput Screening.
AID1346987P-glycoprotein substrates identified in KB-8-5-11 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1346986P-glycoprotein substrates identified in KB-3-1 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1803439Enzyme Assay from Article 10.3109/14756366.2013.793184: \\Design, synthesis and biological evaluation of novel inosine 5'-monophosphate dehydrogenase (IMPDH) inhibitors.\\2014Journal of enzyme inhibition and medicinal chemistry, Jun, Volume: 29, Issue:3
Design, synthesis and biological evaluation of novel inosine 5'-monophosphate dehydrogenase (IMPDH) inhibitors.
AID1797842IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: \\Acridone-based inhibitors of inosine 5'-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethy2007Journal of medicinal chemistry, Jul-26, Volume: 50, Issue:15
Acridone-based inhibitors of inosine 5'-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).
AID1797841IMPDH Type 2 Enzyme Assay from Article 10.1021/jm070568j: \\Probing Binding Requirements of Type I and Type II Isoforms of Inosine Monophosphate Dehydrogenase with Adenine-Modified Nicotinamide Adenine Dinucleotide Analogues.\\2007Journal of medicinal chemistry, Nov-15, Volume: 50, Issue:23
Probing binding requirements of type I and type II isoforms of inosine monophosphate dehydrogenase with adenine-modified nicotinamide adenine dinucleotide analogues.
AID1797840IMPDH Type 1 Enzyme Assay from Article 10.1021/jm070568j: \\Probing Binding Requirements of Type I and Type II Isoforms of Inosine Monophosphate Dehydrogenase with Adenine-Modified Nicotinamide Adenine Dinucleotide Analogues.\\2007Journal of medicinal chemistry, Nov-15, Volume: 50, Issue:23
Probing binding requirements of type I and type II isoforms of inosine monophosphate dehydrogenase with adenine-modified nicotinamide adenine dinucleotide analogues.
AID1347167Vero cells viability qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347155Optimization screen NINDS Rhodamine qHTS for Zika virus inhibitors: Linked NS2B-NS3 protease assay2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1508630Primary qHTS for small molecule stabilizers of the endoplasmic reticulum resident proteome: Secreted ER Calcium Modulated Protein (SERCaMP) assay2021Cell reports, 04-27, Volume: 35, Issue:4
A target-agnostic screen identifies approved drugs to stabilize the endoplasmic reticulum-resident proteome.
AID1347151Optimization of GU AMC qHTS for Zika virus inhibitors: Unlinked NS2B-NS3 protease assay2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347150Optimization screen NINDS AMC qHTS for Zika virus inhibitors: Linked NS2B-NS3 protease assay2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347154Primary screen GU AMC qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347169Tertiary RLuc qRT-PCR qHTS assay for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347149Furin counterscreen qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347168HepG2 cells viability qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347122qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347097qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Saos-2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347099qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB1643 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347138qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D caspase screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347106qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347123qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh41 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347125qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh18 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347103qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for OHS-50 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347096qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347111qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SK-N-MC cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347095qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB-EBc1 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347083qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: Viability assay - alamar blue signal for LASV Primary Screen2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347086qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lymphocytic Choriomeningitis Arenaviruses (LCMV): LCMV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347128qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for OHS-50 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347091qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347082qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: LASV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347141qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D viability screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347425Rhodamine-PBP qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1347139qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D viability screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347101qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-12 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347126qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh30 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347098qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347114qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for DAOY cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347124qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347118qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347102qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh18 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347104qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347112qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for BT-12 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347140qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D viability screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347135qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D viability screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347119qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347127qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Saos-2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347090qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for DAOY cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347094qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-37 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347110qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for A673 cells)2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347137qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D viability screen for Daoy cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347115qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for NB-EBc1 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347121qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347093qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-MC cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347108qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh41 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347117qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for BT-37 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347407qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS Pharmaceutical Collection2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID1347424RapidFire Mass Spectrometry qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1347092qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for A673 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347105qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347109qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for NB1643 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347107qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh30 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347129qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347089qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347116qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347113qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for LAN-5 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347100qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for LAN-5 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347136qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Orthogonal 3D viability screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID95166Inhibitory activity against growth of human erythroleukemia K652 cells1998Journal of medicinal chemistry, Feb-12, Volume: 41, Issue:4
Synthesis of a methylenebis(phosphonate) analogue of mycophenolic adenine dinucleotide: a glucuronidation-resistant MAD analogue of NAD.
AID588217FDA HLAED, serum glutamic pyruvic transaminase (SGPT) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID1186329Cytotoxicity against mock-infected MDBK cells after 48 to 96 hrs by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID92486In vitro inhibition of inosine Inosine-5'-monophosphate dehydrogenase1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID1220784Fraction unbound in mouse plasma by ultracentrifugation method2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID588215FDA HLAED, alkaline phosphatase increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID155457Inhibitory activity against proliferation of peripheral blood mononuclear cells2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Quinolone-based IMPDH inhibitors: introduction of basic residues on ring D and SAR of the corresponding mono, di and benzofused analogues.
AID625289Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for liver disease2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1186340Antiviral activity against HSV1 infected in african green monkey Vero 76 cells assessed as reduction in plaque number after 3 days by plaque reduction assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID342523Inhibition of human IMP dehydrogenase 22008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
Bis(sulfonamide) isosters of mycophenolic adenine dinucleotide analogues: inhibition of inosine monophosphate dehydrogenase.
AID657091Immunosuppressive activity in C57BL/6 mouse T lymphocytes assessed as inhibition of cell proliferation after 72 hrs by MTT assay2012Bioorganic & medicinal chemistry letters, May-01, Volume: 22, Issue:9
Penicacids A-C, three new mycophenolic acid derivatives and immunosuppressive activities from the marine-derived fungus Penicillium sp. SOF07.
AID1422698Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.078 uM assessed as Km for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentratio2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID92622Inhibition of human inosine-5'-monophosphate dehydrogenase 22002Journal of medicinal chemistry, May-23, Volume: 45, Issue:11
Discovery of N-[2-[2-[[3-methoxy-4-(5-oxazolyl)phenyl]amino]-5-oxazolyl]phenyl]-N-methyl-4- morpholineacetamide as a novel and potent inhibitor of inosine monophosphate dehydrogenase with excellent in vivo activity.
AID1717751Antiviral activity against wild type HCoV-OC43 infected in BHK-21 cells incubated for 72 hrs by RT-PCR method2020Journal of medicinal chemistry, 11-25, Volume: 63, Issue:22
Chinese Therapeutic Strategy for Fighting COVID-19 and Potential Small-Molecule Inhibitors against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2).
AID300649Inhibition of mouse 3T3-L1 cell differentiation at 0.2 uM2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID304372Inhibition of human IMPDH 22007Journal of medicinal chemistry, Dec-27, Volume: 50, Issue:26
Dual inhibitors of inosine monophosphate dehydrogenase and histone deacetylases for cancer treatment.
AID1503774Cytotoxicity against human HL cells assessed as cell viability at 50 uM after 72 hrs by resazurin dye based fluorescence assay relative to control2017Journal of natural products, 10-27, Volume: 80, Issue:10
Identification of Privileged Antichlamydial Natural Products by a Ligand-Based Strategy.
AID685500HARVARD: Cytotoxicity in HepG2 cell line2012Proceedings of the National Academy of Sciences of the United States of America, May-29, Volume: 109, Issue:22
Liver-stage malaria parasites vulnerable to diverse chemical scaffolds.
AID436483Antiviral activity against Respiratory syncytial virus A2 infected in Vero-76 cells after 5 days by plaque reduction assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID1223457Drug metabolism in diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1261314Antiviral activity against Reo-1 virus infected in BHK-21 cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID195367Inhibitory activity against the PDGF-induced proliferation of rat aortic smooth muscle cell (RAoSMC)2004Bioorganic & medicinal chemistry letters, Jul-05, Volume: 14, Issue:13
Synthesis and biological evaluation of benzimidazole-4,7-diones that inhibit vascular smooth muscle cell proliferation.
AID436486Antiviral activity against Human poliovirus 1 strain Sabin after 2 days by plaque reduction assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID1220797Volume of distribution at steady state in human2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1763990Cytotoxicity against human FaDu cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID1380563Growth inhibition of Acinetobacter baumannii at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID226618Ratio of IC50(Type I) to IC50(Type II)2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Novel inhibitors of IMPDH: a highly potent and selective quinolone-based series.
AID134854In vivo inhibition of plaques per million after 25 mg/kg oral administration in mice.1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID46396Inhibition of CEM (human leukemia) cell proliferation.2003Bioorganic & medicinal chemistry letters, Oct-20, Volume: 13, Issue:20
3-cyanoindole-based inhibitors of inosine monophosphate dehydrogenase: synthesis and initial structure-activity relationships.
AID1336608Antiviral activity against Para-influenza-3 virus infected in African green monkey Vero cells assessed as protection against virus-induced cytopathic effect measured after 3 to 5 days by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID774867Antiviral activity against Coxsackie B3 virus Nancy in african green monkey Vero cells assessed as inhibition virus-induced cytopathic effect after 48 hrs2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID1223460Uncompetitive substrate inhibition of UGT2B7 in diabetic human kidney microsomes assessed as reduction in enzyme-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1234128Drug metabolism in human liver microsomes assessed as glucuronide formation at 600 uM incubated for 60 mins in presence of UDPGA by LC-MS/MS method2015Journal of medicinal chemistry, Jul-23, Volume: 58, Issue:14
Synthesis and Pharmacokinetic Evaluation of Siderophore Biosynthesis Inhibitors for Mycobacterium tuberculosis.
AID1402051Antibacterial activity against Acinetobacter baumannii ATCC 17978 after 18 to 20 hrs by spectrophotometry based microdilution method2018European journal of medicinal chemistry, Jan-01, Volume: 143Synthesis and antimicrobial activity of amino acid and peptide derivatives of mycophenolic acid.
AID1211279Renal clearance in human2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID538356Cytotoxicity against human K562 cells after 72 hrs by trypan blue exclusion assay2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID1503776Antichlamydial activity against Chlamydia pneumoniae K7 infected in HL cells after 70 hrs by fluorescent microscopic analysis2017Journal of natural products, 10-27, Volume: 80, Issue:10
Identification of Privileged Antichlamydial Natural Products by a Ligand-Based Strategy.
AID538357Selectivity ratio of IC50 for human K562 cells to IC50 for human IMPDH22010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID92617Compound was tested for inhibition of human recombinant type II Inosine Monophosphate Dehydrogenase at pH 8.01996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID588218FDA HLAED, lactate dehydrogenase (LDH) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID1380560Growth inhibition of Escherichia coli at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1220798Half life in human2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID588213Literature-mined compound from Fourches et al multi-species drug-induced liver injury (DILI) dataset, effect in non-rodents2010Chemical research in toxicology, Jan, Volume: 23, Issue:1
Cheminformatics analysis of assertions mined from literature that describe drug-induced liver injury in different species.
AID1408890Cytotoxicity against human MDA-MB-231 cells assessed as reduction in cell viability after 48 hrs by MTT assay2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID1422705Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.156 uM assessed as Kcat for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID264226Cell cycle arrest at G1 phase in HUVEC at 1 uM2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID774868Selectivity index, ratio of CC50 for african green monkey Vero cells to IC50 for Coxsackie B3 virus Nancy2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID1186331Cytotoxicity against mock-infected BHK21 cells after 48 to 96 hrs by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID588219FDA HLAED, gamma-glutamyl transferase (GGT) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID134134In vivo antibody response to sheep red blood cells(SRBC) by plaque-forming cells in the spleen (PFC/spleen) after 25 mg/kg oral administration in mice.1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID45812In vitro secondary T-cell (CEM) proliferation assay2002Journal of medicinal chemistry, May-23, Volume: 45, Issue:11
Discovery of N-[2-[2-[[3-methoxy-4-(5-oxazolyl)phenyl]amino]-5-oxazolyl]phenyl]-N-methyl-4- morpholineacetamide as a novel and potent inhibitor of inosine monophosphate dehydrogenase with excellent in vivo activity.
AID530144Inhibition of Aspergillus fumigatus-mediated dendritic cell maturation assessed as decreased CD83 expression at 10 uM2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID1763992Cytotoxicity against human MDA-MB-231 cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID668551Antiproliferative activity against human CCRF-CEM cells after 48 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and biological activity of novel mycophenolic acid conjugates containing nitro-acridine/acridone derivatives.
AID538354Inhibition of human IMPDH2 expressed in Escherichia coli strain BL21(DE3) after 60 mins2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID1485235Antiviral activity against Yellow fever virus infected in African green monkey Vero cells assessed as inhibition of viral-induced cytopathic effect measured after 3 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID1422699Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.078 uM assessed as Kcat for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1220794Plasma clearance in human2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1689898Immunosuppression-mediated antiproliferative activity against human Jurkat T cells assessed as inhibition of cell growth incubated for 48 hrs by VPD450 staining based flow cytometry2020European journal of medicinal chemistry, Mar-01, Volume: 189Immunosuppressive properties of amino acid and peptide derivatives of mycophenolic acid.
AID774865Selectivity index, ratio of CC50 for human Huh7.5 cells to IC50 for HCV expressing pFL-J6/JFH/JC12013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID1261311Cytotoxicity against african green monkey Vero 76 cells assessed as cell viability after 48 to 96 hrs by crystal violet staining method2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1422680Inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) using IMP as substrate in presence of NAD2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID657090Inhibition of IMPDH2 using inosine 5'-monophosphate as substrate by spectrophotometry2012Bioorganic & medicinal chemistry letters, May-01, Volume: 22, Issue:9
Penicacids A-C, three new mycophenolic acid derivatives and immunosuppressive activities from the marine-derived fungus Penicillium sp. SOF07.
AID1408898Cytotoxicity against human PBMC assessed as cell viability at 10 uM after 48 hrs by MTT assay relative to control2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID328839Suppression of inclusion formation in zebrafish embryo expressing poly Q102-GFP protein after 24 hrs at 1 uM2007The Journal of biological chemistry, Mar-23, Volume: 282, Issue:12
Identification of anti-prion compounds as efficient inhibitors of polyglutamine protein aggregation in a zebrafish model.
AID1223439Drug metabolism in diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID625290Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for liver fatty2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1261342Antiviral activity against Vaccinia virus2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1402052Antibacterial activity against Escherichia coli ATCC 8739 after 18 to 20 hrs by spectrophotometry based microdilution method2018European journal of medicinal chemistry, Jan-01, Volume: 143Synthesis and antimicrobial activity of amino acid and peptide derivatives of mycophenolic acid.
AID91401Inhibitory activity against (IMPDH) inosine 5'-monophosphate dehydrogenase2003Bioorganic & medicinal chemistry letters, May-19, Volume: 13, Issue:10
Discovery of novel low molecular weight inhibitors of IMPDH via virtual needle screening.
AID1261324Antiviral activity against HIV-1 infected in MT4 cells assessed as inhibition of virus-induced cytopathogenicity after 4 days by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1498119Antiviral activity against Parainfluenza virus 3 infected in human HeLa cells assessed as inhibition of virus-induced cytopathicity2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID278370Effect on metabolism of 10 ug/ml RBV in Vero E6 cells assessed as RBV-TP concentration at 2 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1422695Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.078 uM assessed as Km for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentratio2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID580127Antiproliferative activity against human K562 cells after 72 hrs by MTS assay2011Bioorganic & medicinal chemistry, Mar-01, Volume: 19, Issue:5
Cofactor-type inhibitors of inosine monophosphate dehydrogenase via modular approach: targeting the pyrophosphate binding sub-domain.
AID1689899Immunosuppression-mediated antiproliferative activity against anti-CD3/anti-CD28-stimulated human PBMC cells assessed as inhibition of cell growth incubated for 72 hrs by VPD450 staining based flow cytometry2020European journal of medicinal chemistry, Mar-01, Volume: 189Immunosuppressive properties of amino acid and peptide derivatives of mycophenolic acid.
AID1675164Cytotoxicity activity against African green monkey Vero cells assessed as reduction in cell viability by MTT assay2020Journal of natural products, 08-28, Volume: 83, Issue:8
Diverse Types of Diterpenoids with an Aromatized C Ring from the Twigs of
AID1422691Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.039 uM assessed as Kcat for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID304371Inhibition of human IMPDH 12007Journal of medicinal chemistry, Dec-27, Volume: 50, Issue:26
Dual inhibitors of inosine monophosphate dehydrogenase and histone deacetylases for cancer treatment.
AID1223430Uncompetitive substrate inhibition of UGT2B7 in non-diabetic human liver microsomes assessed as reduction in enzyme-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID530146Inhibition of Aspergillus fumigatus-mediated dendritic cell maturation assessed as decreased CD1a expression at 10 uM2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID278369Effect on metabolism of 10 ug/ml RBV in Vero E6 cells assessed as RBV-TP concentration at 1 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1223464Uncompetitive substrate inhibition of UGT2B7 in diabetic human kidney microsomes assessed as reduction in enzyme-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1380565Growth inhibition of Cryptococcus neoformans var. grubii at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1211283Fraction metabolized glucuronidation in human liver microsomes in presence of UDP-glucuronosyltransferase and bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1422690Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.039 uM assessed as Km for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentratio2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID588214FDA HLAED, liver enzyme composite activity2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID134852In vivo inhibition of plaque-forming cells in the spleen (PFC/spleen) after 25 mg/kg oral administration in mice.1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID115874Tested for the inhibition of murine PFC response at 25 mg/kg dose1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID1223461Drug metabolism in diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1181041Immunosuppressive activity in anti-CD3/anti-CD28-induced human PBMC after 96 hrs by flow cytometry2014Journal of natural products, Jul-25, Volume: 77, Issue:7
Nitrogen-containing dihydro-β-agarofuran derivatives from Tripterygium wilfordii.
AID1402053Antibacterial activity against ESBL positive Klebsiella pneumoniae ATCC 700603 after 18 to 20 hrs by spectrophotometry based microdilution method2018European journal of medicinal chemistry, Jan-01, Volume: 143Synthesis and antimicrobial activity of amino acid and peptide derivatives of mycophenolic acid.
AID1717769Antiviral activity against HCoV-NL63 infected in Rhesus macaque LLC-MK2 cells incubated for 72 hrs by RT-PCR method2020Journal of medicinal chemistry, 11-25, Volume: 63, Issue:22
Chinese Therapeutic Strategy for Fighting COVID-19 and Potential Small-Molecule Inhibitors against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2).
AID1503328Antiviral activity against Vaccinia virus Lister ATCC VR 1549 infected in African green monkey Vero 76 cells assessed as protection against virus-induced cytopathic effect after 3 days by crystal violet staining-based plaque reduction assay2017European journal of medicinal chemistry, Dec-01, Volume: 141Inhibitors of Yellow Fever Virus replication based on 1,3,5-triphenyl-4,5-dihydropyrazole scaffold: Design, synthesis and antiviral evaluation.
AID1211220Ratio of unbound intrinsic glucuronidation clearance in human kidney microsomes in presence of 1% bovine serum albumin to unbound intrinsic glucuronidation clearance in human kidney microsomes in absence of bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1204082Antiviral activity against Vaccinia virus infected in African green monkey Vero 76 cells after 3 days by plaque reduction assay2015Bioorganic & medicinal chemistry letters, Jun-01, Volume: 25, Issue:11
N-((1,3-Diphenyl-1H-pyrazol-4-yl)methyl)anilines: A novel class of anti-RSV agents.
AID1261312Antiviral activity against BVDV infected in MDBK cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1485239Antiviral activity against Reovirus 1 infected in African green monkey Vero cells assessed as inhibition of viral-induced cytopathic effect measured after 3 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID264223Antiproliferative activity against Jurkat T cells as measured by [3H]thymidine incorporation2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID1422696Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.078 uM assessed as Kcat for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1220786Fraction unbound in monkey plasma by ultracentrifugation method2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID776764Cytotoxicity against human PBMC after 72 hrs by MTT assay2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID392504Antiviral activity against Cowpox virus2009Bioorganic & medicinal chemistry, Jan-15, Volume: 17, Issue:2
Unified QSAR approach to antimicrobials. 4. Multi-target QSAR modeling and comparative multi-distance study of the giant components of antiviral drug-drug complex networks.
AID1211248Unbound intrinsic glucuronidation clearance in human liver microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of UDP-glucuronosyltransferase and 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID436479Antiviral activity against Yellow fever virus infected in BHK-21 cells assessed as inhibition of virus-induced cytopathicity after 3 to 4 days by MTT assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID1336611Antiviral activity against Coxsackie virus B4 infected in African green monkey Vero cells assessed as protection against virus-induced cytopathic effect measured after 3 to 5 days by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID624637Drug glucuronidation reaction catalyzed by human recombinant UGT1A92005Pharmacology & therapeutics, Apr, Volume: 106, Issue:1
UDP-glucuronosyltransferases and clinical drug-drug interactions.
AID1223438Uncompetitive substrate inhibition of UGT2B7 in non-diabetic human kidney microsomes assessed as reduction in enzyme-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID90471In vitro inhibition of human lymphocyte proliferation in response to staphylococcus protein A(SPA) was determined1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID699264Selectivity index, ratio of CC50 for BHK cells to IC50 for Dengue virus 2 infected in BHK-D2RepT cells2012Journal of medicinal chemistry, Jul-26, Volume: 55, Issue:14
Discovery of novel small molecule inhibitors of dengue viral NS2B-NS3 protease using virtual screening and scaffold hopping.
AID1689902Selectivity index, ratio of IC50 for cytotoxicity against human PBMC by MTT assay to EC50 for antiproliferative activity against human PBMC by VPD450 staining based flow cytometry2020European journal of medicinal chemistry, Mar-01, Volume: 189Immunosuppressive properties of amino acid and peptide derivatives of mycophenolic acid.
AID977602Inhibition of sodium fluorescein uptake in OATP1B3-transfected CHO cells at an equimolar substrate-inhibitor concentration of 10 uM2013Molecular pharmacology, Jun, Volume: 83, Issue:6
Structure-based identification of OATP1B1/3 inhibitors.
AID538353Inhibition of human IMPDH1 expressed in Escherichia coli strain BL21(DE3) after 60 mins2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID1422703Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.156 uM assessed as Vmax for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1422681Inhibition of recombinant human His-tagged IMPDH1 expressed in Escherichia coli BL21 (DE3) using IMP as substrate in presence of NAD2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1223459Ratio of Vmax to Km in diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1223443Drug metabolism in diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID625292Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) combined score2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID624631Drug glucuronidation reaction catalyzed by human recombinant UGT1A102005Pharmacology & therapeutics, Apr, Volume: 106, Issue:1
UDP-glucuronosyltransferases and clinical drug-drug interactions.
AID1186334Cytotoxicity against mock-infected african green monkey Vero 76 cells after 48 to 96 hrs by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID114692Effective dose was measured in mice as concentration required to inhibit 50% inhibition of Murine PFC response1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID1485238Antiviral activity against Sindbis virus infected in African green monkey Vero cells assessed as inhibition of viral-induced cytopathic effect measured after 3 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID278371Effect on metabolism of 10 ug/ml RBV in Vero E6 cells assessed as RBV-TP concentration at 5 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID210432The human T-lymphoblastCEM cell (ATCC) proliferation inhibitory activity was determined2002Bioorganic & medicinal chemistry letters, Oct-21, Volume: 12, Issue:20
Novel amide-based inhibitors of inosine 5'-monophosphate dehydrogenase.
AID1261309Cytotoxicity against MDBK cells assessed as cell viability after 48 to 96 hrs by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID331649Antiproliferative activity against PDGF-induced rat aortic VSMC by colorimetric assay2008Bioorganic & medicinal chemistry letters, May-01, Volume: 18, Issue:9
Design, synthesis and evaluation of 2-phenyl-1H-benzo[d]imidazole-4,7-diones as vascular smooth muscle cell proliferation inhibitors.
AID776770Inhibition of human IMPDH2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID1220783Drug metabolism in human assessed as glucuronide concentration in bile and urine2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID348818Inhibition of human IMPDH12008Bioorganic & medicinal chemistry, Oct-15, Volume: 16, Issue:20
Mycophenolic acid analogs with a modified metabolic profile.
AID530147Effect on polymorphonuclear neutrophil killing of Aspergillus fumigatus assessed as survival rate of fungi at 10 ug/ml (Rvb = 53% +/- 11%)2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID1223425Ratio of Vmax to Km in non-diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID264228Cell cycle arrest at G2/M phase in HUVEC at 1 uM2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID304376Differentiation of K562 cells at 0.25 uM after 5 days by MTS assay2007Journal of medicinal chemistry, Dec-27, Volume: 50, Issue:26
Dual inhibitors of inosine monophosphate dehydrogenase and histone deacetylases for cancer treatment.
AID1364946Cytotoxicity against African green monkey Vero cells by neutral red dye uptake assay2017Bioorganic & medicinal chemistry, 08-15, Volume: 25, Issue:16
The medicinal chemistry of Chikungunya virus.
AID1186336Antiviral activity against Poliovirus type 1 Sabin strain infected in african green monkey Vero 76 cells assessed as reduction in plaque number after 2 days by plaque reduction assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID1409614Overall antiviral activity against SARS-CoV-2 (isolate France/IDF0372/2020) in the Vero E6 cell line at 48 h based on three assays 1) detection of viral RNA by qRT-PCR (targeting the N-gene), 2) plaque assay using lysate 3 days after addition of compound 2020Nature, 07, Volume: 583, Issue:7816
A SARS-CoV-2 protein interaction map reveals targets for drug repurposing.
AID697852Inhibition of electric eel AChE at 2 mg/ml by Ellman's method2012Bioorganic & medicinal chemistry, Nov-15, Volume: 20, Issue:22
Exploration of natural compounds as sources of new bifunctional scaffolds targeting cholinesterases and beta amyloid aggregation: the case of chelerythrine.
AID278365Effect on metabolism of 40 ug/ml RBV in Vero E6 cells assessed as RBV-MP concentration at 5 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1763995Cytotoxicity against human MNNK-1 cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID625280Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for cholecystitis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1223429Ratio of Vmax to Km in non-diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1186339Antiviral activity against Vaccinia Virus infected in african green monkey Vero 76 cells assessed as reduction in plaque number after 3 days by plaque reduction assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID699266Antiviral activity against Dengue virus 2 infected in BHK-D2RepT cells assessed as reduction in viral replication incubated for 48 hrs by luciferase reporter replicon-based assay2012Journal of medicinal chemistry, Jul-26, Volume: 55, Issue:14
Discovery of novel small molecule inhibitors of dengue viral NS2B-NS3 protease using virtual screening and scaffold hopping.
AID1336607Cytotoxicity against African green monkey Vero cells assessed as alterations in cell morphology by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID1211280Fraction unbound in human plasma2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1485237Antiviral activity against Coxsackie B4 virus infected in African green monkey Vero cells assessed as inhibition of viral-induced cytopathic effect measured after 3 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID1060472Binding affinity to human IMPDH2014Bioorganic & medicinal chemistry letters, Jan-01, Volume: 24, Issue:1
NAD-based inhibitors with anticancer potential.
AID530141Induction of IL-2 expression in mouse dendritic cell co-cultured with Aspergillus fumigatus germ tube at 10 uM after 7 days by RT-PCR2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID436476Cytotoxicity against human MT4 cells after 96 hrs by MTT assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID1380564Growth inhibition of Candida albicans at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1631433Antiviral activity against Dengue virus 2 16681 infected in human HuH7 cells after 72 hrs by indirect immunofluorescent flow cytometry2016Journal of medicinal chemistry, 06-23, Volume: 59, Issue:12
The Medicinal Chemistry of Dengue Virus.
AID1498131Antiviral activity against Sindbis Virus infected in African green monkey Vero cells assessed as inhibition of virus-induced cytopathicity2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID98904Inhibitory activity of compound against L1210 leukemia cells in tissue culture1996Journal of medicinal chemistry, Jan-05, Volume: 39, Issue:1
Synthesis and modeling studies with monocyclic analogues of mycophenolic acid.
AID537735Binding affinity to Candida albicans CaMdr1p expressed in yeast AD1-8u2010European journal of medicinal chemistry, Nov, Volume: 45, Issue:11
Analysis of physico-chemical properties of substrates of ABC and MFS multidrug transporters of pathogenic Candida albicans.
AID1211284Glucuronidation clearance in human liver microsomes2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1223452Drug metabolism in non-diabetic human kidney microsomes assessed as UGT2B7*1c mutant-mediated intrinsic clearance of mycophenolic acid acyl glucuronide by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1408889Inhibition of human IMPDH2 using IMP as substrate in presence of NAD+ after 30 mins2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID624635Drug glucuronidation reaction catalyzed by human recombinant UGT1A72005Pharmacology & therapeutics, Apr, Volume: 106, Issue:1
UDP-glucuronosyltransferases and clinical drug-drug interactions.
AID1220799Drug metabolism in gallbladder-cannulated mouse assessed as glucuronide concentration in bile and urine at 0.2 mg/kg, iv up to 24 hrs by LC/MS/MS analysis2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1261317Antiviral activity against Human poliovirus 1 Sabin infected in african green monkey Vero76 cells assessed as inhibition of virus-induced cytopathogenicity by plaque reduction assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID264224Antiproliferative activity against HUVEC in presence of 20 uM guanine as measured by [3H]thymidine incorporation2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID530145Inhibition of Aspergillus fumigatus-mediated dendritic cell maturation assessed as decreased CD40 expression at 10 uM2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID1211285Glucuronidation clearance in human liver microsomes in presence of bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID118202Inhibition of Murine plaque forming cells response, activity expressed as Potency Relative to 1a1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID1380553Inhibition of Mycobacterium tuberculosis GuaB22018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID292040Inhibition of human IMPDH22007Journal of medicinal chemistry, Jul-26, Volume: 50, Issue:15
Acridone-based inhibitors of inosine 5'-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).
AID1223431Drug metabolism in non-diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID261583Inhibition of West Nile viral Rluc-Neo-Rep in BHK21 cell line2006Journal of medicinal chemistry, Mar-23, Volume: 49, Issue:6
Identification of compounds with anti-West Nile Virus activity.
AID1223426Uncompetitive substrate inhibition of UGT2B7 in non-diabetic human liver microsomes assessed as reduction in enzyme-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID278379Decrease in GTP level in Vero E6 cells at 40 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID92606Inhibitory activity against inosine monophosphate dehydrogenase IMPDH type I2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Novel inhibitors of IMPDH: a highly potent and selective quinolone-based series.
AID1503324Cytotoxicity against African green monkey Vero 76 cells assessed as decrease in cell viability after 48 to 96 hrs by MTT assay2017European journal of medicinal chemistry, Dec-01, Volume: 141Inhibitors of Yellow Fever Virus replication based on 1,3,5-triphenyl-4,5-dihydropyrazole scaffold: Design, synthesis and antiviral evaluation.
AID1223437Ratio of Vmax to Km in non-diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID776766Antiproliferative activity against human PBMC assessed as incorporation of [3H]thymidine after 18 hrs by scintillation counting analysis2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID1503775Antichlamydial activity against Chlamydia pneumoniae K7 infected in HL cells assessed as chlamydial inhibition at 50 uM after 70 hrs by fluorescent microscopic analysis2017Journal of natural products, 10-27, Volume: 80, Issue:10
Identification of Privileged Antichlamydial Natural Products by a Ligand-Based Strategy.
AID1402049Antibacterial activity against methicillin-resistant Staphylococcus aureus ATCC 43300 after 18 to 20 hrs by spectrophotometry based microdilution method2018European journal of medicinal chemistry, Jan-01, Volume: 143Synthesis and antimicrobial activity of amino acid and peptide derivatives of mycophenolic acid.
AID303351Inhibition of human IMPDH type 12007Journal of medicinal chemistry, Nov-15, Volume: 50, Issue:23
Probing binding requirements of type I and type II isoforms of inosine monophosphate dehydrogenase with adenine-modified nicotinamide adenine dinucleotide analogues.
AID774863Cytotoxicity against human Huh7.5 cells after 96 hrs by MTT assay2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID1223435Drug metabolism in non-diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID92745In vitro inhibition of Inosine-5'-monophosphate dehydrogenase 2.2002Bioorganic & medicinal chemistry letters, Oct-21, Volume: 12, Issue:20
Novel guanidine-based inhibitors of inosine monophosphate dehydrogenase.
AID1223458Drug metabolism in diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1763991Cytotoxicity against human HT-29 cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID776767Selectivity index, ratio of IC50 to EC50 for human Jurkat cells2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID264230Cell cycle arrest at S phase in HUVEC at 1 uM in presence of 50 uM guanosine2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID436485Cytotoxicity against hamster BHK21 cells assessed as reduction in cell viability after 48 to 96 hrs by MTT assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID1380552Inhibition of Mycobacterium tuberculosis GuaB2 at 50 uM2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID642283Antiviral activity against Vaccinia virus Lister Elstree infected in african green monkey Vero 76 cells assessed as reduction of virus-induced plaque formation after 3 days using crystal violet staining2011Bioorganic & medicinal chemistry, Dec-01, Volume: 19, Issue:23
Quinoline tricyclic derivatives. Design, synthesis and evaluation of the antiviral activity of three new classes of RNA-dependent RNA polymerase inhibitors.
AID625279Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for bilirubinemia2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID436481Cytotoxicity against african green monkey Vero 76 cells by crystal violet staining2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID776768Selectivity index, ratio of IC50 to EC50 for human PBMC2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID278351Inhibition of viral RNA levels in HTNV76-118-infected Vero E6 cells at 10 ug/ml2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID625291Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for liver function tests abnormal2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1402062Antibacterial activity against methicillin-sensitive Staphylococcus aureus ATCC 25923 assessed as inhibition of bacterial growth at 187.5 uM after 18 to 20 hrs by spectrophotometry based microdilution method relative to untreated control2018European journal of medicinal chemistry, Jan-01, Volume: 143Synthesis and antimicrobial activity of amino acid and peptide derivatives of mycophenolic acid.
AID625284Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatic failure2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1709981Toxicity in mouse MIN6 cells assessed by dehydrogenase activity measured after 48 hrs by CCK-8 assay2021ACS medicinal chemistry letters, May-13, Volume: 12, Issue:5
Design and Catalyzed Activation of Mycophenolic Acid Prodrugs.
AID1364945Antiviral activity against Chikungunya virus isolate DRDE-06 infected in African green monkey Vero cells by neutral red dye uptake assay2017Bioorganic & medicinal chemistry, 08-15, Volume: 25, Issue:16
The medicinal chemistry of Chikungunya virus.
AID538362Induction of human K562 cells differentiation assessed as hemoglobin/genomic DNA level at 2 uM after 72 hrs relative to untreated control2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID530151Induction of oxidative burst of polymorphonuclear neutrophil co-cultured with Aspergillus fumigatus germlings at 10 uM after 60 minutes relative to untreated control2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID436277Antimicrobial activity against Candida albicans ATCC 90028 by by standard disk assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID1422704Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.156 uM assessed as Km for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentratio2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID267725Inhibition of human IMPDH12006Bioorganic & medicinal chemistry letters, Jul-01, Volume: 16, Issue:13
Phosphonic acid-containing analogues of mycophenolic acid as inhibitors of IMPDH.
AID1485236Antiviral activity against Punta Toro virus infected in African green monkey Vero cells assessed as inhibition of viral-induced cytopathic effect measured after 3 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID1211227Drug metabolism in human liver microsomes assessed as UGT1A9-mediated unbound intrinsic glucuronidation clearance at 1 uM after 30 to 60 mins by LC-MS/MS analysis2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID977599Inhibition of sodium fluorescein uptake in OATP1B1-transfected CHO cells at an equimolar substrate-inhibitor concentration of 10 uM2013Molecular pharmacology, Jun, Volume: 83, Issue:6
Structure-based identification of OATP1B1/3 inhibitors.
AID1220796Drug metabolism in bile duct-cannulated rat assessed as glucuronide concentration in bile and urine at 0.2 mg/kg, iv up to 24 hrs by LC/MS/MS analysis2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID538358Induction of human K562 cells differentiation assessed as hemoglobin/genomic DNA level at 0.125 uM after 72 hrs relative to untreated control2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID767479Antiproliferative activity against human K562 cells at 1 uM in presence of guanosine2013Bioorganic & medicinal chemistry letters, Sep-15, Volume: 23, Issue:18
Discovery of N-(2,3,5-triazoyl)mycophenolic amide and mycophenolic epoxyketone as novel inhibitors of human IMPDH.
AID1220800Drug metabolism in bile duct-cannulated monkey assessed as glucuronide concentration in bile and urine at 0.2 mg/kg, iv up to 24 hrs by LC/MS/MS analysis2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID278359Effect on metabolism of 10 ug/ml RBV in Vero E6 cells assessed as RBV-MP concentration at 1 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID436274Antimicrobial activity against methicillin-resistant Staphylococcus aureus ATCC 43300 by standard disk assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID92616Compound was tested for inhibition of human recombinant type II Inosine Monophosphate Dehydrogenase at pH 7.41996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID300654Stimulation of mouse 3T3-L1 cells differentiation at 2 to 4 uM in presence of guanosine2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID1774079Stabilization of TTR V3OM mutant (unknown origin) assessed as acid-mediated protein aggregation inhibition ratio at 10 uM incubated for 1 week by absorbance method2021Journal of medicinal chemistry, 10-14, Volume: 64, Issue:19
Repositioning of the Anthelmintic Drugs Bithionol and Triclabendazole as Transthyretin Amyloidogenesis Inhibitors.
AID1261341Antiviral activity against VSV2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1211260Ratio of UGT1A9-mediated unbound intrinsic glucuronidation clearance in human kidney microsomes to UGT1A9-mediated unbound intrinsic glucuronidation clearance in human liver microsomes at 1 uM in presence of 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1261340Antiviral activity against HSV12015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID342522Inhibition of human IMP dehydrogenase 12008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
Bis(sulfonamide) isosters of mycophenolic adenine dinucleotide analogues: inhibition of inosine monophosphate dehydrogenase.
AID668553Antiproliferative activity against mouse L1210 cells after 48 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and biological activity of novel mycophenolic acid conjugates containing nitro-acridine/acridone derivatives.
AID264227Cell cycle arrest at S phase in HUVEC at 1 uM2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID1261316Antiviral activity against human RSV infected in african green monkey Vero76 cells assessed as inhibition of virus-induced cytopathogenicity after 5 days by plaque reduction assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID300648Inhibition of mouse 3T3-L1 cell differentiation assessed as minimal effective concentration at 2 uM by oil-red O staining method2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID676607Growth restoration activity in cdc2-1 rad9-deficient Saccharomyces cerevisiae assessed as growth zone at 0.05 ug/disc incubated at 37 degC for 6 hrs and 28 degC for 2 days2012Bioorganic & medicinal chemistry, Jun-15, Volume: 20, Issue:12
Cleavage mechanism and anti-tumor activity of 3,6-epidioxy-1,10-bisaboladiene isolated from edible wild plants.
AID46583Inhibitory activity against proliferation of CEM cell line2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Quinolone-based IMPDH inhibitors: introduction of basic residues on ring D and SAR of the corresponding mono, di and benzofused analogues.
AID1303381Antiviral activity against Vaccinia virus Elstree infected in Vero 76 cells after 3 days by crystal violet staining based plaque reduction assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID1422689Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.039 uM assessed as Vmax for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID92742Inhibitory activity tested against inosine-5'-monophosphate dehydrogenase 2 (IMPDH-II) enzyme2003Bioorganic & medicinal chemistry letters, Oct-20, Volume: 13, Issue:20
3-cyanoindole-based inhibitors of inosine monophosphate dehydrogenase: synthesis and initial structure-activity relationships.
AID1557349Inhibition of Mycobacterium tuberculosis IMPDH22019MedChemComm, Aug-01, Volume: 10, Issue:8
Inhibitors of inosine 5'-monophosphate dehydrogenase as emerging new generation antimicrobial agents.
AID436275Antimicrobial activity against Mycobacterium intracellular ATCC 27853 by by alamar blue assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID300650Inhibition of [1-14C]sodium acetate uptake at PPARgamma in mouse 3T3-L1 cells at 2 uM2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID1709980Inhibition of IMPDH2 (unknown origin) assessed by conversion of NAD+ to NADH preincubated for 30 mins followed by addition of inosine-5'- monophosphate substrate and NAD at 10 uM measured by plate reader method2021ACS medicinal chemistry letters, May-13, Volume: 12, Issue:5
Design and Catalyzed Activation of Mycophenolic Acid Prodrugs.
AID624630Drug glucuronidation reaction catalyzed by human recombinant UGT1A12005Pharmacology & therapeutics, Apr, Volume: 106, Issue:1
UDP-glucuronosyltransferases and clinical drug-drug interactions.
AID685501HARVARD: Inhibition of liver stage Plasmodium berghei infection in HepG2 cells2012Proceedings of the National Academy of Sciences of the United States of America, May-29, Volume: 109, Issue:22
Liver-stage malaria parasites vulnerable to diverse chemical scaffolds.
AID1226487Antiviral activity against Dengue virus type 2 New Guinea C infected in African green monkey Vero cells assessed as reduction in viral titer at 1 uM after 2 days by Giemsa staining assay relative to control2015Journal of medicinal chemistry, May-14, Volume: 58, Issue:9
5'-Silylated 3'-1,2,3-triazolyl Thymidine Analogues as Inhibitors of West Nile Virus and Dengue Virus.
AID95608Antiproliferative activity against K562 cells2002Journal of medicinal chemistry, Jan-31, Volume: 45, Issue:3
Novel mycophenolic adenine bis(phosphonate) analogues as potential differentiation agents against human leukemia.
AID530143Inhibition of Aspergillus fumigatus-mediated dendritic cell maturation assessed as decreased CD86 expression at 10 uM2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID92740Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Quinolone-based IMPDH inhibitors: introduction of basic residues on ring D and SAR of the corresponding mono, di and benzofused analogues.
AID538360Induction of human K562 cells differentiation assessed as hemoglobin/genomic DNA level at 0.5 uM after 72 hrs relative to untreated control2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID472784Cytotoxicity against MDBK cells after 48 to 96 hrs by MTT assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID92607Inhibitory activity against human Inosine-5'-monophosphate dehydrogenase 1 (IMPDH type I isoform); Range is 33-37 nM1998Journal of medicinal chemistry, Feb-12, Volume: 41, Issue:4
Synthesis of a methylenebis(phosphonate) analogue of mycophenolic adenine dinucleotide: a glucuronidation-resistant MAD analogue of NAD.
AID1261308Cytotoxicity against human MT4 cells assessed as cell viability after 96 hrs by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1223424Drug metabolism in non-diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID663911Cytotoxicity against african green monkey Vero 76 cells after 48 to 96 hrs by MTT assay2012European journal of medicinal chemistry, Jul, Volume: 535-acetyl-2-arylbenzimidazoles as antiviral agents. Part 4.
AID588216FDA HLAED, serum glutamic oxaloacetic transaminase (SGOT) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID472793Antiviral activity against HSV1 KOS infected in african green monkey Vero cells after 3 days by plaque reduction assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID1261328Antiviral activity against WNV infected in BHK-21 cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID360765Antiviral activity against Dengue virus 2 NGC infected in african green monkey Vero cells after 3 days by immunofluorescence assay2007Proceedings of the National Academy of Sciences of the United States of America, Feb-27, Volume: 104, Issue:9
c-Src protein kinase inhibitors block assembly and maturation of dengue virus.
AID538355Selectivity ratio of IC50 for human IMPDH1 to IC50 for human IMPDH22010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID1223440Drug metabolism in diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID267726Inhibition of human IMPDH22006Bioorganic & medicinal chemistry letters, Jul-01, Volume: 16, Issue:13
Phosphonic acid-containing analogues of mycophenolic acid as inhibitors of IMPDH.
AID1763994Cytotoxicity against human SH-SY5Y cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID264225Antiproliferative activity against Jurkat T cells in presence of 20 uM guanine as measured by [3H]thymidine incorporation2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID1336612Antiviral activity against Punta Toro virus infected in African green monkey Vero cells assessed as protection against virus-induced cytopathic effect measured after 3 to 5 days by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID261589Inhibition of West Nile virus VLP replicon in BHK21 cell line2006Journal of medicinal chemistry, Mar-23, Volume: 49, Issue:6
Identification of compounds with anti-West Nile Virus activity.
AID668552Antiproliferative activity against human HL60 cells after 48 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and biological activity of novel mycophenolic acid conjugates containing nitro-acridine/acridone derivatives.
AID1303379Antiviral activity against Respiratory syncytial virus A2 infected in Vero 76 cells after 5 days by crystal violet staining based plaque reduction assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID1211222Ratio of unbound intrinsic glucuronidation clearance in human intestinal microsomes in presence of 1% bovine serum albumin to unbound intrinsic glucuronidation clearance in human intestinal microsomes in absence of bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID472789Antiviral activity against Bovine viral diarrhea virus NADL infected in MDBK cells assessed as protection from virus-induced cytopathogenicity after 3 to 4 days by MTT method2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID1211243Fraction unbound in human liver microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID91402Inhibition of Inosine Monophosphate Dehydrogenase II (IMPDH II)2002Bioorganic & medicinal chemistry letters, Nov-04, Volume: 12, Issue:21
A survey of cyclic replacements for the central diamide moiety of inhibitors of inosine monophosphate dehydrogenase.
AID1422697Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.078 uM assessed as Vmax for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID267727Selectivity for IMPDH1 over IMPDH22006Bioorganic & medicinal chemistry letters, Jul-01, Volume: 16, Issue:13
Phosphonic acid-containing analogues of mycophenolic acid as inhibitors of IMPDH.
AID625287Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatomegaly2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID642215Cytotoxicity against african green monkey Vero 76 cells after 48 to 96 hrs by crystal violet staining2011Bioorganic & medicinal chemistry, Dec-01, Volume: 19, Issue:23
Quinoline tricyclic derivatives. Design, synthesis and evaluation of the antiviral activity of three new classes of RNA-dependent RNA polymerase inhibitors.
AID1380557Antimicrobial activity against Mycobacterium tuberculosis H37Rv in presence of 0.08 to 5 ng/ml anhydrotetracycline-induced guaB2 gene silencing by alamar blue assay2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1211239Fraction unbound in human kidney microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1211182Ratio of UGT1A9-mediated unbound intrinsic glucuronidation clearance in human kidney microsomes to UGT1A9-mediated unbound intrinsic glucuronidation clearance in human liver microsomes at 1 uM2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1689895Immunosuppression-mediated cytotoxicity against anti-CD3/anti-CD28-stimulated human PBMC cells assessed as reduction in cell viability incubated for 72 hrs by MTT assay2020European journal of medicinal chemistry, Mar-01, Volume: 189Immunosuppressive properties of amino acid and peptide derivatives of mycophenolic acid.
AID83755Inhibitory activity of compound against HT-29 human colon adenocarcinoma cell line1996Journal of medicinal chemistry, Jan-05, Volume: 39, Issue:1
Synthesis and modeling studies with monocyclic analogues of mycophenolic acid.
AID1380550Antimicrobial activity against Mycobacterium tuberculosis H37Rv by alamar blue assay2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID210304Inhibitory concentration against human T-lymphoblast CEM cell line (ATCC) by CEM proliferation assay.2002Bioorganic & medicinal chemistry letters, Oct-21, Volume: 12, Issue:20
Novel guanidine-based inhibitors of inosine monophosphate dehydrogenase.
AID1210129Activity of human recombinant UGT1A9 expressed in insect cells assessed as enzyme mediated glucuronidation by LC-MS/MS method2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Glucuronidation of edaravone by human liver and kidney microsomes: biphasic kinetics and identification of UGT1A9 as the major UDP-glucuronosyltransferase isoform.
AID436272Antimicrobial activity against Escherichia coli ATCC 35218 by standard disk assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID1220793Ratio of drug level in blood to plasma in human2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1073348Inhibition of Chikungunya virus IMPDH2014Journal of medicinal chemistry, Feb-27, Volume: 57, Issue:4
Chikungunya virus: emerging targets and new opportunities for medicinal chemistry.
AID1211282Fraction metabolized glucuronidation in human liver microsomes in presence of UDP-glucuronosyltransferase2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID264233Inhibition of tumor-induced angiogenesis in RENCA mouse model2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID1181042Cytotoxicity against human PBMC after 96 hrs by flow cytometry2014Journal of natural products, Jul-25, Volume: 77, Issue:7
Nitrogen-containing dihydro-β-agarofuran derivatives from Tripterygium wilfordii.
AID1234127Drug metabolism in rat liver microsomes assessed as glucuronide formation at 600 uM incubated for 30 mins in presence of UDPGA by LC-MS/MS method2015Journal of medicinal chemistry, Jul-23, Volume: 58, Issue:14
Synthesis and Pharmacokinetic Evaluation of Siderophore Biosynthesis Inhibitors for Mycobacterium tuberculosis.
AID264229Cell cycle arrest at G1 phase in HUVEC at 1 uM in presence of 50 uM guanosine2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID1221821Cytotoxicity against HEK293 cells expressing UGT1A3 assessed as decrease in cell viability at 1 mM measured at 24 hrs by MTT assay2011Drug metabolism and disposition: the biological fate of chemicals, Jan, Volume: 39, Issue:1
Toxicological evaluation of acyl glucuronides of nonsteroidal anti-inflammatory drugs using human embryonic kidney 293 cells stably expressing human UDP-glucuronosyltransferase and human hepatocytes.
AID1186335Antiviral activity against Coxsackievirus B5 infected in african green monkey Vero 76 cells assessed as reduction in plaque number after 3 days by plaque reduction assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID537733Binding affinity to Candida albicans CaCdr1p expressed in yeast AD1-8u2010European journal of medicinal chemistry, Nov, Volume: 45, Issue:11
Analysis of physico-chemical properties of substrates of ABC and MFS multidrug transporters of pathogenic Candida albicans.
AID625285Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatic necrosis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1717752cytotoxicity against golden hamster BHK-21 cells incubated for 72 hrs by MTT assay2020Journal of medicinal chemistry, 11-25, Volume: 63, Issue:22
Chinese Therapeutic Strategy for Fighting COVID-19 and Potential Small-Molecule Inhibitors against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2).
AID668550Antiproliferative activity against human MOLT4 cells after 48 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and biological activity of novel mycophenolic acid conjugates containing nitro-acridine/acridone derivatives.
AID624613Specific activity of expressed human recombinant UGT1A102000Annual review of pharmacology and toxicology, , Volume: 40Human UDP-glucuronosyltransferases: metabolism, expression, and disease.
AID664263Antiviral activity against Vaccinia virus Elstree-Lister ATCC VR-1549 infected in african green monkey Vero-76 cells assessed as reduction in viral plaque after 3 days by crystal violet staining2012European journal of medicinal chemistry, Jul, Volume: 535-acetyl-2-arylbenzimidazoles as antiviral agents. Part 4.
AID624636Drug glucuronidation reaction catalyzed by human recombinant UGT1A82005Pharmacology & therapeutics, Apr, Volume: 106, Issue:1
UDP-glucuronosyltransferases and clinical drug-drug interactions.
AID1223462Drug metabolism in diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID530148Induction of oxidative burst of polymorphonuclear neutrophil co-cultured with Aspergillus fumigatus germlings at 10 uM after 120 minutes relative to untreated control2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID89839Inhibition of human Lymphocyte proliferation. The IC50 values were determined by interpolation using cubic spline determination.1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID292039Inhibition of CEM cell proliferation2007Journal of medicinal chemistry, Jul-26, Volume: 50, Issue:15
Acridone-based inhibitors of inosine 5'-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).
AID1380558Inhibition of human IMPDH2 at 10 uM2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID436273Antimicrobial activity against Pseudomonas aeruginosa ATCC 27853 by standard disk assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID134138In vivo for antibody response to sheep red blood cells(SRBC) by plaque-forming cells in the spleen (PFC/spleen) after 25 mg/kg oral administration in mice.1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID1186327Cytotoxicity against mock-infected human MT4 cells after 96 hrs by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID1408893Cytotoxicity against mouse NIH/3T3 cells assessed as cell viability at 10 uM after 48 hrs by MTT assay relative to control2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID1422679Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.078 uM assessed as Vmax for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1422693Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.039 uM assessed as Km for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentratio2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID292041Inhibition of T cell proliferation in PBMC cells2007Journal of medicinal chemistry, Jul-26, Volume: 50, Issue:15
Acridone-based inhibitors of inosine 5'-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).
AID472786Cytotoxicity against african green monkey Vero cells2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID278374Effect on metabolism of 40 ug/ml RBV in Vero E6 cells assessed as RBV-TP concentration at 2 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID278361Effect on metabolism of 10 ug/ml RBV in Vero E6 cells assessed as RBV-MP concentration at 5 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1223444Drug metabolism in diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1675163Antiviral activity against Zika virus2020Journal of natural products, 08-28, Volume: 83, Issue:8
Diverse Types of Diterpenoids with an Aromatized C Ring from the Twigs of
AID1303377Antiviral activity against CVB-2 infected in Vero 76 cells after 3 days by crystal violet staining based plaque reduction assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID705460Antiapicomplexan activity against Toxoplasma gondii THdhxgTRP tachyzoites harboring insertional mutation assessed as growth inhibition after 6 weeks2012Journal of medicinal chemistry, Oct-11, Volume: 55, Issue:19
Salicylanilide inhibitors of Toxoplasma gondii.
AID278360Effect on metabolism of 10 ug/ml RBV in Vero E6 cells assessed as RBV-MP concentration at 2 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1223463Ratio of Vmax to Km in diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID472785Cytotoxicity against BHK cells after 48 to 96 hrs by MTT assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID588211Literature-mined compound from Fourches et al multi-species drug-induced liver injury (DILI) dataset, effect in humans2010Chemical research in toxicology, Jan, Volume: 23, Issue:1
Cheminformatics analysis of assertions mined from literature that describe drug-induced liver injury in different species.
AID438029Inhibition of Dengue virus IMPDH2009Journal of medicinal chemistry, Dec-24, Volume: 52, Issue:24
The medicinal chemistry of dengue fever.
AID1211196Ratio of UGT1A9-mediated unbound intrinsic glucuronidation clearance in human kidney microsomes to UGT1A9-mediated unbound intrinsic glucuronidation clearance in human intestinal microsomes at 1 uM2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID537734Antifungal activity against yeast AD1-8u expressing Candida albicans CaMdr1p by agar disk diffusion assay2010European journal of medicinal chemistry, Nov, Volume: 45, Issue:11
Analysis of physico-chemical properties of substrates of ABC and MFS multidrug transporters of pathogenic Candida albicans.
AID1402056Antibacterial activity against Pseudomonas aeruginosa ATCC 27853 assessed as inhibition of bacterial growth at 1500 uM after 18 to 20 hrs by spectrophotometry based microdilution method relative to untreated control2018European journal of medicinal chemistry, Jan-01, Volume: 143Synthesis and antimicrobial activity of amino acid and peptide derivatives of mycophenolic acid.
AID1408888Inhibition of human IMPDH2 using IMP at 10 uM as substrate in presence of NAD+ after 30 mins relative to control2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID530142Induction of TNF-alpha expression in mouse dendritic cell co-cultured with Aspergillus fumigatus germ tube at 10 uM after 7 days by RT-PCR2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID1211210Unbound intrinsic glucuronidation clearance in human kidney microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of UDP-glucuronosyltransferase and 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID774869Antiviral activity against Coxsackie B6 virus Schmitt in african green monkey Vero cells assessed as inhibition virus-induced cytopathic effect after 48 hrs2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID1709982Toxicity in human hepatocytes assessed by dehydrogenase activity measured after 48 hrs by CCK-8 assay2021ACS medicinal chemistry letters, May-13, Volume: 12, Issue:5
Design and Catalyzed Activation of Mycophenolic Acid Prodrugs.
AID1211267Ratio of UGT1A9-mediated unbound intrinsic glucuronidation clearance in human intestinal microsomes to UGT1A9-mediated unbound intrinsic glucuronidation clearance in human liver microsomes at 1 uM in presence of 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID625282Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for cirrhosis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID472794Antiviral activity against RSV A2 infected in african green monkey Vero cells after 5 days by plaque reduction assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID1380562Growth inhibition of Pseudomonas aeruginosa at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1422700Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.156 uM assessed as Vmax for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID472790Antiviral activity against Yellow fever virus 17D infected in BHK cells assessed as protection from virus-induced cytopathogenicity after 3 to 4 days by MTT method2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID1364947Selectivity index, ratio of CC50 for African green monkey Vero cells to IC50 for Chikungunya virus DRDE-062017Bioorganic & medicinal chemistry, 08-15, Volume: 25, Issue:16
The medicinal chemistry of Chikungunya virus.
AID436477Cytotoxicity against MDBK cells after 48 to 96 hrs by MTT assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID348820Antiproliferative activity against human K562 cells after 72 hrs by MTS assay2008Bioorganic & medicinal chemistry, Oct-15, Volume: 16, Issue:20
Mycophenolic acid analogs with a modified metabolic profile.
AID115875Tested for the inhibition of murine PFC response at 50 mg/kg dose1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID92608Inhibitory activity against human Inosine-5'-monophosphate dehydrogenase 1 (IMPDH type I)2002Journal of medicinal chemistry, Jan-31, Volume: 45, Issue:3
Novel mycophenolic adenine bis(phosphonate) analogues as potential differentiation agents against human leukemia.
AID1774075Inhibition of 8-anilinonaphthalene-l-sulfonic acid binding to TTR V3OM mutant (unknown origin) expressed in Escherichia coli assessed as ANS saturation ratio at 400 uM incubated for 1 hr in presence of 7.5 uM ANS by fluorescence method (Rvb = 56 +/- 2.3%)2021Journal of medicinal chemistry, 10-14, Volume: 64, Issue:19
Repositioning of the Anthelmintic Drugs Bithionol and Triclabendazole as Transthyretin Amyloidogenesis Inhibitors.
AID1220792Ratio of drug level in blood to plasma in dog2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1211175Drug metabolism in human intestinal microsomes assessed as UGT1A9-mediated unbound intrinsic glucuronidation clearance at 1 uM after 30 to 60 mins by LC-MS/MS analysis2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID115876Tested for the inhibition of murine PFC response at 6.25 mg/kg dose1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID774864Antiviral activity against HCV expressing pFL-J6/JFH/JC1 infected in human Huh7.5 cells assessed as inhibition of viral RNA synthesis after 96 hrs by one-step RT-PCR analysis2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID92748Inhibitory activity against human Inosine-5'-monophosphate dehydrogenase 2 (IMPDH type II isoform); Range is 6-10 nM1998Journal of medicinal chemistry, Feb-12, Volume: 41, Issue:4
Synthesis of a methylenebis(phosphonate) analogue of mycophenolic adenine dinucleotide: a glucuronidation-resistant MAD analogue of NAD.
AID537736Antifungal activity against yeast AD1-8u expressing Candida albicans CaCdr1p by agar disk diffusion assay2010European journal of medicinal chemistry, Nov, Volume: 45, Issue:11
Analysis of physico-chemical properties of substrates of ABC and MFS multidrug transporters of pathogenic Candida albicans.
AID1336610Antiviral activity against Sindbis virus infected in African green monkey Vero cells assessed as protection against virus-induced cytopathic effect measured after 3 to 5 days by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID1689901Selectivity index, ratio of IC50 for cytotoxicity against human Jurkat T cells by MTT assay to EC50 for antiproliferative activity against human Jurkat T cells by VPD450 staining based flow cytometry2020European journal of medicinal chemistry, Mar-01, Volume: 189Immunosuppressive properties of amino acid and peptide derivatives of mycophenolic acid.
AID1211213Unbound intrinsic glucuronidation clearance in human intestinal microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of UDP-glucuronosyltransferase2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1485243Antiviral activity against para influenza 3 virus infected in African green monkey Vero cells assessed as inhibition of viral-induced cytopathic effect measured after 3 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID1186330Antiviral activity against Bovine viral diarrhea virus infected in MDBK cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID1498135Cytotoxicity against African green monkey Vero cells assessed as alteration of cell morphology by microscopic analysis2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID1303380Antiviral activity against Vesicular stomatitis virus Indiana infected in Vero 76 cells after 2 days by crystal violet staining based plaque reduction assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID472792Antiviral activity against Polio virus type1 Sabin strain Chat infected in african green monkey Vero cells after 2 days by plaque reduction assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID278363Effect on metabolism of 40 ug/ml RBV in Vero E6 cells assessed as RBV-MP concentration at 1 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1246236Cytotoxicity against African green monkey Vero 76 cells assessed as cell viability after 48 to 96 hrs by crystal violet staining2015European journal of medicinal chemistry, Aug-28, Volume: 101Synthesis, cytotoxicity and antimicrobial activity of thiourea derivatives incorporating 3-(trifluoromethyl)phenyl moiety.
AID1261315Antiviral activity against human CVB5 infected in african green monkey Vero76 cells assessed as inhibition of virus-induced cytopathogenicity after 3 days by plaque reduction assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1220791Ratio of drug level in blood to plasma in monkey2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1223428Drug metabolism in non-diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1220787Fraction unbound in dog plasma by ultracentrifugation method2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID1211241Fraction unbound in human intestinal microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID492854Inhibition of N-terminal His-tagged Mycobacterium tuberculosis H37Rv IMPDH2010Journal of medicinal chemistry, Jun-24, Volume: 53, Issue:12
Triazole-linked inhibitors of inosine monophosphate dehydrogenase from human and Mycobacterium tuberculosis.
AID1717771Selectivity ratio of CC50 for Rhesus macaque LLC-MK2 cells to EC50 for HCoV-NL63 infected in Rhesus macaque LLC-MK2 cells2020Journal of medicinal chemistry, 11-25, Volume: 63, Issue:22
Chinese Therapeutic Strategy for Fighting COVID-19 and Potential Small-Molecule Inhibitors against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2).
AID1211278Clearance in iv dosed human2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1763989Cytotoxicity against human KKUM213 cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID134137In vivo antibody response to sheep red blood cells(SRBC) by plaques per million after 25 mg/kg oral administration in mice.1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID436278Antimicrobial activity against Cryptococcus neoformans ATCC 90113 by standard disk assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID264231Cell cycle arrest at G2/M phase in HUVEC at 1 uM in presence of 50 uM guanosine2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID409957Inhibition of bovine liver MAOA2008Journal of medicinal chemistry, Nov-13, Volume: 51, Issue:21
Quantitative structure-activity relationship and complex network approach to monoamine oxidase A and B inhibitors.
AID767486Antiproliferative activity against human K562 cells at 1 uM2013Bioorganic & medicinal chemistry letters, Sep-15, Volume: 23, Issue:18
Discovery of N-(2,3,5-triazoyl)mycophenolic amide and mycophenolic epoxyketone as novel inhibitors of human IMPDH.
AID1498134Antiviral activity against Yellow fever virus infected in African green monkey Vero cells assessed as inhibition of virus-induced cytopathicity2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID699265Cytotoxicity against BHK cells incubated for 48 hrs by MTT assay2012Journal of medicinal chemistry, Jul-26, Volume: 55, Issue:14
Discovery of novel small molecule inhibitors of dengue viral NS2B-NS3 protease using virtual screening and scaffold hopping.
AID1763993Cytotoxicity against human A549 cells assessed as growth inhibition measured by SRB assay2021Bioorganic & medicinal chemistry letters, 08-01, Volume: 45Synthesis of propargylamine mycophenolate analogues and their selective cytotoxic activity towards neuroblastoma SH-SY5Y cell line.
AID436478Antiviral activity against Bovine viral diarrhea virus infected in MDBK cells assessed as inhibition of virus-induced cytopathicity after 3 to 4 days by MTT assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID538359Induction of human K562 cells differentiation assessed as hemoglobin/genomic DNA level at 0.25 uM after 72 hrs relative to untreated control2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID1474167Liver toxicity in human assessed as induction of drug-induced liver injury by measuring verified drug-induced liver injury concern status2016Drug discovery today, Apr, Volume: 21, Issue:4
DILIrank: the largest reference drug list ranked by the risk for developing drug-induced liver injury in humans.
AID1303378Antiviral activity against Human poliovirus 1 strain Sabin infected in Vero 76 cells after 2 days by crystal violet staining based plaque reduction assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID668554Antiproliferative activity against activated-human PBMC after 96 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and biological activity of novel mycophenolic acid conjugates containing nitro-acridine/acridone derivatives.
AID767480Inhibition of HDAC in human K562 cells using Boc-Lys[Ac]-AMC as substrate at 10 uM incubated for 10 mins prior to substrate addition measured after 80 mins by fluorescence assay2013Bioorganic & medicinal chemistry letters, Sep-15, Volume: 23, Issue:18
Discovery of N-(2,3,5-triazoyl)mycophenolic amide and mycophenolic epoxyketone as novel inhibitors of human IMPDH.
AID436482Antiviral activity against Coxsackie virus type B2 virus infected in african green monkey Vero-76 cells after 3 days by plaque reduction assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID625288Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for jaundice2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID92743Inhibitory concentration against Inosine-5'-monophosphate dehydrogenase 2 was determined2002Bioorganic & medicinal chemistry letters, Nov-18, Volume: 12, Issue:22
The TosMIC approach to 3-(oxazol-5-yl) indoles: application to the synthesis of indole-based IMPDH inhibitors.
AID90469In vitro inhibition of human lymphocyte proliferation in response to phytohemagglutininin (PHA) was determined1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID668549Antiproliferative activity against human Jurkat cells after 48 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and biological activity of novel mycophenolic acid conjugates containing nitro-acridine/acridone derivatives.
AID311524Oral bioavailability in human2007Bioorganic & medicinal chemistry, Dec-15, Volume: 15, Issue:24
Hologram QSAR model for the prediction of human oral bioavailability.
AID278373Effect on metabolism of 40 ug/ml RBV in Vero E6 cells assessed as RBV-TP concentration at 1 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1422692Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.039 uM assessed as Vmax for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID264222Antiproliferative activity against HUVEC as measured by [3H]thymidine incorporation2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID530149Inhibition of Aspergillus fumigatus-mediated dendritic cell maturation assessed as decreased CD80 expression at 10 uM2008Antimicrobial agents and chemotherapy, Jul, Volume: 52, Issue:7
Impact of mycophenolic acid on the functionality of human polymorphonuclear neutrophils and dendritic cells during interaction with Aspergillus fumigatus.
AID538361Induction of human K562 cells differentiation assessed as hemoglobin/genomic DNA level at 1 uM after 72 hrs relative to untreated control2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID46578Inhibitory activity against CEM cell proliferation2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Novel inhibitors of IMPDH: a highly potent and selective quinolone-based series.
AID300651Antagonist activity at pioglitazone-stimulated PPARgamma transcriptional activity in U2OS cells at 1 uM2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID92624Inhibition of human inosine monophosphate dehydrogenase IMPDH II2003Bioorganic & medicinal chemistry letters, Feb-10, Volume: 13, Issue:3
Novel inhibitors of IMPDH: a highly potent and selective quinolone-based series.
AID1408892Cytotoxicity against human U87MG cells assessed as reduction in cell viability after 48 hrs by MTT assay2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID1223423Drug metabolism in non-diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1774076Inhibition of 8-anilinonaphthalene-l-sulfonic acid binding to TTR V3OM mutant (unknown origin) expressed in Escherichia coli at 400 uM incubated for 1 hr in presence of 75 uM ANS by fluorescence method (Rvb = 91 +/- 0.92%)2021Journal of medicinal chemistry, 10-14, Volume: 64, Issue:19
Repositioning of the Anthelmintic Drugs Bithionol and Triclabendazole as Transthyretin Amyloidogenesis Inhibitors.
AID588212Literature-mined compound from Fourches et al multi-species drug-induced liver injury (DILI) dataset, effect in rodents2010Chemical research in toxicology, Jan, Volume: 23, Issue:1
Cheminformatics analysis of assertions mined from literature that describe drug-induced liver injury in different species.
AID436484Antiviral activity against HSV1 infected in Vero-76 cells after 3 days by plaque reduction assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID1223442Uncompetitive substrate inhibition of UGT2B7 in diabetic human liver microsomes assessed as reduction in enzyme-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID436276Antimicrobial activity against Aspergillus fumigatus ATCC 27853 by by standard disk assay2008Journal of natural products, Nov, Volume: 71, Issue:11
Mycophenolic derivatives from Eupenicillium parvum.
AID625281Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for cholelithiasis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1211189Ratio of UGT1A9-mediated unbound intrinsic glucuronidation clearance in human intestinal microsomes to UGT1A9-mediated unbound intrinsic glucuronidation clearance in human liver microsomes at 1 uM2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID348819Inhibition of human IMPDH22008Bioorganic & medicinal chemistry, Oct-15, Volume: 16, Issue:20
Mycophenolic acid analogs with a modified metabolic profile.
AID1223455Ratio of Vmax to Km in diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1211215Unbound intrinsic glucuronidation clearance in human intestinal microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of UDP-glucuronosyltransferase and 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1717753Selectivity index, ratio of CC50 for golden hamster BHK-21 cells to EC50 for wild type HCoV-OC43 infected in BHK-21 cells2020Journal of medicinal chemistry, 11-25, Volume: 63, Issue:22
Chinese Therapeutic Strategy for Fighting COVID-19 and Potential Small-Molecule Inhibitors against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2).
AID304374Antiproliferative activity against human K562 cells after 72 hrs by MTS assay2007Journal of medicinal chemistry, Dec-27, Volume: 50, Issue:26
Dual inhibitors of inosine monophosphate dehydrogenase and histone deacetylases for cancer treatment.
AID1211245Unbound intrinsic glucuronidation clearance in human liver microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of UDP-glucuronosyltransferase2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1380561Growth inhibition of Klebsiella pneumoniae at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1474166Liver toxicity in human assessed as induction of drug-induced liver injury by measuring severity class index2016Drug discovery today, Apr, Volume: 21, Issue:4
DILIrank: the largest reference drug list ranked by the risk for developing drug-induced liver injury in humans.
AID625283Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for elevated liver function tests2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1186332Antiviral activity against Yellow fever virus infected in BHK21 cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID1211237Fraction unbound in human liver microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1220788Fraction unbound in human plasma by ultracentrifugation method2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID278353Decrease in GTP level in Vero E6 cells at 2.5 ug/ml2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID774866Cytotoxicity against african green monkey Vero cells after 96 hrs by MTT assay2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID1261313Antiviral activity against YFV infected in BHK-21 cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID300652Binding affinity to PPARgamma2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID580126Inhibition of human IMPDH2 by Spectrophotometer2011Bioorganic & medicinal chemistry, Mar-01, Volume: 19, Issue:5
Cofactor-type inhibitors of inosine monophosphate dehydrogenase via modular approach: targeting the pyrophosphate binding sub-domain.
AID472791Antiviral activity against Human coxsackievirus B2 Ohio-1 infected in african green monkey Vero cells after 3 days by plaque reduction assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID1223441Ratio of Vmax to Km in diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1336613Antiviral activity against Yellow fever virus infected in African green monkey Vero cells assessed as protection against virus-induced cytopathic effect measured after 3 to 5 days by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID1186342Selectivity index, ratio of CC50 for african green monkey Vero 76 cells to EC50 for Respiratory syncytial virus2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID538363Induction of human K562 cells differentiation assessed as hemoglobin/genomic DNA level at 4 uM after 72 hrs relative to untreated control2010Bioorganic & medicinal chemistry, Nov-15, Volume: 18, Issue:22
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase and differentiation induction of K562 cells among the mycophenolic acid derivatives.
AID776765Antiproliferative activity against human Jurkat cells assessed as incorporation of [3H]thymidine after 18 hrs by scintillation counting analysis2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID1223432Drug metabolism in non-diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1204074Cytotoxicity against African green monkey Vero 76 cells assessed as cell viability after 48 to 96 hrs by MTT method2015Bioorganic & medicinal chemistry letters, Jun-01, Volume: 25, Issue:11
N-((1,3-Diphenyl-1H-pyrazol-4-yl)methyl)anilines: A novel class of anti-RSV agents.
AID697853Inhibition of horse BChE at 2 mg/ml by Ellman's method2012Bioorganic & medicinal chemistry, Nov-15, Volume: 20, Issue:22
Exploration of natural compounds as sources of new bifunctional scaffolds targeting cholinesterases and beta amyloid aggregation: the case of chelerythrine.
AID300653Activation of PPARgamma transcriptional activity in U20S cells at 0.01 to 1 uM2007Bioorganic & medicinal chemistry letters, Sep-01, Volume: 17, Issue:17
Mycophenolic acid as a latent agonist of PPARgamma.
AID1246240Antiviral activity against Human Vaccinia virus ATCC VR-1549 infected in African green monkey Vero 76 cells assessed as reduction of virus-plaque formation after 3 days by crystal violet staining2015European journal of medicinal chemistry, Aug-28, Volume: 101Synthesis, cytotoxicity and antimicrobial activity of thiourea derivatives incorporating 3-(trifluoromethyl)phenyl moiety.
AID1223453Drug metabolism in non-diabetic human kidney microsomes assessed as UGT2B7*2 mutant-mediated intrinsic clearance of mycophenolic acid acyl glucuronide by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID92741Inhibition of human Inosine-5'-monophosphate dehydrogenase 22002Bioorganic & medicinal chemistry letters, Aug-19, Volume: 12, Issue:16
Rapid synthesis of triazine inhibitors of inosine monophosphate dehydrogenase.
AID774870Selectivity index, ratio of CC50 for african green monkey Vero cells to IC50 for Coxsackie B3 virus Schmitt2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and antiviral activity of a novel class of (5-oxazolyl)phenyl amines.
AID436480Antiviral activity against Reovirus-1 infected in BHK-21 cells assessed as inhibition of virus-induced cytopathicity after 3 to 4 days by MTT assay2009Bioorganic & medicinal chemistry, Jul-01, Volume: 17, Issue:13
Antiviral and cytotoxic activities of aminoarylazo compounds and aryltriazene derivatives.
AID767473Induction of human K562 cell differentiation at 1 uM after 72 hrs by trypan blue exclusion assay in presence of guanosine2013Bioorganic & medicinal chemistry letters, Sep-15, Volume: 23, Issue:18
Discovery of N-(2,3,5-triazoyl)mycophenolic amide and mycophenolic epoxyketone as novel inhibitors of human IMPDH.
AID1223422Drug metabolism in diabetic human kidney microsomes assessed as UGT2B7*1c mutant-mediated intrinsic clearance of mycophenolic acid acyl glucuronide by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1211251Unbound intrinsic glucuronidation clearance in human kidney microsomes at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of UDP-glucuronosyltransferase2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1220801Drug metabolism in bile duct-cannulated dog assessed as glucuronide concentration in bile and urine at 0.2 mg/kg, iv up to 24 hrs by LC/MS/MS analysis2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID776769Cytotoxicity against human Jurkat cells after 48 hrs by MTT assay2013European journal of medicinal chemistry, Nov, Volume: 69Synthesis and biological activity of mycophenolic acid-amino acid derivatives.
AID1303375Antiviral activity against Reovirus type-1 3651 infected in BHK21 cells assessed as inhibition of virus induced cytopathogenicity after 3 to 4 days by MTT assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID1422701Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.156 uM assessed as Km for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentratio2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1717770Cytotoxicity against Rhesus macaque LLC-MK2 cells incubated for 72 hrs by MTT assay2020Journal of medicinal chemistry, 11-25, Volume: 63, Issue:22
Chinese Therapeutic Strategy for Fighting COVID-19 and Potential Small-Molecule Inhibitors against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2).
AID1223456Uncompetitive substrate inhibition of UGT2B7 in diabetic human liver microsomes assessed as reduction in enzyme-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1336609Antiviral activity against Reovirus-1 infected in African green monkey Vero cells assessed as protection against virus-induced cytopathic effect measured after 3 to 5 days by microscopic analysis2017Bioorganic & medicinal chemistry letters, 01-15, Volume: 27, Issue:2
Synthesis and in vitro antiviral evaluation of 4-substituted 3,4-dihydropyrimidinones.
AID1211287Drug metabolism in human kidney microsomes assessed as UGT1A9-mediated unbound intrinsic glucuronidation clearance at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1211254Drug metabolism in human intestinal microsomes assessed as UGT1A8/2B7-mediated unbound intrinsic glucuronidation clearance at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of 2% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1220785Fraction unbound in rat plasma by ultracentrifugation method2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID261586Inhibition of Rluc-FL-WNV replicon in Vero cells2006Journal of medicinal chemistry, Mar-23, Volume: 49, Issue:6
Identification of compounds with anti-West Nile Virus activity.
AID685503HARVARD: Inhibition of blood stage Plasmodium falciparum Dd2 infection2012Proceedings of the National Academy of Sciences of the United States of America, May-29, Volume: 109, Issue:22
Liver-stage malaria parasites vulnerable to diverse chemical scaffolds.
AID472783Cytotoxicity against human MT4 cells after 96 hrs by MTT assay2010Bioorganic & medicinal chemistry, Apr-15, Volume: 18, Issue:8
Antiviral activity of benzimidazole derivatives. II. Antiviral activity of 2-phenylbenzimidazole derivatives.
AID637675Immunosuppressive activity in in mouse T-cells assessed as inhibition of concanavalin A-induced proliferation2012Bioorganic & medicinal chemistry letters, Jan-01, Volume: 22, Issue:1
The design, synthesis and in vitro immunosuppressive evaluation of novel isobenzofuran derivatives.
AID625286Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatitis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1380554Octanol/water partition coefficient logP of the compound2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1186333Antiviral activity against Reovirus type-1 infected in BHK21 cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID1303376Cytotoxicity against mock-infected african green monkey Vero 76 cells assessed as reduction in cell viability after 48 to 96 hrs by MTT assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID1408894Cytotoxicity against mouse NIH/3T3 cells assessed as cell viability at 100 uM after 48 hrs by MTT assay relative to control2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID624611Specific activity of expressed human recombinant UGT1A82000Annual review of pharmacology and toxicology, , Volume: 40Human UDP-glucuronosyltransferases: metabolism, expression, and disease.
AID1261322Selectivity index, ratio of CC50 for african green monkey Vero76 cells to EC50 for human RSV2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1408891Cytotoxicity against human DU145 cells assessed as reduction in cell viability after 48 hrs by MTT assay2018European journal of medicinal chemistry, Oct-05, Volume: 158Discovery of novel human inosine 5'-monophosphate dehydrogenase 2 (hIMPDH2) inhibitors as potential anticancer agents.
AID580128Inhibition of human IMPDH1 by Spectrophotometry2011Bioorganic & medicinal chemistry, Mar-01, Volume: 19, Issue:5
Cofactor-type inhibitors of inosine monophosphate dehydrogenase via modular approach: targeting the pyrophosphate binding sub-domain.
AID1223454Drug metabolism in diabetic human kidney microsomes assessed as UGT2B7*2 mutant-mediated intrinsic clearance of mycophenolic acid acyl glucuronide by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1223436Drug metabolism in non-diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID264232Antiangiogenic activity in mouse at 60 mg/kg/day, sc2006Journal of medicinal chemistry, May-04, Volume: 49, Issue:9
Identification of type 1 inosine monophosphate dehydrogenase as an antiangiogenic drug target.
AID1303374Antiviral activity against Yellow fever virus 17D infected in BHK21 cells assessed as inhibition of virus induced cytopathogenicity after 3 to 4 days by MTT assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID115873Tested for the inhibition of murine PFC response at 12.5 mg/kg dose1996Journal of medicinal chemistry, Oct-11, Volume: 39, Issue:21
Structure-activity relationships for inhibition of inosine monophosphate dehydrogenase by nuclear variants of mycophenolic acid.
AID1211281Ratio of drug level blood to plasma in human2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1223427Drug metabolism in non-diabetic human liver microsomes assessed as UGT2B7-mediated mycophenolic acid acyl glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID1211234Drug metabolism in human kidney microsomes assessed as UGT1A9-mediated unbound intrinsic glucuronidation clearance at 1 uM after 30 to 60 mins by LC-MS/MS analysis2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1498132Antiviral activity against Coxsackie virus B4 infected in African green monkey Vero cells assessed as inhibition of virus-induced cytopathicity2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID303352Inhibition of human IMPDH type 22007Journal of medicinal chemistry, Nov-15, Volume: 50, Issue:23
Probing binding requirements of type I and type II isoforms of inosine monophosphate dehydrogenase with adenine-modified nicotinamide adenine dinucleotide analogues.
AID1261310Cytotoxicity against BHK cells assessed as cell viability after 48 to 96 hrs by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1211274Ratio of UGT1A9-mediated unbound intrinsic glucuronidation clearance in human kidney microsomes to UGT1A9-mediated unbound intrinsic glucuronidation clearance in human intestinal microsomes at 1 uM in presence of 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID303356Antiproliferative activity against human K562 cells after 72 hrs2007Journal of medicinal chemistry, Nov-15, Volume: 50, Issue:23
Probing binding requirements of type I and type II isoforms of inosine monophosphate dehydrogenase with adenine-modified nicotinamide adenine dinucleotide analogues.
AID1422694Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.039 uM assessed as Kcat for substrate using NAD as fixed substrate at 700 uM and IMP as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID278375Effect on metabolism of 40 ug/ml RBV in Vero E6 cells assessed as RBV-TP concentration at 5 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1380551Antimicrobial activity against Mycobacterium tuberculosis SRMV2.6 H37Rv by alamar blue assay2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID1211203Drug metabolism in human liver microsomes assessed as UGT1A9-mediated unbound intrinsic glucuronidation clearance at 1 uM after 30 to 60 mins by LC-MS/MS analysis in presence of 1% bovine serum albumin2012Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 40, Issue:4
Characterization of in vitro glucuronidation clearance of a range of drugs in human kidney microsomes: comparison with liver and intestinal glucuronidation and impact of albumin.
AID1220789Ratio of drug level in blood to plasma in mouse2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID328840Suppression of aggregation of poly Q102-GFP protein expressed in zebrafish embryo at 1 uM by filter trap assay2007The Journal of biological chemistry, Mar-23, Volume: 282, Issue:12
Identification of anti-prion compounds as efficient inhibitors of polyglutamine protein aggregation in a zebrafish model.
AID1303373Cytotoxicity against mock-infected BHK21 cells assessed as reduction in cell viability after 72 hrs by MTT assay2016European journal of medicinal chemistry, Jul-19, Volume: 117A combined in silico/in vitro approach unveils common molecular requirements for efficient BVDV RdRp binding of linear aromatic N-polycyclic systems.
AID1774078Stabilization of TTR V3OM mutant (unknown origin) assessed as acid-mediated protein aggregation inhibition ratio at 4 uM incubated for 1 week by absorbance method2021Journal of medicinal chemistry, 10-14, Volume: 64, Issue:19
Repositioning of the Anthelmintic Drugs Bithionol and Triclabendazole as Transthyretin Amyloidogenesis Inhibitors.
AID1380559Growth inhibition of methicillin-resistant Staphylococcus aureus at 32 ug/ml2018Bioorganic & medicinal chemistry letters, 06-01, Volume: 28, Issue:10
Hit discovery of Mycobacterium tuberculosis inosine 5'-monophosphate dehydrogenase, GuaB2, inhibitors.
AID304373Inhibition of HDAC2007Journal of medicinal chemistry, Dec-27, Volume: 50, Issue:26
Dual inhibitors of inosine monophosphate dehydrogenase and histone deacetylases for cancer treatment.
AID1186337Antiviral activity against Respiratory syncytial virus infected in african green monkey Vero 76 cells assessed as reduction in plaque number after 5 days by plaque reduction assay2014Bioorganic & medicinal chemistry, Sep-01, Volume: 22, Issue:17
Antiviral activity of benzimidazole derivatives. III. Novel anti-CVB-5, anti-RSV and anti-Sb-1 agents.
AID134472In vivo antibody response to sheep red blood cells(SRBC) by plaques per million after 25 mg/kg oral administration in mice.1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID1223434Uncompetitive substrate inhibition of UGT2B7 in non-diabetic human kidney microsomes assessed as reduction in enzyme-mediated mycophenolic acid phenolic 7-O-glucuronide formation by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID637676Inhibition of IMPDH22012Bioorganic & medicinal chemistry letters, Jan-01, Volume: 22, Issue:1
The design, synthesis and in vitro immunosuppressive evaluation of novel isobenzofuran derivatives.
AID1422682Inhibition of recombinant human His-tagged IMPDH2 expressed in Escherichia coli BL21 (DE3) using IMP as substrate in presence of NAD2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1261326Antiviral activity against DENV-2 infected in BHK-21 cells assessed as inhibition of virus-induced cytopathogenicity after 3 to 4 days by MTT assay2015Bioorganic & medicinal chemistry, Nov-01, Volume: 23, Issue:21
Antiviral activity of benzotriazole derivatives. 5-[4-(Benzotriazol-2-yl)phenoxy]-2,2-dimethylpentanoic acids potently and selectively inhibit Coxsackie Virus B5.
AID1422702Uncompetitive inhibition of recombinant Cryptococcus neoformans His-tagged IMPDH expressed in Escherichia coli BL21 (DE3) at 0.156 uM assessed as Kcat for substrate using IMP as fixed substrate at 500 uM and NAD as second substrate with varying concentrat2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
Antifungal benzo[b]thiophene 1,1-dioxide IMPDH inhibitors exhibit pan-assay interference (PAINS) profiles.
AID1689894Immunosuppression-mediated cytotoxicity against human Jurkat T cells assessed as reduction in cell viability incubated for 48 hrs by MTT assay2020European journal of medicinal chemistry, Mar-01, Volume: 189Immunosuppressive properties of amino acid and peptide derivatives of mycophenolic acid.
AID1223433Ratio of Vmax to Km in non-diabetic human kidney microsomes assessed as UGT2B7-mediated mycophenolic acid phenolic 7-O-glucuronide formation per pmol protein by HPLC and LC-MS/MS method2011Drug metabolism and disposition: the biological fate of chemicals, Mar, Volume: 39, Issue:3
Diabetes mellitus reduces activity of human UDP-glucuronosyltransferase 2B7 in liver and kidney leading to decreased formation of mycophenolic acid acyl-glucuronide metabolite.
AID278364Effect on metabolism of 40 ug/ml RBV in Vero E6 cells assessed as RBV-MP concentration at 2 ug/ml after 24 h2007Antimicrobial agents and chemotherapy, Jan, Volume: 51, Issue:1
Activity of ribavirin against Hantaan virus correlates with production of ribavirin-5'-triphosphate, not with inhibition of IMP dehydrogenase.
AID1220790Ratio of drug level in blood to plasma in rat2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID210303Inhibitory activity against human T-lymphoblast CEM cell line using T cell proliferation assay2002Bioorganic & medicinal chemistry letters, Aug-19, Volume: 12, Issue:16
Rapid synthesis of triazine inhibitors of inosine monophosphate dehydrogenase.
AID1498133Antiviral activity against Punta Toro virus infected in African green monkey Vero cells assessed as inhibition of virus-induced cytopathicity2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID90470In vitro inhibition of human lymphocyte proliferation in response to pokeweed mitogen(PWM) was determined1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID1220795Plasma clearance in po dosed human2011Drug metabolism and disposition: the biological fate of chemicals, May, Volume: 39, Issue:5
Human pharmacokinetic prediction of UDP-glucuronosyltransferase substrates with an animal scale-up approach.
AID261590Inhibition of West Nile viral Rluc-Neo-Rep in Vero cells2006Journal of medicinal chemistry, Mar-23, Volume: 49, Issue:6
Identification of compounds with anti-West Nile Virus activity.
AID92749Inhibitory activity against human Inosine-5'-monophosphate dehydrogenase 2 (IMPDH type II)2002Journal of medicinal chemistry, Jan-31, Volume: 45, Issue:3
Novel mycophenolic adenine bis(phosphonate) analogues as potential differentiation agents against human leukemia.
AID1485255Cytotoxicity against African green monkey Vero cells assessed as alterations in cell morphology measured after 5 to 6 days post infection by microscopic analysis2017European journal of medicinal chemistry, Jul-28, Volume: 135Host dihydrofolate reductase (DHFR)-directed cycloguanil analogues endowed with activity against influenza virus and respiratory syncytial virus.
AID1498120Antiviral activity against Reovirus-1 infected in human HeLa cells assessed as inhibition of virus-induced cytopathicity2018Bioorganic & medicinal chemistry, 07-23, Volume: 26, Issue:12
Expedient synthesis and biological evaluation of alkenyl acyclic nucleoside phosphonate prodrugs.
AID1347411qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS Mechanism Interrogation Plate v5.0 (MIPE) Libary2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID1347164384 well plate NINDS Rhodamine confirmatory qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347163384 well plate NINDS AMC confirmatory qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347171Orthogonal mCherry assay for qRT-PCR qHTS of selected Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347172Secondary qRT-PCR qHTS assay for selected Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347158ZIKV-mCherry secondary qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347170Vero cells viability counterscreen for qRT-PCR qHTS assay of selected Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347156DAPI mCherry counterscreen qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1811Experimentally measured binding affinity data derived from PDB1996Cell, Jun-14, Volume: 85, Issue:6
Structure and mechanism of inosine monophosphate dehydrogenase in complex with the immunosuppressant mycophenolic acid.
AID977610Experimentally measured binding affinity data (Ki) for protein-ligand complexes derived from PDB1996Cell, Jun-14, Volume: 85, Issue:6
Structure and mechanism of inosine monophosphate dehydrogenase in complex with the immunosuppressant mycophenolic acid.
AID1159550Human Phosphogluconate dehydrogenase (6PGD) Inhibitor Screening2015Nature cell biology, Nov, Volume: 17, Issue:11
6-Phosphogluconate dehydrogenase links oxidative PPP, lipogenesis and tumour growth by inhibiting LKB1-AMPK signalling.
AID1346059Human inosine monophosphate dehydrogenase 2 (Nucleoside synthesis and metabolism)1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID1346128Human inosine monophosphate dehydrogenase 1 (Nucleoside synthesis and metabolism)1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and immunosuppressive activity of some side-chain variants of mycophenolic acid.
AID1159607Screen for inhibitors of RMI FANCM (MM2) intereaction2016Journal of biomolecular screening, Jul, Volume: 21, Issue:6
A High-Throughput Screening Strategy to Identify Protein-Protein Interaction Inhibitors That Block the Fanconi Anemia DNA Repair Pathway.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (8,436)

TimeframeStudies, This Drug (%)All Drugs %
pre-1990131 (1.55)18.7374
1990's781 (9.26)18.2507
2000's3382 (40.09)29.6817
2010's3125 (37.04)24.3611
2020's1017 (12.06)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials1,506 (16.68%)5.53%
Reviews1,196 (13.25%)6.00%
Case Studies1,426 (15.79%)4.05%
Observational100 (1.11%)0.25%
Other4,801 (53.17%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (880)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
[NCT01042457]Phase 324 participants (Actual)Interventional2009-05-31Completed
A Study to Determine the Safety and Efficacy in Lipoprotein Lipase-Deficient Subjects After Intramuscular Administration of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein LipaseS447X[NCT01109498]Phase 2/Phase 314 participants (Actual)Interventional2007-08-31Active, not recruiting
Measurement and Analysis of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate (MPA)[NCT00351377]Phase 3111 participants (Actual)Interventional2006-06-30Completed
A 6-month, Prospective, Open-label, Randomized, Controlled, Pilot Study Evaluating the Efficacy, Safety and Toxicity of an Optimized Immunosuppressive Regimen of CellCept (Mycophenolate Mofetil, MMF) and Reduced Doses of Both Calcineurin-inhibitors and Pr[NCT01213394]Phase 32 participants (Actual)Interventional2010-10-31Terminated(stopped due to Poor recruitment.)
Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy[NCT00303719]Phase 2342 participants (Actual)Interventional2002-03-26Terminated(stopped due to IRB Study Closure)
A Phase I/II Dose Escalation Trial of Clofarabine, in Addition to Melphalan and Thiotepa as Myeloablative Regimen Followed by an Allogeneic Unmodified Hematopoietic Stem Cell Transplant From HLA-Compatible Related or Unrelated Donors for the Treatment of [NCT00423514]Phase 1/Phase 238 participants (Actual)Interventional2006-11-20Completed
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis[NCT00282347]Phase 3144 participants (Actual)Interventional2006-01-31Completed
Impact of Lymphocyte Anti-metabolite Immunosuppressions on Donor-Specific Anti-HLA Antibody and Kidney Graft Outcome: Open-label, Multi-center, Single Arm, Phase 4 Trial (DoSAKOM)[NCT03794492]Phase 4169 participants (Actual)Interventional2018-03-31Active, not recruiting
Immunosuppressive Medications for Previous Participants in Clinical Trial NIS01 (ITN005CT, NCT00014911)[NCT01309022]0 participants Expanded AccessNo longer available
A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies[NCT04904588]Phase 2300 participants (Anticipated)Interventional2021-09-30Recruiting
Registry of IgA Nephropathy in Chinese Children[NCT03015974]1,200 participants (Anticipated)Observational [Patient Registry]2016-01-31Recruiting
Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases[NCT05805605]Phase 256 participants (Anticipated)Interventional2023-05-01Recruiting
A Randomized, Open-label,Controlled Phase II b Study to Demonstrate Efficacy and Safety of Sirolimus Chronic Rejection After Lung Transplant[NCT04415476]Phase 20 participants (Actual)Interventional2020-06-30Withdrawn(stopped due to Study did not proceed to IRB approval.)
A Pharmacoeconomic Study Comparing the Use of Mycophenolate Mofetil or Cyclophosphamide as Induction Therapy in Lupus Nephritis Patients in Egypt[NCT05195086]122 participants (Actual)Observational2018-07-01Completed
CLAG-M or FLAG-Ida Chemotherapy Followed Immediately by Related/Unrelated Reduced-Intensity Conditioning (RIC) Allogeneic Hematopoietic Cell Transplantation for Adults With Myeloid Malignancies at High Risk of Relapse: A Phase 1 Study[NCT04375631]Phase 1120 participants (Anticipated)Interventional2020-12-03Recruiting
Organ Function Preservation by the Combination Treatment of the optImuM Dose of calcineUrin Inhibitor and Mycophenolate Sodium in Kidney Recipients: OPTIMUM Study[NCT01159080]Phase 4350 participants (Actual)Interventional2010-04-01Completed
A Phase I Study of Intensity Modulated Total Marrow Irradiation (IMTMI) in Addition to Fludarabine/Melphalan Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies[NCT02333162]Phase 130 participants (Anticipated)Interventional2014-12-05Recruiting
Optimizing Haploidentical Aplastic Anemia Transplantation (CHAMP) (BMT CTN 1502)[NCT02918292]Phase 232 participants (Actual)Interventional2017-07-03Completed
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen[NCT02722668]Phase 216 participants (Actual)Interventional2017-05-15Active, not recruiting
A Phase III, Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf (Tacrolimus)/MMF, Modified Release (MR) Tacrolimus/MMF and Neoral (Cyclosporine)/MMF in de Novo Kidney Transplant Recipients[NCT00064701]Phase 3668 participants (Actual)Interventional2003-06-30Completed
A Double-Blind, Placebo -Controlled, Randomized Study to Assess the Efficacy and Safety of Zenapax in Combination With CellCept, Cyclosporine, and Corticosteroids in Patients Undergoing Cardiac Transplantation.[NCT00048165]Phase 4434 participants (Actual)Interventional1999-08-31Completed
A Study of Two Different Treatment Strategies in IgG4-related Disease Patients With Re-elevation of Serum IgG4 Level During Maintenance Remission Period: a Randomized Double Blind Placebo Controlled Multicenter Study[NCT05974683]108 participants (Anticipated)Interventional2023-08-01Not yet recruiting
An Open-label, Single-sequence Study to Investigate the Effects of BMS-986165 at Steady State on the Single Dose Pharmacokinetics of Mycophenolate Mofetil (MMF) in Healthy Male Subjects[NCT03660436]Phase 1131 participants (Actual)Interventional2018-08-14Completed
A Trial to Evaluate the Long Term Prognosis in Rosai-Dorfman Disease[NCT05284942]Phase 420 participants (Anticipated)Interventional2011-10-01Recruiting
A Multi-Center, Randomized, Double Blind, Phase III Trial Evaluating Corticosteroids With Mycophenolate Mofetil vs. Corticosteroids With Placebo as Initial Systemic Treatment of Acute Graft-Vs-Host-Disease (BMT CTN #0802)[NCT01002742]Phase 3236 participants (Actual)Interventional2010-01-31Completed
Attenuating Ischemia Reperfusion Injury After Living Donor Renal Transplantation[NCT01149993]Phase 40 participants (Actual)Interventional2010-06-30Withdrawn(stopped due to FDA clinical hold, IND withdrawn.)
Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine, Nanjing, China.[NCT01269021]176 participants (Actual)Interventional2010-11-30Completed
An Open-label, Balanced, Randomized, Two-treatment, Four-period, Two Sequence, Single Dose, Replicate, Crossover, Bioequivalence Study of Mycophenolate Mofetil 500 mg Tablet of Dr. Reddy's Laboratories Limited Comparing With That of Cellcept 500 mg Tablet[NCT01283841]Phase 148 participants (Actual)Interventional2007-11-30Completed
Randomized Controlled Trial Study of Anti-r-HuEpo Associated PRCA Treated by Cyclosporine and Mycophenolate Mofetil (MMF) Compared With Cyclophosphamide and Prednisolone[NCT01288131]Phase 38 participants (Actual)Interventional2009-01-31Terminated(stopped due to We observed >90 % efficacy in cyclophosphamide and Prednisolone group for treatment of anti-i-HuEpo associated PRCA)
A Three-Part Phase 1 Study to Determine the Potential Drug Interaction Between ACH-0144471 and Midazolam, Fexofenadine and Mycophenolate Mofetil in Healthy Subjects[NCT03108274]Phase 135 participants (Actual)Interventional2017-04-18Completed
A Multicenter, Two Arm, Randomized, Open Label Clinical Study Investigating Renal Function in an Advagraf® Based Immunosuppressive Regimen With or Without Sirolimus in Kidney Transplant Patients[NCT01363752]Phase 4853 participants (Actual)Interventional2011-03-08Completed
Allogeneic Islet Cells Transplanted Into the Omentum[NCT02821026]Phase 1/Phase 24 participants (Actual)Interventional2016-05-31Completed
Effect of Cyclophosphamide Versus Mycophenolate Mofetil in Induction Therapy of Lupus Nephritis in Nepalese Population[NCT03200002]Phase 249 participants (Actual)Interventional2014-01-01Completed
The Mechanisms and Significances of Synergistic Effects of Mycophenolic Acid and Lipopolysaccharide on Interleukin-1β Secretion by Mononuclear[NCT02435368]10 participants (Anticipated)Observational2015-04-30Recruiting
Vorinostat With Gemcitabine/Clofarabine/Busulfan for Allogeneic Transplantation for Aggressive Lymphomas[NCT04220008]Phase 230 participants (Anticipated)Interventional2023-06-01Not yet recruiting
Mycophenolate Mofetil Therapy for Reduction of the HIV Reservoir[NCT03262441]Phase 25 participants (Actual)Interventional2018-02-12Completed
Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836[NCT01220531]0 participants Expanded AccessApproved for marketing
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes[NCT02224872]Phase 218 participants (Actual)Interventional2014-08-31Completed
Effect of Sirolimus or Mycophenolate With Tacrolimus on Survival of Pancreas and Kidney Grafts in Type 1 Diabetic Recipients After Simultaneous Pancreas and Kidney Transplantation[NCT03582878]Phase 4238 participants (Actual)Interventional2004-01-01Completed
To Evaluate Prevention Effect Of Everolimus and Low-dose Tacrolimus in Comparison With Standard-dose Tacrolimus Therapy With Mycophenolic Acid on the New Onset Diabetes Mellitus After Transplantation in the Renal Allograft Recipients[NCT02036554]Phase 4234 participants (Anticipated)Interventional2013-03-31Recruiting
A 12-Month, Randomized, Open-Label, Phase IIA Study Evaluating the Safety and Efficacy of Siplizumab in Combination With Belatacept and MPA Compared to Standard of Care Immunosuppression in de Novo Renal Transplant Recipients (ASCEND)[NCT05669001]Phase 290 participants (Anticipated)Interventional2023-11-30Not yet recruiting
Everolimus in Association With Very Low-dose Tacrolimus Versus Enteric-coated Mycophenolate Sodium With Low-dose Tacrolimus in de Novo Renal Transplant Recipients - A Prospective Single Center Study[NCT02084446]Phase 4120 participants (Actual)Interventional2012-12-31Completed
Open Label, Multi-center, Randomized Study to Compare of Tacrolimus and Steroids in Combination With Mycophenolate Mofetil or Without Mycophenolate Mofetil in Liver Transplantation With Hepatitis B Virus(HBsAg) Positive[NCT02075242]Phase 4170 participants (Anticipated)Interventional2014-01-31Recruiting
A Pilot Randomized Study to Assess the Effect and Safety Profile of Thymoglobulin® in Primary Cardiac Transplant Recipients: A 12-month, Single Center, Randomized, Open-label Study of Efficacy Comparing Immediate Treatment With and Without Thymoglobulin® [NCT03292861]Phase 260 participants (Anticipated)Interventional2018-09-13Enrolling by invitation
Clinical Evaluation of the Long-term Benefit of Enteric-coated Mycophenolate (MPAs) After Liver Transplantation[NCT05707520]500 participants (Anticipated)Observational2022-12-04Recruiting
Shortened-duration Tacrolimus Following Nonmyeloablative, Related Donor BMT With High-dose Posttransplantation Cyclophosphamide[NCT01342289]Phase 1127 participants (Actual)Interventional2011-08-31Completed
The Effect of Everolimus on the Pharmacokinetics of Tacrolimus in Renal Transplant Patients, and the Effect of ABCB1、CYP3A4、CYP3A5、PORGenetic Polymorphism on the Two Drugs[NCT02077556]Phase 414 participants (Actual)Interventional2014-04-30Completed
Open Label, Multicenter Randomized Control Study to Investigate the Incidence of NODAT (New-Onset Diabetes After Transplantation), Safety and Efficacy of Corticosteroids Early Withdrawal in Liver Transplanted Recipients[NCT02095418]152 participants (Anticipated)Interventional2014-02-28Recruiting
Allogeneic Stem Cell Transplantation for Patients With Hematological Malignancies Using Multiple Unrelated Cord Blood Units[NCT00547196]10 participants (Actual)Interventional2005-08-16Active, not recruiting
Efficacy and Safety of Induction Strategies Combined With Low Tacrolimus Exposure in Kidney Transplant Recipients Receiving Everolimus or Sodium Mycophenolate[NCT01354301]Phase 4300 participants (Actual)Interventional2011-05-31Completed
A Phase 1, Open Label, Single Sequence, Drug Interaction Study of the Pharmacokinetics of ASP015K and Mycophenolate Mofetil (MMF) After Separate and Concomitant Administration to Healthy Adult Volunteers[NCT01364987]Phase 124 participants (Actual)Interventional2009-05-31Completed
A Phase I/II Clinical Trial of NK Cells Administration to Prevent Disease Relapse for Patient With High-Risk Myeloid Malignancies Undergoing Haploidentical Stem-Cell Transplantation[NCT01904136]Phase 1/Phase 290 participants (Anticipated)Interventional2014-04-22Completed
A Phase 2a, Randomized, Open-Label, Active Control, Multi-Center Study to Assess the Efficacy and Safety of Bleselumab in Preventing the Recurrence of Focal Segmental Glomerulosclerosis in de Novo Kidney Transplant Recipients[NCT02921789]Phase 267 participants (Actual)Interventional2017-05-22Completed
Initial Systemic Treatment of Acute GVHD: A Phase II Randomized Trial Evaluating Etanercept, Mycophenolate Mofetil (MMF), Denileukin Diftitox (ONTAK), and Pentostatin in Combination With Corticosteroids (BMT CTN #0302)[NCT00224874]Phase 2180 participants (Actual)Interventional2005-09-30Completed
Allogeneic Hematopoietic Cell Transplantation Using a Non-Myeloablative Preparative Regimen of Total Lymphoid Irradiation and Anti-Thymocyte Globulin for Older Patients With Hematologic Malignancies[NCT00185640]Phase 2303 participants (Actual)Interventional2003-03-31Completed
Low Steroid Dose Combined With Mycophenolic Acid (Myfortic) Compared With High Dose Steroid for Minimal Change Nephrotic Syndrome[NCT01197040]Phase 3117 participants (Actual)Interventional2009-10-31Completed
Phase III, Open, Randomized, Parallel-group Clinical Trial, to Evaluate the Efficacy and Safety of Treatment With Prednisone, Cyclosporine, Mycophenolic Acid Versus Prednisone and Mycophenolic Acid in Lupus Nephritis Type III-IV-V.[NCT01299922]Phase 30 participants (Actual)Interventional2011-02-28Withdrawn(stopped due to IT was impossible to find patients)
Investigating New Onset Diabetes Mellitus in Kidney Transplant Recipients Receiving an Advagraf-Based Immunosuppressive Regimen With or Without Corticosteroids - A Multicenter, Two Arm, Randomized, Open Label Clinical Study[NCT01304836]Phase 41,166 participants (Actual)Interventional2011-01-22Completed
Nonmyeloablative Hematopoietic Cell Transplantation (HCT) for Patients With Hematologic Malignancies Using Related, HLA-Haploidentical Donors: A Phase II Trial of Peripheral Blood Stem Cells (PBSC) as the Donor Source[NCT01028716]Phase 246 participants (Actual)Interventional2010-05-19Terminated(stopped due to The study experienced lower accrual rates after the onset of COVID. Upon review of the collected data it was deemed that an adequate amount of subjects has been enrolled to date to assess study aims.)
Use of Zevalin to Enhance the Efficacy of Non-Myeloablative Allogeneic Transplantation in Patients With Relapsed or Refractory CD20+ Non-Hodgkin's Lymphoma[NCT01811368]Phase 220 participants (Anticipated)Interventional2013-03-12Active, not recruiting
New Onset of Type 1 Diabetes Mycophenolate Mofetil-Daclizumab Clinical Trial (Preservation of Pancreatic Production of Insulin Through Immunosuppression-POPPII #1)[NCT00100178]Phase 2126 participants (Actual)Interventional2004-05-31Completed
Open Label Balanced Randomized Two-treatment Two-period Two-sequence Single Dose Two-way Crossover Oral Bioequivalence Study of Mycophenolate Mofetil Capsules 250 mg in Normal Healthy Adult Human Subjects Under Fed Conditions[NCT01080417]84 participants (Actual)Interventional2008-06-30Completed
A 2 Step Approach to Matched Related Hematopoietic Stem Cell Transplantation for Patients With Hematological Malignancies-5+5 Dosing[NCT03712878]Phase 210 participants (Actual)Interventional2018-09-19Active, not recruiting
Transplantation of Umbilical Cord Blood for Patients With Hematological Diseases With Cyclophosphamide/Fludarabine/Total Body Irradiation or Cyclophosphamide/Fludarabine/Thiotepa/Total Body Irradiation Myeloablative Preparative Regimen[NCT00719888]Phase 2135 participants (Actual)Interventional2005-11-18Active, not recruiting
Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial[NCT01092962]Phase 370 participants (Actual)Interventional2010-09-30Completed
Reduced Intensity Myeloablative Total Body Irradiation and Thymoglobulin Followed by Allogeneic Peripheral Blood Stem Cell Transplantation[NCT00709592]Phase 242 participants (Actual)Interventional2008-07-21Completed
Population Pharmacokinetic and Pharmacodynamic Model-based Dosing Strategy of Mycophenolate Mofetil in Pediatric Hematopoietic Stem Cell Transplantation(HSCT) Patients[NCT04868786]Phase 120 participants (Anticipated)Interventional2020-10-26Recruiting
A Phase 2 Pilot, Multicenter, Single Arm Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of GSK1070806 Plus Standard of Care for the Prevention of Delayed Graft Function in Adult Subjects After Renal Transplantation[NCT02723786]Phase 27 participants (Actual)Interventional2016-08-27Terminated(stopped due to Lack of efficacy)
Phase I/II Study Using Prophylactic Donor Lymphocyte Infusion Early Post-Transplant After Allogeneic Hematopoietic Cell Transplantation Using Post-Transplantation Cyclophosphamide for High-Risk Hematologic Malignancies[NCT05327023]Phase 1/Phase 2430 participants (Anticipated)Interventional2022-05-23Recruiting
Evaluating the Safety and Efficacy of Chemotherapy in Patients With Relapsed Small Cell Lung Cancer in Combination With Allopurinol and MycoPhenolate (CLAMP Trial)[NCT05049863]Phase 1/Phase 236 participants (Anticipated)Interventional2023-02-27Recruiting
A Randomized Trial of Treatment in Patients With IgG4-Related Disease[NCT02458196]Phase 260 participants (Anticipated)Interventional2015-04-30Recruiting
Phase II/III Randomized Study to Improve Overall Survival in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus Mycop[NCT02416388]Phase 2/Phase 33,100 participants (Anticipated)Interventional2015-01-31Recruiting
Prospective Multicenter Observational Cohort Study of Comparative Effectiveness of Disease-modifying Treatments for Myasthenia Gravis[NCT03490539]167 participants (Actual)Observational [Patient Registry]2018-05-07Completed
A Comparison of PTCy-ATG and ATG Strategy in Haploidentical HSCT for Acute Graft-versus-host Disease Prophylaxis[NCT03689465]Phase 4260 participants (Anticipated)Interventional2018-10-29Recruiting
CYCLONES - CYClophosphamide LOw Dose and No Extra Steroid[NCT03492255]49 participants (Actual)Interventional2018-04-12Terminated(stopped due to Significative difference between percentage of renal response (primary outcome) between the two study arms.)
A Phase 1/1b Adaptive Dose Escalation Study of Mycophenolate Mofetil (MMF) in Combination With Standard of Care for Patients With Glioblastoma[NCT05236036]Phase 160 participants (Anticipated)Interventional2022-08-08Recruiting
Single-Arm, Open Label, Interventional Phase II Clinical Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy[NCT03674411]Phase 222 participants (Actual)Interventional2019-01-02Active, not recruiting
Belatacept in De Novo Heart Transplantation - Pilot Study[NCT04477629]Phase 212 participants (Anticipated)Interventional2020-08-06Recruiting
REduCing Immunogenicity to PegloticasE (RECIPE) Study[NCT03303989]Phase 235 participants (Actual)Interventional2018-06-14Completed
A Partially Blinded, Prospective, Randomized Multicenter Study Evaluating Efficacy, Safety and Tolerability of Oral Sotrastaurin Plus Standard or Reduced Exposure Tacrolimus vs. Mycophenolic Acid Plus Tacrolimus in de Novo Renal Transplant Recipients[NCT01064791]Phase 2298 participants (Actual)Interventional2009-12-31Completed
A Pilot Study of Reduced Intensity HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Advanced Myelofibrosis[NCT03426969]Early Phase 13 participants (Actual)Interventional2018-01-31Completed
Prospective Multicenter Randomized Openlabel Study to Evaluate the Benefit on Renal Function at 12months Post-transplantation of Immunosuppressive Treatment With Withdrawal of Calcineurin Inhibitor at 3months and Combining Mycophenolate Sodium-Everolimus [NCT02334488]Phase 3329 participants (Actual)Interventional2014-12-11Completed
Prospective, Randomized 2 x 2 Factorial Trial of Rabbit Anti-thymocyte Globulin Induction (Single vs. Alternate Day Administration) at Renal Transplantation, With Delayed Calcineurin-inhibitor Withdrawal vs. Minimization[NCT00556933]Phase 4180 participants (Actual)Interventional2004-04-01Completed
Rituximab Versus Mycophenolate Mofetil in Children With Steriod-dependent Nephrotic Syndrome: A Single-center, Randomized Controlled Trial[NCT05843968]Phase 246 participants (Anticipated)Interventional2023-07-01Recruiting
Mycophenolate Mofetil in Systemic Sclerosis: A Phase 1 Pharmacokinetic Study of Orally Ingested Mycophenolate Mofetil Tablets in Patients Suffering From Systemic Sclerosis[NCT03678987]35 participants (Actual)Observational2018-09-13Completed
A Phase 2/3, Prospective, Randomized, Multi-center, Open-Label, Controlled Trial to Assess the Efficacy & Safety of Cellular Immunotherapy With MDR-102 for Induction of Immune Quiescence™in Recipients of HLA-mismatched, LD Kidney Transplants[NCT03605654]Phase 2/Phase 3172 participants (Anticipated)Interventional2024-12-31Not yet recruiting
Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome[NCT00357565]Phase 220 participants (Anticipated)Interventional2005-11-30Recruiting
A Phase II, Randomized Study to Evaluate the Safety and Efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) For the Treatment of Acute GVHD in Patients Who Receive Allogeneic Hematopoietic Stem Cell Transplantation[NCT00136903]Phase 232 participants (Actual)Interventional2005-04-27Completed
Immunosuppression With Antithymocyte Globulin, Rituximab, Tacrolimus, Mycophenolate Mofetil and Sirolimus, Followed by Withdrawal of Immunosuppression, in Living-donor Renal Transplant Recipients[NCT01318915]Early Phase 110 participants (Actual)Interventional2011-07-25Terminated(stopped due to The stopping rule for incidence of acute rejection was met.)
Multi-center, Open-label, Randomized Controlled Phase 4 Study to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients Undergoing Maintenance Therapy With CNI Plus MPA.[CORAL Study][NCT05193565]Phase 4206 participants (Anticipated)Interventional2021-11-19Recruiting
Clinical Study of MMF in Treatment of IIM-ILD and Its Effect on Peripheral Blood Treg Cells[NCT05129410]Phase 420 participants (Anticipated)Interventional2020-12-01Recruiting
Calcineurin-Sparing in a Steroid-free Maintenance Immunosuppression Protocol After Kidney Transplantation[NCT01062555]Phase 1/Phase 2527 participants (Actual)Interventional2006-10-01Completed
TAILOR Study: Tacrolimus Versus Mycophenolate for Autolmmune Hepatitis Patients With incompLete Response On First Line Therapy: a Randomized Trial[NCT05221411]Phase 486 participants (Anticipated)Interventional2022-01-19Recruiting
A Crossover, Randomized and Multi-center Study to Evaluate the Efficacy and Safety of Abatacept Versus Mycophenolate Mofetil (MMF) in Treatment of PV[NCT05303272]Phase 460 participants (Anticipated)Interventional2021-02-01Recruiting
High-dose Post-transplantation Cyclophosphamide as Graft Versus-host Disease Prophylaxis After Allogeneic Hematopoietic Stem Cell Transplantation[NCT02294552]Phase 2200 participants (Actual)Interventional2014-10-31Completed
MyOra® (Mycophenolate Mofetil) Post-Authorization Safety Study for Prophylaxis in de Novo Renal Transplant Patients[NCT03517982]12 participants (Actual)Observational2014-10-31Completed
Intracoronary Analysis of Cardiac Allograft Vasculopathy in Comparison to Coronary Artery Disease by Means of Optical Coherence Tomography[NCT02254668]Phase 4278 participants (Anticipated)Interventional2013-12-31Recruiting
An Observational Study of Mycophenolate Mofetil Combined With Glucocorticoid in the Treatment of Relapse Vogt-Koyanagi-Harada Disease[NCT05627739]15 participants (Anticipated)Observational2021-10-01Recruiting
A Phase II Study of IL-6 Receptor Blockade to Ameliorate Acute Graft Versus Host Disease and Early Toxicity After Double Unit Cord Blood Transplantation in Adults With Hematologic Malignancies.[NCT03434730]Phase 246 participants (Actual)Interventional2018-02-07Active, not recruiting
A Phase 2, Randomized, Double-blind, Placebo-controlled Evaluation of the Safety and Efficacy of BMS-986165 With Background Treatment in Subjects With Lupus Nephritis[NCT03943147]Phase 216 participants (Actual)Interventional2019-07-15Terminated(stopped due to Insufficient enrollment)
Phase 3 Study of Treatment of IgAN With Multi-glycoside of Tripterygium Wilfordii HOOK. f[NCT02187900]Phase 3300 participants (Anticipated)Interventional2014-06-30Recruiting
Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease[NCT01659606]Phase 240 participants (Anticipated)Interventional2012-07-31Active, not recruiting
Ruxolitinib With Calcineurin and Methotrexate vs. Calcineurin Plus Methotrexate and Mycophenolate Mofetil as Graft Versus Host Disease Prophylaxis for HLA-haploidentical Hematopoietic Stem Cell Transplantation[NCT04838704]Phase 4206 participants (Anticipated)Interventional2021-04-08Recruiting
Reduce Intensity Conditioning (RIC) Allogenic Hematopoietic Stem Cell Transplantation (Allo HSCT) for Patients With Relapsed Multiple Myeloma: A Pilot Study[NCT04205240]Phase 21 participants (Actual)Interventional2020-12-22Terminated(stopped due to Poor accrual)
A Single-Dose, 2-Period, 2-Treatment, 2-Way Crossover Bioequivalence Study of Mycophenolate Mofetil 250 mg Capsules Under Fasting Conditions[NCT00911274]Phase 153 participants (Actual)Interventional2006-10-31Completed
A Pilot Randomized Controlled Trial of De Novo Belatacept-Based Immunosuppression in Lung Transplantation[NCT03388008]Phase 227 participants (Actual)Interventional2019-12-17Completed
12-month Open Label, Randomized, Multicenter Study Evaluating Efficacy, Safety and Tolerability of Oral AEB071 Plus Tacrolimus (Converted to Mycophenolic Acid After 3 Months), vs. Mycophenolic Acid Plus Tacrolimus in de Novo Renal Transplant Recipients[NCT00403416]Phase 1/Phase 2215 participants (Actual)Interventional2006-10-31Completed
Evaluation of Acute Rejection Rates in de Novo Renal Transplant Recipients Following Thymoglobulin Induction, CNI-free, Nulojix (Belatacept)-Based Immunosuppression[NCT02137239]Phase 258 participants (Actual)Interventional2015-12-31Completed
Clinical and Laboratory Evaluation of Acute Rejection, Myocyte Growth, Repair, and Oxidative Stress Following de Novo Cardiac Transplant: A Comparison Between Tacrolimus- and Cyclosporine- Based Immunoprophylactic Regimens With MPA TDM[NCT00157014]Phase 3111 participants (Actual)Interventional2004-05-10Completed
A Phase IV, Single Center Pilot Study Using Alemtuzumab (Campath-1H) Induction Combined With Prednisone-Free, Calcineurin-Inhibitor-Free Immunosuppression in Kidney Transplantation[NCT00166712]Phase 440 participants (Actual)Interventional2005-04-30Terminated(stopped due to Study stopped due to lack of efficacy & funding.)
Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating a CNI-free Regimen With Enteric-Coated Mycophenolate Sodium (EC-MPS) and Everolimus in Comparison to Standard Therapy With Enteric-Coated Mycophenolate Sodium (EC-MPS) a[NCT00154310]Phase 4300 participants (Actual)Interventional2005-06-30Completed
Intra-osseous Co-transplant of Cord Blood and Mesenchymal Stromal Cells: A Feasibility Study[NCT02181478]Early Phase 16 participants (Actual)Interventional2015-07-22Completed
Myfortic (Enteric-coated Mycophenolate Sodium) for the Treatment of Non-infectious Intermediate Uveitis - a Prospective, Controlled, Randomized Multicenter Trial[NCT01092533]Phase 344 participants (Actual)Interventional2010-03-31Completed
Phase II/III, Open-Label, Randomized, Controlled, Multiple-Dose Study of Efficacy and Safety of BMS-224818 (Belatacept) as Part of a Quadruple Drug Regimen in First Renal Transplant Recipients[NCT00035555]Phase 2230 participants (Actual)Interventional2001-03-31Completed
A Single-Dose, Replicate, Comparative Bioavailability Study of Two Formulations of Mycophenolate Mofetil 500 mg Tablets Under Fed Conditions[NCT00908128]Phase 140 participants (Actual)Interventional2006-08-31Completed
Randomized Controlled Trial to Evaluate the Efficacy of Enteric-coated Mycophenolate Sodium Versus Azathioprine for the Induction and Maintenance of Remission of the Extra-renal Lupus Manifestations[NCT01112215]Phase 4240 participants (Actual)Interventional2009-12-31Completed
A 12-Month, Randomized, Controlled, Open-Label, Dose Escalation Study Evaluating Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of an Anti-CD2 Monoclonal Antibody, TCD601(Siplizumab) Compared to Anti-thymocyte Globulin (rATG), as In[NCT04311632]Phase 224 participants (Anticipated)Interventional2021-05-26Recruiting
Matched Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplantation With a Conditioning Regimen of Targeted Busulfan, Cyclophosphamide, and Thymoglobulin[NCT00611351]Phase 25 participants (Actual)Interventional2005-06-07Completed
Allogeneic Peripheral Blood Stem Cell Transplantation With Minimally Myelosuppressive Regimen of Pentostatin and Low-dose Total-body Irradiation[NCT00571662]Phase 276 participants (Actual)Interventional2000-12-08Completed
Booster Effects With Autoimmune Treatments in Patients With Poor Response to Initial COVID-19 Vaccine (ACV01)[NCT05000216]Phase 2257 participants (Actual)Interventional2021-08-13Active, not recruiting
Efficacy of Mycophenolate Mofetil Versus Leflunomide as Maintenance Treatment for IgG4-RD Patients With Internal Organ Involvement[NCT05789017]60 participants (Anticipated)Interventional2022-07-01Recruiting
A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic [NCT05457556]Phase 3435 participants (Anticipated)Interventional2023-03-15Recruiting
Partially HLA-Mismatched Related Donor Hematopoietic Stem Cell Transplantation Using Killer Immunoglobulin Receptor and Human Leukocyte Antigen Based Donor Selection[NCT02880293]44 participants (Actual)Interventional2016-08-23Completed
A Randomized Open-label Study of Fixed-dose Versus Concentration-controlled Mycophenolate Mofetil for the Treatment of Active Lupus Nephritis[NCT03920059]Phase 42 participants (Actual)Interventional2019-08-20Terminated(stopped due to Slow recruitment rate)
Sirolimus-based Immunosuppression Treatment Regimen for Liver Transplantation: A Multicenter, Open-label, Randomized, Controlled Clinical Trial in Liver Transplant Recipients With Hepatocellular Carcinoma[NCT03500848]Phase 2/Phase 3130 participants (Anticipated)Interventional2018-05-01Not yet recruiting
Extended Follow-Up After Islet Transplantation in Type 1 Diabetes (CIT-08)[NCT01369082]75 participants (Actual)Observational2011-05-31Completed
Mycophenolate Mofetil and Prednisolone Versus Prednisolone Alone in Fibrotic Hypersensitivity Pneumonitis: a Randomized Controlled Trial[NCT05626387]Phase 4144 participants (Anticipated)Interventional2022-11-23Recruiting
Low-dose Combination of Mycophenolate Mofetil and Tacrolimus for Refractory Lupus Nephritis: a 12-month Prospective Study[NCT01203709]Phase 420 participants (Actual)Interventional2010-08-31Completed
Multicenter Registry of Pediatric Lupus Nephritis in China[NCT03791827]1,200 participants (Anticipated)Observational2018-12-01Recruiting
JAK Inhibitor Prior to Allogeneic Stem Cell Transplant for Patients With Primary and Secondary Myelofibrosis: A Prospective Study[NCT02251821]Phase 299 participants (Actual)Interventional2014-10-20Active, not recruiting
A Single-center,Randomized,Open-label,12 Months Study,2 Parallel Group to Compare the Efficacy of Everolimus Combination + Tacrolimus in Regression of Left Ventricular Hypertrophy vs Tacrolimus + MMF in Renal Transplant Patients[NCT03415750]Phase 420 participants (Actual)Interventional2016-11-30Completed
Adoptive Immunotherapy in Patients With Relapsed Hematological Malignancy: Effect of Duration and Intensity of Early GVHD Prophylaxis on Long-Term Clinical Outcomes[NCT02593123]Phase 231 participants (Actual)Interventional2015-11-04Completed
Steroid Free Immunosuppression or Calcineurin Inhibitor Minimization After Basiliximab Induction Therapy in Kidney Transplantation: Comparison With a Standard Quadruple Immunosuppressive Regimen[NCT01560572]Phase 4305 participants (Actual)Interventional2011-04-30Completed
A Two Part, Phase 1/2, Safety, PK and PD Study of TOL101, an Anti-TCR Monoclonal Antibody for Prophylaxis of Acute Organ Rejection in Patients Receiving Renal Transplantation[NCT01154387]Phase 1/Phase 285 participants (Anticipated)Interventional2010-07-31Active, not recruiting
An Open-label, Balanced, Randomized, Two-treatment, Two-period, Two Sequence, Single Dose, Crossover, Bioequivalence Study of Mycophenolate Mofetil 500 mg Tablet of Dr. Reddy's Laboratories Limited Comparing With That of Cellcept 500 mg Tablet of Roche La[NCT01283867]Phase 192 participants (Actual)Interventional2007-11-30Completed
A Randomized Controlled Multi-center Trial of Mycophenolate Mofetil for the Patient With High Risk Membranous Nephropathy[NCT01282073]Phase 362 participants (Anticipated)Interventional2011-03-31Recruiting
A Pilot Study to Assess Engraftment Using CliniMACS TCR-α/β and CD19 Depleted Stem Cell Grafts From Haploidentical Donors for Hematopoietic Progenitor Cell Transplantation (HSCT) in Patients With Relapsed Lymphoma[NCT02652468]11 participants (Actual)Interventional2016-03-10Completed
Weaning of Immunosuppression in Nephritis of Lupus[NCT01284725]Phase 3100 participants (Actual)Interventional2011-01-31Active, not recruiting
Myfortic® Combined With Low-dose Steroid in Minimal Change Nephrotic Syndrome[NCT01185197]Phase 420 participants (Actual)Interventional2010-09-30Completed
Clinical Research of Allogeneic Hematopoietic Stem Cell Transplantation for Treatment of Children With Stage 4/M Neuroblastoma: A Prospective, Single-arm, Phase II, Multi-center Trial[NCT05303727]Phase 264 participants (Anticipated)Interventional2022-08-31Not yet recruiting
Budesonide in Liver Transplantation[NCT03315052]Phase 40 participants (Actual)Interventional2019-01-31Withdrawn(stopped due to Delay in IRB approval)
A Study Evaluating Escalating Doses of 90Y-DOTA-BC8 (Anti-CD45) Antibody Followed by Allogeneic Stem Cell Transplantation for High-Risk Acute Myeloid Leukemia (AML) Acute Lymphoblastic Leukemia (ALL), or Myelodysplastic Syndrome (MDS)[NCT01300572]Phase 116 participants (Actual)Interventional2012-01-31Completed
Relationships Between Pharmacokinetic and Pharmacodynamic Strategies for Assessment of the Risks for Acute Rejection and Side Effects of Mycophenolate Mofetil[NCT01292226]Phase 245 participants (Actual)Interventional2006-12-31Completed
Pharmacokinetics of Mycophenolate Mofetil in de Novo Lung Allograft Recipients[NCT01014442]Phase 368 participants (Actual)Interventional2010-01-31Completed
Alloantibodies in Pediatric Heart Transplantation[NCT01005316]290 participants (Actual)Observational2010-01-31Terminated(stopped due to Inability to meet accrual goals within the funding period.)
Early vs. Delayed EVERolimus in de Novo HEART Transplant Recipients: Optimization of the Safety/Efficacy Profile (EVERHEART Study)[NCT01017029]Phase 4182 participants (Actual)Interventional2009-09-30Completed
Open-Label, Randomized, Multicenter, Parallel-Group Efficacy and Safety Study of Tacrolimus Immunosuppressive Therapy After Kidney Transplantation[NCT00717379]Phase 450 participants (Actual)Interventional2007-05-31Completed
A Randomized, Open Label Study Comparing the Effect of CellCept Combined With Low Dose Versus Standard Dose Tacrolimus, and Corticosteroids, on Kidney Function in Renal Transplantation Patients[NCT00758602]Phase 4210 participants (Actual)Interventional2008-09-30Completed
Phase IV, Open-Label, Multicenter, Randomized Study Comparing Mycophenolate Mofetil (MMF) Dose Adjustment Based on Blood MPA Concentration to Standard Care Treatment With MMF in Renal Transplant Recipients Receiving Tacrolimus[NCT00737659]Phase 4138 participants (Anticipated)Interventional2008-08-31Recruiting
Allograft of Hematopoietic Stem Cells With Reduced-intensity Conditioning From a HLA-haploidentical Family Donor: Phase II Study of Combined Immunosuppression Before and After Transplantation[NCT00740467]Phase 250 participants (Anticipated)Interventional2008-01-31Recruiting
A Two Step Approach to Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies[NCT02566304]Phase 235 participants (Anticipated)Interventional2015-11-13Active, not recruiting
The Efficacy and Safety of Bone Marrow-derived Mesenchymal Stem Cells in Kidney Transplantation From Chinese Donation After Citizen Death (DCD): A Multi-center Randomized Controlled Trial[NCT02561767]Phase 1/Phase 2120 participants (Anticipated)Interventional2015-10-31Not yet recruiting
Phase II Pilot Cohort Study to Investigate the Safety and Efficacy of Infliximab as Additional Therapy in the Treatment if Anti-Neutrophil Cytoplasm Antibody Associated Vasculitis[NCT00753103]Phase 237 participants (Actual)Interventional2003-01-31Completed
Patient Reported Outcomes in Renal Transplant Patients Tolerating GI Symptoms Converted to Myfortic (EC-MPS).[NCT00676221]Phase 4110 participants (Anticipated)Interventional2006-07-31Active, not recruiting
A Multicenter, Randomized, Double Blind, Double Dummy Controlled Study to Assess the Tolerability of an Increased Dose of Enteric Coated MPA After Conversion From MMF in Renal Transplant Recipients Who Required MMF Dose Reductions Due to Gastrointestinal [NCT00658333]Phase 430 participants (Actual)Interventional2008-03-31Terminated
A Randomised, Multicenter, Open-label Study Evaluating the Impact on the Fibrosis at Week 52 of an Early Biopsy at (D10) Versus Standard Management in Patients Who Have Undergone de Novo Renal Transplantation and Received a Marginal Organ Transplant[NCT00817687]Phase 466 participants (Actual)Interventional2009-01-31Completed
A Randomised, Double-Blind, Placebo Controlled, Parallel-Group, Multicenter Study to Evaluate the Efficacy and Safety of Two Doses of Ocrelizumab in Patients With WHO or ISN Class III or IV Nephritis Due to Systemic Lupus Erythematosus[NCT00626197]Phase 3381 participants (Actual)Interventional2008-02-15Terminated(stopped due to Study was terminated due to an imbalance of serious and opportunistic infections in the ocrelizumab treated patients versus the placebo arm.)
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies-Increasing GVT Effects Without Increasing Toxicity[NCT03032783]Phase 263 participants (Anticipated)Interventional2017-01-31Recruiting
Reduced Intensity Stem Cell Transplantation (RIST) for Patients With Hematological Malignancies Conditioned With Fludarabine and Busulfan[NCT00619645]Phase 28 participants (Actual)Interventional2007-06-30Completed
A Multicenter Pilot Study of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation With In-vivo T-cell Depletion to Evaluate the Role of NK Cells and KIR Mis-matches in Relapsed or Refractory High-risk Neuroblastoma.[NCT00874315]Phase 20 participants (Actual)Interventional2008-09-30Withdrawn(stopped due to lack of accrual)
Post-Transplant Bendamustine (PT-BEN) for GVHD Prophylaxis[NCT04022239]Phase 1/Phase 240 participants (Anticipated)Interventional2020-03-13Recruiting
Very Low-dose Total Body Irradiation in Combination With Total Lymphoid Irradiation and Anti-Thymocyte Globulin to Improve Donor Engraftment in Patients Undergoing Non-Myeloablative Hematopoietic Cell Transplantation[NCT03734601]Phase 222 participants (Actual)Interventional2018-11-05Completed
A Phase 1/2a, Open-Label, Multicenter, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravenous Administration of RGI-2001 in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (AHSCT)[NCT01379209]Phase 1/Phase 268 participants (Actual)Interventional2011-09-30Completed
Infusion of Off-the-Shelf Ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells to Augment Single or Double Myeloablative Cord Blood Transplantation in Patients With Hematologic Malignancies[NCT01175785]Phase 215 participants (Actual)Interventional2010-08-31Completed
Islet Transplantation in Type I Diabetes With LEA29Y (Belatacept) Maintenance Therapy (CIT-04)[NCT00468403]Phase 210 participants (Actual)Interventional2008-10-31Completed
Tacrolimus and Mycophenolate Mofetil as Post-Grafting Immunosuppression After Conditioning With Fludarabine and Low-Dose Total Body Irradiation for Recipients of HLA-Matched or Mis-Matched Family or Unrelated Donor HCT[NCT02178683]Phase 350 participants (Anticipated)Interventional2010-11-30Recruiting
Multi-center, Open-label, Randomized Controlled Phase 4 Study to Evaluate the Efficacy and Safety of CertiroBell® Compared With Mycophenolate Mofetil in Primary Living Donor Liver Transplant Recipients.[NCT04471441]Phase 4150 participants (Anticipated)Interventional2020-06-30Recruiting
A Multicenter, Randomized, Double-blind, Placebo-controlled TRial Evaluating Immunosuppressive Treatment in Patients With Chronic Virus-Negative Inflammatory cardiomyopaThY (TRINITY Trial)[NCT05570409]Phase 2/Phase 3130 participants (Anticipated)Interventional2023-03-28Recruiting
Allogeneic Stem Cell Transplantation With Alternative Donor in Treatment of Hematologic Malignancy[NCT02487069]876 participants (Actual)Interventional2015-06-30Completed
A Phase II Study of Cytokine Induced Memory-like NK Cell Adoptive Therapy After Haploidentical Donor Hematopoietic Cell Transplantation[NCT02782546]Phase 260 participants (Anticipated)Interventional2017-01-30Recruiting
A Prospective, Multi-center, Single-arm, Interventional Study of Thymoglobuline® Induction Therapy in Adult Recipients of Donated After Cardiac Death Kidney Transplant in China[NCT03099122]Phase 4115 participants (Actual)Interventional2017-08-16Completed
Belatacept for the Management of Moderately Sensitized Patients at Risk for Delayed Graft Function (DGF)[NCT02130817]Phase 40 participants (Actual)Interventional2014-09-24Withdrawn(stopped due to Organ transplant criteria made recruitment difficult. Closed with IRB 10/09/2015.)
A Prospective, Randomized, Controlled Pilot Study of Early-Use Long Acting Tacrolimus (Envarsus XR) in Lung Transplant Recipients[NCT04469842]Early Phase 148 participants (Anticipated)Interventional2023-08-31Not yet recruiting
Comparisons Of Inflammatory Biomarkers And Cardiovascular Risk Scores Before And After Conversion To Full Dose Myfortic® Using Two Hour Neoral® Monitoring.[NCT02058875]Phase 40 participants (Actual)Interventional2014-02-28Withdrawn(stopped due to The study funder retracted their grant funding offer before contract signed.)
A Randomised Clinical Trial Assessing the Efficacy and Safety of Mycophenolate Mofetil Versus Azathioprine for Induction of Remission in Treatment Primary Biliary Cholangitis-Autoimmune Hepatitis Overlap Syndrome[NCT04933292]Phase 478 participants (Anticipated)Interventional2021-06-16Recruiting
Allogeneic Stem Cell Transplantation for Patients With Multiple Myeloma: a Pilot Feasibility Study Using a Novel Protocol[NCT02447055]Early Phase 10 participants (Actual)Interventional2015-12-31Withdrawn
Comparison of Safety and Efficacy of de Novo Everolimus Plus Low Dose of Cyclosporine With Standard Dose of Cyclosporine Plus Cellcept on CMV and BK Virus Infections Prevention in Renal Transplant Patients[NCT04906304]35 participants (Actual)Interventional2020-01-01Completed
Prospective, Multicenter, Randomized, Open-label, Phase 2, Lasting 12 Weeks, Evaluating the Pharmacodynamics, Efficacy and Safety of Basiliximab in de Novo Adult Renal Transplant Patients at Low Risk Receiving Either a Cumulative Dose of Basiliximab of 40[NCT01596062]Phase 216 participants (Actual)Interventional2012-03-31Completed
Mycophenolate Sodium (Myfortic®) in the Treatment of Corticosteroid-refractory Autoimmune Uveitis:Pilot Study[NCT01261169]40 participants (Anticipated)Observational2009-01-31Recruiting
Treg Adoptive Therapy in Subclinical Inflammation in Kidney Transplantation (CTOT-21)[NCT02711826]Phase 1/Phase 214 participants (Anticipated)Interventional2016-09-20Completed
Scleroderma Lung Study III (SLS III): Combining the Anti-fibrotic Effects of Pirfenidone (PFD) With Mycophenolate (MMF) for Treating Scleroderma-related Interstitial Lung Disease[NCT03221257]Phase 251 participants (Actual)Interventional2017-11-28Completed
Maintaining or Stopping Immunosuppressive Therapy in Patients With ANCA Vasculitis and End-stage Renal Disease: a Prospective, Multicenter, Randomized, Open-label, Clinical Trial[NCT03323476]Phase 3136 participants (Anticipated)Interventional2018-02-02Recruiting
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major[NCT03171831]Phase 430 participants (Anticipated)Interventional2017-04-01Recruiting
Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression[NCT03480360]Phase 320 participants (Anticipated)Interventional2018-03-28Active, not recruiting
Multicenter Uveitis Steroid Treatment (MUST) Trial[NCT00132691]Phase 4255 participants (Actual)Interventional2005-09-30Completed
Impact of Cyclosporine or Steroid Withdrawal at 3 Months Post Transplantation on Graft Function, Patient Survival and Cardiovascular Surrogate Markers the First 5 Years After Renal Transplantation.[NCT00903188]Phase 4152 participants (Anticipated)Interventional2008-10-31Recruiting
Phase 4, Randomized, Open-label, Comparative, Multicenter Study to Assess the Safety and Efficacy of Induction Agents, Alemtuzumab, Basiliximab or Rabbit Anti-thymocyte Globulin in Combination With Tacrolimus, MMF, and a Rapid Steroid Withdrawal in Renal [NCT00113269]Phase 4501 participants (Actual)Interventional2005-05-31Completed
Phase-I/II Trial to Assess the Safety and Efficacy of Venetoclax in Addition to Sequential Conditioning With Fludarabine / Amsacrine / Ara-C (FLAMSA) + Treosulfan for Allogeneic Blood Stem Cell Transplantation in Patients With MDS, CMML or sAML (FLAMSACla[NCT05807932]Phase 1/Phase 238 participants (Anticipated)Interventional2023-06-26Recruiting
Phase I/II Study to Reduce Post-transplantation Cyclophosphamide Dosing for Older or Unfit Patients Undergoing Bone Marrow Transplantation for Hematologic Malignancies[NCT04959175]Phase 1/Phase 2320 participants (Anticipated)Interventional2021-09-23Recruiting
A Reduced Intensity Conditioning Regimen With CD3-Depleted Hematopoietic Stem Cells to Improve Survival for Patients With Hematologic Malignancies Undergoing Haploidentical Stem Cell Transplantation[NCT00566696]Phase 273 participants (Actual)Interventional2007-12-14Completed
Controlled, Randomized, Parallel Group Study to Assess Efficacy and Tolerability of Full Dose Enteric-coated Mycophenolate Sodium, in Addition to Cyclosporine for Microemulsion Reduced Dose, in Maintenance Renal Transplant Recipients[NCT00434590]Phase 410 participants (Actual)Interventional2007-03-31Terminated(stopped due to The study has been stopped because of the lack of enrollment)
A Phase II Multicenter Trial to Assess the Safety and Efficacy of Campath-1H and Tacrolimus Followed By Immunosuppression Withdrawal in Liver Transplantation (ITN024ST)[NCT00105235]Phase 227 participants (Actual)Interventional2005-06-30Completed
A Phase II, Randomized Study to Evaluate the Safety and Efficacy of Ex-Vivo Cultured Allogenic Mesenchymal Stem Cells For the Treatment of Extensive Chronic Graft Versus Host Disease[NCT00972660]Phase 252 participants (Anticipated)Interventional2009-09-30Enrolling by invitation
A Prospective Multicenter Open-label Randomized Study to Assess Efficacy and Safety of Everolimus in Combination With Cyclosporine Microemulsion Versus Everolimus Without Calcineurine Inhibitor in Combination With Enteric-coated Mycophenolate Sodium (EC-M[NCT00425308]Phase 330 participants (Actual)Interventional2006-10-31Completed
A Multicenter Randomized Open Label Study to Assess Efficacy and Safety of a Steroid Avoidance Regimen in Comparison to a Treatment With Steroids, in Combination With Enteric-coated Mycophenolate Sodium (EC-MPS) 2.16 g/d for 6 Weeks and Cyclosporine Micro[NCT00413920]Phase 3222 participants (Actual)Interventional2007-04-30Completed
Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Liver Transplant Recipients (MyLiver)[NCT00405652]Phase 334 participants (Actual)Interventional2007-01-31Completed
A Randomized, Multicenter, Parallel-group, Open-label Study to Evaluate the Therapeutic Benefit of an Initially Intensified Dosing Regimen of Mycophenolate Sodium Versus a Standard Dosing Regimen, in Combination With Cyclosporine and Corticosteroids in de[NCT00419926]Phase 4313 participants (Actual)Interventional2006-12-31Completed
A Randomised, Double-Blind, Placebo Controlled, Parallel-Group, Multicenter Study to Evaluate the Efficacy and Safety of Two Doses of Ocrelizumab in Patients With Active Systemic Lupus Erythematosus[NCT00539838]Phase 333 participants (Actual)Interventional2007-12-19Terminated(stopped due to The study was terminated prematurely when the decision was made that ocrelizumab was not likely to benefit this patient population.)
A Randomized, Open Label Study Comparing the Effect of CellCept With Therapeutic Drug Monitoring, Tacrolimus and a Corticosteroid-sparing Regimen Versus Fixed Dose CellCept, Tacrolimus and Corticosteroids Maintained up to 6 Months, on Acute Rejection and [NCT00545402]Phase 4180 participants (Actual)Interventional2007-11-30Completed
Efficacy and Safety of Anti-cytomegalovirus Prophylaxis Versus Pre-emptive Approaches With Valganciclovir in Heart Transplant Recipients Treated With Everolimus or Mycophenolate. A Randomized Open-label Study for Prevention of Cardiaca Allograft Vasculopa[NCT00966836]Phase 3100 participants (Anticipated)Interventional2009-04-30Recruiting
Multi-center, Open Label, Randomized Trial Comparing Single Versus Double Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia (BMT CTN #0501)[NCT00412360]Phase 3224 participants (Actual)Interventional2006-12-31Completed
Phase I Feasibility Study of Clofarabine and Low Dose Total Body Irradiation (TBI) as a Non-myeloablative Preparative Regimen for Stem Cell Transplantation (SCT) for Hematologic Malignancies[NCT01041508]Phase 136 participants (Anticipated)Interventional2010-02-28Completed
Open Randomized Multi-Center Study to Evaluate Safety and Efficacy of Low Molecular Weight Sulfated Dextran (LMW-SD) in Islet Transplantation After Kidney Transplantation (CIT-01B)[NCT00790439]Phase 20 participants (Actual)Interventional2008-07-31Withdrawn(stopped due to Due to funding limitations)
Randomized, Double-blind, Double-dummy Trial of Mycophenolic Acid Versus Azathioprine in the Treatment of Corticosteroid-refractory Myasthenia Gravis[NCT00997412]40 participants (Anticipated)Interventional2009-05-31Active, not recruiting
Phase I Study of CellCept for Advanced Pancreatic Cancer[NCT00997958]Phase 112 participants (Actual)Interventional2004-06-30Completed
Pilot, Single-arm, Non-comparative, Open-label Study of Daclizumab in Combination With Mycophenolate Mofetil and Sirolimus in the Prevention of Acute Rejection in Cardiac Allografts Recipients in Risk of Deteriorated Renal Function[NCT02554955]Phase 436 participants (Actual)Interventional2004-02-29Completed
A Randomized, Open-label Study of the Achievement of a Mycophenolic Acid Therapeutic Window During Treatment With 2 Dosing Regimens of Oral CellCept Administered as a Component of Standard Immunosuppressive Therapy in Patients With Kidney Transplants[NCT00788567]Phase 3136 participants (Actual)Interventional2005-06-30Completed
Open, Single Center, Randomised, Parallel Group Pilot Study to Investigate a Calcineurin Free Immunosuppressive Treatment for de Novo Renal Transplant Recipients: A Comparison of a Rapamycin/MMF/Steroids Regime to a Cyclosporine A Neoral/MMF/Steroids Regi[NCT00812123]Phase 4127 participants (Actual)Interventional2001-01-31Completed
A Randomized, Open-Label, Multicenter, Parallel-Group Study of Belatacept (BMS-224818)-Based Corticosteroid-Free Regimens in Renal Transplant[NCT00455013]Phase 293 participants (Actual)Interventional2007-07-31Completed
Nordic Everolimus (Certican) Trial in Heart and Lung Transplantation: Results at 24 Months[NCT00377962]Phase 4282 participants (Actual)Interventional2005-12-31Completed
A Prospective, Randomised, Double-blind, Placebo-controlled Trial Evaluating the Effects of Mycophenolate Mofetil on 'Surrogate Markers' for Atherosclerosis in Female Patients With SLE.[NCT01101802]Phase 471 participants (Actual)Interventional2006-03-31Completed
Comparative Clinical Study of Reduced Dose of Tacrolimus and Standard Dose of Mycophenolate Mofetil vs. Conventional Dose of Tacrolimus and Reduced Dose of Mycophenolate Mofetil in De Novo Renal Transplant Recipients[NCT03968588]Phase 4108 participants (Actual)Interventional2014-07-29Completed
Pilot Study Using Donor Stem Cells and Campath-1H to Induce Renal Transplant Tolerance (ITN022ST)[NCT00183248]Phase 1/Phase 29 participants (Actual)Interventional2004-09-30Completed
A Multi-center Phase III Study Comparing Nonmyeloablative Conditioning With TBI Versus Fludarabine/TBI for HLA-matched Related Hematopoietic Cell Transplantation for Treatment of Hematologic Malignancies[NCT00075478]Phase 387 participants (Actual)Interventional2003-10-31Completed
A Multicenter Pilot Study to Determine the Pharmacokinetics of Astagraf XL, Prograf and Mycophenolate Mofetil in Renal Transplant Candidates Who Have Undergone Laparoscopic Sleeve Gastrectomy[NCT02221583]Phase 426 participants (Actual)Interventional2014-05-31Completed
Open-label Prospective Randomized Study to Determine the Efficacy and Safety of Two Dosing Regimens of ACTHar in the Treatment of Proliferative Lupus Nephritis.[NCT02226341]Phase 420 participants (Anticipated)Interventional2014-10-31Recruiting
A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Atacicept in Subjects With Lupus Nephritis in Combination With Mycophenolate Mofetil Therapy.[NCT00573157]Phase 2/Phase 36 participants (Actual)Interventional2007-12-31Terminated(stopped due to The study was terminated due to unanticipated safety issues)
Prospective Donor-specific Cellular Alloresponse Assessment for Immunosuppression Minimization in de Novo Renal Transplantation[NCT02540395]184 participants (Actual)Interventional2015-03-31Completed
Targeting Inflammation and Alloimmunity in Heart Transplant Recipients With Tocilizumab (RTB-004)[NCT03644667]Phase 2200 participants (Anticipated)Interventional2018-12-20Recruiting
HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen[NCT02776202]Phase 215 participants (Anticipated)Interventional2016-05-31Recruiting
Minimizing Glucocorticoid Administration in Patients With Proliferative Lupus Nephritis During the Induction of Remission Period-EUROLUPUS vs. RITUXILUP Regimen: A Randomized Study[NCT05207358]Phase 430 participants (Anticipated)Interventional2022-03-02Recruiting
Phase IV Study of Enteric-coated Mycophenolate Sodium in Combination With Tacrolimus in Renal Transplant Patient[NCT00646737]Phase 418 participants (Actual)Interventional2008-05-31Completed
Pilot Safety and Feasibility Trial of Mycophenolate and Sirolimus for Prevention of GVHD in Mismatched Unrelated and Related Donor Hematopoietic Stem Cell Transplantation for Hematologic Malignancies[NCT02728700]Phase 11 participants (Actual)Interventional2016-02-29Terminated(stopped due to Accrual factor)
Sequential, Related Donor Partial Liver Transplantation Followed by Bone Marrow Transplantation for Fibrolamellar or Non-fibrolamellar Hepatocellular Carcinoma (HCC) Including Fibrolamellar HCC[NCT02702960]Phase 20 participants (Actual)Interventional2016-03-31Withdrawn(stopped due to This study was withdrawn due to lack of necessary resources from the liver transplant surgical group.)
The Study of Immunotherapy With Rituximab and Pulse Dexamethasone Followed by With Mycophenolate Mofetil or Placebo in Adult Patients With Persistent and Chronic Immune Thrombocytopenia[NCT02649504]Phase 30 participants (Actual)Interventional2016-12-01Withdrawn
Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients[NCT02809079]Phase 4100 participants (Anticipated)Interventional2016-01-31Enrolling by invitation
Validation of Population Pharmacokinetic Model Derived From Healthy Volunteer in Kidney Transplant Recipients[NCT02808065]40 participants (Anticipated)Observational2016-05-31Recruiting
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study[NCT04009525]Phase 4800 participants (Anticipated)Interventional2019-06-01Recruiting
An Open, Multicentre, Randomised, Parallel Group Pilot-Study to Compare Safety and Efficacy of Discontinuation of Mycophenolate Mofetil From a Tacrolimus/MMF/Steroid Triple Regimen Following Kidney Transplantation[NCT00693381]Phase 3152 participants (Actual)Interventional2003-02-28Completed
A Prospective, Randomized, Multi-Center Double-Blind Study of Early Corticosteroid Cessation vs. Long Term Corticosteroid Therapy With Prograf and CellCept in Primary Renal Transplant Patients[NCT00650468]Phase 4397 participants (Actual)Interventional1999-11-30Completed
A Randomized Trial for the Treatment of Relapsing Aplastic Anemia With Mycophenolate Mofetil (MMF) and Cyclosporine (CSA)[NCT00005935]Phase 2130 participants Interventional2000-06-30Completed
A Multi-Institutional Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Malignant Neuro-Epithelial and Other Solid Tumors[NCT02653196]Early Phase 11 participants (Actual)Interventional2015-09-30Terminated(stopped due to The Principal Investigator left the institution.)
Identification of Drug-drug Interaction Between Tacrolimus and Mycophenolate Mofetil in Healthy Adults[NCT02743247]Phase 118 participants (Actual)Interventional2015-10-31Completed
Haploidentical Hematopoietic Stem Cell Transplantation Using A Novel Clofarabine Containing Conditioning Regimen For Patients With Refractory Hematologic Malignancies[NCT00824135]Phase 134 participants (Actual)Interventional2009-01-31Completed
Everolimus and Mycophenolate Sodium as GvHD Prophylaxis in Allogeneic Stem Cell Transplantation[NCT00856505]Phase 1/Phase 238 participants (Anticipated)Interventional2008-03-31Recruiting
Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Treosulfan Based Preparative Regimen[NCT00796068]Phase 2130 participants (Actual)Interventional2009-02-24Completed
An Open-label, Single-sequence Study to Investigate the Effects of BMS-986256 at Steady State on the Single Dose Pharmacokinetics of Mycophenolate Mofetil in Healthy Male Participants[NCT04039373]Phase 115 participants (Actual)Interventional2019-07-22Completed
Optimized Cord Blood Transplantation for the Treatment of High-Risk Hematologic Malignancies in Adults and Pediatrics[NCT06013423]Phase 254 participants (Anticipated)Interventional2024-02-06Not yet recruiting
A Phase II/III Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Abatacept Versus Placebo on a Background of Mycophenolate Mofetil and Glucocorticosteroids in Subjects With Active Proliferative Glomeru[NCT00430677]Phase 2/Phase 3423 participants (Actual)Interventional2007-06-30Terminated(stopped due to Terminated due to failure to meet the primary efficacy endpoint in the Short-term Period)
Study of the Pharmacokinetics of Mycophenolate Mofetil in Patients Who Have Undergone Orthotopic Liver Transplantation[NCT00007059]20 participants Interventional1998-06-30Completed
Transplantation of Umbilical Cord Blood for Myeloid Leukemia Patients Not in CR With Cyclophosphamide/Fludarabine/Total Body Irradiation Myeloablative Preparative Regimen and UCB NK Cells[NCT00354172]Phase 216 participants (Actual)Interventional2006-02-28Terminated(stopped due to Competing study was started.)
Multi-center, Open-label, Prospective, Randomized, Parallel Group, Long-term Study Investigating a Standard Regimen in de Novo Kidney Transplant Patients Versus a CNI-free Regimen and a CNI-low Dose Regimen[NCT00514514]Phase 3802 participants (Actual)Interventional2007-07-31Completed
A Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf® (Tacrolimus)/ Myfortic® and Advagraf® (Extended Release Tacrolimus) / Myfortic® in de Novo Liver Transplant Recipients[NCT01018914]Phase 444 participants (Actual)Interventional2009-04-30Completed
Gastrointestinal Adverse Effect Outcomes of De Novo African American Kidney Transplant Recipients Treated With Tacrolimus, Corticosteroids and Mycophenolate Mofetil or Enteric Coated Mycophenolate Sodium[NCT00522548]Phase 437 participants (Actual)Interventional2007-03-31Terminated(stopped due to enrollment halted in order to have all patients complete follow-up by Jan 2011)
A 24-month, Multi-center, Randomized, Open-label, Non-inferiority Study of Efficacy and Safety Comparing Two Exposures of Concentration-controlled Everolimus With Reduced Cyclosporine Versus 3.0 g Mycophenolate Mofetil With Standard Dose Cyclosporine in d[NCT00300274]Phase 3721 participants (Actual)Interventional2006-01-31Completed
Mycophenolate Mofetil (MMF) ,Carnitine and Phosphodiesterase Type 5 Inhibitor, Three Potential Treatments for Resistant Proteinuria and for Slowing the Deterioration of Diabetic Nephropathy in Patients With Type II Diabetes Mellitus[NCT01566006]80 participants (Anticipated)Interventional2012-04-30Not yet recruiting
Sirolimus, Mycophenolate Mofetil and Bortezomib as Graft-Versus-Host Disease Prophylaxis After Non-Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation[NCT00548717]Phase 215 participants (Actual)Interventional2007-10-31Terminated(stopped due to First two patients enrolled after trial reopened, developed grade III-IV acute GVHD and subsequently passed away.)
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Haematological Diseases Using a Reduced Intensity Conditioning Regimen[NCT00959231]Phase 260 participants (Anticipated)Interventional2009-01-31Recruiting
Infusion of Expanded Cord Blood T Cells Following Cord Blood Transplantation[NCT00972101]Phase 10 participants (Actual)Interventional2009-09-30Withdrawn(stopped due to Development of other studies led to termination without recruitment.)
Hematopoietic Cell Transplantation for Patients With High-Risk Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), or Myelodysplastic Syndrome (MDS) Using Radiolabeled DOTA-Biotin Pretargeted by BC8 Antibody-Streptavidin Conjugate[NCT00988715]Phase 117 participants (Actual)Interventional2010-04-21Completed
Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation[NCT00506948]Phase 213 participants (Actual)Interventional2006-09-30Terminated(stopped due to Halted due to high incidence of veno-oclusive disease of the liver.)
A Prospective, Randomized, Active Controlled, Parallel Group, Multi-center Trial to Assess the Efficacy and Safety of Mycophenolate Mofetil (MMF) in Inducing Response and Maintaining Remission in Subjects With Lupus Nephritis.[NCT00377637]Phase 3370 participants (Actual)Interventional2005-07-31Completed
Rheumatoid Arthritis: Tolerance Induction by Mixed Chimerism[NCT00282412]Phase 14 participants (Actual)Interventional2002-09-30Terminated(stopped due to No participant enrolled for three years. No plan to continue study.)
A Phase 2, Randomized, Open-Label, Parallel Group, Multi-Center Study to Assess the Safety and Efficacy of Alefacept in de Novo Kidney Transplant Recipients[NCT00543569]Phase 2323 participants (Actual)Interventional2008-02-29Completed
Autologous Followed by Non-myeloablative Allogeneic Transplantation for Non-Hodgkin's Lymphoma[NCT00481832]Phase 250 participants (Actual)Interventional2007-01-31Terminated(stopped due to Accrual Factor)
Research Institute of Nephrology, Jinling Hospital,[NCT01056237]206 participants (Actual)Interventional2010-02-28Completed
Open Label Balanced Randomized Two-treatment Two-period Two-sequence Single Dose Two-way Crossover Oral Bioequivalence Study of Mycophenolate Mofetil Capsules 250 mg in Normal Healthy Adult Human Subjects Under Fasting Conditions[NCT01080443]56 participants (Actual)Interventional2008-06-30Completed
SIMPLE Study: A Prospective and Randomized Trial of a Simplified Immunosuppressive Protocol Utilizing Low Dose EnvarsusXR[NCT04773392]Phase 480 participants (Anticipated)Interventional2021-11-23Recruiting
Combination Therapy Using Mycophenolate Mofetil (CellCept) and Human Interferon beta1a (Rebif) in Early Treatment of Multiple Sclerosis[NCT00618527]Early Phase 131 participants (Actual)Interventional2006-08-31Completed
A Two-Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Advanced Cutaneous T Cell Lymphoma[NCT02548468]Phase 10 participants (Actual)Interventional2015-11-20Withdrawn(stopped due to Slow accrual)
An Open, Randomized, Multicentre Clinical Study to Compare the Safety and Efficacy of a Combination of Sequential Therapy of Tacrolimus (FK506) With Monoclonal Anti-IL2R Antibodies and Mycophenolate Mofetil Versus Tacrolimus (FK506) With Steroids in Liver[NCT00693524]Phase 294 participants (Actual)Interventional2002-11-30Completed
A Multicenter, Three Arm, Randomized, Open Label Clinical Study to Compare Renal Function in Liver Transplant Recipients Receiving an Immunosuppressive Regimen of Advagraf (Immediately or Delayed Post-transplant) and MMF With or Without a Monoclonal Anti-[NCT01011205]Phase 3893 participants (Actual)Interventional2009-09-30Completed
Randomized Trial of GVHD Prophylasxis With Post-transplantation Cyclophocphomide (PTCy) or Thymoglobulin in Unrelated SCT Recepients With Chronic Myeloproliferative Neoplasms and Myelodisplatic Syndrome[NCT02627573]Phase 232 participants (Actual)Interventional2015-07-31Terminated(stopped due to Poor recruitment)
Induction of Mixed Hemopoietic Chimerism in Patients Using Fludarabine, Low Dose TBI, PBSC Infusion and Post Transplant in Immunosuppression w/CSA & Mycophenolate Mofetil[NCT00531635]Phase 1350 participants (Anticipated)Interventional2002-07-31Recruiting
A Prospective, Multicenter, Randomized Controlled Trial of Mycophenolate Mofetil (MMF) in Patients With IgA Nephropathy (IgAN)[NCT00657059]Phase 3151 participants (Actual)Interventional2007-09-30Completed
A Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multicenter, 36-Week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Maintain or Improve Symptom Control With Reduced Corticosteroid in Subjects[NCT00683969]Phase 3136 participants (Anticipated)Interventional2004-08-31Completed
The Use of Thymoglobulin in a Calcineurin Inhibitor and Steroid Minimization Protocol[NCT00706680]Phase 430 participants (Anticipated)Interventional2008-02-29Recruiting
Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients[NCT04101578]2,000 participants (Anticipated)Observational2017-02-08Recruiting
Pharmacokinetic Profile of Myfortic (Enteric Coated Mycophenolate Sodium) in a Rapid Steroid Withdrawal Protocol in Combination With Tacrolimus in Stable Renal Transplant Recipients in the Fed and Fasting State[NCT00585468]Phase 421 participants (Actual)Interventional2007-12-31Completed
Gastrointestinal Mucosal Findings in Patients Receiving Mycophenolic Acid (MPA) as Demonstrated by Small Bowel Capsule Endoscopy (SBCE)[NCT00652834]Phase 423 participants (Actual)Interventional2009-04-30Completed
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Hematologic Malignancies[NCT01760655]Phase 262 participants (Actual)Interventional2012-12-24Completed
A Multi-center, Randomized, Open-label, Parallel Group Study Investigating the Renal Tolerability, Efficacy and Safety of a CNI-free Regimen (Everolimus and MPA) Versus a CNI-regimen With Everolimus in Heart Transplant Recipients[NCT00862979]Phase 4162 participants (Actual)Interventional2009-02-24Completed
The Highly Sensitized Patients: Effects of Rituximab and Mycophenolate Mofetil (MMF) On Anti-Human Leukocyte Antigen (HLA) Antibody Levels In Patients Awaiting Cadaveric Renal Transplant.[NCT00446251]Phase 214 participants (Actual)Interventional2006-12-31Completed
Reduced Intensity Conditioning Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies at High Risk for Transplant Related Mortality With Standard Transplantation[NCT00795132]Phase 247 participants (Actual)Interventional2004-04-30Completed
A Randomized, Open Label Study Comparing the Effect of CellCept Combined With 2 Regimens of Reduced Calcineurin Inhibitors on Kidney Function in Liver Transplant Patients[NCT00717314]Phase 487 participants (Actual)Interventional2008-05-31Completed
A Phase 2 Study in Poor Risk Diffuse Large B-cell Lymphoma of Total Lymphoid Irradiation & Antithymocyte Globulin Followed by Matched Allogeneic Hematopoietic Transplantation as Consolidation to Autologous Hematopoietic Cell Transplantation[NCT00482053]Phase 23 participants (Actual)Interventional2006-10-31Terminated(stopped due to Low accrual)
Sirolimus and Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation[NCT01220297]Phase 23 participants (Actual)Interventional2006-08-31Terminated(stopped due to Low accrual)
Three-arm Clinical Trial for Patients With Hematologic Malignancies and Mismatched Donors - Haploidentical, 1 Antigen Mismatch Related or Unrelated, and Matched Unrelated Donor (MUD)- Using a T-cell Replete Allograft and High-dose Post-transplant Cyclopho[NCT01010217]Phase 2176 participants (Actual)Interventional2009-11-05Completed
Post Transplant Cyclophosphamide for Unrelated and Related Allogeneic Hematopoietic Stem Cell Transplantation for Hematological Malignancies[NCT01349101]Phase 280 participants (Actual)Interventional2011-02-10Completed
Optimum Immunosuppression in Renal Transplant Recipients at High Risk of Developing New Onset Diabetes After Transplantation: A Multicenter, Prospective, Controlled and Randomized Trial.[NCT01002339]Phase 4134 participants (Actual)Interventional2010-02-28Terminated(stopped due to Terminated: higher rate of acute rejection in the Cyclosporin A group)
Low-Dose TBI and Fludarabine Followed by Nonmyeloablative Unrelated Donor Peripheral Blood Stem Cell Transplantation Using Enhanced Postgrafting Immunosuppression for Patients With Hematologic Malignancies and Renal Cell Carcinoma - A Multi-center Trial[NCT00027820]Phase 1/Phase 2106 participants (Actual)Interventional2001-08-31Completed
A Multicenter, Four Arm, Randomized, Open Label Clinical Study Investigating Optimized Dosing in a Prograf®-/Advagraf®-Based Immunosuppressive Regimen in Kidney Transplant Subjects (OSAKA Study)[NCT00717470]Phase 41,252 participants (Actual)Interventional2008-05-14Completed
Mycophenolate Mofetil Maintenance Therapy for Liver Transplantation Following Campath-1H Induction[NCT00849238]0 participants (Actual)InterventionalWithdrawn(stopped due to feasibility issues)
A Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf (Tacrolimus)/MMF and Extended Release (XL) Tacrolimus /MMF and Steroid Withdraw in de Novo Liver Transplant Recipients.[NCT00720408]Phase 348 participants (Actual)Interventional2007-12-31Completed
Triple Arm, Prospectively Randomized Multi Centre Study Phase IV to Evaluate Calcineurin Inhibitor Reduced, Steroid Free Immunosuppression After Renal Transplantation in Non-risk Patients[NCT00724022]Phase 4600 participants (Anticipated)Interventional2008-06-30Completed
Mycophenolate vs. Oral Cyclophosphamide in Scleroderma Interstitial Lung Disease (Scleroderma Lung Study II)[NCT00883129]Phase 2142 participants (Actual)Interventional2009-09-30Completed
A Phase II/III, Randomized, Open-Label, Active Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of Efalizumab Compared With Cyclosporine, Both in Combination With Mycophenolate Mofetil and Corticosteroids, As an Immunosuppressant Regimen [NCT00729768]Phase 2/Phase 30 participants (Actual)InterventionalWithdrawn
A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies[NCT05589896]Phase 1/Phase 212 participants (Anticipated)Interventional2023-11-30Recruiting
A Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic Sclerosis[NCT01413100]Phase 221 participants (Actual)Interventional2011-09-15Active, not recruiting
Randomized, Prospective Single-center Study Comparing a Rapid Discontinuation of Corticosteroids (Steroid Withdrawal) With Corticosteroid Therapy in Kidney Transplantation Using Mycophenolate Mofetil and Tacrolimus Maintenance Therapy[NCT00596947]Phase 418 participants (Actual)Interventional2005-10-31Terminated(stopped due to due to low study enrollment)
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 250 mg Capsules Under Fasting Conditions[NCT00893542]37 participants (Actual)Interventional2005-11-30Completed
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 500 mg Tablets Under Fasting Conditions[NCT00894088]38 participants (Actual)Interventional2006-01-31Completed
A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies[NCT02793544]Phase 280 participants (Actual)Interventional2016-12-31Completed
Patient Reported Outcomes in Renal Transplant Patients With and Without Gastrointestinal Symptoms[NCT00529269]Phase 4200 participants (Anticipated)Interventional2006-12-31Completed
A Single Centre, Prospective, Open-Label, Parallel Group, Randomized Study to Compare the Gastrointestinal Tolerability of Mycophenolate Mofetil (MMF, CellCept) and Enteric-Coated Mycophenolate Sodium (EC-MPS, Myfortic) in Maintenance Transplant Patients [NCT00611494]Phase 4400 participants (Anticipated)Interventional2008-01-31Recruiting
Ph 2, Double-Masked, Randomized, Parallel, Sham Surgery/Placebo Control, Multi-Center Study to Evaluate Systemic IMT Regimens as Graft Rejection Prophylaxis Following Transplantation of hESC Derived RPE Cells in Patients With AMD[NCT02563782]Phase 20 participants (Actual)Interventional2015-08-24Withdrawn(stopped due to Changes to the study design and the cell line)
Predictors of Rejection in Pediatric Kidney Transplantation[NCT04292418]70 participants (Anticipated)Observational2020-05-01Not yet recruiting
Intensified Dosing of Cellcept in Kidney Transplantation Trial[NCT00943228]Phase 440 participants (Actual)Interventional2010-02-28Completed
A Randomized Single-center Trial of Mycophenolate Mofetil for the Prophylaxis of Graft-versus-host Disease in High Risk Allogeneic Stem Cell Transplantation[NCT00563589]30 participants (Anticipated)Interventional2003-08-31Recruiting
A Phase 2a, Randomized, Open-label, Active Control, Multi-Center Study to Assess the Efficacy and Safety of ASKP1240 in de Novo Kidney Transplant Recipients[NCT01780844]Phase 2149 participants (Actual)Interventional2013-03-05Completed
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity[NCT04339777]Phase 266 participants (Anticipated)Interventional2020-09-22Recruiting
A Therapeutic Exploratory Study to Determine the Efficacy and Safety of Calcineurin-Inhibitor-Free De-novo Immunosuppression After Liver Transplantation[NCT00890253]Phase 229 participants (Anticipated)Interventional2010-01-31Recruiting
A Prospective, Randomized, Open Label, Case-Controlled Study on the Efficacy of Mycophenolate Mofetil for IgA Nephropathy Patients With Heavy Proteinuria Despite Angiotensin Blockade[NCT00863252]Phase 440 participants (Actual)Interventional2002-03-31Completed
The Clinical Efficacy and Economic Evaluation of EC-MPS (Myfortic) in the Treatment of Relapse or Resistant Proliferative Lupus Nephritis[NCT01015456]Phase 359 participants (Actual)Interventional2010-01-31Terminated(stopped due to Data Safety Monitoring Board concerning of the participants' safety)
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 250 mg Capsules Under Fed Conditions[NCT00893919]37 participants (Actual)Interventional2005-11-30Completed
Conversion of CellCept to Myfortic: A Prospective Study on the Tolerability and Safety of Myfortic in Liver Transplant Recipients[NCT00336895]29 participants (Actual)Interventional2006-11-30Completed
Randomized, Multi-Center Comparative Trial of Tacrolimus w/Steroids and Standard Daclizumab Induction vs a Novel Steroid-Free Tacrolimus Based Immunosuppression Protocol w/ Extended Daclizumab Induction in Pediatric Renal Transplantation[NCT00141037]Phase 1/Phase 2130 participants (Actual)Interventional2004-03-31Completed
Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating a CNI-free Regimen With Enteric-coated Mycophenolate Sodium and Everolimus in Comparison to Standard Therapy With Enteric-coated Mycophenolate Sodium and Ciclosporin Mic[NCT00332839]Phase 493 participants (Actual)Interventional2005-11-30Terminated(stopped due to The trial was terminated early due to slow enrollment. It was determined that the planned sample size of 300 could not be achieved.)
Umbilical Cord Blood (UCB) Allogeneic Stem Cell Transplant for Hematologic Malignancies[NCT01093586]Phase 214 participants (Actual)Interventional2007-09-30Completed
Prospective and Randomized Study to Evaluate the Effect of Everolimus in the Clinical and Intra-Cardiac Ecography Progression of Heart Graft Vascular Illness.[NCT00695344]Phase 452 participants (Actual)Interventional2006-01-31Active, not recruiting
A Phase 2 Study of Autologous Followed by Nonmyeloablative Allogeneic Transplantation Using Total Lymphoid Irradiation (TLI) and Antithymocyte Globulin (ATG) in Multiple Myeloma Patients[NCT00899847]Phase 29 participants (Actual)Interventional2009-05-31Completed
An Open-label Study to Evaluate the Effect of Iguratimod Concomitant With Conventional Immunosuppressive Drugs on Preventing Antibody-induced Rejection in Human Leukocyte Antigen(HLA) Highly Mismatched Kidney Transplant Recipients[NCT02839941]60 participants (Anticipated)Interventional2016-08-31Recruiting
A Comparison of Effects of Standard Dose vs. Low Dose Advagraf® With IL-2 Receptor Antibody Induction, MMF and Steroids, With or Without ACEi/ARB - Based Antihypertensive Therapy on Renal Allograft Histology, Function, and Immune Response[NCT00933231]Phase 3281 participants (Actual)Interventional2009-08-17Completed
Pilot Study of Unrelated Cord Blood Transplantation in Patients With Poor Risk Haematological Malignancies[NCT00916045]Phase 240 participants (Anticipated)Interventional2009-09-30Terminated(stopped due to Recruitment issues)
Mycophenolate Mofetil and Tacrolimus vs Tacrolimus Alone for the Treatment of Idiopathic Membranous Glomerulonephritis (IMG)[NCT00843856]Phase 440 participants (Actual)Interventional2009-03-03Completed
A 12-month, Multicenter, Randomized, Open-label Study to Investigate Efficacy and Safety of Concentration Controlled Everolimus With Reduced Dose Cyclosporine A Versus Mycophenolate Mofetil With Standard Dose Cyclosporine A in de Novo Renal Transplant Adu[NCT00658320]Phase 3122 participants (Actual)Interventional2008-02-29Completed
A Randomized Multicenter Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Mycophenolate Mofetil (CellCept) for the Treatment of Refractory Interstitial Cystitis (IC)[NCT00451867]Phase 3210 participants (Actual)Interventional2007-03-31Terminated(stopped due to The major and primary reason for the study termination is the observed reduced efficacy of CellCept compared to placebo.)
Comparison of 3g Versus 2g Mycophenolate Mofetil in Combination With Tacrolimus on Progression of Chronic Histology Changes in Kidney Transplant Recipients[NCT01860183]Phase 476 participants (Actual)Interventional2013-05-31Completed
A Prospective, Randomized, Open-Label, Pilot Study To Compare The Effect On Carotid Atherosclerosis Of A Tacrolimus-Based Regimen With Conversion From A Tacrolimus- To A Sirolimus-Based Regimen At 3-4 Months Post-Transplant In De Novo Renal Transplant Rec[NCT00311311]Phase 372 participants (Actual)Interventional2006-04-30Terminated(stopped due to See termination reason in detailed description.)
Non-Myeloablative Allogeneic Bone Marrow Transplantation for Hematologic Malignancies Using Haploidentical Donors: A Phase I Trial of Pre-Transplant Cyclophosphamide[NCT00006042]Phase 10 participants Interventional1999-12-31Completed
A Scandinavian Controlled, Randomized, Open-label, and Multi-centre Study Evaluating if Once-daily Tacrolimus or Twice-daily Cyclosporin, Reduces the 3-year Incidence of Chronic Lung Allograft Dysfunction After Lung Transplantation[NCT02936505]249 participants (Actual)Interventional2016-10-12Active, not recruiting
Gemtuzumab Ozogamicin (GO), Fludarabine, And Low-Dose TBI Followed By Donor Stem Cell Transplantation For Patients With Advanced Acute Myeloid Leukemia Or Myelodysplastic Syndrome[NCT00008151]Phase 20 participants Interventional2000-10-31Completed
Non-Ablative Chemotherapeutic Conditioning Before Allogeneic Stem Cell Transplantation[NCT00008307]Phase 252 participants (Anticipated)Interventional1998-04-30Active, not recruiting
Efficacy and Safety of Alefacept in Combination With Tacrolimus, Mycophenolate Mofetil and Steroids in de Novo Kidney Transplantation - a Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study[NCT00617604]Phase 2218 participants (Actual)Interventional2007-12-31Completed
Mycophenolate Mofetil in the Treatment of Wegener's Granulomatosis and Related Vasculitides[NCT00001764]Phase 150 participants Interventional1998-04-30Completed
Evaluation of the Benefit/Risk Ratio of a Reduction of Tacrolimus Dose in Association With Mycophenolate Mofetil on the Prevention of Complications in Adult Liver Transplantation[NCT00151632]Phase 3195 participants (Actual)Interventional2003-05-31Terminated(stopped due to insufficient enrollment)
Mycophenolate Mofetil Versus Azathioprine for Maintenance Therapy of Lupus Nephritis[NCT02949349]Phase 232 participants (Actual)Interventional2015-07-31Completed
Nature of Anifrolumab Impact on Vaccine-Emergent Immunity in Patients With Moderately to Severely Active Systemic Lupus Erythematosus: A Multi-Centre Open Label Parallel Group Trial:[NCT04726553]Early Phase 120 participants (Anticipated)Interventional2021-01-20Recruiting
A Multicentre Randomised Trial of First Line Treatment Pathways for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Mycophenolate[NCT03156452]Phase 3123 participants (Actual)Interventional2017-10-25Completed
A Study of the Effect of Conversion to Enteric-Coated Mycophenolate Sodium (EC-MPS) on Quality of Life in Patients With Gastrointestinal (GI) Symptoms Related to Mycophenolate Mofetil Therapy After Kidney Transplantation (MYQOL)[NCT00239005]Phase 4134 participants (Actual)Interventional2005-09-30Completed
Conversion Trial From Mycophenolate Mofetil (MMF) to Enteric-Coated Mycophenolate Sodium (EC-MPS) in Stable Transplanted Patients Suffering From GI Adverse Events While on Mycophenolate Mofetil Therapy[NCT00149942]Phase 423 participants (Actual)Interventional2004-10-31Completed
A Pilot Study of Mycophenolate Mofetil (MMF) in Patients With p-ANCA Microscopic Polyangiitis and Mild to Moderate Renal Dysfunction.[NCT00405860]Phase 118 participants (Actual)Interventional2002-12-31Completed
Safety and Tolerability of Enteric-Coated Mycophenolate Sodium in Renal Transplant Patients With GI Intolerance[NCT00150020]Phase 4728 participants (Anticipated)Interventional2004-10-31Completed
An Open-label Study to Assess the Efficacy and Safety of Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Expressed by an Adeno-Associated Viral Vector After Intramuscular Administration in LPL-deficient Adult Subjects[NCT00891306]Phase 2/Phase 35 participants (Actual)Interventional2009-02-28Completed
An Open-Label, Randomized, Prospective Multicenter Study To Compare The Efficacy And Safety Among 3 Immunosuppressant Treatment Regimens In Patients Receiving A Liver Transplant For ESLD Caused By Chronic Hepatitis C[NCT00163657]Phase 4312 participants (Actual)Interventional2002-07-31Completed
A 3 Month, Multicenter, Open-label Study to Evaluate the Impact of the Immunosuppressive Combination of Enteric-Coated Mycophenolate Sodium (EC- MPS), Basiliximab and Cyclosporine for Microemulsion With C2 Monitoring, on Efficacy and Safety Outcomes in de[NCT00154232]Phase 446 participants Interventional2004-06-30Completed
Pilot Trial for Implementation of a Medroxyprogesterone(MPA)Pharmacokinetic(PK) Monitoring Strategy in Patients on Mycophenolate Mofetil(MMF)/FK Based Immunosuppression.[NCT00187941]Early Phase 122 participants (Actual)Interventional2005-08-31Completed
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 500 mg Tablets Under Fed Conditions[NCT00893958]39 participants (Actual)Interventional2006-01-31Completed
A 36 Month Multi-center, Open Label, Randomized, Comparator Study to Evaluate the Efficacy and Safety of Everolimus Immunosuppression Treatment in Liver Transplantation for Hepatocellular Carcinoma Exceeding Milan Criteria[NCT02081755]Phase 4336 participants (Anticipated)Interventional2014-03-31Active, not recruiting
Prospective Cohort Study of Clinical and Imaging Patterns of Neuroinflammation Diseases (CLUE)[NCT04106830]1,000 participants (Anticipated)Observational [Patient Registry]2019-01-01Recruiting
Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Non-Myeloablative Preparative Regimen[NCT00719849]Phase 213 participants (Actual)Interventional2005-11-30Terminated(stopped due to A new protocol was developed to replace this protocol in 2008, with removal of ATG and extension of MMF duration.)
A Phase II Exploratory Study to Determine the Safety and Study the Immunomodulatory Functions of Induction Therapy With Campath, Combined With Chronic Immunosuppression With Mycophenolate Mofetil and Sirolimus[NCT00240994]Phase 235 participants (Actual)Interventional2005-01-31Completed
A Randomized, Open-label Study to Compare the Effect of CellCept Plus Corticosteroids, and Cyclophosphamide Plus Corticosteroids Followed by Azathioprine, on Remission Rate in Patients With Lupus Nephritis[NCT00425438]Phase 352 participants (Actual)Interventional2007-03-31Terminated(stopped due to Study was terminated early for administrative reasons.)
The Research of Standard Diagnosis and Treatment for Henoch-Schonlein Purpura Nephritis in Children[NCT02532777]Phase 2100 participants (Anticipated)Interventional2015-08-31Recruiting
A Randomized, Multicenter, Open-label, 6-month Study to Explore the Efficacy and Safety of Enteric-coated Mycophenolate Sodium in Combination With Two Corticosteroid Regimens for the Treatment of Lupus Nephritis Flare[NCT00423098]Phase 281 participants (Actual)Interventional2007-02-28Completed
Phase II Clinical Trial of Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis[NCT01047072]Phase 20 participants (Actual)InterventionalWithdrawn
A Prospective, Open-label, Controlled Multicenter Trial to Assess the Efficacy and Safety of an Induction Regimen of Cyclosporine Micro Emulsion, Enteric-coated Mycophenolate Sodium (EC-MPS) and Corticosteroids, Followed by Administration of Everolimus an[NCT00371826]Phase 4126 participants (Actual)Interventional2006-03-31Completed
A 4-week, Multicenter, Double-blind, Double-dummy, Randomized, Parallel Group Study to Compare the Gastrointestinal Safety and Tolerability of EC-MPS & MMF When Administered in Combination With Calcineurin Inhibitors in Renal Transplant Recipients Experie[NCT00400400]Phase 4400 participants (Actual)Interventional2006-10-31Completed
Non-myeloablative Allogeneic Transplantation for the Treatment of Multiple Myeloma[NCT00185614]Phase 263 participants (Actual)Interventional2000-08-31Completed
Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating an Intensified Enteric-coated Mycophenolate Sodium (EC-MPS) Dosing Regimen in Comparison to a Standard Dosing Regimen of EC-MPS in Combination With Cyclosporin Microemul[NCT00369278]Phase 3128 participants (Actual)Interventional2006-06-30Completed
Timed Sequential Busulfan and Post Transplant Cyclophosphamide for Allogeneic Transplantation[NCT02861417]Phase 2204 participants (Actual)Interventional2016-08-05Active, not recruiting
A Randomized Controlled Trial of Mycophenolate Mofetil in Patients With IgA Nephropathy[NCT00318474]Phase 3184 participants (Actual)Interventional2002-01-31Terminated(stopped due to DSMB recommended stopping the trial because of lack of effect.)
Transplantation of Unrelated Umbilical Cord Blood for Patients With Hematological Diseases With Cyclophosphamide/Fludarabine/Total Body Irradiation Myeloablative Preparative Regimen[NCT00309842]Phase 2213 participants (Actual)Interventional2005-07-28Completed
Haploidentical Stem Cell Transplant Using Post Transplant Cyclophosphamide for GvHD Prophylaxis: A Pilot Study[NCT02248597]Phase 227 participants (Actual)Interventional2015-02-25Completed
A Multicenter, National, Open-label, Prospective, Randomized Study to Evaluate Efficacy and Tolerability of Enteric-coated Mycophenolate Sodium 1440 mg/Day With Tacrolimus Reduced Dose Versus Enteric-coated Mycophenolate Sodium 720 mg/Day With Tacrolimus [NCT00284934]Phase 394 participants (Actual)Interventional2005-12-31Completed
Steroid Free Immunosuppression in Liver Transplantation[NCT00296244]Phase 440 participants (Actual)Interventional2006-02-28Completed
Single Center, Open-label Randomized Prospective Trial: Effect of Sirolimus on Polycystic Liver Disease[NCT00934791]2 participants (Actual)Interventional2009-02-28Terminated(stopped due to Terminated due to inadequate enrollment)
Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Simultaneous Pancreas-Kidney Transplant Recipients[NCT00267150]Phase 331 participants (Actual)Interventional2005-11-30Completed
Efficacy and Safety Study Comparing Concentration-controlled Everolimus in Two Doses (1.5 and 3.0 mg/Day Starting Doses) With Reduced Cyclosporine Versus 1.44 g Mycophenolic Acid (as Sodium Salt) With Standard Dose Cyclosporine in de Novo Renal Transplant[NCT00251004]Phase 3833 participants (Actual)Interventional2005-10-31Completed
The Highly Sensitized Patients: Effects of Mycophenolate Mofetil (MMF) On Anti-Human Leukocyte Antigen (HLA) Antibody Levels In Patients Awaiting Cadaveric Renal Transplant[NCT00446459]Phase 245 participants (Actual)Interventional2006-04-30Completed
CellCept (Mycophenolate Mofetil, MMF) Maintenance Immunosuppression in Liver Transplant Recipients With Long-term Follow-up Post-transplantation for Non-Autoimmune Liver Disease - A Prospective, Randomized, Multicenter Trial.[NCT00206076]Phase 419 participants (Actual)Interventional2006-08-31Completed
An Open, Multi-center, Randomized Trial Comparing Haploidentical HSCTs From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies[NCT04547049]Phase 3160 participants (Anticipated)Interventional2020-09-01Recruiting
Phase IV Study of Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion (CsA-ME) in Kidney Transplant Patients[NCT00239083]Phase 440 participants (Anticipated)Interventional2005-01-31Completed
[NCT01895049]Phase 4171 participants (Actual)Interventional2013-08-31Completed
A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)[NCT04660539]Phase 3119 participants (Actual)Interventional2021-03-02Active, not recruiting
Regulatory T Cell Modulation in Kidney Transplantation With Biologic Blockade of Dual Effector Pathways, CD28 and IL-6 (CTOT-24)[NCT04066114]Phase 1/Phase 210 participants (Actual)Interventional2019-12-11Completed
Randomized Controlled Study: Effect of Mycophenolatmofetil in Patients With Histologically Proven Chronic Allograft Nephropathy[NCT00204230]86 participants Interventional1999-10-31Terminated
[NCT00411515]Phase 40 participants InterventionalCompleted
A Prospective, Open Label Protocol to Assess the Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients[NCT00238953]Phase 430 participants (Actual)Interventional2005-02-28Completed
A Prospective, Randomized, Open Label, Multicenter Trial of EC-MPS With Steroid Withdrawal vs EC-MPS With Standard Steroid Regimen for the Prevention of Acute Rejection Episodes in de Novo Renal Transplant Recipients.[NCT00238992]Phase 3144 participants Interventional2002-12-31Completed
Multicentre, Controlled, Prospective, Randomized, Open-label Clinical Trial to Compare Enteric-Coated Mycophenolate Sodium (EC-MPS) Plus Reduced Dose Cyclosporine Microemulsion (CsA-ME) Vs EC-MPS Plus Standard Dose CsA-ME in Elderly de Novo Renal Transpla[NCT00239031]Phase 3117 participants (Actual)Interventional2002-03-31Completed
A Prospective, Open-label, Multicenter, International Follow-up Study on the Safety and Efficacy of Enteric-coated Mycophenolate Sodium (EC-MPS) in Kidney Transplant Recipients[NCT00239070]Phase 369 participants (Actual)Interventional2003-04-30Completed
A Prospective, Open-label, Multicenter, International Follow-up Study on the Safety and Efficacy of Enteric-coated Mycophenolate Sodium (EC-MPS) in Kidney Transplant Recipients[NCT00241059]Phase 4183 participants (Actual)Interventional2002-08-31Completed
Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Metastatic Renal Cell Carcinoma[NCT00243009]Phase 21 participants (Actual)Interventional2005-06-30Terminated(stopped due to Due to a lack of a referal base, study was terminated.)
A Study of Low-dose Lenalidomide After Non-myeloablative Allogeneic Stem Cell Transplant With Bortezomib as GVHD Prophylaxis in High Risk Multiple Myeloma[NCT01954784]Phase 18 participants (Actual)Interventional2013-10-07Terminated(stopped due to Funding unavailable)
A Prospective, Open, Randomized Controlled Stage II Trial Investigating the Efficacy and Safety of Thymosin Alpha-1 in Treating Moderate to Severe Immune-related Adverse Events[NCT06178146]Phase 440 participants (Anticipated)Interventional2023-09-01Recruiting
ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia[NCT01971684]120 participants (Actual)Observational2013-08-31Completed
A Phase I/IIa, Open-label, Non-randomized, Study of ASC-101 in Patients With Hematologic Malignancies or Myelodysplastic Syndrome (MDS) Who Are Candidates for Dual-cord Umbilical Cord Blood Transplantation (UCBT)[NCT01983761]Phase 1/Phase 225 participants (Anticipated)Interventional2013-11-30Active, not recruiting
A One-Year Prospective, Randomized, Placebo-Controlled, Double-Blind, Phase II/III Safety Trial of Combination Therapy With IFN Beta-1a (Avonex) and Mycophenolate Mofetil (Cellcept) in Early Multiple Sclerosis[NCT00223301]Phase 2/Phase 324 participants Interventional2004-07-31Completed
MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions[NCT02582775]Phase 217 participants (Actual)Interventional2016-03-31Completed
6-month, Open-label, Randomized, Multicenter, Prospective, Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Everolimus in de Novo Renal Transplant Recipients Participating in the Eurotransplant Senior Program[NCT00956293]Phase 4207 participants (Actual)Interventional2009-07-31Terminated(stopped due to The study was terminated because the required sample size of 240-260 de novo senior renal transplant patients was not achieved within a reasonable time.)
Phase I/II Study of HLA-Matched Non-Myeloablative Peripheral Blood Mobilized Hematopoietic Progenitor Cell Transplantation as Treatment for Patients With Metastatic Renal Cell Carcinoma. A Multi-Center Trial.[NCT00005851]Phase 1/Phase 211 participants (Actual)Interventional2000-02-29Completed
Phase II Pilot Trial of Non-Myeloablative Allogeneic Peripheral Blood Stem Cell (PBSC) Transplantation Using Fludarabine, Low Dose TBI and Post-Transplant Cyclosporine and Mycophenolate Mofetil Followed by Donor Lymphocyte Infusion for Therapy of Advanced[NCT00005941]Phase 20 participants Interventional1999-11-30Completed
Efficacy and Safety of Certican® (Everolimus) in Combination With Myfortic® (EC-MPS, Enteric-coated Mycophenolate Sodium) After Early CNI Elimination Versus Myfortic® in Combination With Prograf® in Renal Transplant Recipients[NCT00965094]Phase 436 participants (Actual)Interventional2009-12-31Completed
A 12-month, Single-blind, Randomized, Parallel Group, Multicenter Study to Investigate the Efficacy and Safety of ERL080A Compared With MMF in de Novo Heart Recipients[NCT00574743]Phase 4162 participants (Actual)Interventional2002-01-31Completed
A Randomized Open-Label Study Comparing the Efficacy and Safety of Sirolimus Combined With Daclizumab, Mycophenolate and Corticosteroids vs Cyclosporine, Mycophenolate and Corticosteroids in Renal Allograft Recipients Receiving Kidneys From Older Donors[NCT00195273]Phase 361 participants (Actual)Interventional2004-11-30Completed
Comparison Between Tacrolimus and Mycophenolate Mofetil for Induction of Remission in Lupus Nephritis[NCT01580865]84 participants (Actual)Interventional2012-05-31Completed
A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability and Clinical Effect of Laquinimod in Active Lupus Nephritis Patients, in Combination With Standard of Care (Mycophenolate Mofetil and Steroid[NCT01085097]Phase 246 participants (Actual)Interventional2010-09-01Completed
Efficacy and Safety of Mycophenolate Mofetil as Maintenance Therapy After Rituximab Treatment in Childhood-onset, Frequently-relapsing or Steroid-dependent Nephrotic Syndrome: a Multicenter Double-blind, Randomized, Placebo-controlled Trial[NCT04531865]Phase 30 participants (Actual)Interventional2021-01-01Withdrawn(stopped due to lack of funding)
Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders[NCT05293509]Phase 20 participants (Actual)Interventional2022-03-02Withdrawn(stopped due to 0 accrual)
Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) for the Treatment of Hematological Diseases [MT2015-32][NCT02661035]Phase 2156 participants (Actual)Interventional2017-03-09Completed
Phase I Study of Escalating Doses of Total Marrow and Lymphoid Irradiation (TMLI) During Conditioning for HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Myelodysplasia or Acute Leukemia[NCT02446964]Phase 124 participants (Anticipated)Interventional2015-06-25Active, not recruiting
Tacrolimus, Mini-dose Methotrexate and Mycophenolate Mofetil Versus Tacrolimus and Methotrexate for the Prevention of Acute Graft-versus-Host-Disease[NCT01951885]Phase 3101 participants (Actual)Interventional2014-07-07Completed
A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA)[NCT01810588]Phase 2273 participants (Actual)Interventional2012-10-16Active, not recruiting
Multicenter, Open-label, Randomized, 24 Months Follow-up, Two Arm Study to Compare the Efficacy of Everolimus in Improving the Cardiovascular Profile in a Regimen With Mycophenolic Acid vs. a Regimen of CNI+MPA in Maintenance Renal Transplant Recipients.[NCT01169701]Phase 471 participants (Actual)Interventional2010-08-31Completed
Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant[NCT03249831]Phase 13 participants (Actual)Interventional2019-01-04Active, not recruiting
A Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf (Tacrolimus)/MMF and Extended Release (XL) Tacrolimus /MMF in de Novo Kidney Transplant Recipients[NCT00717678]Phase 373 participants (Actual)Interventional2007-12-31Completed
Hematopoietic Stem Cell Transplantation From Haploidentical Donors in Patients With Hematological Malignancies Using a Treosulfan-Based Preparative Regimen[NCT04195633]Phase 260 participants (Anticipated)Interventional2021-01-25Recruiting
An Multi-site, Open, Prospective Study to Assess the Efficacy and Safety of Multi-target Therapy in the Treatment of Class Ⅲ,Ⅳ,Ⅴ,Ⅲ+Ⅴand Ⅳ+Ⅴ Lupus Nephritis[NCT00876616]362 participants (Actual)Interventional2009-04-30Completed
Head to Head Comparison of Tacrolimus and Myfortic vs Tacrolimus and Sirolimus Used in Combination in Non-HLA Identical Living Donor Kidney Transplants[NCT01038505]Phase 40 participants (Actual)Interventional2010-01-31Withdrawn(stopped due to Lost funding source)
Pharmacokinetic Evaluation of an Intensified and Decreasing Dosing Regimen of Mycophenolate Sodium in Combination With Tacrolimus During the First 3 Months Post Kidney Transplant (the myFORTic Study)[NCT00941824]Phase 415 participants (Anticipated)Interventional2009-02-28Completed
Open Randomized Mult-Center Study to Evaluate Safety and Efficacy of Low Molecular Weight Sulfated Dextran in Islet Transplantation (CIT-01)[NCT00789308]Phase 224 participants (Actual)Interventional2008-07-11Completed
Multicentre, Open Study for the Setting up of Population Pharmacokinetic Models and Bayesian Estimators for Individual Dose Adjustment of Immunosuppressive Drugs (Cyclosporine, Tacrolimus, Mycophenolate Mofetil, Everolimus) During the First Year Post-graf[NCT00812786]Phase 442 participants (Actual)Interventional2007-07-31Completed
Open Label Randomized Study of Thymoglobulin Versus Daclizumab Induction Therapies for the Reduction of Acute Rejection in Live Donor Kidney Transplant Recipients With a Positive Crossmatch[NCT00275509]Phase 356 participants (Actual)Interventional2007-01-31Completed
Total Marrow Irradiation and Myeloablative Chemotherapy Followed By Double Umbilical Cord BloodTransplantation In Patients With Refractory Acute Leukemia[NCT00686556]Phase 112 participants (Actual)Interventional2012-08-31Completed
"Prospective Randomized Controlled Trial to Compare a Calcineurin Inhibitor Free Immunosuppression With a Low Dose Tacrolimus Based Immunosuppression in Old for Old Kidney Transplantation."[NCT00912678]Phase 490 participants (Actual)Interventional2002-03-31Completed
Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamid and Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia[NCT00669890]Phase 112 participants (Actual)Interventional2004-05-31Terminated(stopped due to PI left institution)
Biomarker-Linked Outcomes of Cellcept in Lupus Arthritis[NCT00594932]Phase 1/Phase 227 participants (Actual)Interventional2006-11-30Completed
Comparative Bioavailability of Myfenax® (Teva) and CellCept® (Roche) in Stable Patients After Renal Transplantation[NCT00991510]Phase 443 participants (Actual)Interventional2009-08-31Terminated(stopped due to Slow recruitment and lack of time to product launch)
A Phase II Study of Optimally Dosed Clofarabine in Combination With Low-Dose TBI to Decrease Relapse Rates After Related or Unrelated Donor Hematopoietic Cell Transplantation in Patients With AML[NCT01252667]Phase 244 participants (Actual)Interventional2011-01-25Completed
Abatacept for Graft Versus Host Disease Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease: a Sickle Transplant Alliance for Research Trial[NCT02867800]Phase 124 participants (Actual)Interventional2016-07-31Active, not recruiting
Allogeneic Stem Cell Transplantation for Children and Adolescents With CML: Conditioning Regimen, Donor Selection, Supportive Care and Diagnostic Procedures[NCT02707393]Phase 2/Phase 313 participants (Actual)Interventional2009-04-30Completed
A 12 Month, Multi-center, Randomized, Open-label Non-inferiority Study Comparing Safety and Efficacy of Concentration-controlled Everolimus With Low Dose Tacrolimus to Mycophenolate Mofetil With Standard Dose Tacrolimus in de Novo Renal Transplant Recipie[NCT01025817]Phase 3613 participants (Actual)Interventional2010-01-31Completed
Efficacy and Safety of Mycophenolate Mofetil in subjectswithSjogren's Syndrome[NCT02691949]Phase 254 participants (Anticipated)Interventional2016-02-29Enrolling by invitation
Sirolimus Associated With Tacrolimus at Low Doses in Elderly Kidney Transplant Patients: A Prospective Randomized Controlled Trial[NCT02683291]Phase 448 participants (Actual)Interventional2014-01-31Completed
A Phase III Multicenter, Randomized Trial Comparing Tacrolimus/Sirolimus/Methotrexate Versus Tacrolimus/Methotrexate or Cyclosporine/Mycophenolate Mofetil for GVHD Prophylaxis After Reduced Intensity Allogeneic Stem Cell Transplantation for Patients With [NCT00928018]Phase 3139 participants (Actual)Interventional2009-06-30Completed
A Three-Month, Open-Label, Two Cohort Study to Investigate the Safety and Tolerability of Myfortic in Combination With Neoral (Cyclosporin) or Prograf (Tacrolimus) in Liver Transplant Recipients With GI Intolerance[NCT00619216]31 participants (Actual)Observational2008-03-31Completed
A Prospective Randomized Trial of Prednisone and Tacrolimus Versus Prednisone, Tacrolimus and Mycophenolate Mofetil in Pediatric Liver Transplantation[NCT00656266]Phase 413 participants (Actual)Interventional2004-11-30Terminated(stopped due to insufficient funding)
A Single-Dose, 2-Period, 2-Treatment, 2-Way Crossover Bioequivalence Study of Mycophenolate Mofetil Capsules Under Fed Conditions[NCT00910663]Phase 160 participants (Actual)Interventional2006-10-31Completed
Selective CD28 Blockade With Lulizumab Compared to CNI Inhibition With Tacrolimus in Renal Transplant Recipients[NCT04903054]Phase 20 participants (Actual)Interventional2022-01-10Withdrawn(stopped due to Limited period of availability of a supply of the study drug and difficulties in enrollment)
Cyclophosphamide Versus Anti-thymocyte Globulin for GVHD Prophylaxis After RIC Allo-SCT[NCT02876679]Phase 294 participants (Actual)Interventional2017-04-06Completed
Evaluation of the Benefit of Antithymocyte Induction Therapy on Hepatic Fibrosis in de Novo Hepatitis C Virus Liver Transplant Patients.[NCT00538265]Phase 4100 participants (Anticipated)Interventional2005-05-31Completed
IIT2021-11-PAQUETTE-OmitMMF: Fludarabine and Total Body Irradiation 800 cGy or 1125 cGy For Allogeneic Stem Cell Transplant Using Graft Versus Host Disease Prophylaxis With Post-Transplant Cyclophosphamide and Tacrolimus, Without Mycophenolate Mofetil[NCT05256537]Phase 260 participants (Anticipated)Interventional2022-04-26Recruiting
Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study[NCT04473911]Phase 125 participants (Actual)Interventional2020-08-14Active, not recruiting
Pharmacogenetics of Mycophenolic Acid in Kidney Transplant Patients[NCT00433953]Phase 144 participants (Actual)Interventional2007-02-28Terminated(stopped due to No longer following patient and no plans to publish.)
Phase II Study of Shortened-duration Tacrolimus Following Nonmyeloablative Peripheral Blood Stem Cell Transplant With High-dose Posttransplantation Cyclophosphamide in Malignancies That Are Challenging to Engraft[NCT02556931]Phase 2117 participants (Actual)Interventional2015-12-31Completed
Pilot Study of Mycophenolate Mofetil in Congenital Uropathies[NCT00193635]Phase 112 participants (Actual)Interventional2002-03-31Completed
Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Cyclosporine Microemulsion Based Regimen in de Novo Living Donor Kidney Transplant Recipients ; A Prospective, Open Label, Multi-center Study[NCT00537862]Phase 4200 participants (Actual)Interventional2006-05-31Completed
An Open, Prospective, Randomised, Controlled, Multi-Center Study Comparing Fixed Dose vs Concentration Controlled Mycophenolate Mofetil Regimens for de Novo Patients Following Transplantation[NCT00166244]Phase 4901 participants (Actual)Interventional2003-05-31Completed
Non-Myeloablative Allogeneic Hematopoietic Cell Transplantation For Patients With Disease Relapse Or Myelodysplasia After Prior Autologous Transplantation[NCT00053196]Phase 282 participants (Actual)Interventional2002-12-31Completed
The Effect of Sirolimus on the Pharmacokinetics of Tacrolimus[NCT00166829]Phase 440 participants Interventional2004-05-31Recruiting
Enteric Coated Mycophenolic Acid (Myfortic) in Liver Transplant Recipients- Effect on Compliance and Calcineurin Inhibitor and Corticosteroid Sparing[NCT00167492]Phase 40 participants (Actual)Interventional2005-09-30Withdrawn
An Open Label, Multi-centre Trial of Alipogene Tiparvovec for the Treatment of LPLD Patients[NCT02904772]Phase 20 participants (Actual)Interventional2016-10-31Withdrawn(stopped due to uniQure, has decided not to renew the Marketing Authorization of Glybera in the EU. This decision is not related to any safety, efficacy or quality issue)
A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies[NCT00423826]0 participants Expanded Access2007-01-31No longer available
Phase II Trial of Non-Myeloablative Allogeneic Peripheral Blood Stem Cell (PBSC) Transplantation Using Fludarabine, Low-Dose TBI, and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil (MMF) Followed by Donor Lymphocyte Infusion[NCT00006233]Phase 20 participants Interventional2000-01-31Completed
Induction of Mixed Hematopoietic Chimerism in Patients Using Fludarabine, Low Dose TBI, PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil[NCT00006251]Phase 1/Phase 221 participants (Actual)Interventional2000-05-31Completed
Open-label Study to Evaluate the Tolerability, Safety and Efficacy of the Equimolar Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium in Patients With Stable Renal Transplant Receiving Tacrolimus[NCT00171392]Phase 3132 participants (Actual)Interventional2004-03-31Completed
Open Label, Randomized, Single-dose, Crossover Study to Evaluate the Pharmacokinetic Characteristics of CKD-4101 Tablet, in Healthy Volunteers[NCT01016626]Phase 128 participants (Actual)Interventional2009-07-31Completed
A Pilot Study of Campath-1H Induction Therapy Combined With Rituximab®, Myfortic™ and a Short Course of Calcineurin Inhibitor Therapy to Allow for a Long Term Calcineurin Inhibitor Free Regimen After Renal Transplantation[NCT00579592]11 participants (Actual)Interventional2006-04-30Terminated(stopped due to Higher than expected rate of acute rejection)
Multicenter, Open-label Follow-up Study on the Safety of Enteric-coated Mycophenolate Sodium in Renal Transplant Patients.[NCT00149916]Phase 3139 participants (Actual)Interventional2000-04-30Completed
An Open Label, Prospective, Randomized, Controlled, Multicenter Study Assessing Fixed Dose vs. Concentration Controlled CellCept Regimens for Patients Following a Single Organ Renal Transplantation in Combination With Full Dose and Reduced Dose Calcineuri[NCT00217152]Phase 412 participants (Actual)Interventional2005-03-31Terminated(stopped due to Roche decided to prematurely terminate study.)
Role of Th1, Th2 and Monokine Responses as Risk Factors of Acute and Chronic Renal Transplant Rejection - Impact of Different Immunosuppressive Protocols[NCT00150891]84 participants (Actual)Observational1998-01-31Completed
Evaluating the Safety of Myfortic (Mycophenolate Sodium) in Patients With Lupus Nephritis: a 12 Month, Single-arm, Observational Study in Taiwan Population[NCT04645589]64 participants (Anticipated)Observational2021-03-16Recruiting
Phase II Trial of Reduced Intensity Conditioning Hematopoietic Stem Cell Transplantation for Primary Immune Deficiencies, Immune Dysregulatory Syndromes, and Inherited Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide[NCT04232085]Phase 227 participants (Anticipated)Interventional2020-02-12Recruiting
A Pilot Study of Campath-1H Induction Therapy Combined With CellCept® Therapy to Allow for a Calcineurin Inhibitor Free Regimen After Renal Transplantation[NCT00214266]Phase 231 participants (Actual)Interventional2005-01-31Completed
A Phase III, Randomised, Single-site Trial on the Minimisation of Immunosuppression In Elderly Renal Transplant Recipients.[NCT05073822]Phase 1/Phase 20 participants (Actual)Interventional2023-02-20Withdrawn(stopped due to Funding not secured)
Induction in Sensitized Kidney Transplant Recipients Without Preexisting Donor-specific antiboDies: a Randomized Multicentre Trial Between a Lymphocyte Depleting and Basiliximab.[NCT05385432]Phase 3244 participants (Anticipated)Interventional2023-09-12Not yet recruiting
Rituximab For Prevention Of Acute Graft-Versus-Host Disease (GVHD) After Unrelated Donor Allogeneic Hematopoietic Cell Transplantation (HCT)[NCT01044745]Phase 220 participants (Actual)Interventional2009-12-10Terminated(stopped due to Study was closed to accrual for safety related to the frequency of BK infections.)
A Randomized Controlled Trial to Compare the Efficacy of Oral Mycophenolate Mofetil With Placebo in Patients With Systemic Sclerosis Related Early Interstitial Lung Disease[NCT02896205]Phase 341 participants (Actual)Interventional2016-10-31Completed
Phase III Multicenter Open-label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 mo Post-Transplant to Prevent Long-term Complications After Pediatric Heart Transplantation[NCT03386539]Phase 3211 participants (Actual)Interventional2018-01-29Active, not recruiting
A Phase II Trial of Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched Peripheral Blood Stem Cells for Patients With Hematologic Malignancies[NCT00782379]Phase 220 participants (Actual)Interventional2008-10-31Completed
Standard Therapy or Individualized Immunosuppression For Lowering Adverse Event Risk[NCT04473924]Phase 20 participants (Actual)Interventional2022-07-31Withdrawn(stopped due to Study has never started as sponsor did not approve for funding.)
A Pilot Study Comparing the Safety and Efficacy of Zortress (Everolimus) With Low Dose Tacrolimus to Early Conversion to Calcineulin Inhibitor-Free Regimen and Mycophenolic Acid With Standard Dose Tacrolimus in Recipients of ECD/DCD Kidneys[NCT01878786]Phase 2/Phase 325 participants (Actual)Interventional2013-06-30Terminated(stopped due to Interim results suggested a concern for patient outcomes and safety)
Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies[NCT01471067]Phase 133 participants (Actual)Interventional2012-07-13Completed
A Study to Investigate the Impact of Pharmacogenetics on CellCept Use, in Patients Participating in a Study in Renal Transplantation[NCT00337493]Phase 4155 participants (Actual)Interventional2005-12-31Completed
Open-Label, Randomized Study Comparing the Patient Reported Severity of GI Side Effects of MMF Versus EC-MPS in Maintenance Heart Transplant Patients.[NCT00468936]Phase 3100 participants (Anticipated)Interventional2007-05-31Recruiting
Randomized Comparative Study on Effects of Immunosuppression on HCV Recurrence After Living Donor Liver Transplantation - Comparison Between Tacrolimus + MMF and Tacrolimus + Steroid[NCT00469131]Phase 379 participants (Actual)Interventional2003-09-30Completed
Mycophenolic Acid Monotherapy in Recipients of HLA-identical Living-Related Transplantation[NCT01053221]Phase 216 participants (Actual)Interventional2006-03-31Terminated(stopped due to Funding loss)
Evaluate the Efficacy and Safety of RaparoBell® Tablet Plus Calcineurin Inhibitors Compared With Mycophenolate Mofetil Plus Calcineurin Inhibitors in ABO Incompatible De Novo Living Kidney Transplant Recipients. [ART Study][NCT04700709]Phase 4158 participants (Anticipated)Interventional2021-01-31Not yet recruiting
Pharmacokinetics of Everolimus and Enteric-Coated Mycophenolatesodium Before and After Withdrawal of Cyclosporine in Stable Renal Transplant Patients[NCT00443937]Phase 415 participants (Anticipated)Interventional2004-01-31Completed
Natural Killer Cells in Allogeneic Cord Blood Transplantation[NCT01619761]Phase 113 participants (Actual)Interventional2013-05-03Active, not recruiting
A Randomized, Prospective, Multicenter Trial to Compare the Effect on Chronic Allograft Nephropathy Prevention of Mycophenolate Mofetil Versus Azathioprine as the Sole Immunosuppressive Therapy for Kidney Transplant Recipients[NCT00494741]Phase 4233 participants (Actual)Interventional2007-05-31Completed
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation With JSP191-Based Conditioning in Participants With GATA2 Deficiency[NCT05907746]Phase 232 participants (Anticipated)Interventional2023-11-29Recruiting
Targeted Astatine-211-Labeled BC8-B10 Monoclonal Antibody as Reduced Intensity Conditioning for Nonmalignant Diseases[NCT04083183]Phase 1/Phase 240 participants (Anticipated)Interventional2020-06-16Recruiting
Mesenchymal Stem Cells Under Basiliximab/Low Dose RATG to Induce Renal Transplant Tolerance[NCT00752479]Phase 1/Phase 24 participants (Actual)Interventional2008-05-31Terminated(stopped due to Necessity of major revision of the protocol)
A Randomized Study To Compare The Safety And Efficacy Of Two Immunosuppressive Regimens In De Novo Renal Allograft Recipients:Sirolimus Plus Mycophenolate Mofetil Plus Corticosteroids Following A Rabbit Anti-Human Thymocyte Globulin Induction (RATG) Vs Ta[NCT00261820]Phase 4160 participants InterventionalCompleted
Benadamustine, Fludarabine and Busulfan Conditioning in Recipients of Haploidentical Stem Cell Transplantation (FluBuBe)[NCT04942730]Phase 240 participants (Anticipated)Interventional2021-01-21Recruiting
SCHEDULE - Scandinavian Heart Transplant Everolimus de Novo Study With Early CNI Avoidance[NCT01266148]Phase 4115 participants (Actual)Interventional2009-11-30Completed
An Open-Label, Prospective, Randomized, Controlled, Multi-Center Study Assessing Fixed Dose Versus Concentration Controlled Cellcept® Regimens for Patients Following a Single Organ Renal Transplantation in Combination With Full Dose and Reduced Dose Calci[NCT00087581]Phase 4720 participants (Actual)Interventional2004-06-30Completed
Study of Tacrolimus vs Mycophenolate Mofetil in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome: a Randomized, Multicenter, Open-label, Parallel-arm Study[NCT04048161]Phase 4270 participants (Actual)Interventional2019-11-12Completed
Phase I/II Study De-intensifying Exposure of Post-transplantation Cyclophosphamide as GVHD Prophylaxis After HLA-haploidentical Hematopoietic Cell Transplantation for Hematologic Malignancies[NCT03983850]Phase 1/Phase 2400 participants (Anticipated)Interventional2019-07-09Recruiting
Mycophenolate Sodium Treatment in Patients With Primary Sjogren's Syndrome - An Open Label Pilot Trial[NCT00542763]Phase 112 participants (Actual)Interventional2005-04-30Completed
Cord Blood Ex-Vivo MSC Expansion Plus Fucosylation to Enhance Homing and Engraftment[NCT03096782]Phase 26 participants (Actual)Interventional2017-10-13Completed
Multitarget Therapy for Idiopathic Membranous Nephropathy[NCT04424862]Phase 482 participants (Actual)Interventional2020-06-09Completed
Autologous Followed by Non-myeloablative Allogeneic Transplantation for Non-Hodgkin's Lymphoma[NCT00882895]Phase 218 participants (Actual)Interventional2009-05-05Active, not recruiting
An Exploratory, Open-Label, Single Center Study to Assess the Efficacy and Dose Titration of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Reducing Donor Specific Antibody (DSA) Strength in Maintenance Kidney Transplant Recipients[NCT01044303]Phase 432 participants (Actual)Interventional2010-01-31Completed
Allogeneic Hematopoietic Cell Transplantation for Patients With Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen[NCT00919503]Phase 298 participants (Actual)Interventional2009-07-31Completed
Nonmyeloablative BMT With Post-transplant Cyclophosphamide, Rituximab and Optimized Donor Selection for B-cell Lymphomas[NCT00946023]Phase 2135 participants (Actual)Interventional2009-07-31Terminated(stopped due to Funding was unavailable to complete the study as originally planned.)
5, 6 or 7 Year Follow-up Control After the SCHEDULE Study (SCANDINAVIAN HEART TRANSPLANT EVEROLIMUS DE NOVO STUDY WITH EARLY CNI AVOIDANCE)[NCT02864706]Phase 495 participants (Actual)Interventional2016-01-18Completed
Conversion From MPA to Zortress (Everolimus) for GI Toxicity Post-renal Transplantation[NCT02974686]Phase 41 participants (Actual)Interventional2016-11-30Terminated(stopped due to low recruitment)
Allogeneic Stem Cell Transplantation For Multiple Myeloma: A Two Step Approach To Reduce Toxicity Involving High Dose Melphalan and Autologous Stem Cell Transplant Followed By PBSC Allografting After Low Dose TBI[NCT00003954]Phase 1/Phase 240 participants (Anticipated)Interventional1999-03-31Completed
Evaluation of Efficacy and Safety of Rituximab in Association With Mycophenolate Mofetil Versus Mycophenolate Mofetil Alone in Patients With Interstitial Lung Diseases (ILD) Non-responders to a First-line Immunosuppressive Treatment[NCT02990286]Phase 3122 participants (Actual)Interventional2017-01-20Completed
Phase II Study of IV Busulfan Combined With 12 cGy of Fractionated Total Body Irradiation (FTBI) and Etoposide (VP-16) as a Preparative Regimen for Allogeneic Bone Marrow Transplantation for Patients With Advanced Hematological Malignancies[NCT00534430]Phase 230 participants (Actual)Interventional2000-02-29Active, not recruiting
A Randomized, Open Label, Phase II Multicenter Study of Non-Myeloablative Autologous Transplantation With Auto-CD34+HPC Versus Currently Available Immunosuppressive/Immunomodulatory Therapy for Treatment of Systemic Lupus Erythematosus[NCT00230035]Phase 20 participants (Actual)Interventional2005-09-30Withdrawn(stopped due to Recommended by DSMB due to lack of accrual)
A One Arm, Open-label Study to Investigate the Tolerability and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Renal Transplant Recipients Who Received Mycophenolate Mofetil (MMF)[NCT00238966]Phase 4187 participants (Actual)Interventional2002-11-30Completed
An Open Label Study to Evaluate the Tolerability and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion (CsA-ME) in Maintenance Renal Transplant Recipients[NCT00239044]Phase 340 participants Interventional2002-12-31Completed
Efficacy and Safety of Two Immunosuppressive Regimens Mycophenolate Sodium (EC-MPS) With Short-term Steroid Use or Free of Steroids Compared With a Regimen of EC-MPS With Standard Steroids in de Novo Kidney Recipients[NCT00240955]Phase 479 participants (Actual)Interventional2004-03-31Completed
A 12-week Multicenter, Randomized, Open Study to Evaluate the Effects of Enteric-coated Mycophenolate Sodium (EC-MPS) in Terms of Quality of Life in Patients With Gastrointestinal (GI) Symptoms Treated With MMF (Mycophenolate Mofetil) After Kidney Transpl[NCT00400647]Phase 4136 participants (Actual)Interventional2006-07-31Completed
Mycophenolate for Pulmonary Sarcoidosis[NCT00262132]Phase 320 participants Interventional2003-09-30Terminated
A Multi-Center Phase II Study of Nonmyeloablative Conditioning With TBI and Fludarabine for HLA-Matched Related Hematopoietic Cell Transplantation for Treatment of Chronic Myeloid Leukemia in Chronic and Accelerated Phase[NCT00110058]Phase 240 participants (Anticipated)Interventional2005-02-28Completed
Pilot Study on Allogeneic Stem Cell Transplantation Following Conditioning With Fludarabine and an Alkylating Agent in Patients With High-Risk Chronic Lymphocytic Leukemia[NCT00281983]Phase 1/Phase 2100 participants (Actual)Interventional2000-06-30Completed
A Cyclophosphamide/Fludarabine/Total Body Irradiation Preparative Regimen for Patients With Hematological Malignancy Receiving Unrelated Donor Umbilical Cord Blood Transplantation[NCT00290641]68 participants (Actual)Interventional2001-04-30Completed
Fludarabine and Low-Dose TBI Dose Escalation to Determine the Optimal Regimen for Achieving High Rates Engraftment of Unrelated Donor Peripheral Blood Stem Cell in Patients With Chronic Myeloid Leukemia - A Multi-Center Trial[NCT00119340]Phase 1/Phase 275 participants (Anticipated)Interventional2005-04-30Completed
A Randomized Double Blinded Placebo-Controlled Phase III Trial Comparing Cyclosporine Plus Steroids With or Without Myfortic as Primary Treatment for Extensive Chronic Graft Versus Host Disease[NCT00298324]Phase 334 participants (Actual)Interventional2006-09-30Terminated(stopped due to Due to slow accrual)
Mycophenolate Mofetil Versus Intravenous Cyclophosphamide Pulses in the Treatment of Crescentic IgA Nephropathy[NCT00301600]40 participants (Actual)Interventional2003-01-31Completed
Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies[NCT00301951]Phase 17 participants (Actual)Interventional2004-09-30Completed
Mycophenolate Mofetil Versus Azathioprine for Maintenance Therapy in ANCA Associated Systemic Vasculitis[NCT00307645]Phase 3160 participants Interventional2003-05-31Terminated
Effect of Enteric-Coated Mycophenolate Sodium (EC-MPS) Plus Valsartan as Part of Intensified Multi-factorial Intervention Compared to EC-MPS Plus Standard Practice of Care on Development of Transplant Nephropathy in Cadaver Donor Kidney Recipients Given B[NCT00308425]Phase 3119 participants (Actual)Interventional2002-10-31Completed
A Phase II Study of Reduced Dose Post Transplantation Cyclophosphamide as GvHD Prophylaxis in Adult Patients With Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation[NCT06001385]Phase 2170 participants (Anticipated)Interventional2023-11-01Not yet recruiting
A Pilot Study of Reduced Intensity HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Advanced Myelofibrosis[NCT03118492]Phase 116 participants (Anticipated)Interventional2017-05-24Recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome[NCT01861106]Phase 2144 participants (Anticipated)Interventional2013-07-24Recruiting
A Phase II Trial of Total Body Irradiation Plus Metabolism-Based Cyclophosphamide Dosing as Preparative Therapy for Allogeneic Hematopoietic Cell Transplant for Patients With Hematological Malignancy[NCT00317785]Phase 250 participants (Anticipated)Interventional2005-05-31Completed
An Open-Label Study to Evaluate the Effect on Quality of Life of Switching Kidney Transplant Patients From Reduced Dose EC-MPS to a Higher Than the Equimolar Dose of CellCept[NCT00420472]Phase 40 participants Interventional2007-03-31Terminated
Hemophagocytic Lymphohistiocytosis[NCT00334672]Phase 3288 participants (Anticipated)Interventional2006-03-31Active, not recruiting
A Pilot Study to Evaluate the Co-infusion of Ex Vivo Expanded Umbilical Cord Blood Progenitors With an Unmanipulated Cord Blood Graft in Patients Undergoing Umbilical Cord Blood Transplantation for Hematologic Malignancies[NCT00343798]Phase 123 participants (Actual)Interventional2006-04-30Completed
Low-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter Trial[NCT00093743]Phase 12 participants (Actual)Interventional2000-01-31Completed
A Comparison of Oral Methylprednisolone Plus Azathioprine or Mycophenolate Mofetil for the Treatment of Bullous Pemphigoid[NCT00431119]Phase 270 participants Interventional1997-10-31Completed
Phase I, Open-label Study of Mycophenolate Mofetil In Systemic Sclerosis[NCT00433186]Phase 130 participants (Anticipated)Interventional2006-03-31Completed
Study to Investigate the Clinical Outcomes of Different Regimens of Myfortic® in De Novo Kidney Tx Pts Using Simulect® and Neoral® With or Without Steroids[NCT00101738]Phase 3342 participants (Actual)Interventional2003-03-31Completed
Interruption of the Calcineurine Inhibitors (ICN) and Introduction of Mycophenolate Mofetil (MMF) in Liver Transplant Patients With Side Effects Due to ICN: Study of the Reduction of the Risks of Rejection by Mycophenolate Mofetil Therapeutic Drug Monitor[NCT00456235]Phase 492 participants (Actual)Interventional2006-09-30Completed
A Randomized, Controlled, Multi-Center Study of Thymoglobulin Induction Therapy With a Calcineurin Inhibitor Sparing Regimen in Liver Transplant Patients[NCT00117689]Phase 275 participants (Actual)Interventional2005-04-30Completed
A Multicenter, Randomized, Open Label, Parallel Study to Evaluate and Compare the Efficacy and Safety of FK506MR vs Prograf® in Combination With MMF and Steroids in Patients Undergoing Kidney Transplantation and a Pharmacokinetics Study.[NCT00481819]Phase 3240 participants (Actual)Interventional2007-07-31Completed
[NCT00120419]Phase 490 participants (Anticipated)Interventional2005-04-30Recruiting
A Randomized, Open-label Study of the Effect of Replacing CNI With Sirolimus in a Standard Care Regimen of CNI, CellCept, and Steroids on Renal Function in Heart Transplant Patients[NCT00121784]Phase 412 participants (Actual)Interventional2005-10-31Terminated(stopped due to Poor recruitment)
Cord Blood Expansion on Mesenchymal Stem Cells[NCT00498316]Phase 198 participants (Actual)Interventional2007-07-03Completed
A Randomized, Multicenter Study to Assess the Efficacy on Diseases Activity of Enteric-coated Mycophenolate Sodium Versus Continuation of Azathioprine in Patients With Systemic Lupus Erythematosus on Azathioprine Maintenance Therapy.[NCT00504244]Phase 312 participants (Actual)Interventional2007-07-31Terminated(stopped due to Insufficient recruitment)
Evaluation of the Long-term Safety and Efficacy of a Tacrolimus-based 5-day Steroid Rapid Withdrawal Immunoprophylactic Regimen in de Novo Renal Transplantation[NCT00133172]Phase 485 participants (Actual)Interventional2005-07-31Terminated(stopped due to Varience of supply chain from that required by protocol)
A Multicenter, Open, Single Arm, Pilot Study to Evaluate Efficacy, Tolerability and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion and Steroids in Pediatric de Novo Renal Transplant Patients[NCT00154206]Phase 415 participants Interventional2004-09-30Completed
[NCT00154245]Phase 420 participants Interventional2004-01-31Completed
A European Multicenter Open-Label Randomised Trial to Evaluate the Efficacy and Safety of Sirolimus and Tacrolimus Compared to MMF and Tacrolimus With Short-Course Induction Therapy, Short-Term Steroids Application in de Novo SPK Transplanted Diabetic Pat[NCT00140543]Phase 3228 participants Interventional2002-02-28Completed
A Non-Myeloablative Conditioning Regimen With Peri-Transplant Rituximab and the Transplantation of Hematopoietic Stem Cells From HLA-Compatible Related or Unrelated Donors in Patients With B Cell Lymphoid Malignancies[NCT00425802]Phase 261 participants (Actual)Interventional2006-11-28Completed
Tacrolimus and Mycophenolate Mofetil vs Tacrolimus and Sirolimus in SPK, Pancreas After Kidney or Pancreas Transplant Alone[NCT00533442]Phase 2170 participants (Actual)Interventional2000-09-30Completed
Hematopoietic Cell Transplantation for Treatment of Patients With Primary Immunodeficiencies and Other Nonmalignant Inherited Disorders Using Low-Dose TBI and Fludarabine With or Without Campath®[NCT00553098]Phase 229 participants (Actual)Interventional2006-06-30Completed
Reduced-intensity, Related-donor Allogeneic BMT With Fludarabine, Busulfan, and High-dose Posttransplantation Cyclophosphamide for Hematologic Malignancies[NCT01135329]Phase 215 participants (Actual)Interventional2010-08-31Terminated(stopped due to The stopping rule was met and hence the study was closed)
Tandem Autologous HCT/Nonmyeloablative Allogeneic HCT From HLA-Matched Related and Unrelated Donors Followed by Bortezomib Maintenance Therapy for Patients With High-Risk Multiple Myeloma[NCT00793572]Phase 232 participants (Actual)Interventional2008-10-31Completed
Sequential Autologous HCT / Nonmyeloablative Allogeneic HCT Using Related, HLA-Haploidentical Donors for Patients With High-Risk Lymphoma, Multiple Myeloma, or Chronic Lymphocytic Leukemia[NCT01008462]Phase 216 participants (Actual)Interventional2010-03-18Completed
Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders[NCT01043640]Phase 246 participants (Actual)Interventional2009-12-31Completed
he Efficacy and Safety of Combining Mycophenolate Mofetil With Methimazole on Remission of Newly Diagnosis Graves' Disease (3M-RGD Trial): an Open-label, Randomized Trial[NCT06068179]Phase 2/Phase 3205 participants (Anticipated)Interventional2023-10-08Not yet recruiting
A 12-month, Prospective, Randomized, Dual Center, Open Label Pilot Study to Evaluate the Safety and Efficacy of Myfortic® (Mycophenolic Acid) Loading Regimens in Combination With Thymoglobulin® [Anti-thymocyte Globulin (Rabbit)] or Simulect® (Basiliximab)[NCT01336296]Phase 461 participants (Actual)Interventional2010-09-30Completed
Comparative Efficacy of Mizoribine With Mycophenolate Mofetil for Living Related Kidney Transplantation Recipients[NCT06114953]Phase 4152 participants (Anticipated)Interventional2023-01-01Recruiting
Multicenter, Open-label Follow-up Study on the Safety of Enteric-coated Mycophenolate Sodium in Maintenance Renal Transplant Patients[NCT00149864]Phase 3264 participants (Actual)Interventional2000-02-29Completed
Multicenter, Double-blind, Randomized, Parallel Group Study on Efficacy and Safety of Enteric-coated Mycophenolate Sodium vs. Mycophenolate Mofetil in de Novo Chinese Renal Transplant Recipients[NCT00149903]Phase 3300 participants Interventional2005-01-31Completed
Pharmacokinetics of Mycophenolate Mofetil Alone and in Combination With Valganciclovir in Renal and Heart Transplant Recipients[NCT00189150]Phase 416 participants (Actual)Interventional2005-04-30Completed
A Randomized, Double-Blind Study to Evaluate the Safety of Continued Treatment With CellCept in Patients With Well-Controlled Myasthenia Gravis Receiving a Stable Dose of Prednisone[NCT00408213]Phase 3136 participants (Anticipated)Interventional2004-06-30Completed
Effects of Mycophenolate Mofetil (MMF) on Surrogate Markers for Cardiovascular Disease in HIV-1 Infected Patients[NCT00247494]Phase 490 participants (Anticipated)Interventional2005-04-30Recruiting
A Dose Finding Study of Total Body Irradiation for Conditioning Patients With Severe Aplastic Anemia Transplanted With Umbilical Cord Blood[NCT00354419]Phase 130 participants (Anticipated)Interventional2006-02-28Terminated(stopped due to Low accrual)
Efficacy and Safety of Maintenance Neoral Compared to Bitherapy Neoral-Imurel or Neoral-CellCept in Renal Transplantation[NCT00461825]Phase 3207 participants Interventional1998-07-31Completed
An Open-label, Balanced, Randomized, Two-treatment, Two-period, Two Sequence, Single Dose, Crossover, Bioequivalence Study in Healthy, Adult,Human, Male Subjects Under Fasting Conditions.[NCT01513044]Phase 168 participants (Actual)Interventional2009-01-31Completed
An Open-label, Balanced, Randomized, Two-treatment, Two-period, Two Sequence, Single Dose, Crossover, Bioequivalence Study in Healthy, Adult,Human, Male Subjects Under Fed Conditions.[NCT01513057]Phase 161 participants (Actual)Interventional2009-01-31Completed
Phase II Study of Evaluating the Efficacy of Total Marrow and Lymphoid Irradiation (TMLI) as the Conditioning Regimen for HLA-Haploidentical Hematopoietic Cell Transplantation in Patients With Myelodysplasia or Acute Leukemia[NCT04262843]Phase 270 participants (Anticipated)Interventional2020-02-07Recruiting
Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders[NCT00053989]Phase 241 participants (Actual)Interventional2002-01-29Completed
Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies After a Non-Myeloablative Conditioning Regimen From HLA-Matched Sibling Donors[NCT00006350]Phase 20 participants Interventional2000-01-31Completed
Comparative Study of the Efficacy of Induction Therapy With Cyclophosphamide or Mycophenolate Mofetil for Non-Life-Threatening Relapses of PR3- or MPO-ANCA Associated Vasculitis[NCT00103792]Phase 390 participants (Anticipated)Interventional2004-12-31Recruiting
An Open Label Study to Evaluate the Effect of CellCept in Combination With Cyclosporine A and Steroids on Renal Function and the Prevention of Acute Rejection in Heart Transplant Patients.[NCT02091414]Phase 336 participants (Actual)Interventional2006-08-31Completed
A Dose-Blinded, 2-Dose Level, Parallel-Group, Multicenter, Long-Term Extension Study to Evaluate the Long-Term Safety, Efficacy, and Immunogenicity of BIIB023 in Subjects With Lupus Nephritis[NCT01930890]Phase 287 participants (Actual)Interventional2013-11-30Terminated(stopped due to Results from pre-specified criteria in study NCT01499355 (211LE201) did not demonstrate sufficient efficacy to warrant continuation of the study)
A Phase I Study Combining Escalating Doses of Radiolabeled BC8 (Anti-CD45) Antibody With Fludarabine, Low Dose TBI, PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to Establish Mixed or Full Donor Chimerism [NCT00008177]Phase 179 participants (Actual)Interventional1999-07-27Completed
Transplantation of Unrelated Donor Hematopoietic Stem Cells for the Treatment of Hematological Malignancies[NCT00281879]Phase 2200 participants (Actual)Interventional2006-02-28Terminated
Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil[NCT00008450]Phase 16 participants (Actual)Interventional1997-08-11Completed
Pharmacokinetics of Mycophenolic Acid and Mycophenolate 7-O-Phenolic Glucuronide in Healthy Subjects With or Without Two Common Genetic Polymorphisms in the Promoter Region of Uridine Diphosphate Glucuronosyltransferase 1A9[NCT00128947]Phase 1130 participants Interventional2005-07-31Completed
Submyeloablative Allogeneic Blood Stem Cell Transplantation With HLA Identical Donor Lymphocyte Infusions From Matched Related and Matched Unrelated Donors for Treatment of Metastatic Renal Cell Carcinoma[NCT00025519]Phase 20 participants (Actual)Interventional2001-06-30Withdrawn
Nonmyeloablative Allogeneic Hematopoietic Cell Transplantation From HLA Matched Sibling Donors For Treatment Of Patients With High Risk Acute Lymphocytic Leukemia In Complete Remission[NCT00027547]Phase 1/Phase 20 participants Interventional2001-07-31Completed
Sibling Donor Cord Blood Banking and Transplantation[NCT00029380]Phase 230 participants (Anticipated)Interventional1999-01-31Completed
Nonmyeloablative PBSC Allografting From HLA Matched Related Donors Using Fludarabine and/or Low Dose TBI With Disease-Risk Based Immunosuppression[NCT00014235]160 participants (Anticipated)Interventional2000-12-31Completed
A Phase I/II Randomized, Double-Blind, Placebo-Controlled Pilot Study of Beta-D-2,6-diaminopurine Dioxolane (DAPD) Versus DAPD Plus Mycophenolate Mofetil (MMF) in Treatment-Experienced Subjects[NCT00038272]Phase 256 participants InterventionalCompleted
Treatment of Patients With Membranous Nephropathy and Renal Insufficiency With Mycophenolate Mofetil and Prednisone: a Pilot Study[NCT00135967]Phase 2/Phase 330 participants Interventional2002-05-31Completed
Randomised, Double-Arm, Controlled, Open-Label Study Comparing Calcineurin Inhibitor-Free Immunosuppression (Zenapax®, CellCept® and Prednisolone) and Cyclosporine A Based Immunosuppression (Sandimmun Neoral®, CellCept® and Prednisolone) on the Outcome of[NCT00138970]Phase 470 participants Interventional2002-01-31Completed
A Prospective Randomized Open-label Study to Compare Mycophenolate Mofetil and A Prospective Randomized Open-label Study to Compare Mycophenolate Mofetil and Corticosteroid With Tacrolimus and Corticosteroid as Immunosuppressive Treatment for Lupus Membra[NCT00404794]Phase 320 participants (Anticipated)Interventional2005-11-30Completed
A Prospective Randomized Open-label Study to Compare Mycophenolate Mofetil and Corticosteroid With Conventional Immunosuppressive Treatment on Proteinuria in Idiopathic Membranous Nephropathy (MN) and Focal Segmental Glomerulosclerosis (FSGS)[NCT00404833]Phase 316 participants (Anticipated)Interventional2003-01-31Completed
[NCT00010361]20 participants Interventional2000-11-30Completed
A Prospective, Randomized Trial of Calcineurin-Inhibitor Withdrawal in Renal Allograft Recipients[NCT00275535]Phase 4165 participants (Actual)Interventional2001-04-30Completed
A Phase I/II Study of the Safety, Tolerability, and Antiretroviral Activity of Mycophenolate Mofetil As an Adjunct to Abacavir Therapy in HIV-Infected Subjects With Treatment Failure and Extensive Prior Antiretroviral Exposure[NCT00021489]Phase 20 participants (Actual)InterventionalWithdrawn
Allogeneic Hematopoietic Cell Transplantation From HLA-matched Donors After Reduced-intensity Conditioning: a Phase II Randomized Study Comparing 2 GVHD Prophylaxis Regimens[NCT01428973]Phase 2200 participants (Actual)Interventional2011-09-30Active, not recruiting
Induction Therapy for Lupus Nephritis With no Added Oral Steroids: An Open Label Randomised Multicentre Controlled Trial Comparing Oral Corticosteroids Plus Mycophenolate Mofetil (MMF) Versus Obinutuzumab and MMF[NCT04702256]Phase 3196 participants (Anticipated)Interventional2021-12-09Recruiting
Calcineurin Inhibitor Sparing Protocol in Living Donor Pediatric Kidney Transplantation[NCT00023231]35 participants (Actual)Interventional2001-02-28Completed
Nonmyeloablative Allogeneic Hematopoietic Cell Transplantation From HLA Matched Unrelated Donors for Treatment of Patients With High Risk Acute Lymphocytic Leukemia in Complete Remission - A Multicenter Trial[NCT00031655]Phase 230 participants (Anticipated)Interventional2001-09-30Completed
Pharmacokinetic Evaluation of Plasmapheresis in Cross Match Positive or ABO Incompatible Kidney Allograft Recipients[NCT00203281]0 participants (Actual)Observational2003-02-28Withdrawn(stopped due to funding withdrawn)
A Randomized Multicenter Trial Comparing Mycophenolate Mofetil and Azathioprine as Remission-maintaining Treatment for Proliferative Lupus Glomerulonephritis. The MAINTAIN Nephritis Trial.[NCT00204022]Phase 3105 participants (Actual)Interventional2001-02-28Completed
A Phase II Multicenter Randomized Study Of Two Non-Myeloablative Stem Cell Transplant Strategies For Low-Grade Lymphoma And Chronic Lymphocytic Leukemia (CLL)[NCT00041288]Phase 210 participants (Actual)Interventional2001-10-31Terminated(stopped due to Poor accrual and difficulty with multicenter logistics)
Prolonged Mycophenolate Mofetil and Truncated Cyclosporine Postgrafting Immunosuppression to Reduce Life-Threatening GVHD After Unrelated Donor Peripheral Blood Cell Transplantation Using Nonmyeloablative Conditioning for Patients With Hematologic Maligna[NCT00078858]Phase 1/Phase 237 participants (Actual)Interventional2003-09-30Completed
Low Dose Total-Body Irradiation And Fludarabine Followed By HLA Matched Allogeneic Stem Cell Transplantation For Hematologic Malgnancies - A Multi-Center Study[NCT00044954]Phase 20 participants Interventional1999-11-30Completed
A Randomized, Open-label Study Comparing the Effects of Low-dose Cyclosporine vs Cyclosporine Withdrawal on Renal Function in Kidney Transplant Patients Treated With CellCept and Daclizumab[NCT00048152]Phase 3539 participants (Actual)Interventional2000-12-31Completed
Lowering Total Immunosuppressive Load in Renal Transplant Recipients More Than 12 Months Posttransplant - Randomised Withdrawal of Mycophenolate Mofetil (CellCept®) or Cyclosporine A (Sandimmun Neoral®)[NCT00148252]Phase 4298 participants Interventional2003-02-28Terminated(stopped due to To high rejection rate in CsA withdrawal arm)
Multicenter, Open-label Follow-up Study on the Safety of Enteric-coated Mycophenolate Sodium in de Novo Renal Transplant Patients[NCT00149929]Phase 3246 participants (Actual)Interventional1999-12-31Completed
Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Renal Transplant Recipients (MyLife)[NCT00149968]Phase 4196 participants (Actual)Interventional2005-04-30Completed
Measurement of Patient Reported Outcomes in Renal Transplant Patients With and Without Gastrointestinal (GI) Symptoms (PROGIS)[NCT00150007]Phase 4335 participants Interventional2004-06-30Completed
Prospective Randomized Study to Characterize Risk Factors of Polyomavirus-related Transplant Nephropathy and the Impact of Three Immunosuppressive Regimens on Nephropathy Incidence[NCT00160966]Phase 4108 participants (Actual)Interventional2004-09-30Completed
The Relationships Between Mycophenolic Acid Levels, T-Cell Subsets and Outcomes in Pediatric Heat Transplant Recipients Receiving Mycophenolate Mofetil (Cellcept)[NCT00166153]30 participants Interventional2003-01-31Terminated
Open-label Study to Evaluate the Tolerability, Safety and Efficacy of the Equimolar Conversion From Mycophenolate Mofetil (MMF) to Enteric-Coated Mycophenolate Sodium (EC-MPS) in Patients With Renal Transplant[NCT00171379]Phase 3162 participants (Actual)Interventional2004-03-31Completed
Pilot Trial for Implementation of a MPA PK Monitoring Strategy[NCT00187915]24 participants (Actual)Interventional2003-07-31Completed
Comparison Of Efficacy Of Two Immunosuppressive Protocols Including Tacrolimus With Or Without Mycophenolate Mofetil In Pediatric Liver Transplantation Aimed In Early Termination Of Steroid Therapy[NCT00195988]Phase 440 participants Interventional2002-09-30Completed
A Prospective, Randomized, Open, Multicentric Study Intended to Evaluate the Efficacy and Tolerability of Sequential Treatment Based on Rabbit Anti-T-lymphocyte Serum, of Mycophenolate Mofetil and of Cyclosporin, Without Concomitant Corticosteroids, After[NCT00200551]Phase 4200 participants Interventional2001-01-31Completed
A Trial of Reduced Intensity Conditioning and Transplantation of Haplotype Mismatched and KIR Class I Epitope-Mismatched Highly Purified CD34 Cells[NCT00085449]Phase 1/Phase 20 participants (Actual)Interventional2006-05-31Withdrawn(stopped due to Funding cut, no patients enrolled)
Post Transplant Infusion of Allogeneic Cytokine Induced Killer Cells as Consolidative Therapy After Non-Myeloablative Allogeneic Transplantation in Patients With Myelodysplasia or Myeloproliferative Disorders[NCT01392989]Phase 244 participants (Actual)Interventional2011-03-31Completed
A Phase II Study of the Combination of Tacrolimus and Mycophenolate Mofetil for the Prevention of Acute Graft-Versus-Host Disease[NCT00096096]Phase 20 participants Interventional2004-08-31Completed
A Prospective Study on the Tolerability and Efficacy of the de Novo Use of Myfortic in Liver Transplant Recipients[NCT00336817]30 participants (Actual)Interventional2006-11-30Completed
Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients[NCT00333437]7 participants (Actual)Interventional2006-05-31Completed
Low-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Cen[NCT00397813]Phase 277 participants (Actual)Interventional2006-01-31Completed
HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide[NCT00358657]Phase 214 participants (Actual)Interventional2006-05-24Terminated(stopped due to Low accrual)
Safety of Tacrolimus And Methotrexate (MTX) Versus Tacrolimus And Mycophenolate Mofetil (MMF) As Graft Versus Host Disease Prophylaxis In Allogeneic Hematopoietic Cell Transplants (HCT)[NCT00360685]89 participants (Actual)Interventional2005-09-30Completed
A 12-month, Prospective, Randomized, Single Center, Open Label Pilot Study to Evaluate the Safety and Efficacy of Myfortic in Combination With Tacrolimus and Thymoglobulin in Early Corticosteroid Withdrawal[NCT00374803]Phase 445 participants (Actual)Interventional2006-04-30Completed
A Single Center, Pilot Study of Corticosteroid Discontinuation in Liver Transplant Recipients Utilizing a Tacrolimus/Mycophenolate Mofetil Based Maintenance Immunosuppression Protocol[NCT00374231]Phase 440 participants (Actual)Interventional2002-10-31Completed
A Non-Myeloablative Conditioning Regimen With Peri-Transplant Rituximab and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With B Cell Lymphoid Malignancies[NCT00387959]Phase 217 participants (Actual)Interventional2006-07-31Completed
A Two Step Approach To Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies From HLA Partially-Matched Related Donors[NCT00429143]Phase 1/Phase 227 participants (Actual)Interventional2006-01-31Completed
A California Cooperative Clinical Study Comparing Allogeneic Hematopoietic Cell Transplantation Using Nonmyeloablative Host Conditioning With Total Lymphoid Irradiation and Anti-thymocyte Globulin Versus Best Standard of Care in Acute Myeloid Leukemia (AM[NCT00568633]Phase 358 participants (Actual)Interventional2007-08-31Terminated(stopped due to Poor accrual)
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Anemia and Other Hemoglobinopathies[NCT00489281]Phase 243 participants (Actual)Interventional2008-06-23Terminated(stopped due to Initiation of CMS BMT study for sickle-cell disease operating under NCT01166009 made further accrual to this study impossible.)
Evaluation of Belatacept as First Line Immunosuppression in De Novo Liver Transplant Recipients[NCT00555321]Phase 2260 participants (Actual)Interventional2008-01-31Terminated
Clinical Outcome of Delayed or Standard Prograf Together With Induction Therapy Followed by Conversion to Advagraf in Donation After Cardiac (or Circulatory) Death (DCD) Kidney Transplant Recipients: A Randomized, Open-label, Multicenter Clinical Trial[NCT03644485]Phase 4284 participants (Actual)Interventional2018-10-21Completed
A Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia[NCT00630253]Phase 1/Phase 231 participants (Actual)Interventional2000-02-17Completed
Phase 0/I Dose Escalation Study of Mycophenolate Mofetil Combined With Radiation Therapy in Glioblastoma[NCT04477200]Phase 168 participants (Anticipated)Interventional2020-08-05Recruiting
Efficacy of Chimeric Monoclonal Anti-CD20 Antibodies (Rituximab Biosimilar) in the Treatment of Childhood Steroid-dependent Nephrotic Syndrome and Development of Cell Biomarkers Predicting Outcome. The RTX 4 Trial.[NCT04402580]Phase 230 participants (Actual)Interventional2019-07-01Terminated(stopped due to due to unexpectedly high rate of relapse in the active comparator arm.)
Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis[NCT00622895]Phase 1/Phase 23 participants (Actual)Interventional2006-09-01Completed
Multicenter, Open-Label, Randomized Study on Steroid-Free Immunosuppression, in Comparison With Daily Steroid Therapy, in Pediatric Renal Transplant : Impact on Growth, Bone Metabolism and Acute Rejection[NCT00707759]Phase 328 participants (Actual)Interventional2008-06-30Completed
Optimization of NULOJIX® (Belatacept) Usage as a Means of Avoiding CNI and Steroids in Renal Transplantation (CTOT-10)[NCT01436305]Phase 219 participants (Actual)Interventional2011-09-30Terminated(stopped due to Secondary to safety concerns plus change in Campath® (alemtuzumab) availability.)
Comparing and Combining Bortezomib and Mycophenolate in SSc Pulmonary Fibrosis Grant Number: R34HL122558[NCT02370693]Phase 29 participants (Actual)Interventional2016-03-31Completed
Transplantation of Two Partially Matched Umbilical Cord Blood Units Following Reduced Intensity Conditioning to Enhance Engraftment and Limit Transplant-Related Mortality in Adults With Hematologic Malignancies[NCT00827099]Phase 25 participants (Actual)Interventional2006-06-30Terminated(stopped due to Unacceptable morbidity & mortality)
A Randomized Phase II Study to Compare the Net Clinical Benefit of Cyclosporine and Sirolimus Combined With MMF or Post-Transplant Cyclophosphamide as GVHD Prophylaxis After HLA-Matched or HLA-Mismatched Unrelated G-CSF Mobilized Blood Cell Transplantatio[NCT03246906]Phase 2160 participants (Anticipated)Interventional2017-09-11Recruiting
The Effects of Mycophenolate Mofetil on Renal Outcomes in Patients With Advanced IgA Nephropathy: a Randomized Open-label Study[NCT01854814]238 participants (Actual)Interventional2013-07-31Completed
"A Feasibility Study of Early Allogeneic Hematopoietic Cell Transplantation for Relapsed or Refractory High-Grade Myeloid Neoplasms"[NCT02756572]Phase 230 participants (Actual)Interventional2016-09-22Completed
Randomized, Open Label, Multicenter Study of Belatacept-based Early Steroid Withdrawal Regimen With Alemtuzumab or rATG Induction Compared to Tacrolimus-based Early Steroid Withdrawal Regimen With rATG Induction in Renal Transplantation[NCT01729494]Phase 4316 participants (Actual)Interventional2012-09-30Completed
A 60 Month, Single-arm, Proof-of-concept Study to Induce Allogeneic Tolerance in Deceased Donor Liver Transplant Recipients Using Siplizumab, an Anti-CD2 Antibody in Combination With Cyclophosphamide and Splenectomy[NCT06019507]Phase 212 participants (Anticipated)Interventional2022-06-29Recruiting
A Phase II Trial of De-escalated PTCy and Ruxolitinib for GVHD Prophylaxis in Patients Undergoing Reduced Intensity Conditioning Allogeneic HCT[NCT05622318]Phase 256 participants (Anticipated)Interventional2023-08-29Recruiting
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome[NCT05027945]Phase 237 participants (Anticipated)Interventional2023-02-23Recruiting
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis[NCT04384692]Phase 245 participants (Anticipated)Interventional2020-12-18Recruiting
Randomized Double-Blind Placebo-Controlled Clinical Trial to Assess the Efficacy of Mycophenolate Mofetil in Subclinical Interstitial Lung Disease Associated With Systemic Sclerosis: a Feasibility Study[NCT05785065]Phase 235 participants (Anticipated)Interventional2023-12-31Not yet recruiting
A Randomized, Placebo-controlled, Double-blind, Multicenter Study Investigating Basiliximab in Combination With MMF, Cyclosporine Microemulsion and Prednisone in the Prevention of Acute Rejection in Pediatric Renal Allograft Recipients[NCT00228020]Phase 3212 participants (Actual)Interventional2001-05-31Completed
APOMYGRE : Multicenter, Randomized, Open-Label Study of MMF Therapeutic Follow-up's Interest in the the 12 First Months in Kidney Transplantation[NCT00199667]Phase 4137 participants Interventional2002-10-31Active, not recruiting
A Randomised Clinical Trial of Mycophenolate Mofetil Versus Cyclophosphamide for Remission Induction in ANCA Associated Vasculitis.[NCT00414128]Phase 2/Phase 3140 participants (Actual)Interventional2007-03-31Completed
A One-year, Randomized, Open Label, Parallel Group Study to Investigate the Safety and the Effect of Enteric-coated Mycophenolate Sodium (EC-MPS) in Combination With Either Full Dose or Reduced Dose Cyclosporine Microemulsion in de Novo Kidney Transplant [NCT00238940]Phase 355 participants (Actual)Interventional2003-02-28Completed
A 6-month, 1-arm, Open-label Study to Investigate the Tolerability and Safety of Converting Stable Renal Transplant Recipients Who Receive Tacrolimus With or Without Corticosteroids From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium ([NCT00238979]Phase 450 participants Interventional2003-01-31Completed
A Prospective, Open-label, Multicenter, Follow-up Study on the Efficacy and Safety of Enteric-coated Mycophenolate Sodium Administered in de Novo Kidney Transplant Patients[NCT00239018]Phase 4144 participants (Actual)Interventional2003-04-30Completed
Enteric-Coated Mycophenolate Sodium (EC-MPS) Administration in Maintenance Renal Transplant Patients Receiving Cyclosporine Microemulsion (CsA-ME) and Steroids, for the Withdrawal of Concomitant Steroid Therapy: a Prospective, Open-label, Exploratory Stud[NCT00239057]Phase 323 participants (Actual)Interventional2002-05-31Completed
[NCT01680952]Phase 4158 participants (Actual)Interventional2012-09-30Completed
Development of Population Pharmacokinetic-Pharmacodynamic (PK-PD) Models of Mycophenolic Acid for Bayesian Dose Individualization in Pediatric Kidney Transplant Patients[NCT00281619]29 participants (Actual)Observational2006-01-31Completed
Nonmyeloablative Bone Marrow Transplants in Hematologic Malignancies: Dose Finding Study for Post-Transplant Immunosuppression[NCT00255710]Phase 160 participants (Anticipated)Interventional2002-07-31Completed
Transplantation of Umbilical Cord Blood From Related and Unrelated Donors[NCT00290628]43 participants (Actual)Interventional1999-10-31Terminated(stopped due to Replaced with another study)
An Open, Prospective Study to Assess the Efficacy and Safety of FK506 Combined MMF in the Treatment of Class III,IV,V + IV or V + III Lupus Nephritis[NCT00298506]120 participants (Actual)Interventional2005-09-30Completed
Non-Myeloablative HLA-Matched Sibling Allogeneic Peripheral Blood Stem Cell Transplantation for Metastatic Renal Cell Carcinoma[NCT00262886]Phase 235 participants (Anticipated)Interventional2001-08-31Completed
Open, Multicenter, Randomized Clinical Trial in Patients With Moderate-Severe Psoriasis (PASI > 10) to Compare the Efficacy of Mycophenolate Mofetil Versus Cyclosporine A.[NCT00295425]Phase 250 participants Interventional2000-05-31Active, not recruiting
Treatment of Necrotizing Vasculitides for Patients Older Than 65 Years Comparison of Two Strategies Combining Steroids With or Without Immunosuppressants[NCT00307671]Phase 4108 participants (Actual)Interventional2005-07-31Completed
A Twelve-month, Randomized, Multicenter, Open-label, Exploratory Study to Investigate the Clinical Outcomes of an Immunosuppressive Regimen of Basiliximab, Cyclosporine Microemulsion (CsA-ME) and Enteric-coated Mycophenolate Sodium (EC-MPS) Free of Steroi[NCT00284921]Phase 360 participants Interventional2004-04-30Terminated
A Trial of Mycophenolate Mofetil in Myasthenia Gravis[NCT00285350]Phase 380 participants Interventional2002-09-30Completed
Non-myeloablative Allogeneic Stem Cell Transplantation With Match Unrelated Donors for Treatment of Hematologic Malignancies and Renal Cell Carcinoma and Aplastic Anemia[NCT00295997]35 participants (Anticipated)Interventional2005-05-31Active, not recruiting
An Open, Randomised, Multicentre Clinical Study to Investigate the Safety and Efficacy of Steroid Withdrawal With Tacrolimus, Mycophenolate Mofetil and Daclizumab Against Tacrolimus, Mycophenolate Mofetil and Steroids in Children After Kidney Transplantat[NCT00296348]Phase 3198 participants (Actual)Interventional2005-11-30Completed
Desensitization of Renal Transplant Candidates[NCT00298883]Phase 19 participants (Actual)Interventional2006-02-28Completed
A Prospective, Randomized, Open-label Study Evaluating the Efficacy of Mycophenolate Mofetil in the Prevention of Relapse of Steroid Dependent Nephrotic Syndrome in Children[NCT01895894]Phase 434 participants (Actual)Interventional2013-09-30Completed
MMF Versus Intravenous CTX Pulses in the Treatment of Adult Severe Henoch-Schonlein Purpura Nephritis[NCT00301613]60 participants (Actual)Interventional2003-01-31Completed
Mycophenolate Mofetil Versus Cyclophosphamide in the Induction Treatment of ANCA Associated Vasculitis[NCT00301652]60 participants (Actual)Interventional2003-06-30Completed
Safety and Efficacy of Low-dose Cyclosporine in Association With Everolimus to Minimize Renal Dysfunction in Heart Transplant Recipients[NCT00420537]Phase 434 participants (Actual)Interventional2006-09-30Terminated(stopped due to A cluster of adverse events in everolimus arm was noted.)
A Prospective, Open Label, Multicenter Study to Assess the Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients[NCT00312143]Phase 435 participants (Actual)Interventional2004-02-29Completed
Myfortic® Monotherapy to Prevention of de Novo Allosensitization in Islet Transplant Recipients Following Complete Graft Loss[NCT01999361]18 participants (Anticipated)Interventional2009-01-31Recruiting
A Phase I Study of 90Y-BC8-DOTA Monoclonal Antibody, Fludarabine and TBI Followed by HLA-Matched, Allogeneic Peripheral Blood Stem Cell Transplant for the Treatment of Multiple Myeloma[NCT01503242]Phase 115 participants (Actual)Interventional2012-01-09Completed
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders[NCT04644016]Phase 231 participants (Anticipated)Interventional2020-11-20Recruiting
HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3 Depletion for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors After Conditioning of TBI, Fludarabine, Cyclophosphamide and Antithymocyte Glo[NCT01509300]Phase 1/Phase 210 participants (Anticipated)Interventional2012-01-31Recruiting
Hematopoietic Stem Cell Transplantation Using Alternate Donor Umbilical Cord Blood Options[NCT01652014]Phase 20 participants (Actual)Interventional2014-01-31Withdrawn(stopped due to Funding unavailable)
Immune Tolerance and Alloreactivity in Liver Transplant Recipients on Different Monotherapy Immunosuppressive Agents[NCT01678937]31 participants (Actual)Observational2007-09-30Completed
A Prospective, Randomised, Double-blind, Placebo-controlled, Parallel Group, Mult-center, 52-week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Achieve Remission With Reduced Corticosteroid in Subjects With Pemphigus Vu[NCT00683930]Phase 396 participants (Actual)Interventional2004-05-31Completed
A Phase I/II Study Evaluating the Safety and Efficacy of Adding a Single Prophylactic Donor Lymphocyte Infusion (DLI) of Natural Killer Cells Early After Nonmyeloablative, HLA-Haploidentical Hematopoietic Cell Transplantation - A Multi Center Trial[NCT00789776]Phase 1/Phase 241 participants (Actual)Interventional2008-10-13Completed
Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950[NCT00579527]Phase 1/Phase 214 participants (Actual)Interventional2005-12-19Completed
[NCT01706471]Phase 460 participants (Actual)Interventional2009-06-30Completed
A Phase 1, Open-Label, Sequential Study of the Effect of Multiple Doses of Isavuconazole on the Pharmacokinetics of a Single Dose of Mycophenolate Mofetil in Healthy Adult Subjects[NCT01711489]Phase 124 participants (Actual)Interventional2012-03-31Completed
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of BMS-188667 (Abatacept) or Placebo on a Background of Mycophenolate Mofetil and Corticosteroids in the Treatment of Subjects With Active Class III or IV Lup[NCT01714817]Phase 3695 participants (Actual)Interventional2013-01-22Terminated(stopped due to Inability to meet protocol objectives.)
Multicenter, Open-label, Parallel Clinical Investigation of the Safety and Efficacy of Advagraf® (Extended Release Tacrolimus) vs. Prograf® (Tacrolimus) in de Novo Kidney Recipients 1 Month After Kidney Transplantation[NCT01742624]Phase 460 participants (Actual)Interventional2012-04-30Completed
A Multi-center, Open, Randomized-control Study to Compare the Effects and Safety of Idarubicin-strengthened Pretreatment Program and Conventional Busulfan Cyclophosphamide Pretreatment Program on High-risk Acute Myeloid Leukemia Patient[NCT01766375]Phase 3200 participants (Anticipated)Interventional2012-08-31Recruiting
Open Label, Multicenter, Non-comparative Study to Evaluate the Efficacy and Safety of MY-REPT Capsule in Primary, Liver Transplantation Recipients.[NCT01766518]Phase 4120 participants (Anticipated)Interventional2009-11-30Recruiting
A Single-Dose, Replicate, Comparative Bioavailability Study of Two Formulations of Mycophenolate Mofetil 500 mg Tablets Under Fasting Conditions[NCT00907907]Phase 140 participants (Actual)Interventional2006-07-31Completed
Phase 3 Open Label Randomised Multicentre Controlled Trial of Rituxmab and Mycophenolate Mofetil Without Oral Steroids for the Treatment of Lupus Nephritis[NCT01773616]Phase 324 participants (Actual)Interventional2015-04-30Terminated(stopped due to Study assessments for patients recruited continuing per protocol so patients receive a minimum of 6 months follow up. No safety concerns have been raised.)
A Phase II Study Evaluating Selective Depletion of CD45RA+ T Cells From Allogeneic Peripheral Blood Stem Cell Grafts From HLA-Matched Related and Unrelated Donors for Prevention of GVHD[NCT02220985]Phase 284 participants (Actual)Interventional2015-02-03Active, not recruiting
Prospective Evaluation of the Efficacy and Safety of Zortress (Everolimus)/Myfortic (Enteric Coated Mycophenolate Sodium) Conversion in High MELD Liver Transplantation[NCT01807767]0 participants (Actual)Interventional2013-03-31Withdrawn(stopped due to funding was withdrawn)
Kidney Graft Function Under the Immunosuppression Strategies With Low Dose of Neoral®(Cyclosporine) and Standard Dose of Myfortic®(Enteric-Coated Mycophenolate Sodium) vs. With Conventional Dose of Neoral®(Cyclosporine) and Reduced Dose of Myfortic®(Enter[NCT01817322]Phase 4140 participants (Actual)Interventional2011-06-30Completed
A Prospective Study to Investigate Mycophenolic Acid (MPA) Exposure Through Area Under the Curve (AUC) in Renal Transplants Recipients Treated With Mycophenolate Mofetil (MMF) and After Conversion to Mycophenolate Sodium (EC-MPS)[NCT01822483]Phase 4100 participants (Actual)Interventional2013-04-30Completed
Open Label, Randomized, Multi-center, Phase 4 Trial to Evaluate the Efficacy and Safety of My-Rept® Tablet(Mycophenolate Mofetil 500mg/Tab.) Versus My-Rept® Capsule(Mycophenolate Mofetil 250mg/Cap.) in Combination With Tacrolimus for 26 Weeks in Kidney Tr[NCT01842269]Phase 4156 participants (Actual)Interventional2013-01-31Completed
A Randomized Phase III Study to Determine the Most Promising Postgrafting Immunosuppression for Prevention of Acute GVHD After Unrelated Donor Hematopoietic Cell Transplantation Using Nonmyeloablative Conditioning for Patients With Hematologic Malignancie[NCT01231412]Phase 3174 participants (Actual)Interventional2010-11-30Completed
A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes[NCT01871441]Phase 24 participants (Actual)Interventional2013-05-17Terminated(stopped due to Trial was closed due to poor accrual.)
To Compare the Efficacy of Tacrolimus and Mycophenolate Mofetil for the Initial Therapy of Active Lupus Nephritis[NCT00371319]Phase 4150 participants (Actual)Interventional2005-09-30Completed
An Open Label Study of the Effects of a Combination of NeoRecormon, CellCept and Prednisone on Hematological Parameters and Cytogenesis in Patients With Low or Intermediate Risk Myelodysplastic Syndromes.¿[NCT00551291]Phase 210 participants (Actual)Interventional2007-08-31Completed
A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Disease and Other Hemoglobinopathies[NCT01850108]21 participants (Actual)Interventional2013-05-31Active, not recruiting
Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)[NCT01626092]3 participants (Actual)Interventional2012-07-11Completed
Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation as Second Transplantation for Patients With Disease Relapse or Myelodysplasia After Prior Autologous Transplantation[NCT01118013]Phase 26 participants (Actual)Interventional2010-12-31Terminated
A Phase II, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescent Patients With Active Class III or IV Lupus Nephritis, Including an Evaluation of Open Label Sa[NCT05039619]Phase 240 participants (Anticipated)Interventional2022-05-12Recruiting
Haploidentical Donor Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation With a TLI Based Conditioning Regimen in Patients With Hematologic Malignancies[NCT01807611]Phase 282 participants (Actual)Interventional2013-05-16Completed
A Randomized, Multicenter, Open-label Phase II Trial to Compare Prophylaxis of Graft Versus Host Disease With Tacrolimus and Mycophenolate Mofetil Versus Ruxolitinib After Post-transplant Cyclophosphamide[NCT04669210]Phase 2128 participants (Actual)Interventional2020-11-03Active, not recruiting
A Phase Ib/2 Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell Transplantation[NCT03192397]Phase 1/Phase 235 participants (Actual)Interventional2017-08-09Active, not recruiting
T-Cell Replete Haploidentical Donor Hematopoietic Stem Cell Plus Natural Killer (NK) Cell Transplantation in Patients With Hematologic Malignancies Relapsed or Refractory Despite Previous Allogeneic Transplant[NCT01621477]Phase 234 participants (Actual)Interventional2012-08-31Terminated(stopped due to Study was terminated in August 2016 due to replacement by a new study.)
Once Daily Dosing to Improve Medication Adherence and Patient Satisfaction in Kidney Transplant Recipients: A Pilot Study[NCT02426502]76 participants (Actual)Interventional2016-04-30Active, not recruiting
Phase 1 Pilot Study Using Autologous CD4+CD25+FoxP3+ T Regulatory Cells and Campath-1H to Induce Renal Transplant Tolerance[NCT01446484]Phase 1/Phase 230 participants (Anticipated)Interventional2011-10-31Recruiting
A Randomised Prospective Open Label Pilot Trial Comparing Mycophenolate Mofetil (MMF) With no Immunosuppression in Adults With Limited Cutaneous Systemic Sclerosis[NCT04927390]Phase 2120 participants (Anticipated)Interventional2021-12-08Active, not recruiting
A Phase II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation From Matched Unrelated Donors in Patients With Advanced Hematologic Malignancies and Hematological Disorders[NCT00544115]Phase 2260 participants (Actual)Interventional2001-10-16Active, not recruiting
An Open-label Study of the Pharmacokinetics of Mycophenolic Acid as Myfortic (Enteric-coated Mycophenolate Sodium) When Used in Combination With Prograf (Tacrolimus) and Corticosteroids in Patients Undergoing de Novo Liver Transplantation[NCT01467011]25 participants (Actual)Observational2010-12-31Completed
Investigating the Links Between Microbiota Composition and Variability Observed in the Pharmacological Response to Immunosuppressive Therapies in Kidney Transplant Patients.[NCT04360031]100 participants (Anticipated)Observational2020-02-10Recruiting
A Multicenter Clinical Trial: Risk Factors of Chinese Kidney Transplant Recipients DSA Based on MPA Immunosuppressive Regimen[NCT04444843]Phase 4300 participants (Anticipated)Interventional2021-01-22Recruiting
Prospective, Multicenter, Randomized Open Study to Evaluate the Progression of Renal Graft Fibrosis According to the Epithelial-mesenchymal Transition (EMT) in de Novo Renal Transplant Recipients Treated Either by a CNI Free Immunosuppressive Regimen With[NCT01079143]Phase 3194 participants (Actual)Interventional2009-09-30Completed
Clinical Evaluation of New Treatment Strategy of Mucous Membrane Pemphigoid Using Large Dose of Prednisolone Plus Intra-lesional of Triamcinolone Acetonide Followed by Combination of Mycophenolate Mofetil, Dapsone and Low Dose Prednisolone[NCT04744623]Phase 2/Phase 310 participants (Actual)Interventional2020-09-30Active, not recruiting
A Randomized Controlled Clinical Trial of Low Dose Thymoglobulin and Extended Delay of Calcineurin Inhibitor Therapy for Renal Protection After Liver Transplantation[NCT00970073]30 participants (Actual)Interventional2009-08-31Completed
Induction of Mixed Hematopoietic Chimerism in Older Patients With B-Cell Malignancies and in Selected Other Diseases, Using Low Dose TBI , PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to be Followed by Do[NCT00003196]63 participants (Actual)Interventional1997-09-30Completed
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Human Leukocyte Antigen Partially-Matched Related Donor[NCT01341301]Phase 225 participants (Actual)Interventional2010-05-31Completed
Nonmyeloablative Conditioning With Pre- and Post-Transplant Rituximab Followed by Related or Unrelated Donor Hematopoietic Cell Transplantation for Patients With Advanced Chronic Lymphocytic Leukemia: A Multi-Center Trial[NCT00104858]Phase 266 participants (Actual)Interventional2004-12-31Completed
Campath (Alemtuzumab) Dose Escalation, Low-Dose TBI and Fludarabine Followed by HLA Class II Mismatched Donor Stem Cell Transplantation for Patients With Hematologic Malignancies: A Multicenter Trial[NCT00118352]Phase 212 participants (Actual)Interventional2005-03-31Completed
A Randomized, Open-label Study of the Effect of a Long-term Calcineurin Inhibitor-free Maintenance Regimen With CellCept and Sirolimus on Preservation of Renal Function and Prevention of Acute Rejection in Recipients of an Orthotropic Liver Transplant[NCT00118742]Phase 4293 participants (Actual)Interventional2005-08-31Completed
Open Label Multicenter Randomized Trial Comparing Standard Immunosuppression With Tacrolimus and Mycophenolate Mofetil With a Low Exposure Tacrolimus Regimen in Combination With Everolimus in de Novo Renal Transplantation in Elderly Patient[NCT03797196]Phase 4374 participants (Anticipated)Interventional2019-07-29Active, not recruiting
A Study to Evaluate the Efficacy of CellCept, Administered in a Sequential Treatment Scheme, in Delaying Progressive Renal Damage in Patients With Lupus Nephritis[NCT02081183]Phase 316 participants (Actual)Interventional2006-03-31Terminated
Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients:A Large, Multiple-Center Prospective Study[NCT05872568]Phase 4270 participants (Anticipated)Interventional2023-06-01Not yet recruiting
Donor Statin Treatment for Prevention of Severe Acute GVHD After Nonmyeloablative Hematopoietic Cell Transplantation[NCT01527045]Phase 247 participants (Actual)Interventional2012-09-25Completed
A Randomized Study to Evaluate The Efficacy of Mycophenolate Mofetil Added to The Systemic Immunosuppressive Regimen First Used For Treatment of Chronic Graft-Versus-Host Disease[NCT00089141]Phase 3151 participants (Actual)Interventional2004-05-31Terminated(stopped due to Low probability of positive outcome)
A Multi-Center Study of Nonmyeloablative Conditioning With TBI or Fludarabine/TBI for HLA-matched Related Hematopoietic Cell Transplantation for Treatment of Hematologic Malignancies With Post Grafting Immunosuppression With Tacrolimus and Mycophenolate M[NCT00089011]Phase 2150 participants (Actual)Interventional2004-04-30Completed
Open-label, Multicenter Phase II Study of Combination Therapy of Imatinib Mesylate and Mycophenolate Mofetil in Children With Steroid-Refractory Sclerotic/Fibrotic Type Chronic Graft-versus-host Disease[NCT01898377]Phase 29 participants (Actual)Interventional2013-08-31Terminated(stopped due to New treatment option introduced for patients with the study indication)
A Randomized Phase II Study to Determine the Most Promising Postgrafting Immunosuppression for Prevention of Acute GVHD After Unrelated Donor G-CSF Mobilized Peripheral Blood Mononuclear Cell (G-PBMC) Transplantation Using Nonmyeloablative Conditioning fo[NCT00105001]Phase 2210 participants (Actual)Interventional2004-11-30Completed
Reduced-Intensity Allogeneic HSC Transplantation From HLA-Matched Related and Unrelated Donors for Patients With Multiple Myeloma - A Multi-Center Trial[NCT00054353]Phase 1/Phase 216 participants (Actual)Interventional2002-10-31Completed
Nonmyeloablative Allogeneic Peripheral Blood Stem Cell Transplantation From HLA Matched Related Donors for Treatment of Older Patients With De Novo or Secondary Acute Myeloid Leukemia in First Complete Remission[NCT00045435]Phase 217 participants (Actual)Interventional2002-04-30Completed
Safety, Efficacy and Feasibility of Haploidentical Stem Cell Transplantation (Haplo-SCT) Using Post-Transplant Cyclophosphamide (PTCy) as an Alternative Donor Source for Patients Who Lack a Matched Sibling/Unrelated Donor Options[NCT03088709]Phase 240 participants (Anticipated)Interventional2017-01-18Recruiting
Pentostatin and Donor Lymphocyte Infusion for Low Donor T-cell Chimerism After Hematopoietic Cell Transplantation - A Multi-center Trial[NCT00096161]Phase 236 participants (Actual)Interventional2003-05-31Completed
Randomized Clinical Trial to Compare Efficacy and Safety of Repeated Courses of Rituximab to That of Maintenance Mycophenolate Mofetil Following Single Course of Rituximab in Maintaining Remission Over 24 Months Among Children With Steroid Dependent Nephr[NCT03899103]Phase 3100 participants (Anticipated)Interventional2019-05-15Active, not recruiting
Tacrolimus In Combination, Tacrolimus Alone Compared (TICTAC Trial): A Prospective Randomized Trial Of Minimized Immunosuppression In Adult Heart Transplant Recipients[NCT00299221]Phase 4150 participants (Actual)Interventional2004-04-30Completed
A Phase III Randomized Open-label Multi-center Study of Ruxolitinib vs. Best Available Therapy in Patients With Corticosteroid-refractory Chronic Graft vs Host Disease After Allogeneic Stem Cell Transplantation (REACH3)[NCT03112603]Phase 3330 participants (Actual)Interventional2017-06-29Completed
A Study of the Safety, Tolerability and Pharmacokinetics of Oral CellCept® (Mycophenolate Mofetil, MMF) in Pediatric Liver Transplant Recipients on Concomitant Treatment With Cyclosporine and Corticosteroids[NCT02630563]Phase 29 participants (Actual)Interventional2003-05-31Terminated(stopped due to Due to extremely slow recruitment, infrequent use of combination triple therapy (MMF, cyclosporine, steroids), study was discontinued; Part 2 was not conducted.)
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies[NCT01384513]Phase 240 participants (Actual)Interventional2011-08-04Completed
A Pilot Study of Post-transplant High Dose Cyclophosphamide (PTCY) as Part of Graft-Versus-Host Disease (GVHD) Prophylaxis in T-Cell Replete HLA-Mismatched Unrelated Donor (MMUD) Ablative and Reduced Intensity Hematopoietic Cell Transplantation (HCT) for [NCT03128359]Phase 238 participants (Actual)Interventional2017-05-30Active, not recruiting
Reduced Intensity, Partially HLA Mismatched Allogeneic BMT for Hematologic Malignancies Using Donors Other Than First-degree Relatives[NCT01203722]Phase 1/Phase 2100 participants (Anticipated)Interventional2010-09-30Recruiting
Randomized, Multicenter, Open-label, Comparative Study of Efficacy and Safety of Treatment With a Calcineurin Inhibitor (CNI), Associating Myfortic ® and Neoral ® Compared to a CNI-free Treatment, Combining Myfortic ® and Certican ® , in Adult Patients Wi[NCT01595984]Phase 390 participants (Actual)Interventional2012-05-03Active, not recruiting
Observational Study of Visual Outcomes in Retinal Disease[NCT01613963]2,000 participants (Anticipated)Observational2012-05-31Enrolling by invitation
A Controlled Randomized Open-label Multicenter Study Evaluating if Early Conversion to Everolimus (Certican) From Cyclosporine (Neoral) in de Novo Renal Transplant Recipients Can Improve Long-term Renal Function and Slow Down the Progression of Chronic Al[NCT00634920]Phase 4204 participants (Actual)Interventional2008-03-31Completed
Belatacept Therapy for the Failing Renal Allograft[NCT01921218]Phase 313 participants (Actual)Interventional2013-08-31Completed
A Prospective Cross-over Study Comparing the Effect of Sirolimus Versus Mycophenolate on Viral Load in Liver Transplant Recipients With Recurrent Chronic HCV Infection[NCT01134952]Phase 411 participants (Actual)Interventional2010-06-30Completed
Allogeneic Nonmyeloablative Hematopoietic Stem Cell Transplant for Patients With BCR-ABL Tyrosine Kinase Inhibitor Responsive Ph+ Acute Leukemia ? A Multi-center Trial[NCT00036738]Phase 228 participants (Actual)Interventional2001-07-13Completed
Non-Myeloablative Conditioning and Unrelated Umbilical Cord Blood Transplantation for Children and Adults With Serious Oncohematologic Diseases[NCT00255684]16 participants (Actual)Interventional2003-12-31Terminated(stopped due to low accrual)
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched Bone Marrow for Patients With Hematologic Malignancies[NCT00134004]Phase 2210 participants (Actual)Interventional2004-10-31Completed
Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Non-Myeloablative Preparative Regimen[NCT00305682]Phase 2295 participants (Actual)Interventional2005-06-30Completed
Donor Specific Regulation (DSR) Guided Tacrolimus Withdrawal to Myfortic Monotherapy in Liver Transplantation[NCT01230502]11 participants (Actual)Interventional2011-11-30Terminated(stopped due to insufficient study population to meet study objective)
First-line Antimetabolites as Steroid-sparing Treatment Uveitis Pilot Trial[NCT01232920]Phase 380 participants (Actual)Interventional2010-10-31Completed
A Randomized, Open Label, Four-way Crossover Study to Compare the Pharmacokinetics of Mycophenolate Mofetil Metabolites From Four Tablet Formulations in Healthy Subjects[NCT02981290]Phase 132 participants (Actual)Interventional2008-07-31Completed
Sirolimus and Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation[NCT00350181]Phase 211 participants (Actual)Interventional2006-08-31Completed
An Open, Multicentre, Randomised, Parallel Group Study to Compare in Marginal Old-for-old Renal Transplant Patients the Safety and Efficacy of Two Treatments: Sequential Mycophenolate Mofetil/Delayed Tacrolimus Plus Steroids vs Tacrolimus Plus Mycophenola[NCT00321113]Phase 3142 participants (Actual)Interventional2004-09-30Completed
A Pilot Study of Corticosteroid-Free, Calcineurin-Sparing Immunosuppression Protocol for HLA-Identical Living Donor Renal Transplant Recipient[NCT00352092]Phase 420 participants (Actual)Interventional2002-06-30Completed
A Randomized, Open-label, Parallel-Group Multicenter Study to Determine the Safety/Efficacy of Mycophenolate Mofetil in Mono & Combination Therapy With Interferon Beta 1a in Patients With Relapsing Remitting Multiple Sclerosis[NCT00324506]Phase 243 participants (Actual)Interventional2006-05-31Completed
A Phase II Trial of High-Dose 90Y-Ibritumomab Tiuxetan (Anti-CD20) Followed by Fludarabine and Low-Dose Total Body Irradiation and HLA-Matched Allogeneic Hematopoietic Transplantation for Patients With Relapsed or Refractory Aggressive B-Cell Lymphoma[NCT01434472]Phase 220 participants (Actual)Interventional2011-11-16Terminated(stopped due to Terminated due to insufficient funding)
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using Two Related Donors[NCT01532635]Phase 24 participants (Actual)Interventional2012-03-31Terminated(stopped due to Slow accrual)
Randomized Controlled Trial of Infliximab (Remicade®) Induction Therapy for Deceased Donor Kidney Transplant Recipients (CTOT-19)[NCT02495077]Phase 2290 participants (Actual)Interventional2015-11-02Completed
Impact of Two Prednisone-free Maintenance Immunosuppressive Regimens With Reduced Dose FK506+Everolimus vs. Standard Dose Tacrolimus (FK506)+ Mycophenolate Mofetil (MMF) on Subpopulation of T and B Cells, Renal Allograft Function and Gene Expression Profi[NCT01653847]88 participants (Actual)Interventional2013-02-28Completed
A 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity Conditioning[NCT05031897]Phase 267 participants (Anticipated)Interventional2021-10-25Recruiting
Non-Myeloablative Allogeneic Bone Marrow Transplant for Hematologic Malignancies[NCT00003572]Phase 220 participants (Anticipated)Interventional1998-08-31Completed
Donor-Alloantigen-Reactive Regulatory T Cell (darTreg) Therapy in Liver Transplantation (RTB-002)[NCT02188719]Phase 115 participants (Actual)Interventional2014-12-17Terminated(stopped due to The trial could not be completed within the grant timeline.)
Induction of Mixed Hematopoietic Chimerism Using Fludarabine, Low Dose TBI , PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to be Followed by Donor Lymphocyte Infusion In Patients With Chronic Myeloid Leuke[NCT00003145]Phase 218 participants (Actual)Interventional1997-08-31Completed
A Randomized Trial Comparing Methotrexate Versus Mycophenolate Mofetil for Remission Maintenance in Wegener's Granulomatosis and Related Vasculitides[NCT00004567]Phase 275 participants Interventional2000-02-29Completed
TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study[NCT04182984]200 participants (Anticipated)Observational [Patient Registry]2019-11-04Recruiting
Bone Marrow Transplantation and High Dose Post-Transplant Cyclophosphamide for Chimerism Induction and Renal Allograft Tolerance (ITN054ST)[NCT02029638]Phase 24 participants (Actual)Interventional2014-01-07Terminated(stopped due to Slow accrual)
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Haploidentical Donor[NCT01982682]Phase 241 participants (Actual)Interventional2013-11-04Completed
A Randomized, Double-Blind, Double-Dummy, Active-Comparator, Multicenter Study to Evaluate the Efficacy and Safety of Rituximab Versus MMF in Patients With Pemphigus Vulgaris[NCT02383589]Phase 3135 participants (Actual)Interventional2015-05-26Completed
De Novo Everolimus Versus Tacrolimus in Combination With Mofetil Mycophenolate and Low Dose Corticosteroids to Reduce Tacrolimus Induced Nephrotoxicity in Liver Transplantation: a Prospective, Multicentric, Randomised Study[NCT02909335]Phase 30 participants (Actual)Interventional2016-11-30Withdrawn
Multi-center, Randomized, Open Label, Comparative, Phase IV Study to Evaluate the Efficacy and Safety of a Combination of Mycophenolate Mofetil and Corticosteroid for 48 Weeks in Advanced IgA Nephropathy[NCT02981212]Phase 4100 participants (Anticipated)Interventional2016-06-30Recruiting
A Multicenter, Randomized, Double-Masked Study To Evaluate The Safety, Tolerability, And Efficacy Of SURF-100 Ophthalmic Solution (A Mycophenolic Acid/Betamethasone Sodium Phosphate Combination) In Subjects With Dry Eye Disease[NCT04734197]Phase 2351 participants (Actual)Interventional2021-01-11Completed
A Randomized Prospective Trial of Conversion to Everolimus Therapy From Calcineurin Inhibitor Based Maintenance Immunosuppression in Association With Mycophenolic Acid in Liver Transplantation: Examination of Impact on Long Term Renal Function.[NCT01936519]24 participants (Actual)Interventional2013-12-16Completed
A 12-month Randomized, Multiple Dose, Open-label, Study Evaluating Safety, Tolerability, Pharmacokinetics/Pharmacodynamics (PK/PD) and Efficacy of an Anti-CD40 Monoclonal Antibody, CFZ533, in Combination With Mycophenolate Mofetil (MMF) and Corticosteroid[NCT02217410]Phase 1/Phase 259 participants (Actual)Interventional2015-02-05Completed
A Phase II Trial of Non-Myeloablative (NMA) Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Donor (MUD) Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia[NCT02833805]Phase 221 participants (Actual)Interventional2016-09-30Completed
Impact of the Microbiota on the Likelihood of Renal Graft Rejection[NCT04736381]70 participants (Anticipated)Observational2021-04-14Not yet recruiting
Gemcitabine/Clofarabine/Busulfan and Allogeneic Transplantation for Aggressive Lymphomas[NCT01701986]Phase 1/Phase 280 participants (Anticipated)Interventional2012-10-25Active, not recruiting
A Phase II Study of Reduced Intensity Allogeneic Bone Marrow Transplantation for the Treatment of Relapsed Non-Hodgkin and Hodgkin Lymphoma[NCT00057954]Phase 26 participants (Actual)Interventional2005-11-09Terminated(stopped due to Slow accrual)
A Phase II Study of Reduced Intensity Allogeneic Bone Marrow Transplant for the Treatment of Myelodysplastic Syndromes[NCT00045305]Phase 217 participants (Actual)Interventional2006-10-24Completed
Hematopoietic Bone Marrow Transplantation for Patients With High-risk Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), or Myelodysplastic Syndrome (MDS) Using Related HLA-Mismatched Donors: A Trial Using Radiolabeled Anti-CD45 Antibody Co[NCT00589316]Phase 126 participants (Actual)Interventional2007-10-05Terminated(stopped due to Terminated due to loss of funding)
Donor-derived Mesenchymal Stromal Cells, Alemtuzumab, Co-stimulation Blockade and Sirolimus for Tolerance Induction in Adult Kidney Allograft Recipients (ITN062ST)[NCT03504241]Phase 18 participants (Actual)Interventional2018-07-30Active, not recruiting
Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease[NCT00004485]Phase 1/Phase 250 participants Interventional1999-12-31Completed
Low-Dose TBI and Fludarabine Followed by Unrelated Donor Stem Cell Transplantation for Patients With Hematologic Malignancies and Renal Cell Carcinoma - A Multi-center Trial[NCT00005799]55 participants (Actual)Interventional1999-11-30Completed
Post Transplant Infusion of Allogeneic CD8 Memory T-Cells as Consolidative Therapy After Non-myeloablative Allogeneic Hematopoietic Cell Transplantation in Patients With Leukemia and Lymphoma[NCT02424968]Phase 218 participants (Actual)Interventional2015-06-30Completed
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen[NCT00365287]Phase 1/Phase 2148 participants (Actual)Interventional2000-06-30Completed
A Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haploidentical Peripheral Blood Stem Cell Transplantation[NCT03018223]Phase 132 participants (Actual)Interventional2017-01-31Completed
Once-daily Regimen With Envarsus® to Optimize Immunosuppression Management and Outcomes in Kidney Transplant Recipients[NCT02954198]40 participants (Actual)Interventional2016-12-01Completed
Open Label Randomized Single Study to Evaluate the Safety & Efficacy of Early CNI Withdrawal in Recipients of Primary Renal Allografts Maintained Long-Term on Mycophenolate Mofetil; MMF (CellCept) and Sirolimus (Rapamune)[NCT00374647]Phase 417 participants (Actual)Interventional2005-03-31Completed
Dosing Requirements of Astagraf XL® in African American Kidney Transplant Recipients Converted From Twice-daily Tacrolimus[NCT02953873]Phase 425 participants (Actual)Interventional2017-05-05Completed
NEw Clinical Endpoints in Patients With Primary Sjögren's Syndrome (pSS): an Interventional Trial Based on stratifYing Patients[NCT05113004]Phase 2300 participants (Anticipated)Interventional2022-01-20Recruiting
Umbilical Cord Blood Transplant With Co-Infusion of T Regulatory Cells[NCT00376519]Phase 13 participants (Actual)Interventional2007-05-31Terminated(stopped due to Slow accrual)
A Multi-center Phase III Study Comparing Myeloablative to Nonmyeloablative Transplant Conditioning in Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia[NCT00322101]Phase 325 participants (Actual)Interventional2006-01-31Completed
A Multicenter, Randomization, Open-label Phase 4 Clinical Trial to Evaluate the Efficacy and Safety of MYREPTIC-N® or MY-REPT® in Stable Patients After Kidney Transplant Recipients[NCT06044493]Phase 4158 participants (Anticipated)Interventional2023-11-01Not yet recruiting
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Obinutuzumab in Patients With ISN/RPS 2003 Class III or IV Lupus Nephritis[NCT02550652]Phase 2126 participants (Actual)Interventional2015-11-13Completed
Multi-Center, Open-Label Randomized Study of Single or Double Myeloablative Cord Blood Transplantation With or Without Infusion of Off-The-Shelf Ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells in Patients With Hematologic Malignancies[NCT01690520]Phase 2163 participants (Actual)Interventional2012-12-11Completed
Compare the Safety and Effective of HLA-mismatched Microtransplantation With HLA-matched Nonmyeloablative Transplantation for Acute Myeloid Leukemia in Intermediate-risk[NCT02461121]Phase 3156 participants (Actual)Interventional2004-05-31Completed
Effect of the Introduction of mTor Inhibitors in the Activation of the Cytomegalovirus (CMV) -Specific Cellular Immune Response to Control Viral Replication in Kidney Transplant Patients[NCT04936971]Phase 446 participants (Anticipated)Interventional2021-09-30Not yet recruiting
A Randomized, Multicenter Study of Belatacept, Everolimus, rATG and Early Steroid Withdrawal Versus Belatacept, Mycophenolate, rATG and Chronic Steroids in Renal Transplantation[NCT04849533]Phase 4120 participants (Anticipated)Interventional2021-04-09Recruiting
Mycophenolate Mofetil Versus Cyclophosphamide in the Induction Therapy of Pediatric Patients With Active Proliferative Lupus Nephritis: A Prospective, Randomized, Multicenter, Open-label, Parallel-arm Study[NCT05495893]Phase 4224 participants (Anticipated)Interventional2022-07-25Recruiting
Comparing the Effects of an Immunosuppressant (Mycophenolate Mofetil or MMF) on the Urinary Sodium Excretion Response to Mental Stress in a Crossover Design (MMF)[NCT02432339]Phase 1/Phase 250 participants (Anticipated)Interventional2014-04-22Recruiting
Pilot Study of Infusion of Fucosylated Regulatory T Cells to Prevent Graft Versus Host Disease[NCT02423915]Phase 15 participants (Actual)Interventional2015-07-30Completed
A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT C[NCT03959241]Phase 3428 participants (Anticipated)Interventional2019-06-25Active, not recruiting
Prospective, Multicenter, Randomized, Evaluating Two Induction Therapies With Simulect® Versus ATG® Fresenius Associated With Tacrolimus and Myfortic® in the Prevention of Treatment Failure, in Sensitized Renal Transplant[NCT02377193]Phase 460 participants (Actual)Interventional2013-09-30Completed
A Single-arm, Single-center Trial of Prophylactic Tocilizumab for Acute GVHD Prevention After Haploidentical HSCT.[NCT04688021]Phase 246 participants (Anticipated)Interventional2020-12-03Recruiting
Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis[NCT02339441]320 participants (Actual)Observational2010-06-30Completed
Allogeneic Hematopoietic Stem Cell Transplantation as Initial Salvage Therapy for Patients With Primary Induction Failure Acute Myeloid Leukemia Refractory to High-Dose Cytarabine-Based Induction Chemotherapy[NCT02441803]Phase 211 participants (Actual)Interventional2015-09-14Active, not recruiting
Prospective Study of Combined ATG Regimen for Prophylaxis of aGVHD in Matched Sibling Donor PBSCT[NCT02677181]Phase 4100 participants (Actual)Interventional2016-01-31Completed
A Prospective Randomized, Controlled Trial of Mycophenolate Mofetil Plus Steroid in the Treatment Of Patients With Progressive Idiopathic Membranous Nephropathy[NCT03170323]Phase 4128 participants (Anticipated)Interventional2018-07-01Recruiting
The Efficacy and Safety of Corticosteroid Combining Noncorticosteroid Systemic Immunomodulatory Therapy in VKH[NCT05120687]200 participants (Anticipated)Observational2021-10-15Recruiting
A Phase I/II Study Evaluating Escalating Doses of 211At-Labeled Anti-CD45 MAb BC8-B10 (211At-BC8-B10) Followed by Related Haplo-Identical Allogeneic Hematopoietic Cell Transplantation for High-Risk Acute Leukemia or Myelodysplastic Syndrome (MDS)[NCT03670966]Phase 1/Phase 230 participants (Anticipated)Interventional2019-07-10Recruiting
A Study Evaluating Escalating Doses of 211^At-Labeled Anti-CD45 MAb BC8-B10 (211^At-BC8-B10) Followed by Allogeneic Hematopoietic Cell Transplantation for High-Risk Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), or Myelodysplastic Syndr[NCT03128034]Phase 1/Phase 275 participants (Anticipated)Interventional2017-10-24Suspended(stopped due to Administrative - FDA Comments)
T Cell-Reduced Unrelated Donor Allogeneic Peripheral Blood Stem Cell Transplantation With Pentostatin and Low-Dose Total Body Irradiation[NCT00816413]Phase 1/Phase 20 participants (Actual)Interventional2008-09-30Withdrawn(stopped due to Screenings yielded inadequate eligible subjects to enroll.)
Effectiveness of Mycophenolate Mofetil Combined With Tacrolimus for Steroid Tapering in Systemic Lupus Erythematosus: A Prospective, Random Control, Open-label, Single Center Clinical Trial[NCT05916781]Phase 4220 participants (Anticipated)Interventional2023-07-01Recruiting
Assessment of Gastrointestinal Tolerability and Efficacy of Enteric-coated Mycophenolate Sodium (Myfortic®) in Heart Transplant Recipients[NCT00574197]Phase 411 participants (Actual)Interventional2006-06-30Completed
Immune Monitoring and Calcineurin Inhibitor (CNI) Withdrawal in Low Risk Recipients of Kidney Transplantation[NCT01517984]Phase 252 participants (Actual)Interventional2010-11-30Terminated(stopped due to Absence of equipoise on the basis of predetermined stopping rules.)
Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid.[NCT02993133]Phase 353 participants (Actual)Interventional2016-12-31Completed
Total Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation[NCT00352976]Phase 2/Phase 383 participants (Actual)Interventional2006-05-18Completed
Mycophenolate Mofetil Versus Cyclosporin A in the Treatment of Primary Biliary Cholangitis-autoimmune Hepatitis Overlap Syndrome Duo to Nonresponse to Standard Therapy[NCT04376528]Phase 489 participants (Anticipated)Interventional2021-06-16Recruiting
Using mTOR Inhibitors in the Prevention of BK Nephropathy[NCT01649609]40 participants (Actual)Interventional2012-03-31Completed
Busulfan and Cyclophosphamide Followed By Allogeneic Hematopoietic Cell Transplantation In Patients With Hematological Malignancies[NCT01685411]5 participants (Actual)Interventional2013-01-31Terminated
ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN PATIENTS WITH HIGH RISK HEMOGLOBINOPATHIES LIKE SICKLE CELL DISEASE AND β-THALESSEMIA-MAJOR USING REDUCED INTENSITY CONDITIONING REGIMEN[NCT02435901]Phase 1/Phase 229 participants (Actual)Interventional2008-12-31Completed
Topical 0.03% Tacrolimus Versus Systemic Mycophenolate Mofetil for Preventing Graft Rejection After Repeat Keratoplasty: One-year Results of a Randomized Clinical Trial[NCT04147390]Phase 2/Phase 358 participants (Anticipated)Interventional2019-11-30Recruiting
Pharmacokinetic Cross-over Study to Evaluate the Influence of Pantoprazole on MPA Bioavailability Administered as Mycophenolate Mofetil and Enteric Coated Mycophenolate Sodium in Maintenance Renal Transplant Patients[NCT01801280]Phase 420 participants (Actual)Interventional2012-01-31Completed
A Novel Immunosuppression Intervention for the Treatment of Amyotrophic Lateral Sclerosis (ALS)[NCT01884571]Phase 231 participants (Actual)Interventional2013-10-31Completed
A 12-Month, Open Label Study of Extended Release Tacrolimus (Envarsus XR®, LCPT) With Mycophenolate, Rabbit Antithymocyte Globulin (rATG) and Early Steroid Withdrawal in de Novo Kidney Transplant Recipients[NCT03828682]Phase 460 participants (Anticipated)Interventional2019-06-21Recruiting
A Pilot Study of Pharmacokinetics-based Mycophenolate Mofetil Dosing for Graft-Versus-Host-Disease Prophylaxis in Pediatric Blood and Marrow Transplantation[NCT01487577]Phase 219 participants (Actual)Interventional2010-06-30Completed
Allogeneic Hematopoietic Stem Cell Transplantation With Nonmyeloablative Conditioning for Patients With Chronic Lymphocytic Leukemia - A Multi-center Trial[NCT00060424]Phase 221 participants (Actual)Interventional2003-03-31Completed
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of BIIB023 in Subjects With Lupus Nephritis[NCT01499355]Phase 2276 participants (Actual)Interventional2012-07-31Terminated(stopped due to Results from pre-specified criteria did not demonstrate sufficient efficacy to warrant continuation of the study.)
Allogeneic Hematopoietic Cell Transplantation of Positively Selected CD34+ Cells and Defined Inoculum of T Cells From Related Haploidentical Donors for Older Patients With Indolent Hematologic Malignancies[NCT00185692]Phase 216 participants (Actual)Interventional2000-08-31Completed
Campath® [Alemtuzumab] Dose Escalation, Low-Dose TBI and Fludarabine Followed by HLA Class I Mismatched Donor Stem Cell Transplantation for Patients With Hematologic Malignancies - A Multi-Center Trial[NCT00040846]Phase 260 participants (Actual)Interventional2001-11-30Completed
Nonmyeloablative Hematopoietic Stem Cell Transplantation for Patients With High-Risk Hematologic Malignancies Using Related, HLA-Haploidentical Donors: A Phase II Trial of Combined Immunosuppression Before and After Transplantation[NCT00049504]Phase 253 participants (Actual)Interventional2002-01-31Completed
Proteomics Combined With Metabolomics Studies on the Efficacy of Telitacicept in Chinese Patients of Systemic Lupus Erythematosus[NCT05666336]Phase 430 participants (Anticipated)Interventional2022-12-31Recruiting
A Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Pediatric and Adolescent-Young Adults Patients With High Risk Solid Tumors[NCT04530487]Phase 240 participants (Anticipated)Interventional2020-08-19Recruiting
Efficacy & Safety of Pharmacokinetically-Driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis- A Double-Blind Placebo Controlled Clinical Trial[NCT05538208]Phase 2105 participants (Anticipated)Interventional2023-08-31Not yet recruiting
A Phase II Trial Evaluating the Safety and Efficacy of Non-myeloablative 90Y-Ibritumomab Tiuxetan (Anti-CD20) Antibody With Fludarabine, Low-Dose Total Body Irradiation (TBI) and HLA Matched Allogeneic Transplantation for Relapsed B-cell Lymphoma[NCT00119392]Phase 242 participants (Actual)Interventional2004-06-30Completed
An Open Label, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Early Calcineurin Inhibitor Withdrawal in Recipients of Primary Renal Allografts Maintained Long-term on Mycophenolate Mofetil (MMF) (CellCept®) and Sirolimus (Rapamune®)[NCT00121810]Phase 4305 participants (Actual)Interventional2003-08-31Completed
S0125, A Phase II Study Of Chimerism-Mediated Immunotherapy (CMI) Using Nonmyeloablative Allogeneic Peripheral Blood Stem Cell Transplantation In Older Patients With Acute Myeloid Leukemia (AML) In First Complete Remission (A BMT Study)[NCT00053014]Phase 25 participants (Actual)Interventional2003-04-30Terminated(stopped due to Poor accrual)
Optimization of NULOJIX® (Belatacept) Usage as a Means of Minimizing CNI Exposure in Simultaneous Pancreas and Kidney Transplantation (CTOT-15)[NCT01790594]Phase 246 participants (Actual)Interventional2013-02-28Terminated(stopped due to Slow accrual within enrollment time period: projected accrual goal not achieved.)
Nonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding Study[NCT00453388]Phase 26 participants (Actual)Interventional2007-02-28Completed
Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis[NCT01399047]Phase 219 participants (Actual)Interventional2011-07-31Completed
Allogeneic Hematopoietic Cell Transplantation for Patients With Treatment-Refractory Crohn's Disease: A Phase 2 Study[NCT01570348]Phase 22 participants (Actual)Interventional2012-07-17Terminated(stopped due to Annual accrual goal not met)
Steroid and Tacrolimus Avoidance Using NULOJIX® (Belatacept) in Renal Transplantation (CTOT-16)[NCT01856257]Phase 271 participants (Actual)Interventional2013-07-31Terminated(stopped due to Safety: Stopping rule not met.)
Advancing Transplantation Outcomes in Children (CTOT-41)[NCT06055608]Phase 2200 participants (Anticipated)Interventional2023-11-01Not yet recruiting
Effect of Azole/Echinocandin Use on Tacrolimus Pharmacokinetics in Kidney Transplant Recipients[NCT06044558]507 participants (Actual)Observational2022-09-01Completed
A Multi-centre Phase II Trial of GVHD Prophylaxis Following Unrelated Donor Stem Cell Transplantation Comparing Thymoglobulin vs. Calcineurin Inhibitor or Sirolimus-based Post-transplant Cyclophosphamide[NCT04888741]Phase 2400 participants (Anticipated)Interventional2021-02-22Recruiting
An Intention-to-Treat Study of Salvage Chemotherapy Followed by Allogeneic Hematopoietic Stem Cell Transplant for the Treatment of High-Risk or Relapsed Hodgkin Lymphoma[NCT00574496]Phase 225 participants (Actual)Interventional2007-11-13Completed
Head to Head Comparison of Myfortic vs. Cellcept in the Treatment of Kidney Transplant Recipients Using Our Current Center Standardized Concomitant Immunosuppressive Protocol[NCT00533624]Phase 2/Phase 3150 participants (Actual)Interventional2004-12-31Completed
"MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem Cells"[NCT01033552]Phase 1/Phase 232 participants (Actual)Interventional2010-01-31Completed
Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis: A Phase 2a, Single-centered, Randomized, Placebo-controlled, Double-blind, Proof-of-concept Pilot Study.[NCT01670565]Phase 220 participants (Actual)Interventional2012-08-31Completed
A Pilot Study Comparing Two Different Sirolimus-based Transition Regimens in African-American Renal Transplant Recipients[NCT01005706]40 participants (Actual)Interventional2009-08-31Completed
Comparison of Pharmocokinetics of Mycophenolate Mofetil and Enteric Coated Mycophenolate Sodium in Calcineurininhibitor-free Treated Patients After Renal Transplantation[NCT01033864]Phase 423 participants (Actual)Interventional2009-11-30Completed
A Phase 1/2 Multi-Center Trial of Vorinostat for Graft vs Host Disease Prevention in Children, Adolescents and Young Adults Undergoing Allogeneic Blood and Marrow Transplantation[NCT03842696]Phase 1/Phase 249 participants (Anticipated)Interventional2020-02-04Recruiting
Pharmacokinetically-driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis[NCT05101447]Phase 20 participants (Actual)Interventional2023-07-31Withdrawn(stopped due to funding not secured)
Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies[NCT04776850]Early Phase 10 participants (Actual)Interventional2020-12-29Withdrawn(stopped due to Competing protocol opened in Adult SCT that will include pediatric patients and is now multi-center. No patients enrolled on study.)
Evaluation of the Effectiveness and Safety of Immunosuppressive and Biological Therapy of Atopic Dermatitis in Childhood[NCT04895423]Phase 4160 participants (Anticipated)Interventional2021-11-25Not yet recruiting
Non-interventional Clinical Study With Target of Kidney Function Follow-up in Routine Clinical Practice on De Novo Kidney Transplant Recipients Who Are on CellCept Immunosuppressive Combination Therapy in Routine Clinical Practice[NCT01672957]128 participants (Actual)Observational2011-09-30Completed
Pilot Study of JAK Inhibitor Therapy Followed by Reduced Intensity Haploidentical Transplantation for Patients With Myelofibrosis[NCT04370301]Phase 210 participants (Anticipated)Interventional2021-02-09Recruiting
An Investigator-Initiated, Phase II, Randomized, Withdrawal Study of Mycophenolate Mofetil (MMF) in Patients With Stable, Quiescent Systemic Lupus Erythematosus (SLE)[NCT01946880]Phase 2102 participants (Actual)Interventional2013-11-20Terminated(stopped due to Slow enrollment.)
Mechanisms and Treatment of Chronic Allograft Injury (CAI) Due to Calcineurin Inhibitor (CNI) Toxicity[NCT01473732]2 participants (Actual)Interventional2012-03-31Terminated(stopped due to terminated after 2 patients due to difficulty in enrollment)
A Multi-center Phase II Trial Randomizing Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN #1203; Progress I)[NCT02208037]Phase 2279 participants (Actual)Interventional2014-08-31Completed
A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation[NCT01529827]Phase 294 participants (Actual)Interventional2012-02-28Completed
In-Vivo Activated T-Cell Depletion to Prevent GVHD[NCT00594308]10 participants (Actual)Interventional2007-10-31Terminated(stopped due to Treatment ineffective)
A Pilot Study of an Immunosuppressive and Myeloablative Preparative Regimen for Allogeneic Unrelated Donor Hematopoietic Stem Cell Transplantation (HSCT) for Severe Sickle Cell Disease[NCT01279616]Phase 28 participants (Actual)Interventional2010-09-30Terminated(stopped due to PI moving to a different institution.)
Trial Of Double Umbilical Cord Blood Transplantation[NCT00763490]Phase 220 participants (Actual)Interventional2008-12-31Completed
A Phase II Study to Assess Immunosuppression With Sirolimus Combined With Cyclosporine (CSP) and Mycophenolate Mofetil (MMF) for Prevention of Acute GVHD After Non-Myeloablative HLA Class I or II Mismatched Donor Hematopoietic Cell Transplantation- A Mult[NCT01251575]Phase 277 participants (Actual)Interventional2010-12-01Completed
Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen[NCT02199041]Phase 224 participants (Actual)Interventional2014-07-11Terminated(stopped due to The study was halted early due to slow accrual.)
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies in Remission From HLA Partially-Matched Related Donors[NCT01350245]Phase 228 participants (Actual)Interventional2010-07-31Completed
Dose-Intense Yttrium-90 Ibritumomab Tiuxetan (Zevalin)-Containing Non-Myeloablative Conditioning for Allogeneic Stem Cell Transplantation in B-cell Malignancies[NCT01490723]Phase 220 participants (Actual)Interventional2013-01-31Completed
A 3-month, Multicenter, Randomized, Open Label Study to Evaluate the Impact of Early Versus Delayed Introduction of Everolimus on Wound Healing in de Novo Kidney Transplant Recipients With a Follow-up Evaluation at 12 Month After Transplant (NEVERWOUND St[NCT01410448]Phase 3383 participants (Actual)Interventional2011-11-30Completed
Safety and Efficacy of Mycophenolic Acid Withdrawal With Conversion to Zortress (Everolimus) in Renal Transplant Recipients With BK Virus Infection[NCT01624948]Phase 440 participants (Actual)Interventional2012-09-30Completed
Remission Induction Therapy for Refractory Systemic Lupus Erythematosus With Autologous Hematopoietic Stem Cell Transplantation (AHSCT) Versus Rituximab (antiCD20) Followed by Maintenance Therapy With Mycophenolate Mofetil (MMF)[NCT05063513]Phase 2/Phase 30 participants (Actual)Interventional2009-07-31Withdrawn
Clofarabine Pre-conditioning Followed by Hematopoietic Stem Cell Transplant With Post-Transplant Cyclophosphamide for Non-remission Acute Myeloid Leukemia[NCT04002115]Phase 22 participants (Actual)Interventional2020-06-03Terminated(stopped due to terminated due to low accrual)
A Randomized, Double-Blind, Placebo-Controlled Study of Belimumab and Rituximab Combination Therapy for the Treatment of Diffuse Cutaneous Systemic Sclerosis[NCT03844061]Phase 230 participants (Anticipated)Interventional2019-07-29Recruiting
A Phase II, Multicenter, Randomized, Open-label Study to Investigate the Safety and Efficacy of Mycophenolate Mofetil and Rilonacept (Anti-interleukin-1) in Patients With Alcoholic Hepatitis[NCT01903798]Phase 24 participants (Actual)Interventional2014-12-31Completed
Comparison of the Efficacy and Safety of Sirolimus, Everolimus or Mycophenolate in Renal Transplant Recipients Receiving Induction With Anti-thymocyte Globulin, Tacrolimus and Prednisone[NCT03468478]Phase 41,209 participants (Actual)Interventional2017-06-18Completed
A Phase 2, Double-Blind, Placebo-Controlled Trial of Mycophenolate Mofetil Alone or With Voclosporin for Systemic Lupus: Examining Distinct Immunophenotypes to Validate and Enhance Rational Treatment (DIVERT) (ALE10)[NCT05306873]Phase 2120 participants (Anticipated)Interventional2022-10-28Recruiting
PRESERVE: A Multi-Center Trial Using Banked, Cryopreserved HLA-Mismatched Unrelated Donor Bone Marrow and Post-Transplantation Cyclophosphamide in Allogeneic Transplantation for Patients With Hematologic Malignancies[NCT05170828]Phase 10 participants (Actual)Interventional2022-09-30Withdrawn(stopped due to Change in Study Design)
Immunosuppressive Drugs and Gut Microbiome: Pharmacokinetic- and Microbiome Diversity Effects[NCT04207177]Phase 4100 participants (Anticipated)Interventional2019-10-30Active, not recruiting
A Randomized, Double-Blind, Parallel-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Human Umbilical Cord Mesenchymal Stem Cells in Patients With Lupus Nephritis[NCT03580291]Phase 2230 participants (Anticipated)Interventional2018-08-01Not yet recruiting
A Randomized Open-label Study to Evaluate the Efficacy and Safety of Tacrolimus and Corticosteroids in Comparison With Mycophenolate Mofetil and Corticosteroids in Subjects With Class III/IV±V Lupus Nephritis[NCT02630628]Phase 4130 participants (Actual)Interventional2015-12-05Active, not recruiting
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major Treatment: a Multi-center, Open, Randomized, Controlled Clinical Study[NCT02342145]Phase 440 participants (Anticipated)Interventional2014-06-30Recruiting
A Multicenter, Two Arms, Randomized, Open Label Clinical Phase IV Study Investigating the Proportion of CMV Seropositive Kidney Transplant Recipients Who Will Develop a CMV Infection Within the First 6 Months Post-transplantation When Treated With an Immu[NCT02328963]Phase 4186 participants (Actual)Interventional2014-05-02Completed
Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)[NCT05736419]Phase 224 participants (Anticipated)Interventional2023-02-09Recruiting
A National Prospective Study of Patients With Hepatitis Induced by Immune Checkpoint Inhibitors; Characterization of Liver Injury, Outcome of Therapy and Randomization to Either Prednisolone or Mycophenolate Mofetil Treatment in Case of Relapse[NCT04810156]Phase 260 participants (Anticipated)Interventional2021-04-07Recruiting
Randomized, Open-Label Trial of Tacrolimus/Everolimus vs. Tacrolimus/Enteric-Coated Mycophenolate Sodium to Prevent Biopsy-Proven Acute Rejection and Chronic Allograft Injury in Adult, Primary Kidney Transplantation[NCT01680861]Phase 332 participants (Actual)Interventional2012-11-30Completed
Reappraisal of Second-line Therapies of Refractory Autoimmune Hemolytic Anemia in Systemic Lupus Erythematosus[NCT05057481]Phase 330 participants (Anticipated)Interventional2021-09-15Active, not recruiting
A Randomised, Open-label Clinical Trial Assessing the Efficacy and Safety of Mycophenolate Mofetil Versus Azathioprine for Induction of Remission in Treatment Naive Autoimmune Hepatitis[NCT02900443]Phase 470 participants (Anticipated)Interventional2017-01-31Active, not recruiting
A Pilot Study of Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease[NCT02678143]Phase 11 participants (Actual)Interventional2016-04-26Terminated(stopped due to Closed early due to competing studies)
The Efficacy and Safety of Mycophenolate Mofetil in the Treatment of Lymphocytic Myocarditis in Comparison With Azathioprine[NCT05237323]Phase 350 participants (Anticipated)Interventional2020-10-01Recruiting
"A Phase I/II Clinical Trial of Off-the-shelf NK Cell Administration in Combination With Allogeneic SCT to Decrease Disease Relapse in Patients With High-risk Myeloid Malignancies Undergoing Matched Related, Matched Unrelated, One Antigen Mismatched Unrel[NCT05115630]Phase 1/Phase 224 participants (Anticipated)Interventional2022-04-08Recruiting
A Prospective, Open-label, Multicenter, Randomized Study to Evaluate the Benefits and Risks of Conversion of Existing Adolescent Renal Allograft Recipients Aged 12 to Less Than 18 Years of Age to a Belatacept-based Immunosuppressive Regimen as Compared to[NCT04877288]Phase 3102 participants (Anticipated)Interventional2021-07-21Recruiting
Phase I/II Study of Sorafenib Added to Busulfan and Fludarabine Conditioning Regimen in Patients With Relapsed/Refractory AML Undergoing Stem Cell Transplantation[NCT03247088]Phase 1/Phase 274 participants (Anticipated)Interventional2017-07-30Recruiting
A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease[NCT03121001]Phase 250 participants (Anticipated)Interventional2017-03-20Recruiting
A Phase I/II Study of Nonmyeloablative Conditioning and Transplantation of Human Leukocyte Antigen (HLA)-Matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated Bone Marrow for Patients With Refractory SLE[NCT02080195]Phase 1/Phase 21 participants (Actual)Interventional2016-09-13Terminated(stopped due to Study was unable to accrue subjects)
Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Reduced-Intensity Preparative Regimen (A Multi-Center Trial Coordinated by the FHCRC)[NCT00723099]Phase 273 participants (Actual)Interventional2008-06-25Completed
A Phase I/II Study of Autologous Stem Cell Transplantation Followed by Nonmyeloablative Allogeneic Stem Cell Transplantation for Patients With Relapsed or Refractory Lymphoma - A Multi-center Trial[NCT00005803]Phase 1/Phase 276 participants (Actual)Interventional1999-09-30Completed
A Phase I/II Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplant for the Treatment of Patients With Hematologic Malignancies Using Busulfan, Fludarabine and Total Body Irradiation[NCT00245037]Phase 1/Phase 2147 participants (Actual)Interventional2005-06-30Completed
Safety and Efficacy of Basiliximab, Delayed Dose Tacrolimus Plus ECMPA, Versus Standard Dose Tacrolimus, ECMPA Plus Corticosteroids in Patients Undergoing Liver Transplant[NCT02123108]Phase 459 participants (Actual)Interventional2011-01-31Completed
A National, Multi-center, Randomized, Open Label Study to Evaluate the Efficacy and Safety of Everolimus Combined With Enteric-coated Mycophenolate Sodium Compared to the Standard Treatment Combining Tacrolimus and Enteric-coated Mycophenolate Sodium in d[NCT01625377]Phase 3188 participants (Actual)Interventional2012-12-31Completed
First-line Antimetabolites as Steroid-sparing Treatment (FAST) Uveitis Trial[NCT01829295]Phase 3216 participants (Actual)Interventional2013-08-31Completed
A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) for Children With Juvenile Myelomonocytic Leukemia (JMML)[NCT01824693]Phase 230 participants (Actual)Interventional2013-06-24Completed
A Randomized Study Comparing the Incidence of Acute Clinical or Subclinical Rejection With Two Dosing Regimens of CellCept in de Novo Renal Transplant Recipients Receiving Induction, Cyclosporine and Brief Steroid Therapy[NCT02005562]Phase 3252 participants (Actual)Interventional2006-05-31Completed
Prospective Randomised Marker-based Trial to Assess the Clinical Utility and Safety of Biomarker-guided Immunosuppression Withdrawal in Liver Transplantation[NCT02498977]Phase 4116 participants (Actual)Interventional2015-10-31Active, not recruiting
Does the Mycophenolate Improve the Ability of Weaning Patients Off the Treatment in Chronic Inflammatory Demyelinating Polyradiculopathy (CIDP)[NCT02494505]Phase 340 participants (Actual)Interventional2013-11-18Completed
A Randomized Study to Evaluate Antibody Response to an Additional Dose of SARS-CoV-2 Vaccination With and Without Immunosuppression Reduction in Kidney and Liver Transplant Recipients[NCT05077254]Phase 2400 participants (Anticipated)Interventional2021-12-06Recruiting
A Pilot Study of Allogeneic Hematopoietic Cell Transplantation for Patients With High Grade Central Nervous System Malignancies[NCT04521946]Phase 10 participants (Actual)Interventional2021-01-14Withdrawn(stopped due to No participants enrolled.)
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism[NCT00176865]Phase 219 participants (Actual)Interventional2002-08-31Completed
Allogeneic Hematopoietic Stem Cell Transplantation for Induction of Mixed Hematopoietic Chimerism in Patients Infected With Human Immunodeficiency Virus-1 Using a Non-Marrow Ablative Conditioning Regimen Containing Total Body Irradiation in Combination Wi[NCT00112593]5 participants (Actual)Interventional1999-11-30Completed
Randomized Conversion Of Epstein-Barr Virus (EBV)+ Kidney Transplant Recipients Of Living Or Standard Criteria Donors At Three Months Post Transplantation To Belatacept With MPA Or Belatacept With Low-Dose Tacrolimus (50% Of Dose) Compared To Patients Rem[NCT02213068]Phase 428 participants (Actual)Interventional2014-07-31Completed
Pediatric Blood & Marrow Transplant Consortium (PBMTC) Phase II Myeloablative Haploidentical BMT With Post-transplantation Cyclophosphamide for Pediatric Patients With Hematologic Malignancies[NCT02120157]Phase 235 participants (Actual)Interventional2015-07-02Completed
12 Month, Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating a Standard Regimen in de Novo Kidney Transplant Patients Versus a Certican® Based Regimen Either in Combination With Cyclosporin A or Tacrolimus[NCT01843348]Phase 3612 participants (Actual)Interventional2012-12-27Completed
Decitabine Followed by Bone Marrow Transplant and High-Dose Cyclophosphamide for the Treatment of Relapsed and Refractory Acute Myeloid Neoplasms[NCT01707004]Phase 220 participants (Actual)Interventional2013-05-16Completed
A Phase II Trial Combining Radiolabeled BC8 (Anti-CD45) Antibody With Fludarabine and Low Dose TBI Followed by Related or Unrelated PBSC Infusion and Post-Transplant Immunosuppression for Patients With Advanced AML or High Risk Myelodysplastic Syndrome[NCT00119366]Phase 218 participants (Actual)Interventional2003-05-31Terminated(stopped due to Funding ended before target accrual was reached; participants are no longer being examined or receiving intervention.)
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)
Changes in GI-related Quality of Life Index (GIQLI), After Patients Are Converted From MMF to Enteric-coated Mycophenolate Sodium
Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Overall Treatment Effects for for Health-related Quality of Life Assessed by the Patient
Overall Treatment Effects for GI Symptoms Assessed by the Patient
Overall Treatment Effects for GI Symptoms Assessed by the Physician
Acute Graft-Versus-Host Disease
Neutrophil and Donor Cell Engraftment
Overall Survival
Serious Adverse Events
Transplant Related Mortality
Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0
British Isles Lupus Assessment Group (BILAG) Index Score Over 52 Weeks
Change From Baseline in Anti-double-stranded DNA at Week 52
Change From Baseline in the Systemic Lupus Erythematosus Expanded Health Survey Physical Function Score at Week 52
Percentage of Participants Who Achieved a Complete Renal Response at Week 24 and Maintained it to Week 52
Percentage of Participants Who Achieved a Complete Renal Response at Week 52
Percentage of Participants With a Baseline Urine Protein to Creatinine Ratio of > 3.0 Who Achieved a Urine Protein to Creatinine Ratio of < 1.0 at Week 52
Time to Achieve a Complete Renal Response
Change From Baseline in C3 and C4 Complement Levels at Week 52
Percentage of Participants Who Achieved a Complete Renal Response (CRR), a Partial Renal Response (PRR), or no Renal Response (NRR) at Week 52
Percentage of Participants With Acute Graft-vs-host-disease (GVHD)
Percentage of Participants With Chronic GVHD
Percentage of Participants With Graft-Failure-Free Survival
Percentage of Participants With Overall Survival (OS)
Percentage of Participants With Platelet Recovery
Percentage of Participants With Primary Graft Failure
Percentage of Participants With Secondary Graft Failure
Frequencies of Infections Categorized by Infection Type
Health Related Quality of Life (HR-QoL) - Medical Outcomes Study Short Form (MOS SF-36)
Health Related Quality of Life (HR-QoL) - PedsQL Stem Cell Transplant Module
Immune Reconstitution of Flow Cytometry
Immune Reconstitution of Quantitative Immunoglobulins
Participants With Grade 3-5 Toxicities by SOC
Percentage of Participants With Cytomegalovirus (CMV), Epstein Barr Virus (EBV) or Post-Transplant Lymphoproliferative Disease (PTLD)
Percentage of Participants With Neutrophil Recovery
Graft Survival at One Year
Kaplan-Meier Estimate of Graft Survival at the End of the Study
Kaplan-Meier Estimate of Patient Survival at the End of the Study
Number of Participants Experiencing Multiple Rejection Episodes
Number of Participants Requiring Anti-lymphocyte Antibody Therapy for Treatment of Rejection
Number of Participants Who Crossed Over Due to Treatment Failure
Number of Participants With Clinically Treated Acute Rejection Episodes
Number of Participants With Treatment Failure
Patient Survival at One Year
Percentage of Participants With Efficacy Failure
Time to First Biopsy-confirmed Acute Rejection Episode
Change From Month 1 in Creatinine Clearance at Month 6 and Month 12
Change From Month 1 in Serum Creatinine at Month 6 and Month 12
Percentage of Participants With Biopsy Confirmed Acute Rejection at 6 and 12 Months
Severity of Acute Rejection
Number of Participants Who Developed Acute Rejection Episode Within 6 Months Post-Transplant
Number of Participants Who Developed Acute Rejection Episode Within the 12 Months PT
Mean Maintenance Doses of Mycophenolate Mofetil, Cyclosporine, and Cumulative Dose of Corticosteroids at 6 and 12 Months PT
Median Change From Baseline for LDL/HDL Ratio
Median Change From Baseline for Lipid Profile (Total Cholesterol, Low Density Lipoproteins, High Density Lipoproteins, and Triglycerides)
Median Time to First Acute Rejection Episode Within the First 6 Months and 12 Months PT
Number of Acute Rejection Episodes Per Participant Within the First 6 Months and 12 Months PT
Number of Participant Who Died Within 6 Months 12 Months and 3 Years PT
Number of Participants Using Monomurab Cluster of Differentiation 3, Orthoclone Polyclonal Antithymocyte Globulin or Antilymphocyte Globulin in the First 6 Months and 12 Months PT
Number of Participants With Any Adverse Events and Any Serious Adverse Event, and Adverse Events Leading to Premature Withdrawal
Number of Participants With Malignancies and Opportunistic Infections
Number of Participants With Marked Laboratory Abnormalities: Biochemistry Parameters
Number of Participants With Marked Laboratory Abnormalities: Hematology Parameters
Number of Participants With Worst ISHLT Biopsy Grade Within First 6 Months and 12 Months PT
Cumulative Incidence of a Severe/Life-threatening/Fatal Infections
Disease-Free Survival (DFS) Post-Randomization
Incidence of Chronic GVHD
Incidence of Cytomegalovirus (CMV) Reactivation
Incidence of Epstein-Barr Virus (EBV)-Associated Lymphoma
Incidence of GVHD Flares Requiring Increased Therapy
Incidence of Systemic Infections
Incidence of Topical/Non-absorbable Therapy
Treatment Related Mortality (TRM)
Cumulative Steroid Dose
GVHD-free Survival
Overall GVHD-free Survival Post-randomization
Percentage of Surviving Participants With Complete Response (CR)
Blood CD4+ T Cells Per mm^3 Blood
Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 Effector Memory CD4+ T Cells Over 12 Months
Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 T Cells Over 12 Months
Change in Cell-associated Intact HIV DNA (Ca-iDNA) Levels Per 10^6 T Cells Over 12 Months
Incidence of Opportunistic Infection
Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe?
Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant
Number of Participants With Grade II-IV or Grade III-IV Acute GVHD
Number of Participants With Major Toxicities Related to Transplant
Number of Patients That Expired Due to Non-relapsed-related Mortality Following Transplant
Number of Patients That Expired Due to Transplant Related Mortality
Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant
Number of Patients That Have Survived at One Year
Number of Patients With Primary or Secondary Graft Failure Following Transplant
Participants That Were GVHD Free, Relapse Free Survival (GRFS)
Participants With Chronic GVHD at One Year
Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB)
Survival Rate at Day 180 After Allogeneic Transplant From Umbilical Cord Blood (UCB)
Percentage of Participants With Efficacy Failure Through 12 Months Post Transplant
Percentage of Participants With Recurrence of Focal Segmental Glomerulosclerosis (rFSGS) or Death or Graft Loss or Lost to Follow-up Through 3 Months Post Transplant
Percentage of Participants With Biopsy Proven rFSGS Through 3, 6 and 12 Months Post-Transplant
Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) Through 3, 6, and 12 Months Post Transplant
Percentage of Participants With rFSGS or Death or Graft Loss or Lost to Follow-up Through 6 and 12 Months Post Transplant
Cumulative Incidence of Systemic Infections
Number of Complete Response (CR) at Day 28 of Therapy
Number of Patients With Acute Graft-versus-host Disease (GVHD) Flares at Day 90
Number of Patients With Chronic Graft-versus-host Disease (GVHD)
Proportion of Treatment Failure
Number of Partial Response (PR), Mixed Response (MR), and Progression
Number of Patients Discontinuing Immune Suppression Without Flare
Number of Patients Surviving at 6 and 9 Months Post Randomization
Acute Graft vs Host Disease (GvHD)
Acute Graft vs Host Disease (GvHD), All Evaluable
Incidence of Relapse
Transplant-related Mortality
Event-free Survival (EFS)
Overall Survival (OS)
Disease-free Survival
Incidence of Grades III/IV Acute Graft Versus Host Disease
Incidence of Primary Graft Failure
Non-relapse Mortality at 1 Year
Number of Platelet Transfusions
Number of Red Blood Cell Transfusions
Percentage of Participants With Chronic Graft Versus Host Disease
Point Estimate of Overall Survival at 3 Years
Relapse of Malignancy After Transplantation
Time to Neutrophil Recovery
Time to Platelet Recovery
Toxicity of Treatment Regimen Determined by Number of Adverse Events Per Organ System
Mean Stimulated C-peptide Area Under the Curve
Acute Graft-Versus-Host Disease (GVHD)
Chronic Graft-Versus-Host Disease (GVHD)
Donor Lymphocyte Infusion
Engraftment of Donor Hematopoietic Stem Cells, as Measured by Time in Days to Neutrophil and Platelet Count Recovery Following Allogeneic PBSCT.
Event-free Survival
Relapse
Survival
Treatment Related Mortality
The Comparison of Functional Immune Reconstitution at 6-9 Months Following Transplant as Measured by Antibody Response to Vaccination With Inactivated Hepatitis A or B Vaccine.
Maximum Plasma Concentration (Cmax) of GSK1070806
Number of Participants Having Infections
Number of Participants Requiring Dialysis During the First 7 Days Post Transplant
Number of Participants Who Are Dialysis Independent at Visits up to 12 Months Post-transplant
Number of Participants With Dialysis Events in the First 30 Days Post-transplant
Number of Participants With Episodes of Biopsy-proven Acute Rejection
Area Under the Plasma Concentration Time Curve (AUC) From Time 0 to the Last Measurable Concentration (AUC[0-t]) and AUC From Time 0 to Infinite Time (AUC[0-inf]) of GSK1070806
Baseline and Change From Baseline in Serum Levels of Free, Total, and GSK1070806 Bound Interleukin 18 (IL-18) Over Time Post-transplant
Number of Participants Having Any Abnormal Clinical Chemistry Results of Potential Clinical Importance
Number of Participants Having Any Abnormality in Hematology Results of Potential Clinical Importance
Number of Participants Having Any Abnormality of Potential Clinical Importance of Vital Signs Results
Number of Participants in the First 7 Days With: Primary Non Function, Functional DGF, Intermediate Graft Function, Immediate Graft Function
Number of Participants With Adverse Event (AE) and Serious Adverse Event (SAE)
Serum Concentrations of GSK1070806
Serum Creatinine at Baseline and Change From Baseline Over Time Post Transplant
Serum Interferon Gamma-induced Protein 10 (IP-10) and Serum Monokine Induced Gamma Interferon (Mig) Levels at Baseline and Change From Baseline Over Time Post Transplant
Urine Volume at Baseline and Change From Baseline Over Time Post Transplant
Acute Rejection Per Kidney Biopsy (Banff Grading Criteria)
Acute Tubular Necrosis (ATN) Rate, Defined as the Requirement for Dialysis Within 7 Days Post-transplantation.
Graft Survival
Lymphoid Cell Sub-type CD3 Absolute Numbers
New-onset Diabetes and Hyperglycemia After Transplantation (NODAT)
New-onset Polyomavirus (BK Virus) Disease Per Kidney Biopsy
Patient Survival
Ratio of CD4/CD8 Lymphoid Cells
Requirement for Additional Immunosuppression (Such as Corticosteroids, Antimetabolites or Other Immunosuppressive Agents)
Safety Profile
Average of Renal Function
Chronic Allograft Nephropathy (Cumulative Calcineurin-inhibitor Nephrotoxicity/Transplant Nephropathy) Per Protocol Surveillance Kidney Biopsies (Banff Grading Criteria).
Immunosuppression-free Duration in Days, Defined as Time From Completion of Immunosuppression Withdrawal to End of Trial Participation or to Time of Restarting Immunosuppression
Percent of Participants Requiring Anti-lymphocyte Therapy (OKT3, ATG) for an Acute Rejection Event
Percent of Participants Successfully Withdrawn From Immunosuppression and Remained Off Immunosuppression for at Least 52 Weeks
Percent of Participants With Chronic T Cell-mediated or Antibody-mediated Rejection
Percent of Transplant Participants Who Died
Percent of Transplanted Participants Who Achieve Either Sirolimus Monotherapy or Monotherapy on a Mycophenolic Compound Within 52 Weeks Post-transplant
Percent of Transplanted Participants Who Achieve MMF or Mycophenolic Acid Monotherapy Within 52 Weeks Post-transplant in Those Participants Intolerant of Sirolimus
Percent of Transplanted Participants Who Achieve Sirolimus Monotherapy Within 52 Weeks Post-transplant
Percent of Transplanted Participants Who Remain Off Immunosuppression for at Least 52 Weeks Including Those in Whom the 52 Week Biopsy Was Not Performed
Percent of Transplanted Participants Who Remain Off Immunosuppression for the Duration of the Study as Defined as Completion of All Schedules of Events/Followed Through August 25, 2017
Percent of Transplanted Participants With Acute Rejection or Presumed Acute Rejection
Percent of Transplanted Participants With Graft Loss
Time From Completion of Immunosuppression Withdrawal to First Episode of Acute Rejection or Presumed Acute Rejection
Time From Transplant to the First Episode of Acute Rejection Requiring Treatment
Histological Severity of Biopsies Demonstrating Acute Rejection as Measured by Banff 2007 Grade
Number of Adverse Events, Including Number of Post-transplant Infections, Wound Complications, Lymphocoele, Post-transplant Diabetes Mellitus, and Malignancies
Participant Diastolic Blood Pressure Over Time
Participant Glucose Level Over Time
Participant Renal Function as Measured by GFR Using CKD-EPI
Participant Systolic Blood Pressure Over Time
Participant Total Cholesterol Over Time
Phase I: Acute Rejection-Free Survival
Phase I: The Minimization of Negative Side Effects - Graft Survival
Phase I: The Minimization of Negative Side Effects - Patient Survival
Phase II: Acute Rejection-Free Survival
Phase II: The Minimization of Negative Side Effects - Graft Survival
Phase II: The Minimization of Negative Side Effects - Patient Survival
Percent Change From Baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR) at Week 24 in the Blinded Treatment Period (Part B)
The Number of Participants Experiencing Averse Events in the Blinded Treatment Period (Part B)
The Number of Participants With Abnormal Laboratory Parameters of Clinical Significance in the Blinded Treatment Period (Part B)
The Number of Participants With Clinically Significant ECG Abnormalities in the Blinded Treatment Period (Part B)
The Number of Participants With Complete Renal Response (CRR) at Week 24 in the Blinded Treatment Period (Part B)
The Number of Participants With Complete Renal Response (CRR) at Week 52 in the Blinded Treatment Period (Part B)
The Number of Participants With Complete Renal Response (CRR) Plus Successful Corticosteroid Taper to ≤ 7.5 mg/Day at Week 24 in the Blinded Treatment Period (Part B)
The Number of Participants With Complete Renal Response (CRR) Plus Successful Corticosteroid Taper to ≤ 7.5 mg/Day at Week 52 in the Blinded Treatment Period (Part B)
The Number of Participants With Partial Renal Response (PRR) at Week 24 in the Blinded Treatment Period (Part B)
The Number of Participants With Partial Renal Response (PRR) at Week 52 in the Blinded Treatment Period (Part B)
The Percent Change in Vital Sign Measurements in the Blinded Treatment Period (Part B)
Number of Patients With a Partial Response
Number of Patients With Grade II-IV Acute Graft-versus Host Disease (GvHD (aGVHD)
AUC0-inf - Area Under Concentration-time Curve From Time Zero to Infinity (Extrapolated)
AUC0-t - Area Under Concentration-time Curve From Time Zero to Time of Last Non-zero Concentration
Cmax - Maximum Observed Concentration
Donor-specific HLA Antibodies, Re-transplantation, or Death
Percentage of Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR) at 6 Months
Percentage of Participants With Adverse Events (AEs)
Percentage of Participants With Serious Adverse Events (SAEs)
Time to Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR).
Absolute Calculated Glomerular Filtration Rate (cGFR): Mean
Absolute Values of Blood Pressure: Mean
Absolute Values of Blood Pressure: Median
Absolute Values of Fasting Lipid Values: Mean
Absolute Values of Fasting Lipid Values: Median
Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR) at 6, 12 and 24 Months
Mean and Mean Change From Baseline in Blood Glucose
Mean and Mean Change From Baseline in Whole Blood HbA1c
Mean Change From Month 3 in cGFR
Mean Changes From Baseline Values for Blood Pressure
Mean Changes From Baseline Values of Lipid Values
Median Calculated Glomerular Filtration Rate (cGFR)
Number of Participants Deaths Post Transplant
Number of Participants Who Experience Graft Loss Post Transplant
Number of Participants Who Survive With a Functioning Graft
Percentage of Participants With BANFF Grade by Severity Grades. BANFF Type (Grade) for Acute/Active Rejection
Percentage of Participants With De Novo Donor Specific Anti-HLA Antibodies (DSA)
Percentage of Participants With Donor Specific Anti-HLA Antibodies (DSA)
Percentage of Participants With Events of Special Interest (ESIs)
Percentage of Participants With New Onset Diabetes After Transplant
Percentage of Particpants With Laboratory Test Abnormalities (LTAs)
Time to Event: Graft Loss and Death
Treatment Differences in Therapeutic Modalities
Urine Protein Creatinine Ratio (UPr/Cr)
Mean Cases of Acute Rejection (MCAR) Per Patient
Mean Cases of Acute Rejection (MCAR) Per Patient (Pediatric Population)
Number of Cardiac Rejection Episodes Requiring Treatment
Number of Cardiac Rejection Episodes Requiring Treatment (Pediatric Population)
Number of Patients Requiring Antilymphocyte Antibodies or Steroids for Treatment of Severe Acute Rejection
Number of Patients Requiring Antilymphocyte Antibodies or Steroids for Treatment of Severe Acute Rejection (Pediatric Population)
Time to First Acute Rejection Episode Following de Novo Cardiac Transplant
Time to First Acute Rejection Episode Following de Novo Cardiac Transplant (Pediatric Population)
Changes in Circulating Markers of Inflammation and Oxidation: Cystatin-C (Pediatric Population)
Changes in Circulating Markers of Inflammation and Oxidation: F2 Isoprostanes (Pediatric Population)
Changes in Circulating Markers of Inflammation and Oxidation: hsCRP (Pediatric Population)
Changes in Circulating Markers of Inflammation and Oxidation: Nitrotyrosine (Pediatric Population)
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: BNP
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Cystatin-C
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: E-selectin
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: F2 Isoprostanes
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Fibrinogen
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: GSH/GSSG
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Homocysteine
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: hsCRP
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: IL-18
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: IL-6
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: MCP-1
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Nitrotyrosine
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Osteopontin
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: s-ICAM
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: T-bars
Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Troponin T
Number of Acute Rejection Episodes by International Society of Heart and Lung Transplantation (ISHLT) Criteria
Number of Acute Rejection Episodes by International Society of Heart and Lung Transplantation (ISHLT) Criteria (Pediatric Population)
Number of Patients With Successful Steroid Taper or Withdrawal at Weeks 26 and 52
Number of Patients With Successful Steroid Taper or Withdrawal at Weeks 26 and 52 (Pediatric Population)
Number of Patients With Treatment Failure and Crossover for Treatment Failure
Number of Patients With Treatment Failure and Crossover for Treatment Failure (Pediatric Population)
The Change in the Markers of Growth, Apoptosis, Inflammation and Oxidation Measured in Endomyocardial Biopsies
The Change in the Markers of Growth, Apoptosis, Inflammation and Oxidation Measured in Endomyocardial Biopsies (Pediatric Population)
The Incidence of Biopsy-proven Acute Allograft Rejection During the First 12 Months of Transplant.
Renal Function (Nankivell Formula) at Month 12 Post Transplantation.
Changes in Cardiovascular Risk From Month 4.5 to Final Assessment at Month 12
Number of Participants Who Experienced an Adverse Event or Serious Adverse Event
Number of Participants With Occurrence of Biopsy Proven Acute Rejection (BPAR), Graft Loss or Death
Number of Participants With Occurrence of Treatment Failures
Number of Participants With an Episode of Clinically-suspected and Biopsy-proven Acute Rejection (CSPAR)
Mean Iohexol Clearance
Mean LDL Cholesterol, HDL Cholesterol, Total Cholesterol, Triglyceride, and Non-HDL Levels
Number of Participants Meeting Marked Abnormality Criteria for Select Hemolytic, Blood Chemistry, and Urinalysis Laboratory Test Results
Number of Participants With Death as Outcome, Serious Adverse Events (SAEs), Treatment-related SAEs, Discontinuations Due to SAEs, Adverse Events (AEs), Treatment-related AEs, and Discontinuations Due to AEs
Number of Participants With Hypertension
Number of Participants With Posttransplant Diabetes Mellitus
Percentage of Participants Who Had Chronic Allograft Nephropathy
Percentage of Participants Who Used Antihypertensive Medication
Percentage of Participants With Acute Rejection or Presumed Acute Rejection (PAR)
Percentage of Participants With Biopsy-proven Acute Rejection (BPAR) or Who Received Treatment for Acute Rejection
Percentage of Participants With Biopsy-proven Acute Rejection (BPAR) Through Months 6 and 12
AUC0-inf - Area Under the Concentration-time Curve From Time Zero to Infinity (Extrapolated)
AUC0-t - Area Under the Concentration-time Curve From Time Zero to Time of the Last Non-zero Concentration
Cmax - Maximum Observed Concentration
Event-free Survival
Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)
Overall Survival
Transplantation-related Mortality at 100 Days Post-transplantation
Incidence of Acute and Chronic Graft-versus-host Disease
Kinetics of Immunologic Reconstitution
Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting
Responses to Therapy
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Determine the Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) With Diagnosis of Engraftment Loss.
Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Diagnosed With Chronic Graft vs Host Disease (GVHD)
Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Diagnosed With Opportunistic Infections
Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) With Acute Graft Vs Host Disease (GVHD)
Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort)Diagnosed With Differences in the Rates of Engraftment Syndrome.
Differences in the Rates of Achieving Donor Chimerisms (the Percentage of DNA in the Sample Which Comes From the Donor) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort).
Differences in the Rates of T-cell Recovery Kinetics Following Stem Cell Transplantation (SCT) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort).
The Difference Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Day 60 Donor-derived (dd) Cluster of Differentiation (CD)3 10E3per microL Counts.
The Probability of Overall Survival (OS) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort)
Number of Participants With Absolute Neutrophil Count >= 500/Mcl for 3 Consecutive Measurements on Different Days and Platelet Count > 20,000/mm^3 With no Platelet Transfusions in the Preceding 7 Days
Number of Participants With Grade III-IV Acute GVHD as Determined by International Bone Marrow Transplant Registry (IBMTR) Severity Index Criteria
Number of Participants With Graft Failure
Number of Participants With Severe Chronic GVHD
Number of Participants With Treatment-related Mortality
Overall Survival (OS)
Progression-free Survival
Achievement of Remission
Disease-free Survival
Duration of Remission
Overall Survival
Rates of Donor Chimerism
Rates of Engraftment
The MTD of Radiation Delivered Via 90Y-DOTA-BC8 When Combined With FLU and 2 Gy TBI as a Preparative Regimen for Patients Aged ≥ 18 With Advanced AML, ALL, and High-risk MDS.
Estimation of Absorbed Radiation Doses to Normal Organs, Marrow and Tumor
Rates of Non-relapse Mortality
Percentage of Participants Surviving
Percentage of Participants With Acute Rejection
Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR)
Percentage of Participants With Graft Loss
Time to Rejection
Free MPA (mcg/mL) by Visit
IMPDH Expression I by Visit and Timepoint
IMPDH Expression II by Visit and Timepoint
Inosine MonoPhosphate DeHydrogenase (IMPDH) Activity by Visit and Timepoint
Interleukin 8 (IL-8) Expression by Visit and Timepoint
MPA Area Under the Concentration - Time Curve From Time 0 to 12 Hours (AUC0-12) (mcg/mL) by Visit
Percentage of Participants With Gastrointestinal Toxicities
Percentage of Participants With Hematologic Toxicity
Percentage of Participants With Infection
Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Gastrointestinal Toxicity
Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Hematologic Toxicity
Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Infection
Spearman's Rank Correlation Coefficient Between IMPDH I Expression and Free Fraction
Spearman's Rank Correlation Coefficient Between IMPDH I Expression and MPA Levels
Spearman's Rank Correlation Coefficient Between IMPDH II Expression and Free Fraction
Spearman's Rank Correlation Coefficient Between IMPDH II Expression and MPA Levels
Spearman's Rank Correlation Coefficient Between IMPDH Inhibition and Risk of Acute Rejection
Spearman's Rank Correlation Coefficient Between MPA Levels and IMPDH Activity
Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Gastrointestinal Toxicity
Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Hematologic Toxicity
Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Infection
Total Mycophenolate Acid (MPA) by Visit and Timepoint
Tumor Necrosis Factor (TNF) Expression by Visit and Timepoint
AUC0-12 of Free MPA at Day 20
AUC0-12 of Free MPA at Day 4
AUC0-12 of Free MPA at Day 8
AUC0-12 of Free MPA at Day 90
Cmax of Free MPA at Day 20
Cmax of Free MPA at Day 4
Cmax of Free MPA at Day 8
Cmax of Free MPA at Day 90
Cmin of Free MPA at Day 20
Cmin of Free MPA at Day 4
Cmin of Free MPA at Day 8
Cmin of Free MPA at Day 90
Forced Expiratory Volume in 1 Second (FEV1) at Day 90 Post-Transplantation
Forced Vital Capacity (FVC) at Day 90 Post-Transplantation
Free Fraction of Free MPA at Day 20
Free Fraction of Free MPA at Day 4
Free Fraction of Free MPA at Day 8
Free Fraction of Free MPA at Day 90
Percent of Predicted FEV1 at Day 90 Post-Transplantation
Percentage of Participants With Opportunistic Infections
Area Under the Curve From Time 0 to 12 Hours (AUC0-12) of MPA, MPAG and AcMPAG at Day 4
AUC0-12 of MPA, MPAG and AcMPAG at Day 20
AUC0-12 of MPA, MPAG and AcMPAG at Day 8
AUC0-12 of MPA, MPAG and AcMPAG at Day 90
Change From Baseline in Intracellular Adenosine-Tri-Phosphate (iATP) Levels
Change From Baseline in T-Cell Phenotype
CL of MPA, MPAG and AcMPAG at Day 20
CL of MPA, MPAG and AcMPAG at Day 8
CL of MPA, MPAG and AcMPAG at Day 90
Clearance (CL) of MPA, MPAG and AcMPAG at Day 4
Cmax of MPA, MPAG and AcMPAG at Day 20
Cmax of MPA, MPAG and AcMPAG at Day 8
Cmax of MPA, MPAG and AcMPAG at Day 90
Cmin of MPA, MPAG and AcMPAG at Day 20
Cmin of MPA, MPAG and AcMPAG at Day 8
Cmin of MPA, MPAG and AcMPAG at Day 90
Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 20
Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 4
Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 8
Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 90
Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 20
Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 4
Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 8
Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 90
Maximum Concentration (Cmax) of Mycophenolic Acid (MPA), Mycophenolic Acid Glucuronide (MPAG) and Acyl Glucuronide Metabolite of Mycophenolic Acid (AcMPAG) at Day 4
Minimum Concentration (Cmin) of MPA, MPAG and AcMPAG at Day 4
Time to Maximum Concentration (Tmax) of MPA, MPAG, AcMPAG and Free MPA at Day 4
Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 20
Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 8
Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 90
Volume of Distribution (Vz) of MPA, MPAG and AcMPAG at Day 4
Vz of MPA, MPAG and AcMPAG at Day 20
Vz of MPA, MPAG and AcMPAG at Day 8
Vz of MPA, MPAG and AcMPAG at Day 90
Percentage of Participants -Overall Participant and Graft Survival
Percentage of Participants Experiencing Acute Rejection
Percentage of Participants Positive for de Novo Donor-Specific Alloantibody Production in the First Year Post-Transplantation
Percentage of Participants Positive for Event of Death, Graft Loss or Rejection With Hemodynamic Compromise at 12 Months Post-Transplantation
Percentage of Participants Positive for Severe Infection(s)
Percentage of Participants With Occurrence of Re-Hospitalization(s)
Percentage of Participants With the Presence of Anti-HLA IgG Antibodies by Luminex SA Testing
Percentage of Participants With the Presence of Anti-MICA Antibodies by Luminex TM Assay
Percentage of Participants- Mortality While on Transplantation Wait-List
Presence of C4d on Endomyocardial Biopsy (EMB)
Time From Participant Listing on Organ Wait-List to Receiving Organ Transplant, Death or De-Listing
Time to Acute Rejection
Time to Diagnosis of Chronic Rejection
Time to New-Onset Diabetes Mellitus
Time to Post-Transplantation Lymphoproliferative Disorder
Time to Production of Post-Transplant de Novo Donor-specific Alloantibodies
Percentage of Participants -Quantification of Anti-HLA IgG Antibodies by Luminex SA Testing
Hazard Cox's Model Analysis of Pericardial/Pleural Effusions
Participants With at Least One Occurrence of Composite Treatment Failure Events
Participants With at Least One Occurrence of Safety Composite Endpoint After 6 Months by Treatment Group
Absolute and Percent Frequencies of Patients With LDL ≥ 100 mg/mL at 1, 3 and 6 Months, by Treatment Group
Partcipants With at Least One Occurrence of Each Safety Composite Endpoint Event After 6 Months by Treatment Group
Participants With CMV Infection and CMV Syndrome/Disease After 6 Months by Treatment Group
Chronic Allograft Damage Index (CADI) Score at Month 12 After Transplantation
Glomerular Filtration Rate (GFR) at Month 12 After Transplantation
Percentage of Participants With Treatment Failure at 12 Months Post-Transplant
Time to First Acute Rejection Post-Transplant
Time to First Acute Rejection Post-Transplant - Number of Participants With an Event
Glomerular Filtration Rate (GFR) (mL/Min)
Participant and Graft Survival
Percentage of Participants Experiencing Acute Rejection, Graft Loss, or Death at 6 and 12 Months Post-Transplant
Serum Creatinine (Micromoles Per Liter [µmol/L])
Average Daily Doses (mg) of Enteric-coated Mycophenolate Acid (MPA) and Mycophenolate Mofetil (MMF) by Treatment Duration Intervals
Number of Participants With Response (Yes/no)
Percentage of Participants Who Achieved Overall Response
Mean Absolute Counts of Cluster of Differentiation (CD) 19 Positive (+) Cells Per Visit
Number of Participants Who Achieved Complete Renal Response (CRR)
Percentage of Participants With CD19+ Absolute B Cell Counts <10 Cells Per Microliter (Cells/uL)
Percentage of Participants With CD19+ Absolute B Cell Counts <20 Cells/uL by Visit
Percentage of Participants With CD19+ Absolute B Cell Counts Less Than the Lower Limit of Normal (LLN) by Visit
Number of Patients Alive 24 Months Post Day 100 Transplant
Number of Patients With Day 100 Transplant-related Mortality
Disease Progression
Event-free Survival (EFS) at 1 Year
Full-dose Donor Chimerism (FDC) at Day 28 Following TLI/ATG/TBI Conditioning.
Non-relapse Mortality (NRM)
Overall Survival (OS)
Graft vs Host Disease (GvHD)
Area Under the Curve (AUC) of CD25 Saturation by Basiliximab From Day 0 to Day 84
AUC of Basiliximab Binding to CD25 Receptors From Day 0 to Day 84
Estimated Glomerular Filtration Rate (eGFR) at Day 8 and Week 24
Percentage of Participants With Biopsy Proven Acute Rejection (BPAR) According to Type and Severity
Percentage of Participants With of Biopsy Proven Acute Rejection (BPAR)
Percentage of Participants With of Treatment Failures
Percentage of T-cells That Bind Basiliximab to CD25 Receptors
Proportion of CD3+, CD4+, CD8+, CD19+ and CD56+ T Cells
Saturation Rate of CD25 Antigen Saturation by Basiliximab
3.0% or Greater Improvement From Baseline in FVC-%.
Forced Vital Capacity Volume (FVC, in ml)
Greater Than 5% Improvement in FVC-%
Health Assessment Questionnaire Modified for Scleroderma (HAQ-DI)
High Resolution Computerized Tomography (HRCT) Measures of Quantitative Interstitial Lung Disease Score in the Lobe of Maximal Involvement (QILD-LM)
High Resolution Computerized Tomography (HRCT) Measures of Quantitative Interstitial Lung Disease Score in the Whole Lung (QILD-WL)
High Resolution Computerized Tomography (HRCT) Measures of Quantitative Lung Fibrosis Score in the Lobe of Maximal Involvement (QLF-LM)
High Resolution Computerized Tomography (HRCT) Measures of Quantitative Lung Fibrosis Score in the Whole Lung (QLF-WL)
High Resolution Computerized Tomography (HRCT) Measures of Total Lung Capacity (TLC)
Mahler Modified Transitional Dyspnea Index (TDI)
Modified Rodnan Skin Score (mRSS)
Number of Participants With Treatment-related Adverse Events as Assessed by System Organ Classification Using Preferred Medical Dictionary for Regulatory Activities (MedDRA) Terms.
Percent Predicted Forced Vital Capacity (FVC-%)
Percent Predicted Single-breath Diffusing Capacity for Carbon Monoxide (DLCOHb-%)
St. George's Respiratory Questionnaire (SGRQ)
Time to Withdrawal From the Study Drug or Treatment Failure
Cataract - Incident Cataract
Change in Best-corrected Visual Acuity (Change in the Numbers of Letters Read From a Standard ETDRS Eye Chart) From Baseline to 24 Months in Eyes With Uveitis
Change in Self-reported Vision-related Function as Measured by the National Eye Institute 25-Item Visual Function Questionnaire (NEI-VFQ 25) Vision Targeted Composite Score From Baseline to 24 Months
Change in SF-36 Mental Component Score From Baseline to 24 Months
Change in SF-36 Physical Component Score From Baseline to 24 Months
Diabetes Mellitus
Glaucoma - Incident
Hyperlipidemia - Incident
Hypertension Diagnosis Requiring Treatment
Intraocular Pressure - Incident IOP Elevation >= 10 mmHg Above Baseline
Intraocular Pressure - Incident IOP Greater Than or Equal to 24 mm Hg
Intraocular Pressure - Incident IOP Greater Than or Equal to 30 mm Hg
Intraocular Pressure - IOP-lowering Surgery
Intraocular Pressure (IOP) - Incident Use of IOP-lowering Medical Therapy (Percentage of Eyes With Uveitis That Were Not Being Treated With IOP-lowering Medical Therapy at Baseline and Underwent IOP Lowering Therapy During the 24 Month Follow-up.
Macular Edema
Mortality
Uveitis Activity
Clinically Treated Acute Rejection
Efficacy Failure
Graft Survival at 12 Months
Overall Graft Survival
Overall Patient Incidence of BCAR
Overall Patient Survival
Patient Incidence of Biopsy-confirmed Acute Rejection (BCAR) at 6 Months
Patient Survival at 12 Months
Time to First BCAR
Renal Function Abnormalities Based on Creatinine Clearance
Renal Function Abnormalities Based on Serum Creatinine
Disease-Free Survival (DFS)
Event-free Survival (EFS)
Incidence of Regimen-related Mortality
Overall Survival (OS)
Incidence of Non-hematologic Regimen-related Toxicities
To Estimate the Cumulative Incidence of Relapse for Research Participants Who Receive This Study Treatment.
To Estimate the Rate of Overall Grade III-IV Acute GVHD, and the Rate and Severity of Chronic GVHD in Research Participants.
Proportion of Participants Successfully Withdrawn and Remain Off Immunosuppressants
Proportion of Participants Successfully Withdrawn From Immunosuppressants
Proportion of Participants Who Had Graft Loss or Death
Proportion of Participants Who Have Graft Loss or Death
Number of Events: Immunosuppression-related Complications
Change in Glomerular Filtration Rate Estimated by Iohexol Plasma Clearance 12 Months After Randomization Between the 2 Groups of Patients.
Change in the Glomerular Filtration Rate Estimated by Iohexol Plasma Clearance 12 Months After Randomization Between the 2 Groups of Patients Who Completed Trial
Assessing Cardiovascular Risk Factors Based on Fasting C-reactive Protein (CRP).
Assessing Cardiovascular Risk Factors Based on Fasting Glucose.
Assessing Cardiovascular Risk Factors Based on Fasting High-density Lipoprotein (HDL) Cholesterol, Low-density Lipoprotein (LDL) Cholesterol.
Assessing Cardiovascular Risk Factors Based on Fasting Total Cholesterol.
Assessing Cardiovascular Risk Factors Based on Fasting Triglycerides.
Change in Renal Function Assessed by Creatinine Clearance at Month 3, Month 6 and Month 12
Change in Renal Function Assessed by Proteinuria at Month 3, Month 6 and Month 12
Change in Renal Function Assessed by Serum Creatinine at Month 3, Month 6 and Month 12
Number of Participants With Biopsy-proven Acute Rejection (BPAR) at Month 6 and Month 12.
Number of Participants With Treatment Failures Assessed by Biopsy-proven Acute Rejection (BPAR), Graft Loss/Re-transplantation, Death or Lost to Follow-up at Month 12.
Number of Participants With the Occurrence of Treatment Failures at 6 Months Post-transplantation
Number of Participants Requiring Steroids in Non-steroid Treatment Group
Number of Participants With Subclinical Histological Rejections
Number of Participants With Treatment Failure at 3 Months by Graft Recovery Status
Number of Participants With Treatment Failure, BPAR, Clinical Acute Rejection (AR) and Treated AR at 3 Months
The Number of Participants With BPAR, Clinical Acute Rejection (AR) and Treated AR at 6 Months
The Number of Participants With Subclinical Rejection as Evaluated by a Change in Liver Enzymes
Changes in Gastrointestinal Symptom Severity and Health Related Quality of Life
Number of Patients With Treatment Failure 6-months Post Transplant Measured by the Combined Incidence of Biopsy Proven Acute Rejection, Graft Loss, and Death
Comparison of Overall Treatment Failure at Days 21 and 84 Post-transplantation Assessed by Biopsy Proven Acute Rejection (BPAR), GFL, and Death
Renal Function Assessed by Glomerular Filtration Rate (GFR)at Each Visit
Graft Survival
Overall Survival (OS) at Month 12 - Percentage of Participants With an Event
Overall Survival at Month 12
Percentage of Participants With Graft Loss
Percentage of Participants With Treated Biopsy Proven Acute Rejection (BPAR) According to Banff Criteria up to 12 Months Post-Transplant
Percentage of Participants by Graft Histology at 12 Months Post-Transplant - Central Review
Number of Participants With Engraftment Syndrome
Percentage of Participants With Disease-free Survival
Percentage of Participants With Overall Survival
Percentage of Participants With Relapse
Percentage of Participants With Treatment-related Mortality
Percentage of Participants With Acute Graft-versus-host Disease (GVHD)
Percentage of Participants With Chronic GVHD
Percentage of Participants With Neutrophil and Platelet Engraftment
Time to Neutrophil and Platelet Engraftment
Number of Participants Using Antihyperlipidemic Medications at Month 12 - Intent to Treat Population
Number of Participants With Acute Rejection of Transplant up to End of Month 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Number of Participants With Composite of Death, Graft Loss and Acute Rejection up to Month 12 - Intent to Treat Population
Number of Participants With Composite of Death, Graft Loss and Acute Rejection up to Month 6 - Intent to Treat Population
Number of Participants With Delayed Graft Function - Intent to Treat Population
Number of Participants With New Onset Diabetes Mellitus From Baseline to Month 12 Post Transplantation - Intent to Treat Population
Mean (Standard Deviation) in Calculated Glomerular Filtration Rate (GFR) mL/Min/1.73m^2 at Month 3, Month 6 and Month 12 Post Transplantation - Intent to Treat Population
Mean (Standard Deviation) in Calculated Glomerular Filtration Rate (GFR) mL/Min/1.73m^2 at Months 24, 36 and 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Mean Change From Baseline (BL) to Month 12 Post Transplantation in Lipid Values - Intent to Treat Population
Mean Systolic, Diastolic and Arterial Blood Pressure at Baseline and Month 12 - Intent to Treat Population
Number of Corticosteroid-free Participants at 6 and 12 Months Post Transplantation - Intent to Treat Population
Number of Corticosteroid-free Participants at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Number of Participants Who Switched Between MMF and Sirolimus During Long Term Extension up to Study Completion
Number of Participants Who Used Anti-hypertension Medications at Baseline and at 12 Months Post Transplantation - Intent to Treat Population
Number of Participants Who Were Both Calcineurin Inhibitor-free (CNI-free)and Corticosteroid-free at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Number of Participants Who Were Corticosteroid-free at Months 6 and 12 and Number of Participants Who Were Both Calcineurin Inhibitor-free (CNI-free)and Corticosteroid-free at Months 6 and 12 Post Transplantation - Intent to Treat Population
Number of Participants With Acute Rejection (AR) of Transplant up to 6 Months Post Transplantation - Intent to Treat (ITT) Population
Number of Participants With Acute Rejection of Transplant up to Month 12 Post Transplantation - Intent to Treat Population
Number of Participants With Graft Loss or Death at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Number of Participants With Graft Loss or Death up to Month 6 and Month 12 Post Transplantation - Intent to Treat Population
Change in Forced Expiratory Volume in 1 Second (FEV1) From Baseline to End of Study (Month 24) in the Lung Transplant Subgroup
Change in Forced Vital Capacity (FVC) From Baseline to End of Study (Month 24) in the Lung Transplant Subgroup
Mean Days of Hospitalization From Baseline to End of Study (Month 24)
Number of Patients in Need of Dialysis From Month 12 to End of Study (Month 24)
Number of Patients With Biopsy-proven Acute Rejection From Month 12 to End of Study (Month 24)
Change in Left Ventricular Function (Diameter and Thickness Parameters) From Baseline to End of Study (Month 24) in the Heart Transplant Subgroup
Change in Left Ventricular Function (Filling and Ejection Fraction Parameters) From Baseline to End of Study (Month 24) in the Heart Transplant Subgroup
Change in Measured Glomerular Filtration Rate (mGFR) From Baseline to End of Study (Month 24)
Change in Measured Glomerular Filtration Rate (mGFR) From Baseline to Month 12
Change in Serum Creatinine From Baseline to End of Study (Month 24)
Number of Patients Discontinued From the Study Due to Adverse Events From Month 12 to End of Study (Month 24)
Number of Patients Who Died and Number of Patients With Graft Loss From Month 12 to End of Study (Month 24)
Graft Survival at Three Years Post-Transplant
Number of Chronic Allograft Nephropathies
Number of Graft-versus-host Disease (GVHD) Events
Number of Kidney Biopsy-proven Acute Rejection
Overall Kidney Graft Survival at One Year Post-Transplant
Overall Participant Survival at One Year Post Kidney Transplant
Participant Survival at Three Years Post Kidney Transplant
Incidence of Chronic Extensive GVHD
Incidence of Grades II-IV Acute GVHD
Incidence of Graft Rejection
Incidence of Non-relapse Mortality
Incidence of Relapse-related Mortality
Incidence of Relapse/Progression
Overall Survival
Progression-free Survival
Duration (Days) Until Participants Obtained Platelet Engraftment
Incidence of Acute or Chronic Graft-versus-host Disease (GVHD)
Incidence of Clinically Significant Infections
Incidence of Relapse or Disease Progression
Non-relapse Mortality
Number of Participants Surviving by 1 Year
Number of Participants Surviving up to 2 Years Without Disease Progression
Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100
Number of Participants With Graft Failure/Rejection
Number of Participants With Secondary Graft Failure
Number of Patients With Non-relapse Mortality (NRM)
The Number of Participants Alive at Two-years Follow up.
Change in SLICC/ACR Damage Index From Baseline During Short-term Period
Number of Months CRR Was Maintained During Short-term Period
Number of Participants Achieving Renal Response (RR) at Month 12 During Short-term Period
Number of Participants With a Treatment-emergent Seropositive Result During the Long-term Extension Period
Number of Participants With Confirmed Complete Renal Response (CRR) During Short-term Period
Participants Achieving a Confirmed Complete Renal Response (CRR) at Month 12 During Short-term Period
Participants Achieving Renal Improvement (RI) or CRR at Month 12 During Short-term Period
Time to Achieve First Confirmed Renal Improvement (RI) During Short-term Period (as Determined by Kaplan-Meier Methodology)
Time to First Confirmed Complete Renal Response (CRR) During the Short-term (Double-blind) Period
Baseline and Post Baseline Systemic Lupus International Collaborating Clinics (SLICC)/American College of Rheumatology (ACR) Damage Index During Short-term Period
Baseline Fatigue as Measured by Fatigue Severity Scale-Krupp During Short-term Period
Baseline Fatigue as Measured by the Fatigue Visual Analog Scale During Short-term Period
Baseline Mental Component Summary of the Short SF-36 During Short-term Period
Baseline Physical Component Summary of the Short Form (SF)-36 During Short-term Period
Baseline Quantitative Immunoglobulins During the Short-term Period
Baseline Renal Function Over Time During Short-term Period
Change From Baseline in Mental Component Summary of the SF-36 During Short-term Period
Change From Baseline in Physical Component Summary of the SF-36 During Short-term Period
Change in Fatigue From Baseline as Measured by Fatigue Severity Scale-Krupp During Short-term Period
Change in Fatigue From Baseline as Measured by the Fatigue Visual Analog Scale During Short-term Period
Change in Quantitative Immunoglobulin From Baseline During Short-term Period
Change in Renal Function From Baseline Over Time During Short-term Period
Mean Change From Baseline in SLICC/ACR Damage Index
Number of Participants Achieving Complete Response by ACCESS Definition
Number of Participants Achieving Patient Response (PR) at Month 12 During the Short-term Period
Number of Participants Achieving Patient Response of Complete or Partial Response, Based on the June 2010 Food and Drug Administration Guidance Document for Lupus Nephritis
Number of Participants Achieving Renal Response
Number of Participants With Death, Serious Adverse Events (SAE), Treatment-related Adverse Events SAEs, Discontinuations Due to SAEs, Adverse Events (AEs), Treatment-related AEs, and Discontinuations Due to AEs During Long-term Extension Period
Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period
Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period (Continued)
Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period (Continued)
Number of Participants With Positive Abatacept-induced Responses (ECL Method) Over Time During the Short-term Period
Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths, and Discontinuations Due to AEs Reported During the Short-term Period
Participants With AEs of Special Interest During the Short-term Period
Participants With Marked Abnormalities Urinalysis During the Short-term Period
Participants With Marked Hematology Abnormalities During the Short-term Period
Participants With Marked Laboratory Abnormalities During the Short-term Period
Participants With Marked Liver and Kidney Function Abnormalities During the Short-term Period
Vital Signs Summary During the Short-term Period: Diastolic Blood Pressure (DBP)
Vital Signs Summary During the Short-term Period: Heart Rate
Vital Signs Summary During the Short-term Period: Systolic Blood Pressure (SBP)
Vital Signs Summary During the Short-term Period: Temperature
Number of Participants (Patients) Who Attained Neutrophil Engraftment
Number of Participants (Patients) Who Attained Platelet Engraftment
Number of Participants (Patients) Who Died by 12 Months
Number of Participants (Patients) Who Died by 24 Months
Number of Participants (Patients) Who Died Due to Transplant.
Number of Participants (Patients) Who Experienced Relapse by 12 Months
Number of Participants (Patients) Who Experienced Relapse by 24 Months
Number of Participants (Patients) Who Were Disease-free and Alive at 12 Months
Number of Participants (Patients) Who Were Disease-free and Alive at 6 Months
Number of Participants (Patients) With Acute Graft-versus-host Disease (GVHD) Grade II-IV
Number of Participants (Patients) With Acute Graft-versus-Host Disease at Grade III-IV
Number of Participants (Patients) With Chronic Graft-Versus-Host Disease
Number of Participants (Patients) With Successful Natural Killer Cell Expansion
Number of Patients Who Were Disease-free and Alive at 24 Months
Chimerism After Double Umbilical Cord Blood Transplant (UCBT)
GFR at Month 12 Utilizing Cockcroft-Gault Formula
GFR at Month 12 Utilizing Modification of Diet in Renal Disease (MDRD) Method
GFR at Month 60 Utilizing Cockcroft-Gault Formula
GFR at Month 60 Utilizing Modification of Diet in Renal Disease (MDRD) Method
GFR Calculated Via Nankivell Formula at Month 60
GFR Via Nankivell Formula at Month 12 - All Regimens
GFR Via Nankivell Method at Month 12 - CNI-Free vs Standard Regimen
Mean Change in Serum Creatinine From Month 3 to Month 12
Mean Change in Serum Creatinine From Month 3 to Month 60
Change From BL2 (Month 3) to Month 12 in Cardiovascular Risk (Framingham Score; 10-year Cardiovascular Risk)
Efficacy Event Data After Month 12 to Month 60
Efficacy Event Data Baseline 2 (Month 3) to Month 12
Efficacy Event Data From Baseline 2 (Month 3) to Month 6
Gastrointestinal Toxicity Due to Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Formulation Manufactured by Sandoz
Modification of Diet and Renal Disease (MDRD) Measured Glomerular Filtration Rates in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
Serum Creatinine in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
The Incidence of Intolerance (Defined as Transient Dose Reduction or Transient Discontinuation of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
The Incidence of Rejection in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz.
The Incidence of the Requirement of Full Dose Proton Pump Inhibitors in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
Gastrointestinal Symptom Rating Scale (GSRS) Score Changes From Baseline to 24 Weeks After Transplant in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
Pharmacokinetics (Mycophenolic Acid Area Under the Curve) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients
Pharmacokinetics (Mycophenolic Acid Maximum Concentration) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients
Pharmacokinetics (Mycophenolic Acid Trough Levels) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients
The Incidence of the Occurrence of Lower Gastrointestinal Symptoms Per GSRS Scale Ratings in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
The Incidence of the Occurrence of Upper Gastrointestinal Symptoms Per GSRS Scale Ratings in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Formulation Manufactured by Sandoz
Change From Baseline in the Average Maximum Intimal Thickness at Month 12
Percentage of Participants With Cardiac Allograft Vasculopathy (CAV) at Month 12
Percentage of Participants With Composite Efficacy Failure at 12 Months
Percentage of Participants With Composite Efficacy Failure at 24 Months
Percentage of Participants With Graft Loss/Re-transplant, Death or Loss to Follow-up at 12 Months
Percentage of Participants With Graft Loss/Re-transplant, Death or Loss to Follow-up at 24 Months
Renal Function Calculated by Glomerular Filtration Rate (GFR) at 24 Months
Renal Function Measured by Glomerular Filtration Rate (GFR) at 12 Months
Percentage of Participants With Biopsy-proven Acute Rejection (BPAR of ISHLT Grade ≥ 3A), Acute Rejection (AR) Associated With Hemodynamic Compromise (HDC), Graft Loss/Re-transplant and Death at Month 24
Percentage of Participants With Biopsy-proven Acute Rejection (BPAR of ISHLT Grade ≥ 3A), Acute Rejection Associated With Hemodynamic Compromise (HDC), Graft Loss/Re-transplant and Death at Month 12
Donor Stem Cell Engraftment, Including Donor-host Hematopoietic Chimerism Studies Post Transplant
Incidence of 100 Day Mortality
Incidence of Chronic GVHD
Overall Survival
The Rate of Renal Insufficiency
To Determine the Rate of Grade II-IV Acute GVHD When Sirolimus and Mycophenolate Mofetil or Sirolimus, Mycophenolate Mofetil and Bortezomib is Used for GVHD Prophylaxis After Allogeneic Stem Cell Transplantation in Patients With Hematologic Malignancies
To Correlate the Serum Concentrations of Mycophenolate Mofetil and Its Metabolites With Acute GVHD Incidence
Number of Participants With Acute Graft-versus-host Disease (aGVHD)
Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Deaths
Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Participants With End-stage Renal Disease (ESRD)
Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Participants With Sustained Doubling of Serum Creatinine
Maintenance Phase: Participants With Major Extra-renal Flare
Induction Phase: Change From Baseline in Short-Form Health Survey (SF-36) Domain and Component Scores
Induction Phase: Change From Baseline to Week 24 in 24-hour Urine Protein
Induction Phase: Change From Baseline to Week 24 in Serum Albumin
Induction Phase: Change From Baseline to Week 24 in Serum Creatinine
Induction Phase: Change in Renal British Isles Lupus Assessment Group (BILAG) Score
Induction Phase: Number of Participants Achieving Complete Remission
Induction Phase: Number of Patients Showing Treatment Response
Maintenance Phase: Events Contributing to the Primary Endpoint: Kaplan-Meier Estimates of Percentage of Participants Not Receiving Rescue Therapy
Maintenance Phase: Events Contributing to the Primary Endpoint: Kaplan-Meier Estimates of Percentage of Participants Renal Flare Free, by Time Interval
Maintenance Phase: Kaplan-Meier Estimates of Percentage of Participants Treatment Failure Free, by Time Interval
Survival
Percentage of Participants With BCAR at Month 12 Assessed by Local Review
Percentage of Participants With Biopsy-confirmed Acute Rejection (BCAR) at Month 6 Assessed by Local Review
Time to First BCAR Assessed by Central Review
Time to First BCAR Assessed by Local Review
Time to First T-cell Mediated BCAR Assessed by Central Review
Time to First T-cell Mediated BCAR Assessed by Local Review
Change From Week 4 in GFR by Iothalamate Clearance at Month 6
Change From Week 4 in Glomerular Filtration Rate Estimated by the MDRD Method at Month 6 and Month 12
Change From Week 4 in Serum Creatinine at Month 6 and 12
Gastrointestinal Quality of Life Index Score Over Time
Gastrointestinal Symptom Rating Scale Scores Over Time
Graft Survival at Month 6 and Month 12
Maximum Grade of T-cell Mediated Rejection as Assessed by Central Review
Maximum Grade of T-cell Mediated Rejection Assessed by Local Review
Patient Survival at Month 6 and Month 12
Percentage of Participants With Anti-lymphocyte-treated Rejection at Months 6 and 12
Percentage of Participants With BCAR at Month 6 and 12 Assessed by Central Review
Percentage of Participants With Clinically Treated Acute Rejection at Month 6 and Month 12
Percentage of Participants With Efficacy Failure at 6 and 12 Months Assessed by Central Review
Percentage of Participants With Efficacy Failure at 6 and 12 Months Assessed by Local Review
Percentage of Participants With Multiple Rejection Episodes at Months 6 and 12
Percentage of Participants With T-cell Mediated BCAR at Month 6 and 12 Assessed by Central Review
Percentage of Participants With T-cell Mediated BCAR at Month 6 and 12 Assessed by Local Review
Percentage of Participants With Treatment Failure at Month 6 and 12
Achieving Full Donor Chimerism
Event-free Survival (EFS)
Incidence of Acute Graft Versus Host Disease (GvHD)
Incidence of Chemotherapy-associated Pneumonitis
Incidence of Chronic Graft Versus Host Disease (GvHD)
Median Time to Neutrophile Engraftment
Median Time to Platelet Engraftment
Overall Mortality Rate
Overall Survival (OS)
Relapse Rate
Area Under the Curve (AUC) From Time Zero to 12 Hours Post-Dose [AUC (0-12)] of Mycophenolic Acid (MPA)
Area Under the Curve From Time Zero to 12 Hours Post-Dose [AUC (0-12)] of Mycophenolic Acid Glucuronide (MPAG)
Maximum Observed Plasma Concentration (Cmax) of Mycophenolic Acid (MPA)
Maximum Observed Plasma Concentration (Cmax) of Mycophenolic Acid Glucuronide (MPAG)
Minimum Observed Plasma Concentration (Cmin) of Mycophenolic Acid (MPA)
Minimum Observed Plasma Concentration (Cmin) of Mycophenolic Acid Glucuronide (MPAG)
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mycophenolic Acid (MPA)
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mycophenolic Acid Glucuronide (MPAG)
GI Mucosal Lesions Change and Clinical Symptoms Using The Gastrointestinal Symptom Rating Scale (GSRS) Score
Calculated Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula at Month 18
Reciprocal Creatinine Slope Between Month 6 and Month 18
Calculated Glomerular Filtration Rate (cGFR) According to Cockcroft-Gault at Month 12 and 18
Occurrence of Major Cardiac Events (MACE) From Month 6 to 18
Occurrence of Treatment Failures From Month 6 to 9 and Month 9 to 18
Serum Creatinine at Month 6, 8, 9, 10 12 and 18
The Number of Subjects Who Experience a Change From Baseline in Their Panel of Reactive Antibody (PRA) Titers at 12 Months Post Rituximab Infusion.
The Number of Subjects Who Experience a Decrease in Their Panel of Reactive Antibodies (PRA) at 6 Months Post Rituximab Infusion.
The Number of Subjects With a Negative Crossmatch at the Time of Transplant.
Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments
Number of Participants Who Experienced Transplantation-related Mortality (TRM)
Two Year Overall Survival
Change From Baseline in Corrected Creatinine Clearance (mL/Min) at Week 52
Percentage of Participants Experiencing Acute Rejection, Graft Loss, Death, or a Decrease From BL in Creatinine Clearance of ≥20% at Week 52
Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) at Week 52
Percentage of Participants With Decrease in Glomerular Filtration Rate (GFR) of Greater Than 20%
Changes From Baseline in Creatinine Clearance (Milliliters Per Minute [mL/Min])
Percentage Change in Creatinine Clearance From Baseline
Percentage of Participants With Graft Loss or Death at Week 52
Event-free Survival (EFS) Per Protocol
Incidence of Chronic Graft vs Host Disease (GvHD)
Median Time to Neutrophil Engraftment After Allogeneic Transplant
Median Time to Neutrophil Engraftment After Autologous Transplant
Median Time to Platelet Engraftment After Allogeneic Transplant
Median Time to Platelet Engraftment After Autologous Transplant
Overall Survival (OS)
Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)
Acute GvHD (Grade 3 to 4)
Disease-free Survival (DFS)
Overall Survival
Veno-occlusive Disease (VoD)
cGVHD
Disease Free Survival
Engraftments
Grade III-IV aGVHD
Number of Participants With Non Related Mortality (NRM)
Number of Participants With Non-relapse Mortality (NRM)
Blood Pressure
Blood Pressure
Changes of Carotid Intima-media Thickness Over Time
Lipidic Profile (Cholesterol)
Lipidic Profile (HDL-c)
Lipidic Profile (LDL-c)
Lipidic Profile (Triglycerides)
Number of Antihypertensive Drugs Patients Reported Taking.
Patients Treated With Insulin or Oral Antidiabetic Drugs
Percentage of Patients Using Acetylsalicylic Acid (ASA)
Percentage of Patients Using Statins
Primary Outcome Measure (Glucose Intolerance)
Proteinuria
Rejection
Renal Function
"Primary Outcome Measure New Onset Diabetes After Renal Transplantation (NODAT)"
Fibrosis Score, as Measured by Thoracic High Resolution Computerized Tomography (HRCT)
Forced Vital Capacity (FVC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value
Health-related Quality of Life as Measured by the Patient Responses to the Health Assessment Questionnaire Disability Index (HAQ-DI)
Single-breath Diffusing Capacity for Carbon Monoxide (DLCO), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value
Skin Involvement, as Measured by the Modified Rodnam Skin Thickness Scores (mRSS)
Tolerability, as Assessed by the Time to Withdrawal From the Study Drug or Meeting Protocol-defined Criteria for Treatment Failure.
Total Lung Capacity (TLC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value
Toxicity, as Measured by Adverse Events, Serious Adverse Events, and Death
Transitional Dyspnea Index Score
Number of Participants With Cytomegalovirus Infection or Disease
Gastrointestinal Side Effects and Quality of Life (-Subscales of GSRS)
Gastrointestinal Side Effects and Quality of Life (Total Score of GSRS)
Comparison by Treatment Assignment in the Number of Biopsy-Proven Acute Rejections Within 12 Months Post Kidney Transplantation
The Difference in Linear Growth by Treatment Assignment at 1 Year Post Kidney Transplantation
Number of Participants Who Experienced Adverse Events and Death
Disease Free
Disease Free
Hematologic Engraftment
Incidence of Acute Graft-versus-host Disease (GVHD)
Incidence of Chronic GVHD
Occurrence of Serious Infections
Overall Survival
Overall Survival
Overall Survival
Recurrence or Relapse
Recurrence or Relapse
Toxicity Related to UCB Transplantation and Cytoreduction as Assessed by CTC v3.0
Transplant Related Mortality
Transplant Related Mortality
Complete Response Rate (CRR)
Event-free Survival (EFS)
Incidence of Graft Versus Host Disease (GvHD)
Overall Response Rate (ORR)
Overall Survival (OS)
Partial Response Rate (PRR)
Median Time to Engraftment After Allo-PBSC Transplant
Median Time to Engraftment After Auto-PBSC Transplant
Number of Patient Who Gained Remission From the Nephrotic Syndrome
Number of Patients Achieved Remission
Core Study: Number Participants With Combined Graft Loss, Death or Loss to Follow-up
Core Study: Renal Function Measured by Calculated Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula
Extension Study: Renal Function Measured by Calculated Glomerular Filtration Rate (cGFR) Using the Modification of Diet in Renal Disease (MDRD) Formula
Extension Study: Renal Function Measured by Calculated Glomerular Filtration Rate (cGFR) Using the Modification of Diet in Renal Disease (MDRD) Formula
Core Study: Number of Patients With Composite Efficacy Endpoint
Extension Study: Cyclosporine Trough Levels
Extension Study: Everolimus Trough Levels
Extension Study: Number of Participants With Adverse Events and Serious Adverse Events
Extension Study: Number of Participants With Combined Efficacy Endpoint: Graft Loss, Death, Loss to Follow-up and/or Treated Biopsy Proven Acute Rejection (BPAR)
Extension Study: Renal Function Measured by Calculated Glomerular Filtration Rate (GFR) Using the Nankivell Formula
Annual Change Rate in Total Plaque Volume (TPV) From Pre-conversion Baseline to 12 Months Post-transplant
CIMT at Pre-conversion Baseline
TPV at Pre-conversion Baseline
Annual Change Rate in Carotid Intima Media Thickness (CIMT) From Pre-conversion Baseline at 12, 18, 24 and 36 Months Post-transplant
Annual Rate of Change in TPV From Pre-conversion Baseline to 18, 24 and 36 Months Post Transplant
Change From Pre-conversion Baseline in Adiponectin at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Endothelin-1 at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Fasting Lipid Parameters at 12, 18, 24 and 36 Months Post-transplant
Change From Pre-conversion Baseline in Fibrinogen at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Glucose at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Glycosylated Hemoglobin(HbA1C) at Months 12, 24, and 36 Post-transplant
Change From Pre-conversion Baseline in High Sensitivity C-Reactive Protein (hsCRP) at Months 12, 24 and 36 Post-transplant.
Change From Pre-conversion Baseline in Homocysteine at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Insulin at Months 12, 24, and 36 Post-transplant
Change From Pre-conversion Baseline in Interleukin-6 (IL-6) at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Lipoprotein(a) at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Tumor Necrosis Factor Alpha (TNF-alpha) at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Uric Acid at Months 12, 24 and 36 Post-transplant
Change From Pre-conversion Baseline in Vitamin B12 at Months 12, 24 and 36 Post-transplant
Number of Participants Who Used Anti-hypertensive Medications
Number of Participants Who Used Lipid Lowering Therapies
GFR Measured by Iothalamate Clearance at Month 6
Graft Survival
Patient Survival
Percentage of Participants With Acute Rejection Diagnosed by Signs and Symptoms at Month 6
Percentage of Participants With Anti-Lymphocyte Antibody Therapy for Treatment of Rejection at Month 6
Percentage of Participants With Biopsy Confirmed Acute Mixed T-Cell Mediated and Antibody-Mediated Rejection at Month 6
Percentage of Participants With Biopsy Confirmed Acute Rejection (T-Cell Mediated or Antibody Mediated) at Month 6
Percentage of Participants With Biopsy Confirmed Antibody-Mediated Acute Rejection at Month 6
Percentage of Participants With Biopsy-Confirmed Acute T-cell Mediated Rejection as Assessed by Central Review at Month 6
Percentage of Participants With Biopsy-confirmed Acute T-cell Mediated Rejection at Month 6 Assessed by Local Review
Percentage of Participants With Clinically Treated Acute Rejection at Month 6
Percentage of Participants With Delayed Graft Function
Percentage of Participants With Efficacy Failure at Month 6
Percentage of Participants With Steroid-resistant Acute Rejection at Month 6
Percentage of Participants With Treatment Failure at Month 6
Change From Month 1 in Creatinine Clearance
Change From Month 1 in Glomerular Filtration Rate (GFR)
Change From Month 1 in Serum Creatinine
Maximum Histological Grade of All Biopsies After Local Review
Number of Participants With Adverse Events
Mycophenolic Acid (MPA) Maintenance Treatment
Changes in Gastrointestinal (GI) Symptoms as Measured by the Gastrointestinal Symptom Rating Scale (GSRS).
Changes in Gastrointestinal Symptoms as Measured by the Gastrointestinal Quality of Life Index (GIQLI).
Freedom From Acute Rejection or HCV Recurrence or Treatment Failure
Freedom From HCV Recurrence Within First Year That Requires HCV Antiviral Therapy and Freedom From Treatment Failure
Incidence of Chronic Graft-versus-host-disease (GVHD) at 1 Year
Incidence of Clinically Significant Infections at 1 Year
Incidence of Clinically Significant Infections at 2 Years
Incidence of Clinically Significant Infections at 6 Months
Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD) at Day 100
Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD) at Day 100
Incidence of Neutrophil Engraftment at Day 42
Incidence of Non-relapse Mortality at 6 Months
Incidence of Platelet Engraftment at 6 Months
Incidence of Relapse at 1 Year
Incidence of Relapse at 2 Years
Probability of Progression-free Survival at 1 Year
Probability of Progression-free Survival at 2 Years
Probability of Survival at 1 Year
Probability of Survival at 2 Years
Chimerism
The Proportion of Participants With Graft Loss or Death Within 12 Months Post Kidney Transplantation
Duration of Exposure to Study Medication
Change From Baseline in Overall Disease Activity With British Isles Lupus Assessment Group (BILAG)
Change From Baseline in Overall Disease Activity With Systematic Lupus Erythematosus Disease Activity Index (SLEDAI)
Cumulative Dose of Prednisone Equivalent Corticosteroids (CS)
Number of Patients With Adverse Events and Infections
Number of Patients With Complete Remission
Number of Patients With Complete Remission
Number of Patients With Moderate to Severe Flares
Number of Patients With Partial Remission
Number of Patients With Treatment Failure
Calculated Glomerular Filtration Rate (cGFR) After Kidney Transplant to Evaluate Kidney Function (12 Months Analysis)
Creatinine Clearance (CrCl) Calculated by the Cockcroft-Gault Formula (12 Months Analysis)
Mean Serum Creatinine (12 Months Analysis)
Mean Urine Albumin/Creatinine Ratio (ACR) as Measurement of Proteinuria (12 Months Analysis)
Number of Participants Hospitalized for Reasons Other Than Primary Transplantation (12 Months Analysis)
Number of Participants Hospitalized for Reasons Other Than Primary Transplantation (36 Months Analysis)
Number of Participants With Antibody-mediated Rejection Per Treatment Group (12 Months Analysis)
Number of Participants With Biopsy Proven Acute Rejection (BPAR) Per Treatment Group (12 Months Analysis)
Number of Participants With Composite Endpoint of Treatment Failure (12 Months Analysis)
Number of Participants With Erythropoietin Usage (12 Months Analysis)
Number of Participants With Erythropoietin Usage (36 Months Analysis)
Calculated Glomerular Filtration Rate (cGFR) After Kidney Transplant to Evaluate Kidney Function (36 Months Analysis)
Change in Bone Mineral Density Between Week 2 and Month 24 (36 Months Analysis)
Creatinine Clearance Calculated by the Cockcroft-Gault Formula (36 Months Analysis)
Mean Serum Creatinine (36 Months Analysis)
Mean Short-form 36 Health Survey (SF-36) Score as a Measure of Quality of Life Assessment (12 Months Analysis)
Mean Short-form 36 Health Survey (SF-36) Score as a Measure of Quality of Life Assessment (36 Months Analysis)
Mean Urine Albumin/Creatinine Ratio [ACR] as Measurement of Proteinuria (36 Months Analysis)
Number of Participants With Antibody-mediated Rejection Per Treatment Group (36 Months Analysis)
Number of Participants With Any Wound Problems (36 Months Analysis)
Number of Participants With Biopsy Proven Acute Rejection (BPAR) Influenced by Demographic Characteristics and Morbidities (12 Months Analysis)
Number of Participants With Biopsy Proven Acute Rejection (BPAR) Influenced by Demographic Characteristics and Morbidities (36 Months Analysis)
Number of Participants With Biopsy Proven Acute Rejection (BPAR) Per Treatment Group (36 Months Analysis)
Number of Participants With Composite Endpoint of Treatment Failure (36 Months Analysis)
Number of Participants With Employment Status (12 Months Analysis)
Number of Participants With Employment Status (36 Months Analysis)
Number of Participants With Histological Evidence Chronic Allograft Nephropathy (CAN) (12 Months Analysis)
Number of Participants With Histological Evidence Chronic Allograft Nephropathy (CAN) (36 Months Analysis)
Number of Participants With New Onset Diabetes Mellitus After Transplantation (NODAT) and Impaired Fasting Glucose (36 Months Analysis)
Number of Participants With Notable Abnormal Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) as Measurement of Effect of Treatment on Cardiovascular Health (12 Months Analysis)
Number of Participants With Notable Abnormal Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) as Measurement of Effect of Treatment on Cardiovascular Health (36 Months Analysis)
Number of Participants With Notable Abnormalities in Total Cholesterol and Triglycerides as Measurement of Effect of Treatment on Cardiovascular Health (12 Months Analysis)
Number of Participants With Notable Abnormalities in Total Cholesterol and Triglycerides as Measurement of Effect of Treatment on Cardiovascular Health (36 Months Analysis)
Number of Participants With Post Transplant Diabetes Mellitus (PTDM) and Impaired Fasting Glucose (12 Months Analysis)
Number of Participants With Sub Clinical Acute Rejection (12 Months Analysis)
Number of Participants With Sub Clinical Acute Rejection (36 Months Analysis)
Number of Participants With Wound Problems(12 Months Analysis)
Number of Patient Survival and Graft Survival (12 Months Analysis)
Number of Patient Survival and Graft Survival (36 Months Analysis)
Change From Baseline in Lower and Upper GI Symptom Burden Measured by GI Symptom Rating Scale Score
Change From Baseline (BL) to Day 30 in the Gastrointestinal Quality of Life Index (GIQLI) Total Score and Subscale Scores
Change From Baseline to Day 30 in the Severity of Gastrointestinal Symptoms Overall Total Score
Change in Gastrointestinal Symptom Rating Scale Subscale Scores After 30 Days of Treatment
Number of Participants With Biopsy-proven Acute Rejection (BPAR) and Treated Acute Rejection (TAR)
Number of Participants With Reported Dose Changes or Interruption of Study Medication During the 30 Days of Treatment
The Number of Participants Who Responded to the Conversion to Mycophenolate Sodium (EC-MPS) Therapy
Acute Graft-vs-Host-Disease (aGvHD)
Event-free Survival (EFS)
Overall Survival (OS)
Relapse Rate
Number of Participants With Any Treatment Failure
Time to First Occurrence of a Mycophenolic Acid (MPA) Plasma Concentration of ≥ 40 mg*h/L
Number of Participants With Single Treatment Failures
Renal Function as Measured by Glomerular Filtration Rate (GFR)
Renal Function as Measured by Serum Creatinine
Change in Proteinuria - Uprotein/Creatinine Ratio
Number of Participants Who Died Due to Transplant
Number of Participants Who Were Alive at 1 Year Transplant Overall Survival
Number of Participants With Chronic Graft-Versus-Host Disease
Number of Participants With Neutrophil Engraftment
Number of Participants With Platelet Engraftment
Percentage Chimerism at 1 Year
Percentage Chimerism at 2 Years
Percentage Chimerism at 6 Months
Percentage Chimerism on Day 100
Percentage Chimerism on Day 21
Number of Participants With Acute Graft-Versus-Host Disease
12 Month Disease Free Survival Probability
Overall Survival
Progression Free Survival
Rate of Acute GvHD
Relapse-free Mortality
Number of Participants With Graft and Patient Survivals at 6 Months
Number of Participants With Treatment Failure Parameters (Biopsy-Proven Acute Rejection (BPAR), Graft Loss, Death, or Loss to Follow-up) at 6 Months
Renal Function Assessed by Change in Estimated Glomerular Filtration Rate(eGFR)
Renal Function at 3 Months Assessed by Change in Estimated Glomerular Filtration Rate (eGFR)
Acute Rejection Rate
Incidence and Severity of HCV Recurrence Post-OLT
Infection as an Adverse Effect of Steroids
New-onset Diabetes Mellitus (NODM) as Secondary Outcome
Graft Survival Rate
Patient Survival Rate
Changes in Gastrointestinal Symptom Severity and/or Health-related Quality of Life After Conversion From MMF to Enteric Coated Mycophenolate Sodium
Gastrointestinal Symptoms Under MMF-based Immunosuppressive Therapy
Non-inferiority Analysis of Renal Function, Calculated by Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula
Non-inferiority Analysis on Percentage of Participants With Composite Efficacy Endpoints
Percentage of Participants With the Composite Incidence of Graft Loss, Death or Loss to Follow up at 12 Months Post-transplantation
Number of Participants With Composite Efficacy Endpoints - 12 Month Analysis
The Number of Kidney Transplant up to 12 Months.
The Number of Pariticpants With a White Blood Cell Count Below 2.0 Thousand (Low) or Total IgG/IgM Titers Below Range (620-1490 mg/dL).
The Number of Subjects With a 10% Decrease in PRA Level at Month 8.
The Number of Subjects With Significant Infections up to Month 12.
The Number of Transplants With a Negative Crossmatch at Transplant.
Number of Biopsy Proven Rejections at 12 Months
Number of Participants With Adverse Events Including Infections at 12 Months
Patient and Graft Survival at 12 Months
Number of Participants Who Experienced Adverse Events, Serious Adverse Events and Death
Assessment of GFR by the Cockcroft-Gault Method (LOCF)
Renal Function Assessed as Glomerula Filtration Rate (GFR) - Nankivell Method - 9 Months After Renal Transplantation (LOCF)
Change in Renal Function (Creatinine Slope)
Participants Who Had Occurrence of Biopsy Proven Acute Rejection, Graft Loss or Death.
Participants Who Had Occurrence of Treatment Failure.
Mean Creatinine Clearance Rate
Mean Creatinine Clearance Rate - 3 Months
Number of Patients With Acute Rejection
Patient and Graft Survival
Number of Participants With Adverse Events (AEs)
Percent Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 24
Incidence of Hepatotoxicity as Measured by Bilirubin
Incidence of Hepatotoxicity as Measured by Veno-occlusive Disease (VOD)
Incidence of Infection
Incidence of Pulmonary Toxicity Measured by Pulmonary Edema
Incidence of Pulmonary Toxicity Measured by Pulmonary Hemorrhage
Incidence of Pulmonary Toxicity Measured by Respiratory Failure
Length of Hospitalization
Length of Time on Continuous Infusion Narcotics
Overall Survival
Percentage of Participants Using Total Parenteral Nutrition (TPN) Within 100 Days
Percentage of Severe (Grade 3-4) Mucositis Graded According to the World Health Organization (WHO) Grading Scale
Progression-free Survival
Time to Neutrophil Engraftment
Time to Platelet Engraftment
Cumulative Incidence of Participants With Acute GVHD
Incidence of Chronic GVHD
Incidence of Chronic GVHD
Incidence of Hepatotoxicity as Measured by Elevated Liver Enzymes
Incidence of Nephrotoxicity
Change From Baseline in Left Ventricular Mass Index (LVMI)
Percentage of Participants With Major Cardiovascular Events (MACE)
Change From Baseline in Cardiovascular Biomarkers, C-reactive Protein (CRP)
Change From Baseline in Cardiovascular Biomarkers: Troponin I and Collagen Type 1 C-telopeptide (ICTP)
Change From Baseline in Mean 24 Hour Systolic and Diastolic Blood Pressure
Change From Baseline in the Cardiovascular Biomarker, Glycosylated Hemoglobin (HbA1c)
Change From Baseline in the Cardiovascular Biomarker, Myeloperoxidase (MPO)
Change From Baseline in the Cardiovascular Biomarker, N-terminal Pro-brain Natriuretic Peptide Fraction (NT-proBNP)
Change From Baseline in the Cardiovascular Biomarker, Type 1 Procollagen Amino-terminal-propeptide (PINP)
Percentage of Participants With Biopsy-proven Acute Rejection (BPAR), Graft Loss, Death and Lost to Follow up
Pulse Wave Velocity (PWV)
Renal Function as Measured by Creatinine Clearance
Renal Function as Measured by Estimated Glomerular Filtration Rate (eGFR)
Renal Function Measured by Serum Creatinine
6-month Acute Antibody-mediated Rejection Rate (AMR)
6-month Acute Cellular-mediated Rejection Rate (CMR)
6-month Cumulative Rejection Incidence (Either CMR, AMR or Both)
Arthritis Complete Response
Major and Partial Clinical Response
Major Arthritis Response
Area Under the Plasma Concentration-time Curve (AUC(0-6h)) of Mycophenolate Mofetil
Area Under the Plasma Concentration-time Curve (AUC(0-tau)) of Mycophenolate Mofetil
Degree of Fluctuation of the Concentration Levels of Mycophenolate Mofetil Over One Dosing Interval (PTF)
Maximum Observed Plasma Concentration (Cmax) of Mycophenolate Mofetil
Minimum Observed Plasma Concentration (Cmin) of Mycophenolate Mofetil
Plasma Concentrations of Mycophenolate Mofetil in Pre-Administration Samples (Cpd)
Time Corresponding to Occurrence of Cmax (Tmax) of Mycophenolate Mofetil
Summary of Participants With Adverse Events
Number of Non-Relapse Mortalities (NRM)
Number of Participants Surviving Overall
Number of Participants Surviving Progression-free.
Number of Participants Who Graft Rejected.
Number of Participants With Minimal Residual/Recurring Disease (MRD) Post-transplant
Part 1: Number of Participants With Dose Limiting Toxicities (DLT)
Part 2: Number of Participants With Relapsed Disease
Pharmacokinetics (PK) of Clofarabine
Pharmacokinetics (PK) of Clofarabine
Estimated Glomerular Filtration Rate (eGFR)
Number of Participants With Incidence of Adverse Events, Serious Adverse Events, and Tacrolimus-associated Adverse Events
Number of Participants With Incidence of CMV (Viremia, Syndrome and Disease)
Number of Participants With Incidence of Composite Efficacy Failure
Number of Participants With Incidence of New Onset of Diabetes Mellitus
Number of Participants With Incidence of Proteinuria Events
Number of Participants With Incidence Rates of BKV Viremia, BKV Viruria, or BKV Nephropathy
To Compare 2-year Overall Survival of Patients With Lymphoma Undergoing RIC SCT Between Those Receiving Tacrolimus/Sirolimus/Methotrexate and Those Receiving Tacrolimus/Methotrexate or Cyclosporine/Mycophenolate Mofetil
To Compare 2-year Progression-free Survival Between the Two Treatment Arms
To Compare the 2-year Cumulative Incidence of Chronic GVHD Between the Two Treatment Arms.
To Compare the 180-day Cumulative Incidence of Grades II-IV and Grades III-IV Acute GVHD Between the Two Treatment Arms
To Compare the 2-year Cumulative Incidences of Disease Progression and of Non-relapse Mortality Between the Two Treatment Arms
To Compare the 2-year of Overall Survival, Progression-free Survival, Cumulative Incidences of Progression and Non-relapse Mortality Between the Treatment Arms for Each Histology Studied.
AUC0-inf
AUC0-t
Cmax
Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60)
Number of Number of Participants Who Experience Graft Failure, Day 360 (D60)
Number of Number of Participants Who Experience Graft Failure, Day 360 (D90)
Number of Number of Participants With Severe Chronic GVHD, Day 360 (D60)
Number of Number of Participants With Severe Chronic GVHD, Day 360 (D90)
Number of Participants Who Experience Disease Relapse, Days 60-180 (D60)
Number of Participants Who Experience Disease Relapse, Days 90-180 (D90)
Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D90)
Number of Participants Who Experience Graft Failure, Days 60-180 (D60)
Number of Participants Who Experience Graft Failure, Days 90-180 (D90)
Number of Participants Who Experience Non-relapse Mortality, Day 360 (D60)
Number of Participants Who Experience Non-relapse Mortality, Day 360 (D90)
Number of Participants Who Experience Non-relapse Mortality, Days 60-180 (D60)
Number of Participants Who Experience Non-relapse Mortality, Days 90-180 (D90)
Number of Participants Who Experience Relapse, Day 360 (D60)
Number of Participants Who Experience Relapse, Day 360 (D90)
Number of Participants With Chronic GVHD, Days 60-180 (D60)
Number of Participants With Chronic GVHD, Days 90-180 (D90)
Number of Participants With Grades III-IV Acute GVHD, Days 60-180 (D60)
Number of Participants With Grades III-IV Acute GVHD, Days 90-180 (D90)
Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D60 Cohort)
Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D90 Cohort)
Event-free Survival
Number of Participants With Grades II-IV Acute GVHD
Overall Survival
Transplant-related Mortality (TRM)
Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 30 Post-transplantation
Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 60 Post-transplantation
Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 90 Post-transplantation
Disease Free Survival at 12 Months
Disease Free Survival at Day 100
Incidence of Graft Rejection for Patients at Day 100
Non-relapse Mortality at 1 Year After Peripheral Blood Stem Cell Transplantation (PBSCT)
Non-relapse Mortality at Day 100 After Peripheral Blood Stem Cell Transplantation (PBSCT)
Number of Patients Who Experienced Severe Graft-versus-host Disease (GVHD)(Grade 3 or 4)
Overall Survival at 12 Months
Overall Survival at Day 100
Average Level of Protenuria at Week 52
Measured Glomerular Filtration Rate (mGFR), 12 Months After Heart Transplantation
Calculated Glomerular Filtration Rate From Pre-Transplantation to Week 52
Change in Calculated Glomerular Filtration Rate From Pre-transplantation to Week 52
Change in Quality of Life - Euro Quality of Life 5D (EQ-5D)
Change in Quality of Life Assessed by SF-36 (Minnesota Living With Heart Failure Questionnaire ([MLHF)]) From Pre-transplant to Week 52 of Treatment
Lipid Profile at 12 Months
Number of Rejections Leading to Hemodynamic Compromise
Occurrence of Treatment Failures up to 12 Months After Transplant
Progression of Chronic Allograft Vasculopathy (CAV) Based on Incidence of Chronic Allograft Vasculopathy (CAV) From Baseline to Week 52
Progression of Chronic Allograft Vasculopathy (CAV) Based on Maximal Intimal Thickness (MIT) From Baseline to Week 52
Overall Survival
Disease-free Survival
Time to Engraftment
Percent Change in Mean Fluorescence Index (MFI) of Donor Specific Antibodies (DSA) With Increasing Doses of Enteric-Coated Mycophenolate Sodium (EC-MPS)
To Assess the Rate of Rejection, Infection and Renal Function as Mycophenolic Acid Dose is Increased.
Disease Response at One Year Following Hematopoietic Cell Transplantation
Immune Reconstitution Following Hematopoietic Cell Transplantation
Non-relapse Mortality
Number of Participants With Infections
Number of Patients With Grade II-IV Acute Graft-versus-host Disease
Number of Patients With of Chronic Graft-versus-host Disease
Overall Survival
Preliminary Efficacy
Donor Chimerism CD3 at 100 Days Post Transplant
Donor Chimerism CD33 at Day 100 Post Transplant
Graft Failure
Incidence of Chronic GVHD
Incidence of Grades II-IV Acute Graft-versus-Host-Disease (GVHD)
Incidence of Grades III-IV Acute GVHD
Non-relapse Mortality
Engraftment
Overall Survival
Overall Survival
Progression-free Survival
Progression-free Survival
Relapse
Relapse
Change From Baseline in the Euro Quality of Life 5D
Change From Baseline in Visual Analog Scale (VAS)
Measured Glomerular Filtration Rate (mGFR)
Percent of Participants With Incidence of Coronary Allograft Vasculopathy (CAV)
Progression of Cardiac Allograft Vasculopathy (CAV) Recorded by Intravascular Ultrasound (IVUS)
Myocardial Structure and Function
Number of Participants With Beck Depression Inventory (BDI)
Quality of Life by SF-36 Change From Pre-transplantation to 5-7 Year Follow-up
Cumulative Incidence of Relapse With Transplant-related Death as the Competing Risk: Diagnosis Groups Are Compared.
Disease-free Survival at Five Years Post-transplant
Overall Survival at 5 Years Post-Transplant.
Overall Survival Comparing Diagnosis Groups
Immune Reconstruction/CD4+ Count at 1 Year
Immune Reconstruction/CD4+ Count at 3 Months
Immune Reconstruction/CD4+ Count at 6 Months
Incidence of Chronic GVHD at 1 Year
Incidence of Moderate to Severe Grades II to IV Graft Versus Host Disease (GVHD) at 100 Days
Overall Survival at 1 Year
Time to Neutrophil Engraftment
Time to Platelet Engraftment
Event-Specific Survival Comparisons
Overall Kidney Transplant Function at 12, 36, and 60 Months Post-transplant.
Overall Pancreas Transplant Function at 12, 36, and 60 Months Post-transplant.
Clinical Significant Infection, Requiring Treatment, Within 100 Days Post Transplant
Disease Response by 1 Year Post Transplant
Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant
Greater Than 50% CD33+ Donor Chimerisms at 1 Year Post Transplant
Immune Reconstitution by 1 Year Post Transplant
Number of Patients Diagnosed With Acute GVHD
Number of Patients Diagnosed With Chronic GVHD
Number of Patients Diagnosed With Overall Grade 1 or Grade 2 Acute GVHD
Number of Patients Diagnosed With Overall Grade III or Grade IV Acute GVHD
Number of Patients Who Achieve Greater Than 50% Donor T-cell Chimerism
Overall Survival
Graft Failure
Number of Patients Surviving Overall
Number of Patients Surviving Progression-free
Number of Patients With Chronic GVHD
Number of Patients With Grade II-IV Acute GVHD
Number of Patients With Toxicities Related to Bortezomib Maintenance Therapy
Number of Patients With Non-relapse Mortality
Event-Free Survival (EFS)
Number of Patients Who Engrafted
Number of Patients Who Had Infections
Number of Patients With Relapsed/Progressive Disease
Overall Survival
Non-relapse Mortality (NRM)
Number of Patients With Grade II-IV Acute Graft-versus-Host-Disease and/or Chronic Extensive Graft-versus-Host-Disease
Donor Cell Chimerism Following Transplant
Donor Cell Chimerism Following Transplant
Donor Cell Chimerism Following Transplant
Donor Cell Chimerism Following Transplant
Donor Cell Chimerism Following Transplant
Number of Patients Who Died Peri-Transplant
Number of Patients With Donor Derived Engraftment
Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)
Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)
Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)
Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)
Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)
Difference in Renal Function
Incidence of Biopsy-confirmed Acute Rejection by Banff '97 Criteria (Updated 2007) 3, 6 and 12 Months Post Transplant
Incidence of Chronic Alloantibody Rejection or Chronic Allograft Arteriopathy by Banff '97
Number of Patients Requiring Anti-lymphocyte Therapy for Acute Rejection
Severity of Acute Rejection by Banff '97 Criteria
Acute GvHD
Day 100 TRM
OS
PFS
Percentage of Participants Discontinuing Immunosuppressants (MMF) for More Than 14 Consecutive Days or 30 Cumulative Days Within 24 Weeks of Transplantation
Percentage of Participants Lost To Follow Up Within 24 Weeks of Transplantation
Percentage of Participants Requiring Use of Additional Immunosuppressants Not Specified in the Protocol Within 24 Weeks of Transplantation
Percentage of Participants With Graft Loss Within 24 Weeks of Transplantation
Percentage of Participants With Abnormal Serum Creatinine and BUN Values at Baseline and During Treatment
Percentage of Participants With Abnormal Serum Creatinine and BUN Values at Baseline and Normal Values During Treatment
Percentage of Participants With Biopsy Proven Acute Rejection (BPAR) by Week
Percentage of Participants With Normal Serum Creatinine and Blood Urea Nitrogen (BUN) Values At Baseline (BL) And During Treatment
Percentage of Participants With Normal Serum Creatinine and BUN Values at Baseline and Abnormal Values During Treatment
Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Number of Participants Who Discontinued Study Treatment or Withdrew From Study Due to an AE
Full Donor Chimerism (FDC)
Overall Survival (OS)
Event-free Survival (EFS) Rate
Number of Participants That Experience Grade 2 to 4 aGvHD Within 100 Days and 1 Year
Drug Discontinuation Due to Side Effects
Incidence of Biopsy-proven Acute Cellular Rejection During the Study Period
Incidence of Cytomegalovirus Infection or Disease During the Study Period
Incidence of Graft Loss or Death During the Study Period
Number of Participants With Bone Marrow Suppression
Number of Participants With Clinically Significant Decrease in Serum Creatinine From Baseline Through Week 12
Number of Participants With Neurotoxicity
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale)
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale)
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale)
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale)
GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale)
Mean Change From Baseline in Forced Vital Capacity (FVC)
Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
Mean Change in Six Minute Walk Distance
Change in Shortness of Breath (Self-reported)
Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils)
Number of Patients Who Engrafted
Number of Patients Who Had Infections
Number of Patients With HCT Failure.
Number of Patients With Progression-free Survival
Number of Patients With Relapse/Progression
Graft Failure
Graft Rejection
Immune Reconstitution
Incidence of Chronic GVHD
Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)
Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)
Number of Patients With Infections
Number of Patients With Transplant Related Mortality
Proportion of Patients Who Achieve Greater Than 5% Donor T-cell Chimerism
Incidence of Acute Graft-vs-host Disease (aGVHD)
Incidence of Severe Mucositis
Overall Survival
GI Toxicities
Incidence of All Biopsy Proven Acute Rejection.
Incidence of Post Transplant Infections
Renal Function at 12 Months
Patient and Allograft Survival 12 Months
Incidence of Biopsy Confirmed Acute Rejection at 12 Months.
Patient Survival.
Time Post Transplant Corticosteroid Withdrawal
Survival at 1 Year After Transplantation
Engraftment Rates
Incidence of Grades III-IV GVHD
Lymphoid Recovery
Optimal Dose of CD3+ Donor Lymphocytes (T-cells) for Consistent Engraftment Without GVHD
Overall Survival of Participants
Complete Donor Hematopoietic Cell Chimerism
Disease-free Survival (DFS)
Early Graft Loss
Non-relapse Mortality
Overall Survival (OS)
Patients Completing the Intended Therapy in Both Arms
Relapse Rate
Transplant-related Mortality
Transplant-related Mortality
Donor Chimerism at 1 Year
Donor Chimerism at 30 Days
Belatacept Pharmacokinetic (PK) Parameter: Maximum Serum Concentration
Belatacept Pharmacokinetic (PK) Parameter: Time to Achieve the Maximum Plasma Concentration
Belatacept PK Parameter: Amount Excreted in Ascites Fluid Over Days 1 to 14
Belatacept PK Parameter: Area Under the Serum Concentration-time Curve to the End of the Dosing Period (AUCtau)
Belatacept PK Parameter: Minimum Plasma Concentration
Belatacept PK Parameter: Terminal Half-life
Belatacept PK Parameter: Total Body Clearance
Belatacept PK Parameter: Volume of Distribution
Percentage of Participants Surviving With Functional Graft by End of Study (Includes LTE Data)
Percentage of Participants Who Experienced at Least One Episode of Acute Rejection (AR), Graft Loss, or Death by 6 Months Post-transplant
Percentage of Participants Who Experienced at Least One Episode of Acute Rejection, Graft Loss, or Death by 12 Months
Percentage of Participants Who Experienced at Least One Episode of Acute Rejection, Graft Loss, or Death by End of Study (Includes LTE Data)
Belatacept Trough Concentration Before Each Infusion During the LTE
Glycosylated Hemoglobin (HbA1C) Values: 12-month Treatment Phase
Mean Change From Baseline in Calculated GFR During the LTE
Mean Change From Baseline in Calculated GFR, by Modification of Diet in Renal Disease (MDRD) Equation: 12-month Treatment Phase
Mean Change From Baseline in Measured Glomerular Filtration Rate (GFR): 12-month Treatment Phase
Mean Change From Baseline Serum Creatinine at Months 1, 2, 3, 6 and 12
Mean Change in Baseline Values of Cystatin C at 2 and 12 Months
Number of Participants (Who Were HCV Positive at Baseline) With HCV Ribonucleic Acid (RNA) Levels >2.4*10^6 U/mL and >4.7*10^6 U/mL: 12-month Treatment Phase
Number of Participants (Who Were HCV Positive at Baseline) With HCV RNA Levels >2.4 * 10^6 U/mL and >4.7 * 10^6 U/mL During the LTE
Number of Participants at Risk of First Acute Rejection as Determined by Kaplan-Meier Method by 12 Months
Number of Participants Having Acute Rejection by Rejection Activity Index During the LTE
Number of Participants Having Acute Rejections During the LTE
Number of Participants Having Acute Rejections: 12-month Treatment Phase
Number of Participants Who Had Acute Rejection by Banff Grade by 12 Months
Number of Participants Who Had Adverse Events (AEs), Death, Serious AEs (SAEs) or Were Discontinued Due to AEs (Includes Long Term Extension [LTE] Data)
Number of Participants Who Had Adverse Events of Special Interest During 12-month Treatment Phase
Number of Participants Who Had AEs of Special Interest During the LTE
Number of Participants Who Had AEs, Death, SAEs or Were Discontinued Due to AEs: 12-month Treatment Phase
Number of Participants Who Received Anti-hypertensive Therapy at Month 12
Number of Participants With Acute Rejections by Rejection Activity Index (RAI) by 12 Months
Number of Participants With Central Biopsy Proven Acute Rejection by Treatment Type by 12 Months
Number of Participants With Central Biopsy Proven Acute Rejection by Treatment Type During the LTE
Number of Participants With Marked Electrolytes, Protein and Metabolic Test Abnormalities During the LTE
Number of Participants With Marked Electrolytes, Protein and Metabolic Test Abnormalities: 12-month Treatment Phase
Number of Participants With Marked Hematology Abnormalities During the LTE
Number of Participants With Marked Hematology Abnormalities: 12-month Treatment Phase
Number of Participants With Marked Liver and Kidney Function Abnormalities During the LTE
Number of Participants With Marked Liver and Kidney Function Abnormalities: 12-month Treatment Phase
Percentage of Participants (Who Were Hepatitis C Virus [HCV] Positive at Baseline) With HCV Recurrence (Assessed by Central Pathologist) by 12 Months
Percentage of Participants (Who Were Hepatitis C Virus [HCV] Positive at Baseline) With HCV Recurrence (Assessed by Central Pathologist) During the LTE
Percentage of Participants Meeting the Definition of Dyslipidemia, Hypertriglyceridemia or Hypercholesterolemia at Any Given Time: 12-month Treatment Phase
Percentage of Participants Surviving With Functional Graft: 12-month Treatment Phase
Percentage of Participants Who Develop Dyslipidemia, Hypertriglyceridemia and Hypercholesterolemia After Randomization and Transplantation: 12-month Treatment Phase
Percentage of Participants Who Developed Hypertension in 12-month Treatment Phase
Percentage of Participants Who Have Hypertension at Any Given Time During the 12-month Treatment Phase
Percentage of Participants With New Onset Diabetes Mellitus (NODM): 12-month Treatment Phase
Summary Statistics for Diastolic Blood Pressure: 12-month Treatment Phase
Summary Statistics for Lipid Parameters - Serum Triglyceride: 12-month Treatment Phase
Summary Statistics for Lipid Parameters- Serum Cholesterol: 12-month Treatment Phase
Summary Statistics for Lipid Parameters- Serum Low Density Lipoprotein Cholesterol (LDL): 12-month Treatment Phase
Summary Statistics for Lipid Parameters-Serum Total Non-High Density Lipoprotein (Non-HDL) Cholesterol: 12-month Treatment Phase
Summary Statistics for Lipid Parameters; Serum HDL Cholesterol: 12-month Treatment Phase
Summary Statistics for Mean Arterial Pressure: 12-month Treatment Phase
Summary Statistics for Systolic Blood Pressure: 12-month Treatment Phase
Number of Participants Experiencing Graft Failure
Number of Participants Experiencing Overall Survival
Number of Participants With Acute Graft-Versus-Host Disease (GVHD)
Number of Participants With Chronic Graft-Versus-Host Disease (GVHD)
Number of Participants With Transplant Related Deaths
EFS
Event-free Survival (EFS)
Incidence of Graft Rejection
Overall Survival
The Percent of Participants With Definite and Probable Viral, Fungal, and Bacterial Infections
Treatment-related Mortality
Incidence and Severity of Graft-versus-host Disease (GVHD)
Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36)
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ)
Skin Score
Stimulation of Growth After 12 Months (Delta Z-score)
Count of Participants With Acute Cellular Rejection Grade Equal to or Greater Than IA, by the Banff 2007 Criteria
Count of Participants With Antibody Mediated Rejection
Count of Participants With Biopsy Proven Acute Rejection at Any Time Post-Transplant
Count of Participants With CAN/IFTA Grade I, II or III at Any Time Post-transplant
Count of Participants With de Novo Anti-donor HLA Antibodies at Wk 52
Count of Participants With Delayed Graft Function Post-Transplant
Count of Participants With EBV Infection as Reported on the Case Report Form as Adverse Events
Count of Participants With Infections Requiring Hospitalization or Systemic Therapy Reported as Serious Adverse Events
Count of Participants With Rejection
Count of Participants With Treated Diabetes Between Day 14 and Wk 52
Count of Participants With Use of Anti-hypertensive Medications at Wk 52
Mean Glomerular Filtration Rate (GFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52
Number of Events of Death or Graft Loss
Count of Participants by Chronic Kidney Disease (CKD) Stage Post-Transplant
Count of Participants by Severity of First Acute Cellular Rejection by Wk 52
Count of Participants With BKV and CMV Viremia (Local Center Monitoring) Reported as Adverse Events
Count of Participants With CKD Stage 4 or 5
Count of Participants With Either New Onset Diabetes After Transplant (NODAT) or Impaired Fasting Glucose (IFG) at Wk 52 Based on Criteria Specified by the ADA and WHO
Count of Participants With Estimated Glomerular Filtration Rate (GFR) < 60 mL/Min/1.73 m^2 by CKD EPI
Count of Participants With Fever > 39 Degrees Celsius and Blood Pressure < 90mm Hg Within 24 Hours of Onset of Transplant Procedure
Count of Participants With Use of Lipid Lowering Medications at Baseline and Wks 24, 52, 104 and 156
Fasting Lipid Profile (Total Cholesterol, Non-HDL Cholesterol, LDL, HDL, and Triglyceride) at Baseline and Wks 24, 52, 104 and 156
HbA1c Measured at Days 28 & 84, and Weeks 24, 36, 52, 72, 104 and 156
HbA1c Measured at Days 28 & 84, and Weeks 24, 36, 52, 72, 104 and 156
Mean Calculated eGFR Using MDRD 4 Variable Model
Number of All Adverse Events (AEs) and Serious Adverse Events (SAEs)
Standardized Blood Pressure Measurement at Wk 52
The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine
Total Daily Prescribed Pill Number at Days 28 and 84, and Wks 24, 36, 52, 72, 104 and 156
Total Daily Prescribed Pill Number at Days 28 and 84, and Wks 24, 36, 52, 72, 104 and 156
Type of Treatment of Rejection
Change of Lung Function Measured by Forced Vital Capacity (FVC) Between Baseline and 24 Weeks
Change of Skin Fibrosis Measured by the Rodnan Skin Score Between Baseline and 24 Weeks
Number of Participants With Serious Adverse Events
Number of Participants With 100 Day Transplant-related Mortality (TRM)
Number of Patients That Engrafted Blood Counts by 30 Days After Transplant
Acute Graft Versus Host Disease Among Patients Who Received Early Transplant
Demonstrate the Feasibility of Collecting Resource Utilization Data for Trial Participants
Event-free Survival (EFS) Among Patients Who Received Early Transplant
Event-free Survival (EFS) Among Patients Who Received Early Transplant
Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - AGE
Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - TREATMENT RELATED MORTALITY
Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant
Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant
Overall Survival (OS) Among Patients Who Received Early Transplant.
Overall Survival (OS) Among Patients Who Received Early Transplant.
Relapse-free Survival (RFS) Among Patients Who Received Early Transplant
Relapse-free Survival (RFS) Among Patients Who Received Early Transplant
Treatment Related Mortality Among Patients Who Received Early Transplant vs Patients Who Did Not Receive Early Transplant
Demonstrate the Feasibility of Collecting Patient-reported Outcomes for Trial Participants
Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - GENDER
Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Enrollment and Incidence of Early Transplant
Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Relapsed-free Survival 6 Months After Transplant
Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 28
Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 84
# of Patients Developing Denovo Donor Specific Antibody (DSA) Post-transplant
# Patients Experiencing a First Biopsy-proven ACR With Severity Banff > or = 2a Grade
# Patients Experiencing a Graft Loss But Not Including Patients Who Died With Functioning Graft (Death-censored Graft Loss)
# Patients With Composite Endpoint of Either Experiencing Death, Graft Loss, or eGFR < 45ml/Min at 24 Months
# Patients With Composite Endpoint of Experiencing Either Death, Graft Loss, or eGFR < 45ml/Min
Biopsy Proven Acute Antibody Mediated Rejection
Biopsy Proven Acute Cellular Rejection
Biopsy Proven Acute Rejection
Biopsy Proven Mixed Acute Rejection
Delayed Graft Function
Discontinuation of Mycophenolate
Discontinuation of Study Treatment (Belatacept or Tacrolimus)
eGFR (MRDRD) < 45 ml/Min/1.73m2
Leukopenia (WBC < 2000/mm3)
Mean eGFR (MDRD) (ml/Min/1.73m2)
New Onset Diabetes After Transplantation (NODAT)
Patient Death
Proteinuria UPC Ratio > 0.8
Requirement of T-cell Depleting Therapy for Biopsy Proven Acute Rejection (BPAR)
Steroid Therapy
Time to First BPAR
Duration of Prednisone Maintenance Dosing
Percentage of Patients Achieving Responder Status at Week 52
Time to Initial Response
Time to Sustained Response
Number of Non-relapse Participant Mortalities
Number of Participants Who Experienced Chronic Extensive GVHD
Number of Participants Who Experienced Graft Failure
Number of Participants With Dose Limiting Toxicities
Number of Participants With Grades III-IV Acute GVHD
Number of Participants With Relapsed Disease
Number of Subjects Surviving Post-transplant.
Immune Reconstitution Efficacy - Naive CD4 T Cells
Immune Reconstitution Efficacy - Naive CD8 T Cells
Immune Reconstitution Efficacy - Response to Mitogens
Immune Reconstitution Efficacy - Total CD3 T Cells
Immune Reconstitution Efficacy - Total CD4 T Cells
Immune Reconstitution Efficacy - Total CD8 T Cells
Survival at 1 Year Post-CTTI
Survival at 2 Years Post-CTTI
Adjusted Mean Change From Baseline in Disease Activity as Measured by BILAG 2004 Over Time During Year 1 of the Double-blind Period
Adjusted Mean Change From Baseline in UPCR at Day 365 of the Double-blind Period in Overall Population
Adjusted Mean Change From Baseline in Urine Protein/Creatinine Ratio (UPCR) at Day 365 of the Double-blind Period in Nephrotic Participants
AUC (TAU): Area Under the Serum Concentration Time Curve Over a Dosing Interval
Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (Percentage of Blood)
Percentage of Nephrotic Participants in Complete Renal Response of Lupus Glomerulonephritis at Day 365 of the Double-blind Period
Percentage of Nephrotic Participants in Complete Renal Response of Lupus Glomerulonephritis at Day 729 of the Double-blind Period
Percentage of Participants in Complete Renal Response (CR) of Lupus Glomerulonephritis at Day 365 of the Double-blind Period
Percentage of Participants in Overall Population in Complete Renal Response of Lupus Glomerulonephritis at Day 729 of the Double-blind Period
Adjusted Mean Change From Baseline in Disease Activity as Measured by BILAG 2004 Over Time During the Double-blind Period
Adjusted Mean Change From Baseline in eGFR Over Time
Adjusted Mean Change From Baseline in UPCR Over Time
Cmax: Maximum Observed Serum Concentration Following Participants Receiving Active Abatacept IV
Cmin (ug/mL): Trough Level Serum Concentration of Abatacept Prior to the Administration of the IV Infusion
Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (g/L)
Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (mmol/L)
Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (U/L)
Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (Umol/L)
Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (x10^9 Cells/L)
Median Percent Change From Baseline in UPCR Over Time
Median Time to Complete Renal Response During the Double-blind Period in All Participants
Median Time to Complete Renal Response During the Double-blind Period in Nephrotic Participants
Median Time to First Sustained Change to No Response During the Double-blind Period
Median Time to First Treatment Failure and Overall Treatment Failure During the Double-blind Period
Median Time to Partial Renal Response During the Double-blind Period in All Participants
Median Time to Partial Renal Response During the Double-blind Period in Nephrotic Participants
Number of Participants With Abatacept Induced Antibody Response Over Time in the Double-blind Period
Number of Participants With Any Adverse Events (AEs) During Year 1 of the Double-blind Period
Number of Participants With Any Adverse Events (AEs) During Year 2 of the Double-blind Period and Long-term Extension
Number of Participants With Marked Electrolyte Laboratory Abnormalities During Year 1 of the Double Blind Period
Number of Participants With Marked Electrolyte Laboratory Abnormalities in the Double Blind Period
Number of Participants With Marked Hematology Laboratory Abnormalities During Year 1 of the Double Blind Period
Number of Participants With Marked Hematology Laboratory Abnormalities in the Double Blind Period
Number of Participants With Marked Liver and Kidney Function Laboratory Abnormalities During Year 1 of the Double Blind Period
Number of Participants With Marked Liver and Kidney Function Laboratory Abnormalities in the Double Blind Period
Number of Participants With Marked Urinalysis Laboratory Abnormalities During Year 1 of the Double Blind Period
Number of Participants With Marked Urinalysis Laboratory Abnormalities in the Double Blind Period
Number of Participants With Other Marked Chemistry Laboratory Abnormalities During Year 1 of the Double Blind Period
Number of Participants With Other Marked Chemistry Laboratory Abnormalities in the Double Blind Period
Number of Participants With Sustained Change From Higher Level of Response to no Response During the Double-blind Period
Percentage of Participants in Treatment Failure Over Time During the Double-blind Period
Percentage of Participants With Ranked Outcome of Complete Renal Response, Partial Renal Response (PR), and No Renal Response (NR) During the Double-blind Period
Summary Statistics for Diastolic Blood Pressure
Summary Statistics for Heart Rate
Summary Statistics for Systolic Blood Pressure
AUC0-inf - Area Under the Concentration-time Curve From Time Zero to Infinity (Extrapolated)
AUC0-t - Area Under the Concentration-time Curve From Time Zero to Time of Last Non-zero Concentration (Per Participant)
Cmax - Maximum Observed Concentration
Number of Non-Relapse Mortalities
Number of of Participants Surviving Overall
Number of Participants With Relapse/Progression
Number of Patients With Chronic Extensive GVHD
Number of Patients With Grades II-IV Acute GVHD
Number of Patients With Grades III-IV Acute GVHD
Number of Participants With Disease-free Survival (DFS)
Number of Participants With Relapse of Disease
Percentage of Participants With at Least One Adverse Event (AE)
Mean Number of Blood Transfusions Per Visit
Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
Donor (Allogeneic) Hematopoietic Engraftment
Transplant-Related Mortality
Event-free Survival
Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD)
Number of Transplant Recipients With Malignant Relapse
Number of Transplant Recipients With Successful Engraftment
Number of Transplant Recipients With Transplant-related Mortality (TRM)
Overall Survival
Disease-Free Survival (DFS)
Event-Free Survival (EFS)
Incidence of Malignant Relapse
Number of Participants With Transplant Related Mortality (TRM)
One-year Survival (OS)
Incidence and Severity of Acute Graft Versus Host Disease (GVHD)
Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)
Neutrophil Engraftment - The Days Till ANC Recovery
Two-year Overall Survival
Change in EMT Score
Change in Urine Protein/Creatinine Ratio (Without Imputation)
Change From Baseline (M3) in Estimated Glomerular Filtration Rate (eGFR)
Change in Estimated Glomerular Filtration Rate (eGFR) at M12 From Baseline (M3) - ANCOVA Model
Change in Interstitial Fibrosis/Tabular Atrophy (IF/TA) Grade
Change in Percentage of Interstitial Fibrosis (IF) by Numerical Quantification
Incidence (Number) of BPAR
Incidence (Number) of Participants With Graft Losses
Incidence (Number) of Subclinical Rejections and Borderline Lesions
Interstitial Fibrosis/Tabular Atrophy (IF/TA)
Number of Participants With Epithelial-mesenchymal Transition (EMT) Score
Number of Participants With Epithelial-mesenchymal Transition (EMT) Status
Number of Participants With Progression of Renal Fibrosis Using Numerical Quantification
Number of Participants With Progression of Renal Graft Fibrosis (Primary Comparison - ITT Population
Risk Factors of IF/TA Progression
Severity of BPAR
Treatment Failures
Type of Biopsy Proven Acute Rejection (BPAR)
Allograft Rejection Rates at 30 Days
Estimated Glomerular Filtration Rate (eGFR) at 12 Months Post-surgery
Graft Survival
Patient Survival
Number of Participants That Experience One Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplant (HSCT)
Number of Participants With Grade II-III and III-IV Acute GVHD and Chronic GVHD
Number of Participants With Regimen-related Toxicity and Infections
Number of Participants With Relapse/Progression
Number of Participants With Treatment-related Mortality
Number of Patients Achieving Complete Response and Partial Response (Overall Response Rate)
Overall Survival
Donor and Host Polymorphisms of the FCgammaRIIIa Receptor and CD32 and Their Impact on Disease Response and Relapse
Rituxan Concentration
Disease Progression/Relapse
Incidence of Grade III-IV Acute GVHD
Incidence of Graft Rejection
Incidence of High-dose Corticosteroid Utilization.
Incidence of Infection
Incidence of Non-relapse Mortality
Change From Baseline in Glomerular Filtration Rate (GFR) at 12 Months Posttransplant
Change From Baseline in Glomerular Filtration Rate (GFR) at 24 Months Posttransplant
Change From Baseline in Glomerular Filtration Rate (GFR) at 6 Months Posttransplant
Change From Baseline in Creatinine Clearance
Number of Donors Discontinuing Atorvastatin Due to Toxicity
Number of Non-relapse Mortalities
Number of Patients Requiring Secondary Systemic Immunosuppressive Therapy
Number of Patients Surviving Overall
Number of Patients With Chronic Extensive GVHD
Number of Patients With Grade III-IV Acute Graft-versus-host Disease (GVHD) Post-transplant
Number of Patients With Grades II-IV Acute Graft-versus-host-disease (GVHD)
Number of Patients With Recurrent or Progressive Malignancy
Bronchiolitis Obliterans
Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy
Death
Death or Recurrent Malignancy
Definitive Absence of Efficacy Success
End of Systemic Treatment
Non-relapse Mortality
Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy
Recurrent Malignancy
Withdrawal of Prednisone
Incidence of Chronic Extensive GVHD
Incidence of Grade III/IV GVHD
Incidences of Grades II-IV Acute GVHD
Incidences of Graft Rejection
Overall Survival
Rate and Duration of Steroid Use for the Treatment of Chronic GVHD
Rates of Disease Progression
Rates of Relapse-related Mortality
Number of Non-Relapse Mortalities
Number of Participants Surviving Overall
Number of Participants Surviving Without Progression
Number of Participants Utilizing High-Dose Corticosteroids
Number of Participants With Grades II-IV Acute GVHD
Engraftment
Incidence of Acute GVHD (Grades III-IV)
Incidence of Chronic (Extensive) GVHD
Non-relapse Mortality
PFS
Relapse Rate
Response Rate
OS
Disease-free Survival-incidence of Survival Without Relapse
Incidence of Rejection
Incidence of Relapse
Nonrelapse Mortality (NRM)-Incidence of Nonrelapse Death
Overall Survival
Incidence of Acute and Chronic GVHD
Incidence of Grade IV Acute GVHD
Incidence of Infections
Incidence of Relapse/Progression
Percentage Patients With an Increase of at Least 10 Percentage Points in Donor T-cell Chimerism
Survival
Incidence of GVHD
Mean International Society for Heart and Lung Transplantation Biopsy Score Over the First 6 Months Post-transplantation
Mean ISHLT Biopsy Score Over First Year Post-transplant
Number of Patients With Allograft Vasculopathy (CAD) at One Year Post Transplant
Number of Patients With Cytomegalovirus (CMV) at One Year Post-transplant
Percent of Patients Alive at One Year Post-transplant
Best Overall Response (BOR) at Cycle 7 Day 1
BOR During Cross-over Treatment With Ruxolitinib
Duration of Response Through Study Completion
Efficacy of Ruxolitinib Versus Investigator's Choice Best Available Therapy (BAT) in Participants With Moderate or Severe Steroid Refractory Chronic Graft Versus Host Disease (SR-cGvHD) Assessed by Overall Response Rate (ORR) at the Cycle 7 Day 1 Visit
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
ORR at the End of Cycle 3
Overall Survival (OS)
Rate of Failure-free Survival (FFS)
Rate of FFS at Study Completion
Rate of Participants With Clinically Relevant Improvement of the Modified Lee cGvHD Symptom Scale Score
Utilization of Medical Resources
AUCinf of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
AUClast of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Change From Baseline in EQ-5D-5L
Change From Baseline in Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT)
CL/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Cmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Cumulative Incidence of Malignancy Relapse/Recurrence (MR)
Cumulative Incidence of Non-relapse Mortality (NRM)
Percentage of Participants Successfully Tapered Off of All Corticosteroids
Percentage of Participants With a ≥ 50% Reduction in Daily Corticosteroid Dose
t1/2 of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Tmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Vz/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
Area Under the Plasma Concentration Time Curve From 0-12 Hours for Mycophenolic Acid and Mycophenolic Acid Glucuronide Phenolic Glucuronide of Mycophenolic Acid
Area Under the Plasma Concentration-Time Curve From 0 to 12 Hours of Mycophenolic Acid Normalized for Dose And for Body Surface Area
Maximum Plasma Concentration for Mycophenolic Acid and Mycophenolic Acid Glucuronide
Number of Participants With Adverse Events and Serious Adverse Events
Plasma Concentration of Mycophenolic Acid and Its Metabolite Mycophenolic Acid Glucuronide at Each Time Point
Time to Maximum Plasma Concentration for Mycophenolic Acid and Mycophenolic Acid Glucuronide
Acute Graft Versus Host Disease (aGVHD) of Grades 2-4 According to the Consensus Grading
Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS) at 1 Year
Overall Survival (OS) at 1 Year
Measured Glomerular Filtration Rate
Measured Glomerular Filtration Rate
Time to First Malignancy
Time to Treatment Failure
Calculated Glomerular Filtration Rate
Health-related Quality of Life (QoL) as Measured by EuroQoL EQ-5D
Lipid Profile for Apolipoprotein
Lipid Profile for HDL-C, LDL-C,Total Cholesterol, and Triglycerides
Number of Antihypertensive Drugs Taken
Number of Lipid-lowering Drugs Taken
Percentage of Participants on Antihypertensive Drugs
Percentage of Participants on Lipid-lowering Drugs
Percentage of Participants Who Developed CAN (Chronic Allograft Nephropathy)
Percentage of Participants Who Had Donor Specific Antibodies (DSA)
Percentage of Participants With Biopsy Proven Acute Rejection (BPAR)
Percentage of Participants With Graft Loss or Death
Percentage of Participants With Treatment Failures
Progression of Measured Glomerular Filtration Rate
Proteinuria (Measured as Urine Albumin/Creatinine Ratio (mg/mmol))
Number of Infectious Complications
Number of Participants With Donor-specific Antibody Formation
Time to Initiation of Dialysis
Glomerular Filtration Rate (GFR)
Glomerular Filtration Rate (GFR)
Number of Participants With Anti-human Leukocyte Antigen (HLA) Alloantibodies
Delta Alanine Aminotransferase
Delta Alkaline Phosphatase
Delta Bilirubin
Delta Cholesterol Fasting Level
Delta Hemoglobin
Delta Hepatitis C Viral Load
Delta Platelet Count
Delta Tacrolimus Trough Level
Delta Triglyceride Fasting Level
Final Hepatitis C Viral Load
Sirolimus Trough Level
Relapse Free Survival
Transplant-related Mortality
Transplant-related Mortality
Leukemia-free Survival
Overall Survival
Number of Participants Who Developed Acute Graft Versus Host Disease
Number of Participants Who Survived 100 Days or Longer
Graft Failure Rate
Hematologic and Non-hematologic Toxicities as Measured by NCI Common Toxicity Criteria for Adverse Events, v 3.0 Weekly Until 1 Year After Transplantation
Progression-free Survival
Relapse Rate
Transplant-related Mortality
Number of Participants Experiencing Progression-free Survival
Number of Participants Experiencing Progression-free Survival at 2 Years
Number of Participants Experiencing Relapse (Incidence of Relapse)
Number of Participants Experiencing Relapse (Incidence of Relapse) at 2 Years
Number of Participants Who Were Alive at 1 Year Post Transplant
Number of Participants Who Were Alive at 2 Years Post Transplant
Number of Participants Who Were Dead at 6 Months After Study Completion
Number of Participants With Acute Graft-versus-host Disease (GVHD)
Number of Participants With Chronic Graft-Versus-Host Disease
Number of Participants With Neutrophil Engraftment
Number of Participants With Platelet Engraftment
Percentage of Donor Chimerism at 100 Days
Percentage of Donor Chimerism at 180 Days
Percentage of Donor Chimerism at 21 Days
Percentage of Donor Chimerism at 365 Days
Modification of Diet in Renal Disease (MDRD) Estimation of Glomerular Filtration Rate (GFR)
Modification of Diet in Renal Disease (MDRD) Estimation of Glomerular Filtration Rate (GFR)
Change in Best Spectacle-corrected Visual Acuity (BSCVA)
Number of Participants Achieving Treatment Success
Time to Control of Inflammation
Number of Eyes With Resolution of Macular Edema
Absolute Neutrophil Count (ANC) Engraftment
Hematopoietic Toxicity
Overall Survival
Platelet Engraftment
Progression-free Survival
Response Rates
Treatment-related Mortality
BANFF Grades of First AMR.
Change in eGFR Between 3 Months and 24 Months as Measured by CKD-EPI
Change in eGFR Between 3 Months and 24 Months as Measured by MDRD
Change in eGFR Between 6 Months and 24 Months as Measured by CKD-EPI
Change in eGFR Between 6 Months and 24 Months as Measured by MDRD
Change in eGFR Between Post-transplant Nadir and 24 Months as Measured by CKD-EPI
Change in eGFR Between Post-transplant Nadir and 24 Months as Measured by MDRD
Creatinine Reduction Ratio (CRR), Defined as the First Creatinine on Day 2 Divided by he First Creatinine After Surgery
Creatinine Reduction Ratio (CRR), Defined as the First Creatinine on Day 5 Divided by the First Creatinine After Surgery.
Days From Transplantation Until Event (ACR, AMR, or Hospitalization for Infection and/or Malignancy)
Duration of Delayed Graft Function (DGF), Defined as Time From Transplantation to the Last Required Dialysis Treatment.
eGFR Values as Measured by CKD-EPI
eGFR Values as Measured by MDRD
Number of Dialysis Sessions.
Percent of Participants That Required at Least One Dialysis Treatment.
Percent of Participants With Any Infection Requiring Hospitalization or Resulting in Death.
Percent of Participants With BANFF Chronicity Scores > or Equal 2 on the 24 Month Biopsy.
Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection (AMR)
Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection (AMR).
Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection AMR or Suspicious for AMR
Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection AMR or Suspicious for AMR.
Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR)
Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR) or Borderline Rejection
Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR) or Borderline Rejection.
Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR).
Percent of Participants With BK Viremia That Require a Change in Immunosuppression or Anti-viral Treatment as Per Standard of Care at the Site.
Percent of Participants With CMV Viremia That Require a Change in Immunosuppression or Anti-viral Treatment as Per Standard of Care at the Site
Percent of Participants With de Novo DSA.
Percent of Participants With Death or Graft Failure.
Percent of Participants With Impaired Wound Healing Manifested by Wound Dehiscence, Wound Infection, or Hernia at the Site of the Transplant Incision
Percent of Participants With Locally Treated Rejection, Defined as Treatment Administered for Rejection Based on Clinical Signs or Biopsy Findings.
Percent of Participants With Locally Treated Rejection, Defined as Treatment Administered for Rejection Based on Clinical Signs or Biopsy Findings.
Percent of Participants With Malignancy.
Percent of Participants With Mycobacterial or Fungal Infections
Percent of Participants With Only Graft Failure.
Percent of Participants With Primary Non-Function (PNF), Defined as Dialysis-dependency for More Than 3 Months.
The Difference Between the Mean eGFR (Modified MDRD) in the Experimental vs. Control Groups.
The Percent of Participants Who Need Dialysis After Week 1.
The Percent of Participants Whose Day 2 Serum CRR Was Less Than 30%.
The Percent of Participants Whose Day 5 Serum CRR Was Less Than 70%.
The Percent of Participants With a Serum Creatinine of More Than 3 mg/dL.
Change From Baseline (Immediately After Surgery) in Serum Creatinine.
eGFR Values as Measured by CKD-EPI
eGFR Values as Measured by MDRD
Acute Rejection
Patient Survival
Renal Allograft Survival
Change in Glomerular Filtration Rate (GFR)
Change in T Cell & B Cell Generation
Percent of Participants With Adverse Events (AEs) Attributable to the Donor Alloantigen Reactive Tregs (darTregs) Infusion
Percent of Participants With Grade 3 or Higher Infectious Adverse Event(s)
Percent of Participants With Grade 3 or Higher Wound Complication(s) Adverse Event(s)
Percent of Participants With Biopsy-Proven Acute and/or Chronic Rejection
Percent of Participants With Grade 2 or Higher Hematologic Adverse Events (AEs) of Anemia, Neutropenia, and/or Thrombocytopenia
Duration in Days of Adverse Events (AEs)- Including Infection, Wound Complications, Post-transplant Diabetes, Hemorrhagic Cystitis and Malignancy
Duration in Days of Graft-versus-Host Disease in Transplanted Participants
Number of Days From Neutrophil Nadir to Absolute Neutrophil Recovery
Number of Days From Transplant to Platelet Count Recovery
Number of Days Post-Transplant to the First Episode of Acute Rejection Requiring Treatment
Number of Participants Experiencing an Incidence of Graft-versus-Host Disease Post-Transplant
Number of Participants Free From Return to Immunosuppression for the Duration of the Study
Number of Transplanted Participants Who Developed Donor- Specific Antibody After Initiation of Immunosuppression Withdrawal
Number of Transplanted Participants Who Developed Donor-Specific Antibody During Study Participation
Number of Transplanted Participants Who Died
Number of Transplanted Participants Who Remained Off Immunosuppression for at Least 52 Weeks, Including Those in Whom the 52 Week Biopsy Was Not Performed
Number of Transplanted Participants With Acute Renal Allograft Rejection
Number of Transplanted Participants With Chronic T Cell-Mediated or Antibody-Mediated Rejection
Number of Transplanted Participants With Engraftment Syndrome
Percent of Participants Who Achieved Operational Tolerance
Histological Severity of Biopsies Demonstrating Acute Rejection as Defined by Banff 2007 Classification Renal Allograft Pathology
Number of Adverse Events (AEs) by Severity- Including Infection, Wound Complications, Post-Transplant Diabetes, Hemorrhagic Cystitis and Malignancy
Number of Adverse Events (AEs)- Including Infection, Wound Complications, Post-transplant Diabetes, Hemorrhagic Cystitis and Malignancy
Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach
Count of Participants That Experienced Death as a Result of Graft-versus-host Disease (GVHD)
Number of Participants With Successful Engraftment
Median Pace of T Cell Immune Recovery at 28 Days Post Hematopoietic Stem Cell Transplantation (HSCT)
Median Pace of T Cell Immune Recovery at 90 Days Post Hematopoietic Stem Cell Transplantation (HSCT)
Cumulative Oral Corticosteroid Dose
Percentage of Participants (Excluding Telemedicine [TM] Participants) Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score
Percentage of Participants With Anti-Drug Antibodies (ADA)
Time to Initial Sustained Complete Remission
Time to Protocol Defined Disease Flare
Total Number of Protocol Defined Disease Flares
Change in Health-Related Quality of Life (HRQoL), as Measured by the Dermatology Life Quality Index (DLQI) Score
Percentage of Participants With Adverse Events, Serious Adverse Events, and Corticosteroid-Related Adverse Events
Percentage of Participants With Immunoglobulin (Ig) Levels Below Lower Limit of Normal (LLN)
Recipient and Donor Genotyping for Selected Variants of CYP3A5, ABCB1 (MDR1), and CYP4A Genes
Renal Function as Measured by 24 Hour Urine Creatinine Clearance
Renal Function as Measured by Cockcroft Gault Creatinine Clearance
Renal Function as Measured by Iothalamate Clearance
Renal Function as Measured by Modification of Diet in Renal Disease (MDRD) Estimated Glomerular Filtration Rate (eGFR)
Renal Function as Measured by Serum Creatinine Level
Anti-CFZ533 Antibodies - Part I
Mean AUClast Pharmacokinetic Parameter - Part I
Mean Cmax Pharmacokinetic Parameter- Part I
Mean Tmax Pharmacokinetic Parameter - Part I
Anti-CFZ533 Antibodies - Part II
Anti-CFZ533 Antibodies - Part II
CFZ533 Plasma PK Concentrations - Part II
Efficacy as Defined by the Frequency and Severity (Banff Classification) of Treated Biopsy Proven Acute Rejection (tBPAR) Adjudicated Data - Part II
eGFR - Part II
Free CD40 and Total CD40 on B Cells - Part II
Total sCD40 Plasma Concentrations - Part II
Total Soluble CD40 and Total Soluble CD154 Concentrations in Plasma - Part 1
GVHD-free Relapse-free Survival (GRFS)
Number of Participants Who Experience Chronic GVHD
Number of Participants Who Experience Grades II-IV Acute GVHD
Number of Participants Who Experience Grades III-IV Acute GVHD
Number of Participants Who Experience Primary Graft Failure
Number of Participants Who Experience Secondary Graft Failure
Number of Participants With Full Donor Chimerism
Overall Survival and Engraftment at One Year
Overall Survival at One Year
Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts
Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts
Transplant-related Mortality
100-day Overall Survival
Progression-free Survival
Proportion of Participants With Successful Engraftment
Complete Response Rate
Number of Patients Who Developed Disease Progression After Achieving Complete Response
Overall Survival
Proportion of Graft Versus Host Disease
Time to Engraftment for Neutrophil
Time to Engraftment for Platelet
Two-Year Disease-free Survival of Study Participants Who Completed the Study Regimen
Chronic Graft vs Host Disease (GvHD)
Disease Progression (TDP)
Event-free Survival (EFS
Full-dose Donor Chimerism (FDC)
Incidence of Acute Graft vs Host Disease (GvHD)
LOWSKY Grade 3 or Higher Toxicities
Non-relapse Mortality (NRM)
Overall Survival (OS)
Incidence of Chronic GVHD
Incidence of Grade II-IV Acute Graft vs. Host Disease (GVHD)
Overall Survival (OS)
Progression Free Survival (PFS)
Percent of Participants Experiencing Acute Allograft Rejection
Percent of Participants Who Experienced Kidney Transplant Graft Loss
Self-reported Medication Adherence From Baseline to 6 Months.
Subject Specific Change on Medication Side Effect Scale
Dose Modifications
Dose-normalized Trough
Number of Days to Reach Therapeutic Trough Goal
Total Daily Dose
Weight-Based Dose Requirement
Donor Cell Engraftment
Incidence and Severity of Acute and Chronic Graft-vs-host Disease
Incidence of Disease Progression/Relapse
Non-relapse Mortality
Overall Survival
Progression-free Survival
Change From Baseline in Anti-Double Stranded Deoxyribonucleic Acid (Anti-dsDNA) Antibody Levels at Week 52
Change From Baseline in C4 Levels at Week 52
Change From Baseline in Complement Component 3 (C3) Levels at Week 52
Percentage of Participants Who Achieve Protocol Defined Complete Renal Response (CRR) at Week 52
Percentage of Participants Who Achieve Protocol Defined CRR at Week 24
Percentage of Participants Who Achieve Protocol Defined Modified CRR (mCRR1) at Week 52
Percentage of Participants Who Achieve Protocol Defined Overall Response (OR) at Week 52
Percentage of Participants Who Achieve Protocol Defined Partial Renal Response (PRR) at Week 52
Percentage of Participants Who Achieve Protocol Defined Second mCRR (mCRR2) at Week 52
Percentage of Participants Who Achieve Protocol Defined Third mCRR (mCRR3) at Week 52
Percentage of Participants With Anti-Drug Antibody (ADA) to Obinutuzumab
Area Under the Plasma Concentration Versus Time Curve (AUC) of Obinutuzumab
Change From Baseline of Participant's Global Assessment of Disease Activity Visual Analog Scale (VAS) Score
Maximum Observed Plasma Concentration (Cmax) of Obinutuzumab
Percent Change From Baseline in Circulating CD19-Positive B-Cell Levels
Percentage of Participants With Adverse Events (AEs)
Percentage of Participants With Adverse Events of Special Interest: Infusion Related Reactions, Grade 3 or Higher Infections, Drug-related Neutropenia and Drug-related Thrombocytopenia
Percentage of Participants With First Protocol Defined CRR Over the Course of 52 Weeks
Percentage of Participants With First Protocol Defined Overall Response Over the Course of 52 Weeks
Systemic Clearance of Obinutuzumab
Terminal Plasma Half-Life (t1/2) of Obinutuzumab
Volume of Distribution Under Steady State (Vss) of Obinutuzumab
Non-relapse Mortality
Overall Survival
Proportion of Participants With Chronic Graft Versus Host Disease
Proportion of Patients With Severe Acute Graft Versus Host Disease
Time to Neutrophil Engraftment
Time to Platelet Engraftment (20k)
GI Tolerability as Measured by GSRS
Allograft Survival Rate
Incidence of Acute Rejection
Participant Survival Rate
Percentage of Participants in the Experimental Arm Off Tacrolimus
Percentage of Participants With New Donor Specific Antibodies (DSAs)
Estimated GFR Using the Chronic Kidney Disease Epidemiology (CKD-EPI) Equation
Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 100 Days
Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 180 Days
Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
Average IgG Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
Average IgG Levels as a Measure of Immune Reconstitution After Transplant, by 180 Days
Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 100 Days
Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 180 Days
Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
Average Immunoglobulin G (IgG) Levels as a Measure of Immune Reconstitution After Transplant, by 100 Days
Number of Participant With Neutrophil Recovery
Number of Participants Experiencing Acute Graft-versus-host Disease (GVHD)
Number of Participants Experiencing Chronic GVHD
Number of Participants Experiencing Grade ≥3 Regimen Related Toxicity
Number of Participants Experiencing Infections by Day 100
Number of Participants Experiencing Infections by Day 180
Number of Participants Experiencing Infections by Day 365
Number of Participants Experiencing Overall Survival
Number of Participants With Secondary Graft Failure at 100 Days
Number of Participants With BK Viral Load <600 Copies/mL
Number of Participants With Incidence of BK Nephropathy
Count of Participants Who Achieved Neutrophil Engraftment
Count of Participants With Disease Free Survival
Count of Participants With Disease Free Survival
Counts of Participants With Disease Free Survival
Number of Participant Who Were Alive at 2 Years Post Transplant
Number of Participant Who Were Alive at 5 Years Post Transplant
Number of Participant Who Were Alive at 7 Years Post Transplant
Percentage of Participants With Chronic Graft-Versus-Host Disease
Percentage of Participants With Chronic Graft-Versus-Host Disease
Percentage of Participants With Engraftment Failure
Percentage of Participants With Relapse
Percentage of Participants With Relapse
Percentage of Participants With Treatment-Related Toxicity
Percentage of Participants With Treatment-Related Toxicity
Percentage of Participants With Acute Graft-Versus-Host Disease by Grade
Number of Participants With Sustained Cell Engraftment of Donor Cells
Assessment of Treatment Related Mortality and Morbidity
Event Free Survival; Number of Participants Who Survived at 2 Years
Dose-normalized AUC of Mycophenolic Acid
Collection of Blood for Future Analysis of Peripheral Blood Mononuclear Cells (PBMCs)
Collection of Cerebrospinal Fluid for Future Analysis of Cytokine Levels
Mean Rate of Change in Grip Strength Treatment Compared to Pre-Treatment
Mean Rate of Change of ALSFRS-R Scores During Treatment Compared to Pre-Treatment
Mean Rate of Change of Hand-Held Dynamometry (HHD) During Treatment Compared to Pre-Treatment
Mean Rate of Change of Slow Vital Capacity (SVC) During Treatment Compared to Pre-Treatment
Mean Rate of Change of T-cell Subsets in Blood Treatment Compared to Pre-Treatment
Number of Participants With an Average Increase in ALSFRS-R Score of One Point Per Month
Number of Participants in Overall Survival.
Number of Participants Who Experienced Nonrelapse Mortality.
Number of Participants Who Experienced Relapse.
Number of Participants With Grade ≥3 Toxicities Scored According to the CTCAE Version 4.0.
Number of Participants With Acute Grade II-IV GVHD, Acute Grade III-IV GVHD, and Chronic GVHD.
Number of Participants With Neutrophil and Platelet Engraftment.
Pharmacokinetic Analysis of MMF AUC to Evaluate MMF Dose Relationships to Drug Exposure.
Pharmacokinetic Analysis of MMF Clearance to Evaluate MMF Dose Relationships to Drug Exposure.
Pharmacokinetic Analysis of MMF Steady State Concentration to Evaluate MMF Dose Relationships to Drug Exposure.
Overall Survival
Rate of Relapse
Transplant-related Mortality
Acute Grade II-IV GVHD and Chronic (Extensive) GVHD
Rate and Types of Infections
Duration of Renal Response in Participants Who Achieve Partial or Complete Renal Response at Any Time During the Study
Percentage of Participants Who Achieve a Complete or Partial Renal Response at Week 52
Percentage of Participants Who Achieve Complete Renal Response at Week 52
Percentage of Participants With Active Urinary Sediment at Baseline Who Have Inactive Urinary Sediment at Week 52
Percentage of Participants With uPCR > 3.0 mg/mg at Baseline Who Achieve uPCR <1.0 mg/mg at Week 52
Time to Renal Response (Partial or Complete) in Participants Who Achieve Renal Response at Week 52
Duration of Renal Response in Participants Who Achieve Complete Renal Response at Week 52
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Discontinuation During the Run-In Period
Number of Participants With AEs, SAEs and AEs Leading to Study Discontinuation During the Double-Blind Period
Acute Graft-versus-Host Disease (GVHD) Grade 2-4 Risk From Time of Transplant Until Day 90 Post-transplant
Engraftment of Haploidentical CD34+ Selected Blood Stem Cells in Older Patients or Those With Medical Co-morbidities Following Total Lymphoid Irradiation and Antithymocyte Globulin Transplant Conditioning
Evaluate the Risk for Disease Progression and Relapse
Evaluate the Risk of Transplant Related Mortality.
Evaluate the Risk/Incidence of Infections
Determine Whether Engraftment Can be Maintained With a Single Dose Fludarabine, DLI and Continued MMF/CSP, Defined as Rejection Rate < 20%.
Evaluate the Risk of Occurrence of Acute and Chronic GVHD
Donor Engraftment (Chimerism)
Incidence of Grades III-IV Acute GVHD
Non-relapse-related Mortality
Response Rates
Treatment Related Mortality (TRM)
Engraftment and Hematopoietic Toxicity
Incidence and Severity of Acute Graft-versus-host Disease (GVHD) and Chronic GVHD.
Overall and Progression-free Survival
Mean Percent Change in Calculated Creatinine Clearance From Baseline to Months 6, 12, and 24
Mean Percent Change in Calculated Glomerular Filtration Rate From Baseline to Months 6, 12, and 24 (Nankivell Equation)
Mean Percent Change in Glomerular Filtration Rate From Baseline to Month 12
Mean Percent Change in Glomerular Filtration Rate From Baseline to Month 24
Mean Percent Change in Serum Creatinine From Baseline to Months 6, 12, and 24
Overall Survival
Serious Adverse Events
Count of Participants Diagnosed With Epstein-Barr Virus (EBV) Infection as an Adverse Event
Count of Participants Diagnosed With Malignancy as an Adverse Event
Count of Participants With an Infectious Disease Serious Adverse Event(s) Requiring Hospitalization or Systemic Therapy
Count of Participants With Defined CKD Stage 4 or 5 at Wk 52 Post-Transplant
Count of Participants With Delayed Graft Function at Wk 52 Post-Transplant
Count of Participants With eGFR < 60 mL/Min/1.73 m^2 Measured by CKD-EPI at Wk 52 Post-Transplant
Count of Participants With Evidence of Pancreatic Loss at Week 52 Post-Transplant
Count of Participants With Evidence of Partial Pancreatic Graft Function at Week 52 Post-Transplant
Count of Participants With Full Pancreatic Graft Function (Insulin Independent) at Wk 52 Post-Transplant
Count of Participants With Successful Discontinuation of Tacrolimus in Recipients Randomized to the Investigational Arm
Mean Calculated eGFR Using MDRD 4 Variable Model at Wk 52 Post-Transplant
Mean Estimated Glomerular Filtration Rate (eGFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52 Post-Transplant
The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine Post-Transplant
Count of Participant Diagnosed With BK Polyoma Virus (BKV) and Cytomegalovirus (CMV) Viremia As Adverse Events
Count of Participants With Acute Rejection (AR) of Kidney or Pancreatic Transplant During the First 52 Wks Post-Transplant
Count of Participants With Biopsy-Proven Humoral Rejection During the First 52 Weeks Post-Transplant
Count of Participants With De Novo Anti-Donor Antibodies or Anti-Human Leukocyte Antigen (HLA) Antibodies During the First 52 Weeks Post-Transplant
Count of Participants With Event of Death, Graft Loss, or Undetectable C-peptide
Count of Participants With the Occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Count of Participants With Use of Anti-hypertensive Medication From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
Count of Participants With Use of Lipid Lowering Medications at Baseline, Wk 28 and Wk 52 Post-Transplant
Fasting Blood Sugar (FBS) From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
Fasting Lipid Profile at Baseline (Pre-Transplant)
Fasting Lipid Profile at Wk 28 Post-Transplant
HbA1c at Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
Lipid Profile at Wk 52 Post-Transplant
Severity Grade of First Biopsy-Proven Acute Rejection (AR) During the First 52 Weeks Post-Transplant
Standardized Blood Pressure Measurement From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
Type of Treatment(s) Participants Received for Biopsy-Proven Pancreatic Allograft Rejection During the First 52 Weeks Post-Transplant
Type of Treatment(s) Participants Received for Biopsy-Proven Renal Allograft Rejection During the First 52 Weeks Post-Transplant
Incidence of Adverse Events
Incidence of Grades III-IV Acute GVHD
Incidence of Transplant-related Mortality
Number of Patients Who Engraft at Each Dose of TBI Used
Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose
Unified Batten Disease Rating Scale Behavior Subscale Change
Unified Batten Disease Rating Scale Capability Subscale Change
Unified Batten Disease Rating Scale Physical Subscale Change
Unified Batten Disease Rating Scale Seizure Subscale Change
Development of Infectious Complications
Disease Activity
EFS
Event-free Survival (EFS)
Incidence and Severity of GVHD
Incidence of Disease-modifying Drugs for CD Initiated Post-transplant
Incidence of Graft Rejection
Overall Survival
Quality of Life Measured Using the Previously Validated Short Inflammatory Bowel Disease Questionnaire
Regimen-related Toxicity Graded According to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4
Treatment-related Mortality (TRM)
Count of Participant Deaths or Graft Loss by Wk 52 Post-Transplant
Count of Participants With Acute Cellular Rejection Grade ≥ IA Defined by Banff 2007 Criteria By Wk 52 Post-Transplant
Count of Participants With Antibody Mediated Rejection by Wk 52 Post-Transplant
Count of Participants With Biopsy Proven Acute Rejection By Wk 52 Post-Transplant
Count of Participants With Defined CKD Stage 4 or 5 at Wk 52 Post-Transplant
Count of Participants With Delayed Graft Function at Wk 52 Post-Transplant
Count of Participants With eGFR < 60 mL/Min/1.73 m^2 Measured by CKD-EPI at Wk 52 Post-Transplant
Count of Participants With Epstein-Barr Virus (EBV) Infection as Reported on the Case Report Form as Adverse Events
Count of Participants With Graft Rejection by Wk 52 Post-Transplant
Count of Participants With Infections Requiring Hospitalization or Systemic Therapy by Wk 52 Post-Transplant
Count of Participants With Treated Diabetes Between Day 14 and Wk 52 Post-Transplant
Count of Participants With Use of Anti-hypertensive Medication at Wk 52 Post-Transplant
Mean Calculated eGFR Using MDRD 4 Variable Model at Wk 52 Post-Transplant
Mean Estimated Glomerular Filtration Rate (eGFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52 Post-Transplant
The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine Post-Transplant
Count of Participants by Severity of First Acute Cellular Rejection by Wk 52 Post-Transplant
Count of Participants Experiencing ≥ 1 Adverse Event (AEs) or Serious Adverse Events (SAEs) by Wk 52
Count of Participants With BK Polyoma Virus (BKV) and Cytomegalovirus (CMV) Viremia (Local Center Monitoring) as Adverse Events by Wk 52 Post-Transplant
Count of Participants With Either New Onset Diabetes After Transplant (NODAT) or Impaired Fasting Glucose (IFG) at Week 52 Post-Transplant -Based on Criteria Specified by the ADA and WHO
Count of Participants With Fever > 39 Degrees Celsius and Blood Pressure < 90 mmHg Within 24 Hours of Onset of Transplant Procedure
Count of Participants With Use of Lipid Lowering Medications at Baseline and Wk 28 and Wk 52 Post-Transplant
Fasting Lipid Profile at Baseline (Pre-Transplant)
Fasting Lipid Profile at Wk 28 Post-Transplant
Fasting Lipid Profile at Wk 52 Post-Transplant
Hemoglobin A1c (HbA1c) Measurements Over Time
Standardized Blood Pressure Measurement at Wk 52 Post-Transplant
Total Daily Prescribed Pill Count
Type of Rejection Classified by Pathologist - For Cause Kidney Biopsies
Type of Treatment for Detected Graft Rejection
Disease Relapse or Progression as Measured by CT Scan or PET
Overall Survival
Progression-free Survival at 1 Year
Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
Change in Forced Vital Capacity (FVC)
Change in in Short Form-36 (SF-36) Questionnaire:Mental Component Summary
Change in Modified Rodnan Skin Score (MRSS)
Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI)
Change in Short Form-36 (SF-36) Questionnaire: Physical Component Summary
Number of Adverse Events and Serious Adverse Events
Effectiveness and Safety of a Particular Drug Regimen to Prevent Kidney Rejection
Dose-Normalized C0
Dose-Normalized Cmax (mg/L)
Dose-Normalized Cmin
Dose-Normalized MPA AUC0-12
Maximum Plasma Concentration (Cmax)
Minimum Plasma Concentration (Cmin)
MPA Area Under the Curve From 0 to 12 Hours (AUC0-12)
Pre-dose Trough Concentration (C0)
Percentage of Participants By Time to Maximum Plasma Concentration (Tmax)
Regression Coefficients For Participants Receiving MMF
Creatinine Clearance at 1 Month After Transplantation
Creatinine Clearance at Month 12 After Transplantation
Creatinine Clearance at Month 6 After Transplantation
GFR at Month 12 After Transplantation
GFR at Month 6 After Transplantation
Glomerular Filtration Rate (GFR) at Month 1 After Transplantation
Mean Dose of Mycophenolate Mofetil
Percentage of Participants Who Received Other Immunosuppressive Agents in Combination With Mycophenolate Mofetil
Percentage of Participants With Acute Rejection
Percentage of Participants With Graft Survival
All-Cause Mortality
Cumulative Excess Systemic Steroid Dose From Time of Clinically Significant Disease Reactivation to Return to Pre-Flare Dose or End of Trial Participation
Cumulative Systemic Steroid Dose by Week 60
Mortality Related to Systemic Lupus Erythematosus (SLE)
Number of Infection-Related Adverse Events (AEs)
Number of Malignancies Reported as Adverse Events (AEs).
Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60
Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup
Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF<2000 mg/Day Subgroup
Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup
Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup
Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60
Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup
Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup
Number of Participants Experiencing Any British Isles Lupus Assessment Group (BILAG) A Flare by Week 60
Number of Participants Experiencing Clinically Significant Disease Reactivation by Week 60
Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF <2000 mg/Day Subgroup
Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup
Number of Participants in the Lupus Nephritis Subgroup Experiencing a British Isles Lupus Assessment Group (BILAG) A Renal Flare by Week 60
Number of Serious Adverse Events (SAEs).
The Addition of Aggressive Adjunctive Therapy to Mycophenolate Mofetil (MMF) or Change in MMF Therapy to Cytotoxic Drug Due to Flare by Week 60
Time From Clinically Significant Disease Reactivation to Improvement in British Isles Lupus Assessment Group (BILAG) From Maximum Level During Flare
Time From Clinically Significant Disease Reactivation to Recovery to Baseline British Isles Lupus Assessment Group (BILAG) Score or BILAG C
Time From Clinically Significant Disease Reactivation to Return to Pre-Flare Steroid Dose
Time to Clinically Significant Disease Reactivation
Time to First Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare
Time to First Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare
Change From Baseline in the Functional Assessment of Chronic Illness Therapy (FACIT-F) Fatigue Scale (FS): Total Score
Change From Baseline in the Lupus Quality of Life (QoL)Score
Change From Baseline in the Short Form Health Survey (SF-36) Physical Component Summary (PCS) Score
Change From Baseline in the Short Form Health Survey (SF-36) Physical Functioning (PF) Score
Change From Baseline in the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Disease Damage Index for Systemic Lupus Erythematosus (SLICC/DI): Total Score
Number of Grade 3, 4, or 5 Adverse Events (AEs)
Number of Grade 3, 4, or 5 Adverse Events (AEs) Related to Mycophenolate Mofetil (MMF)
Number of Grade 3, 4, or 5 Adverse Events (AEs) Related to Systemic Lupus Erythematosus (SLE)
Number of Grade 3, 4, or 5 Hematological Adverse Events (AEs).
Percentage of Participants With Chronic GVHD
Percentage of Participants With Chronic GVHD Requiring Immunosupressive Therapy
Percentage of Participants With Disease Relapse or Progression
Percentage of Participants With Disease-free Survival
Percentage of Participants With Grade II-IV Acute GVHD
Percentage of Participants With Grade III-IV Acute GVHD
Percentage of Participants With GVHD-free Survival
Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS)
Percentage of Participants With Overall Survival
Percentage of Participants With Transplant-Related Mortality (TRM)
Percentage of Participants With Neutrophil Recovery
Percentage of Participants With Platelet Recovery
Donor Cell Engraftment
Clinical Response
Median Time to Neutrophil Engraftment
Progression Free Survival (PFS) at One Year
Transplant Related Mortality (TRM)
Number of Days for Absolute Neutrophil Count to Recover
Number of Patients Engrafting at Day +30 by Short Tandem Repeat (STR) on Peripheral Blood Mononuclear Cells (PBMC's).
Number of Patients With Acute Grade II-IV GVHD
Patients Who Experience Serious Transplant Related Toxicities as Evaluated by Bone Marrow Transplant-adjusted NCI Common Toxicity Criteria.
Time to Resolution of Cytopenias: Platelet Transfusion Independence
Percentage of Participants Alive at 1 Year After Transplant
Percentage of Patients Alive at the End of the Trial
Cumulative Incidence of Neutrophil and Platelet Engraftment
Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Number of Non-Relapse Mortalities
Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD)
Number of Patients With Grade III-IV Acute GVHD
Number of Participants With Event-free Survival (EFS)
Number of Participants With Malignant Relapse
Number of Participants With Neutrophil Engraftment
Number of Participants With Overall Survival (OS)
Number of Participants With Secondary Graft Failure
Number of Participants With Transplant-related Morbidity
Number of Participants With Transplant-related Mortality (TRM)
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Disease-Free Survival (DFS)
Probability of Overall Survival at 15 Months Post-treatment
Overall Survival (OS)
Treatment-Related Mortality (TRM)
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) (Calculated With Modified Diet in Renal Disease (MDRD)-4 Formula - ITT
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) (Calculated With Modified Diet in Renal Disease (MDRD)-4 Formula - Modified ITT
Change From Baseline in Serum Creatinine - ITT
Change From Baseline in Serum Creatinine - Modified ITT
Duration of DGF
Participant/Graft Survival Rate: Percentage of Participants With Failure Events of Death or Graft Loss - Worst-case Scenario
Patient Survival Rate: Percentage of Deaths - Worst-case Scenario
Percentage of Participants Who Experienced Treatment Failure - Worst-case Scenario
Percentage of Participants With a New Onset of Diabetes
Percentage of Participants With a New Onset of Malignancy
Percentage of Participants With Acute Rejection (AR)
Percentage of Participants With Delayed Graft Function (DGF) -
Percentage of Participants Without Wound Healing Complications - Worst-case Scenario
Percentage of Participants Without Wound Healing Complications - Worst-case Scenario
Graft Survival Rate: Percentage of Participants With Graft Loss - Worst-case Scenario
Percentage of Participants With BPAR - Worst-case Scenario
Percentage of Participants With Proteinuria
Cholesterol
Evaluation for the Development of BK Virus Nephropathy or Doubling of BK Viremia Levels
Evidence of Reduction of BK Viruria and/or Clearance of BK Viremia
Median Between the Calculated Mean Residual Expression of NFAT-regulated Genes
p70S6 Kinase Phosphorylation
Proteinuria
Complete Remission (CR) Rate at Day 30 Post HSCT
Neutrophil Engraftment
Non-relapse Related Mortality
Rate of Acute Graft-versus-host Disease (GVHD)
Rate of Chronic GVHD
Severity of Acute Graft-versus-host Disease (GVHD)
Severity of Chronic GVHD
Number of Patients Reported Ascites
Survival at Day 29 of the Assigned Treatment
Incidence of Cytomegalovirus Infection or Disease
BPAR (Biopsy-proven Acute Rejection) Incidence During the First 12 Months Post-transplant
Discontinuance of Any Study Medication (Tacrolimus, Everolimus, or EC-MPS)
eGFR (Calculated Glomerular Filtration Rate), i.e., Renal Function, at 1 Month Post-transplant.
eGFR (Renal Function) at 6 Months Post-transplant
eGFR (Renal Function) at Month 3 Post-transplant
Graft Loss (Return to Permanent Dialysis or Death)
Incidence of Chronic Allograft Nephropathy (CAI) at 12 Months Post-transplant
Chronic Graft Versus Host Disease (GVHD)
The Feasibility of the Conditioning Regimen and Post Transplantation Cyclophosphamide in Refractory SLE Patients With Donors Having Various Degrees of Matching
Acute Graft Versus Host Disease (GVHD)
Graft Failure
Survival
Median Time to ANC > 500
Number of Participants With Graft Failure/Rejection
Overall Survival
Percent of Patients With Acute GVHD Grades III-IV
Percent of Patients With Chronic GVHD
Percent of Patients With Grade II-IV Acute Graft Versus Host Disease
Percent of Patients With Non-relapse Mortality
Percent of Patients With Non-relapse Mortality
Time to Platelet Engraftment of > 20,000 Cells Per mm3
Engraftment of HLA Identical PBSC Allografts
Non-Relapse Mortality
Overall Survival (OS)
Progression Free-survival (PFS)
Acute Graft-Versus-Host Disease (aGVHD) Outcome
Chronic Graft-Versus-Host Disease (cGVHD) Outcome
Non-relapse Mortality
Overall Survival
Progression-Free Survival
Regimen-Related Toxicities
Relapse Mortality
Participants Experiencing Acute Rejection Episode
Participants Experiencing Adverse Event Attributable to Study Drug
Participants Experiencing Graft Failure
Renal Recovery/ Function
Survival
Change From Baseline (Randomization) in Creatinine Clearance Estimated Using the Adjusted Cockcroft-Gault Formula
Change From Baseline (Randomization) in Glomerular Filtration Rate Estimated by Abbreviated Modification of Diet in Renal Disease (MDRD) Formula
Change From Baseline (Randomization) in Glomerular Filtration Rate Estimated by CKD-EPI Formula
Change From Baseline (Randomization) in Renal Function
Change From Baseline (Randomization) in Serum Creatinine
Change From Baseline (Randomization) in Urine Protein/Creatinine Ratio
Number of Patients in Different Stages of Chronic Kidney Diseases According to the K/DOQI Classification System
Number of Patients Reported With Different Categories of Severity of BPAR According to Banff Classification
Number of Patients With Any Adverse Events, Serious Adverse Events, Death and Premature Discontinuation
Number of Patients With Death or Graft Loss
Number of Patients With Treated or Not Treated Biopsy Proven Acute Rejection (BPAR)
Number of Patients With Treated or Untreated BPAR With RAI Score Greater Than 3
Number of Patients With Treatment Failures
Number of Participants Achieving Treatment Success After Switching to Other Medication (Phase II, 0-6 Months)
Number of Participants Achieving Treatment Success at 12 Months on Same Medication (Phase I, 6-12 Months)
Number of Participants Achieving Treatment Success at 6 Months (Phase I, 0-6 Months)
Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100
Percent Probability of 18 Months-relapse Event Between Arms
Percent Probability of Event-free Survival (EFS)
Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
Graft Loss - Percentage of Participants With an Event
Participant Survival
Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) Before Week 12 or Acute Subclinical Rejection on Protocol Biopsy at Week 12
Time to Graft Loss
Time to Occurrence of First BPAR Between Day 0 and Week 52
Time to Occurrence of First BPAR Between Day 0 and Week 52 - Percentage of Participants With an Event
Creatinine Clearance Values Estimated With the Cockcroft-Gault Equation (Milliliters Per Minute [mL/Min])
Creatinine Clearance Values Estimated With the Modification of Diet in Renal Disease (MDRD) Simplified Equation
Graft Histology - Percentage of Participants With at Least One Borderline Lesion at Week 12 and Week 52
Graft Histology - Percentage of Participants With at Least One Chronic Graft Nephropathy at Week 12 and Week 52
Percentage of Participants With at Least One BPAR at Week 12 and Week 52
Serum Creatinine Values [Micromoles Per Liter (µmol/L)]
Incidence of Chronic Graft Versus Host Disease (cGVHD)
Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD)
Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD)
Number of Subjects Alive at 100 Days
Number of Subjects Alive at One Year
Number of Subjects With Mixed Chimerism
Percentage of Donor Chimerism at 100 Days
Percentage of Donor Chimerism at 180 Days
Percentage of Donor Chimerism at 365 Days
Death From GVHD
Death From Regimen Toxicity or Opportunistic Infection
Overall Survival
Progression of HIV
Reconstitution of HIV-specific Immunity
Successful Induction of Mixed Hematopoietic Chimerism as Assessed by the Percentage of Peripheral Blood T Cells That Are of Donor Origin
Acute Rejection
Change in eGFR (MDRD) at 2 Years Post-transplant Compared to Baseline at Month 3 (Conversion)
Graft Survival
Patient Survival
Cumulative Incidence of Chronic GVHD
Cumulative Incidence of Non-relapse Mortality
Incidence of Donor Cell Engraftment
Number of Participants With Donor Cell Engraftment
Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4
Primary and Secondary Graft Failure
Steroid and Non-steroid Immunosuppressants
Steroid and Non-steroid Immunosuppressants Use Duration
Survival
Survival
Time to Neutrophil and Platelet Recovery
Duration of Wound Healing
Glomular Filtration Rate (GFR) mL/Min Via Cockcroft- Gault Method at Month 12 Post Transplant
Glomular Filtration Rate (GFR) Via Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Method at Month 12 Post Transplant
Glomular Filtration Rate (GFR) Via Modification of Diet in Renal Disease (MDRD) Method at Month 12 Post Transplant
Glomular Filtration Rate (GFR) mL/Min Via Nankivell Method at Month 12 - Standard Regimen vs Certican Regimens
Percent of Participants With Delayed Graft Function and Slow Graft Function
Percent of Participants With Delayed Graft Function by Day
Percent of Participants With Viral Infections
Percent of Participants With Wound Healing Complications During Study
Percentage of Participants With Composite Treatment Failure Endpoints - Difference Between Groups at Month 12
Percentage of Participants With Treatment Failure Endpoints at Month 12
Cumulative Incidence of Chronic GVHD According to BMTCTN
Cumulative Incidence of Grade III-IV Acute GVHD
Number of Participants With Complete Remission After Transplantation
Number of Participants With Primary Graft Failure
Overall Survival (OS)
Percentage of Participants With Platelet Recovery by Day 30
Progression Free Survival
Time to Neutrophil Recovery
Two-year Disease-free Survival of Study Participants Who Completed the Study Regimen
Number of Participants With 100% Donor Chimerism at Day 28 and Day 84
Participant Disease Response Within 4 Weeks After Transplant

Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)

Changes in GI symptom severity was measured by changes in the Gastrointestinal Symptom Rating Scale (GSRS) total score from baseline visit to the visit at 6-8 weeks. This total score was calculated as the average of the 15 single items (each ranging from 1-7 score points) and thus also had a range from 1-7 score points. Higher values indicate more unfavorable conditions. (NCT00351377)
Timeframe: Baseline and 6 - 8 weeks

InterventionScores on a scale (Mean)
Baseline (N= 111)6-8 Weeks (N= 102)Change From Baseline (N = 102)
Enteric-coated Mycophenolate Sodium2.282.02-0.28

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Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium

The PGWB consists of 22 single items (each ranging from 0-5) with 7 dimensions (including the total score) to be calculated. Lower scores indicate more unfavorable conditions. The total raw score is calculated by summing up all of the single items and thus has a hypothetical range from 0-110 score points. This raw score is further transformed using the formula: (raw score / 110) x 100 to fit a range from 0-100. (NCT00351377)
Timeframe: Baseline and 6-8 weeks

InterventionScores on a scale (Mean)
Baseline (N= 110)6-8 Weeks (N= 103)Change From Baseline (N= 103)
Enteric-coated Mycophenolate Sodium65.366.82.0

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Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium

The change from baseline to the 6-8 week visit for each of the six subscores (each ranging from 0-5) of the PGWB were analyzed individually. Each of the subscores was transformed to fit a range from 0-100. Lower scores indicate more unfavorable conditions, so an increase in score indicates an improvement in symptoms. (NCT00351377)
Timeframe: Baseline and 6-8 weeks

InterventionScores on a scale (Mean)
Anxiety [n=103]Depressed mood [n=104]Positive well-being [n=104]Self control [n=104]General health [n=104]Vitality [n=104]
Enteric-coated Mycophenolate Sodium1.31.50.71.53.42.7

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Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium

Changes in GI symptom severity was measured by changes in the total scores of 5 subscales (reflux, diarrhea, constipation, abdominal pain and indigestion) of the Gastrointestinal Symptom Rating Scale (GSRS) from baseline visit to the visit at 6-8 weeks. The GSRS is a 15-item instrument with a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort). (NCT00351377)
Timeframe: Baseline and 6-8 weeks

InterventionScores on a scale (Mean)
Reflux [n =102]Diarrhea [n =104]Constipation [n = 104]Abdominal pain [n =102]Indigestion [n = 104]
Enteric-coated Mycophenolate Sodium-0.24-0.270.02-0.51-0.42

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Overall Treatment Effects for GI Symptoms Assessed by the Patient

"Assessed using the Overall Treatment Effects for GI symptoms questionnaire. The question was: Has there been any change in the participant's GI symptoms since his/her last study visit? Please indicate if there has been any change in his/her symptoms. The possible answers were: Improved, about the same, or worse. The questionnaire was completed by the patient." (NCT00351377)
Timeframe: 6-8 week

InterventionParticipants (Number)
ImprovedAbout the sameWorsenedMissing data
Enteric-coated Mycophenolate Sodium3853911

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Overall Treatment Effects for GI Symptoms Assessed by the Physician

"Assessed using the Overall Treatment Effects for GI symptoms questionnaire. The question was: Has there been any change in the participant's GI symptoms since his/her last study visit? Please indicate if there has been any change in his/her symptoms. The possible answers were: Improved, about the same, or worse. The questionnaire was completed by the physician." (NCT00351377)
Timeframe: 6-8 week

InterventionParticipants (Number)
ImprovedAbout the sameWorsenedMissing data
Enteric-coated Mycophenolate Sodium4943136

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Acute Graft-Versus-Host Disease

Grade III-IV graft versus host disease (NCT00303719)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
High Risk Patients2
Standard Risk Patients79

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Neutrophil and Donor Cell Engraftment

"Successful sustained engraftment is defined as primary neutrophil engraftment by day 42 and e90% donor cells at day 100, with or without DLI.~Engraftment based on absolute neutrophil count of donor origin > 0.5 x 10e9 /L for 3 days by day 42" (NCT00303719)
Timeframe: Day 42 and Day 100

InterventionParticipants (Count of Participants)
High Risk Patients12
Standard Risk Patients289

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Overall Survival

(NCT00303719)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
High Risk Patients8
Standard Risk Patients181

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Serious Adverse Events

Safety by development of severe adverse events within 100 days of transplant (NCT00303719)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
High Risk Patients0
Standard Risk Patients47

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Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0

(NCT00423514)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL31
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL7

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British Isles Lupus Assessment Group (BILAG) Index Score Over 52 Weeks

The BILAG Index assesses 86 clinical signs and symptoms and laboratory measures of systemic lupus erythematosus in 8 organ system domains: General, mucocutaneous, neurological, musculoskeletal, cardiorespiratory, vasculitis, renal, and hematologic. Most of the 86 items are rated on the following scale: 0=Not present, 1=Improving, 2=Same, 3=Worse, 4=New. Some items are rated as either Yes or No. A single alphabetic score of A (very active) through E (not or never active) for each of the 8 domains is determined from the rating of the individual items in each domain. The total BILAG score is the sum of the scores of the 8 domains where A=9, B=3, C=1, D=0, and E=0. The total score ranges from 0 to 72 with a higher score indicating greater lupus activity. To calculate a BILAG score over the 52 week treatment period of the study, the area under the response-time curve of BILAG scores assessed every 4 weeks was divided by the number of days in the time curve minus the Baseline BILAG score. (NCT00282347)
Timeframe: Baseline to Week 52

InterventionUnits on a scale (Mean)
Rituximab-8.49
Placebo-8.58

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Change From Baseline in Anti-double-stranded DNA at Week 52

(NCT00282347)
Timeframe: Baseline to Week 52

InterventionIU/mL (Mean)
Rituximab0.45
Placebo1.06

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Change From Baseline in the Systemic Lupus Erythematosus Expanded Health Survey Physical Function Score at Week 52

The systemic lupus erythematosus Expanded Health Survey is based on the Short Form 36 Health survey with additional questions specific to lupus. The physical function component score of the survey can range from 0-100. A higher score indicates better health. A positive change score indicates improvement. (NCT00282347)
Timeframe: Baseline to Week 52

InterventionUnits on a scale (Mean)
Rituximab4.8
Placebo5.7

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Percentage of Participants Who Achieved a Complete Renal Response at Week 24 and Maintained it to Week 52

A participant had a complete renal response if they met the following 3 criteria: (1) Normalization of serum creatinine as evidenced by a serum creatinine level ≤ the upper limit of the normal range of central laboratory values or a serum creatinine level ≤ 15% greater than Baseline, if Baseline serum creatinine was within the normal range of the central laboratory values; (2) Inactive urinary sediment (as evidenced by < 5 red blood cells/high-power field and absence of red cell casts; (3) Urinary protein to creatinine ratio < 0.5. (NCT00282347)
Timeframe: Week 24 to Week 52

InterventionPercentage of participants (Number)
Rituximab1.4
Placebo6.9

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Percentage of Participants Who Achieved a Complete Renal Response at Week 52

A participant had a complete renal response if they met the following 3 criteria: (1) Normalization of serum creatinine as evidenced by a serum creatinine level ≤ the upper limit of the normal range of central laboratory values or a serum creatinine level ≤ 15% greater than Baseline, if Baseline serum creatinine was within the normal range of the central laboratory values; (2) Inactive urinary sediment (as evidenced by < 5 red blood cells/high-power field and absence of red cell casts; (3) Urinary protein to creatinine ratio < 0.5. (NCT00282347)
Timeframe: Week 52

InterventionPercentage of participants (Number)
Rituximab26.4
Placebo30.6

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Percentage of Participants With a Baseline Urine Protein to Creatinine Ratio of > 3.0 Who Achieved a Urine Protein to Creatinine Ratio of < 1.0 at Week 52

(NCT00282347)
Timeframe: Baseline to Week 52

InterventionPercentage of participants (Number)
Rituximab47.4
Placebo53.7

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Time to Achieve a Complete Renal Response

(NCT00282347)
Timeframe: Baseline to Week 52

InterventionWeeks (Median)
Rituximab11.99
Placebo12.12

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Change From Baseline in C3 and C4 Complement Levels at Week 52

(NCT00282347)
Timeframe: Baseline to Week 52

,
Interventionmg/dL (Mean)
C3 ComplementC4 Complement
Placebo25.96.6
Rituximab37.59.9

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Percentage of Participants Who Achieved a Complete Renal Response (CRR), a Partial Renal Response (PRR), or no Renal Response (NRR) at Week 52

A participant had a CRR if they met the following 3 criteria: (1) Normalization of serum creatinine (SC) as evidenced by a SC level ≤ the upper limit of the normal range of central laboratory values or a SC level ≤ 15% greater than Baseline, if Baseline SC was within the normal range of the central laboratory values; (2) Inactive urinary sediment (as evidenced by < 5 red blood cells/high-power field (RBCs/HPF) and absence of red cell casts; (3) Urinary protein (UP) to creatinine ratio (CR) < 0.5. A participant had a PRR if they met the following 3 criteria: (1) A SC level ≤ 15% above Baseline; (2) RBCs/HPF ≤ 50% above Baseline and no RBC casts; (3) 50% improvement in the UP to CR, with 1 of the following conditions met: If the Baseline UP to CR was ≤ 3.0, then a UP to CR of < 1.0 or if the Baseline UP to CR was > 3.0, then a UP to CR of ≤ 3.0. A participant had a NRR if they did not achieve either a CRR or PRR. (NCT00282347)
Timeframe: Week 52

,
InterventionPercentage of participants (Number)
CRRPRRNRR
Placebo30.615.354.2
Rituximab26.430.643.1

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Percentage of Participants With Acute Graft-vs-host-disease (GVHD)

Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). The time of onset of grades II-IV and grades III-IV acute GVHD were recorded. This endpoint is evaluated through 100 days post-transplant. Cumulative percentage of acute GVHD post-transplant are estimated using the cumulative incidence function, treating death prior to acute GVHD as the competing risk. (NCT02918292)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT16.1

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Percentage of Participants With Chronic GVHD

The event for this secondary endpoint is any chronic GVHD based on 2014 NIH Consensus Criteria. This includes mild, moderate and severe chronic GVHD. The analyses of Chronic GVHD use the site-reported data. The cumulative percentage of chronic GVHD is computed using the cumulative incidence function, treating death prior to chronic GVHD as a competing risk. Eight organs will be scored on a 0-3 scale to reflect degree of chronic GVHD involvement. Liver and pulmonary function test results and use of systemic therapy for treatment of chronic GVHD will also be recorded. This secondary endpoint of chronic GVHD will include mild, moderate and severe chronic GVHD based on NIH Consensus Criteria. (NCT02918292)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT25.8

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Percentage of Participants With Graft-Failure-Free Survival

Events for Graft-Failure-Free Survival (GFFS) including death, primary graft failure, secondary graft failure. The time to this event is the time from transplant to death from any cause, or graft failure, or last follow-up, or 1 year from transplant, whichever occurs first. For patients experiencing primary graft failure, Day 0.1 was used for primary graft failure event date to count the event in. The one-year GFFS probability and its 95% confidence interval were estimated using the Kaplan-Meier estimator and Greenwood's formula. (NCT02918292)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT77.4

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Percentage of Participants With Overall Survival (OS)

Overall survival (OS) is the primary endpoint of this study. The time to this event is the time from transplant to death from any cause or last follow-up or 1 year from transplant, whichever occurs first. The one-year OS probability and its 95% confidence interval were estimated using the Kaplan-Meier estimator and Greenwood's formula. (NCT02918292)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT80.6

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Percentage of Participants With Platelet Recovery

Platelet recovery is defined by achieving a platelet count > 20 x 10^9/L with no platelet transfusions in the preceding seven days. The first day of the sustained platelet count will be defined as the day of platelet engraftment. The cumulative percentage of platelet engraftment was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to platelet engraftment treated as a competing risk. (NCT02918292)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT77.4

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Percentage of Participants With Primary Graft Failure

Primary graft failure is defined by the lack of neutrophil engraftment by Day 56 post-HSCT or failure to achieve at least 5% donor chimerism (whole blood or marrow) on any measurements up to and including Day +56. For this protocol, lineage-specific, myeloid, and T cell chimerisms are required. Myeloid engraftment might not proceed at the same rate as T cell engraftment. If myeloid has greater than or equal to 5% donor, even if T cell compartment does not, this is not considered primary graft failure. Secondary graft failure is defined by initial neutrophil engraftment (ANC greater than or equal to 0.5 x 10^8/L measured for 3 consecutive measurements on different days) followed by sustained subsequent decline in ANC to less than 0.5 x 10^9/L for three consecutive measurements on different days or initial whole blood or marrow donor chimerism greater than or equal to 5%, but then declining to less than 5% on subsequent measurements or second infusion/transplant given for graft failure. (NCT02918292)
Timeframe: Day 56

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT12.9

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Percentage of Participants With Secondary Graft Failure

"Secondary graft failure is defined as any one of the following:~Initial neutrophil engraftment (ANC greater than or equal to 0.5 x10^9/L measured for three consecutive measurements on different days) followed by sustained subsequent decline in ANC to less than 0.5 x 10^9/L for three consecutive measurements on different days;~Initial whole blood or marrow donor chimerism greater than or equal to 5%, but then declining to less than 5% on subsequent measurements;~Second infusion/transplant given after Day 56 for graft failure." (NCT02918292)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Haplo Bone Marrow HSCT3.2

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Frequencies of Infections Categorized by Infection Type

The number of systemic infections is reported. Infections are categorized by infection type. A participant can report multiple types of infections, so the categories are not mutually exclusive for participants. All grade 2 and grade 3 infections, as defined by the BMT CTN Technical MOP, occurring post transplantation were reported on the study. (NCT02918292)
Timeframe: 1 Year

Interventioninfections (Number)
Bacterial infectionViral infectionFungal infectionProtozoal infectionOther infection
Haplo Bone Marrow HSCT2632303

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Immune Reconstitution of Flow Cytometry

Quantitative assessments of peripheral blood CD3, CD4, CD8, CD19, and CD56 positive lymphocytes will be done through flow cytometric analysis. (NCT02918292)
Timeframe: Baseline, Days 100, 180, and 365

Interventioncells/uL (Mean)
CD3 at BaselineCD3 at Day 100CD3 at 6 MonthsCD3 at 1 YearCD4 at BaselineCD4 at Day 100CD4 at 6 MonthsCD4 at 1 YearCD8 at BaselineCD8 at Day 100CD8 at 6 MonthsCD8 at 1 YearCD19 at BaselineCD19 at Day 100CD19 at 6 MonthsCD19 at 1 YearCD56 at BaselineCD56 at Day 100CD56 at 6 MonthsCD56 at 1 Year
Haplo Bone Marrow HSCT862550.8640.51121434.1122.3172.6472.7326.9272.9333.3569.7106.2221.1204.8264.6124.6237.6260.3293.2

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Immune Reconstitution of Quantitative Immunoglobulins

Quantitative immunoglobulins of IgA, IgG, IgM were done at baseline and 1-year post-transplant. (NCT02918292)
Timeframe: baseline and 1-year

Interventionmg/dL (Mean)
IgA at BaselineIgA at 1 YearIgG at BaselineIgG at 1 YearIgM at BaselineIgM at 1 Year
Haplo Bone Marrow HSCT172.3111.6987.51004102.896

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Participants With Grade 3-5 Toxicities by SOC

Toxicities are evaluated for the study participants at Day 28, Day 56, Day 100, Day 180 and Day 365 post-transplant and graded using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. Grade 3-5 toxicities are reported with higher grade indicating worse outcomes. Toxicities are summarized here by system organ class (SOC). A participant can report multiple toxicities, so the categories are not mutually exclusive for participants. (NCT02918292)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Abnormal Liver SymptomsBlood and Lymphatic DisordersCardiovascular DisordersChemistry/InvestigationsGI DisordersGeneral DisordersHemorrhagic DisordersHepatic DisordersImmune System DisordersMetabolism and Nutrition DisordersMusculoskeletal and Connective Tissue DisordersNervous System DisordersRenal DisordersRespiratory, Thoracic and Mediastinal DisordersTotal (any of above SOC)
Haplo Bone Marrow HSCT711521053617145823

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Percentage of Participants With Cytomegalovirus (CMV), Epstein Barr Virus (EBV) or Post-Transplant Lymphoproliferative Disease (PTLD)

CMV viremia and disease, EBV viremia, and PTLD are monitored and reported per protocol. The cumulative percentage of each outcome was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to event treated as a competing risk. (NCT02918292)
Timeframe: 1 Year

Interventionpercentage of participants (Number)
Cumulative Percentage of Participants with EBVCumulative Percentage of Participants with CMVCumulative Percentage of Participants with PTLD
Haplo Bone Marrow HSCT9.722.66.5

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Percentage of Participants With Neutrophil Recovery

Neutrophil recovery is achieving an absolute neutrophil count (ANC) > 0.5 x10^9/L for three consecutive measurements on different days, with the first of the three days being defined as the day of neutrophil engraftment. The cumulative percentage of neutrophil engraftment was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to neutrophil engraftment treated as a competing risk. (NCT02918292)
Timeframe: Day 28 and 56

Interventionpercentage of participants (Number)
Day 28Day 56
Haplo Bone Marrow HSCT93.593.5

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Graft Survival at One Year

"Graft survival defined as any participant who did not meet the criteria for graft loss, where graft loss is defined as any re-transplant, permanent return to dialysis (> 30 days), patient death, or participant whose outcome at one year was unknown.~Participants were only counted once regardless of how many criteria were met." (NCT00064701)
Timeframe: One year

Interventionpercentage of participants (Number)
Tacrolimus91.5
Tacrolimus Modified Release95.3
Cyclosporine95.3

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Kaplan-Meier Estimate of Graft Survival at the End of the Study

"Graft survival was defined as any participant who did not meet the definition of graft loss, where graft loss was any retransplant or the permanent return to dialysis (more than 30 days) or patient death.~Graft survival was censored at the time of last follow-up contact." (NCT00064701)
Timeframe: End of study (maximum time on study was 1,941 days).

Interventionpercentage of participants (Number)
Tacrolimus82.7
Tacrolimus Modified Release84.7
Cyclosporine83.9

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Kaplan-Meier Estimate of Patient Survival at the End of the Study

Patient survival was defined as any participant who was alive at the end of the study. Patient survival was censored at the time of last follow-up contact. (NCT00064701)
Timeframe: End of study (maximum time on study was 1,941 days).

Interventionpercentage of participants (Number)
Tacrolimus91.2
Tacrolimus Modified Release93.2
Cyclosporine91.7

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Number of Participants Experiencing Multiple Rejection Episodes

This analysis includes rejection episodes that were either confirmed by biopsy by the clinical site pathologist or were clinically treated. (NCT00064701)
Timeframe: one year

Interventionparticipants (Number)
Tacrolimus2
Tacrolimus Modified Release4
Cyclosporine8

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Number of Participants Requiring Anti-lymphocyte Antibody Therapy for Treatment of Rejection

"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Participants with histologically-proven Banff Grade II or III rejection or participants with steroid-resistant rejection were treated with anti-lymphocyte antibody treatment according to institutional practice.~Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel." (NCT00064701)
Timeframe: one year

Interventionparticipants (Number)
Tacrolimus6
Tacrolimus Modified Release8
Cyclosporine18

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Number of Participants Who Crossed Over Due to Treatment Failure

Participants were allowed to cross over to an alternative primary immunosuppressive regimen (either to the tacrolimus or cyclosporine treatment arms) to address an adverse event which led to randomized study drug discontinuation or in the case of severe or refractory rejection. Crossover to the modified release tacrolimus treatment arm was not permitted. (NCT00064701)
Timeframe: one year

Interventionparticipants (Number)
Tacrolimus6
Tacrolimus Modified Release10
Cyclosporine39

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Number of Participants With Clinically Treated Acute Rejection Episodes

A clinically treated acute rejection episode was any biopsy-confirmed or suspected rejection episode that was treated with immunosuppressive therapy. (NCT00064701)
Timeframe: one year

Interventionparticipants (Number)
Tacrolimus25
Tacrolimus Modified Release39
Cyclosporine45

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Number of Participants With Treatment Failure

Treatment failure was defined as the discontinuation of randomized study drug for any reason. Participants who met the definition of treatment failure were to be followed throughout the 12-month treatment period. (NCT00064701)
Timeframe: one year

Interventionparticipants (Number)
Tacrolimus33
Tacrolimus Modified Release31
Cyclosporine61

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Patient Survival at One Year

Patient survival is defined as any participant who is known to be alive one year after the skin closure date. Participants who died or whose outcome was unknown at one year were considered to be non-survivors. (NCT00064701)
Timeframe: One year

Interventionpercentage of participants (Number)
Tacrolimus93.9
Tacrolimus Modified Release97.2
Cyclosporine97.2

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Percentage of Participants With Efficacy Failure

"Efficacy failure is defined as any participant who died, experienced a graft failure (permanent return to dialysis [> 30 days] or retransplant), had a biopsy-confirmed (Banff Grade ≥ I) acute rejection (BCAR), or was lost to follow-up.~Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel." (NCT00064701)
Timeframe: one year

Interventionpercentage of participants (Number)
Tacrolimus15.1
Tacrolimus Modified Release14.0
Cyclosporine17.0

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Time to First Biopsy-confirmed Acute Rejection Episode

"Time to first biopsy-confirmed acute rejection episode defined as the number of days from skin closure (Day 0) to the date of biopsy. Rejection episodes were confirmed by biopsy by the clinical site pathologist and graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel.~Acute rejection is defined as a grade ≥ I." (NCT00064701)
Timeframe: one year

Interventiondays (Median)
Tacrolimus156.00
Tacrolimus Modified Release11.00
Cyclosporine52.00

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Change From Month 1 in Creatinine Clearance at Month 6 and Month 12

Renal function was assessed by creatinine clearance, calculated using the Cockcroft-Gault formula. (NCT00064701)
Timeframe: Month 1, Month 6, and Month 12

,,
InterventionmL/min (Mean)
At 6 months [N=184, 184, 167]At 12 months [N=173, 182, 145]
Cyclosporine-1.79-0.25
Tacrolimus0.831.50
Tacrolimus Modified Release0.472.62

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Change From Month 1 in Serum Creatinine at Month 6 and Month 12

Renal function was assessed by the change from Month 1 in serum creatinine six months and 12 months after transplant. (NCT00064701)
Timeframe: Month 1, Month 6, and Month 12

,,
Interventionmg/dL (Mean)
At 6 months [N=184, 184, 169]At 12 months [N=173, 182, 147]
Cyclosporine-0.01-0.04
Tacrolimus-0.09-0.08
Tacrolimus Modified Release-0.08-0.14

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Percentage of Participants With Biopsy Confirmed Acute Rejection at 6 and 12 Months

"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel.~Acute rejection is defined as a grade ≥ I." (NCT00064701)
Timeframe: Six months and 12 months

,,
Interventionpercentage of participants (Number)
At 6 MonthsAt 12 Months
Cyclosporine11.813.7
Tacrolimus3.87.5
Tacrolimus Modified Release7.910.3

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Severity of Acute Rejection

"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade IA: Significant interstitial infiltration and foci of moderate tubulitis; Grade IB: Significant interstitial infiltration and foci of severe tubulitis; Grade IIA: Mild to moderate intimal arteritis in at least 1 arterial cross section Grade IIB: Severe intimal arteritis comprising >25% of the luminal area lost in at least 1 arterial cross section; Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel." (NCT00064701)
Timeframe: one year

,,
Interventionparticipants (Number)
Grade I-AGrade I-BGrade II-AGrade II-BGrade III
Cyclosporine146612
Tacrolimus84310
Tacrolimus Modified Release113611

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Number of Participants Who Developed Acute Rejection Episode Within 6 Months Post-Transplant

The acute rejection episode was a composite end-point of acute rejection and treatment failure within 6 months post-transplant (PT). Participants with acute rejection included participants with a biopsy histology of International Society of Heart and Lung Transplant (ISHLT) Grade IIIA, IIIB, or IV and participants with hemodynamic compromise (HDC) who were treated for acute rejection (whether or not a biopsy was done and regardless of the grade of the biopsy). Participants who had treatment failure included participants who died within 6 months of transplantation before experiencing acute rejection or who were re-transplanted within 6 months of the primary transplantation and who did not experience an acute rejection or who were lost to follow-up. (NCT00048165)
Timeframe: Up to 6 months PT

Interventionparticipants (Number)
Daclizumab77
Placebo104

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Number of Participants Who Developed Acute Rejection Episode Within the 12 Months PT

The acute rejection episode was a composite end-point of acute rejection and treatment failure within 6 months. Participants with acute rejection included participants with a biopsy histology of ISHLT Grade IIIA, IIIB, or IV and participants with hemodynamic compromise (HDC) who were treated for acute rejection (whether or not a biopsy was done and regardless of the grade of the biopsy). Participants who had treatment failure included participants who died within 6 months of transplantation before experiencing acute rejection or who were re-transplanted within 6 months of the primary transplantation and who did not experience an acute rejection or who were lost to follow-up (NCT00048165)
Timeframe: Up to 12 months PT

Interventionparticipants (Number)
Daclizumab97
Placebo116

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Mean Maintenance Doses of Mycophenolate Mofetil, Cyclosporine, and Cumulative Dose of Corticosteroids at 6 and 12 Months PT

The maintenance doses of mycophenolate mofetil (1.5g twice a day daily), cyclosporine (1-4 mg/kg IV or 2-6 mg/kg oral [PO]/nasogastric [NG] within 72 hours post-operative]), and cumulative dose of corticosteroids (500-1000 mg IV methylprednisolone pre-operative switched to oral at 0.5-1 mg/kg/day. Tapered to 0.2 mg/kg/d by Day 28, 0.1-0.15 mg/kg/day from Days 36 to 90, and 0.1-0.15 mg/kg/day from Days 120 to 180)at 6 and 12 months PT were reported. Maintenance dose was calculated as total dose per day summed over all days that a participant was administered drug within the specified time interval, divided by number of days that a participant took drug within that time interval. (NCT00048165)
Timeframe: Within 6 months and 12 months PT

,
Interventionmg (Mean)
MMF dose, 6 months PT (n=193, 201)MMF dose,12 months PT (n=188, 194)IV Cyclosporine dose, 6 months PT (n=2, 1)IV Cyclosporine dose, 12 months PT (n=4, 2)PO/NG Cyclosporine dose, 6 months PT (n=184, 182)PO/NG Cyclosporine dose, 12 months PT (n=170, 170)Cumulative corticosteroids,6 months PT(n=203, 206)Cumulative corticosteroids,12 months PT(n=195,200)
Daclizumab2522.22394.186.1193.5321.9294.7848.11199.6
Placebo24502380.138.146.7331.1305.7955.41288.9

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Median Change From Baseline for LDL/HDL Ratio

(NCT00048165)
Timeframe: From Baseline (Day -2) to 3 months, and 6 months

,
Interventionratio (Median)
LDL/HDL ratio,change at 3 months (n=91,89)LDL/HDL ratio,change at 6 months (n=91, 99)
Daclizumab-0.44-0.50
Placebo-0.12-0.14

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Median Change From Baseline for Lipid Profile (Total Cholesterol, Low Density Lipoproteins, High Density Lipoproteins, and Triglycerides)

Lipid profile included total cholesterol, low density lipoproteins (LDL), high density lipoproteins (HDL), and triglycerides (all total cholesterol, LDL, HDL, and triglycerides with unit milligram per decilitre [mg/dL]), were reported. The median change from baseline (Day -2) in lipid profile values at 3 months and 6 months was reported. (NCT00048165)
Timeframe: From Baseline (Day -2) to 3 months and 6 months

,
Interventionmg/dL (Median)
Total cholesterol, change at 3 months (n=119,119)Total cholesterol, change at 6 months (n=126,130)LDL, Change at 3 months (n=92,89)LDL, Change at 6 months (n=91,99)HDL, change at 3 months (n=97, 101)HDL, change at 6 months (n=102,112)Triglycerides,change at 3 months (n=104,108)Triglycerides,change at 6 months (n=109,117)LDL/HDL ratio,change at 3 months (n=91,89)LDL/HDL ratio,change at 6 months (n=91, 99)
Daclizumab0.650.280.250.030.340.230.260.20-0.44-0.50
Placebo0.910.610.340.210.310.200.460.44-0.12-0.14

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Median Time to First Acute Rejection Episode Within the First 6 Months and 12 Months PT

The median time to first acute rejection episode within first 6 months and 12 months PT was reported. (NCT00048165)
Timeframe: Within 6 months and 12 months PT

,
Interventiondays (Median)
Within 6 months (n= 77, 104)Within 12 months (n=97, 116)
Daclizumab6196
Placebo2126

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Number of Acute Rejection Episodes Per Participant Within the First 6 Months and 12 Months PT

The number of participants with 0,1, 2, 3 or 4 episodes at 6 and 12 months PT were reported. An episode of acute rejection was defined according to the date of positive biopsy of Grade IIIA or worse or the date of start of treatment for HDC, whichever came first. (NCT00048165)
Timeframe: Within 6 months and 12 months PT

,
Interventionparticipants (Number)
Within 6 months, 0 episodeWithin 6 months, 1 episodeWithin 6 months, 2 episodesWithin 6 months, 3 episodesWithin 6 months, 4 episodesWithin 12 months, 0 episodeWithin 12 months, 1 episodeWithin 12 months, 2 episodesWithin 12 months, 3 episodesWithin 12 months, 4 episodes
Daclizumab139631220119682333
Placebo114821921102901952

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Number of Participant Who Died Within 6 Months 12 Months and 3 Years PT

The survival of the graft and participants at 6,12 months and 3 years PT was reported (NCT00048165)
Timeframe: At 6 months, 12 months , 3 years PT

,
Interventionparticipants (Number)
Within 6 monthsWithin 12 monthsWithin 3 years
Daclizumab1621NA
Placebo1012NA

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Number of Participants Using Monomurab Cluster of Differentiation 3, Orthoclone Polyclonal Antithymocyte Globulin or Antilymphocyte Globulin in the First 6 Months and 12 Months PT

The number of participants who received monomurab Cluster of differentiation 3, orthoclone, polyclonal antithymocyte globulin or antilymphocyte globulin for treatment of biopsy proven rejection or HDC within 6 and 12 months PT were reported. (NCT00048165)
Timeframe: Within 6 months and 12 months PT

,
Interventionparticipants (Number)
Within 6 monthsWithin 12 months
Daclizumab1723
Placebo1921

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Number of Participants With Any Adverse Events and Any Serious Adverse Event, and Adverse Events Leading to Premature Withdrawal

An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Pre-existing conditions which worsened during this study were reported as AEs. A serious adverse event (SAE) is any untoward medical occurrence that at any dose results in death, are life threatening, requires hospitalization or prolongation of hospitalization or results in disability/incapacity, and congenital anomaly/birth defect. (NCT00048165)
Timeframe: Up to 12 months

,
Interventionparticipants (Number)
Any AEsAny SAE'sAny AEs leading to premature discontinuation
Daclizumab21410814
Placebo20710211

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Number of Participants With Malignancies and Opportunistic Infections

The opportunistic infections included infections with Cytomegalovirus, Aspergillus, Candida, Pneumocystis, Cryptococcus, Listeria, Herpes simplex, Herpes zoster. For malignancy, participants with any type of malignancy whose date of onset or diagnosis was after randomization was reported. (NCT00048165)
Timeframe: Up to 12 months

,
Interventionparticipants (Number)
Participants with malignanciesParticipants with opportunistic infections
Daclizumab1171
Placebo1180

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Number of Participants With Marked Laboratory Abnormalities: Biochemistry Parameters

A marked reference range was predefined by Roche. The marked reference range is broader than the standard reference range. Values falling outside the marked reference range (low or high) that also represent a defined change from Baseline were considered marked laboratory abnormalities (i.e. potentially clinically relevant). Biochemistry included blood urea nitrogen, creatinine, serum glutamic oxalacetic transaminase (SGOT), serum glutamic pyruvic transaminase (SGPT), gamma-glutamyl transferase (GGT), phosphorous, total bilirubin, direct bilirubin, total protein, albumin, glucose, alkaline phosphatase, low density lipoprotein (LDH), uric acid, carbon dioxide, magnesium, sodium, potassium, chloride, calcium, LDL, HDL, total cholesterol, and triglycerides. (NCT00048165)
Timeframe: Up to 12 months

,
Interventionparticipants (Number)
SGPT-high (n=200, 200)ALP-high (n=201, 200)SGOT-high (n=201, 200)GGT-high (n=180, 175)LDH-high (n=194, 191)Total bilirubin-high (n=201, 200)BUN-high (n= 210, 200)Creatinine-high (n=211, 203)Albumin-low (n=198,197)Total protein-high (n=195,196)Total protein-low(n=195,196)Cholesterol-high (n=174, 174)Triglycerides-high (n=164, 164)Carbondioxide-high (n=206, 197)Carbondioxide-low (n=206, 197)Chloride-high (n=211, 203)Chloride-low (n=211, 203)Potassium-high (n=211, 204)Potassium-low (n=211, 204)Sodium-high (n=211, 203)Sodium-low (n=211, 203)Calcium-low (n=207, 201)Glucose fasting-high (n=210, 203)Glucose fasting-low (n=210, 203)Phosphate-high (n=199, 194)Phosphate-low (n=199,194)Uric acid high (n=191, 188)
Daclizumab4815229053289366475853352712142742839283543223
Placebo457257454209564500794302153367211144273482620

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Number of Participants With Marked Laboratory Abnormalities: Hematology Parameters

A marked reference range was predefined by Roche. The marked reference range is broader than the standard reference range. Values falling outside the marked reference range (low or high) that also represent a defined change from Baseline were considered marked laboratory abnormalities (i.e. potentially clinically relevant). Hematology included hemoglobin, hematocrit, white blood cell count (WBC) with differential (including granulocytes or neutrophils, basophils, eosinophils, monocytes, lymphocytes), platelets, and erythrocyte count (NCT00048165)
Timeframe: Up to 12 months

,
Interventionparticipants (Number)
Hematocrit-high (n=210, 203)Hematocrit-low (n=210, 203)Hemoglobin-high (n=210, 204)Hemoglobin-low (n=210, 204)Platelets-high (n=210, 203)Platelets-low (n=210, 203)RBC-high (n=209, 203)RBC-low (n=209, 203)Basophils-high (n=199, 195)Eosinophils-high (n=199, 195)Lymphocytes-high (n=199, 198)Lymphocytes-low (n=199, 198)Monocytes-high (n=199, 198)Monocytes-low (n=199, 198)Neutrophils-low (n= 199, 198)WBC-high (n=207, 201)WBC-low (n=207, 201)
Daclizumab011601072301116402157514336143
Placebo011701121222106402157620335729

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Number of Participants With Worst ISHLT Biopsy Grade Within First 6 Months and 12 Months PT

The number of participants with Worst International Society of Heart and Lung Transplant (ISHLT) grade within first 6 months and 12 months PT were reported. ISHLT is a standardized grading method to determine the acute cellular rejection on endomyocardial biopsy ; where 0= no rejection, IA= focal (perivascular or interstitial) infiltrate without necrosis, IB= diffuse but sparse infiltrate without necrosis, II=one focus only with aggressive infiltration and/or focal myocyte damage, IIIA=multifocal aggressive infiltrates and/or myocyte damage, IIIB= diffuse inflammatory process with necrosis, IV= diffuse aggressive polymorphous and/or infiltrate and/or edema and/or hemorrhage and/or vasculitis, with necrosis (NCT00048165)
Timeframe: Within 6 months and 12 months PT

,
Interventionparticipants (Number)
Within 6 months, Grade 0Within 6 months, Grade IAWithin 6 months, Grade IBWithin 6 months, Grade IIWithin 6 months, Grade IIIAWithin 6 months, Grade IIIBWithin 6 months, Grade IVWithin 12 months, Grade 0Within 12 months, Grade IAWithin 12 months, Grade IBWithin 12 months, Grade IIWithin 12 months, Grade IIIAWithin 12 months, Grade IIIBWithin 12 months, Grade IV
Daclizumab96426554881756225163111
Placebo551283874151439214785151

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Cumulative Incidence of a Severe/Life-threatening/Fatal Infections

(NCT01002742)
Timeframe: Year 1

Interventionpercentage of participants (Number)
Placebo42.9
Mycophenolate Mofetil44.5

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Disease-Free Survival (DFS) Post-Randomization

DFS includes death or progression/relapse of malignancy (NCT01002742)
Timeframe: Year 1

Interventionpercentage of participants (Number)
Placebo63
Mycophenolate Mofetil53.9

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Incidence of Chronic GVHD

(NCT01002742)
Timeframe: 12 months post-randomization

Interventionpercentage of participants (Number)
Placebo43.3
Mycophenolate Mofetil41.5

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Incidence of Cytomegalovirus (CMV) Reactivation

(NCT01002742)
Timeframe: Year 1

Interventionpercentage of participants (Number)
Placebo39
Mycophenolate Mofetil44

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Incidence of Epstein-Barr Virus (EBV)-Associated Lymphoma

(NCT01002742)
Timeframe: 12 months

Interventionparticipants (Number)
Placebo4
Mycophenolate Mofetil6

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Incidence of GVHD Flares Requiring Increased Therapy

Flares are defined as any progression of acute GVHD after an initial response (i.e., earlier CR or PR) that requires re-escalation of steroid dosing, or initiation of additional topical or systemic therapy. (NCT01002742)
Timeframe: Day 90

Interventionparticipants (Number)
Placebo16
Mycophenolate Mofetil8

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Incidence of Systemic Infections

Number of participants that experienced at least one infection. (NCT01002742)
Timeframe: 6 Months

Interventionparticipants (Number)
Placebo77
Mycophenolate Mofetil81

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Incidence of Topical/Non-absorbable Therapy

(NCT01002742)
Timeframe: Day 56

Interventionparticipants (Number)
Placebo81
Mycophenolate Mofetil77

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Cumulative Steroid Dose

The cumulative steroid dose for each patient will be calculated by adding the doses (end of each week's dose) for each of the first four weeks of treatment, divided by the number of days of survival during this interval. The cumulative steroid dose was calculated for all patients per treatment arm and compared. (NCT01002742)
Timeframe: Days 28 and 56

,
Interventionmg/kg (Number)
Day 28Day 56
Mycophenolate Mofetil0.600.17
Placebo0.630.20

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GVHD-free Survival

Success is defined as alive and free of GVHD at day 56 after randomization, all others are considered to be a study failure. (NCT01002742)
Timeframe: Day 56

,
Interventionparticipants (Number)
GVHD freeStudy Failure
Mycophenolate Mofetil6947
Placebo6059

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Overall GVHD-free Survival Post-randomization

(NCT01002742)
Timeframe: Months 6 and 12

,
Interventionpercentage of participants (Number)
6 Months12 Months
Mycophenolate Mofetil72.057.8
Placebo73.464.7

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Percentage of Surviving Participants With Complete Response (CR)

CR is defined as a score of 0 for the GVHD grading in all evaluable organs. (NCT01002742)
Timeframe: Days 14, 28, and 56

,
Interventionpercentage of participants (Number)
Day 14Day 28Day 56
Mycophenolate Mofetil4446.660.3
Placebo49.644.553.8

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Blood CD4+ T Cells Per mm^3 Blood

Frequency of participants with any time point with <200 CD4+ T cells per mm^3 from 4 sampled time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months

Interventionparticipants (Number)
Mycophenolate Mofetil0

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Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 Effector Memory CD4+ T Cells Over 12 Months

Regression slope of change in cell-associated HIV DNA (ca-DNA) as measured by multiplexed digital droplet PCR in study participants on MMF calculated from 3 time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months

Interventionlog10 caDNA copies per 10^6 T-cells/week (Mean)
Mycophenolate Mofetil0.001

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Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 T Cells Over 12 Months

Regression slope of change in cell-associated HIV DNA (ca-DNA) as measured by multiplexed digital droplet PCR in study participants on MMF calculated from 4 time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months

Interventionlog10 caDNA copies per 10^6 T-cells/week (Mean)
Mycophenolate Mofetil-0.00033

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Change in Cell-associated Intact HIV DNA (Ca-iDNA) Levels Per 10^6 T Cells Over 12 Months

Regression slope of change in cell-associated intact HIV DNA (ca-iDNA) as measured by multiplexed digital droplet PCR in study participants on MMF calculated from 4 time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months

Interventionlog10 caDNA copies per 10^6 T-cells/week (Mean)
Mycophenolate Mofetil.0024

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Incidence of Opportunistic Infection

Number of participants experiencing opportunistic infection (NCT03262441)
Timeframe: 12 months

Interventionparticipants (Number)
Mycophenolate Mofetil0

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Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe?

Feasibility will be met with the following conditions: the patient has the transplant, is assessed for the safety endpoint, and survives one year. The safety monitoring plan is included to monitor graft failure (day 60), grade 2-4 acute graft versus host disease (day100), 6 month mortality (day 180), and chronic graft versus host disease (day 180). (NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant18

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Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant

CBC drawn daily with a WBC differential once the total WBC is greater than 100 until ANC > 500 for three days or two consecutive measurements over a three day period; then CBC drawn weekly with differential. (NCT02224872)
Timeframe: 1 year

Interventiondays (Median)
Bone Marrow Transplant18.85

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Number of Participants With Grade II-IV or Grade III-IV Acute GVHD

Participants were graded during clinical visits based on evidence and extent of skin rash, liver involvement, and GI tract involvement (NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant17

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Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant

Donor chimerism will be measured in the peripheral blood around day 30 and day 60. Patients with >5% donor chimerism around day 60 will be considered as having engrafted. (NCT02224872)
Timeframe: 60 days

InterventionParticipants (Count of Participants)
Bone Marrow Transplant7

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Number of Patients That Have Survived at One Year

(NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant18

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Number of Patients With Primary or Secondary Graft Failure Following Transplant

"Graft failure: < 5% donor chimerism in blood and/or bone marrow on ~Day 30 or after and on all subsequent measurements.~Primary graft failure: < 5% donor chimerism in blood and/or bone marrow by ~ Day 56 Secondary graft failure: achievement of > 5% donor chimerism, followed by sustained <5% donor chimerism in blood and/or bone marrow." (NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant17

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Participants That Were GVHD Free, Relapse Free Survival (GRFS)

(NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant14

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Participants With Chronic GVHD at One Year

(NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant3

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Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB)

Number of surviving patients at Day 100 post-transplant divided by number of patients undergone transplantation. (NCT00547196)
Timeframe: From transplant to Day 100 post-transplant

Interventionpercentage of surviving patients (Number)
Regimen I (FTBI, Cyclophosphamide, Fludarabine)100
Regimen III (TBI, Cyclophosphamide, Fludarabine)100
Regimen IV (Fludarabine, Melphalan)100

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Survival Rate at Day 180 After Allogeneic Transplant From Umbilical Cord Blood (UCB)

Number of surviving patients at Day 180 post-transplant divided by number of patients undergone transplantation. (NCT00547196)
Timeframe: From transplant up to Day 180 post-transplant

Interventionpercentage of surviving patients (Number)
Regimen I (FTBI, Cyclophosphamide, Fludarabine)60
Regimen III (TBI, Cyclophosphamide, Fludarabine)100
Regimen IV (Fludarabine, Melphalan)50

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Percentage of Participants With Efficacy Failure Through 12 Months Post Transplant

Efficacy failure was defined as BPAR, death, graft loss or lost to follow-up through 12 months post transplant. (NCT02921789)
Timeframe: 12 Months post transplant

InterventionPercentage of participants (Number)
SOC Regimen32.3
Bleselumab Regimen32.0

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Percentage of Participants With Recurrence of Focal Segmental Glomerulosclerosis (rFSGS) or Death or Graft Loss or Lost to Follow-up Through 3 Months Post Transplant

rFSGS was defined as nephrotic range proteinuria with a protein/creatinine ratio (≥ 3.0 g/g). Death, graft loss or lost to follow-up was imputed as rFSGS. (NCT02921789)
Timeframe: At 3 Months post transplant

InterventionPercentage of participants (Number)
SOC Regimen31.3
Bleselumab Regimen18.5

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Percentage of Participants With Biopsy Proven rFSGS Through 3, 6 and 12 Months Post-Transplant

Percentage of participants with biopsy-proven rFSGS determined by a blinded central review of images from electron microscopy (EM) and slides for light microscopy (LM) by an independent pathologist. (NCT02921789)
Timeframe: At 3, 6 and 12 Months post transplant

,
InterventionPercentage of participants (Number)
Month 3Month 6Month 12
Bleselumab Regimen31.830.429.2
SOC Regimen35.736.736.7

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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) Through 3, 6, and 12 Months Post Transplant

All episodes of kidney dysfunction based on clinical signs and symptoms were evaluated for possible BPAR. BPAR was confirmed if participants Banff criteria >=1. (NCT02921789)
Timeframe: At 3, 6 and 12 Months post transplant

,
InterventionPercentage of participants (Number)
Month 3Month 6Month 12
Bleselumab Regimen26.926.929.2
SOC Regimen20.020.024.1

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Percentage of Participants With rFSGS or Death or Graft Loss or Lost to Follow-up Through 6 and 12 Months Post Transplant

rFSGS was defined as nephrotic range proteinuria with a protein/creatinine ratio (≥ 3.0 g/g). Death, graft loss or lost to follow-up was imputed as rFSGS. (NCT02921789)
Timeframe: At 6 and 12 Months post transplant

,
InterventionPercentage of participants (Number)
Month 6Month 12
Bleselumab Regimen18.523.1
SOC Regimen31.335.5

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Cumulative Incidence of Systemic Infections

(NCT00224874)
Timeframe: Measured at Day 270

Interventionpercentage of participants (Number)
Etanercept47
Mycophenolate Mofetil44
Denileukin Diftitox62
Pentostatin57

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Number of Complete Response (CR) at Day 28 of Therapy

Complete response at day 28 after randomization. CR was defined as resolution of all signs and symptoms of Graft-Versus-Host Disease (GVHD) in all evaluable organs in comparison to Day 1 scoring. (NCT00224874)
Timeframe: Measured at Day 28

Interventionparticipants (Number)
Etanercept12
Mycophenolate Mofetil27
Denileukin Diftitox25
Pentostatin16

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Number of Patients With Acute Graft-versus-host Disease (GVHD) Flares at Day 90

Flares were defined as any increase in symptoms of or therapy for acute GVHD after an initial response (i.e., progression from an earlier CR or PR). (NCT00224874)
Timeframe: Measured at Day 90

Interventionparticipants (Number)
Etanercept16
Mycophenolate Mofetil12
Denileukin Diftitox15
Pentostatin15

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Number of Patients With Chronic Graft-versus-host Disease (GVHD)

Number of patients with limited and extensive chronic GVHD at 9 months (NCT00224874)
Timeframe: Measured at 9 months

Interventionparticipants (Number)
Etanercept11
Mycophenolate Mofetil19
Denileukin Diftitox15
Pentostatin12

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Proportion of Treatment Failure

(NCT00224874)
Timeframe: Measured at Day 56

Interventionpercentage of participants (Number)
Etanercept24
Mycophenolate Mofetil9
Denileukin Diftitox26
Pentostatin29

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Number of Partial Response (PR), Mixed Response (MR), and Progression

Partial response, mixed response, and progression at Day 28 after randomization. Partial response was defined as improvement in one or more organs involved with Graft-Versus-Host Disease (GVHD) symptoms without progression in others. Mixed response was defined as improvement in one or more organs with deterioration in another organ manifesting symptoms of GVHD or development of symptoms of GVHD in a new organ. Progression was defined as deterioration in at least one organ without any improvement in others. (NCT00224874)
Timeframe: Measured at Day 28

,,,
Interventionparticipants (Number)
Partial ResponseMixed ResponseProgression
Denileukin Diftitox303
Etanercept1037
Mycophenolate Mofetil841
Pentostatin1024

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Number of Patients Discontinuing Immune Suppression Without Flare

Immunosuppression discontinuation was defined as the discontinuation of corticosteroids and all additional immunosuppressives, except cyclosporine or tacrolimus, for treatment of acute GVHD without subsequent flare by Day 90 post-initiation of therapy and later by discontinuation of all immunosuppressive medications, including cyclosporine or tacrolimus. (NCT00224874)
Timeframe: Measured at Days 90, 180, and 270 post-treatment

,,,
Interventionparticipants (Number)
Day 90Day 180Day 270
Denileukin Diftitox5810
Etanercept71216
Mycophenolate Mofetil41317
Pentostatin2810

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Number of Patients Surviving at 6 and 9 Months Post Randomization

(NCT00224874)
Timeframe: Measured at 6 and 9 months

,,,
Interventionparticipants (Number)
Month 6Month 9
Denileukin Diftitox2824
Etanercept2622
Mycophenolate Mofetil3229
Pentostatin2421

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Acute Graft vs Host Disease (GvHD)

"The incidence of acute GvHD after transplantation was assessed per Glucksberg GvHD grade, a compound scale based on the following combinations of disease stages.~Skin Stages~0: No rash~1: Maculopapular (MP) rash <25% of body surface area~2: MP rash on 25-50% of body surface area~3: Generalized erythroderma (ED)~4: Generalized ED with bullous formation and desquamation~Liver Stages (Bilirubin in mg/dL)~0: <2~1: 2-3~2: 3.01-6~3: 6.01-15.0~4: >15~Gastrointestinal (GI) Stages (diarrhea)~0: None or < 500 mL/day~1: 500-999 mL/day~2: 1000-1499 mL/day~3: >1500 mL/day~4: Severe abdominal pain, with or without ileus~Glucksberg Overall grade~Grade 1: Skin 1/2; GI 0; Liver 0; Karnofsky performance scale (KPS) 90-100%~Grade 2: Skin 1-3; GI 1; Liver 1; KPS 70-80~Grade 2: Skin 2/3; GI 2/3; Liver 2-4; KPS 50-60~Grade 4: Skin 2-4; GI 2-4; Liver 2-4; KPS 30-40" (NCT00185640)
Timeframe: 100 days post-transplant

Interventionpercentage of participants (Number)
Non-myeloablative Transplantation2.7

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Acute Graft vs Host Disease (GvHD), All Evaluable

"The incidence of acute GvHD after transplantation was assessed per Glucksberg GvHD grade, a compound scale based on the following combinations of disease stages.~Skin Stages~0: No rash~1: Maculopapular (MP) rash <25% of body surface area~2: MP rash on 25-50% of body surface area~3: Generalized erythroderma (ED)~4: Generalized ED with bullous formation and desquamation~Liver Stages (Bilirubin in mg/dL)~0: <2~1: 2-3~2: 3.01-6~3: 6.01-15.0~4: >15~Gastrointestinal (GI) Stages (diarrhea)~0: None or < 500 mL/day~1: 500-999 mL/day~2: 1000-1499 mL/day~3: >1500 mL/day~4: Severe abdominal pain, with or without ileus~Glucksberg Overall grade~Grade 1: Skin 1/2; GI 0; Liver 0; Karnofsky performance scale (KPS) 90-100%~Grade 2: Skin 1-3; GI 1; Liver 1; KPS 70-80~Grade 2: Skin 2/3; GI 2/3; Liver 2-4; KPS 50-60~Grade 4: Skin 2-4; GI 2-4; Liver 2-4; KPS 30-40" (NCT00185640)
Timeframe: 100 days post-transplant

Interventionpercentage of participants (Number)
Non-myeloablative Transplantation11

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Incidence of Relapse

Reports the overall rate of disease relapse, occurring any time within 3 years after transplant (NCT00185640)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Non-myeloablative Transplantation53

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Event-free Survival (EFS)

Reports the number and proportion of participants who neither died due to any cause nor experienced relapse. (NCT00185640)
Timeframe: 3 and 5 years

InterventionParticipants (Count of Participants)
3 years5 years
Non-myeloablative Transplantation4438

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Overall Survival (OS)

(NCT00185640)
Timeframe: 3 and 5 years

Interventionpercentage of participants (Number)
3 years5 years
Non-myeloablative Transplantation7064

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Disease-free Survival

Defined as being alive and in remission by < 5% blasts by bone marrow morphology for patients with myeloid malignancies and CT or PET imaging for patients with lymphoid malignancies at the time of the assessment (NCT01028716)
Timeframe: 3 years from the date of transplant

Interventionpercent of participants (Number)
Treatment (Nonmyeloablative HCT, TBI)40

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Incidence of Grades III/IV Acute Graft Versus Host Disease

Grading determined by organ system stages. Grade III/IV acute graft versus host disease is defined as skin: stage IV, liver: stages II-IV, and/or gastrointestinal tract: stages II-IV. (NCT01028716)
Timeframe: At day 84

Interventionpercent (Number)
Treatment (Nonmyeloablative HCT, TBI)82

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Incidence of Primary Graft Failure

Defined as < 5% donor CD3 chimerism. Chimerism will be measured by short tandem repeat-polymerase chain reaction on peripheral blood sorted into CD3 and CD33 cell fractions. (NCT01028716)
Timeframe: At day 84

InterventionParticipants (Count of Participants)
Treatment (Nonmyeloablative HCT, TBI)0

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Non-relapse Mortality at 1 Year

Cumulative incidence of death without evidence of disease progression at 1 year (NCT01028716)
Timeframe: Up to 1 year

Interventionpercent of participants (Number)
Treatment (Nonmyeloablative HCT, TBI)22

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Number of Platelet Transfusions

Number platelet transfusions given to the patient between day 0 and day 100 post transplant (NCT01028716)
Timeframe: Day 0-100

Interventiontransfusions (Median)
Treatment (Nonmyeloablative HCT, TBI)6

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Number of Red Blood Cell Transfusions

Number of units of RBCs given to the patient between day 0 and day 100 post transplant (NCT01028716)
Timeframe: Day 0-100

Interventiontransfusions (Median)
Treatment (Nonmyeloablative HCT, TBI)8

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Percentage of Participants With Chronic Graft Versus Host Disease

Scored according to the National Cancer Institute criteria. Mild-to-severe chronic GVHD at 2 years. (NCT01028716)
Timeframe: Up to 2 years post-transplant

Interventionpercentage of participants (Number)
Treatment (Nonmyeloablative HCT, TBI)26

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Point Estimate of Overall Survival at 3 Years

Kaplan Meier estimate of the percentage of participants with overall survival at 3 years (NCT01028716)
Timeframe: 3 years

Interventionpercent (Number)
Treatment (Nonmyeloablative HCT, TBI)45.3

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Relapse of Malignancy After Transplantation

Defined by either morphological or cytogenetic evidence of acute leukemia consistent with pre-transplant features, or radiologic evidence of lymphoma progression. When in doubt, the diagnosis of recurrent or progressive lymphoma should be documented by tissue biopsy. (NCT01028716)
Timeframe: Up to 7 years

Interventionpercent (Number)
Treatment (Nonmyeloablative HCT, TBI)29

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Time to Neutrophil Recovery

Achievement of an absolute neutrophil count greater or equal to 500/mm^3 for three consecutive measurements on different days. The first of the three days will be designated the day of neutrophil recovery. (NCT01028716)
Timeframe: Up to day 84 post-transplant

Interventiondays (Median)
Treatment (Nonmyeloablative HCT, TBI)16

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Time to Platelet Recovery

The first day of a sustained platelet count > 20,000/mm^3 with no platelet transfusions in the preceding seven days. (NCT01028716)
Timeframe: Up to day 84 post-transplant

Interventiondays (Median)
Treatment (Nonmyeloablative HCT, TBI)23

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Toxicity of Treatment Regimen Determined by Number of Adverse Events Per Organ System

Assessed by Common Terminology Criteria for Adverse Events version 3.0. The incidence of all adverse events greater or equal to grade 3 was determined. (NCT01028716)
Timeframe: Up to day 90

Interventionevents (Number)
CardiacFeverRashGastrointestinalInfectionsCMV ReactivationFebrile NeutropeniaMetabolic/laboratoryMusculoskeletalNeurologicPainPulmonaryRenal/Genitourinary
Treatment (Nonmyeloablative HCT, TBI)139417552625243561010

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Mean Stimulated C-peptide Area Under the Curve

The primary outcome is the area under the stimulated C-peptide curve (AUC) based on data collected at time 0 to 2 hours of a 4-hour mixed meal glucose tolerance test (MMTT) conducted at the primary endpoint visit. The timed measurements are done at: 0, 15, 30 60, 90, and 120 minutes. (NCT00100178)
Timeframe: 2 years

Interventionpmol/ml (Geometric Mean)
MMF and DZB0.28
Placebo Control0.27
MMF Alone0.25

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Acute Graft-Versus-Host Disease (GVHD)

(NCT00709592)
Timeframe: 2 year rate (%)

Interventionpercentage of participant (Number)
A:Thymoglobulin: 1.7 mg/kg/Day27.2
B:Thymoglobulin: 2.5 mg/kg/Day4.5

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Chronic Graft-Versus-Host Disease (GVHD)

(NCT00709592)
Timeframe: 2 year GVHD rate

Interventionpercentage of participants (Number)
A:Thymoglobulin: 1.7 mg/kg/Day23.8
B:Thymoglobulin: 2.5 mg/kg/Day31.8

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Donor Lymphocyte Infusion

(NCT00709592)
Timeframe: 2 year rate of DLI

Interventionpercentage of participants (Number)
A:Thymoglobulin: 1.7 mg/kg/Day8.9
B:Thymoglobulin: 2.5 mg/kg/Day45.5

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Engraftment of Donor Hematopoietic Stem Cells, as Measured by Time in Days to Neutrophil and Platelet Count Recovery Following Allogeneic PBSCT.

(NCT00709592)
Timeframe: Up to 52 weeks post transplant.

InterventionDays (Median)
A:Thymoglobulin: 1.7 mg/kg/Day12
B:Thymoglobulin: 2.5 mg/kg/Day12

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Event-free Survival

(NCT00709592)
Timeframe: 2 years

Interventionpercentage of participants (Number)
A:Thymoglobulin: 1.7 mg/kg/Day62.2
B:Thymoglobulin: 2.5 mg/kg/Day44.5

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Relapse

Patients with different disease relapses was determined according to current clinical standards based on the disease. For example, AML or MDS relapse is determined by a bone marrow biopsy. Multiple myeloma relapse requires a number of labs and/or biopsy to diagnose such as SPEP, UPEP, immunofixation, serum and urine light chains. In lymphoma disease is followed using CT and/or PET scans. (NCT00709592)
Timeframe: 2 year relapse rate (%)

InterventionPercent patients relapsing (Number)
A:Thymoglobulin: 1.7 mg/kg/Day28
B:Thymoglobulin: 2.5 mg/kg/Day50

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Survival

(NCT00709592)
Timeframe: 2-year survival rate (%)

Interventionpercentage of patient surviving (Number)
A:Thymoglobulin: 1.7 mg/kg/Day71.3
B:Thymoglobulin: 2.5 mg/kg/Day62.4

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The Comparison of Functional Immune Reconstitution at 6-9 Months Following Transplant as Measured by Antibody Response to Vaccination With Inactivated Hepatitis A or B Vaccine.

A positive test result will indicate immune reconstitution, while a negative test results will indicate lack of immune reconstitution. Participants not done (ND) will be counted with the negative (Neg). (NCT00709592)
Timeframe: Up to 9 months following transplant

,
Interventionparticipants (Number)
PositiveNegative/Not Done
A:Thymoglobulin: 1.7 mg/kg/Day811
B:Thymoglobulin: 2.5 mg/kg/Day319

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Maximum Plasma Concentration (Cmax) of GSK1070806

Serial blood samples were collected to evaluate PK of GSK1070806 at Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion. Log-transformed geometric mean and 95% confidence interval have been presented. (NCT02723786)
Timeframe: Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion

InterventionLog (nanograms per milliliter) (Geometric Mean)
GSK1070806 3 mg/kg IV36315.1

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Number of Participants Having Infections

Number of participants having infections were summarized. (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
GSK1070806 3 mg/kg IV5

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Number of Participants Requiring Dialysis During the First 7 Days Post Transplant

The requirement of dialysis (except as needed for hyperkalaemia during the first 24 hours [hrs]) were used to assess the frequency of delayed graft function (DGF) in donation after circulatory death (DCD) renal transplant recipients treated with GSK1070806. The 'Analysis Population' (AP) is defined as participants in the 'All Subjects' Population who have been declared to have DGF or have reached 7 days. (NCT02723786)
Timeframe: Up to Day 7

InterventionParticipants (Count of Participants)
GSK1070806 3 mg/kg IV4

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Number of Participants Who Are Dialysis Independent at Visits up to 12 Months Post-transplant

Number of participants who are dialysis independent at visits up to 12 months post transplant was evaluated to assess the effect of GSK1070806 on dialysis dependency and graft survival. (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
GSK1070806 3 mg/kg IV2

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Number of Participants With Dialysis Events in the First 30 Days Post-transplant

Number of participants with dialysis events in the first 30 days post transplant was evaluated to assess the effect of GSK1070806 on dialysis dependency and graft survival. The AP Population is defined as participants having Baseline and at least one post-Baseline assessment. (NCT02723786)
Timeframe: Up to 30 days

InterventionParticipants (Count of Participants)
GSK1070806 3 mg/kg IV5

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Number of Participants With Episodes of Biopsy-proven Acute Rejection

Number of participants with episodes of biopsy-proven acute rejection were evaluated to assess the effect of GSK1070806 on acute rejection risk, and rejection/Pharmacodynamic (PD) biomarkers. (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
GSK1070806 3 mg/kg IV1

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Area Under the Plasma Concentration Time Curve (AUC) From Time 0 to the Last Measurable Concentration (AUC[0-t]) and AUC From Time 0 to Infinite Time (AUC[0-inf]) of GSK1070806

Blood samples were collected to evaluate PK of GSK1070806 at Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion. Log-transformed geometric mean and 95% confidence interval have been presented. (NCT02723786)
Timeframe: Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion

InterventionLog (Hour*nanograms per milliliter) (Geometric Mean)
AUC (0-t), n=7AUC (0-inf), n=6
GSK1070806 3 mg/kg IV26131338.241032450.7

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Baseline and Change From Baseline in Serum Levels of Free, Total, and GSK1070806 Bound Interleukin 18 (IL-18) Over Time Post-transplant

IL-18 is itself rapidly secreted from intracellular stores following inflammasome mediated-activation. The appearance of IL-18 marks the initiation of the inflammatory response leading to further injury. Blood samples were collected at indicated time points to assess serum levels of free, total, and GSK1070806 bound IL-18. Baseline value was the latest pre-dose assessment value. Change from Baseline was post Baseline value minus Baseline value. (NCT02723786)
Timeframe: Baseline and at 0.75 hours, 4-8 hours, Day 1, Day 2, Day 30, Day 90, 6 months and 12 months post reperfusion

InterventionPicograms per milliliter (Mean)
Serum Free IL-18, Baseline (pre-operative), n=5Serum Free IL-18, Day 0, 0.75 hour, n=5Serum Free IL-18, Day 0, 4-8 hour, n=5Serum Free IL-18, Day 1, n=5Serum Free IL-18, Day 2, n=4Serum Free IL-18, Day 30, n=4Serum Free IL-18, Day 90, n=2Serum Bound IL-18, Baseline (pre-operative), n=5Serum Bound IL-18, Day 0, 0.75 hour, n=5Serum Bound IL-18, Day 0, 4-8 hour, n=5Serum Bound IL-18, Day 1, n=5Serum Bound IL-18, Day 2, n=4Serum Bound IL-18, Day 30, n=4Serum Bound IL-18, Day 90, n=2Serum Total IL-18, Baseline (pre-operative), n=7Serum Total IL-18, Day 0, 0.75 hour, n=6Serum Total IL-18, Day 0, 4-8 hour, n=6Serum Total IL-18, Day 1, n=6Serum Total IL-18, Day 2, n=5Serum Total IL-18, Day 30, n=6Serum Total IL-18, Day 90, n=7Serum Total IL-18, 6 months, n=7Serum Total IL-18, 12 months, n=6
GSK1070806 3 mg/kg IV26.840-22.620-22.540-23.890-5.263-27.925-2.25021.156362.084314.864472.204485.740617.543946.020130.6857572.3333576.3667636.5667660.66001175.50001423.54291303.71431091.0833

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Number of Participants Having Any Abnormal Clinical Chemistry Results of Potential Clinical Importance

Blood samples were collected to evaluate clinical chemistry parameters. Number of participants with abnormal chemistry results of potential clinical importance (high or low) in any of these parameters at any time post Baseline visit have been presented. PCI (high or low) was considered if albumin (low<30), calcium (low<2, high>2.75), creatinine (high: CHB>44.2 increase), glucose (low<3, high>9), magnesium (low<0.5, high>1.23), phosphorus (low<0.8, high>1.6), potassium (low<3, high>5.5), sodium (low: 130, high>150), Total carbon dioxide (CO2) (low:18, high>32), Alanine aminotransferase (ALT) (high>=2*upper limit of normal [ULN]), Aspartate aminotransferase (AST) (high: >=2*ULN), Alkaline phosphatase (ALP) (high:>=2*ULN), Total bilirubin (high: >2*ULN), Total bilirubin+ALT (high: 1.5*ULN total bilirubin with >=2*ULN ALT). (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
Albumin, LowCalcium, LowGlucose, HighPotassium, LowPotassium, HighTotal Bilirubin, HighSodium, LowALT, HighALP, HighAST, High
GSK1070806 3 mg/kg IV6751312111

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Number of Participants Having Any Abnormality in Hematology Results of Potential Clinical Importance

Blood samples were collected to evaluate hematology parameters. Number of participants with abnormality in any hematology parameter results of potential clinical importance (high or low) observed at any time post Baseline are presented. PCI (high or low) was considered if hematocrit (high:>0.54;low:change from baseline [CFB] 0.075 decrease), hemoglobin (high:180; low: CFB 25 decrease), lymphocytes (low: 0.8), neutrophil count (low: 1.5), platelet count (low: 100; high: 550), White blood cells (low: 3; high:20). (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
Lymphocytes, LowHematocrit, HighWhite Blood Cells, HighWhite Blood Cells, LowPlatelet Count, LowTotal neutrophils, Low
GSK1070806 3 mg/kg IV711111

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Number of Participants Having Any Abnormality of Potential Clinical Importance of Vital Signs Results

Vital signs parameters included analysis of systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR) and body temperature. Number of participants with any abnormality of potential clinical importance (high or low) in any of these vitals signs at any time post Baseline visit have been presented. PCI (high or low) was considered if SBP (low: <85, high:>160), DBP (low: <45, high>100), HR (low: <40, high: >110) and temperature (low: <35.5, high: >37.5). (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
SBP, HighSBP, LowDBP, HighDBP, LowHR, HighTemperature, HighTemperature, Low
GSK1070806 3 mg/kg IV5121112

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Number of Participants in the First 7 Days With: Primary Non Function, Functional DGF, Intermediate Graft Function, Immediate Graft Function

Number of participants in the first 7 days with primary non function, functional DGF, intermediate graft function and immediate graft function were evaluated to access graft function in DCD renal transplant recipients treated with GSK1070806. The AP Population is defined as participants in the 'All Subjects' Population who have been declared to have DGF or have reached 7 days. (NCT02723786)
Timeframe: Up to Day 7

InterventionParticipants (Count of Participants)
Primary Non Function3 day Functional DGF7 day Functional DGF3 day Intermediate Graft Function7 day Intermediate Graft Function3 day Immediate Graft Function7 day Immediate Graft Function
GSK1070806 3 mg/kg IV1350110

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Number of Participants With Adverse Event (AE) and Serious Adverse Event (SAE)

AE is any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Any untoward event resulting in death, life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, congenital anomaly/birth defect or any other situation according to medical or scientific judgment were categorized as SAE. (NCT02723786)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
Any AEAny SAE
GSK1070806 3 mg/kg IV76

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Serum Concentrations of GSK1070806

Serial blood samples were collected to evaluate PK of GSK1070806 at Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion. PK Population included participants in the 'All Subjects' Population for whom a serum PK sample is obtained and analyzed for GSK1070806. (NCT02723786)
Timeframe: Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion

InterventionNanograms per milliliter (Mean)
Pre-operative, n=70.75 hours, n=64-8 hours, n=624 hours, n=6168 hours, n=5Day 30, n=6Day 90, n=76 months, n=712 months, n=6
GSK1070806 3 mg/kg IV0.058783.360033.350933.328260.017366.75047.01083.419.2

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Serum Creatinine at Baseline and Change From Baseline Over Time Post Transplant

Blood samples were collected to measure serum creatinine at the indicated timepoints to assess graft function in DCD renal transplant recipients treated with GSK1070806. Baseline value was the latest pre-dose assessment value. Change from Baseline was post Baseline value minus Baseline value. NA indicates data is not available as standard deviation could not be calculated due to n=1. The AP Population is defined as participants having Baseline and at least one post-Baseline assessment. (NCT02723786)
Timeframe: Baseline and up to 12 months

InterventionMicromoles per liter (Mean)
Screening, n=7Day 0, n=7Day 1, n=7Day 2, n=7Day 3, n=7Day 4, n=7Day 5, n=6Day 6, n=6Day 7, n=6Day 8, n=5Day 9, n=3Day 10, n=3Day 11, n=3Day 12, n=2Day 13, n=2Day 14, n=2Day 15, n=2Day 16, n=2Day 17, n=2Day 18, n=2Day 19, n=2Day 20, n=2Day 21, n=1Day 22, n=1Day 23, n=1Day 24, n=1Day 25, n=1Day 26, n=1Day 27, n=1Day 28, n=1Day 30, n=7Day 90, n=76 months, n=712 months, n=6
GSK1070806 3 mg/kg IV679.0-39.3-44.7-99.0-36.7-104.6-57.2-75.7-43.5-150.8-24.0-53.0-97.0110.038.04.0-51.0-61.5-107.5-145.0-173.5-183.5-117.0-91.0-95.0-115.0-152.0-155.0-175.0-191.0-478.3-489.9-467.4-490.5

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Serum Interferon Gamma-induced Protein 10 (IP-10) and Serum Monokine Induced Gamma Interferon (Mig) Levels at Baseline and Change From Baseline Over Time Post Transplant

The interferon-gamma -inducible chemokine IP10 and the interferon-gamma -inducible chemokine Mig have been identified as an early predictive marker of antibody-mediated kidney graft rejection. Baseline value was the latest pre-dose assessment value. Change from Baseline was calculated as post Baseline value minus Baseline value. (NCT02723786)
Timeframe: Baseline and at 0.75 hours, 4-8 hours, Day 1, Day 2, Day 30, Day 90, 6 months and 12 months post reperfusion

InterventionPicograms per milliliter (Mean)
IP-10, Baseline, n=7IP-10, Day 0, 0.75 hour, n=6IP-10, Day 0, 4-8 hour, n=6IP-10, Day 1, n=6IP-10, Day 2, n=5IP-10, Day 30, n=6IP-10, Day 90, n=7IP-10, 6 months, n=7IP-10, 12 months, n=5Mig, Baseline, n=7Mig, Day 0, 0.75 hour, n=6Mig, Day 0, 4-8 hour, n=6Mig, Day 1, n=6Mig, Day 2, n=5Mig, Day 30, n=6Mig, Day 90, n=7Mig, 6 months, n=7Mig, 12 months, n=5
GSK1070806 3 mg/kg IV518.83817-48.36607-262.30099-214.27224-91.07498-215.96831221.97286145.05039241.29317175.76865-14.02145-49.28436-67.61716-133.99600-159.17646-78.69081-43.68148-30.52711

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Urine Volume at Baseline and Change From Baseline Over Time Post Transplant

Urine volume at Baseline and over time post transplant was measured to assess graft function in DCD renal transplant recipients treated with GSK1070806. Baseline value was the latest pre-dose assessment value. Change from Baseline was post Baseline value minus Baseline value. All Subjects Population comprised of participants who received the dose of study medication. (NCT02723786)
Timeframe: Baseline (Pre-operative) and up to Day 28

InterventionLiter (Mean)
Baseline (Pre-operative), n=5Day 0, n=4Day 1, n=5Day 2, n=5Day 3, n=5Day 4, n=5Day 5, n=4Day 6, n=4Day 7, n=4Day 8, n=3Day 9, n=2Day 10, n=2Day 11, n=2Day 12, n=1Day 13, n=1Day 14, n=1Day 15, n=1Day 16, n=1Day 17, n=1Day 18, n=1Day 19, n=1Day 20, n=1Day 21, n=1Day 22, n=1Day 23, n=1Day 24, n=1Day 25, n=1Day 26, n=1Day 27, n=1Day 28, n=1
GSK1070806 3 mg/kg IV0.6700-0.51500.58621.28200.88401.14800.82701.11681.29851.20901.21350.85850.49851.40001.69001.18001.55001.85001.50001.90001.60001.25000.75001.05001.15001.45002.05002.00001.55001.4600

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Acute Rejection Per Kidney Biopsy (Banff Grading Criteria)

(NCT00556933)
Timeframe: Two years

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus7
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus4
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil8
Divided-dose rATG(1.5mg/kgx4), Sirolimus/Mycophenolate Mofetil9

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Acute Tubular Necrosis (ATN) Rate, Defined as the Requirement for Dialysis Within 7 Days Post-transplantation.

(NCT00556933)
Timeframe: Seven days

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus3
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus1
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil6
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil2

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Graft Survival

Graft failure = permanent return of patient to dialysis. (NCT00556933)
Timeframe: Two years

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus0
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus1
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil2
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil0

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Lymphoid Cell Sub-type CD3 Absolute Numbers

(NCT00556933)
Timeframe: One year

InterventionCD3 Cell Numbers/mm^3 (Mean)
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus446
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus375
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil392
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil266

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New-onset Diabetes and Hyperglycemia After Transplantation (NODAT)

(NCT00556933)
Timeframe: Six months

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus10
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus7
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil5
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil12

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New-onset Polyomavirus (BK Virus) Disease Per Kidney Biopsy

(NCT00556933)
Timeframe: Two years

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus1
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus2
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil0
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil2

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Patient Survival

(NCT00556933)
Timeframe: Two years

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus0
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus1
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil0
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil1

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Ratio of CD4/CD8 Lymphoid Cells

(NCT00556933)
Timeframe: One year

InterventionRatio of cell counts (Mean)
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus0.84
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus0.84
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil0.98
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil1.01

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Requirement for Additional Immunosuppression (Such as Corticosteroids, Antimetabolites or Other Immunosuppressive Agents)

(NCT00556933)
Timeframe: Two years

Interventionparticipants (Number)
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus7
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus5
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil6
Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil11

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Safety Profile

Number of events: cytomegalovirus (CMV) disease, opportunistic infections (bacteremia, abscess, pneumonia, fungal), Post-transplantation Lymphoproliferative Disorder (PTLD), wound healing problems within 30 days, and lymphoceles. (NCT00556933)
Timeframe: Two years

InterventionEvents (Number)
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus11
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus14
Ingle-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil10
Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil17

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Average of Renal Function

Calculated Glomerular Filtration Rate (GFR) by using the abbreviated MDRD (aMDRD) formula and patient serum creatinine and demographic data; averaged values from months four through 24. (NCT00556933)
Timeframe: Two years

,,,
Interventionml/min/1.73m2 (Mean)
Average GFR, months 1-3Average GFR, months 4-6Average GFR, months 7-9Average GFR, months 10-12Average GFR, months 13-15Average GFR, months 16-18Average GFR, months 19-21Average GFR, months 22-24
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus50.157.355.055.657.356.354.857.0
Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil50.454.855.856.658.556.656.857.6
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus50.457.354.855.856.857.656.656.9
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil53.660.261.057.461.862.360.262.9

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Chronic Allograft Nephropathy (Cumulative Calcineurin-inhibitor Nephrotoxicity/Transplant Nephropathy) Per Protocol Surveillance Kidney Biopsies (Banff Grading Criteria).

Protocol kidney biopsies collected at approximately 12 and 24 months were scored by a transplant renal pathologist blinded to treatment group assignment for evidence of rejection, BK virus nephropathy, antibody-mediated rejection, recurrent disease, inflammation, and Banff 2005 categories of chronic renal injury. Chronic injury categories were arteriolar hyaline thickening (ah), allograft glomerulopathy (cg), interstitial fibrosis (ci), tubular atrophy (ct), and vascular fibrous intimal thickening (cv). Severity scores within each category could be 0 (<5%; none or minimal), 1 (>5% - <25%; mild), 2 (>25% - <50%, moderate), or 3 (>50%, severe). The proportions of patients in each severity grade (0, 1, 2, and 3) for both the individual categories and a composite were compared using Fisher's exact test. (NCT00556933)
Timeframe: Two years

,,,
Interventionpercentage of participants (Number)
Banff histopathology cumulative grade = 0Banff histopathology cumulative grade = 1Banff histopathology cumulative grade = 2Banff histopathology cumulative grade = 3
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus404696
Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil514171
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus4337164
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil534520

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Immunosuppression-free Duration in Days, Defined as Time From Completion of Immunosuppression Withdrawal to End of Trial Participation or to Time of Restarting Immunosuppression

Time (in days) from when the participant is off all immunosuppression to the end of trial participation or re-initiation of immunosuppression, whichever is earliest. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionDays (Median)
Transplanted374

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Percent of Participants Requiring Anti-lymphocyte Therapy (OKT3, ATG) for an Acute Rejection Event

Anti-lymphocyte therapy is a drug that targets specific cells in the immune system called lymphocytes (white blood cells). This therapy helps stop the participant's immune system from attacking the donor kidney. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)20

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Percent of Participants Successfully Withdrawn From Immunosuppression and Remained Off Immunosuppression for at Least 52 Weeks

Participants are considered successfully withdrawn from immunosuppression if they remained off immunosuppression for at least 52 weeks without evidence of rejection, as determined by a biopsy performed 52 weeks after completion of immunosuppression withdrawal. All participants who failed to complete immunosuppression withdrawal, regardless of reason, or failed to have a biopsy 52 weeks after completion of immunosuppression withdrawal, were considered to have failed. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks after discontinuation of all immunosuppression

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)20

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Percent of Participants With Chronic T Cell-mediated or Antibody-mediated Rejection

This assessment included participants who experienced chronic T cell-mediated rejection or chronic antibody mediated rejection as well as progressive interstitial fibrosis/tubular atrophy (IF/TA), transplant glomerulopathy or chronic obliterative arteriopathy without an alternative, non-rejection-related cause. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)0

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Percent of Transplant Participants Who Died

Death after receiving a kidney transplant. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)0

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Percent of Transplanted Participants Who Achieve Either Sirolimus Monotherapy or Monotherapy on a Mycophenolic Compound Within 52 Weeks Post-transplant

Participants that were treated with only sirolimus or treated with only mycophenolate mofetil (MMF) or mycophenolic acid within 52 weeks after transplantation. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks post-transplantation

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)70

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Percent of Transplanted Participants Who Achieve MMF or Mycophenolic Acid Monotherapy Within 52 Weeks Post-transplant in Those Participants Intolerant of Sirolimus

Participants that were treated with only mycophenolate mofetil (MMF) or mycophenolic acid within 52 weeks after transplantation in those who could not tolerate sirolimus. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks post-transplantation

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)33

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Percent of Transplanted Participants Who Achieve Sirolimus Monotherapy Within 52 Weeks Post-transplant

Participants that were treated with only sirolimus within 52 weeks after transplantation in those who could tolerant sirolimus. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks post-transplantation

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)86

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Percent of Transplanted Participants Who Remain Off Immunosuppression for at Least 52 Weeks Including Those in Whom the 52 Week Biopsy Was Not Performed

Participants are considered successfully withdrawn from immunosuppression if they remained off immunosuppression for at least 52 weeks without evidence of rejection. A biopsy performed 52 weeks after completion of immunosuppression withdrawal confirmed that there was no sub-clinical evidence of rejection. This result considers a participant off all immunosuppression for at least 52 weeks with or without the confirmatory week 52 biopsy as a success. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks after discontinuation of all immunosuppression

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)20

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Percent of Transplanted Participants Who Remain Off Immunosuppression for the Duration of the Study as Defined as Completion of All Schedules of Events/Followed Through August 25, 2017

Participants that remained off all immunosuppression through the completion of study participation. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through study completion (up to 4.4 years post-transplant)

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)10

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Percent of Transplanted Participants With Acute Rejection or Presumed Acute Rejection

Participants with either biopsy proven acute rejection per Banff guidelines or participants that were treated for acute rejection in the absence of a biopsy. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionPercent of participants (Geometric Least Squares Mean)
Induction (Rituximab and ATG)90

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Percent of Transplanted Participants With Graft Loss

A participant is considered to have graft loss when the donated kidney needs to be removed, the participant is retransplanted with another donor kidney, or chronic dialysis is instituted. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionPercent of participants (Number)
Induction (Rituximab and ATG)0

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Time From Completion of Immunosuppression Withdrawal to First Episode of Acute Rejection or Presumed Acute Rejection

Time (in days) from when the participant is off all immunosuppression to the first episode of biopsy proven or presumed acute rejection. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionDays (Median)
Induction (Rituximab and ATG)380

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Time From Transplant to the First Episode of Acute Rejection Requiring Treatment

Time (in days) from transplant to the start date of the first dose of treatment for acute rejection. This includes acute rejection episodes requiring treatment that are not biopsy proven. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionDays (Median)
Induction (Rituximab and ATG)1008.5

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Histological Severity of Biopsies Demonstrating Acute Rejection as Measured by Banff 2007 Grade

"Biopsy-confirmed 1.) acute cellular rejection and 2.) acute antibody-mediated rejection was classified according to Banff 2007 criteria of renal allograft pathology for renal allograft rejection. A Banff result of indeterminate was not classified as rejection.~Acute cellular rejection occurs when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection is defined as a grade ≥ IA. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe.~Acute antibody-mediated rejection-or humoral rejection-is defined as a grade ≥1. Severity is graded as I, II, or III, with I being the mildest form of antibody-mediated rejection and III being the most severe." (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionBiopsies (Number)
Acute Cellular Rejection (Type IA)Acute Cellular Rejection (Type IB)Acute Cellular Rejection (Type IIA)Acute Cellular Rejection (Type IIB)Acute Cellular Rejection (Type III)Acute Antibody-Mediated Rejection (Type I)Acute Antibody-Mediated Rejection (Type II)Acute Antibody-Mediated Rejection (Type III)
Induction (Rituximab and ATG)73000000

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Number of Adverse Events, Including Number of Post-transplant Infections, Wound Complications, Lymphocoele, Post-transplant Diabetes Mellitus, and Malignancies

Adverse events that are reported as being a post-transplant infection, wound complication, lymphocoele (a collection of fluid in the lymphatic system), post-transplant diabetes mellitus or malignancy. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)

InterventionEvents (Number)
Post-Transplant InfectionWound ComplicationsLymphocoelePost-Transplant Diabetes MellitusMalignancy
Induction (Rituximab and ATG)41000

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Participant Diastolic Blood Pressure Over Time

Diastolic blood pressure measures the pressure in the arteries when the heart is at rest and is thus filled with blood. A normal diastolic blood pressure is lower than 80 mmHg. High blood pressure, as known as hypertension, is a risk factor for coronary artery disease, stroke, heart failure, and other complications if left unmanaged. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant

InterventionmmHg (Median)
26 Weeks Post-Transplant52 Weeks Post-Transplant104 Weeks Post-Transplant156 Weeks Post-Transplant208 Weeks Post-Transplant
Induction (Rituximab and ATG)77.577797673

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Participant Glucose Level Over Time

This is a measure of glucose found in the blood. Glucose, a sugar, is an energy source that the body relies on to properly function. If levels are too high for a long period of time, diabetes can develop. Diabetes can result in many long-term complications such as eye, kidney, and nerve damage, stroke, and cardiovascular complications. Fasting levels for glucose should be around 70-99 mg/dL and less than 140 mg/dL within 2 hours after a meal. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant

Interventionmg/dL (Median)
26 Weeks Post-Transplant52 Weeks Post-Transplant104 Weeks Post-Transplant156 Weeks Post-Transplant208 Weeks Post-Transplant
Induction (Rituximab and ATG)103.510896.5104.583.5

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Participant Renal Function as Measured by GFR Using CKD-EPI

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. A value less than 15 indicates kidney failure, 15 to 29 indicates severe loss of kidney function, 30 to 44 indicates moderate to severe loss of kidney function, 45 to 59 mild to moderate loss of kidney function, 60 to 89 indicates mild loss of kidney function, and 90 or higher indicates normal kidney function. The equation developed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) is used to estimate GFR from serum creatinine. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant

InterventionmL/min/1.73m^2 (Median)
26 Weeks Post-Transplant52 Weeks Post-Transplant104 Weeks Post-Transplant156 Weeks Post-Transplant208 Weeks Post-Transplant
Induction (Rituximab and ATG)60.370.963.756.756.0

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Participant Systolic Blood Pressure Over Time

Systolic blood pressure measures the pressure on the blood vessels when the heart is beats and thus is pushing blood to the rest of the body. A normal systolic blood pressure is lower than 120 mmHg. High blood pressure, as known as hypertension, is a risk factor for coronary artery disease, stroke, heart failure, and other complications if left unmanaged. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant

InterventionmmHg (Median)
26 Weeks Post-Transplant52 Weeks Post-Transplant104 Weeks Post-Transplant156 Weeks Post-Transplant208 Weeks Post-Transplant
Induction (Rituximab and ATG)131.5135137140132

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Participant Total Cholesterol Over Time

Total cholesterol measures the amount of cholesterol found in the blood. Cholesterol is a waxy substance your body needs to build cells, but too much can be a problem since it can build-up in arteries. Narrowed arteries can result in heart attack or stroke. A value less than 200 mg/dL is considered good. The value closest to and within 12 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant

Interventionmg/dL (Median)
26 Weeks Post-Transplant52 Weeks Post-Transplant104 Weeks Post-Transplant156 Weeks Post-Transplant208 Weeks Post-Transplant
Induction (Rituximab and ATG)188141.5165175173

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Phase I: Acute Rejection-Free Survival

Percent of participants at 6 months without acute rejection. (NCT01062555)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Phase I Arm 180
Phase I Arm 285

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Phase I: The Minimization of Negative Side Effects - Graft Survival

Percent of participants at 6 months with a functioning graft (without graft failure). (NCT01062555)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Phase I Arm 199
Phase I Arm 299

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Phase I: The Minimization of Negative Side Effects - Patient Survival

The percentage of patients alive at 6 month post transplant. (NCT01062555)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Phase I Arm 199
Phase I Arm 298

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Phase II: Acute Rejection-Free Survival

The percentage of patients without acute rejection at 7 years post transplant or at the end of study activities. Not all participants completed 7-year follow-up. (NCT01062555)
Timeframe: up to 7 years

Interventionpercentage of participants (Number)
Phase II Arm 176
Phase II Arm 281

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Phase II: The Minimization of Negative Side Effects - Graft Survival

The percentage of patients with a functioning graft (without graft failure) at 7 years post transplant or at the end of study activities. Not all participants completed 7-year follow-up. (NCT01062555)
Timeframe: up to 7 years

Interventionpercentage of participants (Number)
Phase II Arm 180
Phase II Arm 278

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Phase II: The Minimization of Negative Side Effects - Patient Survival

The percentage of patients alive at 7 years post transplant or at the end of study activities. Not all participants completed 7-year follow-up. (NCT01062555)
Timeframe: up to 7 years

Interventionpercentage of participants (Number)
Phase II Arm 189
Phase II Arm 285

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Percent Change From Baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR) at Week 24 in the Blinded Treatment Period (Part B)

The percent change from baseline in UPCR based on 24-hour urine collections. 24-hour urine specimens measure the levels of proteins and creatinine in urine and will be used for the UPCR at baseline (week 12) and week 24. (NCT03943147)
Timeframe: Week 24

InterventionPercent change from baseline (Number)
Open Label MMF + BMS-986165-34.86

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The Number of Participants Experiencing Averse Events in the Blinded Treatment Period (Part B)

An adverse event (AE) is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug and that does not necessarily have a causal relationship with this treatment. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861651

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The Number of Participants With Abnormal Laboratory Parameters of Clinical Significance in the Blinded Treatment Period (Part B)

The number of participants with abnormal laboratory parameters (Chemistry, hematology, coagulation, immunohematology) that have been considered clinically significant. Clinically relevant laboratory results are determined by the investigator. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Clinically Significant ECG Abnormalities in the Blinded Treatment Period (Part B)

The number of participants with clinically significant abnormalities in electrocardiograms (ECGs) parameters. The following ECG parameters will be measured: HR, PR-interval, QRS-duration, QT-interval, QTc-interval. A single 12-lead ECG will be recorded after the participant has been supine for at least 5 minutes. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Complete Renal Response (CRR) at Week 24 in the Blinded Treatment Period (Part B)

The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline. (NCT03943147)
Timeframe: Week 24

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Complete Renal Response (CRR) at Week 52 in the Blinded Treatment Period (Part B)

The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline. (NCT03943147)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Complete Renal Response (CRR) Plus Successful Corticosteroid Taper to ≤ 7.5 mg/Day at Week 24 in the Blinded Treatment Period (Part B)

The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline who was also able to successfully taper corticosteroid use to ≤ 7.5 mg/day. (NCT03943147)
Timeframe: Week 24

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Complete Renal Response (CRR) Plus Successful Corticosteroid Taper to ≤ 7.5 mg/Day at Week 52 in the Blinded Treatment Period (Part B)

The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline who was also able to successfully taper corticosteroid use to ≤ 7.5 mg/day. (NCT03943147)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Partial Renal Response (PRR) at Week 24 in the Blinded Treatment Period (Part B)

The number of participants with partial renal response (PRR) defined as ≥ 50% reduction from baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR). 24-hour urine specimens measure the levels of proteins and creatinine in urine and will be used for the UPCR at baseline (week 12) and week 24. (NCT03943147)
Timeframe: Week 24

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Number of Participants With Partial Renal Response (PRR) at Week 52 in the Blinded Treatment Period (Part B)

The number of participants with partial renal response (PRR) defined as ≥ 50% reduction from baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR). 24-hour urine specimens measure the levels of proteins and creatinine in urine and will be used for the UPCR at baseline (week 12) and week 52. (NCT03943147)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Open Label MMF + BMS-9861650

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The Percent Change in Vital Sign Measurements in the Blinded Treatment Period (Part B)

The percent change from baseline in Vital sign measurements including: blood pressure, heart rate, respiratory rate, and temperature. Blood pressure and heart rate are measured after the participant has been resting quietly for at least 5 minutes. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B

InterventionPercent change from baseline (Number)
Diastolic Blood Pressure(mmHg)Systolic Blood Pressure(mmHgHeart Rate(beats/min)Respiratory Rate(breaths/min)Temperature(C)
Open Label MMF + BMS-986165-3.455.2216.876.25-1.35

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Number of Patients With a Partial Response

The proportion of each type of response with a 95% CI will be reported for all evaluable patients, assuming a binomial distribution. (NCT04205240)
Timeframe: Approximately 11 months

Interventionpatients (Number)
Treatment (Conditioning Regimen, Stem Cell Transplant)1

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Number of Patients With Grade II-IV Acute Graft-versus Host Disease (GvHD (aGVHD)

The event will be onset of grade II-IV aGvHD and time to aGvHD will be defined as the period of time from transplantation to the event of aGvHD. Death and early relapse without aGvHD will be competing risks. Cumulative incidence rate of aGVHD with 95% confidence intervals will be estimated from the cumulative incidence curves. To evaluate the association between patient characteristics and aGVHD, the Gray's test accounting for competing risks will be used to compare the cumulative incidence curves and a proportional hazards model for the sub distribution of competing risks will be used to estimate the hazard ratio. The cumulative incidence of chronic GVHD (cGVHD) will be similarly analyzed. (NCT04205240)
Timeframe: Up to 6 weeks

Interventionparticipants (Number)
Treatment (Conditioning Regimen, Stem Cell Transplant)0

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AUC0-inf - Area Under Concentration-time Curve From Time Zero to Infinity (Extrapolated)

Bioequivalence based on AUC0-inf (NCT00911274)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*hr/mL (Mean)
Mycophenolate Mofetil (Test)15.7365
CellCept® (Reference)15.7289

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AUC0-t - Area Under Concentration-time Curve From Time Zero to Time of Last Non-zero Concentration

Bioequivalence based on AUC0-t (NCT00911274)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*hr/mL (Mean)
Mycophenolate Mofetil (Test)14.8297
CellCept® (Reference)14.6761

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Cmax - Maximum Observed Concentration

Bioequivalence based on Cmax (NCT00911274)
Timeframe: Blood samples collected over 72 hour period

Interventionµg/mL (Mean)
Mycophenolate Mofetil (Test)8.5858
CellCept® (Reference)9.2170

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Donor-specific HLA Antibodies, Re-transplantation, or Death

The Outcome Measure is a composite primary endpoint of the development of donor-specific HLA antibodies, re-transplantation, or death. Testing for donor-specifc HLA antibodies was performed at study-specified time points using the single antigen bead assay at the study core lab. Donor-specific HLA antibodies were defined as reactivity with a mean fluorescence intensity (MFI) ≥ 2,000. (NCT03388008)
Timeframe: 365 days

,
Interventionparticipants (Number)
DeathDonor-specific HLA antibodiesRe-transplantation
Belatacept-based Immunosuppression530
Standard of Care030

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Percentage of Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR) at 6 Months

Number of Participants with Clinically-suspected biopsy-proven acute rejection (CSBPAR) at 6 Months (NCT02137239)
Timeframe: 6 Months

InterventionPercentage of participants (Number)
Treatment A7.7
Treatment B9.4

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Percentage of Participants With Adverse Events (AEs)

Percentage of participants with AEs up to 24 months post-transplant (NCT02137239)
Timeframe: Up to 24 months Post-Transplant

InterventionPercentage of participants with AEs (Number)
Treatment A100.0
Treatment B97.0

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Percentage of Participants With Serious Adverse Events (SAEs)

Percentage of participants with SAEs up to 24 months post-transplant (NCT02137239)
Timeframe: Up to 24 months Post-Transplant

InterventionPercentage of participants with SAEs (Number)
Treatment A52.0
Treatment B60.6

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Time to Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR).

Time to Clinically suspected biopsy proven acute rejection (NCT02137239)
Timeframe: Up to 24 Months

InterventionMonths (Mean)
Treatment ANA
Treatment BNA

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Absolute Calculated Glomerular Filtration Rate (cGFR): Mean

Absolute (mean and median) cGFR values at 3, 6, 12 and 24 months post-transplant, as determined from the 4-variable Modification of Diet in Renal Disease (MDRD) formula (NCT02137239)
Timeframe: Up 24 Months post-transplant

,
InterventionmL/min/1.73 m^2 (Mean)
At 3 MonthsAt 6 MonthsAt 12 MonthsAt 24 Months
Treatment A69.266.066.271.8
Treatment B62.263.962.068.7

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Absolute Values of Blood Pressure: Mean

Absolute (mean and median) values for SBP and DBP at 3, 6, 12 and 24 months posttransplant; (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionmmHg (Mean)
Diastolic Month 3Systolic Month 3Diastolic Month 6Systolic Month 6Diastolic Month 12Systolic Month 12Diastolic Month 24Systolic Month 24
Treatment A78.7134.277.4128.178.7131.078.1130.9
Treatment B77.7131.079.4133.080.1131.078.5131.7

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Absolute Values of Blood Pressure: Median

Absolute (mean and median) values for SBP and DBP at 3, 6, 12 and 24 months posttransplant; (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionmmHg (Median)
Diastolic Month 3Systolic Month 3Diastolic Month 6Systolic Month 6Diastolic Month 12Systolic Month 12Diastolic Month 24Systolic Month 24
Treatment A78.5135.575.5127.077.0130.078.0130.0
Treatment B80.0131.080.0131.081.0126.079.0130.0

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Absolute Values of Fasting Lipid Values: Mean

"Absolute (mean and median) values at 3, 6, 12 and 24 months post-transplant for the following:~Serum total cholesterol (TC) Serum high density lipoprotein (HDL) cholesterol Serum low density lipoprotein (LDL) cholesterol Serum triglycerides (TG)" (NCT02137239)
Timeframe: Up to 24 Months

,
Interventionmg/dL (Mean)
TC Month 3TC Month 6TC Month 12TC Month 24HDL Month 3HDL Month 6HDL Month 12HDL Month 24LDL Month 3LDL Month 6LDL Month 12LDL Month 24TG Month 3TG Month 6TG Month 12TG Month 24
Treatment A181.2197.7189.0193.250.646.449.450.196.9115.2107.597.9171.6180.0162.4263.4
Treatment B174.4175169.9168.250.453.949.651.396.593.788.091.5137.8138.3161.3145.0

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Absolute Values of Fasting Lipid Values: Median

"Absolute (mean and median) values at 3, 6, 12 and 24 months post-transplant for the following:~Serum total cholesterol (TC) Serum high density lipoprotein (HDL) cholesterol Serum low density lipoprotein (LDL) cholesterol Serum triglycerides (TG)" (NCT02137239)
Timeframe: Up to 24 Months

,
Interventionmg/dL (Median)
TC Month 3TC Month 6TC Month 12TC Month 24HDL Month 3HDL Month 6HDL Month 12HDL Month 24LDL Month 3LDL Month 6LDL Month 12LDL Month 24TG Month 3TG Month 6TG Month 12TG Month 24
Treatment A167.0187.0184.0193.045.045.550.049.089.099.5104.0103.5147.0157.5154.0159.0
Treatment B173.0178.0171.5166.049.049.047.049.095.0100.091.586.0128.0126.0130.0114.0

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Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR) at 6, 12 and 24 Months

"Clinically-suspected biopsy-proven acute rejection (CSBPAR) at 6, 12 and 24 Months~Change in the incidence of CSBPAR at 6, 12 and 24 months post transplant, in the belatacept + EVL(Treatment A) as compared to TAC + MMF (Treatment B)." (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionPercentage of CSBPAR (Number)
CSBPAR at 6 MonthsCSBPAR at 12 monthsCSBPAR at 24 Months
Treatment A7.711.515.4
Treatment B9.412.512.5

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Mean and Mean Change From Baseline in Blood Glucose

Mean fasting blood glucose levels, and mean changes from baseline values at Months 6, 12 and 24 months post- transplant (NCT02137239)
Timeframe: Up to 24 months

,
Interventionmg/dL (Mean)
Mean Value at 6 monthsChange from baseline at 6 monthsMean Value at 12 monthsChange from baseline at 12 monthsMean Value at 24 monthsChange from baseline at 24 months
Treatment A107.24.9101.1-1.3127.522.3
Treatment B107.24.8127.520.6111.815.0

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Mean and Mean Change From Baseline in Whole Blood HbA1c

Mean whole blood HbA1C concentrations, and mean changes from baseline values at Months 6, 12 and 24 months post-transplant. (NCT02137239)
Timeframe: Up to 24 months

,
Interventionmg/dL (Mean)
Mean Value at 6 monthsChange from baseline at 6 monthsMean Value at 12 monthsChange from baseline at 12 monthsMean Value at 24 monthsChange from baseline at 24 months
Treatment A6.110.346.180.476.240.66
Treatment B6.130.486.210.326.290.41

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Mean Change From Month 3 in cGFR

The mean change from Month 3 cGFR at 3, 6, 12 and 24 months post-transplant (NCT02137239)
Timeframe: Up 24 Months post-transplant

,
InterventionmL/min/1.73 m^2 (Mean)
At 3 MonthsAt 6 MonthsAt 12 MonthsAt 24 Months
Treatment A0-3.2-3.13.1
Treatment B02.81.46.3

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Mean Changes From Baseline Values for Blood Pressure

Mean changes from baseline values for SBP and DBP at 6, 12 and 24 months post-transplant (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionmmHg (Mean)
Diastolic Month 6Systolic Month 6Diastolic Month 12Systolic Month 12Diastolic Month 24Systolic Month 24
Treatment A1.0-4.02.3-1.10.9-2.3
Treatment B4.8-0.75.4-3.22.1-4.2

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Mean Changes From Baseline Values of Lipid Values

"Mean changes from baseline values in the following:~Serum total cholesterol (TC) Serum high density lipoprotein (HDL) cholesterol Serum low density lipoprotein (LDL) cholesterol Serum triglycerides (TG)" (NCT02137239)
Timeframe: at months 12 and 24

,
Interventionmg/dL (Mean)
TC Month 12TC Month 24HDL Month 12HDL Month 24LDL Month 12LDL Month 24TG Month 12TG Month 24
Treatment A25.726.65.46.225.717.43.3106.8
Treatment B-2.810.01.94.810.815.7-86.1-13.6

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Median Calculated Glomerular Filtration Rate (cGFR)

Median cGFR values at 3, 6, 12 and 24 months post-transplant, as determined from the 4-variable Modification of Diet in Renal Disease (MDRD) formula (NCT02137239)
Timeframe: Up 24 Months post-transplant

,
InterventionmL/min/1.73 m^2 (Median)
At 3 MonthsAt 6 MonthsAt 12 MonthsAt 24 Months
Treatment A64.064.066.073.5
Treatment B62.067.062.568.0

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Number of Participants Deaths Post Transplant

Number of participant deaths at 6, 12 and 24 months post transplant (NCT02137239)
Timeframe: up to 24 months

,
InterventionParticipants (Count of Participants)
At 6 MonthsAt 12 MonthsAt 24 Months
Treatment A000
Treatment B000

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Number of Participants Who Experience Graft Loss Post Transplant

Number of all participants who experience graft loss at 6, 12 and 24 months post transplant (NCT02137239)
Timeframe: At 6, 12 and 24 months

,
InterventionParticipants (Count of Participants)
At 6 MonthsAt 12 MonthsAt 24 Months
Treatment A111
Treatment B111

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Number of Participants Who Survive With a Functioning Graft

Number of all participants who survive with a functioning graft at 6, 12 and 24 months post transplant (NCT02137239)
Timeframe: At 6, 12 and 24 months

,
InterventionParticipants (Count of Participants)
At 6 MonthsAt 12 MonthsAt 24 Months
Treatment A252525
Treatment B313131

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Percentage of Participants With BANFF Grade by Severity Grades. BANFF Type (Grade) for Acute/Active Rejection

"Treatment differences in the severity grades to treat all episodes of CSBPAR at 6, 12, and 24 months post-transplant.~Type 1A - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>4 mononuclear cells/Tubular cross section or group of 10 Tubular cell). Type 1B - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>10 mononuclear cells/Tubular cross section or group of 10 Tubular cell).Type 2A - Cases with mild to moderate intimal arteritis.Type 2B - Cases with severe intimal arteritis comprising >25% of the luminal area. Type 3 - Cases with transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (v3 with accompanying lymphocytic inflammation)" (NCT02137239)
Timeframe: At 6, 12 and 24 Months

,
InterventionPercentage of Participants (Number)
6 Months: Mild Acute (1A)6 Months: Mild Acute (1B)6 Months: Moderate Acute (2A)6 Months: Moderate Acute (2B)6 Months: Severe Acute12 Months: Mild Acute (1A)12 Months: Mild Acute (1B)12 Months: Moderate Acute (2A)12 Months: Moderate Acute (2B)12 Months: Severe Acute24 Months: Mild Acute (1A)24 Months: Mild Acute (1B)24 Months: Moderate Acute (2A)24 Months: Moderate Acute (2B)24 Months: Severe Acute
Treatment A3.807.7007.707.70011.507.700
Treatment B03.16.3003.13.16.3003.13.16.300

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Percentage of Participants With De Novo Donor Specific Anti-HLA Antibodies (DSA)

Characterization of any de novo DSA detected by IgM and IgG subclasses, and by the presence or absence of complement fixing properties. (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionPercentage (Number)
Baseline Class 1 DSABaseline Class 2 DSABaseline Both Class 1 and 2 DSADe Novo 12 Month Class 1 DSADe Novo 12 Month Class 2 DSADe Novo 12 Month Both Class 1 and 2 DSADe Novo 24 Month Class 1 DSADe Novo 24 Month Class 2 DSADe Novo 24 Month Both Class 1 and 2 DSA
Treatment A1000000000
Treatment B0000003.4500

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Percentage of Participants With Donor Specific Anti-HLA Antibodies (DSA)

Percentage of participants with, and titers of pre-existing (pre-transplant) DSA on Day 1 (pre-transplant, pre-dose), and at Months 12 and 24 posttransplant (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionPercentage of Participants (Number)
Baseline Class 1 DSABaseline Class 2 DSABaseline Both Class 1 and 2 DSA12 Month Class 1 DSA12 Month Class 2 DSA12 Month Both Class 1 and 2 DSA24 Month Class 1 DSA24 Month Class 2 DSA24 Month Both Class 1 and 2 DSA
Treatment A10008.00008.0000
Treatment B00003.0303.033.030

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Percentage of Participants With Events of Special Interest (ESIs)

"Percentage of participants which have one of the following events of special interest:~Serious Infections Post-Transplant Lymphoproliferative Disorder (PTLD) Progressive multifocal leukoencephalopathy (PML) Malignancies (Other than PTLD) including non-melanoma skin carcinomas (Malignancies) Tuberculosis Infections Central Nervous System (CNS) Infections Viral Infections Infusion Related reactions within 24 hours since belatacept infusion" (NCT02137239)
Timeframe: Up to 24 Months

,
InterventionPercentage of participants with ESIs (Number)
Serious InfectionsPTLDPMLMalignanciesTBCNS InfectionsViral InfectionsInfusion Related Reactions
Treatment A164.004.00001
Treatment B15.23.003.00000

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Percentage of Participants With New Onset Diabetes After Transplant

Percentage of participants with New Onset Diabetes After Transplantation (NODAT) at 6, 12, and 24 months post-transplant. (NCT02137239)
Timeframe: up to 24 months

,
InterventionPercentage of participants (Number)
Up to 6 MonthsUp to 12 MonthsUp to 24 Months
Treatment A11.511.515.4
Treatment B6.36.312.5

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Percentage of Particpants With Laboratory Test Abnormalities (LTAs)

Percentage of participants with laboratory tests with marked laboratory abnormalities (NCT02137239)
Timeframe: At 24 Months

,
InterventionPercentage of participants (Number)
Hemoglobin (Low)Leukocytes (low)Lymphocyte (Absolute) (low)Neutrophils (Absolute) (low)Aspartate Aminotransferase (High)Creatinine (High)Inorganic Phosphorus (low)Potassium (high)Sodium (low)Albumin (low)Glucose (high)Triglycerides (high)Uric Acid (high)
Treatment A12.0084.004.016.024.04.04.008.012.08.0
Treatment B6.13.069.73.003.012.1003.012.100

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Time to Event: Graft Loss and Death

The Number of days to participant Graft Loss and death for any reason (NCT02137239)
Timeframe: Up to 728 Days

,
InterventionDays (Number)
Graft Loss
Treatment A107
Treatment B2

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Treatment Differences in Therapeutic Modalities

Treatment Received for Biopsy Proven Acute Rejection (Banff Grade IA or Higher), or Humoral (Antibody Mediated) Rejection Treatment regimen: Categorical analysis of CSBPAR episodes by treatment received. (NCT02137239)
Timeframe: at 6, 12 and 24 Months

,
InterventionPercentage of participants with CSBPARs (Number)
Corticosteroids (6 months)Lymphocyte depleting agent (6 months)Plasmapheresis (6 months)IVIG (6 months)Rituximab (6 months)Corticosteroids (12 months)Lymphocyte depleting agent (12 months)Plasmapheresis (12 months)IVIG (12 months)Rituximab (12 months)Corticosteroids (24 months)Lymphocyte depleting agent (24 months)Plasmapheresis (24 months)IVIG (24 months)Rituximab (24 months)
Treatment A7.7000015.43.800019.23.8000
Treatment B9.46.303.1012.56.303.1012.56.303.10

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Urine Protein Creatinine Ratio (UPr/Cr)

Urine protein to creatinine ratio (UPr/Cr) at 3, 6, 12 and 24 months post-transplant. (NCT02137239)
Timeframe: Up 24 Months post-transplant

,
Interventionmg Protein/mg Creatinine (Mean)
At 3 MonthsAt 6 MonthsAt 12 MonthsAt 24 Months
Treatment A0.31460.38960.28350.3940
Treatment B0.14120.14610.18490.1685

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Mean Cases of Acute Rejection (MCAR) Per Patient

"MCAR represents the average number of acute rejections among all patients in each treatment group. Results were based on rejection episodes with endomyocardial biopsies.~Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection." (NCT00157014)
Timeframe: 52 Weeks

InterventionMCAR per patient (Mean)
Tacrolimus - Adult0.15
Cyclosporine - Adult0.17

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Mean Cases of Acute Rejection (MCAR) Per Patient (Pediatric Population)

"MCAR represents the average number of acute rejections among all patients in each treatment group. Results were based on rejection episodes with endomyocardial biopsies.~Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection." (NCT00157014)
Timeframe: 52 Weeks

InterventionMCAR per patient (Mean)
Tacrolimus - Pediatric0.60
Cyclosporine - Pediatric0.50

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Number of Cardiac Rejection Episodes Requiring Treatment

The number of rejection episodes requiring treatment (medications started/ stopped, non-medication treatment, or both) regardless of biopsy grade or presence of hemodynamic compromise. (NCT00157014)
Timeframe: 52 Weeks

InterventionRejection Episodes (Number)
Tacrolimus - Adult12
Cyclosporine - Adult11

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Number of Cardiac Rejection Episodes Requiring Treatment (Pediatric Population)

A summary of rejection episodes requiring treatment regardless of biopsy grade or presence of hemodynamic compromise. (NCT00157014)
Timeframe: 52 Weeks

InterventionRejection Episodes (Number)
Tacrolimus - Pediatric3
Cyclosporine - Pediatric3

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Number of Patients Requiring Antilymphocyte Antibodies or Steroids for Treatment of Severe Acute Rejection

Severe Acute Rejection is defined as rejection with ISHLT Grade 4. (NCT00157014)
Timeframe: 52 Weeks

InterventionPatients (Number)
Tacrolimus - Adult0
Cyclosporine - Adult0

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Number of Patients Requiring Antilymphocyte Antibodies or Steroids for Treatment of Severe Acute Rejection (Pediatric Population)

Severe Acute Rejection was defined as rejection with ISHLT Grade 4. (NCT00157014)
Timeframe: 52 Weeks

InterventionPatients (Number)
Tacrolimus - Pediatric0
Cyclosporine - Pediatric0

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Time to First Acute Rejection Episode Following de Novo Cardiac Transplant

"Acute Rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Time to first acute rejection is defined as: date of onset - date of transplant." (NCT00157014)
Timeframe: 52 Weeks

InterventionDays (Mean)
Tacrolimus - Adult55.0
Cyclosporine - Adult35.60

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Time to First Acute Rejection Episode Following de Novo Cardiac Transplant (Pediatric Population)

"Acute Rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Time to first acute rejection is defined as: date of onset - date of transplant." (NCT00157014)
Timeframe: 52 Weeks

InterventionDays (Mean)
Tacrolimus - Pediatric56.3
Cyclosporine - Pediatric49.0

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Changes in Circulating Markers of Inflammation and Oxidation: Cystatin-C (Pediatric Population)

Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionmg/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Pediatric0.770.84-0.01
Tacrolimus - Pediatric0.860.87-0.11

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Changes in Circulating Markers of Inflammation and Oxidation: F2 Isoprostanes (Pediatric Population)

Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionpg/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Pediatric104.6866.48-30.07
Tacrolimus - Pediatric106.0669.71-38.31

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Changes in Circulating Markers of Inflammation and Oxidation: hsCRP (Pediatric Population)

Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionmg/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Pediatric12.082.43-13.94
Tacrolimus - Pediatric30.4626.31-7.85

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Changes in Circulating Markers of Inflammation and Oxidation: Nitrotyrosine (Pediatric Population)

Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
InterventionnM (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Pediatric12701.2141147.6221514.62
Tacrolimus - Pediatric233.085462.995148.42

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: BNP

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~BNP= Brain Natriuretic Peptide" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionng/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult4240.81856.8-1446.7
Tacrolimus - Adult4314.8670.1-4018.4

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Cystatin-C

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionmg/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult1.291.480.27
Tacrolimus - Adult1.211.290.06

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: E-selectin

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionng/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult98.9680.93-19.16
Tacrolimus - Adult90.4068.60-18.58

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: F2 Isoprostanes

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionpg/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult53.9450.44-3.43
Tacrolimus - Adult52.0330.08-13.29

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Fibrinogen

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventiong/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult4.43.8-0.5
Tacrolimus - Adult4.43.4-1.1

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: GSH/GSSG

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~GSH/GSSG= ratio of reduced to oxidised glutathione" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
InterventionRatio (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult58.8353.72-5.55
Tacrolimus - Adult55.0751.69-2.07

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Homocysteine

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionμmol/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult15.915.80.7
Tacrolimus - Adult14.213.50.3

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: hsCRP

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~hsCRP= high-sensitivity C Reactive Protein" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionmg/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult21.833.95-18.69
Tacrolimus - Adult32.853.01-34.32

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: IL-18

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionpg/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult496.2427.2-71.0
Tacrolimus - Adult574.0534.65.2

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: IL-6

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~IL= Interleukin" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionpg/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult2.540.90-1.56
Tacrolimus - Adult3.360.98-2.84

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: MCP-1

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~MCP-1= monocyte chemoattractant protein-1" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionpg/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult193.63180.90-16.49
Tacrolimus - Adult233.05229.9642.92

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Nitrotyrosine

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
InterventionnM (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult482.43368.95-99.79
Tacrolimus - Adult422.63451.8871.44

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Osteopontin

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionng/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult11.1410.490.20
Tacrolimus - Adult11.888.77-2.22

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: s-ICAM

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~s-ICAM= soluble-intracellular adhesion molecule" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionng/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult674.46503.71-183.96
Tacrolimus - Adult766.58590.30-227.58

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: T-bars

"Change is defined as Week 52 assessment - Pre-Transplant assessment.~T-bars = thiobarbituric acid reactive substances" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionnmol/mL (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult3.913.14-0.77
Tacrolimus - Adult3.783.25-0.64

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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Troponin T

Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks

,
Interventionug/L (Mean)
Pre-TransplantWeek 52Change from Pre-Transplant at Week 52
Cyclosporine - Adult0.280.04-0.27
Tacrolimus - Adult0.300.03-0.32

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Number of Acute Rejection Episodes by International Society of Heart and Lung Transplantation (ISHLT) Criteria

"Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Patients may report more than one acute rejection." (NCT00157014)
Timeframe: 52 Weeks

,
InterventionRejection Episodes (Number)
Total Acute Rejection EpisodesAcute Rejection Episodes with ISHLT Grade ≥3AAcute Rejection Episodes w/ Hemodynamic Compromise
Cyclosporine - Adult872
Tacrolimus - Adult836

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Number of Acute Rejection Episodes by International Society of Heart and Lung Transplantation (ISHLT) Criteria (Pediatric Population)

"Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Patients may report more than one rejection episode." (NCT00157014)
Timeframe: 52 Weeks

,
InterventionRejection Episodes (Number)
Total Acute Rejection EpisodesAcute Rejection Episodes with ISHLT Grade ≥3AAcute Rejection Episodes w/ Hemodynamic Compromise
Cyclosporine - Pediatric330
Tacrolimus - Pediatric330

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Number of Patients With Successful Steroid Taper or Withdrawal at Weeks 26 and 52

A successful steroid taper or withdrawal was defined as steroids (prednisone) being discontinued or tapered to the suggested dose level after week 26. (NCT00157014)
Timeframe: 26 Weeks and 52 Weeks

,
InterventionPatients (Number)
Week 26Week 52
Cyclosporine - Adult1629
Tacrolimus - Adult2233

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Number of Patients With Successful Steroid Taper or Withdrawal at Weeks 26 and 52 (Pediatric Population)

A successful steroid taper or withdrawal was defined as steroids (prednisone) being discontinued or tapered to the suggested dose level after week 26. (NCT00157014)
Timeframe: 26 Weeks and 52 Weeks

,
InterventionPatients (Number)
Week 26Week 52
Cyclosporine - Pediatric31
Tacrolimus - Pediatric21

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Number of Patients With Treatment Failure and Crossover for Treatment Failure

"Treatment failure was defined as death, re-transplantation, withdrawal due to an Adverse Event, or a switch of main immunosuppressant medication, whichever came first.~Crossover was defined as a switch from originally administered primary immunosuppressant (tacrolimus or cyclosporine) to the alternate primary immunosuppressant." (NCT00157014)
Timeframe: 52 Weeks

,
InterventionPatients (Number)
Treatment FailuresCrossover for Treatment Failures
Cyclosporine - Adult118
Tacrolimus - Adult62

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Number of Patients With Treatment Failure and Crossover for Treatment Failure (Pediatric Population)

"Treatment failure was defined as death, re-transplantation, withdrawal due to an Adverse Event, or a switch of main immunosuppressant medication, whichever came first.~Crossover was defined as a switch from originally administered primary immunosuppressant (tacrolimus or cyclosporine) to the alternate primary immunosuppressant." (NCT00157014)
Timeframe: 52 Weeks

,
InterventionPatients (Number)
Treatment FailuresCrossover for Treatment Failures
Cyclosporine - Pediatric33
Tacrolimus - Pediatric10

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The Change in the Markers of Growth, Apoptosis, Inflammation and Oxidation Measured in Endomyocardial Biopsies

"The markers assessed were p-ERK ½ (phosphorylated extracellular signal-regulated kinase), p-JNK (phosphorylated jun N-terminal kinase) and p-p38 MAPK (phosphorylated mitogen-activated protein kinase).~The data for each biopsy marker were expressed as a ratio of its densitometry / densitometry of glyceraldehyde-3-phosphate dehydrogenase (GAPDH).~Change is defined as Week 52 assessment- Week 2 assessment." (NCT00157014)
Timeframe: 2 Weeks and 52 Weeks

,
InterventionDensitometry / Densitometry of GAPDH (Mean)
p-ERK ½ - Week 2p-ERK ½ - Week 52p-ERK ½ - Change from Week 2p-JNK - Week 2p-JNK - Week 52p-JNK - Change from Week 2p-p38 MAPK - Week 2p-p38 MAPK - Week 52p-p38 MAPK - Change from Week 2
Cyclosporine - Adult0.900.79-0.051.231.460.220.540.770.23
Tacrolimus - Adult0.700.870.051.101.330.030.480.630.14

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The Change in the Markers of Growth, Apoptosis, Inflammation and Oxidation Measured in Endomyocardial Biopsies (Pediatric Population)

"The markers assessed were p-ERK ½, p-JNK and p-p38 MAPK.~The data for each biopsy marker were expressed as a ratio of its densitometry / densitometry of glyceraldehyde-3-phosphate dehydrogenase (GAPDH).~Change is defined as Week 52 assessment- Week 2 assessment." (NCT00157014)
Timeframe: 2 Weeks and 52 Weeks

,
InterventionDensitometry / Densitometry of GAPDH (Mean)
p-ERK ½ - Week 2p-ERK ½ - Week 52p-ERK ½ - Change from Week 2p-JNK - Week 2p-JNK - Week 52p-JNK - Change from Week 2p-p38 MAPK - Week 2p-p38 MAPK - Week 52p-p38 MAPK - Change from Week 2
Cyclosporine - Pediatric1.671.22-0.270.910.820.040.430.580.34
Tacrolimus - Pediatric1.740.93-0.091.170.57-0.210.830.24-0.04

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The Incidence of Biopsy-proven Acute Allograft Rejection During the First 12 Months of Transplant.

The incidence of rejection is determined by the proportion of patients experiencing biopsy proven acute allograft rejection during the first 12 months post-transplant. (NCT00166712)
Timeframe: Within 12 months post kidney transplant

InterventionParticipants (Number)
Groups4
Group 26

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Renal Function (Nankivell Formula) at Month 12 Post Transplantation.

Renal function at the end of the trial assessed as mean absolute values of the glomerular filtration rate (GFR) calculated by Nankivell formula 12 months after renal transplantation. The Nankivell formula: GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)^2 + C ; where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. Estimated GFR is expressed in mL/min per 1.73m^2. (NCT00154310)
Timeframe: at Month 12 post transplantation

InterventionmL/min /1.73m^2 (Mean)
Everolimus + Mycophenolate Sodium71.84
Cyclosporine + Mycophenolate Sodium61.24

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Changes in Cardiovascular Risk From Month 4.5 to Final Assessment at Month 12

An updated 1991 Framingham coronary prediction algorithm was used to estimate the total risk of developing coronary heart diseases (CHD) over the course of 10 years. Risk was calculated separately for male and females. To calculate risk, points were assigned for each of the following risk factors: age, levels of LDL cholesterol, HDL cholesterol, blood pressure, cigarette smoking, and diabetes mellitus. The sum of the individual risk factor points gives a total point score, which ranges from -5 to 18 for men and -16 to 24 for women. Higher points indicate a higher risk for CHD. (NCT00154310)
Timeframe: Month 4.5 and Month 12

,
InterventionPoints (Mean)
Male (n= 55, 37)Female (n= 22, 35)Total Population (n= 77, 72)
Cyclosporine + Mycophenolate Sodium0.10.80.4
Everolimus + Mycophenolate Sodium0.50.00.4

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Number of Participants Who Experienced an Adverse Event or Serious Adverse Event

Additional information about the number of participants who experienced Adverse Events (greater than 5%) or Serious Adverse Events can be found in the Adverse Event section. (NCT00154310)
Timeframe: Aes from end of core study period (month 12) to end of follow-up period (month 60)

,
InterventionParticipants (Number)
Adverse EventsSerious Adverse Events
Cyclosporine + Mycophenolate Sodium14586
Everolimus + Mycophenolate Sodium15595

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Number of Participants With Occurrence of Biopsy Proven Acute Rejection (BPAR), Graft Loss or Death

The number of participants with occurrence of biopsy proven acute rejection (BPAR), graft loss, or death up to Month 12 during the randomized treatment period. BPAR was defined as a biopsy graded IA, IB, IIA, IIB or III according to Banff 97 classification. A graft core biopsy was performed prior to 24 hours following initiation of graft rejection therapy. The allograft is presumed to be lost on the day the patient starts dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss. (NCT00154310)
Timeframe: Up to Month 12

,
InterventionParticipants (Number)
BPAR: YesBPAR: NoGraft Loss: YesGraft Loss: NoDeath: YesDeath: No
Cyclosporine + Mycophenolate Sodium514101461145
Everolimus + Mycophenolate Sodium1513901540154

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Number of Participants With Occurrence of Treatment Failures

Treatment failures defined as a composite endpoint of biopsy proven acute rejection, graft loss, death, loss to follow up and discontinuations due to lack of efficacy or toxicity, or conversion to another regimen (at least one condition must be present). (NCT00154310)
Timeframe: up to or at Month 12

,
InterventionParticipants (Number)
Treatment failure: YesTreatment failure: No
Cyclosporine + Mycophenolate Sodium23123
Everolimus + Mycophenolate Sodium29125

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Number of Participants With an Episode of Clinically-suspected and Biopsy-proven Acute Rejection (CSPAR)

No participant was to receive treatment for acute rejection without a biopsy to confirm the diagnosis. CSPAR=Clinically-suspected rejection, defined as an increase in serum creatinine ≥0.5 mg/dL compared with the baseline value in the absence of other factors known to adversely affect renal function, and biopsy-proven rejection, which includes all cases in which a biopsy was read by the central pathologist as demonstrating acute rejection regardless of the reason why the biopsy was performed. (NCT00035555)
Timeframe: By Month 6 posttransplant (From Day 1 to Month 6)

InterventionParticipants (Number)
Belatacept: More Intensive (MI) Regimen5
Belatacept: Less Intensive (LI) Regimen4
Cyclosporine Regimen6

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Mean Iohexol Clearance

Iohexol, a true glomerular filtration marker, is used to measure glomerular filtration rate. (NCT00035555)
Timeframe: By Months 1, 6, and 12 posttransplant (Day 1 to Months 1, 6, and 12)

,,
InterventionmL/min per 1.73 m^2 (Mean)
Month 1 (n=53, 51, 48)Month 6 (n=41, 41, 31)Month 12 (n=32, 37, 27)
Belatacept: Less Intensive (LI) Regimen60.264.562.1
Belatacept: More Intensive (MI) Regimen59.762.266.3
Cyclosporine Regimen54.056.053.5

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Mean LDL Cholesterol, HDL Cholesterol, Total Cholesterol, Triglyceride, and Non-HDL Levels

LDL=low-density lipoprotein; HDL=high-density lipoprotein. Total cholesterol=LDL + HDL + very low-density (VLDL) cholesterol. VLDL=triglycerides divided by 5. Non-HDL cholesterol=Total cholesterol minus HDL cholesterol. (NCT00035555)
Timeframe: By Months 1, 6, and 12 posttransplant (Day 1 to Months 1, 6, and 12)

,,
Interventionmg/dL (Mean)
LDL cholesterol: Month 1 (n=69, 69, 66)LDL cholesterol: Month 6 (n=63, 66, 55)LDL cholesterol: Month 12 (n=61, 60, 52)HDL cholesterol: Month 1 (n=68, 68, 64)HDL cholesterol: Month 6 (n=62, 65, 62)HDL cholesterol: Month 12 (n=60, 57, 48)Total cholesterol: Month 1 (n=69, 69, 65)Total cholesterol: Month 6 (n=63, 65, 54)Total cholesterol: Month 12 (n=60, 58, 50)Triglycerides: Month 1 (n=69, 69, 65)Triglycerides: Month 6 (n=63, 65, 54)Triglycerides: Month 12 (n=60, 58, 50)Non-HDL: Month 1 (n=68, 68, 64)Non-HDL cholesterol: Month 6 (n=62, 64, 51)Non-HDL cholesterol: Month 12 (n=59, 56, 48)
Belatacept: Less Intensive (LI) Regimen120121125685656210202201147168152142143144
Belatacept: More Intensive (MI) Regimen129125120645453222204198168177176159150145
Cyclosporine Regimen137131125706259239224212185198186169165151

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Number of Participants Meeting Marked Abnormality Criteria for Select Hemolytic, Blood Chemistry, and Urinalysis Laboratory Test Results

Normal laboratory values: Hemoglobin (g/dL): Males (18-64 years) 13.8-17, (65 years and older) 11.8-16.8; Females (18-64 years) 12.0-15.6, F (65 years and older) 11.1-15.5. Platelets (per mm^3) 130,000-400,000. Leukocytes (18 years and older) 3.8-10.8 1000/uL. ALT (u/L)(13 years and older) 0-48. (NCT00035555)
Timeframe: Days 8 and Months 1, 3, 6, 9, and 12 posttransplant (from Day 1)

,,
InterventionParticipants (Number)
Hemoglobin, lowHemoglobin, highPlatelet count, lowPlatelet count, highLeukocytes, lowLeukocytes, highAlanine aminotransferase (ALT), lowALT, high
Belatacept: More Intensive (MI) Regimen13NA1NA1NANA9
Belatacept:Less Intensive (LI) Regimen7NA0NA4NANA3
Cyclosporine Regimen9NA3NA5NANA7

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Number of Participants With Hypertension

Hypertension is defined as diastolic blood pressure ≥90 mm Hg and/or systolic blood pressure ≥140 mm Hg or, the use of any antihypertensive medication. (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)

,,
InterventionParticipants (Number)
At Month 6At Month 12
Belatacept: Less Intensive (LI) Regimen1512
Belatacept: More Intensive (MI) Regimen1614
Cyclosporine Regimen1811

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Number of Participants With Posttransplant Diabetes Mellitus

Posttransplant diabetes mellitus is defined as the need for treatment of hyperglycemia with either an oral agent or insulin for a total of >4 weeks or hemoglobin A1c (HbA1c) >7% in a participant not known to be diabetic prior to transplantation (NCT00035555)
Timeframe: By Months 1, 3, 6, 9, and 12 posttransplant (Day 1 to Months 1, 3, 6, 9, and 12 )

,,
InterventionParticipants (Number)
Up to Month 1: All eventsMonth 1: Hyperglycemia medicationMonth 1: HbA1c >7%Up to Month 3: All eventsUp to Month 3: Hypoglycemic medicationUp to Month 3: HbA1c >7%Up to Month 6: All eventsUp to Month 6: Hyperglycemia medicationUp to Month 6: HbA1c >7%Up to Month 9: All eventsUp to Month 9: Hyperglycemia medicationUp to Month 9: HbA1c >7%Up to Month 12: All eventsUp to Month 12: Hyperglycemia medicationUp to Month 12: HbA1c >7%
Belatacept: Less Intensive (LI) Regimen000101303303404
Belatacept: More Intensive (MI) Regimen505505606827827
Cyclosporine Regimen110321422523734

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Percentage of Participants Who Had Chronic Allograft Nephropathy

Based on postbaseline biopsies (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)

,,
InterventionPercentage of participants (Number)
Month 6 (n= 32, 33, 27)Month 12 (n=52, 54, 45)
Belatacept: Less Intensive (LI) Regimen9.120.4
Belatacept: More Intensive (MI) Regimen18.828.8
Cyclosporine Regimen33.344.4

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Percentage of Participants Who Used Antihypertensive Medication

Hypertension is defined as diastolic blood pressure ≥90 mm Hg and/or systolic blood pressure ≥140 mm Hg (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)

,,
InterventionPercentage of participants (Number)
Month 6: Total requiring at least 1 medicationMonth 6: Requiring 1 medicationMonth 6: Requiring 2 medicationsMonth 6: Requiring 3 medicationsMonth 6: Requiring 4 medicationsMonth 6: Requiring 5 medicationsMonth 6: Requiring 6 medicationsMonth 6: Requiring >6 medicationsMonth 12: Total requiring at least 1 medicationMonth 12: Requiring 1 medicationMonth 12: Requiring 2 medicationsMonth 12: Requiring 3 medicationsMonth 12: Requiring 4 medicationsMonth 12: Requiring 5 medicationsMonth 12: Requiring 6 medicationsMonth 12: Requiring >6 medications
Belatacept: Less Intensive (LI) Regimen78.627.128.614.37.11.40.00.071.626.99.09.07.50.00.00.0
Belatacept: More Intensive (MI) Regimen87.724.723.323.311.02.72.70.079.721.723.223.28.75.81.40.0
Cyclosporine Regimen88.421.731.921.711.61.40.00.086.420.322.022.015.33.40.00.0

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Percentage of Participants With Acute Rejection or Presumed Acute Rejection (PAR)

Throughout this study, acute rejection=clinically-suspected and biopsy-proven acute rejection (BPAR). Clinically-suspected rejection is defined as an increase in serum creatinine ≥0.5 mg/dL compared with the baseline value in the absence of other factors known to adversely affect renal function. BPAR includes all cases in which a biopsy was read by the central pathologist as demonstrating acute rejection regardless of the reason why the biopsy was performed. PAR is defined as an elevation in SCr ≥0.5 mg/dL compared with the baseline value in the absence of other factors known to adversely affect renal function that led the investigator to suspect that the participant had experienced acute rejection, and in whom either the biopsy did not confirm acute rejection and the participant received treatment for acute rejection or the participant received treatment for acute rejection without a biopsy to confirm the diagnosis. (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)

,,
InterventionPercentage of participants (Number)
Month 6: Acute rejection or PARMonth 12: Acute rejection or PARMonth 6: PARMonth 12: PAR
Belatacept: Less Intensive (LI) Regimen91034
Belatacept: More Intensive (MI) Regimen111155
Cyclosporine Regimen101113

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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR) or Who Received Treatment for Acute Rejection

BPAR includes all cases in which a biopsy read by the central pathologist demonstrates acute rejection, regardless of the reason that the biopsy was performed. A participant was reported as having had an episode of treated acute rejection if he or she received antirejection therapy during an episode of rejection (clinically-suspected or biopsy-proven rejection). (NCT00035555)
Timeframe: By Months 3, 6, and 12 posttransplant (Day 1 to Months 3, 6, and 12)

,,
InterventionPercentage of participants (Number)
By Month 3By Month 6By Month 12
Belatacept: Less Intensive (LI) Regimen29.632.438.0
Belatacept: More Intensive (MI) Regimen21.623.028.4
Cyclosporine Regimen17.824.727.4

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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR) Through Months 6 and 12

BPAR includes all cases in which a biopsy read by the central pathologist demonstrates acute rejection, regardless of the reason that the biopsy was performed. (NCT00035555)
Timeframe: Through Months 6 and 12 posttransplant (From Day 1 to Months 6 and 12)

,,
InterventionPercentage of participants (Number)
Up to 6 monthsUp to 12 months
Belatacept: Less Intensive (LI) Regimen23.929.6
Belatacept: More Intensive (MI) Regimen14.918.9
Cyclosporine Regimen17.817.8

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AUC0-inf - Area Under the Concentration-time Curve From Time Zero to Infinity (Extrapolated)

Bioequivalence based on AUC0-inf (NCT00908128)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*hr/mL (Mean)
Mycophenolate Mofetil24.5141
CellCept®24.9890

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AUC0-t - Area Under the Concentration-time Curve From Time Zero to Time of the Last Non-zero Concentration

Bioequivalence based on AUC0-t (NCT00908128)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*hr/mL (Mean)
Mycophenolate Mofetil23.3091
CellCept®23.5366

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Cmax - Maximum Observed Concentration

Bioequivalence based on Cmax (NCT00908128)
Timeframe: Blood samples collected over 72 hour period

Interventionµg/mL (Mean)
Mycophenolate Mofetil7.4578
CellCept®8.2195

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Event-free Survival

(NCT00611351)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic2

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Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)

(NCT00611351)
Timeframe: at day 100 post transplantation

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic4

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Overall Survival

(NCT00611351)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic2

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Transplantation-related Mortality at 100 Days Post-transplantation

(NCT00611351)
Timeframe: at the 100 days post-transplant

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic2

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Incidence of Acute and Chronic Graft-versus-host Disease

Incidence of acute and chronic graft-versus-host disease. Acute GVHD usually occurs during the first three months following transplant. Chronic GVHD usually develops after the third month post-transplant. (NCT00571662)
Timeframe: twice weekly until day 100 up to 1 year post transplant

InterventionPercent of Particpants (Number)
Acute GVHDChronic GVHD
Cohort I3133

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Kinetics of Immunologic Reconstitution

Rate of return of immune cells after allogeneic transplantation (NCT00571662)
Timeframe: at day 100 post transplantation

,
Interventionpercentage of cells in peripheral blood (Median)
CD3 cellsCD4 cellsCD8 cells
Day + 28 Post Transplant73.51.7
Study Baseline1355

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Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting

the efficacy of the regimen as determined by engraftment rate and establishment of donor hematopoietic chimerism at day +28 and day +70. (NCT00571662)
Timeframe: days +28 and +70

Interventionpercent of participants (Median)
Day 28Day 70
Cohort I8590

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Responses to Therapy

event-free and overall survival at 12 months (NCT00571662)
Timeframe: every 6 mo. up to 2 years

InterventionPercent of Participants (Number)
Event free survivalOverall survival
Cohort I5259

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Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)

(NCT00571662)
Timeframe: Conditioning regimen to count recovery (D + 28 post transplant)

InterventionParticipants (Count of Participants)
Absolute neutrophil count < 500/mm^3platelet count < 20,000/mm^3Grade 3 or 4 FeverGrade 3 or 4 hypokalemiaGrade 3 or 4 bacteremiaGrade 3 or 4 infectionGrade 3 or 4 renal toxicityGrade 3 or 4 thromboembolism
Cohort I4029212611

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Determine the Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) With Diagnosis of Engraftment Loss.

Number of patients with engraftment loss. (NCT02593123)
Timeframe: 60 Days Following Stem Cell Transplant

InterventionParticipants (Count of Participants)
Arm I (MMF-15, Sargramostim)0
Arm II (MMF-30, Filgrastim)1

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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Diagnosed With Chronic Graft vs Host Disease (GVHD)

The differences in the rates of chronic GVHD between patients randomized to MMF-30 (control cohort) and MMF-15 (investigational cohort) (NCT02593123)
Timeframe: 60 Days following stem cell transplant

InterventionParticipants (Count of Participants)
Arm I (MMF-15, Sargramostim)9
Arm II (MMF-30, Filgrastim)7

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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Diagnosed With Opportunistic Infections

The number of patients diagnosed with an opportunistic infections. (NCT02593123)
Timeframe: 60 Days following stem cell transplant

InterventionParticipants (Count of Participants)
Arm I (MMF-15, Sargramostim)7
Arm II (MMF-30, Filgrastim)9

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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) With Acute Graft Vs Host Disease (GVHD)

The number of patients diagnosed with acute acute GVHD between patients randomized to MMF-30 (control cohort) and MMF-15 (investigational cohort) (NCT02593123)
Timeframe: 60 Days following stem cell transplant

InterventionParticipants (Count of Participants)
Arm I (MMF-15, Sargramostim)4
Arm II (MMF-30, Filgrastim)8

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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort)Diagnosed With Differences in the Rates of Engraftment Syndrome.

Number of patients diagnosed with engraftment syndrome. (NCT02593123)
Timeframe: 60 Days Following Stem Cell Transplant

InterventionParticipants (Count of Participants)
Arm I (MMF-15, Sargramostim)0
Arm II (MMF-30, Filgrastim)0

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Differences in the Rates of Achieving Donor Chimerisms (the Percentage of DNA in the Sample Which Comes From the Donor) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort).

Number of patients that achieved donor chimerisms by day 100. (NCT02593123)
Timeframe: 100 Days following Stem Cell Transplant

InterventionParticipants (Count of Participants)
Arm I (MMF-15, Sargramostim)15
Arm II (MMF-30, Filgrastim)11

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Differences in the Rates of T-cell Recovery Kinetics Following Stem Cell Transplantation (SCT) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort).

Number of T Cells 10E3 per microL per arm indicating rates of T-cell recovery by Day 100 following SCT. (NCT02593123)
Timeframe: 100 Days Following Stem Cell Transplantation

Intervention10^3 *cells* per microliter (Mean)
Arm I (MMF-15, Sargramostim)313.6
Arm II (MMF-30, Filgrastim)222.2

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The Difference Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Day 60 Donor-derived (dd) Cluster of Differentiation (CD)3 10E3per microL Counts.

Day 60 donor-derived (dd) cluster of differentiation (CD)3 counts measured by 10E3per microL. (NCT02593123)
Timeframe: 60 Days Following Stem Cell Transplant

Intervention10^3 *cells* per microliter (Mean)
Arm I (MMF-15, Sargramostim)383.0
Arm II (MMF-30, Filgrastim)254.1

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The Probability of Overall Survival (OS) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort)

Overall survival (days to event or survival: time-to-event; survival: categorical) (NCT02593123)
Timeframe: Randomization up to 2 years

InterventionProbablility of 2 year survival (Number)
Arm I (MMF-15, Sargramostim)0.78
Arm II (MMF-30, Filgrastim)0.5

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Number of Participants With Absolute Neutrophil Count >= 500/Mcl for 3 Consecutive Measurements on Different Days and Platelet Count > 20,000/mm^3 With no Platelet Transfusions in the Preceding 7 Days

To determine engraftment of neutrophils and platelets at 28 days following alpha/beta T-cell depletion using Human Leukocyte Antigen (HLA) haploidentical donors for stem cell transplant in relapsed lymphoma. (NCT02652468)
Timeframe: At day 28 after transplantation

InterventionParticipants (Count of Participants)
Peripheral Blood Stem Cell Transplant11

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Number of Participants With Grade III-IV Acute GVHD as Determined by International Bone Marrow Transplant Registry (IBMTR) Severity Index Criteria

The number of participants with grade III - IV acute Graft versus host disease (GVHD) by Day +100 is reported. (NCT02652468)
Timeframe: Day +100

InterventionParticipants (Count of Participants)
Peripheral Blood Stem Cell Transplant3

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Number of Participants With Graft Failure

Graft failure - defined as < 5% donor chimerism in the CD3 and/or CD33 selected cell populations at any time during the study follow up period once initial engraftment has been achieved. (NCT02652468)
Timeframe: Up to 2 years after graft

InterventionParticipants (Count of Participants)
Peripheral Blood Stem Cell Transplant0

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Number of Participants With Severe Chronic GVHD

The number of participants with severe chronic GVHD by Day +180 will be reported. (NCT02652468)
Timeframe: Day +180

InterventionParticipants (Count of Participants)
Peripheral Blood Stem Cell Transplant0

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Overall Survival (OS)

(NCT02652468)
Timeframe: Median follow up of 1689 days

Interventiondays (Median)
Peripheral Blood Stem Cell Transplant352

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Progression-free Survival

Progression-free survival will be analyzed as time before any progression by either Positron Emission Tomography/Computed Tomography (PET/CT) or bone marrow, (NCT02652468)
Timeframe: Median follow up of 1689 days

Interventiondays (Median)
Peripheral Blood Stem Cell Transplant352

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Achievement of Remission

"Number of participants who are in complete remission (CR) 4 weeks after transplant. CR is defined as complete resolution of all signs of myelodysplasia or leukemia for at least 4 weeks with all of the following:~Normal bone marrow with blasts <5% with normal cellularity, normal megakaryopoiesis, > 15% erythropoiesis and > 25% granulocytopoiesis~Normalization of blood counts (no blasts, platelets > 100000/mm3, granulocytes >1500/mm3)~No extramedullary disease." (NCT01300572)
Timeframe: 4 weeks after transplant

InterventionParticipants (Count of Participants)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)13

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Disease-free Survival

Number of study participants who are alive and remains in complete remission after transplant. (NCT01300572)
Timeframe: 100 days after transplant

Interventionparticipants (Number)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)7

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Duration of Remission

"Median time to relapse after achieving complete remission (CR). CR is defined as complete resolution of all signs of myelodysplasia or leukemia for at least 4 weeks with all of the following:~Normal bone marrow with blasts <5% with normal cellularity, normal megakaryopoiesis, > 15% erythropoiesis and > 25% granulocytopoiesis~Normalization of blood counts (no blasts, platelets > 100000/mm3, granulocytes >1500/mm3)~No extramedullary disease.~Relapse Criteria:~After CR: >5% blasts in the bone marrow and/or peripheral blood~After partial remission (PR): increase of blasts cells in the marrow to >50% of those during PR~Extramedullary disease confirmed cytologically or histologically." (NCT01300572)
Timeframe: 1 year

Interventiondays (Median)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)213

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Overall Survival

Number of participants who are still alive after transplant with or without disease. (NCT01300572)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)7

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Rates of Donor Chimerism

Number of participants who has 100% donor chimerism within 100 days after transplant (NCT01300572)
Timeframe: Up to 100 days post-transplant

InterventionParticipants (Count of Participants)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)14

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Rates of Engraftment

Average number of days to ANC >= 500 after transplant (NCT01300572)
Timeframe: Up to 84 days post-transplant

Interventiondays (Mean)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)16

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The MTD of Radiation Delivered Via 90Y-DOTA-BC8 When Combined With FLU and 2 Gy TBI as a Preparative Regimen for Patients Aged ≥ 18 With Advanced AML, ALL, and High-risk MDS.

The MTD will be defined as the dose that is associated with a true DLT rate of 25%. The highest dose achieved was 28 Gy but none of the patients experienced a DLT. Thus, the MTD was not reached. (NCT01300572)
Timeframe: Within the first 30 days following transplant

InterventionGy (Number)
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)28

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Estimation of Absorbed Radiation Doses to Normal Organs, Marrow and Tumor

The amount of energy absorbed per unit weight of the organ or tissue is called absorbed dose and is expressed in units of gray (Gy). One gray dose is equivalent to one joule radiation energy absorbed per kilogram of organ or tissue weight. (NCT01300572)
Timeframe: Approximately day -20 to day -12 prior to transplant

InterventionGy (Mean)
Average absorbed dose to the marrowAverage absorbed dose to the liverAverage abosorbed dose total bodyAverage absorbed dose to the spleen
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)11.417.23.170

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Rates of Non-relapse Mortality

Transplant-related deaths within 100 days after transplant (NCT01300572)
Timeframe: Within the first 100 days following transplant

InterventionParticipants (Count of Participants)
Severe refractory GVHDBacterial infection
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant)11

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Percentage of Participants Surviving

(NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28

Interventionpercentage of participants (Number)
MMF Monotherapy97.7

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Percentage of Participants With Acute Rejection

Diagnosis of acute rejection was suspected in any participant with an increase in serum creatinine greater than or equal to (≥) 25 percent (%). All suspected acute rejections were confirmed by biopsy. The start date of acute rejection was identified as the date of biopsy. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28

Interventionpercentage of participants (Number)
MMF Monotherapy9.1

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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR)

BPAR was defined according to 1997 Banff Criteria as a biopsy Banff grade of IA, IB, IIA, IIB, or III. Grade IA was defined as significant interstitial infiltration with greater than (>)25% of parenchyma affected, and foci of moderate tubulitis with >4 mononuclear cells per tubular cross section or group of 10 tubular cells. Grade IB was defined as significant interstitial infiltration with >25% parenchyma affected, and foci of severe tubulitis with >10% mononuclear cells per tubular cross section or group of 10 tubular cells. Grade IIA was defined as mild to moderate intimal arteritis. Grade IIB was defined as severe intimal arteritis comprising >25% of the luminal area. Grade III was defined as transmural arteritis and/or arterial fibrinoid changes and necrosis of medial smooth muscle cells. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28

Interventionpercentage of participants (Number)
MMF Monotherapy4.5

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Percentage of Participants With Graft Loss

An allograft was presumed to be lost if a participant started dialysis and was not able to subsequently be removed from dialysis. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28

Interventionpercentage of participants (Number)
MMF Monotherapy2.3

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Time to Rejection

The mean time, in days, from the date of enrollment to date of biopsy confirming acute rejection. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28

Interventiondays (Mean)
MMF Monotherapy23.67

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Free MPA (mcg/mL) by Visit

Drug quantification of free MPA in the plasma was measured at T = 0, 40, and 120 mins. (NCT01292226)
Timeframe: Weeks 2, 4, 12, 24, safety follow-up (Week 28), and any unscheduled visits

Interventionmcg/mL (Mean)
T=0, Week 2 (n=40)T=0, Week 4 (n=39)T=0, Week 12 (n=33)T=0, Week 24 (n=32)T=0, safety follow-up (n=3)T=0, unscheduled visit (n=7)T=0, overall mean value (n=154)T=40, Week 2 (n=41)T=40, Week 4 (n=42)T=40, Week 12 (n=35)T=40, Week 24 (n=35)T=40, safety follow-up (n=3)T=40, unscheduled visit (n=7)T=40, overall mean value (n=163)T=120, Week 2 (n=43)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, Week 24 (n=35)T=120, safety follow-up (n=3)T=120, unscheduled visit (n=7)T=120, overall mean value (n=165)
MMF Monotherapy0.030.040.030.030.010.040.030.110.110.110.380.060.090.170.110.110.160.100.100.070.12

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IMPDH Expression I by Visit and Timepoint

IMPDH I gene expression was measured by real time polymerase chain reaction (QRT-PCR) based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of messenger ribonucleic acid (mRNA) copies per cell (copies/cell). (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits

Interventionnumber of mRNA copies/cell (Mean)
T=0, BL (n=44)T=0, Week 2 (n=41)T=0, Week 4 (n=42)T=0, Week 12 (n=34)T=0, Week 24 (n=34)T=0, safety follow-up (n=3)T=0, unscheduled visit (n=7)T=0, mean value (n=205)T=120, BL (n=0)T=120, Week 2 (n=43)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, Week 24 (n=34)T=120, safety follow-up (n=3)T=120, unscheduled visit (n=7)T=120, mean value (n=164)
MMF Monotherapy2.3232.293.164.101.9511803.484.00181.4804146.171692.111556.063.02107.301038.521899.45

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IMPDH Expression II by Visit and Timepoint

IMPDH II gene expression was measured by QRT-PCR based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of mRNA copies/cell. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits

Interventionnumber of mRNA copies/cell (Mean)
T=0, BL (n=44)T=0, Week 2 (n=41)T=0, Week 4 (n=42)T=0, Week 12 (n=34)T=0, Week 24 (n=34)T=0, safety follow-up (n=3)T=0, unscheduled visit (n=7)T=0, mean value (n=205)T=120, BL (n=0)T=120, Week 2 (n=43)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, Week 24 (n=34)T=120, safety follow-up (n=3)T=120, unscheduled visit (n=7)T=120, mean value (n=164)
MMF Monotherapy115.33113.43117.16112.43114.21123.86116.57114.820304.64148.32143.11109.00140.51167.19180.71

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Inosine MonoPhosphate DeHydrogenase (IMPDH) Activity by Visit and Timepoint

"IMPDH activity in peripheral blood mononuclear cells (PBMCs) was measured at 2 timepoints per visit, 0 and 120 minutes and presented in enzyme units. The unit of measure of enzyme activity is U. One U is defined as the amount of the enzyme that produces a certain amount of enzymatic activity that is, the amount that catalyzes the conversion of 1 micro mole of substrate per minute under pre-specified conditions (temperature, pH)." (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits

Interventionenzyme units (Mean)
T=0, BL (n=44)T=0, Week 2 (n=42)T=0, Week 4 (n=42)T=0, Week 12 (n=35)T=0, Week 24 (n=34)T=0, safety follow-up (n=0)T=0, unscheduled visit (n=7)T=0, mean value (n=204)T=120, BL (n=0)T=120, Week 2 (n=42)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, Week 24 (n=34)T=120, safety follow-up (n=0)T=120, unscheduled visit (n=7)T=120, mean value (n=160)
MMF Monotherapy5.013.963.896.749.5806.005.6503.063.103.756.3903.893.97

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Interleukin 8 (IL-8) Expression by Visit and Timepoint

IL-8 gene expression was measured by QRT-PCR based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of mRNA copies/cell. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits

Interventionnumber of mRNA copies/cell (Mean)
T=0, BL (n=44)T=0, Week 2 (n=41)T=0, Week 4 (n=42)T=0, Week 12 (n=34)T=0, Week 24 (n=34)T=0, safety follow-up (n=3)T=0, unscheduled visit (n=7)T=0, mean value (n=205)T=120, BL (n=0)T=120, Week 2 (n=43)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, Week 24 (n=34)T=120, safety follow-up (n=3)T=120, unscheduled visit (n=7)T=120, mean value (n=164)
MMF Monotherapy4532.7229960.4154101.301829.8710391.601254.2919148.9420748.320.01028.9321227.1112022.839418.65397887.30220.1417512.31

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MPA Area Under the Concentration - Time Curve From Time 0 to 12 Hours (AUC0-12) (mcg/mL) by Visit

The AUC0-12 of MPA was estimated on the validated limited sampling strategy, AUC (milligrams multiplied by height over liter [mg.h/L]) = 7.182 + 4.607 multiplied by (*) concentration at 0 minutes (C0)+ 0.998 * the concentration at 40 minutes (C0.67) + 2.149 * the concentration at 120 minutes (C2). (NCT01292226)
Timeframe: Predose and 40 minutes and 2 hours postdose at Weeks 2, 4, 12, and 24, and at the Safety follow-up (Week 28)

Interventionmcg*hr/mL (Mean)
Week 2 (n=41)Week 4 (n=42)Week 12 (n=35)Week 24 (n=35)Follow-up (n=3)
MMF Monotherapy38.339.739.739.829.7

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Percentage of Participants With Gastrointestinal Toxicities

Gastrointestinal adverse events (AEs) according to WHO worst grade observed. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, 24, and 28 (Safety Follow-up Visit)

Interventionpercentage of participants (Number)
Abdominal pain (moderate)Abdominal pain (severe)Anal fissure (mild)Diarrhoea (mild)Diarrhoea (moderate)Diarrhoea (severe)Dyspepsia (mild)Gastritis (moderate)Gastritis erosive (moderate)Gingival hyperplasia (mild)Haemorrhoids (mild)Intra-abdominal haematoma (mild)Nausea (moderate)Stomatitis (mild)Vomiting (mild)Vomiting (moderate)Vomiting (severe)
MMF Monotherapy2.222.222.224.442.222.222.222.222.222.222.222.222.222.222.224.444.44

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Percentage of Participants With Hematologic Toxicity

Hematological toxicities graded according to WHO worst grade observed (Grade 1=mild, Grade 2=moderate). (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, 24, and 28 (Safety Follow-Up)

Interventionpercentage of participants (Number)
Grade 1 hemoglobin decreasedGrade 1 leukocytes decreasedGrade 2 leukocytes decreasedGrade 1 granulocytes decreasedGrade 2 granulocytes decreasedGrade 1 platelets decreasedGrade 1 bilirubin increasedGrade 2 bilirubin increasedGrade 1 hypoglycemiaGrade 1 alkaline phosphatase increasedGrade 2 alkaline phosphatase increasedGrade 1 aspartate aminotransferase increasedGrade 1 alanine aminotransferase increasedGrade 2 alanine aminotransferase increasedGrade 1 cholesterol increasedGrade 2 cholesterol increasedGrade 1 triglycerides increasedGrade 2 triglycerides increasedGrade 1 blood urea nitrogen increasedGrade 2 blood urea nitrogen increased
MMF Monotherapy13.3311.118.896.676.676.674.442.224.4413.332.226.678.892.2226.678.8924.442.222.222.22

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Percentage of Participants With Infection

Infections were graded according to the World Health Organization (WHO) worst grade observed. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, 24, and 28 (Safety Follow-Up)

Interventionpercentage of participants (Number)
Acarodermatitis (mild)Bronchitis (moderate)Cytomegalovirus (CMV) infection (mild)CMV infection (moderate)CMV infection (severe)CMV viraemia (mild)Gastroenteritis proteus (severe)Gastrointestinal infection (severe)Legionella infection (life-threatening)Oral herpes (mild)Sepsis (life-threatening)Tracheitis (mild)Urethritis (mild)Urinary tract infection (mild)Urinary tract infection (moderate)
MMF Monotherapy2.222.2213.338.894.442.222.222.222.222.222.222.222.2228.892.22

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Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Gastrointestinal Toxicity

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
IMPDH I expression (n=334)IMPDH II expression (n=351)
MMF Monotherapy0.0820.030

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Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Hematologic Toxicity

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
IMPDH I expression (n=334)IMPDH II expression (n=351)
MMF Monotherapy-0.116-0.004

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Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Infection

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
IMPDH I expression (n=334)IMPDH II expression (n=351)
MMF Monotherapy0.0450.047

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Spearman's Rank Correlation Coefficient Between IMPDH I Expression and Free Fraction

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
p Free fraction, T=0 (n=140)p Free fraction, T=120 (n=143)
MMF Monotherapy0.0470.080

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Spearman's Rank Correlation Coefficient Between IMPDH I Expression and MPA Levels

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
Free MPA, T=0 (n=141)Free MPA, T=120 (n=143)Total MPA, T=0 (n=147)Total MPA, T=120 (n=143)
MMF Monotherapy0.073-0.0240.037-0.140

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Spearman's Rank Correlation Coefficient Between IMPDH II Expression and Free Fraction

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
p Free fraction, T=0 (n=144)p Free fraction, T=120 (n=153)
MMF Monotherapy-0.0270.015

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Spearman's Rank Correlation Coefficient Between IMPDH II Expression and MPA Levels

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
Free MPA, T=0 (n=145)Free MPA, T=120 (n=153)Total MPA, T=0 (n=152)Total MPA, T=120 (n=153)
MMF Monotherapy0.0070.073-0.0010.084

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Spearman's Rank Correlation Coefficient Between IMPDH Inhibition and Risk of Acute Rejection

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
IMPDH I expression, T=0 (n=189)IMPDH I expression T=120 (n=145)IMPDH II expression, T=0 (n=196)IMPDH II expression (T=120, n=155)MPDH Activity, T=0 (n=198)IMPDH Activity, T=120 (n=155)
MMF Monotherapy0.0300.083-0.062-0.010-0.121-0.004

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Spearman's Rank Correlation Coefficient Between MPA Levels and IMPDH Activity

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
Free MPA, T=0 (n=144)Free MPA, T=120 (n=153)Total MPA, T=0 (n=152)Total MPA, T=120 (n=153)
MMF Monotherapy0.0630.0280.034-0.050

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Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Gastrointestinal Toxicity

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
Free MPA (n=300)Total MPA (n=308)AUC MPA (n=152)
MMF Monotherapy0.1060.1420.187

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Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Hematologic Toxicity

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
Free MPA (n=300)Total MPA (n=308)AUC MPA (n=152)
MMF Monotherapy-0.004-0.037-0.038

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Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Infection

The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24

Interventioncorrelation coefficient (Number)
Free MPA (n=300)Total MPA (n=308)AUC MPA (n=152)
MMF Monotherapy-0.0200.0630.030

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Total Mycophenolate Acid (MPA) by Visit and Timepoint

Drug quantification of total MPA (micrograms per milliliter [mcg/mL]) in the plasma was measured at time (T) = 0 minutes (min), 40 mins, and 120 mins. (NCT01292226)
Timeframe: Weeks 2, 4, 12, 24, and 28 (Safety Follow-Up Visit), and any unscheduled visits

Interventionmcg/mL (Mean)
T=0, Week 2 (n=41)T=0, Week 4 (n=42)T=0, Week 12 (n=35)T=0, Week 24 (n=35)T=0, safety follow-up (n=3)T=0, unscheduled visit (n=7)T=0, overall mean value (n=163)T=40, Week 2 (n=43)T=40, Week 4 (n=42)T=40, Week 12 (n=35)T=40, Week 24 (n=35)T=40, safety follow-up (n=3)T=40, unscheduled visit (n=7)T=40, overall mean value (n=165)T=120, Week 2 (n=41)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, safety follow-up (n=3)T=120, Week 24 (n=35)T=120, unscheduled visit (n=7)T=120, overall mean value (n=163)
MMF Monotherapy1.751.871.791.901.161.321.796.446.967.236.475.714.066.638.868.958.574.9110.047.038.92

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Tumor Necrosis Factor (TNF) Expression by Visit and Timepoint

TNF gene expression was measured by QRT-PCR based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of mRNA copies/cell. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits

Interventionnumber of mRNA copies/cell (Mean)
T=0, BL (n=44)T=0, Week 2 (n=41)T=0, Week 4 (n=42)T=0, Week 12 (n=34)T=0, Week 24 (n=34)T=0, safety follow-up (n=3)T=0, unscheduled visit (n=7)T=0, mean value (n=205)T=120, BL (n=0)T=120, Week 2 (n=43)T=120, Week 4 (n=42)T=120, Week 12 (n=35)T=120, Week 24 (n=34)T=120, safety follow-up (n=3)T=120, unscheduled visit (n=7)T=120, mean value (n=164)
MMF Monotherapy4532.7229960.4154101.301829.8710391.601254.2919148.9420748.3201028.9321227.1112022.839418.65397887.30220.1417512.31

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AUC0-12 of Free MPA at Day 20

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

Interventionhours*mcg/L (Mean)
MMF - Cystic Fibrosis48.530
MMF - COPD, Emphysema, IPF, or A1AD89.315

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AUC0-12 of Free MPA at Day 4

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours times micrograms per liter (hours*[mcg/L]). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

Interventionhours*mcg/L (Mean)
MMF - Cystic Fibrosis135.703
MMF - COPD, Emphysema, IPF, or A1AD80.272

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AUC0-12 of Free MPA at Day 8

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

Interventionhours*mcg/L (Mean)
MMF - Cystic Fibrosis61.155
MMF - COPD, Emphysema, IPF, or A1AD83.683

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AUC0-12 of Free MPA at Day 90

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

Interventionhours*mcg/L (Mean)
MMF - Cystic Fibrosis68.874
MMF - COPD, Emphysema, IPF, or A1AD99.212

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Cmax of Free MPA at Day 20

Cmax was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis51.822
MMF - COPD, Emphysema, IPF, or A1AD100.094

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Cmax of Free MPA at Day 4

Cmax was expressed in micrograms per liter (mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis135.275
MMF - COPD, Emphysema, IPF, or A1AD84.014

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Cmax of Free MPA at Day 8

Cmax was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis59.571
MMF - COPD, Emphysema, IPF, or A1AD95.553

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Cmax of Free MPA at Day 90

Cmax was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis87.293
MMF - COPD, Emphysema, IPF, or A1AD101.472

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Cmin of Free MPA at Day 20

Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 20 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis12.193
MMF - COPD, Emphysema, IPF, or A1AD20.237

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Cmin of Free MPA at Day 4

Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 4 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis40.188
MMF - COPD, Emphysema, IPF, or A1AD23.711

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Cmin of Free MPA at Day 8

Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 8 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis18.161
MMF - COPD, Emphysema, IPF, or A1AD15.669

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Cmin of Free MPA at Day 90

Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 90 post-transplantation

Interventionmcg/L (Mean)
MMF - Cystic Fibrosis8.102
MMF - COPD, Emphysema, IPF, or A1AD18.831

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Forced Expiratory Volume in 1 Second (FEV1) at Day 90 Post-Transplantation

FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. (NCT01014442)
Timeframe: Day 90 post-transplantation

InterventionL (Mean)
MMF - Cystic Fibrosis3.087
MMF - COPD, Emphysema, IPF, or A1AD2.834

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Forced Vital Capacity (FVC) at Day 90 Post-Transplantation

FVC at Day 90 post-transplantation is reported. (NCT01014442)
Timeframe: Day 90 post-transplantation

InterventionL (Mean)
MMF - Cystic Fibrosis3.445
MMF - COPD, Emphysema, IPF, or A1AD3.619

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Free Fraction of Free MPA at Day 20

MPA Free fraction (in %) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

Interventionpercentage of free fraction (Mean)
MMF - Cystic Fibrosis0.2922
MMF - COPD, Emphysema, IPF, or A1AD0.3052

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Free Fraction of Free MPA at Day 4

MPA Free fraction (in percent [%]) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

Interventionpercentage of free fraction (Mean)
MMF - Cystic Fibrosis0.5789
MMF - COPD, Emphysema, IPF, or A1AD0.3208

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Free Fraction of Free MPA at Day 8

MPA Free fraction (in %) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

Interventionpercentage of free fraction (Mean)
MMF - Cystic Fibrosis0.3595
MMF - COPD, Emphysema, IPF, or A1AD0.3594

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Free Fraction of Free MPA at Day 90

MPA Free fraction (in %) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

Interventionpercentage of free fraction (Mean)
MMF - Cystic Fibrosis0.2270
MMF - COPD, Emphysema, IPF, or A1AD0.3020

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Percent of Predicted FEV1 at Day 90 Post-Transplantation

FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Percent predicted FEV1 [%] = (FEV1 [L] / Predicted normal value FEV1 [L]) * 100% (NCT01014442)
Timeframe: Day 90 post-transplantation

Interventionpercentage of predicted FEV1 (Mean)
MMF - Cystic Fibrosis81.75
MMF - COPD, Emphysema, IPF, or A1AD90.17

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Percentage of Participants With Opportunistic Infections

Opportunistic infections included all infections which occurred due to aspergillus, candida, pneumocystis, cryptococcus, listeria, herpes zoster, herpes simplex, cytomegalovirus pathogens. (NCT01014442)
Timeframe: Up to Day 90

Interventionpercentage of participants (Number)
MMF - Cystic Fibrosis18.2
MMF - COPD, Emphysema, IPF, or A1AD20.0

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Area Under the Curve From Time 0 to 12 Hours (AUC0-12) of MPA, MPAG and AcMPAG at Day 4

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours time milligrams per liter (hours*[mg/L]). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

,
Interventionhours*(mg/L) (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD27.174829.1413.672
MMF - Cystic Fibrosis23.4601095.9810.414

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AUC0-12 of MPA, MPAG and AcMPAG at Day 20

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
Interventionhours*(mg/L) (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD28.2901095.379.482
MMF - Cystic Fibrosis18.925812.653.252

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AUC0-12 of MPA, MPAG and AcMPAG at Day 8

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
Interventionhours*(mg/L) (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD23.352888.969.081
MMF - Cystic Fibrosis17.841881.506.064

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AUC0-12 of MPA, MPAG and AcMPAG at Day 90

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
Interventionhours*(mg/L) (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD31.864972.697.219
MMF - Cystic Fibrosis32.365776.287.776

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Change From Baseline in Intracellular Adenosine-Tri-Phosphate (iATP) Levels

iATP was expressed in ng/mL. (NCT01014442)
Timeframe: Baseline, Days 4, 8, 20 and 90 post-transplantation

,
Interventionng/mL (Mean)
Day 4Day 8Day 20Day 90
MMF - COPD, Emphysema, IPF, or A1AD85.4144.9-45.9-114.7
MMF - Cystic Fibrosis24.0162.9-42.4-164.6

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Change From Baseline in T-Cell Phenotype

Reported values are change in the T-cell phenotype status from baseline to Day 20 and 90 for cluster of differentiation (CD) 3, CD19, CD4, CD4CD25, CD28, CD45RA, CD45RO, CD69, CD127, and CD152. (NCT01014442)
Timeframe: Baseline, Days 20 and 90 post-transplantation

,
Interventionpercentage of lymphocytes (Mean)
CD3: Change at Day 20CD3: Change at Day 90CD19: Change at Day 20CD19: Change at Day 90CD4: Change at Day 20CD4: Change at Day 90CD4CD25: Change at Day 20CD4CD25: Change at Day 90CD28: Change at Day 20CD28: Change at Day 90CD45RA: Change at Day 20CD45RA: Change at Day 90CD45RO: Change at Day 20CD45RO: Change at Day 90CD69: Change at Day 20CD69: Change at Day 90CD127: Change at Day 20CD127: Change at Day 90CD152: Change at Day 20CD152: Change at Day 90
MMF - COPD, Emphysema, IPF, or A1AD-3.01-2.811.880.61-14.71-1.93-0.53-0.10-4.44-1.04-5.11-4.88-2.83-0.430.01-0.04-3.90-2.03-10.97-15.59
MMF - Cystic Fibrosis-1.341.743.290.5913.299.002.720.894.5410.155.35-1.57-0.08-1.925.68-0.46-1.36-0.19-3.78-0.28

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CL of MPA, MPAG and AcMPAG at Day 20

CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in L/hour. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
InterventionL/hour (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD58.2951.4788232.34
MMF - Cystic Fibrosis83.0681.9338680.68

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CL of MPA, MPAG and AcMPAG at Day 8

CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in L/hour. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
InterventionL/hour (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD81.5781.7847253.81
MMF - Cystic Fibrosis90.9212.3235410.86

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CL of MPA, MPAG and AcMPAG at Day 90

CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in L/hour. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
InterventionL/hour (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD36.9221.2433223.56
MMF - Cystic Fibrosis38.0911.5701231.28

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Clearance (CL) of MPA, MPAG and AcMPAG at Day 4

CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in liters per hour (L/hour). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

,
InterventionL/hours (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD56.7091.7615140.96
MMF - Cystic Fibrosis68.4711.6754206.60

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Cmax of MPA, MPAG and AcMPAG at Day 20

Cmax was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD6.762124.4501.434
MMF - Cystic Fibrosis5.28493.8820.624

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Cmax of MPA, MPAG and AcMPAG at Day 8

Cmax was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD6.106100.1281.361
MMF - Cystic Fibrosis5.124100.1271.007

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Cmax of MPA, MPAG and AcMPAG at Day 90

Cmax was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD7.294113.4531.163
MMF - Cystic Fibrosis12.915104.9381.375

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Cmin of MPA, MPAG and AcMPAG at Day 20

Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 20 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD1.23469.0230.528
MMF - Cystic Fibrosis0.78258.4840.261

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Cmin of MPA, MPAG and AcMPAG at Day 8

Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 8 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD0.77257.1890.517
MMF - Cystic Fibrosis0.75054.5660.386

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Cmin of MPA, MPAG and AcMPAG at Day 90

Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 90 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD1.57080.4880.466
MMF - Cystic Fibrosis1.10357.9570.449

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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 20

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
Interventionhours/L (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.019230.74400.006420.0000597
MMF - Cystic Fibrosis0.014020.60590.002390.0000399

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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 4

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

,
Interventionhours/L (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.020160.61650.010560.0000594
MMF - Cystic Fibrosis0.016520.77200.007390.0000943

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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 8

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
Interventionhours/L (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.016240.62700.006440.0000580
MMF - Cystic Fibrosis0.012560.61570.004260.0000428

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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 90

AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
Interventionhours/L (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.031720.91900.007000.00001023
MMF - Cystic Fibrosis0.031260.78870.007480.0000752

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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 20

Dose-normalized Cmax was determined (in 1/L) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
Intervention1/L (Mean)
MPAMPAGAcMPAGFree MP
MMF - COPD, Emphysema, IPF, or A1AD0.004560.084300.0009690.0000666
MMF - Cystic Fibrosis0.003950.070440.0004820.0000439

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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 4

Dose-normalized Cmax was determined (in 1 per liter [1/L]) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

,
Intervention1/L (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.004220.067490.0013880.0000625
MMF - Cystic Fibrosis0.005570.082860.0011220.0000929

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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 8

Dose-normalized Cmax was determined (in 1/L) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
Intervention1/L (Mean)
MPAMPAGAcMPAFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.004350.070700.0009670.0000663
MMF - Cystic Fibrosis0.003560.069800.0007020.0000409

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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 90

Dose-normalized Cmax was determined (in 1/L) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
Intervention1/L (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD0.007420.109480.0011340.0001043
MMF - Cystic Fibrosis0.012610.106710.0013770.0000945

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Maximum Concentration (Cmax) of Mycophenolic Acid (MPA), Mycophenolic Acid Glucuronide (MPAG) and Acyl Glucuronide Metabolite of Mycophenolic Acid (AcMPAG) at Day 4

Cmax was expressed in milligrams per liter (mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 after transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD5.60891.2071.810
MMF - Cystic Fibrosis7.914117.2101.583

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Minimum Concentration (Cmin) of MPA, MPAG and AcMPAG at Day 4

Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 4 post-transplantation

,
Interventionmg/L (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD1.09447.4930.733
MMF - Cystic Fibrosis1.26766.9170.602

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Time to Maximum Concentration (Tmax) of MPA, MPAG, AcMPAG and Free MPA at Day 4

(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

,
Interventionhour (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD2.8924.8913.4051.405
MMF - Cystic Fibrosis1.8233.7132.3791.472

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Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 20

(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
Interventionhour (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD2.4634.9073.4111.366
MMF - Cystic Fibrosis2.2933.7622.8421.236

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Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 8

(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
Interventionhour (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD1.6593.6472.5461.446
MMF - Cystic Fibrosis2.1383.6692.8021.272

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Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 90

(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
Interventionhour (Mean)
MPAMPAGAcMPAGFree MPA
MMF - COPD, Emphysema, IPF, or A1AD2.1434.4203.5641.485
MMF - Cystic Fibrosis1.4503.0952.2341.490

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Volume of Distribution (Vz) of MPA, MPAG and AcMPAG at Day 4

Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation

,
InterventionLiter (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD287.7412.062705.38
MMF - Cystic Fibrosis345.4611.6811065.02

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Vz of MPA, MPAG and AcMPAG at Day 20

Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation

,
InterventionLiter (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD257.579.5331571.93
MMF - Cystic Fibrosis476.0612.7983006.18

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Vz of MPA, MPAG and AcMPAG at Day 8

Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation

,
InterventionLiter (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD331.5112.7401173.38
MMF - Cystic Fibrosis465.4512.1681432.88

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Vz of MPA, MPAG and AcMPAG at Day 90

Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation

,
InterventionLiter (Mean)
MPAMPAGAcMPAG
MMF - COPD, Emphysema, IPF, or A1AD192.539.201980.49
MMF - Cystic Fibrosis139.489.254811.15

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Percentage of Participants -Overall Participant and Graft Survival

This measure looks at the participants who did not die and/or did not receive a subsequent heart transplant. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized93.8
Cohort B: Sensitized, Crossmatch Positive93.8
Cohort B: Sensitized, Crossmatch Negative87.4

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Percentage of Participants Experiencing Acute Rejection

Acute rejection is defined as any one of the following types of rejection: acute cellular rejection (International Society for Heart and Lung Transplant (ISHLT)) system for grading rejection grade 2R or greater), acute refractory cellular rejection (acute cellular rejection unresponsive to two sequential courses of corticosteroids), acute antibody mediated rejection (histological evidence of unequivocal acute capillary injury, with complement deposition and margination of macrophages with or without neutrophils), acute mixed rejection (evidence of acute antibody mediated rejection with ISHLT grade 1R or greater), or acute clinical rejection (clinically-based acute rejection, no matter the ISHLT grade, leading to an acute augmentation of immunosuppression). (NCT01005316)
Timeframe: Transplantation to the end of study.

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized27.8
Cohort B: Sensitized, Crossmatch Positive75.0
Cohort B: Sensitized, Crossmatch Negative49.6

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Percentage of Participants Positive for de Novo Donor-Specific Alloantibody Production in the First Year Post-Transplantation

A de novo donor-specific alloantibody (DSA) is a newly developed alloantibody that is against the donor organ. This measure includes all de novo DSA (≥1000 MFI) regardless of is persistence or timing within the first year post-transplant. Alloantibodies are important mediators of acute and chronic rejection. (NCT01005316)
Timeframe: Transplantation to first year post transplant (up to 12 months post transplant).

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized22.7
Cohort B: Sensitized, Crossmatch Positive50.0
Cohort B: Sensitized, Crossmatch Negative37.8

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Percentage of Participants Positive for Event of Death, Graft Loss or Rejection With Hemodynamic Compromise at 12 Months Post-Transplantation

"This is a composite outcome of death, graft loss or rejection with hemodynamic compromise.~Rejection was considered to be with hemodynamic compromise if the rejection event had new onset echocardiographically measured from fractional shortening <26% with ≥5% fall from last echocardiogram or the rejection event had new onset of heart failure." (NCT01005316)
Timeframe: 12 months post-transplantation

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized5.2
Cohort B: Sensitized, Crossmatch Positive12.5
Cohort B: Sensitized, Crossmatch Negative11.8

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Percentage of Participants Positive for Severe Infection(s)

Severe infections are defined as a clinical illness considered likely infectious in origin that leads to hospitalization. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized34.0
Cohort B: Sensitized, Crossmatch Positive43.8
Cohort B: Sensitized, Crossmatch Negative30.7

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Percentage of Participants With Occurrence of Re-Hospitalization(s)

Hospitalization is defined as any hospitalization lasting greater than 24 hours. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized66.0
Cohort B: Sensitized, Crossmatch Positive75.0
Cohort B: Sensitized, Crossmatch Negative62.2

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Percentage of Participants With the Presence of Anti-HLA IgG Antibodies by Luminex SA Testing

Luminex SA testing was used to detect the presence of anti-HLA IgG Antibodies for all samples at a central laboratory. (NCT01005316)
Timeframe: Pre-transplantation

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized22.7
Cohort B: Sensitized, Crossmatch Positive81.3
Cohort B: Sensitized, Crossmatch Negative68.5

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Percentage of Participants With the Presence of Anti-MICA Antibodies by Luminex TM Assay

Major histocompatibility complex class I chain-related gene A (MICA) is an antigen that is a potential marker of rejection. Luminex TM assay was used to detect its presence. (NCT01005316)
Timeframe: Pre-Transplantation

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized8.2
Cohort B: Sensitized, Crossmatch Positive18.8
Cohort B: Sensitized, Crossmatch Negative11.0

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Percentage of Participants- Mortality While on Transplantation Wait-List

Death that occurred while on the transplantation wait-list, and thus before receiving a heart transplant. (NCT01005316)
Timeframe: Pre-transplantation

Interventionpercentage of participants (Number)
Enrolled, Not Transplanted39.2

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Presence of C4d on Endomyocardial Biopsy (EMB)

The biopsy of the heart stained positive for the presence of C4d, a potential marker of rejection. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

Interventionpercentage of participants (Number)
Cohort A: Non-Sensitized18.6
Cohort B: Sensitized, Crossmatch Positive62.5
Cohort B: Sensitized, Crossmatch Negative34.6

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Time From Participant Listing on Organ Wait-List to Receiving Organ Transplant, Death or De-Listing

Time (in days) from listing on the organ wait-list to receiving an organ transplant, death or de-listing. This measure is calculated as time from listing on the organ wait-list until the earliest time among transplantation, death and de-listing. (NCT01005316)
Timeframe: Study enrollment to transplantation

InterventionDays (Mean)
Enrolled128.3

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Time to Acute Rejection

Time (in days) to acute rejection. Acute rejection is defined as any one of the following types of rejection: acute cellular rejection (International Society for Heart and Lung Transplant (ISHLT) system for grading rejection grade 2R or greater), acute refractory cellular rejection (acute cellular rejection unresponsive to two sequential courses of corticosteroids), acute antibody mediated rejection (histological evidence of unequivocal acute capillary injury, with complement deposition and margination of macrophages with or without neutrophils), acute mixed rejection (evidence of acute antibody mediated rejection with ISHLT grade 1R or greater), or acute clinical rejection (clinically-based acute rejection, no matter the ISHLT grade, leading to an acute augmentation of immunosuppression). Time to acute rejection is time from transplantation to first acute rejection date. (NCT01005316)
Timeframe: Transplantation to the end of study.

InterventionDays (Mean)
Cohort A: Non-Sensitized151.0
Cohort B: Sensitized, Crossmatch Positive74.5
Cohort B: Sensitized, Crossmatch Negative124.7

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Time to Diagnosis of Chronic Rejection

Time (in days) to the diagnosis of chronic rejection. Chronic rejection is defined as stenosis, irregularity, or ectasia of the epicardial vessels, or severe peripheral pruning of the distal coronary artery tree. Time to diagnosis is time from transplantation until the first diagnosis of chronic rejection. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

InterventionDays (Mean)
Cohort A: Non-Sensitized606.8
Cohort B: Sensitized, Crossmatch PositiveNA
Cohort B: Sensitized, Crossmatch Negative398.9

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Time to New-Onset Diabetes Mellitus

Time (in days) to new-onset diabetes mellitus. New-onset diabetes mellitus is defined as the new onset of insulin dependency or the need for oral hypoglycemic agents lasting more than 30 days post-transplant. Time to new-onset diabetes is time from transplantation until the diagnosis of new-onset diabetes. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

InterventionDays (Mean)
Cohort A: Non-Sensitized73
Cohort B: Sensitized, Crossmatch Positive48
Cohort B: Sensitized, Crossmatch Negative283.4

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Time to Post-Transplantation Lymphoproliferative Disorder

Time (in days) post-transplant lymphoproliferative disorder (PTLD). PTLD is defined as histopathological evidence of lymphoid proliferation (nodal or extranodal) fulfilling the criteria of the revised classification of the WHO 2008 (Swerdlow 2008). Time to PTLD is time from transplantation until the diagnosis of PTLD. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).

InterventionDays (Mean)
Cohort A: Non-Sensitized910
Cohort B: Sensitized, Crossmatch PositiveNA
Cohort B: Sensitized, Crossmatch Negative118.7

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Time to Production of Post-Transplant de Novo Donor-specific Alloantibodies

Time (in days) from transplant to development of de novo donor-specific alloantibodies (DSA). This measure is calculated as time from transplant until the earliest time of development of any de novo DSA. The DSA is a newly developed alloantibody that is against the donor organ. Alloantibodies are important mediators of acute and chronic rejection. (NCT01005316)
Timeframe: Transplantation to first year post transplant (up to 12 months post transplant).

InterventionDays (Mean)
Cohort A: Non-Sensitized28.1
Cohort B: Sensitized, Crossmatch Positive15.4
Cohort B: Sensitized, Crossmatch Negative55.1

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Percentage of Participants -Quantification of Anti-HLA IgG Antibodies by Luminex SA Testing

Quantification of anti-HLA IgG antibodies is measured in mean fluorescence intensity (MFI). The maximum MFI for the given subject is provided. (NCT01005316)
Timeframe: Pre-transplantation

,,
Interventionpercentage of participants (Number)
MissingNoneMFI 1000-3999MFI 4000-7999MFI ≥8000
Cohort A: Non-Sensitized077.320.61.01.0
Cohort B: Sensitized, Crossmatch Negative2.429.129.914.224.4
Cohort B: Sensitized, Crossmatch Positive018.86.312.562.5

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Hazard Cox's Model Analysis of Pericardial/Pleural Effusions

Pericardial effusions: any pericardial effusion defined as at least moderate (i.e. measuring at least 2.0 cm in diastole, in the point of largest distance between the pericardial leaflets), with or without signs of hemodynamic compromise, or leading to drainage or to prolonged hospitalization. Pleural effusions: need for surgical drainage tubes for longer than 7 days after surgery and subsequent pleural effusions leading to drainage. CI = confidence interval, HR = hazard ratio, MDRD = Modification of Diet in Renal Disease (NCT01017029)
Timeframe: 6 months

Interventionparticipants (Number)
Immediate Introduction of Everolimus30
Delayed Introduction of Everolimus18

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Participants With at Least One Occurrence of Composite Treatment Failure Events

Comparison of 6-months cumulative incidence of composite treatment failure events (BPAR ≥ 2R, rejection with hemodynamic compromise, graft loss, or death) between delayed everolimus arm and immediate everolimus arm (NCT01017029)
Timeframe: 6 months

Interventionparticipants (Number)
Immediate Introduction of Everolimus33
Delayed Introduction of Everolimus26

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Participants With at Least One Occurrence of Safety Composite Endpoint After 6 Months by Treatment Group

Comparison of 6-month cumulative incidence of safety composite endpoint (wound healing delay) related to initial transplant surgery, pleural/pericardial effusions and occurrence of acute renal insufficiency, defined as estimated glomerular filtration rate (eGFR) ≤ 30 mL/min/1.73 m2, between delayed everolimus arm and immediate everolimus arm (NCT01017029)
Timeframe: 6 months

Interventionparticipants (Number)
Immediate Introduction of Everolimus40
Delayed Introduction of Everolimus30

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Absolute and Percent Frequencies of Patients With LDL ≥ 100 mg/mL at 1, 3 and 6 Months, by Treatment Group

LDL = low density lipoprotein (NCT01017029)
Timeframe: 6 months

,
Interventionparticipants (Number)
Month 1Month 3Month 6
Delayed Introduction of Everolimus383736
Immediate Introduction of Everolimus413734

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Partcipants With at Least One Occurrence of Each Safety Composite Endpoint Event After 6 Months by Treatment Group

(NCT01017029)
Timeframe: 6 months

,
Interventionparticipants (Number)
Wound healing complicationPleural effusionPericardial effusioneGFR ≤ 30 mL/min/1.73 m2
Delayed Introduction of Everolimus81188
Immediate Introduction of Everolimus101307

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Participants With CMV Infection and CMV Syndrome/Disease After 6 Months by Treatment Group

CMV infection is defined as pp65 antigenemia or DNAemia (NCT01017029)
Timeframe: 6 months

,
Interventionparticipants (Number)
CMV infectionsCMV syndrome/disease
Delayed Introduction of Everolimus636
Immediate Introduction of Everolimus463

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Chronic Allograft Damage Index (CADI) Score at Month 12 After Transplantation

CADI scoring was defined for 6 histological categories: interstitial inflammatory cell infiltration (0 equals (=) no or mild inflammation, 1=approximately (~)25 percent (%) cell infiltration, 2=26-50% cell infiltration, and 3=greater than (>)50% cell infiltration); interstitial fibrosis (0=none, 1=~25% interstitial affected, 2=26-50% interstitial affected, and 3=>50% interstitial affected); tubular atrophy (0=none, 1=~15% proximal tubular atrophy [PTA], 2=16-30% PTA, and 3=>30% PTA); mesangial matrix proliferation (MMP; 0=none, 1=25% non-glomerulosclerosis [NGS] combined with moderate MMP, 2=25-50% NGS combined with MMP, and 3=>50% NGS combined with MMP); glomerular sclerosis (0=none, 1=~15% glomerulus affected, 2=16-50% glomerulus affected, and 3=>50% glomerulus affected); endothelial proliferation (EP; 0=none, 1=EP to less than (<)25% remaining artery/small artery membrane [RA/SAM], 2=EP to 26-50% [RA/SAM], and 3=>50% [RA/SAM]). CADI score was the sum of the 6 histological findings. (NCT00758602)
Timeframe: Month 12

Interventionscore on a scale (Mean)
MMF, Standard Dose Tacrolimus1.82
MMF, Low Dose Tacrolimus2.13

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Glomerular Filtration Rate (GFR) at Month 12 After Transplantation

GFR was determined using the Cockcroft-Gault formula to calculate the creatinine clearance, at Month 12 after renal transplantation. For males, creatinine clearance [milliliters per minute (mL/min)] = [(140 minus age) multiplied by (*) (body weight in kg) divided by [72 * serum creatinine mg per deciliter (mg/dL)]. For females, creatinine clearance (mL/min) = 0.85 * [(140 minus age) * (body weight in kg)] divided by [72 * serum creatinine (mg/dL)]. (NCT00758602)
Timeframe: Month 12

InterventionmL/min (Mean)
MMF, Standard Dose Tacrolimus77.08
MMF, Low Dose Tacrolimus80.12

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Percentage of Participants With Treatment Failure at 12 Months Post-Transplant

Treatment failure was defined by the occurrence of any of the following: use of additional maintenance immunosuppressive medication not specified in the assigned treatment group; discontinuation of any of the assigned immunosuppressants for more than 14 consecutive days or 30 cumulative days; graft loss or return to chronic dialysis; or death. (NCT00758602)
Timeframe: Month 12

Interventionpercentage of participants (Number)
MMF, Standard Dose Tacrolimus9.6
MMF, Low Dose Tacrolimus6.6

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Time to First Acute Rejection Post-Transplant

The median time, in days, between randomization and acute rejection. (NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52

Interventiondays (Median)
MMF, Standard Dose Tacrolimus40
MMF, Low Dose Tacrolimus18

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Time to First Acute Rejection Post-Transplant - Number of Participants With an Event

(NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52

Interventionparticipants (Number)
MMF, Standard Dose Tacrolimus2
MMF, Low Dose Tacrolimus4

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Glomerular Filtration Rate (GFR) (mL/Min)

The mean GFR values in mL/min at BL, Weeks 2, 4, 13, 26, 39, and 52. (NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52

,
InterventionmL/min (Mean)
Baseline (n=95,96)Week 2 (n=101,102)Week 4 (n=99,98)Week 13 (n=88,91)Week 26 (n=84,83)Week 39 (n=83,77)Week 52 (n=86,82)
MMF, Low Dose Tacrolimus15.4171.0272.8775.7176.7778.0080.12
MMF, Standard Dose Tacrolimus14.3767.9571.7778.1375.9277.3477.08

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Participant and Graft Survival

The percentage of participants surviving with grafts intact at 6 and 12 months after renal transplant. (NCT00758602)
Timeframe: Months 6 and 12

,
Interventionpercentage of participants (Number)
6 months post-transplant12 months post-transplant
MMF, Low Dose Tacrolimus100.099.1
MMF, Standard Dose Tacrolimus100.0100.0

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Percentage of Participants Experiencing Acute Rejection, Graft Loss, or Death at 6 and 12 Months Post-Transplant

(NCT00758602)
Timeframe: Months 6 and 12

,
Interventionpercentage of participants (Number)
Acute rejection, 6 months post-transplantAcute rejection, 12 months post-transplantGraft loss, 6 months post-transplantGraft loss, 12 months post-transplantDeath, 6 months post-transplantDeath, 12 months post-transplant
MMF, Low Dose Tacrolimus5.25.20.00.00.00.9
MMF, Standard Dose Tacrolimus2.62.60.00.00.00.0

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Serum Creatinine (Micromoles Per Liter [µmol/L])

The mean serum creatinine values in µmol/L at Baseline (BL), Weeks 2, 4, 13, 26, 39, and 52. (NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52

,
Interventionµmol/L (Mean)
Baseline (n=95,96)Week 2 (n=101,102)Week 4 (n=99,98)Week 13 (n=88,91)Week 26 (n=84,83)Week 39 (n=83,77)Week 52 (n=86,82)
MMF, Standard Dose Tacrolimus662.93154.57118.4797.52105.70102.22103.10
MMFl, Low Dose Tacrolimus639.70115.71107.08100.1097.5195.1694.19

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Average Daily Doses (mg) of Enteric-coated Mycophenolate Acid (MPA) and Mycophenolate Mofetil (MMF) by Treatment Duration Intervals

The average daily doses of enteric-coated mycophenolate acid (MPA) and mycophenolate mofetil (MMF) at baseline and during the last 2 weeks of treatment. 1000 mg MMF = 720 mg enteric-coated MPA (MPA equivalent dose). (NCT00658333)
Timeframe: Baseline and week 4 to week 6

,
Interventionmg (Mean)
MMF Baseline Dose (n=15, 15)Week 4 to Week 6 (n=13, 14)
Enteric-coated Mycophenolate Acid1033.30914.5
Mycophenolate Mofetil1216.71567.6

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Number of Participants With Response (Yes/no)

The primary variable was the response (yes/no) with positive response being defined as an increase of 360 mg/day enteric-coated mycophenolate acid (MPA)from the baseline daily dose, tolerated and maintained for a 4 week duration until the end of the study (Week 6). Tolerability was defined as the overall assessment of improvement or no change in the intensity of physician assessed gastrointestinal (GI) symptoms at end of study as reported on the physician administered evaluation of GI symptomatology. (NCT00658333)
Timeframe: 6 weeks

,
InterventionParticipants (Number)
YesNo
Enteric-coated Mycophenolate Acid105
Mycophenolate Mofetil96

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Percentage of Participants Who Achieved Overall Response

Overall response rate (ORR) equals (=) CRR + PRR. CRR was defined as: 1. Normal serum creatinine (and with no more than a 25% increase from baseline) 2. Improvement in urinary protein:urinary creatinine ratio to ≤0.5. PRR was defined as at least 50 % reduction in proteinuria from Baseline, without more than 25% increase of serum creatinine at Week 48, compared with Baseline. If Baseline urine protein:urine creatinine ratio is >3, a urine protein:urine creatinine ratio of <3 needs to be achieved. (NCT00626197)
Timeframe: Week 48

InterventionPercentage of Participants (Number)
OCR 400 mg + SOC66.7
OCR 1000 mg + SOC67.1
Placebo + SOC54.7

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Mean Absolute Counts of Cluster of Differentiation (CD) 19 Positive (+) Cells Per Visit

CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. CD19+ cells were measured as cells per microliter (cells/uL). (NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32

,,,,,
InterventionCells/uL (Mean)
BaselineDay 15Week 4Week 16Week 32Week 48Day 1 Pre-infusionDay 1 Post-infusionDay 15 Pre-infusionDay 15 Post-infusionWeek 16 Pre-infusionWeek 16 Post-infusionWeek 32 Pre-infusionWeek 32 Post-infusion
OCR + SOC240.821.42.35.17.32408.420.92.313.60.9
OCR 1000 mg + SOC224.12.21.322.12.6222.85.72.10.92.10.820.7
OCR 400 mg + SOC256.31.91.42.67.811.7256.311.11.912.51.25.11
Placebo + SOC203.5262.7209.7125.9116.6110203.5103.1264.491.2127.852.712546.2
Placebo-Euro Lupus (EL)186.3163.3143.374.768.561.3186.3104.3164.565.876.330.972.227.6
Placebo-Mycophenolate Mofetil (MMF)213.7327.4253.4154.3141.2134.9213.7102.4322.5106.3156.564.2153.456.9

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Number of Participants Who Achieved Complete Renal Response (CRR)

CRR was defined as: 1. Normal serum creatinine (and with no more than a 25 percent [%] increase from Baseline); 2. Improvement in urinary protein:urinary creatinine ratio to less than or equal to (≤) 0.5. PRR was defined as at least 50 percent (%) reduction in proteinuria from Baseline, without more than 25% increase of serum creatinine at Week 48, compared with Baseline. If Baseline urine protein:urine creatinine ratio was greater than (>) 3, a urine protein:urine creatinine ratio of less than (<) 3 needed to be achieved. (NCT00626197)
Timeframe: Week 48

,,
InterventionPercentage of Participants (Number)
CRRPRR
OCR 1000 mg + SOC31.535.6
OCR 400 mg + SOC42.724
Placebo + SOC34.720

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Percentage of Participants With CD19+ Absolute B Cell Counts <10 Cells Per Microliter (Cells/uL)

CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. 0 represents 0% of participants. (NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32

,,,,,
InterventionPercentage of Participants (Number)
BaselineDay 15Week 4Week 16Week 32Week 48Day 1 Pre-infusionDay 1 Post-infusionDay 15 Pre-infusionDay 15 Post-infusionWeek 16 Pre-infusionWeek 16 Post-infusionWeek 32 Pre-infusionWeek 32 Post-infusion
OCR + SOC2.998.699.396.593.693.42.982.598.61009710094.4100
OCR 1000 mg + SOC1.597.110097.195.698.51.583.897.11009710095.2100
OCR 400 mg + SOC4.210098.595.891.888.74.281.21001009710093.7100
Placebo + SOC4.34.54.44.37.45.94.3104.47.54.511.53.315
Placebo-Euro Lupus (EL)00004.38.703.70009.5022.7
Placebo-Mycophenolate Mofetil (MMF)6.87.57.36.78.94.46.814711.9712.55.110.5

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Percentage of Participants With CD19+ Absolute B Cell Counts <20 Cells/uL by Visit

CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. n = number of participants analyzed at the specified visit. 0 represents 0% of participants. (NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32

,,,,,
InterventionPercentage of Participants (Number)
BaselineDay 15Week 4Week 16Week 32Week 48Day 1 Pre-infusionDay 1 Post-infusionDay 15 Pre-infusionDay 15 Post-infusionWeek 16 Pre-infusionWeek 16 Post-infusionWeek 32 Pre-infusionWeek 32 Post-infusion
OCR + SOC7.399.310098.696.594.97.289.199.310098.510097.6100
OCR 1000 mg + SOC398.510098.610098.5391.298.610098.5100100100
OCR 400 mg + SOC11.310010098.693.291.511.38710010098.510095.2100
Placebo + SOC7.17.610.311.413.217.67.122.97.417.910.4418.335
Placebo-Euro Lupus (EL)03.87.488.726.1025.94128.357.14.850
Placebo-Mycophenolate Mofetil (MMF)11.41012.213.315.613.311.420.99.321.411.632.510.326.3

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Percentage of Participants With CD19+ Absolute B Cell Counts Less Than the Lower Limit of Normal (LLN) by Visit

CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32

,,,,,
InterventionPercentage of Participants (Number)
BaselineDay 15Week 4Week 16Week 32Week 48Day 1 Pre-infusionDay 1 Post-infusionDay 15 Pre-infusionDay 15 Post-infusionWeek 16 Pre-infusionWeek 16 Post-infusionWeek 32 Pre-infusionWeek 32 Post-infusion
OCR + SOC3510010010098.698.534.898.510010010010099.2100
OCR 1000 mg + SOC33.310010010010010032.8100100100100100100100
OCR 400 mg + SOC36.610010010097.397.236.697.110010010010098.4100
Placebo + SOC27.128.839.757.152.951.527.155.730.058.256.780.35088.3
Placebo-Euro Lupus (EL)23.126.940.7728778.323.159.3286870.895.285.795.5
Placebo-Mycophenolate Mofetil (MMF)29.5303948.935.637.829.5.532.652.448.872.530.884.2

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Number of Patients Alive 24 Months Post Day 100 Transplant

Patients will be followed for survival for 24 months post day 100 transplant. (NCT00619645)
Timeframe: 24 months post day 100 transplant

InterventionParticipants (Count of Participants)
RIST for Heme Malignancies3

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Disease Progression

Transplant recipients will be assessed for disease progression at 1 year after hematopoietic cell transplantation (HCT). The outcome is reported as the number of transplant recipients who experienced disease progression. (NCT03734601)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
TBI+TLI7

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Event-free Survival (EFS) at 1 Year

Event-free survival (EFS) is defined as the number of transplant recipients remaining alive at 12 months after transplant and who did not experience disease relapse defined as blasts < 5%. The outcome is expressed as the number of transplant recipients remaining alive at 12 months after transplant without disease relapse, a number without dispersion. (NCT03734601)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
TBI+TLI16

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Full-dose Donor Chimerism (FDC) at Day 28 Following TLI/ATG/TBI Conditioning.

Subsequent to TLI/ATG/TBI conditioning, the proportion of participants with full dose donor chimerism (FDC) will be determined by Day 28. FDC is defined as achieving ≥ 95% donor type in the CD3+ lineage within 28 days of donor cell infusion, as assessed by short tandem repeat (STR) testing. The outcome will be expressed as the number of participants that achieve FDC by Day 28, a number without dispersion. (NCT03734601)
Timeframe: Day 28

InterventionParticipants (Count of Participants)
TBI+TLI13

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Non-relapse Mortality (NRM)

Non-relapse mortality (NRM) is defined as death without known disease relapse or recurrence. The outcome is expressed as the number of transplant recipients whose cause of death was not disease relapse or recurrence, a number without dispersion. (NCT03734601)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
TBI+TLI2

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Overall Survival (OS)

Overall survival (OS) is defined as the number of transplant recipients remaining alive at 12 months after transplant. The outcome is expressed as the number of transplant recipients who remained alive at 12 months after treatment, a number without dispersion. (NCT03734601)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
TBI+TLI16

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Graft vs Host Disease (GvHD)

"Recipients will be monitored for Grade 2 to 4 graft vs host disease (GvHD). The outcome is reported as the number of transplant recipients who experienced acute GvHD grades 2 to 4, the number of transplant recipients who experienced chronic and extensive GvHD. In addition, the number of transplant recipients with chronic and extensive GvHD that was refractory to treatment (persistent) is reported. Per protocol, the result for chronic extensive and persistent GvHD is based on the subset of participants that had chronic and extensive GvHD." (NCT03734601)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Acute GvHDChronic extensive GvHDChronic extensive and persistent GvHD
TBI+TLI583

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Area Under the Curve (AUC) of CD25 Saturation by Basiliximab From Day 0 to Day 84

CD25 saturation is the percentage of T cells expressing CD25. Mean AUC of CD25 was calculated only for patients who received two Simulect® injections. (NCT01596062)
Timeframe: Day 84 (Week 12) after transplantation

InterventionWeeks * Percentage of saturated CD25 (Mean)
Simulect 40mg + Neoral + Myfortic + Steroids8.4
Simulect 80mg + Neoral + Myfortic + Steroids11.1
Simulect 80mg + Certican + Myfortic + Steroids9.7

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AUC of Basiliximab Binding to CD25 Receptors From Day 0 to Day 84

Mean AUC was calculated only for patients who received two Simulect injections. (NCT01596062)
Timeframe: Day 84 (Week 12) post-transplantation

InterventionWeeks * Percentage of T cells (Mean)
Simulect 40mg + Neoral + Myfortic + Steroids7.0
Simulect 80mg + Neoral + Myfortic + Steroids9.9
Simulect 80mg + Certican + Myfortic + Steroids8.4

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Estimated Glomerular Filtration Rate (eGFR) at Day 8 and Week 24

(MDRDa formula) with imputation by last observation carried forward (LOCF) (NCT01596062)
Timeframe: Day 8, Week 24

,,
InterventionmL/min/1.73m^2 (Mean)
Day 8Week 24 (n= 3, 6, 6)
Simulect 40mg + Neoral + Myfortic + Steroids35.031.9
Simulect 80mg + Certican + Myfortic + Steroids49.954.4
Simulect 80mg + Neoral + Myfortic + Steroids53.855.7

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Percentage of Participants With Biopsy Proven Acute Rejection (BPAR) According to Type and Severity

Antibody mediated acute rejection: C4d deposition, presence of circulating antidonor antibody, morphologic evidence of acute tissue injury such as acute tubular necrosis-like minimal inflammation or capillary and/or glomerular inflammation and/or thromboses or arterial inflammation. Cellular acute rejection: acute T-cell mediated rejection Type IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells) Type IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells) Type IIA: Mild to moderate intimal arteritis. Type IIB: Severe intimal arteritis comprising > 25% of the lumenal area. Type III: Transmural (full vessel wall thickness) arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (with accompanying lymphocytic inflammation). (NCT01596062)
Timeframe: Day 84 (Week 12), Week 24 post-transplantation

,,
InterventionPercentage of participants (Number)
Day 84 (Week 12): Antibody mediated AR - NoDay 84 (Week 12): Antibody mediated AR - YesDay 84 (Week 12): Cellular AR - NoDay 84 (Week 12): Cellular AR - YesDay 84 (Week 12): Banff type lADay 84 (Week 12): Banff type lBDay 84 (Week 12): Banff type llADay 84 (Week 12): Banff type llBDay 84 (Week 12): Banff type lll (C AR)Week 24: Antibody mediated AR - NoWeek 24: Antibody mediated AR - YesWeek 24: Cellular AR - NoWeek 24: Cellular AR - YesWeek 24: Banff type lAWeek 24: Banff type lBWeek 24: Banff type llAWeek 24: Banff type llBWeek 24: Banff type lll
Simulect 40mg + Neoral + Myfortic + Steroids66.733.3100.00.00.00.00.00.00.066.733.3100.00.00.00.00.00.00.0
Simulect 80mg + Certican + Myfortic + Steroids85.714.357.142.914.314.30.014.30.085.714.357.142.90.028.60.014.30.0
Simulect 80mg + Neoral + Myfortic + Steroids100.00.083.316.70.016.70.00.00.0100.00.083.316.70.016.70.00.00.0

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Percentage of Participants With of Biopsy Proven Acute Rejection (BPAR)

BPAR is one of the components of treatment failure. One assessment of efficacy was BPAR. Renal graft biopsies were performed and the renal tissue was examined to determine if there was acute rejection of the renal transplant. (NCT01596062)
Timeframe: Day 84 (Week 12), Week 24 post-transplantation

,,
InterventionPercentage of participants (Number)
Day 84 (Week 12): NoDay 84 (Week 12):YesWeek 24: NoWeek 24:Yes
Simulect 40mg + Neoral + Myfortic + Steroids66.733.366.733.3
Simulect 80mg + Certican + Myfortic + Steroids42.957.142.957.1
Simulect 80mg + Neoral + Myfortic + Steroids83.316.783.316.7

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Percentage of Participants With of Treatment Failures

Treatment failure was defined either as a BPAR, a graft loss, a death or a loss to follow-up. An extended treatment failure was also defined including treated borderline lesions, BPAR, graft loss, death or loss to follow-up. Treated borderline lesions were considered as acute rejection by investigators and DMC experts. (NCT01596062)
Timeframe: Day 84 (Week 12), Week 24

,,
InterventionPercentage of participants (Number)
Day84 (Week 12): BPAR and/or borderline lesions-NoDay84 (Week 12):BPAR and/or borderline lesions-YesDay 84 (Week 12): Graft loss - NoDay 84 (Week 12): Graft loss - YesDay 84 (Week 12): Death - NoDay 84 (Week 12): Death - YesDay 84 (Week 12): Loss to follow-up - NoDay 84 (Week 12): Loss to follow-up - YesWeek 24: BPAR or borderline lesions - NoWeek 24: Graft loss - NoWeek 24: Death - NoWeek 24: Loss to follow-up - NoWeek 24: Loss to follow-up - YesWeek 24: BPAR or borderline lesions - YesWeek 24: Graft loss - YesWeek 24: Death - Yes
Simulect 40mg + Neoral + Myfortic + Steroids66.733.366.733.3100.00.0100.00.066.766.7100.0100.00.033.333.30.0
Simulect 80mg + Certican + Myfortic + Steroids14.385.7100.00.0100.00.0100.00.014.3100.0100.0100.00.085.70.00.0
Simulect 80mg + Neoral + Myfortic + Steroids66.733.3100.00.0100.00.0100.00.066.7100.0100.0100.00.033.30.00.0

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Percentage of T-cells That Bind Basiliximab to CD25 Receptors

This is the percentage of T cells binding basiliximab at all timepoints. (NCT01596062)
Timeframe: Day 0, Day 1, Day 4, Day 6, Day 14, Day 21, Day 28, Day 42, Day 56 and Day 84 (Week 12) post-transplantation

,,
InterventionPercentage of T cells (Mean)
Day 0 (before injection)Day 0 (2 hours after injection)Day 1 (n= 3, 5, 7)Day 4 (before injection): (n= 3, 6, 6)Day 4 (2 hours after injection): (n= 3, 5, 5)Day 6: (n= 3, 6, 6)Day 14: (n= 3, 6, 6)Day 21: (n= 3, 6, 6)Day 28: (n= 3, 6, 6)Day 42: (n= 3, 6, 5)Day 56: (n= 3, 6, 5)Day 84 (Week 12): (n= 3, 6, 5)
Simulect 40mg + Neoral + Myfortic + Steroids0.095.370.0100.0100.091.7100.089.076.347.353.06.7
Simulect 80mg + Certican + Myfortic + Steroids4.781.691.453.081.298.393.885.882.778.875.215.8
Simulect 80mg + Neoral + Myfortic + Steroids0.096.264.859.083.499.089.087.791.084.088.857.7

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Proportion of CD3+, CD4+, CD8+, CD19+ and CD56+ T Cells

Cell counts of various subpopulations of T, B and NK lymphocytes (CD3, CD4, CD8, CD19 and CD56) (flow cytometry). (NCT01596062)
Timeframe: Day 0, Day 6, Day 42, Day 84 (Week 12)

,,
Intervention10^9 cells/L (Mean)
CD3 cells count at Day 0 (before injection)CD3 cells count at Day 6: (n= 3, 6, 6)CD3 cells count at Day 42: (n= 3, 6, 5)CD3 cells count at Day 84 (Week 12): (n= 3, 6, 6)CD4 cells count at Day 0 (before injection)CD4 cells count at Day 6: (n= 3, 6, 6)CD4 cells count at Day 42: (n= 3, 6, 5)CD4 cells count at Day 84 (Week 12): (n= 3, 6, 6)CD8 cells count at Day 0 (before injection)CD8 cells count at Day 6: (n= 3, 6, 6)CD8 cells count at Day 42: (n= 3, 6, 5)CD8 cells count at Day 84 (Week 12): (n= 3, 6, 6)CD19 cells count at Day 0 (before injection)CD19 cells count at Day 6: (n=3, 6, 6)CD19 cells count at Day 42: (n= 3, 6, 5)CD19 cells count at Day 84 (Week 12): (n= 3, 6, 6)CD56 cells count at Day 0 (before injection)CD56 cells count at Day 6: (n= 3, 6, 6)CD56 cells count at Day 42: (n= 3, 6, 5)CD56 cells count at Day 84 (Week 12): (n= 3, 6, 6)
Simulect 40mg + Neoral + Myfortic + Steroids0.50.60.50.80.40.50.30.60.10.20.10.20.10.10.10.20.10.10.10.1
Simulect 80mg + Certican + Myfortic + Steroids0.70.91.11.10.50.60.80.70.20.20.40.30.10.20.20.10.20.20.20.1
Simulect 80mg + Neoral + Myfortic + Steroids0.91.11.21.20.60.70.80.80.30.30.30.30.10.20.20.10.20.10.20.1

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Saturation Rate of CD25 Antigen Saturation by Basiliximab

CD25 saturation is the percentage of T cells expressing CD25 (NCT01596062)
Timeframe: Day 0, Day 1, Day 4, Day 6, Day 14, Day 21, Day 28, Day 42, Day 56 and Day 84 (Week 12) post-transplantation

,,
Interventionpercentage of T cells (Mean)
Day 0 (before injection)Day 0 (2 hours after injection)Day 1Day 4 (before injection); (n= 3, 6, 6)Day 4 (2 hours after injection): (n= 3, 6, 5)Day 6: (n= 3, 6, 6)Day 14: (n= 3, 6, 6)Day 21: (n= 3, 6, 6)Day 28: (n= 3, 6, 6)Day 42: (n= 3, 6, 5)Day 56: (n= 3, 6, 5)Day 84 (Week 12): (n= 3, 6, 5)
Simulect 40mg + Neoral + Myfortic + Steroids0.093.795.7100.096.798.798.3100.095.378.365.00.0
Simulect 80mg + Certican + Myfortic + Steroids0.094.799.093.784.894.097.392.592.799.294.214.2
Simulect 80mg + Neoral + Myfortic + Steroids0.096.797.796.296.3100.096.794.5100.0100.093.367.5

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3.0% or Greater Improvement From Baseline in FVC-%.

The time (in months) required for each treatment arm to achieve a 3.0% or greater improvement from baseline in the FVC-% over the 18-month treatment period. (NCT03221257)
Timeframe: Baseline to 18 months

Interventionmonths (Median)
Placebo (Plac) + Mycophenolate (MMF)17.8
Pirfenidone (PFD) + Mycophenolate (MMF)12.3

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Forced Vital Capacity Volume (FVC, in ml)

Change from baseline to month 18 in the Forced Vital Capacity volume (FVC, in ml) (NCT03221257)
Timeframe: Baseline to 18 months

Interventionml (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)121.53
Pirfenidone (PFD) + Mycophenolate (MMF)112.33

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Greater Than 5% Improvement in FVC-%

The percentage of subjects in each treatment arm achieving greater than a 5% improvement in FVC-% over the 18-month treatment period. (NCT03221257)
Timeframe: Baseline to 18 months

InterventionParticipants (Count of Participants)
Placebo (Plac) + Mycophenolate (MMF)6
Pirfenidone (PFD) + Mycophenolate (MMF)9

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Health Assessment Questionnaire Modified for Scleroderma (HAQ-DI)

"Change from baseline to month 18 as a subjective measure of dyspnea and quality of life.~HAQ-DI ranges from 0 (no disability) to 3 (severe disability)." (NCT03221257)
Timeframe: Baseline to 18 months

Interventionscore on a scale (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)-0.03
Pirfenidone (PFD) + Mycophenolate (MMF)-0.17

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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Interstitial Lung Disease Score in the Lobe of Maximal Involvement (QILD-LM)

"Change from screening to month 18 in computer-generated scoring of HRCT data for the percentage of imaging pixels exhibiting features characteristic for any of three patterns of ILD (including QGG, QLF and QHC) within the lobe of maximal involvement at baseline.~Individual image scores range 0 to 100%, with higher percentages representing greater extent of quantitative interstitial lung disease." (NCT03221257)
Timeframe: Screening to 18 months

Interventionpercent (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)2.99
Pirfenidone (PFD) + Mycophenolate (MMF)-0.99

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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Interstitial Lung Disease Score in the Whole Lung (QILD-WL)

"Change from screening to month 18 in computer-generated scoring of HRCT data from the whole lung for the percentage of imaging pixels that exhibit features of any of the three patterns of interstitial lung disease (ILD) including quantitative ground-glass opacity (QGG), lung fibrosis (QLF) and quantitative honeycombing (QHC).~Individual image scores range 0 to 100%, with higher percentages representing greater extent of quantitative interstitial lung disease." (NCT03221257)
Timeframe: Screening to 18 months

Interventionpercent (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)2.36
Pirfenidone (PFD) + Mycophenolate (MMF)-1.15

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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Lung Fibrosis Score in the Lobe of Maximal Involvement (QLF-LM)

"Change from screening to month 18 in computer-generated scoring of HRCT data for the percentage of imaging pixels that exhibit features characteristic for lung fibrosis within the lobe of maximal involvement at baseline.~Individual image score range 0 to 100%, with higher percentages representing greater extent of quantitative lung fibrosis." (NCT03221257)
Timeframe: Screening to 18 months

Interventionpercent (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)2.57
Pirfenidone (PFD) + Mycophenolate (MMF)0.13

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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Lung Fibrosis Score in the Whole Lung (QLF-WL)

"Change from screening to month 18 in computer-generated scoring of HRCT data from the whole lung for the percentage of imaging pixels that exhibit features characteristic for lung fibrosis.~Individual image scores range 0 to 100%, with higher percentages representing greater extent of quantitative lung fibrosis." (NCT03221257)
Timeframe: Screening to 18 months

Interventionpercent (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)1.46
Pirfenidone (PFD) + Mycophenolate (MMF)-0.11

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High Resolution Computerized Tomography (HRCT) Measures of Total Lung Capacity (TLC)

"Change from screening to 18 months in quantitative HRCT measurement of TLC at maximal inspiration (HRCT-TLC).~Higher scores indicates a better outcome." (NCT03221257)
Timeframe: Screening to 18 months

Interventionml (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)70.69
Pirfenidone (PFD) + Mycophenolate (MMF)191.99

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Mahler Modified Transitional Dyspnea Index (TDI)

The change from baseline to 18 months in dyspnea. The TDI score for each of three domains ranges from -3 (major deterioration) to +3 (major improvement). The sum of all domains yields the TDI total score (-9 to +9). (NCT03221257)
Timeframe: Baseline to 18 months

Interventionscore on a scale (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)1.13
Pirfenidone (PFD) + Mycophenolate (MMF)1.99

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Modified Rodnan Skin Score (mRSS)

Change from baseline to month 18 in the mRSS. mRSS scores have a range from 0 to 51, with higher score indicating greater skin involvement. (NCT03221257)
Timeframe: Baseline to 18 months

Interventionscore on a scale (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)-5.42
Pirfenidone (PFD) + Mycophenolate (MMF)-4.96

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Percent Predicted Forced Vital Capacity (FVC-%)

Change from baseline to month 18 in the mean forced vital capacity (represented as the percentage of the age-; height-; gender-; and race-adjusted predicted value, i.e. FVC-%). (NCT03221257)
Timeframe: Baseline to 18 months

Interventionpercent predicted (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)2.24
Pirfenidone (PFD) + Mycophenolate (MMF)2.09

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Percent Predicted Single-breath Diffusing Capacity for Carbon Monoxide (DLCOHb-%)

Change from baseline to month 18 in DLCO, calculated as a percent of the age-; height-; gender-; race-; and hemoglobin-adjusted predicted value (DLCOHb-%). The raw DLCO value and adjusting it for all of these factors and presenting it as a percent of predicted (expected) is the outcome measure (DLCOHb-%). (NCT03221257)
Timeframe: Baseline to 18 months

Interventionpercent predicted (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)1.25
Pirfenidone (PFD) + Mycophenolate (MMF)1.24

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St. George's Respiratory Questionnaire (SGRQ)

Change from baseline to month 18 as a subjective measure of dyspnea and quality of life. SGRQ ranges from 0 (no impairment) to 100 (maximum impairment). (NCT03221257)
Timeframe: Baseline to 18 months

Interventionscore on a scale (Least Squares Mean)
Placebo (Plac) + Mycophenolate (MMF)-4.77
Pirfenidone (PFD) + Mycophenolate (MMF)-6.11

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Time to Withdrawal From the Study Drug or Treatment Failure

The time from start of treatment to withdrawal or removal from active drug therapy (MMF or Plac/PFD separately) for any reason will be plotted over the course of the 18-month treatment as a measure of tolerability and toxicity. Median times to withdrawal are not available for reporting as less than half of the participants discontinued the study drugs. (NCT03221257)
Timeframe: Baseline to 18 months

Interventiondays (Median)
Placebo (Plac) + Mycophenolate (MMF)NA
Pirfenidone (PFD) + Mycophenolate (MMF)NA

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Cataract - Incident Cataract

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Fluocinolone Acetonide Implant90.7
Systemic Therapy44.9

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Change in Best-corrected Visual Acuity (Change in the Numbers of Letters Read From a Standard ETDRS Eye Chart) From Baseline to 24 Months in Eyes With Uveitis

Best-corrected visual acuity was measured as the number of letters read from standard logarithmic visual acuity charts by study-certified examiners who were masked to treatment. Visual acuity was measured at all study visits. The primary outcome was eye-specific change in visual acuity from baseline to 2-year follow-up. Positive change values indicate improved vision while negative change values indicate vision has gotten worse. A change of 7.5 letters is considered clinically meaningful. (NCT00132691)
Timeframe: 24 months

Interventionletters (Mean)
Flucinolone Acetonide Implant6.0
Systemic Therapy3.2

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Change in SF-36 Mental Component Score From Baseline to 24 Months

Self-reported health related QoL was measured with the SF 36 survey. The mental component score for the SF 36 is a summary measure of mental health primarily based on the social functioning, role emotional, mental health and vitality domains. The score is scaled to a population norm with a mean of 50 and standard deviation of 10. Higher scores represent better outcomes. The mean change in scores between baseline and 24 months was calculated for each treatment group. (NCT00132691)
Timeframe: 24 months

Interventionunits on a scale (Mean)
Flucinolone Acetonide Implant2.55
Systemic Therapy-1.1

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Change in SF-36 Physical Component Score From Baseline to 24 Months

Self-reported health related QoL was measured with the SF 36 survey. The physical component score for the SF 36 is a summary measure of physical health primarily based on the physical functioning, role physical, bodily pain and general health domains of the survey. The score is scaled to a population norm with a mean of 50 and standard deviation of 10. Higher scores represent better outcomes. The mean change in scores between baseline and 24 months was calculated for each treatment group. A 3 to 5 point difference is considered to be clinically meaningful. (NCT00132691)
Timeframe: 24 months

Interventionunits on a scale (Mean)
Flucinolone Acetonide Implant1.15
Systemic Therapy-1.8

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Diabetes Mellitus

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of participants (Number)
Flucinolone Acetonide Implant1.0
Systemic Therapy3.6

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Glaucoma - Incident

Glaucoma was diagnosed by a glaucoma specialist through review of visual fields, clinical data, and fundus images. (NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Fluocinolone Acetonide Implant16.5
Systemic Therapy4.0

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Hyperlipidemia - Incident

LDL greater than or equal to 160 mg/mL (NCT00132691)
Timeframe: 24 months

Interventionpercentage of participants at risk (Number)
Flucinolone Acetonide Implant9.8
Systemic Therapy11.0

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Hypertension Diagnosis Requiring Treatment

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of participants (Number)
Flucinolone Acetonide Implant4.6
Systemic Therapy10.5

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Intraocular Pressure - Incident IOP Elevation >= 10 mmHg Above Baseline

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Fluocinolone Acetonide Implant51.8
Systemic Therapy15.5

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Intraocular Pressure - Incident IOP Greater Than or Equal to 24 mm Hg

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Flucinolone Acetonide Implant53.1
Systemic Therapy18.7

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Intraocular Pressure - Incident IOP Greater Than or Equal to 30 mm Hg

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Flucinolone Acetonide Implant32.8
Systemic Therapy6.3

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Intraocular Pressure - IOP-lowering Surgery

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Flucinolone Acetonide Implant26.2
Systemic Therapy3.7

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Intraocular Pressure (IOP) - Incident Use of IOP-lowering Medical Therapy (Percentage of Eyes With Uveitis That Were Not Being Treated With IOP-lowering Medical Therapy at Baseline and Underwent IOP Lowering Therapy During the 24 Month Follow-up.

The percentage of subjects who used topical or systemic treatment for elevated IOP at any time during the 2 year follow-up and were not on IOP-lowering therapy at baseline is reported. (NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis at risk (Number)
Flucinolone Acetonide Implant61.1
Systemic Therapy20.1

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Macular Edema

center point macular thickness >= 240 micrometers assessed on OCT (Stratus OCT-3 [Carl Zeiss Meditec, Dublin, CA]) as graded by Central Reading Center (NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis (Number)
Flucinolone Acetonide Implant22
Systemic Therapy30

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Mortality

(NCT00132691)
Timeframe: 24 months

Interventionpercentage of participants (Number)
Flucinolone Acetonide Implant1.6
Systemic Therapy0

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Uveitis Activity

Uveitis activity was determined by clinician assessment at each study visit. The study ophthalmologist evaluated each eye as active, inactive/never had uveitis or cannot assess. (NCT00132691)
Timeframe: 24 months

Interventionpercentage of eyes with uveitis (Number)
Fluocinolone Acetonide Implant12
Systemic Therapy29

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Clinically Treated Acute Rejection

"Clinically treated acute rejection is defined as patient incidence of any rejection (suspected or otherwise) for which treatment was provided. Data is reported as the percentage of patients with Clinically Treated Acute Rejection.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients22.9
Conventional High-Risk Patients21.7
Alemtuzumab Low- Risk Patients12.8
Conventional Low-Risk Patients26.9

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Efficacy Failure

"Efficacy Failure is a composite measure of biopsy confirmed acute rejection, graft loss and death. Data is reported as the percentage of patients with Efficacy Failure.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients22.9
Conventional High-Risk Patients29.0
Alemtuzumab Low- Risk Patients15.9
Conventional Low-Risk Patients24.6

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Graft Survival at 12 Months

"Graft survival is defined as no graft loss (re-transplant, return to dialysis for more than 30 days or death) with 12 months of skin closure.~Kaplan Meier analysis was used to estimate percentage of patients with event. Patients with no event by the time of the scheduled visit or whose first event was after premature discontinuation of randomized study drug or tacrolimus were censored on the scheduled day of assessment, on the day of premature treatment discontinuation or last evaluation day, whichever came first." (NCT00113269)
Timeframe: 12 months

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients95.6
Conventional High-Risk Patients92.1
Alemtuzumab Low- Risk Patients97.5
Conventional Low-Risk Patients95.1

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Overall Graft Survival

"Overall graft survival is defined as not having graft loss (re-transplant, return to dialysis for more than 30 consecutive days, or death) at any time following skin closure. Data is reported as the percentage of patients with Overall Graft Survival.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients88.6
Conventional High-Risk Patients82.6
Alemtuzumab Low- Risk Patients90.9
Conventional Low-Risk Patients91.2

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Overall Patient Incidence of BCAR

"Overall patient incidence of BCAR is defined as a suspected new rejection at any time following skin closure confirmed by a Banff Grade ≥ 1A as assigned by a local pathologist. Incidence is reported as the percentage of patients with BCAR. The Banff 97 scale is a classification system for interpreting histology of allograft biopsies. The grades range from Mild (1A & 1B) to Moderate (2A & 2B) to Severe (3).~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients15.7
Conventional High-Risk Patients13.0
Alemtuzumab Low- Risk Patients9.8
Conventional Low-Risk Patients21.6

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Overall Patient Survival

"Overall patient survival is defined as not dead at any time following skin closure. Data is reported as the percentage of patients with Overall Patient Survival.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients95.7
Conventional High-Risk Patients89.9
Alemtuzumab Low- Risk Patients93.9
Conventional Low-Risk Patients95.3

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Patient Incidence of Biopsy-confirmed Acute Rejection (BCAR) at 6 Months

"A BCAR is a suspected new rejection w/in 6 mos. of skin closure, confirmed by Banff Grade ≥1A assigned by a pathologist. The Banff 97 classification system is used for interpreting histology of allograft biopsies, including Mild (1A/1B), Moderate (2A/2B) & Severe (3).~Kaplan Meier analysis was used to estimate % of pts. w/event. Patients w/no event at time of scheduled visit or whose 1st event was after premature discontinuation of study drug/tacrolimus were censored on the scheduled day of a) assessment, b) of premature treatment discontinuation or c) last evaluation, whichever came 1st." (NCT00113269)
Timeframe: 6 months

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients6.2
Conventional High-Risk Patients9.4
Alemtuzumab Low- Risk Patients1.9
Conventional Low-Risk Patients17.5

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Patient Survival at 12 Months

"Patient survival is defined as not dead within 12 months after skin closure.~Kaplan Meier analysis was used to estimate percentage of patients with event. Patients with no event by the time of the scheduled visit or whose first event was after premature discontinuation of randomized study drug or tacrolimus were censored on the scheduled day of assessment, on the day of premature treatment discontinuation or last evaluation day, whichever came first." (NCT00113269)
Timeframe: 12 months

InterventionPercentage of Patients (Number)
Alemtuzumab High-Risk Patients98.6
Conventional High-Risk Patients96.9
Alemtuzumab Low- Risk Patients98.1
Conventional Low-Risk Patients98.7

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Time to First BCAR

"Time to first BCAR is defined as the number of days from skin closure to the first episode of BCAR.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)

InterventionDays (Median)
Alemtuzumab High-Risk Patients226
Conventional High-Risk Patients49
Alemtuzumab Low- Risk Patients469
Conventional Low-Risk Patients13

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Renal Function Abnormalities Based on Creatinine Clearance

"Increases in creatinine clearance usually indicates an improvement.~Change in creatinine clearance from month 1 was calculated.~Change from 1 month is calculated by month 36 - month 1." (NCT00113269)
Timeframe: 1 month and End of Study (36 months)

,,,
InterventionmL/s (Mean)
Month 1 (N= 65; 65; 162; 161)Month 36 (N= 48; 47; 125; 131)Change from Month 1 (N= 48; 46; 125; 129)
Alemtuzumab High-Risk Patients0.8820.9760.031
Alemtuzumab Low- Risk Patients0.9061.0110.080
Conventional High-Risk Patients0.8330.8620.039
Conventional Low-Risk Patients0.8911.0390.140

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Renal Function Abnormalities Based on Serum Creatinine

"Decrease in serum creatinine usually indicates an improvement.~Change in creatinine clearance from month 1 was calculated.~Change from 1 month is calculated by month 36 - month 1." (NCT00113269)
Timeframe: 1 month and End of Study (36 months)

,,,
Interventionmcmol/L (Mean)
Month 1 (N= 67; 65; 163; 162)Month 36 (N= 49; 47; 126; 131)Change from Month 1 (N= 49; 46; 126; 129)
Alemtuzumab High-Risk Patients151.7136.61.8
Alemtuzumab Low- Risk Patients139.0121.7-8.0
Conventional High-Risk Patients155.9152.03.4
Conventional Low-Risk Patients138.2117.1-18.7

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Disease-Free Survival (DFS)

Estimate the one-year disease-free survival (DFS) for research participants who receive this study treatment. DFS is defined as time from transplantation to the occurrence of relapse or death due to relapse. Patients who are alive at the time of analysis or die due to other causes will be censored at the time of their events. The estimated percentage of participants with DFS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: One year post-transplant

InterventionPercentage of participants (Number)
High-Risk Hematologic Malignancies70.1

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Event-free Survival (EFS)

To determine if one year event-free survival can be improved in pediatric patients undergoing a haploidentical transplant by using a reduced intensity conditioning regimen and a targeted dose T cell depleted donor product. EFS is defined as time from transplantation to the occurrence of relapse or death due to any cause. Patients who are alive at the time of analysis will be censored. The estimated percentage of participants with EFS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: one year post-transplant

InterventionPercentage of participants (Number)
High-Risk Hematologic Malignancies54.8

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Overall Survival (OS)

Estimate the one-year overall survival (OS) for research participants who receive this study treatment. OS is defined as time from transplantation to death due to any cause. Patient who are alive at the time of analysis will be censored. The estimated percentage of participants with OS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: one year post-transplant

InterventionPercentage of participants (Number)
High-Risk Hematologic Malignancies71.0

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To Estimate the Cumulative Incidence of Relapse for Research Participants Who Receive This Study Treatment.

The Cumulative Incidence of Relapse will be reported as the proportion of number of relapses since transplant to the total number of patients at risk at five years post-transplant. (NCT00566696)
Timeframe: five years post-transplant

InterventionPercentage of participants (Number)
The Cumulative Incidence of Relapse at five year pEstimate±SE
High-Risk Hematologic Malignancies30.08.6

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To Estimate the Rate of Overall Grade III-IV Acute GVHD, and the Rate and Severity of Chronic GVHD in Research Participants.

The rate of Overall Grade III-IV Acute AVHD will be report as the proportion of patients who have Grade III-IV Acute GVHD to the total patients with transplant. The rate of Chronic GVHD will be report as the proportion of patients who have Chronic GVHD to the total patients with transplant. The Severity of Chronic GVHD will be reported using CTCAE grading system, as a number. (NCT00566696)
Timeframe: five years post-transplant

InterventionPercentage of participants (Number)
Rate of Overall Grade III-IV Acute AVHDRate of limited grade Chronic GVHD
High-Risk Hematologic Malignancies22.589.68

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Proportion of Participants Successfully Withdrawn and Remain Off Immunosuppressants

This measure of tolerance induction includes the proportion of participants who qualify for immunosuppression withdrawal as determined by a review of individual clinical results by a protocol withdrawal committee, were successfully withdrawn from immunosuppressants, and remained off immunosuppressants at the time the trial ended. Successful withdrawal definition: participants who remain off immunosuppression for at least 8 weeks and do not restart immunosuppressant drugs after successful withdrawal. (NCT00105235)
Timeframe: From 1 year post- transplantation until study completion or participant termination (participants followed up to 48 months post-transplant)

InterventionProportion of Participants (Number)
Alemtuzumab0.1

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Proportion of Participants Successfully Withdrawn From Immunosuppressants

This measure of tolerance induction includes the proportion of participants who qualify for immunosuppression withdrawal as determined by a review of individual clinical results by a protocol withdrawal committee. Successful withdrawal definition: participants who remain off immunosuppression for at least 8 weeks. (NCT00105235)
Timeframe: From 1 year post- transplantation until study completion or participant termination (participants followed up to 48 months post-transplant)

InterventionProportion of Participants (Number)
Alemtuzumab0.2

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Proportion of Participants Who Had Graft Loss or Death

Proportion of participants who had liver graft loss or who died or terminated from the study within 2 years of initiating immunosuppression withdrawal (NCT00105235)
Timeframe: Within 2 years after initiation of immunosuppression withdrawal

InterventionProportion of Participants (Number)
Alemtuzumab0

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Proportion of Participants Who Have Graft Loss or Death

Proportion of participants who had liver graft loss or who died within 1 year of undergoing transplantation. Note: Participants who discontinued treatment or terminated the study prior to 1 year post transplantation are considered treatment failures and are included in this measure. (NCT00105235)
Timeframe: Within 1 year of post-transplantation

InterventionProportion of Participants (Number)
Alemtuzumab0.22

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Change in Glomerular Filtration Rate Estimated by Iohexol Plasma Clearance 12 Months After Randomization Between the 2 Groups of Patients.

Primary efficacy endpoint: between treatment analysis of change in iohexol plasmatic clearance (mL/min) from baseline to Month 12 (M12) (NCT00425308)
Timeframe: From Baseline to Month 12

Intervention(mL/min) (Least Squares Mean)
Cyclosporine-4.07
Enteric-coated Mycophenolate Sodium (EC-MPS)10.30

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Change in the Glomerular Filtration Rate Estimated by Iohexol Plasma Clearance 12 Months After Randomization Between the 2 Groups of Patients Who Completed Trial

Primary efficacy endpoint: between treatment analysis of change in iohexol plasmatic clearance (mL/min) from baseline to Month 12 (M12) (NCT00425308)
Timeframe: From Baseline to Month 12

Intervention(mL/min) (Least Squares Mean)
Cyclosporine1.46
Enteric-coated Mycophenolate Sodium (EC-MPS)12.00

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Assessing Cardiovascular Risk Factors Based on Fasting C-reactive Protein (CRP).

Blood chemistry - C-reactive Protein (CRP) (mg/L) (NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12

,
Interventionmg/L (Mean)
Baseline [N = 13; N = 14]Month 3 [N = 11; N = 11]Month 6 [N = 11; N = 9]Month 12 [N = 14; N = 12]
Cyclosporine11.46.38.034.2
Enteric-coated Mycophenolate Sodium (EC-MPS)3.010.35.612.6

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Assessing Cardiovascular Risk Factors Based on Fasting Glucose.

Blood chemistry - fasting glycemia (mmol/L) (NCT00425308)
Timeframe: From Baseline to Month 1, 3, 6, 9, and 12

,
Interventionmmol/L (Mean)
Baseline [N = 15; N = 15]Month 1 [N = 15; N = 15]Month 3 [N = 14; N = 15]Month 6 [N = 14; N = 14]Month 9 [N = 12; N = 13]Month 12 [N = 14; N = 13]
Cyclosporine5.55.25.65.55.34.8
Enteric-coated Mycophenolate Sodium (EC-MPS)5.25.35.25.15.65.4

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Assessing Cardiovascular Risk Factors Based on Fasting High-density Lipoprotein (HDL) Cholesterol, Low-density Lipoprotein (LDL) Cholesterol.

(NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12

,
Interventionmmol/L (Mean)
Baseline LDL [N = 10; N = 14]Baseline HDL [N = 10; N = 14]Month 3 LDL [N = 12; N = 14]Month 3 HDL [N = 12; N = 13]Month 6 LDL [N = 12; N = 12]Month 6 HDL [N = 12; N = 12]Month 12 LDL [N = 10; N = 11]Month 12 HDL [N = 10; N = 11]
Cyclosporine3.41.53.21.73.21.63.01.7
Enteric-coated Mycophenolate Sodium (EC-MPS)3.11.53.41.53.31.43.21.5

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Assessing Cardiovascular Risk Factors Based on Fasting Total Cholesterol.

Blood chemistry - total cholesterol (mmol/L) (NCT00425308)
Timeframe: From Baseline to Month 1, 3, 6, 9, and 12

,
Interventionmmol/L (Mean)
Baseline [N = 13; N = 14]Month 1 [N = 11; N = 14]Month 3 [N = 13; N = 14]Month 6 [N = 12; N = 12]Month 9 [N = 9; N = 12]Month 12 [N = 12; N = 11]
Cyclosporine5.75.05.35.55.45.6
Enteric-coated Mycophenolate Sodium (EC-MPS)6.05.75.65.75.45.8

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Assessing Cardiovascular Risk Factors Based on Fasting Triglycerides.

(NCT00425308)
Timeframe: From Baseline to Month 1, 3, 6, 9, and 12

,
Interventionmmol/L (Mean)
Baseline [N = 11; N = 14]Month 1 [N = 10; N = 14]Month 3 [N = 13; N = 14]Month 6 [N = 12; N = 12]Month 9 [N = 10; N = 12]Month 12 [N = 12; N = 11]
Cyclosporine2.42.12.01.91.92.3
Enteric-coated Mycophenolate Sodium (EC-MPS)1.82.02.22.12.22.3

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Change in Renal Function Assessed by Creatinine Clearance at Month 3, Month 6 and Month 12

Change in creatinine clearance, Nankivell formula (mL/min/1.73m²) from baseline to M12 (NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12

,
InterventionmL/min/1.73m² (Mean)
Baseline [N = 12; N = 13]Month 3 [N = 10; N = 14]Month 6 [N = 10; N = 12]Month 12 [N = 7; N = 11]
Cyclosporine56.357.350.348.7
Enteric-coated Mycophenolate Sodium (EC-MPS)60.367.168.170.1

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Change in Renal Function Assessed by Proteinuria at Month 3, Month 6 and Month 12

Change in proteinuria (g/24h) from baseline to M12 (NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12

,
Interventiong/24h (Mean)
Baseline [N = 9; N = 10]Month 3 [N = 9; N = 8]Month 6 [N = 6; N = 5]Month 12 [N = 7; N = 10]
Cyclosporine0.440.530.440.48
Enteric-coated Mycophenolate Sodium (EC-MPS)0.470.240.330.75

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Change in Renal Function Assessed by Serum Creatinine at Month 3, Month 6 and Month 12

(NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12

,
Interventionµmol/L (Mean)
Baseline [N = 13; N = 14]Month 3 [N = 12; N = 14]Month 6 [N = 12; N = 13]Month 12 [N = 13; N = 14]
Cyclosporine161.7156.6170.8227.7
Enteric-coated Mycophenolate Sodium (EC-MPS)153.2138.9139.8130.4

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Number of Participants With Biopsy-proven Acute Rejection (BPAR) at Month 6 and Month 12.

(NCT00425308)
Timeframe: Month 6 and 12

,
Interventionparticipants (Number)
Yes: treatment failure Month 6No: treatment failure Month 6Yes: treatment failure Month 12No: treatment failure Month 12
Cyclosporine00112
Enteric-coated Mycophenolate Sodium (EC-MPS)00014

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Number of Participants With Treatment Failures Assessed by Biopsy-proven Acute Rejection (BPAR), Graft Loss/Re-transplantation, Death or Lost to Follow-up at Month 12.

(NCT00425308)
Timeframe: Month 12

,
Interventionparticipants (Number)
BPAR 12 monthsGraft Loss 12 monthsDeath 12 monthsLost to Follow-up 12 months
Cyclosporine1010
Enteric-coated Mycophenolate Sodium (EC-MPS)0000

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Number of Participants With the Occurrence of Treatment Failures at 6 Months Post-transplantation

Treatment failures defined as Biopsy Proven Acute Rejection (BPAR), graft loss, death or loss to follow-up. Only BPAR from other biopsies than the protocol defined biopsy at Month 3 are described. Acute rejection: an episode of acute renal dysfunction diagnosed as rejection on the basis of biopsy or clinical observations, treated with anti-rejection medication. BPAR: renal transplant biopsy finding of acute cellular or antibody mediated rejection. Graft loss: allograft will be presumed to be lost on the day the patient starts dialysis and is not able to subsequently be removed from dialysis. (NCT00413920)
Timeframe: 6 months post transplantation

InterventionNumber of participants (Number)
Without Steroids20
With Steroids16

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Number of Participants Requiring Steroids in Non-steroid Treatment Group

(NCT00413920)
Timeframe: Months 3 and 6

InterventionNumber of participants (Number)
3 Months6 Months
Without Steroids2520

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Number of Participants With Subclinical Histological Rejections

The number of participants with subclinical histological rejections was determined by renal biopsy screening at 3 months in 125 patients, providing adequate samples for 112 biopsies. (NCT00413920)
Timeframe: Month 3

,
InterventionNumber of participants (Number)
Sample quality inadequateSubclinical rejectionBorderline lesionsBPAR
With Steroids617512
Without Steroids712210

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Number of Participants With Treatment Failure at 3 Months by Graft Recovery Status

"The number of participants with treatment failure defined as a Biopsy Proven Acute Rejection (BPAR), a graft loss, a death, or a loss to follow-up at 3 months by graft recovery status.~Delayed graft function is defined as the need for dialysis within the first 7 days post-transplantation, excluding the first post-transplantation day.~Slow graft function is defined as a serum creatinine value > 250 µmol/L at day 5." (NCT00413920)
Timeframe: Month 3

,
InterventionNumber of participants (Number)
Delayed Graft Function [N= 25,24]Slow Graft Function [N= 36,23]Immediate Graft Function [N= 51,63]
With Steroids413
Without Steroids863

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Number of Participants With Treatment Failure, BPAR, Clinical Acute Rejection (AR) and Treated AR at 3 Months

A treatment failure is a Biopsy Proven Acute Rejection (BPAR), a graft loss, a death, or a loss to follow-up. Only BPAR from other biopsies than the protocol defined biopsy at Month 3 are described. Acute rejection: an episode of acute renal dysfunction diagnosed as rejection on the basis of biopsy or clinical observations, treated with anti-rejection medication. BPAR: renal transplant biopsy finding of acute cellular or antibody mediated rejection. Graft loss: The allograft will be presumed lost on the day the patient starts dialysis and is not able to subsequently be removed from dialysis. (NCT00413920)
Timeframe: Month 3

,
InterventionNumber of participants (Number)
Treatment FailureBiopsy Proven Acute RejectionGraft LossDeathLoss to Follow-upAcute RejectionTreated Acute Rejection
With Steroids851201916
Without Steroids17105203231

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The Number of Participants With BPAR, Clinical Acute Rejection (AR) and Treated AR at 6 Months

If a participant experienced several BPAR, only the rejection with highest grade is taken into account. Only events that occurred before study treatment discontinuation are taken into account. Only BPAR from other biopsies than the protocol defined biopsy at Month 3 are described. Acute rejection: an episode of acute renal dysfunction diagnosed as rejection on the basis of biopsy or clinical observations, treated with anti-rejection medication. BPAR: renal transplant biopsy finding of acute cellular or antibody mediated rejection. (NCT00413920)
Timeframe: Month 6

,
InterventionNumber of participants (Number)
Biopsy Proven Acute RejectionGraft LossDeathLoss to Follow-upAcute RejectionTreated Acute Rejection
With Steroids83502119
Without Steroids135203736

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The Number of Participants With Subclinical Rejection as Evaluated by a Change in Liver Enzymes

The number of participants with subclinical rejection episodes as defined by a steroid-sensitive, clinically relevant increase of AST, ALT, gamma-GT, AP or bilirubin (i.e., elevation of one or more of these enzymes that was considered clinically relevant and showed resolution upon treatment with a slight increase of steroid dosage). (NCT00405652)
Timeframe: 12-20 weeks

InterventionParticipants (Number)
Enteric-coated Mycophenolate Sodium6

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Number of Patients With Treatment Failure 6-months Post Transplant Measured by the Combined Incidence of Biopsy Proven Acute Rejection, Graft Loss, and Death

To evaluate therapeutic benefit by comparing the efficacy defined as the number of participants with treatment failure (biopsy-proven acute rejection [BPAR], graft loss [GFL] or death) at 6 months post-transplant. BPAR was defined as a biopsy graded IA, IB, IIA, IIB or III using Banff 2000 classification. A graft core biopsy was performed within 24 hours of initiation of anti-rejection therapy. GFL was defined as the day the allograft was presumed lost (the day the patient started dialysis, the day of nephrectomy or the day of irreversible graft loss demonstrated by imaging techniques.) (NCT00419926)
Timeframe: 6 months

Interventionnumber of participants (Number)
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen33
Standard Mycophenolate Sodium (Myfortic) Dosing Regimen36
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen26
Standard Mycophenolate Sodium (Myfortic) Dosing Regimen35

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Comparison of Overall Treatment Failure at Days 21 and 84 Post-transplantation Assessed by Biopsy Proven Acute Rejection (BPAR), GFL, and Death

The overall treatment differences of the number of participants with at least one occurrence of the composite event BPAR, GFL or death at study days 21 and 84 post-transplantation. BPAR was defined as a biopsy graded IA, IB, IIA, IIB or III using Banff 2000 classification. A graft core biopsy was performed within 24 hours of initiation of anti-rejection therapy. GFL was defined as the day the allograft was presumed lost (the day the patient started dialysis, the day of nephrectomy or the day of irreversible graft loss demonstrated by imaging techniques.) (NCT00419926)
Timeframe: 21 and 84 days

,
Interventionnumber of participants (Number)
Day 84Day 21
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen20331426
Standard Mycophenolate Sodium (Myfortic) Dosing Regimen21342033

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Renal Function Assessed by Glomerular Filtration Rate (GFR)at Each Visit

The Modification of Diet in Renal Disease (MDRD) formula was used to calculate the GFR. Serum creatinine levels, age, sex and race were used to estimate the GFR levels in mL/min/1.73m^2. (NCT00419926)
Timeframe: at 21 days, 84 days and 180 days

,
Intervention(mL/min/1.73m^2) (Mean)
At 21 daysAt 84 daysAt 180 days
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen47.352.153.5
Standard Mycophenolate Sodium (Myfortic) Dosing Regimen46.851.851.3

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Graft Survival

The median time, in months, between randomization and graft loss event. Participants were censored at the date of last follow up, the date of last contact or premature withdrawal, and date of death. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12, 28 days after Month 12 or last dose of study treatment, and 6 and 12 months after the last dose of study treatment.

Interventionmonths (Median)
Adjusted MMF + Tacrolimus + CS12.9
Fixed-Dose MMF + Tacrolimus + CS12.9

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Overall Survival (OS) at Month 12 - Percentage of Participants With an Event

OS was defined as the time between the date of randomization and death up to Month 12. Participants were censored at the date of last follow up and the date of last contact or premature withdrawal. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12

Interventionpercentage of participants (Number)
Adjusted MMF + Tacrolimus + CS8.9
Fixed-Dose MMF + Tacrolimus + CS11.1

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Overall Survival at Month 12

The median time, in months, between randomization and OS event. Participants were censored at the date of last follow up and the date of last contact or premature withdrawal. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12

Interventionmonths (Median)
Adjusted MMF + Tacrolimus + CS12.9
Fixed-Dose MMF + Tacrolimus + CS12.9

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Percentage of Participants With Graft Loss

Graft survival was defined as the time between the randomization date and the graft loss date. Participants were censored at the date of last follow up, the date of last contact or premature withdrawal, and date of death. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12, 28 days after Month 12 or last dose of study treatment, and 6 and 12 months after the last dose of study treatment.

Interventionpercentage of participants (Number)
Adjusted MMF + Tacrolimus + CS2.2
Fixed-Dose MMF + Tacrolimus + CS5.6

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Percentage of Participants With Treated Biopsy Proven Acute Rejection (BPAR) According to Banff Criteria up to 12 Months Post-Transplant

Banff criteria required at least 2 of the 3 following features for a histopathological diagnosis of acute rejection: portal inflammation, bile duct inflammation, and venous endothelial inflammation. Each item was graded from 0 to 3 where 0 equals (=) mild, 2 = moderate, and 3 = severe. The sum of the 3 individual scores, from 0 to 9, corresponded to the Rejection Activity Index (RAI). If RAI = 0, 1, or 2, there was no evidence of rejection. If RAI = 3, there was borderline acute rejection. If RAI = 4 or 5, there was mild acute rejection. If RAI = 6 or 7, there was moderate acute rejection. If RAI = 8 or 9, there was severe acute rejection. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12, 28 days after Month 12 or last dose of study treatment, and 6 and 12 months after the last dose of study treatment

Interventionpercentage of participants (Number)
Adjusted MMF + Tacrolimus + CS8.0
Fixed-Dose MMF + Tacrolimus + CS8.2

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Percentage of Participants by Graft Histology at 12 Months Post-Transplant - Central Review

The percentage of participants with biopsies of grafts evaluated by central review and scored according to Banff criteria at Month 12 post-transplant. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12

,
Interventionpercentage of participants (Number)
Normal liverMinor lesionsAcute rejectionChronic rejectionChronic hepatitisVascular lesionsPathology of biliary obstructionLobular hepatitisRecurrence of initial autoimmune diseaseOther
Adjusted MMF + Tacrolimus + CS4.84.80.07.126.235.74.84.80.035.7
Fixed-Dose MMF + Tacrolimus + CS11.417.10.02.922.911.45.72.90.045.7

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Number of Participants With Engraftment Syndrome

(NCT00412360)
Timeframe: Day 100 post-transplant

InterventionParticipants (Count of Participants)
Single UCB Transplant11
Double UCB Transplant7

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Percentage of Participants With Disease-free Survival

Disease-free survival is defined as survival without relapse of the primary disease. (NCT00412360)
Timeframe: 1 year post-randomization

Interventionpercentage of participants (Number)
Single UCB Transplant70
Double UCB Transplant64

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Percentage of Participants With Overall Survival

Overall survival is defined as survival of death from any cause. (NCT00412360)
Timeframe: 1 year post-randomization

Interventionpercentage of participants (Number)
Single UCB Transplant73
Double UCB Transplant65

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Percentage of Participants With Relapse

Relapse is defined by either morphological or cytogenetic evidence of AML, ALL, CML, or MDS consistent with pre-transplant features. Testing for recurrent malignancy in the blood, marrow or other sites will be used to assess relapse after transplantation. (NCT00412360)
Timeframe: 1 year post-randomization

Interventionpercentage of participants (Number)
Single UCB Transplant12
Double UCB Transplant14

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Percentage of Participants With Acute Graft-versus-host Disease (GVHD)

"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL~2-3 mg/dL~3.01-6 mg/dL~6.01-15.0 mg/dL~>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT00412360)
Timeframe: Day 100 post-randomization

,
Interventionpercentage of participants (Number)
Acute GVHD Grade II-IVAcute GVHD Grade III-IV
Double UCB Transplant5623
Single UCB Transplant5713

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Percentage of Participants With Chronic GVHD

Incidences of chronic GVHD will be graded per Shulman et al. 1980. This reference categorizes chronic GVHD as either limited or extensive. For this outcome, participants developing either type are considered to have a chronic GVHD event. (NCT00412360)
Timeframe: 1 year post-randomization

,
Interventionpercentage of participants (Number)
Chronic GVHDExtensive Chronic GVHD
Double UCB Transplant3215
Single UCB Transplant309

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Percentage of Participants With Neutrophil and Platelet Engraftment

Neutrophil engraftment is defined as achieving an absolute neutrophil count greater than 500x10^6/liter for three consecutive measurements on different days. The first of the three days will be designated the day of neutrophil engraftment. Platelet engraftment is defined as achieving platelet counts greater than 50,000/microliter for consecutive measurements over 7 days without requiring platelet transfusions. The first of the 7 days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 7 days. (NCT00412360)
Timeframe: Days 42 and 100

,
Interventionpercentage of participants (Number)
Neutrophil Engraftment at Day 42Platelet Engraftment at Day 100
Double UCB Transplant8865
Single UCB Transplant8976

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Time to Neutrophil and Platelet Engraftment

Platelet engraftment is defined as achieving platelet counts greater than 50,000/microliter for consecutive measurements over 7 days without requiring platelet transfusions. The first of the 7 days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 7 days. (NCT00412360)
Timeframe: 2 years post-transplant

,
Interventiondays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Double UCB Transplant2384
Single UCB Transplant2158

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Number of Participants Using Antihyperlipidemic Medications at Month 12 - Intent to Treat Population

Participants using > = 1 antihyperlipidemic medication at Month 12. (NCT00455013)
Timeframe: Month 12

Interventionparticipants (Number)
Belatacept, Mycophenolate Mofetil (MMF)11
Belatacept, Sirolimus10
Tacrolimus, MMF9

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Number of Participants With Acute Rejection of Transplant up to End of Month 48 Post Transplantation - Intent to Treat Population in Long Term Extension

AR defined as a clinicopathological event requiring clinical evidence and biopsy confirmation by central pathologist. One or more conditions were met and a renal biopsy revealed histologic evidence of rejection: unexplained rise of serum creatine (SCr) >= 25 % from baseline plus one or more of the following: unexplained decreased urine output; fever and graft tenderness; a SCr that remained elevated within 14 days after transplantation and clinical suspicion of AR; reason other than those listed and participant was treated for this episode. Day 1 was day of transplantation. Banff grade used Banff 97 working classification of kidney transplant pathology. ITT population was all randomized and transplanted participants. (NCT00455013)
Timeframe: End of Month 12 to end of Month 48 Post Transplantation

Interventionparticipants (Number)
Belatacept, Mycophenolate Mofetil (MMF)0
Belatacept, Sirolimus0
Tacrolimus, MMF0

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Number of Participants With Composite of Death, Graft Loss and Acute Rejection up to Month 12 - Intent to Treat Population

Subjects with graft loss or death prior to Month 12 were considered having an event of AR, therefore, the incidence of AR was reported as a composite of AR, death, and graft loss. (NCT00455013)
Timeframe: Day 1 up to Month 12

Interventionparticipants (Number)
Belatacept, Mycophenolate Mofetil (MMF)7
Belatacept, Sirolimus3
Tacrolimus, MMF1

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Number of Participants With Composite of Death, Graft Loss and Acute Rejection up to Month 6 - Intent to Treat Population

Participants with graft loss or death prior to Month 6 were considered having an event of AR, therefore, the incidence of AR was reported as a composite of AR, death, and graft loss. (NCT00455013)
Timeframe: Day 1 up to Month 6

Interventionparticipants (Number)
Belatacept, Mycophenolate Mofetil (MMF)6
Belatacept, Sirolimus2
Tacrolimus, MMF1

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Number of Participants With Delayed Graft Function - Intent to Treat Population

Delayed graft function (DGF) is defined as participant requiring dialysis within the first week (Day 1-8) post transplantation. Participants losing their graft less than 48 hours post transplant and receiving chronic dialysis were not considered as having DGF. Day 1 was day of transplantation. Intent to treat population defined as all participants randomized and transplanted (NCT00455013)
Timeframe: From Day 1 up to and including Day 8 post transplantation

Interventionparticipants (Number)
Belatacept, Mycophenolate Mofetil (MMF)6
Belatacept, Sirolimus4
Tacrolimus, MMF2

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Number of Participants With New Onset Diabetes Mellitus From Baseline to Month 12 Post Transplantation - Intent to Treat Population

"Baseline defined as day before transplantation. A participant who did not have diabetes prior to randomization and received an antidiabetic medication for a duration of at least 30 days or a participant who meets the following criteria and did not have diabetes prior to randomization: Symptoms of diabetes plus casual plasma glucose (PG) concentration ≥ 200 mg/dL (11.1 mmol/L); or fasting plasma glucose ≥ 126 mg/dL (7.0 mmol/L); or 2-hour PG ≥ 200 mg/dL (11.1 mmol/L) during an oral glucose tolerance test and a confirmatory laboratory test based on measurements of venous PG must have been done on another day in the absence of unequivocal hyperglycemia accompanied by acute metabolic decompensation.~Intent to treat population included all participants randomized and transplanted." (NCT00455013)
Timeframe: Baseline to Month 12

Interventionparticipants (Number)
Belatacept, Mycophenolate Mofetil (MMF)0
Belatacept, Sirolimus2
Tacrolimus, MMF1

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Mean (Standard Deviation) in Calculated Glomerular Filtration Rate (GFR) mL/Min/1.73m^2 at Month 3, Month 6 and Month 12 Post Transplantation - Intent to Treat Population

Blood urea nitrogen (BUN) in mg/dL; Albumin (Alb) in g/dL;Serum creatinine (SCr) in mg/dL; Age in years. Glomerular filtration rate (GFR) was calculated based upon serum creatinine (SCr) using the Modification of Diet in Renal Disease (MDRD) formula as suggested by Levey et al: MDRD GFR = 170 x [SCr/0.95]^(-0.999) x [Age]^(-0.176) x [0.762 if participant was female] x [1.180 if participant was black] x [BUN]^(-0.170) x [Alb]^(+0.318). Intent to Treat (ITT) population is defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: Months 3, 6 and 12 post transplantation

,,
InterventionmL/min/1.73m^2 (Mean)
Month 3 post transplantation(n=29,24,27)Month 6 post transplantation(n=29,24,25)Month 12 post transplantation(n=27, 23, 29)
Belatacept, Mycophenolate Mofetil (MMF)57.857.563.6
Belatacept, Sirolimus60.558.761.8
Tacrolimus, MMF51.251.754.0

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Mean (Standard Deviation) in Calculated Glomerular Filtration Rate (GFR) mL/Min/1.73m^2 at Months 24, 36 and 48 Post Transplantation - Intent to Treat Population in Long Term Extension

GFR was calculated based upon serum creatinine (SCr) using the Modification of Diet in Renal Disease (MDRD) formula as suggested by Levey et al: MDRD GFR = 170 x [SCr/0.95]^(-0.999) x [Age]^(-0.176) x [0.762 if participant was female] x [1.180 if participant was black] x [BUN]^(-0.170) x [Alb]^(+0.318). Age in years, Alb = Albumin in g/dL; SCr = in mg/dL; BUN =Blood urea nitrogen in mg/dL. Intent to Treat population is defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: Months 24, 36 and 48 post transplantation

,,
InterventionmL/Min/1.73m^2 (Mean)
Month 24 (N=27, 18, 27)Month 36 (N=25, 16, 22)Month 48 (N=18, 14, 15)
Belatacept, Mycophenolate Mofetil (MMF)60.662.859.6
Belatacept, Sirolimus66.069.972.2
Tacrolimus, MMF52.255.555.7

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Mean Change From Baseline (BL) to Month 12 Post Transplantation in Lipid Values - Intent to Treat Population

Baseline (BL) was value obtained day prior to transplantation. Lipid values measured in milligrams/deciliter (mg/dL) included: high density lipoprotein cholesterol (HDL-C), low density lipoprotein cholesterol (LDL-C), non-HDL cholesterol (non-HDL-C), total cholesterol (TC), triglycerides. Intent to treat population included all participants randomized and transplanted. (NCT00455013)
Timeframe: Baseline to Month 12

,,
Interventionmg/dL (Mean)
HDL-C change from BL to Month 12Non-HDL-C change from BL to Month 12LDL-C change from BL to Month 12Total cholesterol change from BL to Month 12Triglycerides change from BL to Month 12
Belatacept, Mycophenolate Mofetil (MMF)1.516.023.917.5-11.4
Belatacept, Sirolimus-3.716.125.012.5-1.1
Tacrolimus, MMF-0.520.534.020.0-14.2

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Mean Systolic, Diastolic and Arterial Blood Pressure at Baseline and Month 12 - Intent to Treat Population

Systolic, diastolic and mean arterial blood pressures were measured in millimeters of mercury (mm Hg). Baseline was defined as value obtained before transplantation. Intent to treat population included all participants randomized and transplanted. (NCT00455013)
Timeframe: Baseline and 12 months post transplantation

,,
Interventionmm Hg (Mean)
Systolic blood pressure at baselineSystolic blood pressure at Month 12Diastolic blood pressure at baselineDiastolic blood pressure at Month 12Mean Arterial pressure at baselineMean Arterial pressure at Month 12
Belatacept, Mycophenolate Mofetil (MMF)133.1129.378.673.396.891.9
Belatacept, Sirolimus126.9131.072.375.190.593.7
Tacrolimus, MMF141.8138.275.377.697.597.8

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Number of Corticosteroid-free Participants at 6 and 12 Months Post Transplantation - Intent to Treat Population

Participants were said to be corticosteroid-free at Month 6 if they were not receiving corticosteroids for greater than (>) 7 consecutive days during Days 141 through Days 196, and at Month 12 if not receiving corticosteroids for > 7 days during Days 337 through 392. Intent to treat population included all randomized and transplanted participants. (NCT00455013)
Timeframe: Day 1 through Month 12

,,
Interventionparticipants (Number)
At 6 Months post transplantationAt 12 Months post transplantation
Belatacept, Mycophenolate Mofetil (MMF)2724
Belatacept, Sirolimus2320
Tacrolimus, MMF2828

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Number of Corticosteroid-free Participants at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension

In the LTE, a participant was considered corticosteroid-free if they were not receiving corticosteroids for >7 consecutive days during Day 701 and Day 756, Day 1065 and Day 1120, as well as Day 1429 and Day 1484, respectively. (NCT00455013)
Timeframe: End of Month 12 to end of Long Term Extension (Year 4)

,,
Interventionparticipants (Number)
Month 24 (N=27, 18, 27)Month 36 (N=26, 16, 23)Month 48 (N=19, 14, 16)
Belatacept, Mycophenolate Mofetil (MMF)222216
Belatacept, Sirolimus151412
Tacrolimus, MMF252016

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Number of Participants Who Switched Between MMF and Sirolimus During Long Term Extension up to Study Completion

Long Term extension was the period from the end of Month 12 to the end of Month 48 post transplantation and the completion of the study 31 July 2012. At any time in the study, participants who were unable to tolerate MMF in the Bela-MMF and Tac-MMF groups could discontinue (DC) MMF and switch to sirolimus and remain in the study and those in the Bela-Siro group who were unable to tolerate sirolimus could DC sirolimus and switch to MMF and remain in the study. Study completion=data base (DB) lock. (NCT00455013)
Timeframe: End of Month 12 to end of Study (Month 48)

,,
Interventionparticipants (Number)
By Month 24 Switched between MMF and SirolimusBy Month 36 Switched between MMF and SirolimusBy Month 48 Switched between MMF and SirolimusUp to DB Lock Switched between MMF and Sirolimus
Belatacept, Mycophenolate Mofetil (MMF)0000
Belatacept, Sirolimus1233
Tacrolimus, MMF0000

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Number of Participants Who Used Anti-hypertension Medications at Baseline and at 12 Months Post Transplantation - Intent to Treat Population

Baseline was defined as day prior to transplantation. Number of anti-hypertension medications taken were categorized from 1 to 6 and greater than (>)6. Intent to treat population included all participants randomized and transplanted. (NCT00455013)
Timeframe: Baseline and Month 12

,,
Interventionparticipants (Number)
Total using at least 1 medication at baselineTotal using at least 1 medication at Month 12Participants using 1 medication at baselineParticipants using 1 medication at Month 12Participants using 2 medications at baselineParticipants using 2 medications at Month 12Participants using 3 medications at baselineParticipants using 3 medications at Month 12Participants using 4 medications at baselineParticipants using 4 medications at Month 12Participants using 5 medications at baselineParticipants using 5 medications at Month 12Participants using 6 medications at baselineParticipants using 6 medications at Month 12Participants using >6 medications at baselineParticipants using >6 medications at Month 12
Belatacept, Mycophenolate Mofetil (MMF)31251011568631302100
Belatacept, Sirolimus242095585522102000
Tacrolimus, MMF2820548138033201010

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Number of Participants Who Were Both Calcineurin Inhibitor-free (CNI-free)and Corticosteroid-free at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension

Participants were considered corticosteroid-free at Months 24, 36, and 48 if they were not receiving corticosteroids for >7 consecutive days during Day 701 and Day 756, Day 1065 and Day 1120, as well as Day 1429 and Day 1484, respectively. Participants were considered CNI-free at Months 24, 36, and 48 if they were not receiving CNI during Day 701 and Day 756, Day 1065 and Day 1120, as well as Day 1429 and Day 1484, respectively. Participants in the tacrolimus arm were not relevant to this analysis because tacrolimus is a calcinurin inhibitor. (NCT00455013)
Timeframe: Months 24, 36, 48

,,
Interventionparticipants (Number)
Month 24 (N=27, 18, 27)Month 36 (N=26, 16, 23)Month 48 (N=19, 14, 16)
Belatacept, Mycophenolate Mofetil (MMF)222216
Belatacept, Sirolimus151412
Tacrolimus, MMF000

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Number of Participants Who Were Corticosteroid-free at Months 6 and 12 and Number of Participants Who Were Both Calcineurin Inhibitor-free (CNI-free)and Corticosteroid-free at Months 6 and 12 Post Transplantation - Intent to Treat Population

Participants were said to be CNI-free at Month 6 or 12 if they were not receiving a CNI during Day 141 to Day 196, or Day 337 to Day 392. Participants in the tacrolimus arm were not relevant to this analysis because tacrolimus is a calcinurin inhibitor. Participants were corticosteroid-free (CS-free) at Month 6 if they were not receiving corticosteroids for > 7 consecutive days during Days 141 through Days 196, and at Month 12 if not receiving corticosteroids for > 7 days during Days 337 through 392. Day 1 was day of transplantation. Intent to treat population included all randomized and transplanted participants. (NCT00455013)
Timeframe: Day 1 to Month 12 post transplantation

,,
Interventionparticipants (Number)
CS-free Month 6 (N=33, 26, 30)CS-free Month 12 (N=32, 26, 30)CNI-free + CS-free Month 6 (N=32, 26, 30)CNI -free + CS-free Month 12 (N=32,26,30)
Belatacept, Mycophenolate Mofetil (MMF)27242524
Belatacept, Sirolimus23201918
Tacrolimus, MMF282811

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Number of Participants With Acute Rejection (AR) of Transplant up to 6 Months Post Transplantation - Intent to Treat (ITT) Population

AR is clinicopathological event requiring clinical evidence and biopsy confirmation by central pathologist. One or more conditions were met and a renal biopsy revealed histologic evidence of rejection: unexplained rise of serum creatine (SCr) greater than or equal to 25% from baseline plus one or more of the following: unexplained decreased urine output; fever, graft tenderness; SCr that remained elevated 14 days post-transplantation and clinical suspicion of AR; other reason and participant treated for episode. Day 1=transplantation. Banff 97 working classification of kidney transplant pathology: Type I=tubulointerstitial AR without arteritis (IA: interstitial infiltration with >25% of parenchyma affected and moderate tubulitis with >4 mononuclear cells/tubular cross section; IB: >10 mononuclear cells; Type II vascular AR with (IA) intimal arteritis (IIA=mild - moderate; IIB=severe; Type III=severe rejection with transmural arterial changes, necrosis of smooth muscle cells. (NCT00455013)
Timeframe: Day 1 to Month 6 post-transplantation

,,
Interventionparticipants (Number)
Total Number of Participants with ARMild Acute IAMild Acute IBModerate Acute IIAModerate Acute IIB
Belatacept, Mycophenolate Mofetil (MMF)40021
Belatacept, Sirolimus10001
Tacrolimus, MMF10010

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Number of Participants With Acute Rejection of Transplant up to Month 12 Post Transplantation - Intent to Treat Population

AR defined as a clinicopathological event requiring clinical evidence and biopsy confirmation by central pathologist. One or more conditions were met and a renal biopsy revealed histologic evidence of rejection: unexplained rise of serum creatine (SCr) >= 25 % from baseline plus one or more of the following: unexplained decreased urine output; fever and graft tenderness; a SCr that remained elevated within 14 days after transplantation and clinical suspicion of AR; reason other than those listed and participant was treated for this episode. Day 1 was day of transplantation. Banff grade used Banff 97 working classification of kidney transplant pathology. ITT population was all randomized and transplanted participants. (NCT00455013)
Timeframe: Day 1 to Month 12 post transplantation

,,
Interventionparticipants (Number)
Total number of participants with ARMild Acute IAMild Acute IBModerate Acute IIAModerate Acute IIB
Belatacept, Mycophenolate Mofetil (MMF)50032
Belatacept, Sirolimus10001
Tacrolimus, MMF10010

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Number of Participants With Graft Loss or Death at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension

Graft loss was defined as either functional loss or physical loss. Functional loss was defined as either: sustained level of SCr greater than or equal to (>=) 6.0 mg/dL (530 micromoles/Liter; micromol/L) for >= 4 weeks as determined by the local laboratory; regularly scheduled dialysis treatments over a period of 56 days; impairment of renal function to such a degree that the participant undergoes re-transplant. Day 1 was day of transplantation. ITT population defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: End of Month 12 to end of Long Term Extension (Year 4)

,,
Interventionparticipants (Number)
Graft Loss or Death at Month 24 (N=27,19,27)Graft Loss or Death at Month 36 (N=27,19,27)Graft Loss or Death at Month 48 (N=27,19,27)
Belatacept, Mycophenolate Mofetil (MMF)011
Belatacept, Sirolimus122
Tacrolimus, MMF000

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Number of Participants With Graft Loss or Death up to Month 6 and Month 12 Post Transplantation - Intent to Treat Population

Graft loss was defined as either functional loss or physical loss. Functional loss was defined as either: sustained level of SCr greater than or equal to (>=) 6.0 mg/dL (530 micromoles/Liter; micromol/L) for >= 4 weeks as determined by the local laboratory; regularly scheduled dialysis treatments over a period of 56 days; impairment of renal function to such a degree that the participant undergoes re-transplant. Day 1 was day of transplantation. ITT population defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: Day 1 to Month 6 and Month 12 post transplantation

,,
Interventionparticipants (Number)
Graft loss up to Month 6 post transplantationGraft loss up to Month 12 post transplantationDeath up to Month 6 post transplantationDeath up to Month 12 post transplantation
Belatacept, Mycophenolate Mofetil (MMF)1211
Belatacept, Sirolimus1200
Tacrolimus, MMF0000

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Change in Forced Expiratory Volume in 1 Second (FEV1) From Baseline to End of Study (Month 24) in the Lung Transplant Subgroup

Forced expiratory volume in 1 second (FEV1) was measured by spirometry conducted according to internationally accepted standards. FEV1 is the volume delivered in the first second of a forced vital capacity (FVC) maneuver. A positive change score indicates improved lung function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)

InterventionLiters (Mean)
Everolimus + CNI Reduction-0.2
Control-0.1

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Change in Forced Vital Capacity (FVC) From Baseline to End of Study (Month 24) in the Lung Transplant Subgroup

Forced vital capacity (FVC) was measured by spirometry conducted according to internationally accepted standards. FVC is the volume delivered during an expiration made as forcefully and completely as possible starting from full inspiration. A positive change score indicates improved lung function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)

InterventionLiters (Mean)
Everolimus + CNI Reduction-0.2
Control-0.1

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Mean Days of Hospitalization From Baseline to End of Study (Month 24)

(NCT00377962)
Timeframe: Baseline to end of study (Month 24)

InterventionDays (Mean)
Everolimus + CNI Reduction8.5
Control16.2

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Number of Patients in Need of Dialysis From Month 12 to End of Study (Month 24)

(NCT00377962)
Timeframe: Month 12 to end of study (Month 24)

InterventionParticipants (Number)
Everolimus + CNI Reduction0
Control2

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Number of Patients With Biopsy-proven Acute Rejection From Month 12 to End of Study (Month 24)

Biopsy-proved acute rejection was defined as a treated acute rejection confirmed by biopsy, graded locally according to the International Society for Heart & Lung Transplantation (ISHLT) criteria. A treated acute rejection was defined as an acute rejection clinically suspected, whether biopsy-proven or not, which had been treated and confirmed by the investigator according to the response to therapy. (NCT00377962)
Timeframe: Month 12 to end of study (Month 24)

InterventionParticipants (Number)
Everolimus + CNI Reduction6
Control5

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Change in Left Ventricular Function (Diameter and Thickness Parameters) From Baseline to End of Study (Month 24) in the Heart Transplant Subgroup

Left ventricular function was assessed by echocardiography which was performed according to local routine practice. Echocardiography parameters were left ventricular end diastolic diameter (LVEDD), left ventricular end systolic diameter (LVESD), interventricular septal wall thickness (IVSTd), and posterior wall thickness (PWTd). A positive change score indicates improved left ventricular function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)

,
Interventioncm (Mean)
LVEDDLVESDIVSTdPWTd
Control-0.00.1-0.1-0.1
Everolimus + CNI Reduction-0.10.1-0.4-0.5

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Change in Left Ventricular Function (Filling and Ejection Fraction Parameters) From Baseline to End of Study (Month 24) in the Heart Transplant Subgroup

Left ventricular function was assessed by echocardiography which was performed according to local routine practice. Echocardiography parameters were filling fraction (FF) and ejection fraction (EF). A positive change score indicates improved left ventricular function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)

,
Interventionpercentage (Mean)
EFFF
Control0.10
Everolimus + CNI Reduction-0.60

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Change in Measured Glomerular Filtration Rate (mGFR) From Baseline to End of Study (Month 24)

Renal function was assessed by determining the measured glomerular filtration rate (mGFR) using creatinine ethylenediamine tetraacetic acid (Cr-EDTA) clearance or an equivalent method. A positive change score indicates improved renal function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)

,
InterventionmL/min (Mean)
Month 0Month 24Change
Control49.146.8-2.4
Everolimus + CNI Reduction49.352.53.2

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Change in Measured Glomerular Filtration Rate (mGFR) From Baseline to Month 12

Renal function was assessed by determining the measured glomerular filtration rate (mGFR) using creatinine ethylenediamine tetraacetic acid (Cr-EDTA) clearance or an equivalent method. A positive change score indicates improved renal function. (NCT00377962)
Timeframe: Baseline to Month 12

,
InterventionmL/min (Mean)
BaselineMonth 12Change from Baseline
Control48.047.5-0.5
Everolimus + CNI Reduction48.653.24.6

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Change in Serum Creatinine From Baseline to End of Study (Month 24)

Renal function was assessed by determining serum creatinine using standard laboratory methods. A positive change score indicates improved renal function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)

,
Interventionμmol/L (Mean)
Month 0Month 24Change
Control1291323
Everolimus + CNI Reduction1261260

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Number of Patients Discontinued From the Study Due to Adverse Events From Month 12 to End of Study (Month 24)

(NCT00377962)
Timeframe: Month 12 to end of study (Month 24)

,
InterventionParticipants (Number)
Total discontinued due to AE(s)Pulmonary embolismSkin problemsHypercholesterolemiaStrokeMuscular painDiarrheaEdema
Control00000000
Everolimus + CNI Reduction82111111

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Number of Patients Who Died and Number of Patients With Graft Loss From Month 12 to End of Study (Month 24)

Number of patients not alive and number of patients with loss of their graft. (NCT00377962)
Timeframe: Month 12 to end of study (Month 24)

,
InterventionParticipants (Number)
DeathGraft Loss
Control00
Everolimus + CNI Reduction30

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Graft Survival at Three Years Post-Transplant

"Number of participants that did not experience kidney graft failure[1] at three years post-transplant~[1]Graft failure is defined as the institution of chronic dialysis (at least 6 consecutive weeks, excluding participants with delayed graft function), transplant nephrectomy, or retransplantation." (NCT00183248)
Timeframe: Three years post kidney transplant

Interventionparticipants (Number)
DBMCs4
Control Group5

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Number of Chronic Allograft Nephropathies

"Number of chronic allograft nephropathies[1,2,3] at 3 years post kidney transplant.~Chronic allograft nephropathy is defined as renal biopsies with Banff 97 Grade I or greater[2] with higher numeric scores indicating more severe nephropathy~The Banff 97 diagnostic category for renal allograft biopsies is an international standardized histopathological classification[3]~Reference: Racusen LC, Solez K, Colvin RB et al. The Banff 97 working classification of renal allograft pathology. Kidney Int, 55: 713-723, 1999" (NCT00183248)
Timeframe: Three years post kidney transplant

InterventionNephropathy Events (Number)
DBMCs6
Control Group2

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Number of Graft-versus-host Disease (GVHD) Events

A disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Symptoms include jaundice, skin rash or blisters, a dry mouth, or dry eyes. Also called graft-versus-host disease. (NCT00183248)
Timeframe: Three years post kidney transplant

InterventionGVHD Events (Number)
DBMCs0
Control Group0

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Number of Kidney Biopsy-proven Acute Rejection

"Biopsy-proven acute renal (kidney) rejection[1,2].~Diagnosis of acute rejection was made by renal biopsy using the Banff 97 criteria. The Banff 97 diagnostic category for renal allograft biopsies is an international standardized histopathological classification. Acute rejection is defined by a renal biopsy demonstrating a Banff 97 classification of Grade IA or greater, with higher scores indicating more severe rejection[2]~Ref: Racusen LC et al. The Banff 97 working classification of renal allograft pathology. Kidney Int, 55: 713-723, 1999" (NCT00183248)
Timeframe: Three years post kidney transplant

InterventionRejection Events (Number)
DBMCs1
Control Group1

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Overall Kidney Graft Survival at One Year Post-Transplant

"Number of participants that did not experience kidney graft failure[1] at one year post-transplant~[1]Graft failure is defined as the institution of chronic dialysis (at least 6 consecutive weeks, excluding participants with delayed graft function), transplant nephrectomy, or retransplantation." (NCT00183248)
Timeframe: One year post kidney transplant

Interventionparticipants (Number)
DBMCs4
Control Group5

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Overall Participant Survival at One Year Post Kidney Transplant

(NCT00183248)
Timeframe: One year post kidney transplant

Interventionparticipants (Number)
DBMCs4
Control Group5

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Participant Survival at Three Years Post Kidney Transplant

(NCT00183248)
Timeframe: Three years post kidney transplant

Interventionparticipants (Number)
DBMCs4
Control Group5

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Incidence of Chronic Extensive GVHD

Percentage patients with chronic extensive GVHD, estimated by cumulative incidence methods (NCT00075478)
Timeframe: 3 years after transplant

Interventionpercentage of participants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)72
Arm II (TBI, Transplant, GVHD Prophylaxis)48

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Incidence of Grades II-IV Acute GVHD

Percentage patients with grades II-IV GHVD, estimated by cumulative incidence methods (NCT00075478)
Timeframe: 120 days after transplant

Interventionpercentage of participants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)46
Arm II (TBI, Transplant, GVHD Prophylaxis)32

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Incidence of Graft Rejection

Donor CD3 chimerism less than 5% (NCT00075478)
Timeframe: 1 year after transplant

Interventionparticipants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)0
Arm II (TBI, Transplant, GVHD Prophylaxis)2

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Incidence of Non-relapse Mortality

Percentage of NRM as estimated by cumulative incidence methods with competing risks (NCT00075478)
Timeframe: 3 years after transplant

Interventionpercentage of participants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)7
Arm II (TBI, Transplant, GVHD Prophylaxis)9

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Incidence of Relapse/Progression

Percentage of relapse estimated by cumulative incidence methods (NCT00075478)
Timeframe: 3 years after transplant

Interventionpercentage of participants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)40
Arm II (TBI, Transplant, GVHD Prophylaxis)55

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Overall Survival

Percentage of patients surviving as estimated by Kaplan-Meier. (NCT00075478)
Timeframe: 3 years after transplant

Interventionpercentage of participants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)65
Arm II (TBI, Transplant, GVHD Prophylaxis)54

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Progression-free Survival

Percentage of patients with progression-free survival, estimated by cumulative incidence methods (NCT00075478)
Timeframe: 3 years after transplant

Interventionpercentage of participants (Number)
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)53
Arm II (TBI, Transplant, GVHD Prophylaxis)36

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Duration (Days) Until Participants Obtained Platelet Engraftment

Summarized using a range and median of measure in days until participants reached platelet engraftment. Platelet engraftment was defined as the first of 7 consecutive days when the platelet count exceeded 20 x 109/L (untransfused). (NCT00796068)
Timeframe: At 6 months

Interventiondays (Median)
Arm I (Low Risk for Graft Failure)31
Arm II (High Risk for Graft Failure)31

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Incidence of Acute or Chronic Graft-versus-host Disease (GVHD)

Overall GVHD is determined based on the organ stage, response to treatment and whether GVHD was a major cause of death. Chronic GVHD will be defined according to the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease. Described as mild, moderate, or severe as graded according to the attached Organ Scoring Sheet. Symptoms consistent with both chronic and acute GVHD occurring after day 100 will be considered overlap chronic GVHD syndrome. (NCT00796068)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)7
Arm II (High Risk for Graft Failure)6

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Incidence of Clinically Significant Infections

Collected and graded according to the modified National Cancer Institute Common Toxicity Criteria. (NCT00796068)
Timeframe: At 6 months

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)53
Arm II (High Risk for Graft Failure)61

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Incidence of Relapse or Disease Progression

Cumulative incidence estimates using non-relapse mortality as competitive event. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)17
Arm II (High Risk for Graft Failure)11

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Non-relapse Mortality

Death of any cause other than relapse or disease progression was considered. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)8
Arm II (High Risk for Graft Failure)10

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Number of Participants Surviving by 1 Year

Overall survival was measured from the first day of CBT infusion until death from any cause. (NCT00796068)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)51
Arm II (High Risk for Graft Failure)47

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Number of Participants Surviving up to 2 Years Without Disease Progression

Progression-free survival is the time interval from start of treatment to documented evidence of disease progression. Disease progression was defined by European LeukemiaNet 2017 criteria and International Working Group (IWG) within 3 years of transplant. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)37
Arm II (High Risk for Graft Failure)41

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Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100

Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. Diagnosing and grading acute GVHD is based on clinical findings (the organ stage, response to treatment and whether GVHD was a major cause of death) and frequently varies between transplant centers and independent reviewers. (NCT00796068)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)36
Arm II (High Risk for Graft Failure)37

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Number of Participants With Graft Failure/Rejection

"Patients will be considered primary graft failure provided they meet any criteria listed below, in the absence of documented disease persistence/recurrence or active bone marrow infection (ex. Cytomegalovirus (CMV)):~i. Absence of 3 consecutive days with neutrophils >500/u1 combined with host CD3+ peripheral blood chimerism ≥50% at day 42 ii. Absence of 3 consecutive days with neutrophils >500/u1 under any circumstances at day 55 iii. Death after day 28 with neutrophil count <100/u1 without any evidence of engraftment (< 5% donor CD3+) iv. Primary autologous count recovery with < 5% donor CD3+ peripheral blood chimerism at count recovery and without relapse" (NCT00796068)
Timeframe: Up to 100 days

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)2
Arm II (High Risk for Graft Failure)5

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Number of Participants With Secondary Graft Failure

Secondary graft failure is defined as decline of neutrophil count to <500/ul with loss of donor chimerism after day 55 (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)0
Arm II (High Risk for Graft Failure)0

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Number of Patients With Non-relapse Mortality (NRM)

Defined as death from any cause without sign of disease progression or relapse. Loss to follow up are censored, whereas disease relapse or progression are considered competing risks. (NCT00796068)
Timeframe: At day -200

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)7
Arm II (High Risk for Graft Failure)6

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The Number of Participants Alive at Two-years Follow up.

Overall survival of participants after two-years of follow up. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)50
Arm II (High Risk for Graft Failure)44

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Change in SLICC/ACR Damage Index From Baseline During Short-term Period

SLICC/ACR score or damage index is a measure of cumulative damage due to Systemic Lupus Erythematosus (SLE). Damage is defined as non-reversible change (not related to active inflammation) occurring since onset of lupus, ascertained by clinical assessment and present for at least 6 months. A score of 0=no damage, early damage is defined as ≥1. The total maximum score is 48, and increasing score indicates increasing disease severity. Change from baseline=Postbaseline - baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Postbaseline (Month 12 or 28 days after last dose)

InterventionUnits on a scale (Mean)
Abatacept 30/10 mg/kg0.17
Abatacept 10/10 mg/kg0.11
Placebo0.13

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Number of Months CRR Was Maintained During Short-term Period

Durability of CRR, defined as the number of months (number of consecutive planned visits beyond Day 15) a participant met the definition of CRR during the double-blind treatment period. Refer to outcome 1 for description of CRR. (NCT00430677)
Timeframe: Day 1 (randomization) to 12 Months

InterventionMonths (Median)
Abatacept 30/10 mg/kg0
Abatacept 10/10 mg/kg0
Placebo0

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Number of Participants Achieving Renal Response (RR) at Month 12 During Short-term Period

RR is defined as meeting BOTH of the following criteria:RENAL FUNCTION: Less than or equal to 25% increase from baseline;PROTEINURIA: Greater than or equal to 50% improvement in the urine protein/creatinine ratio with one of the following - urine protein/creatinine ratio (UPCR) <113 mg/mmol,, if the baseline ratio was <=339 mg/mmol OR UPCR <339 mg/mmol,if the baseline ratio > 339 mg/mmol. A participant was considered as achieving RR if response criteria at both months 11 and 12 (Days 337 and 365, respectively) were met. For 95% CI within each group, normal approximation is used if n>=5. (NCT00430677)
Timeframe: Month 12

InterventionParticipants (Number)
Abatacept 30/10 mg/kg45
Abatacept 10/10 mg/kg39
Placebo33

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Number of Participants With a Treatment-emergent Seropositive Result During the Long-term Extension Period

Collected in at least 1 sample. Assessment includes immunogenicity (detection of serum antibodies which bind to CTLA4-Ig in the in vitro assays) and exposure to corticosteroids (NCT00430677)
Timeframe: Day 365 to end of long-term extension period

InterventionParticipants (Number)
Abatacept 10 mg/kg17

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Number of Participants With Confirmed Complete Renal Response (CRR) During Short-term Period

Confirmed at 2 consecutive visits. CRR defined as meeting all of 5 criteria. Renal function (RF): (Glomerular filtration rate [GFR] calculated using Modification of Diet in Renal Diseases equation equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: Day 1 to 12 months

InterventionParticipants (Number)
Abatacept 30/10 mg/kg22
Abatacept 10/10 mg/kg27
Placebo20

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Participants Achieving a Confirmed Complete Renal Response (CRR) at Month 12 During Short-term Period

Confirmed at 2 consecutive visits. CRR defined as meeting all of 5 criteria. Renal function (RF): (Glomerular filtration rate [GFR] calculated using Modification of Diet in Renal Diseases equation equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: At Month 12 from Day 1

InterventionParticipants (Number)
Abatacept 30/10 mg/kg9
Abatacept 10/10 mg/kg11
Placebo8

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Participants Achieving Renal Improvement (RI) or CRR at Month 12 During Short-term Period

CRR defined as meeting all of 5 criteria. RF: (Glomerular filtration rate [GFR] calculated using MDRD equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: At Month 12 from Day 1

InterventionParticipants (Number)
Abatacept 30/10 mg/kg38
Abatacept 10/10 mg/kg37
Placebo31

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Time to Achieve First Confirmed Renal Improvement (RI) During Short-term Period (as Determined by Kaplan-Meier Methodology)

RI is defined as meeting all of the following criteria. Renal function: If MDRD is abnormal at screening, within 10% of the MDRD at screening; if MDRD is 60-89 at screening, greater than or equal to 50% improvement based on the screening value or 90% or greater of MDRD at screening; if MDRD is 15-59 at screening, if MDRD is normal at screening-within 10% of the MDRD at screening. Proteinuria: improvement greater than or equal to 50% from screening. Hematuria: red blood cell (RBC)count within normal limit of central laboratory. Pyuria: white blood cell (WBC) count within normal limit of central laboratory. Cylindruria: No RBC or WBC casts. (NCT00430677)
Timeframe: Day 1 (randomization) to 12 months.

InterventionDays (Median)
Abatacept 30/10 mg/kg141
Abatacept 10/10 mg/kg136
Placebo144

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Time to First Confirmed Complete Renal Response (CRR) During the Short-term (Double-blind) Period

Confirmed at 2 consecutive visits. CRR defined as meeting all of 5 criteria. Renal function (RF): (Glomerular filtration rate [GFR] calculated using Modification of Diet in Renal Diseases equation equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: Day 1 (randomization) to 12 months.

Interventiondays (Number)
Abatacept 30/10 mg/kgNA
Abatacept 10/10 mg/kgNA
PlaceboNA

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Baseline and Post Baseline Systemic Lupus International Collaborating Clinics (SLICC)/American College of Rheumatology (ACR) Damage Index During Short-term Period

SLICC/ACR score or damage index is a measure of cumulative damage due to Systemic Lupus Erythematosus (SLE). Damage is defined as nonreversible change (not related to active inflammation) occurring since onset of lupus, ascertained by clinical assessment and present for at least 6 months. A score of 0=no damage, early damage is defined as ≥1. The total maximum score is 48, and increasing score indicates increasing disease severity. (NCT00430677)
Timeframe: Baseline (Day 1), Post baseline (Month 12 or 28 days after last dose)

,,
InterventionUnits on a scale (Mean)
Baseline (n=68, 67, 70)Post Baseline Mean (n=68, 67, 70)
Abatacept 10/10 mg/kg0.270.40
Abatacept 30/10 mg/kg0.340.53
Placebo0.290.44

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Baseline Fatigue as Measured by Fatigue Severity Scale-Krupp During Short-term Period

The reduction of fatigue assessed by Fatigue Severity Scale (FSS). The FSS questionnaire is comprised of 9 statements inquiring about the examinee's sleep habits over the preceding week. Participants are asked to rate their level of agreement (toward seven) or disagreement (toward zero) with the nine statements. A score of 36 and above (out of a maximum of 63) indicates the presence of significant fatigue. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Baseline (Day 1) for Day 85 (n=90, 94, 95)Baseline (Day 1) for Day 169 (n=92, 94, 97)Baseline (Day 1) for Day 253 ( n=92, 94, 97)Baseline (Day 1) for Day 365 ( n=92, 94, 97)
Abatacept 10/10 mg/kg39.1439.1439.1439.14
Abatacept 30/10 mg/kg40.6040.4140.4140.41
Placebo39.6439.5939.5939.59

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Baseline Fatigue as Measured by the Fatigue Visual Analog Scale During Short-term Period

A visual analogue scale (VAS) is a psychometric response scale for measurement of subjective characteristics or attitudes that cannot be directly measured. The VAS for Fatigue (VAS-F) consists of a 100 mm line, with 0 (No Fatigue) on 1 end and 100 (Extreme Fatigue) on the other end, which a participant marks to indicate how much fatigue he or she feels. The marked point in mm is converted into a numeric value from 0 to 100, where 0=no fatigue and 100=maximum fatigue. Increasing numbers=increasing fatigue. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Baseline (Day 1) for Day 85 (n=90, 94, 95)Baseline (Day 1) for Day 169 (n=92, 94, 97)Baseline (Day 1) for Day 253 (n=92, 94, 97)Baseline (Day 1) for Day 365 (n=92, 94, 97)
Abatacept 10/10 mg/kg41.9541.9541.9541.95
Abatacept 30/10 mg/kg48.8648.8048.8048.80
Placebo48.9348.2548.2548.25

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Baseline Mental Component Summary of the Short SF-36 During Short-term Period

The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Baseline (Day 1) for Day 85 ( n=89, 91, 93)Baseline (Day 1) for Day 169 (n=92,94,96)Baseline (Day 1) for Day 253 ( n=92,94,96)Baseline (Day 1) for Day 365 ( n=92,94,96)
Abatacept 10/10 mg/kg43.8041.8441.8441.84
Abatacept 30/10 mg/kg42.1844.0844.0844.08
Placebo42.6842.5942.5942.59

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Baseline Physical Component Summary of the Short Form (SF)-36 During Short-term Period

The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Baseline (Day 1) for Day 85 (n=89, 91, 93)Baseline (Day 1) for Day 169 (n=92, 94, 96)Baseline (Day 1) for Day 253 (n=92, 94, 96)Baseline (Day 1) for Day 365 (n=92, 94, 96)
Abatacept 10/10 mg/kg43.8044.1744.1744.17
Abatacept 30/10 mg/kg42.1842.0442.0442.04
Placebo42.6842.4842.4842.48

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Baseline Quantitative Immunoglobulins During the Short-term Period

A quantitative immunoglobulins (Igs) test is used to detect abnormal levels of the three major classes of Igs (IgG, IgA, and IgM). Abnormal test results typically indicate that there is something affecting the immune system which requires further testing. (NCT00430677)
Timeframe: Baseline (Day 1)

,,
Interventionmg/dL (Mean)
Immunoglobulin IgAImmunoglobulin IgGImmunoglobulin IgM
Abatacept 10/10 mg/kg218.04864.1297.10
Abatacept 30/10 mg/kg246.28939.80100.96
Placebo230.231013.1798.49

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Baseline Renal Function Over Time During Short-term Period

Baseline (BL) renal function, as estimated by calculation of the MDRD (Modification of Diet in Renal Disease) equation, over time. Renal MDRD is an equation (calculation) used to estimate Glomerular Filtration Rate (GFR) in participants with impaired renal function based on serum creatinine, age, race, and gender. GFR (mL/min/1.73 m^2) = 175 * (Scr)^-1.154 * (Age)^-0.203 * (0.742 if female) * (1.212 if African American) (conventional units). mL, milliliters; min, minute; m^2, meters squared; Scr, serum creatinine. A negative value indicates worsening. (NCT00430677)
Timeframe: Baseline (Day 1), Day 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365

,,
Interventionmilliliters per minute (mL/min)/1.73 m^2 (Mean)
Baseline (Day 1) for Day 15 (n=98,94,98)Baseline (Day 1) for Day 29 (n=98,95,98)Baseline (Day 1) for Day 57 (n=94, 89, 96)Baseline (Day 1) for Day 85 (n=90, 91, 93)Baseline (Day 1) for Day 113 (n=91, 83, 91)Baseline (Day 1) for Day 141 (n=89, 83, 90)Baseline (Day 1) for Day 169 (n=83, 82, 85)Baseline (Day 1) for Day 197 (n=84, 81, 87)Baseline (Day 1) for Day 225 (n=84, 81, 84)Baseline (Day 1) for Day 253 (n=81, 76, 81)Baseline (Day 1) for Day 281 (n=78, 77, 80)Baseline (Day 1) for Day 309 (n=77, 76, 78)Baseline (Day 1) for Day 337 (n=74, 75, 79)Baseline (Day 1) for Day 365 (n=75, 73, 78)
Abatacept 10/10 mg/kg99.0998.5698.76100.22101.23101.05101.95101.85101.64101.25102.64100.64101.00101.30
Abatacept 30/10 mg/kg92.5793.0891.5494.5394.4393.6294.8394.5594.8794.8994.1794.7495.5195.04
Placebo91.0191.2391.5892.7692.4192.8392.5692.6993.2593.3893.1392.7192.9093.03

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Change From Baseline in Mental Component Summary of the SF-36 During Short-term Period

The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Day 85 (n=89, 91, 93)Day 169 (n=92, 94, 96)Day 253 (n=92, 94, 96)Day 365 (n=92, 94, 96)
Abatacept 10/10 mg/kg3.105.084.834.23
Abatacept 30/10 mg/kg1.072.902.452.62
Placebo1.873.692.992.84

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Change From Baseline in Physical Component Summary of the SF-36 During Short-term Period

The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Day 85 (n=89, 91, 93)Day 169 (n=92, 94, 96)Day 253 (n=92, 94, 96)Day 365 (n=92, 94, 96)
Abatacept 10/10 mg/kg2.614.074.805.00
Abatacept 30/10 mg/kg4.174.184.234.24
Placebo2.863.393.453.77

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Change in Fatigue From Baseline as Measured by Fatigue Severity Scale-Krupp During Short-term Period

The reduction of fatigue assessed by Fatigue Severity Scale (FSS). The FSS questionnaire is comprised of 9 statements inquiring about the examinee's sleep habits over the preceding week. Participants are asked to rate their level of agreement (toward seven) or disagreement (toward zero) with the nine statements. A score of 36 and above (out of a maximum of 63) indicates the presence of significant fatigue. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Least Squares Mean)
Day 85 (n=90, 94, 95)Day 169 (n=92, 94, 97)Day 253 ( n=92, 94, 97)Day 365 ( n=92, 94, 97)
Abatacept 10/10 mg/kg-1.40-1.69-2.95-3.21
Abatacept 30/10 mg/kg-1.54-2.68-3.54-4.20
Placebo-0.67-1.08-3.06-4.79

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Change in Fatigue From Baseline as Measured by the Fatigue Visual Analog Scale During Short-term Period

"A visual analogue scale is a psychometric response scale for measurement of subjective characteristics or attitudes that cannot be directly measured.~The VAS for Fatigue (VAS-F) consists of a 100 mm line, with 0 (No Fatigue) on 1 end and 100 (Extreme Fatigue) on the other end, which a participant marks to indicate how much fatigue he or she feels. The marked point in mm is converted into a numeric value from 0 to 100, where 0=no fatigue and 100=maximum fatigue. Increasing numbers=increasing fatigue." (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365

,,
InterventionUnits on a scale (Mean)
Day 85 (n=90, 94, 95)Day 169 (n=92, 94, 97)Day 253 (n=92, 94, 97)Day 365 (n=92, 94, 97)
Abatacept 10/10 mg/kg-4.94-9.52-11.90-12.32
Abatacept 30/10 mg/kg-9.18-7.18-8.78-12.21
Placebo-6.20-4.71-7.35-11.07

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Change in Quantitative Immunoglobulin From Baseline During Short-term Period

"A quantitative immunoglobulin (Ig) test is used to detect abnormal levels of the 3 major classes of Ig (IgG, IgA, and IgM). Abnormal test results typically indicate that something is affecting the immune system and further testing is required.~Please refer to Outcome 31 for the respective baseline values" (NCT00430677)
Timeframe: Day 365

,,
Interventionmg/dL (Mean)
Ig A (n=76, 73, 78)IgG (n=76, 73, 78)IgM (n=76, 73, 78)
Abatacept 10/10 mg/kg-34.4827.21-19.38
Abatacept 30/10 mg/kg-32.8341.41-17.76
Placebo-26.5123.42-20.62

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Change in Renal Function From Baseline Over Time During Short-term Period

Mean change from baseline in renal function, as estimated by calculation of the MDRD equation, over time. Renal MDRD is an equation (calculation) used to estimate Glomerular Filtration Rate (GFR) in participants with impaired renal function based on serum creatinine, age, race, and gender. GFR (mL/min/1.73 m^2) = 175 * (Scr)^-1.154 * (Age)^-0.203 * (0.742 if female) * (1.212 if African American) (conventional units). mL, milliliters; min, minute; m^2, meters squared; Scr, serum creatinine. A positive value indicates improvement. Change from baseline=Post-baseline-baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Day 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365

,,
Interventionmilliliters per minute (mL/min)/1.73 m^2 (Mean)
Day 15; (n=98,94,98)Day 29 (n=98, 95, 98)Day 57 (n=94 89 96)Day 85 (n=90, 91, 93)Day 113 (n=91, 83, 91)Day 141 (n=89, 83, 90)Day 169 (n=83, 82, 85)Day 197 (n=84, 81, 87)Day 225 (n=84, 81, 84)Day 253 (n=81, 76, 81)Day 281 (n=78, 77, 80)Day 309 (n=77, 76, 78)Day 337 (n=74, 75, 79)Day 365 (n=75, 73, 78)
Abatacept 10/10 mg/kg1.154.517.286.2010.237.9010.5510.677.727.226.819.5310.1511.03
Abatacept 30/10 mg/kg-0.242.265.968.565.318.338.127.717.324.705.686.345.345.17
Placebo0.621.702.382.894.123.627.346.797.195.865.386.004.395.68

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Mean Change From Baseline in SLICC/ACR Damage Index

SLICC=Systemic Lupus International Collaborating Clinics; ACR=American College of Rheumatology. The SLICC/ACR Damage Index measures organ damage (nonreversible change, unrelated to active inflammation) occurring since onset of lupus, ascertained by clinical assessment and present for at least 6 months unless otherwise stated. The index assesses 47 items in 12 systems: Ocular, Neuropsychiatric, Renal, Pulmonary, Cardiovascular, Gastrointestinal, Peripheral Vascular, Musculoskeletal, Skin, Premature Gonadal Failure, Diabetes, Malignancy. Scores range from 0 to 2, and the same lesion cannot be scored twice. If damage is noted for a particular item, it is scored 1. No damage is scored 0. Some items may score 2 points if they occur more than once, so that the maximum possible score is 47. Scores can only increase with time, but scores rarely reach over 12. It is usually completed (or updated) yearly. (NCT00430677)
Timeframe: Day 365 to termination of the long-term extension phase

,,
InterventionUnits on a scale (Mean)
Day 365 (n=69, 65, 74)Day 729 (n=66, 65, 69)Day 1093 (n=41, 38, 44)
Abatacept 10/10 mg/kg-0.17-0.28-0.16
Abatacept 30/10 mg/kg-0.12-0.21-.27
Placebo-0.08-0.100.02

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Number of Participants Achieving Complete Response by ACCESS Definition

The Abatacept and Cyclophosphamide Combination Efficacy and Safety Study (ACCESS) defines complete response as a response meeting all of the following criteria: serum creatinine ≤upper limit of normal as defined by the central laboratory or ≤125% of the higher value at either screening or baseline; urine protein/creatinine ratio <50 mg/mmoL; and prednisone or prednisone-equivalent dose tapered to 10 mg per day. (NCT00430677)
Timeframe: End of short-term period (Day 365) to termination of the long-term extension period

,,
Interventionparticipants (Number)
Day 365Day 645 (n=55, 56, 61)
Abatacept 10/10 mg/kg3028
Abatacept 30/10 mg/kg2927
Placebo2525

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Number of Participants Achieving Patient Response (PR) at Month 12 During the Short-term Period

"PR is either CRR, Partial Renal Response(PRR),or no Response(NR).~CRR= Serum creatinine(SC)is normal, Inactive urinary sediment, No cellular casts, Urinary protein/creatinine (UPCR) ratio <56.5 mg/mmoL; PRR= SC is normal OR SC not >25% above BL, RBCs at reference range, UPCR <56.5 mg/mmoL OR ≥50% improvement in UPCR with one of the following: UPCR <113 or <339 mg/mmoL, based on the BL ratio; NR= Not achieving either a CRR or a PRR. Participants achieved response if criteria at both months 11 and 12 (Days 337 and 365) were met. Participants who Early discontinuations were categorized as NR." (NCT00430677)
Timeframe: Month 12

,,
InterventionParticipants (Number)
No Renal ResponsePartial Renal ResponseComplete Renal Response
Abatacept 10/10 mg/kg69921
Abatacept 30/10 mg/kg611424
Placebo661420

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Number of Participants Achieving Patient Response of Complete or Partial Response, Based on the June 2010 Food and Drug Administration Guidance Document for Lupus Nephritis

Patient response=complete, partial, or no response. Complete response=serum creatinine (SCr) normal, inactive urinary sediment, no cellular casts, urinary protein/creatinine (UPCR) ratio<56.5 mg/mmol. Partial response=SCr normal or ≤25% above baseline value, RBCs at reference range, UPCR <56.5 mg/mmoL OR ≥50% improvement in UPCR with one of the following: UPCR <113 or <339 mg/mmoL, based on the baseline ratio. No response=Not achieving complete or partial response criteria. (NCT00430677)
Timeframe: At Day 365 (end of Short-term Period) and Day 645

,,
InterventionParticipants (Number)
Day 365Day 645 (n=59, 59, and 62)
Abatacept 10/10 mg/kg3929
Abatacept 30/10 mg/kg4345
Placebo4236

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Number of Participants Achieving Renal Response

Renal response=serum creatinine level ≤25% above baseline value and greater than or equal to 50% improvement in the urine protein/creatinine ratio with 1 of the following: urine protein/creatinine ratio (UPCR) <113 mg/mmol, if the baseline ratio was <= 339 mg/mmol OR UPCR <339 mg/mmol,if the baseline ratio >339 mg/mmol. (NCT00430677)
Timeframe: At Day 365 (end of short-term period) and Day 645

,,
Interventionparticipants (Number)
Day 365Day 645 (n=59, 59, 62)
Abatacept 10/10 mg/kg6659
Abatacept 30/10 mg/kg4647
Placebo7462

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Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period

preRX=pretreatment; LLN=lower limit of normal; ULN=upper limit of normal. Hemoglobin (g/dL): >3g/dL decrease from preRX value. Hematocrit(%): <0.75*preRX. Erythrocytes (*10^6 c/uL): <0.75*preRX. Platelet count (*10^9 c/L): <0.67*LLN, or >1.5*ULN, or if preRX 1.25*ULN, or if preRX ULN; if preRX>ULN, use >1.2*preRX or 7.50*10^3 c/uL. Monocytes (absolute) (*10^3 c/uL): If value >2000/mm^3. Basophils (absolute)(*10^3 c/uL): If value >.750*10^3 c/uL. Eosinophils (absolute) (*10^3 c/uL): If value >.750*10^3 c/uL. ALP (U/L): >2*ULN, or if preRX>ULN, use >3* preRX. AST (U/L): >3*ULN, or if preRX>ULN, use >4*preRX. ALT (U/L): >3*ULN, or if preRX>ULN, use >4*preRX. GGT (U/L):>2*ULN, or if preRX >ULN, use >3*preRX. Bilirubin, total (mg/dL): >2*ULN, or if preRX>ULN, use >4*preRX. BUN (mg/dL): >1.5*preRX. (NCT00430677)
Timeframe: From start of study drug on Day 365 up to 56 days after last dose in the long-term extension period

InterventionParticipants (Number)
Hemoglobin (low)Hemoglobin (high)Hematocrit (n=210) (low)Hematocrit (n=210) (high)Erythrocytes (low)Erythrocytes (high)Platelet count (n=210) (low)Platelet count (n=210) (high)Leukocytes (low)Leukocytes (high)Neutrophils + Bands (absolute) (low)Neutrophils + Bands (absolute) (high)Lymphocytes (absolute) (low)Lymphocytes (absolute) (high)Monocytes (absolute) (low)Monocytes (absolute) (high)Basophils (absolute) (low)Basophils (absolute) (high)Eosinophils (absolute) (low)Eosinophils (absolute) (high)Alkaline phosphatase (ALP) (low)ALP (high)Aspartate aminotransferase (AST) (low)AST (high)Alanine aminotransferase (ALT) (low)ALT (high)G-glutamyl transferase (GGT) (low)GGT (high)Bilirubin, total (low)Bilirubin, total (high)Blood urea nitrogen (BUN) (low)BUN (high)Creatinine (low)Creatinine (high)
Abatacept 10 mg/kg13NA11NA10NA104094NA590NA0NA0NA8NA3NA3NA5NA14NA0NA9NA16

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Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period (Continued)

LLN=lower limit of normal; ULN=upper limit of normal; preRX=pretreatment. Sodium, serum (mEq/L): <0.95*LLN or >1.05*ULN, or if preRXULN if preRX>ULN, use >1.05*preRX or 1.1*ULN, or if preRX ULN if preRX>ULN, use >1.1*preRX or 1.1*ULN, or if preRXULN. Calcium, total (mg/dL): <0.8*LLN or >1.2*ULN, or if preRXULN if preRX>ULN, use >1.25*preRX or 220 mg/dL. Glucose, fasting serum (mg/dL): <0.8*LLN or >1.5*ULN, or if preRX ULN if preRX>ULN, use >2.0*preRX or 2*preRX. Triglycerides (mg/dL): >=2.5*ULN, or if preRX>ULN, use >=2.5*preRX. Triglycerides, fasting (mg/dL): >=2*ULN, or if preRX>ULN, use >2.0*preRX. (NCT00430677)
Timeframe: From start of study drug on Day 365 up to 56 days after last dose in the long-term extension period

InterventionParticipants (Number)
Sodium, serum (low)Sodium, serum (high)Potassium, serum (n=210) (low)Potassium, serum (n=210) (high)Chloride, serum (low)Chloride, serum (high)Calcium, total (n=210) (low)Calcium, total (n=210) (high)Glucose, serum (low)Glucose, serum (high)Glucose, fasting serum (low) (n=143)Glucose, fasting serum (high) (n=143)Protein, total (low)Protein, total (high)Albumin (low)Albumin (high)Cholesterol, total (low) (n=32)Cholesterol, total (high) (n=32)Triglycerides (low) (n=20)Triglycerides (high) (n=20)Triglycerides, fasting (low) (n=18)Triglycerides, fasting (high) (n=18)Protein, urine (low)Protein, urine (high)Glucose, urine (low)Glucose, urine (high)Blood, urine (low)Blood, urine (high)Leukocyte esterase, urine (low) (n=185)Leukocyte esterase, urine (high) (n=185)
Abatacept 10 mg/kg1211300122322219012NA32NA20NA18NANA9NA1NA35NA27

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Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period (Continued)

preRX=pretreatment. Protein, urine: If missing preRX, use >=2, or if value >=4, or if preRX =0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 OR 3 then use >=4. Glucose, urine: If missing preRX, use >=2, or if value >=4, or if preRX=0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 or 3, use >=4. Blood, urine: If missing preRX, use >=2, or if value >=4, or if preRX =0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 or 3, use >=4. Leukocyte esterase, urine: If missing preRX, use >=2, or if value >=4, or if preRX=0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 or 3, use >=4. (NCT00430677)
Timeframe: From start of study drug on Day 365 to up to 56 days after last dose in the long-term extension period

InterventionParticipants (Number)
Protein, urine (low)Protein, urine (high)Glucose, urine (low)Glucose, urine (high)Blood, urine (low)Blood, urine (high)Leukocyte esterase, urine (low) (n=185)Leukocyte esterase, urine (high) (n=185)
Abatacept 10 mg/kgNA9NA1NA35NA27

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Number of Participants With Positive Abatacept-induced Responses (ECL Method) Over Time During the Short-term Period

A validated, sensitive electrochemiluminescence (ECL) immunoassay based on Meso-Scale Discovery instrumentation was used to evaluate immunogenicity. The ECL assay differentiated between two antibody specificities: (1) the 'Ig and/or Junction (Jn) Region' and (2) 'CLTA4 and possibly Ig'. A sample was considered positive if it had a titer of 10 or greater and if immunodepletion was observed with abatacept with or without CTLA4-T. (NCT00430677)
Timeframe: Day 169, Day 365

,
InterventionParticipants (Number)
CTLA4 and possibly Ig; Day 169 (n=90)CTLA4 and possibly Ig; Day 365 (n=74)CTLA4 and possibly Ig;Overall on TRT visits (n=90)CTLA4 and possibly Ig; Overall Post visits (n=20)CTLA4 and possibly Ig; Overall (n=96)Ig/Jn region; Day 169 (n=90)Ig/Jn region; Day 365 (n=78)Ig/Jn region; Overall on TRT visits (n=90)Ig/Jn region; Overall on Post visits (n=20)Ig/Jn region; Overall (n=95)
Abatacept 10/10 mg/kg1015600011
Abatacept 30/10 mg/kg2137901101

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Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths, and Discontinuations Due to AEs Reported During the Short-term Period

AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition that may not have a causal relationship with treatment. SAE=a medical event that at any dose results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization. Related=possibly, probably, or certainly related to and of unknown relationship to study drug. (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.

,,
InterventionParticipants (Number)
DeathsSAEsRelated SAEsAEsRelated AEsDiscontinued due to AEs
Abatacept 10/10 mg/kg22819895313
Abatacept 30/10 mg/kg53320936114
Placebo7311594559

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Participants With AEs of Special Interest During the Short-term Period

AEs of special interest were prospectively identified to be those that may be associated with the use of immunomodulatory agents. They are a subset of all AEs and may be either serious or non-serious. (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.

,,
InterventionParticipants (Number)
Infections and InfestationsMalignanciesAutoimmune DisordersAcute Infusional AEsPeri-infusional AEs
Abatacept 10/10 mg/kg70151818
Abatacept 30/10 mg/kg75042323
Placebo75131717

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Participants With Marked Abnormalities Urinalysis During the Short-term Period

PTV=pretreatment value. Criteria for marked abnormality: Protein, glucose, blood, leukocyte esterase , if missing PTV then use >=2+ (or, if value >=4, or if PTV=0 or 0.5, >=2 or if PTV=1, >=3, or if PTV=2 or 3, >=4). (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.

,,
InterventionParticipants (Number)
Urine Protein (n=99,98,99)Urine Glucose (n=99,98,99)Urine Blood (n=99,98,99)Urine Leukocyte esterase (n=85,91,90)
Abatacept 10/10 mg/kg33198
Abatacept 30/10 mg/kg611610
Placebo80178

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Participants With Marked Hematology Abnormalities During the Short-term Period

LLN=lower limit of normal; ULN=upper limit of normal; PTV=pretreatment value. Normal ranges are provided by the Central Laboratory and may vary according to sex and age. Low(↓)Hemoglobin:>3g/dL decrease from PTV; ↓Hematocrit:<0.75xPTV;↓Erythrocyte count:<0.75xPTV; high(↑)Platelet count:>1.5xULN;↓Platelet count:<0.67xLLN;↓Leukocyte count:<0.75X LLN;↑Leukocyte count:>1.25xULN;↓Absolute(AB)Neutrophils+Bands:<1.00x10^3c/uL;↑AB Lymphocyte count:>7.50x10^3 c/uL; ↓AB lymphocyte count:<0.750x10^3 c/uL;↑AB monocyte count:>2000/mm^3;↑AB basophil count:>400/mm^3;↑AB eosinophil count:>0.750x10^3 c/uL. (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.

,,
InterventionParticipants (Number)
Low Hemoglobin (n=98, 98, 99)Low Hematocrit (n=97, 98, 99)Low Erythrocyte Count (n=98, 98, 99)High Platelet Count (n=98, 98, 98)Low Platelet Count (n=98, 98, 98)High Leukocyte Count (n=98, 98, 99)Low Leukocyte Count (n=98,98,99)Low Absolute Neutrophils + Bands (n=98,98,99)High Absolute Lymphocyte Count (n=98,98,99)Low Absolute Lymphocyte Count (n=98, 98, 99)High Absolute Monocyte Count (n=98, 98, 99)High Absolute Basophil Count (n=98, 98, 99)High Absolute Eosinophil Count (n=98, 98, 99)
Abatacept 10/10 mg/kg3110310182232103
Abatacept 30/10 mg/kg6541115131047100
Placebo9771111167053100

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Participants With Marked Laboratory Abnormalities During the Short-term Period

LLN=lower limit of normal; ULN=upper limit of normal; PTV=pretreatment value. Normal ranges are provided by the Central Laboratory and may vary according to sex and age. ↑Serum Sodium:>1.05x ULN;↓Serum Potassium:<0.9x LLN;↑Serum Potassium:>1.1x ULN;↓Total Calcium:<0.8X LLN;↑Total Calcium:>1.2x ULN; ↓Serum Glucose(SG):<65 mg/dL;↑SG:>220 mg/dL;↓Fasting SG:<0.8x LLN;↑Fasting SG:>1.5x ULN;↓Total Protein:<0.9x LLN;↓Albumin:<0.9x LLN;↑Total Cholesterol:>2x PTV;↑Triglycerides:>=2.5x ULN;↑Fasting Triglycerides:>=2x ULN (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.

,,
InterventionParticipants (Number)
High Serum Sodium (n=98, 98, 99)Low Serum Potassium (n=98, 98, 99)High Serum Potassium (n=98, 98, 99)Low Total Calcium (n=98, 98, 99)High Total Calcium (n=98, 98, 99)Low Serum Glucose (n=98, 98, 99)High Serum Glucose (n=98, 98, 99)Low Fasting Serum Glucose (n=72, 73, 68)High Fasting Serum Glucose (n=72, 73, 68)Low Total Protein (n=98, 98, 99)Low Albumin (n=98, 98, 99)High Total Cholesterol (n=93,93,96)High Triglycerides (n=71, 79, 75)High Fasting Triglycerides (n=65, 64, 63)
Abatacept 10/10 mg/kg21401216221186000
Abatacept 30/10 mg/kg112401122311810223
Placebo01521118200254100

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Participants With Marked Liver and Kidney Function Abnormalities During the Short-term Period

"ULN=upper limit of normal; PTV=pretreatment value. Normal ranges are provided by the Central Laboratory and may vary according to sex and age.~Alkaline Phosphatase:>2x ULN; ↑Aspartate Aminotransferase: >3x ULN; ↑Alanine Aminotransferase : >3x ULN; G-Glutamyl Transferase : >2x ULN; ↑Total Bilirubin : >2x ULN or if PTV > ULN then > 4x PTV; ↑Blood Urea Nitrogen >2x PTV; ↑Creatinine >1.5x PTV." (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.

,,
InterventionParticipants (Number)
High Alkaline PhosphataseHigh Aspartate AminotransferaseHigh Alanine AminotransferaseHigh G-Glutamyl TransferaseHigh Total BilirubinHigh Blood Urea NitrogenHigh Creatinine
Abatacept 10/10 mg/kg1047016
Abatacept 30/10 mg/kg12770516
Placebo10150612

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Vital Signs Summary During the Short-term Period: Diastolic Blood Pressure (DBP)

(NCT00430677)
Timeframe: 0 - 12 Months

,,
Interventionmm Hg (Mean)
Day 1: DBP-before infusion; n=76,81,73Day 1: DBP-1hour after infusion; n=69,78,70Day 1: DBP-2.5 hours after infusion; n=79,81,76Day 15: DBP-before infusion; n=78,78,74Day 15: DBP- 1hour after infusion; n=72,75,71Day 15: DBP-2.5 hours after infusion; n=80,78,78Day 29: DBP-before infusion; n=77,79,75Day 29: DBP-1hour after infusion; n=72,75,74Day 29: DBP-2.5 hours after infusion; n=80,79,82Day 57: DBP-before infusion; n=74,76,70Day 57: DBP-1hour after infusion; n=74,69,68Day 57: DBP-2.5 hours after infusion; n=81,73,73Day 85: DBP- before infusion; n=73,71,67Day 85: DBP-1hour after infusion; n=73,69,72Day 113: DBP-before infusion; n=72,70,70Day 113: DBP-1hour after infusion; n=75,70,75Day 141: DBP-before infusion; n=66,70,66Day 141: DBP-1hour after infusion; n=72,71,70Day 169: DBP-before infusion; n=70,70,66Day 169: DBP-1hour after infusion; n=75,71,71Day 197: DBP-before infusion; n=68,70,64Day 197: DBP-1hour after infusion; n=72,73,70Day 225: DBP-before infusion; n=70,64,63Day 225: DBP-1hour after infusion; n=72,65,68Day 253: DBP-before infusion; n=63,66,63Day 253: DBP-1hour after infusion; n=69,67,67Day 281: DBP-before infusion; n=63,66,58Day 281: DBP-1hour after infusion; n=66,65,64Day 309: DBP-before infusion; n=63,63,59Day 309: DBP-1hour after infusion; n=67,66,64Day 337: DBP-before infusion; n=63,64,61Day 337: DBP-1hour after infusion; n=68,65,67
Abatacept 10/10 mg/kg80.679.679.080.078.778.078.878.078.278.775.376.175.275.677.076.374.074.774.174.475.874.573.674.575.974.973.374.572.972.973.473.3
Abatacept 30/10 mg/kg80.879.679.381.179.681.479.679.978.580.078.979.579.778.178.576.178.877.875.576.576.876.174.575.076.174.577.675.978.176.478.078.1
Placebo80.480.180.982.680.482.881.380.879.578.778.780.278.778.176.776.976.576.177.076.475.875.475.776.077.175.776.676.677.476.676.276.1

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Vital Signs Summary During the Short-term Period: Heart Rate

(NCT00430677)
Timeframe: 0 - 12 Months

,,
Interventionbeats per minute (Mean)
Day 1: before infusion; n=99,98,100Day 1: 1hour after infusion; n=98,98,98Day 1: 2.5 hours after infusion; n=97,96,96Day 15: before infusion; n=97,93,99Day 15: 1hour after infusion; n=96,94,98Day 15: 2.5 hours after infusion; n=93,92,95Day 29: before infusion; n=94,93,99Day 29: 1hour after infusion; n=93,92,99Day 29: 2.5 hours after infusion; n=92,90,97Day 57: before infusion; n=93,88,84Day 57: 1hour after infusion; n=93,84,94Day 57: 2.5 hours after infusion; n=91,84,91Day 85: before infusion; n=89,86,91Day 85: 1hour after infusion; n=88,84,89Day 113: before infusion; n=88,82,92Day 113: 1hour after infusion; n=87,80,91Day 141: before infusion; n=84,82,89Day 141: 1hour after infusion; n=84,81,86Day 169: before infusion; n=85,82,86Day 169: 1hour after infusion; n=84,82,85Day 197: before infusion; n=83,82,84Day 197: 1hour after infusion; n=82,82,85Day 225: before infusion; n=83,76,81Day 225: 1hour after infusion; n=83,76,81Day 253: before infusion; n=78,77,81Day 253: 1hour after infusion; n=78,77,81Day 281: before infusion; n=75,76,77Day 281: 1hour after infusion; n=74,76,76Day 309: before infusion; n=76,75,77Day 309: 1hour after infusion; n=76,74,77Day 337: before infusion; n=73,75,80Day 337: 1hour after infusion; n=73,73,79
Abatacept 10/10 mg/kg79.279.682.281.080.181.780.879.982.182.281.683.482.380.480.480.279.379.178.078.878.178.777.678.079.179.378.377.676.577.476.477.4
Abatacept 30/10 mg/kg82.481.381.883.181.381.583.781.781.383.481.082.584.883.382.181.882.881.480.179.581.779.880.179.181.478.880.178.079.877.978.577.6
Placebo82.681.983.883.782.684.783.081.784.782.881.883.882.583.583.082.682.681.379.181.479.379.678.980.281.579.579.378.778.278.478.978.3

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Vital Signs Summary During the Short-term Period: Systolic Blood Pressure (SBP)

(NCT00430677)
Timeframe: 0 - 12 Months

,,
InterventionmmHg (Mean)
Day 1: SBP-before infusion; n=76,81,73Day 1: SBP-1hour after infusion; n=69,78,70Day 1: SBP-2.5 hours after infusion; n=79,81,76Day 15: SBP-before infusion; n=78,78,74Day 15: SBP- 1hour after infusion; n=72,75,71Day 15: SBP-2.5 hours after infusion; n=80,78,78Day 29: SBP-before infusion; n=77,79,75Day 29: SBP-1hour after infusion; n=72,75,74Day 29: SBP-2.5 hours after infusion; n=80,79,82Day 57: SBP-before infusion; n=74,76,70Day 57: SBP-1hour after infusion; n=74,69,68Day 57: SBP-2.5 hours after infusion; n=81,73,73Day 85: SBP- before infusion; n=73,71,67Day 85: SBP-1hour after infusion; n=73,69,72Day 113: SBP-before infusion; n=72,70,70Day 113: SBP-1hour after infusion; n=75,70,75Day 141: SBP-before infusion; n=66,70,66Day 141: SBP-1hour after infusion; n=72,71,70Day 169: SBP-before infusion; n=70,70,66Day 169: SBP-1hour after infusion; n=75,71,71Day 197: SBP-before infusion; n=68,70,64Day 197: SBP-1hour after infusion; n=72,73,70Day 225: SBP-before infusion; n=70,64,63Day 225: SBP-1hour after infusion; n=72,65,68Day 253: SBP-before infusion; n=63,66,63Day 253: SBP-1hour after infusion; n=69,67,67Day 281: SBP-before infusion; n=63,66,58Day 281: SBP-1hour after infusion; n=66,65,64Day 309: SBP-before infusion; n=63,63,59Day 309: SBP-1hour after infusion; n=67,66,64Day 337: SBP-before infusion; n=63,64,61Day 337: SBP-1hour after infusion; n=68,65,67
Abatacept 10/10 mg/kg127.8126.7127.8125.5125.9124.9126.2125.9126.4124.8122.1123.6118.7119.6121.7121.0119.2117.9117.6118.2118.9117.4118.8119.5120.2117.1116.5119.4116.3114.1117.3117.7
Abatacept 30/10 mg/kg128.2127.7127.1127.6126.9130.1125.6126.2126.3126.4124.6127.0122.0121.8122.6121.5121.4120.9118.5120.3119.0117.7118.2116.6120.6118.1120.7119.5119.4119.6119.9120.4
Placebo126.2126.0126.2129.2128.2131.2126.9128.6127.6123.0124.4126.1122.2122.0119.9120.1118.8120.4118.6118.9118.7120.4119.5118.8120.9119.7120.3121.0120.5119.2119.3119.3

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Vital Signs Summary During the Short-term Period: Temperature

(NCT00430677)
Timeframe: 0 - 12 Months

,,
Interventiondegree celcius (Mean)
Day 1: before infusion; n=99,98,99Day 1: 1hour after infusion; n=98,96,97Day 1: 2.5 hours after infusion; n=97,93,97Day 15: before infusion; n=97,94,99Day 15: 1hour after infusion; n=96,91,98Day 15: 2.5 hours after infusion; n=93,91,95Day 29: before infusion; n=94,93,99Day 29: 1hour after infusion; n=93,91,98Day 29: 2.5 hours after infusion; n=92,90,97Day 57: before infusion; n=93,88,93Day 57: 1hour after infusion; n=93,83,94Day 57: 2.5 hours after infusion; n=90,84,90Day 85: before infusion; n=89,86,91Day 85: 1hour after infusion; n=87,84,89Day 113: before infusion; n=88,82,92Day 113: 1hour after infusion; n=87,79,91Day 141: before infusion; n=84,81,89Day 141: 1hour after infusion; n=84,80,86Day 169: before infusion; n=85,81,86Day 169: 1hour after infusion; n=84,80,84Day 197: before infusion; n=83,82,85Day 197: 1hour after infusion; n=82,81,84Day 225: before infusion; n=83,76,81Day 225: 1hour after infusion; n=83,75,81Day 253: before infusion; n=78,77,80Day 253: 1hour after infusion; n=78,77,81Day 281: before infusion; n=75,76,77Day 281: 1hour after infusion; n=74,76,76Day 309: before infusion; n=76,75,77Day 309: 1hour after infusion; n=75,73,77Day 337: before infusion; n=73,75,79Day 337: 1hour after infusion; n=73,72,79
Abatacept 10/10 mg/kg36.536.536.536.536.536.536.436.536.536.436.436.536.436.436.436.436.436.436.436.436.336.336.436.436.436.436.436.436.336.436.436.4
Abatacept 30/10 mg/kg36.636.636.636.536.636.636.536.536.536.536.136.536.436.436.536.436.536.536.536.436.536.436.536.536.536.536.536.536.536.536.536.5
Placebo36.536.636.636.536.536.636.536.536.536.536.636.536.536.536.536.536.536.536.536.536.536.536.436.536.536.536.536.536.536.536.536.5

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Number of Participants (Patients) Who Attained Neutrophil Engraftment

"Defined as absolute neutrophils (ANC) > 5 x 10^8/Liter for 3 consecutive days.~ANC is the real number of white blood cells (WBCs) that are neutrophils. The absolute neutrophil count is commonly called the ANC. The ANC is not measured directly. It is derived by multiplying the WBC count times the percent of neutrophils in the differential WBC count. The percent of neutrophils consists of the segmented (fully mature) neutrophils) + the bands (almost mature neutrophils). The normal range for the ANC = 1.5 to 8.0 (1,500 to 8,000/mm3)." (NCT00354172)
Timeframe: Day 42 Post Transplant

InterventionParticipants (Number)
Evaluable Patients13

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Number of Participants (Patients) Who Attained Platelet Engraftment

Platelet engraftment is defined as platelet counts > 50 x 10^9/Liter for 3 consecutive days. (NCT00354172)
Timeframe: 1 Year Post Transplant

InterventionParticipants (Number)
Evaluable Patients5

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Number of Participants (Patients) Who Died by 12 Months

Number of patients who died after receiving treatment within 12 months post transplant. (NCT00354172)
Timeframe: 1 year Post Transplant

InterventionParticipants (Number)
Evaluable Patients14

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Number of Participants (Patients) Who Died by 24 Months

Number of patients who died after receiving treatment within 24 months post transplant. (NCT00354172)
Timeframe: 2 years post-transplant

InterventionParticipants (Number)
Evaluable Patients15

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Number of Participants (Patients) Who Died Due to Transplant.

Patients who had transplant-related mortality (TRM). TRM = adverse event(s) that occur(s) after the patient has received a transplant, the principal investigator decides it is related to the procedure and the patient dies within 6 months. (NCT00354172)
Timeframe: 6 Months Post Transplant

InterventionParticipants (Number)
Evaluable Patients4

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Number of Participants (Patients) Who Experienced Relapse by 12 Months

Number of patients who experienced recurrence or progression of disease from the time of transplant. (NCT00354172)
Timeframe: 1 Year Post Transplant

InterventionParticipants (Number)
Evaluable Patients10

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Number of Participants (Patients) Who Experienced Relapse by 24 Months

Number of patients who experienced recurrence or progression of disease from the time of transplant. (NCT00354172)
Timeframe: 2 Years Post transplant

InterventionParticipants (Number)
Evaluable Patients11

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Number of Participants (Patients) Who Were Disease-free and Alive at 12 Months

Number of patients who were alive and free of disease (malignancy) at 12 months after transplant. (NCT00354172)
Timeframe: 12 Months Post transplant

InterventionParticipants (Number)
Evaluable Patients1

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Number of Participants (Patients) Who Were Disease-free and Alive at 6 Months

Number of patients who were alive and free of disease (malignancy) at 6 months after transplant. (NCT00354172)
Timeframe: 6 Months Post Transplant

InterventionParticipants (Number)
Evaluable Patients2

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Number of Participants (Patients) With Acute Graft-versus-host Disease (GVHD) Grade II-IV

Graft-versus-host disease (GVHD) is a common complication of transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. The acute or fulminant form of the disease (aGVHD) is normally observed within the first 100 days post-transplant, and is a major challenge to transplants owing to associated morbidity and mortality. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of I to a high of IV. Patients with grade IV GVHD usually have a poor prognosis. (NCT00354172)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Number)
Evaluable Patients6

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Number of Participants (Patients) With Acute Graft-versus-Host Disease at Grade III-IV

Graft-versus-host disease (GVHD) is a common complication of transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. The acute or fulminant form of the disease (aGVHD) is normally observed within the first 100 days post-transplant, and is a major challenge to transplants owing to associated morbidity and mortality. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of I to a high of IV. Patients with grade IV GVHD usually have a poor prognosis. (NCT00354172)
Timeframe: Day 100 post transplant

InterventionParticipants (Number)
Evaluable Patients1

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Number of Participants (Patients) With Chronic Graft-Versus-Host Disease

The chronic form of graft-versus-host-disease (cGVHD) normally occurs after 100 days. The appearance of moderate to severe cases of cGVHD adversely influences long-term survival. (NCT00354172)
Timeframe: Day 100 through 1 Year Post Transplant

InterventionParticipants (Number)
Evaluable Patients1

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Number of Participants (Patients) With Successful Natural Killer Cell Expansion

Defined by an absolute circulating donor-derived natural killer cell count of >100 cells/microliter 10-13 days after infusion with <5% donor T and B cells in the mononuclear population (NCT00354172)
Timeframe: 10-13 Days Post Infusion

InterventionParticipants (Number)
Evaluable Patients3

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Number of Patients Who Were Disease-free and Alive at 24 Months

Number of patients who were alive and free of disease (malignancy) at 24 months after transplant. (NCT00354172)
Timeframe: 24 Months Post transplant

InterventionParticipants (Number)
Evaluable Patients0

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Chimerism After Double Umbilical Cord Blood Transplant (UCBT)

Calculation of Median (range) of percentage of donor cells engrafted (present) in the recipient (patient). (NCT00354172)
Timeframe: Day 21, Day 100, 6 Months

InterventionPercentage of Engrafted Cells (Median)
Day 21Day 1006 Months
Evaluable Patients9210096.5

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GFR at Month 12 Utilizing Cockcroft-Gault Formula

Cockcroft-Gault formula: For men: GFR= ((140-age) × body weight in kg)∕(72 x serum creatinine in mg∕dl)For women: GFR= (0.85×(140-age) × body weight in kg)∕(72 x serum creatinine in mg/dl), ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen60.18
CNI Free Regimen64.87
CNI Low Regimen61.16

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GFR at Month 12 Utilizing Modification of Diet in Renal Disease (MDRD) Method

"Change in GFR (Modification of Diet in Renal Disease calculated using the -MDRD formulat:~For men: GFR = 170 × (serum creatinine -0,999)×(age-0,176) x (urea nitrogen -0,17) × (albumin0,318) For women: GFR = 170 × (serum creatinine -0,999) × (age-0,176) × (urea nitrogen -0,17) x (albumin0,318) × 0.762 with urea nitrogen = urea / 2.144. ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate." (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen50.23
CNI Free Regimen56.36
CNI Low Regimen50.24

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GFR at Month 60 Utilizing Cockcroft-Gault Formula

Cockcroft-Gault formula: For men: GFR= ((140-age) × body weight in kg)∕(72 x serum creatinine in mg∕dl) For women: GFR= (0.85×(140-age) × body weight in kg)∕(72 x serum creatinine in mg/dl), ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 60 post-transplant

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen55.92
CNI Free Regimen61.6
CNI Low Regimen52.91

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GFR at Month 60 Utilizing Modification of Diet in Renal Disease (MDRD) Method

"Change in GFR (Modification of Diet in Renal Disease calculated using the -MDRD formulat:~For men: GFR = 170 × (serum creatinine -0,999)×(age-0,176) x (urea nitrogen -0,17) × (albumin0,318) For women: GFR = 170 × (serum creatinine -0,999) × (age-0,176) × (urea nitrogen -0,17) x (albumin0,318) × 0.762 with urea nitrogen = urea / 2.144. ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate." (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 60 post-transplant

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen47.56
CNI Free Regimen53.41
CNI Low Regimen44.79

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GFR Calculated Via Nankivell Formula at Month 60

Change in GFR using the Nankivell formula (GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)² + C where where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kilograms, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. The calculated GFR is expressed in mL/min per 1.73m², last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate. (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 60

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen60.24
CNI Free Regimen66.98
CNI Low Regimen58.74

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GFR Via Nankivell Formula at Month 12 - All Regimens

Change in GFR using the Nankivell formula (GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)² + C where where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kilograms, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. The calculated GFR is expressed in mL/min per 1.73m², last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate. (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen63.03
CNI Free Regimen68.59
CNI Low Regimen63.08

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GFR Via Nankivell Method at Month 12 - CNI-Free vs Standard Regimen

Demonstrate superiority of CNI-Free vs Standard Regimen in GFR using the Nankivell formula (GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)² + C where where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kilograms, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. The calculated GFR is expressed in mL/min per 1.73m², last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate. P-values are not adjusted (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant

Interventionml/min/1.73m² (Least Squares Mean)
Standard Regimen63.03
CNI Free Regimen68.59
CNI Low Regimen63.08

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Mean Change in Serum Creatinine From Month 3 to Month 12

Change in venous blood serum creatinine. Last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant

Interventionmg/dl (Least Squares Mean)
Standard Regimen1.66
CNI Free Regimen1.58
CNI Low Regimen1.76

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Mean Change in Serum Creatinine From Month 3 to Month 60

Change in venous blood serum creatinine. Last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 60 post-transplant

Interventionmg/dl (Least Squares Mean)
Standard Regimen1.94
CNI Free Regimen1.69
CNI Low Regimen2.01

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Change From BL2 (Month 3) to Month 12 in Cardiovascular Risk (Framingham Score; 10-year Cardiovascular Risk)

The Framingham Score (based on LDL cholesterol level) estimates the coronary heart disease risk (%) of developing one of the following coronary heart diseases: angina pectoris, myocardial infarction, or coronary disease death, over the course of 10 years. (NCT00514514)
Timeframe: From Baseline 2 (Month 3) to Month 12

,,
InterventionPercent risk (Mean)
Baseline 1/Visit 1 (n=165,171,161)Baseline 2/Month 3 (n=165,171,161)Month 12 (n=158,166,156)Change from Baseline 2 to Month 12 (n=158,166,156)
CNI Free Regimen10.28.89.10.4
CNI Low Regimen9.59.38.7-0.7
Standard Regimen10.910.39.4-0.7

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Efficacy Event Data After Month 12 to Month 60

Efficacy events were: Biopsy-proven acute rejection (BPAR), graft loss, death, and treatment failure (defined as composite endpoint of BPAR, graft loss, death, loss to follow-up, discontinuation due to lack of efficacy or due to toxicity). (NCT00514514)
Timeframe: Events starting after Month 12

,,
InterventionParticipants (Number)
BPARGraft lossDeathLost to follow-upDiscontinuation due to adverse eventTherapy failure (composite endpoint)
CNI Free Regimen137415835
CNI Low Regimen123913436
Standard Regimen1377171038

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Efficacy Event Data Baseline 2 (Month 3) to Month 12

Efficacy events were: Biopsy-proven acute rejection (BPAR), graft loss, death, and treatment failure (defined as composite endpoint of BPAR, graft loss, death, loss to follow-up, discontinuation due to lack of efficacy or due to toxicity). (NCT00514514)
Timeframe: From Baseline 2 (Month 3) to Month 12

,,
InterventionParticipants (Number)
BPARGraft lossDeathLost to follow-upDiscontinuation due to lack of efficacyDiscontinuation due to adverse eventTherapy failure composite
CNI Free Regimen2022034458
CNI Low Regimen1312012735
Standard Regimen1313022534

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Efficacy Event Data From Baseline 2 (Month 3) to Month 6

Efficacy events were: Biopsy-proven acute rejection (BPAR), graft loss, death, and treatment failure (defined as composite endpoint of BPAR, graft loss, death, loss to follow-up, discontinuation due to lack of efficacy or due to toxicity). (NCT00514514)
Timeframe: From Baseline 2 (Month 3) to Month 6

,,
InterventionParticipants (Number)
BPARGraft lossDeathLost to follow-upDiscontinuation due to lack of efficacyDiscontinuation due to adverse eventTherapy failure composite
CNI Free Regimen1511022637
CNI Low Regimen1010011319
Standard Regimen60101814

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Gastrointestinal Toxicity Due to Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Formulation Manufactured by Sandoz

At 24 weeks, we assessed the number of patients at this timepoint who required permanent dose decrease or discontinuation of either enteric coated mycophenolate sodium or mycophenolate mofetil or its generic equivalent formulation manufactured by Sandoz related to gastrointestinal toxicity. (NCT00522548)
Timeframe: 6 months

Interventionparticipants (Number)
Myfortic Comparator Group9
CellCept Comparator Group4

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Modification of Diet and Renal Disease (MDRD) Measured Glomerular Filtration Rates in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz

Modification of Diet and Renal Disease (MDRD) Measured Glomerular Filtration Rates were used as part of a measure of renal function and measured at 24 weeks in patients who were still participating in the study at that point. (NCT00522548)
Timeframe: 6 months

InterventionMDRD (mL/min/1.73m2) (Mean)
Myfortic Comparator Group59.6
CellCept Comparator Group54.8

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Serum Creatinine in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz

Serum creatinine lab values were used as part of a measure of renal function at 24 weeks for any patient that was still participating in the study at this time-point. (NCT00522548)
Timeframe: 6 months

Interventionserum creatinine mg/dL (Mean)
Myfortic Comparator Group1.9
CellCept Comparator Group1.9

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The Incidence of Intolerance (Defined as Transient Dose Reduction or Transient Discontinuation of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz

The incidence of intolerance was defined as transient dose reduction or transient discontinuation of enteric coated mycophenolate sodium or mycophenolate mofetil or its generic equivalent manufactured by Sandoz meaning doses could subsequently be resumed or increased back to original starting dose, once intolerance resolved. (NCT00522548)
Timeframe: 6 months

Interventionparticipants (Number)
Myfortic Comparator Group1
CellCept Comparator Group0

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The Incidence of Rejection in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz.

All rejection episodes of the kidney transplant were proven by kidney transplant biopsy and were measured at 24 weeks for all patients still participating in the study at that timepoint. (NCT00522548)
Timeframe: 6 months

Interventionparticipants (Number)
Myfortic Comparator Group2
CellCept Comparator Group1

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The Incidence of the Requirement of Full Dose Proton Pump Inhibitors in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz

If patients were not on any medications from a medication class known as H-2 antagonists, they were started on ranitidine 150 mg orally twice daily after transplant (with dose adjusted for renal function). Patients were excluded if they were on proton pump inhibitor at time of study screening, however some patients required addition of proton pump inhibitor post-study enrollment. If a patient had upper gastrointestinal side effects such as acid reflux unreleived on ranitidine therapy or nausea, they were switched to a proton pump inhibitor. (NCT00522548)
Timeframe: 6 months

Interventionparticipants (Number)
Myfortic Comparator Group3
CellCept Comparator Group1

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Gastrointestinal Symptom Rating Scale (GSRS) Score Changes From Baseline to 24 Weeks After Transplant in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz

The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item self-administered questionnaire to assess symptoms associated with common gastrointestinal (GI) disorders, and has been validated in renal transplant recipients. It uses a seven-graded Likert scale, where 1 represents the most positive option and 7 the most negative one and the patient grades symptoms based on the past 7 days. A score of ≥ 2 indicates the presence of GI symptoms. Higher values indicate more unfavorable conditions. The questionairre is divided into 5 sub-scales: diarrhea, indigestion, constipation, abdominal pain, and reflux and a mean score is calculated for each dimension. The lowest mean score possible for each dimension is 1 and the highest is 7. Patients completed a GSRS survey at baseline (pre-transplant to two days after transplant), and at weeks 1,4,12 and 24 after transplant. (NCT00522548)
Timeframe: baseline (pre-transplant to two days after transplant) and at 6 months after transplant.

,
InterventionParticipants (Count of Participants)
Worsening of GSRS Score pre-transplant-wk 24No Worsening of GSRS Score pre-transplant-wk 24
CellCept Comparator Group410
Myfortic Comparator Group413

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Pharmacokinetics (Mycophenolic Acid Area Under the Curve) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients

Pharmacokinetics is the study of how a drug is absorbed and broken down in the body. Pharmacokinetics of Enteric-Coated Mycophenolate Sodium or Mycophenolate Mofetil are measured by a level called mycophenolic acid level. Mycophenolic acid levels in this pharmacokinetic study were drawn starting in the morning at time zero (immediately prior to morning dose, also known as trough), then at 0.5, 1,1.5, 2, 2.5, 3,3.5,4,6, 8 and 12 hours post dose. Data was also dose-normalized (concentration was divided by dose). A mycophenolic acid area under the curve value, also known as AUC represents drug exposure. (NCT00522548)
Timeframe: 1 and 6 months

,
Interventionmg*hr/L (Mean)
Mycophenolic Acid AUC (mg*hr/L) at week 4Mycophenolic Acid AUC (mg*hr/L) at week 24
CellCept Comparator Group33.842.4
Myfortic Comparator Group23.921.1

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Pharmacokinetics (Mycophenolic Acid Maximum Concentration) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients

Pharmacokinetics is the study of how a drug is absorbed and broken down in the body. Pharmacokinetics of Enteric-Coated Mycophenolate Sodium or Mycophenolate Mofetil are measured by a level called mycophenolic acid level. Mycophenolic acid levels in this pharmacokinetic study were drawn starting in the morning at time zero (immediately prior to morning dose, also known as trough), then at 0.5, 1,1.5, 2, 2.5, 3,3.5,4,6, 8 and 12 hours post dose. Data was also dose-normalized (concentration was divided by dose).A mycophenolic acid drawn at the peak level is called C Max or maximum concentration. (NCT00522548)
Timeframe: 1 and 6 months

,
Interventionmg/L (Mean)
Mycophenolic Acid C max (mg/L) at week 4Mycophenolic Acid C max (mg/L) at week 24
CellCept Comparator Group10.513.9
Myfortic Comparator Group4.36.4

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Pharmacokinetics (Mycophenolic Acid Trough Levels) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients

Pharmacokinetics is the study of how a drug is absorbed and broken down in the body. Pharmacokinetics of enteric coated mycophenolate sodium and mycophenolate mofetil are measured by a level called mycophenolic acid level. Mycophenolic acid levels in this pharmacokinetic study were drawn starting in the morning at time zero (immediately prior to morning dose, also known as trough), then at 0.5, 1,1.5, 2, 2.5, 3,3.5,4,6, 8 and 12 hours post dose. Data was also dose-normalized (concentration was divided by dose). (NCT00522548)
Timeframe: 1 and 6 months

,
Interventionmg/L (Mean)
Mycophenolic Acid Trough (mg/L) at week 4Mycophenolic Acid Trough(mg/L) at week 24
CellCept Comparator Group1.91.5
Myfortic Comparator Group2.41.9

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The Incidence of the Occurrence of Lower Gastrointestinal Symptoms Per GSRS Scale Ratings in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz

The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item self-administered questionnaire to assess symptoms associated with common gastrointestinal (GI) disorders, and has been validated in renal transplant recipients. It uses a seven-graded Likert scale, where 1 represents the most positive option and 7 the most negative one and the patient grades symptoms based on the past 7 days. A score of ≥ 2 indicates the presence of GI symptoms. Higher values indicate more unfavorable conditions. The questionairre is divided into 5 sub-scales: diarrhea, constipation, abdominal pain, indigestion and reflux and a mean score is calculated for each dimension. The lowest mean score possible for each dimension is 1 and the highest is 7. Patients completed a GSRS survey at baseline (pre-transplant to two days after transplant), and at weeks 1,4,12 and 24 post-transplant. Patients who withdrew from the study only had data included up to the point of withdraw. No values were carried forward. (NCT00522548)
Timeframe: 6 months

,
InterventionParticipants (Count of Participants)
Number of Participants with Lower GI symptomsNumber of Participants without Lower GI symptoms
CellCept Comparator Group95
Myfortic Comparator Group89

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The Incidence of the Occurrence of Upper Gastrointestinal Symptoms Per GSRS Scale Ratings in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Formulation Manufactured by Sandoz

The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item self-administered questionnaire to assess symptoms associated with common gastrointestinal (GI) disorders, and has been validated in renal transplant recipients. It uses a seven-graded Likert scale, where 1 represents the most positive option and 7 the most negative one and the patient grades symptoms based on the past 7 days. A score of ≥ 2 indicates the presence of GI symptoms. Higher values indicate more unfavorable conditions. The questionairre is divided into 5 sub-scales: diarrhea, constipation, abdominal pain, indigestion and reflux and a mean score is calculated for each dimension. The lowest mean score possible for each dimension is 1 and the highest is 7. Patients completed a GSRS survey at baseline (pre-transplant to two days after transplant), and at weeks 1,4,12 and 24 post-transplant. (NCT00522548)
Timeframe: 6 months

,
InterventionParticipants (Count of Participants)
Number of Participants with Upper GI symptomsNumber of Participants without Upper GI symptoms
CellCept Comparator Group113
Myfortic Comparator Group89

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Change From Baseline in the Average Maximum Intimal Thickness at Month 12

Maximum intimal thickness was assessed using Intravascular Ultrasound (IVUS). IVUS is a technique for taking ultrasound pictures of the wall of an artery from inside the artery itself. It shows the thickness of the artery wall and any narrowing of the artery. (NCT00300274)
Timeframe: Baseline, Month 12

Interventionmm (Mean)
Everolimus 1.5 mg0.03
Everolimus 3.0 mg0.04
Mycophenolate Mofetil0.07

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Percentage of Participants With Cardiac Allograft Vasculopathy (CAV) at Month 12

Cardiac allograft vasculopathy is defined as a 0.5 mm increase in maximum intimal thickness as measured by Intravascular Ultrasound (IVUS) in at least one matched slice between baseline and Month 12. (NCT00300274)
Timeframe: 12 Months

InterventionPercentage of participants (Number)
Everolimus 1.5 mg12.5
Everolimus 3.0 mg21.6
Mycophenolate Mofetil26.7

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Percentage of Participants With Composite Efficacy Failure at 12 Months

"Composite efficacy failure was defined as Biopsy Proven Acute Rejection(BPAR) of International Society for Heart and Lung Transplantation(ISHLT) grade ≥3A, Acute Rejection associated with Hemodynamic Compromise, Graft loss/Retransplant, Death or Loss to follow-up.~Identification of acute rejection was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤30% or 25% lower than Baseline or Fractional shortening ≤20% or 25% lower than Baseline and/or use of inotropic treatment." (NCT00300274)
Timeframe: 12 Months

InterventionPercentage of participants (Number)
Everolimus 1.5 mg35.1
Everolimus 3.0 mg35.1
Mycophenolate Mofetil33.6

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Percentage of Participants With Composite Efficacy Failure at 24 Months

"Composite efficacy failure was defined as Biopsy Proven Acute Rejection (BPAR) of International Society for Heart and Lung Transplantation grade ≥ 3A, Acute Rejection associated with Hemodynamic Compromise, Graft loss/Retransplant, Death or Loss to follow-up.~Identification of acute rejections was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤ 30% or 25% lower than Baseline or Fractional shortening ≤ 20% or 25% lower than Baseline and/or use of inotropic treatment." (NCT00300274)
Timeframe: 24 Months

InterventionPercentage of participants (Number)
Everolimus 1.5 mg39.4
Everolimus 3.0 mg41.1
Mycophenolate Mofetil41.3

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Percentage of Participants With Graft Loss/Re-transplant, Death or Loss to Follow-up at 12 Months

Loss to follow-up for this composite endpoint included participants who did not experience graft loss/re-transplant or death and whose last day of contact was prior to Day 316 (start day of the Month 12 visit window). (NCT00300274)
Timeframe: 12 Months

InterventionPercentage of participants (Number)
Everolimus 1.5 mg11.7
Everolimus 3.0 mg11.9
Mycophenolate Mofetil8.9

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Percentage of Participants With Graft Loss/Re-transplant, Death or Loss to Follow-up at 24 Months

Loss to follow-up for this composite endpoint included participants who did not experience graft loss/re-transplant or death and whose last day of contact was prior to Day 631 (start day of 24 Month visit window). (NCT00300274)
Timeframe: 24 Months

InterventionPercentage of participants (Number)
Everolimus 1.5 mg15.2
Everolimus 3.0 mg16.1
Mycophenolate Mofetil15.1

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Renal Function Calculated by Glomerular Filtration Rate (GFR) at 24 Months

"GFR was calculated using the Modification of Diet and Renal Disease (MDRD) formula:~GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R~C is the serum concentration of creatinine [mg/dL] A is age [years] G=0.742 when gender is female, otherwise G=1 R=1.21 when race is black, otherwise R=1" (NCT00300274)
Timeframe: 24 Months

InterventionmL/min/1.73^2 (Mean)
Everolimus 1.5 mg59.50
Everolimus 3.0 mg61.84
Mycophenolate Mofetil64.52

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Renal Function Measured by Glomerular Filtration Rate (GFR) at 12 Months

"GFR was calculated using the Modification of Diet and Renal Disease (MDRD) formula:~GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where C is the serum concentration of creatinine [mg/dL] A is age [years] G=0.742 when gender is female, otherwise G=1 R=1.21 when race is black, otherwise R=1" (NCT00300274)
Timeframe: 12 Months

InterventionmL/min/1.73^2 (Mean)
Everolimus 1.5 mg59.21
Everolimus 3.0 mg59.78
Mycophenolate Mofetil64.37

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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR of ISHLT Grade ≥ 3A), Acute Rejection (AR) Associated With Hemodynamic Compromise (HDC), Graft Loss/Re-transplant and Death at Month 24

"Identification of acute rejections was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤ 30% or 25% lower than Baseline or Fractional shortening ≤ 20% or 25% lower than Baseline, and/ or use of inotropic treatment." (NCT00300274)
Timeframe: 24 Months

,,
InterventionPercentage of participants (Number)
AR associated with HDCBPAR of ISHLT grade ≥ 3ADeathGraft loss/re-transplant
Everolimus 1.5 mg4.324.110.62.5
Everolimus 3.0 mg3.628.611.93.0
Mycophenolate Mofetil5.227.39.23.7

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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR of ISHLT Grade ≥ 3A), Acute Rejection Associated With Hemodynamic Compromise (HDC), Graft Loss/Re-transplant and Death at Month 12

"Identification of acute rejections was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤ 30% or 25% lower than Baseline or Fractional shortening ≤ 20% or 25% lower than Baseline, and/or use of inotropic treatment." (NCT00300274)
Timeframe: 12 Months

,,
InterventionPercentage of participants (Number)
AR associated with HDCBPAR of ISHLT ≥ 3ADeathGraft loss/re-transplant
Everolimus 1.5 mg3.922.37.81.4
Everolimus 3.0 mg3.025.610.13.0
Mycophenolate Mofetil2.624.74.81.8

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Donor Stem Cell Engraftment, Including Donor-host Hematopoietic Chimerism Studies Post Transplant

Engraftment of donor cells by week 4 after transplantation. Assessment of donor stem cell engraftment will include donor-host hematopoietic chimerism analyses at 4 weeks and every 3 months after transplantation. Chimerism measured from peripheral blood or from bone marrow. (NCT00548717)
Timeframe: 30 days

Interventionparticipants (Number)
Siro/MMF9
Siro/MMF/Bort2

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Incidence of 100 Day Mortality

(NCT00548717)
Timeframe: 100 days

Interventionpercentage of participants (Number)
Siro/MMF31
Siro/MMF/Bort0

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Incidence of Chronic GVHD

"Chronic GVHD will be graded according to the modified Seattle criteria. Chronic GVHD divided into limited and extensive and evaluated across the following systems: skin, cutaneous structures, liver, mouth, eyes, esophagus, intestines, lung, musculoskeletal, serous, nervous, urologic, vagina, hematopoietic, and immune.~Localized skin involvement with or without hepatic dysfunction is classified as limited disease.~Generalized skin involvement or limited disease plus eye involvement, oral involvement, hepatic dysfunction with abnormal liver histology, or involvement of any other target organ was classified as extensive disease.~Lee SJ, Vogelsang G, Flowers ME. Chronic graft-versus-host disease. Biol Blood Marrow Transplant.~2003;9:215-33." (NCT00548717)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Siro /MMF15
Siro/MMF/Bort50

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Overall Survival

(NCT00548717)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Siro/MMF39
Siro/MMF/Bort0

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The Rate of Renal Insufficiency

"Renal function will be measured weekly after transplant for 4 weeks, at 8 weeks and then at 3 month intervals from transplantation. Sentinel renal events such as the occurrence of thrombotic microangiopathy will be noted.~The rationale for the substitution of mycophenolate mofetil for tacrolimus is to continue to prevent acute GVHD, but minimize renal toxicity after transplantation. We will thus monitor renal toxicity closely in this study. In our previous experience, the rate of grade III-V renal toxicity by day 100 after transplantation was about 10%. Here, grade III is defined as a creatinine level between 3.1 to 6 times the normal level, grade IV is defined as a creatinine level 6 times or more the normal level, and grade V is defined as a fatality due to renal toxicity." (NCT00548717)
Timeframe: 1 year

Interventionparticipants (Number)
Siro/MMF1
Siro/MMF/Bort0

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To Determine the Rate of Grade II-IV Acute GVHD When Sirolimus and Mycophenolate Mofetil or Sirolimus, Mycophenolate Mofetil and Bortezomib is Used for GVHD Prophylaxis After Allogeneic Stem Cell Transplantation in Patients With Hematologic Malignancies

(NCT00548717)
Timeframe: 150 days

Interventionpercentage of participants (Number)
Siro/MMF77
Siro/MMF/Bort100

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To Correlate the Serum Concentrations of Mycophenolate Mofetil and Its Metabolites With Acute GVHD Incidence

This outcome measure was presented as a comparison between incidence of Grade 0-I aGVHD and Grade II-IV aGVHD across 5 time points (Weeks 1,2,3,8, and 12). (NCT00548717)
Timeframe: 1 year

Interventionng/mL (Mean)
Week 1 MMF level (0-I aGVHD), n=3Week 1 MMF level (II-IV aGVHD), n=8Week 2 MMF level (0-I aGVHD), n=3Week 2 MMF level (II-IV aGVHD), n=6Week 3 MMF level (0-I aGVHD), n=3Week 3 MMF level (II-IV aGVHD), n=6Week 8 MMF level (0-I aGVHD), n=3Week 8 MMF level (II-IV aGVHD), n=4Week 12 MMF level (0-I aGVHD), n=2Week 12 MMF level (II-IV aGVHD), n=4
Siro/MMF2.432.703.403.072.572.852.372.131.752.20

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Number of Participants With Acute Graft-versus-host Disease (aGVHD)

Participants who had acute graft-versus-host disease (aGVHD) within 100 days post transplant. Physical exam and bloodwork every week (for the first 90-100 days after the transplant). (NCT00506948)
Timeframe: Baseline to 100 days post transplant

Interventionparticipants (Number)
Thymoglobulin + Sirolimus + MMF6

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Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Deaths

Treatment Failure was adjudicated by a clinical endpoints committee (CEC) and was defined as the time to the earliest occurrence of any one of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or a requirement for rescue therapy for exacerbation or deterioration of Lupus nephritis (LN). (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

InterventionDeaths (Number)
Mycophenolate Mofetil (MMF)0
Azathioprine (AZA)1

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Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Participants With End-stage Renal Disease (ESRD)

Time to treatment failure, adjudicated by the Clinical Endpoints Committee (CEC), was defined as any 1 the following: death, ESRD, sustained doubling of serum creatinine, renal flare (proteinuric or nephritic), or requirement for rescue therapy to treat deterioration or exacerbation of Lupus nephritis. ESRD is defined as progression to chronic hemodialysis or renal transplant. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

Interventionparticipants (Number)
Mycophenolate Mofetil (MMF)0
Azathioprine (AZA)3

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Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Participants With Sustained Doubling of Serum Creatinine

Sustained doubling of serum creatinine concentration is defined as the first serum creatinine value that is twice the mean of the lowest 2 values from screening to end of induction, as confirmed by a second serum creatinine value obtained at least 4 weeks after the initial doubling. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

Interventionparticipants (Number)
Mycophenolate Mofetil1
Azathioprine5

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Maintenance Phase: Participants With Major Extra-renal Flare

A major extra-renal flare is defined as a British Isles Lupus Assessment Group (BILAG) Score category A in one extrarenal organ or three organs with concurrent category B scores. BILAG indices provide a scoring system for the assessment of lupus disease activity in terms of the need for steroid treatment in 8 organs/systems. Eighty-six items were scored resulting in a classification of A (severe activity), B (moderate activity), C (mild activity), D (no current activity) and E (no activity ever observed) for each organ system. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

Interventionparticipants (Number)
Mycophenolate Mofetil7
Azathioprine6

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Induction Phase: Change From Baseline in Short-Form Health Survey (SF-36) Domain and Component Scores

The SF-36 is a 36 item quality of life questionnaire. The short-form version has eleven questions that permit the participant to rate how they feel that particular day. The SF-36 consists of eight scaled scores and two component scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 score with the higher scores indicating better quality of life. (NCT00377637)
Timeframe: Baseline and 24 weeks

,
InterventionScores on a scale (Mean)
Physical Component Summary [n=139, 137]Mental Component Summary [n=139, 137]Bodily Pain Score [n=141, 137]General Health Score [n=139, 137]Mental Health Score [n=141, 137]Physical functioning Score [n=141, 137]Role-Emotional Score [n=141, 137]Role-Physical Score [n=141, 137]Social Function Score [n=141, 137]Vitality Score [n=141, 137]
Intravenous Cyclophosphamide6.45.716.811.59.89.318.434.018.211.6
Mycophenolate Mofetil5.26.713.49.19.311.623.428.617.714.2

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Induction Phase: Change From Baseline to Week 24 in 24-hour Urine Protein

24-hour urine protein was measured at Baseline and Week 24. (NCT00377637)
Timeframe: Baseline, Week 24

,
Interventionmg/day (Mean)
Baseline [n=180, 180]Week 24 [n= 150, 144]Change from Baseline to Week 24 [n= 146, 142]
Intravenous Cyclophosphamide4451.41831.6-2513.7
Mycophenolate Mofetil4208.91599.0-2510.6

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Induction Phase: Change From Baseline to Week 24 in Serum Albumin

(NCT00377637)
Timeframe: Baseline, Week 24

,
Interventiong/L (Mean)
Baseline [n=184, 185]Week 24 [n=155, 151]Change from Baseline to Week 24 [n=154, 151]
Intravenous Cyclophosphamide28.638.39.0
Mycophenolate Mofetil30.538.47.5

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Induction Phase: Change From Baseline to Week 24 in Serum Creatinine

(NCT00377637)
Timeframe: Baseline, Week 24

,
Interventionµmol/L (Mean)
Baseline [n= 184, 185]Week 24 [n= 155, 151]Change from Baseline to Week 24 [n= 154, 151]
Intravenous Cyclophosphamide92.783.5-5.1
Mycophenolate Mofetil108.677.6-18.9

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Induction Phase: Change in Renal British Isles Lupus Assessment Group (BILAG) Score

"BILAG indices provide a scoring system for the assessment of lupus disease activity in terms of the need for steroid treatment in 8 organs/systems. Eighty-six items were scored resulting in a classification of A (severe activity), B (moderate activity), C (mild activity), D (no current activity) and E (no activity ever observed) for each organ system. The BILAG individual system summaries were calculated by a program supplied by ADS-Limathon (Sheffield, UK).~The score at baseline was compared to the score at the 24 week endpoint for each treatment group, reported here for the renal system." (NCT00377637)
Timeframe: Baseline, 24 weeks

,
InterventionPercentage of participants (Number)
Shift from Baseline=A to 24 Week Endpoint=AShift from Baseline=A to 24 Week Endpoint=BShift from Baseline=A to 24 Week Endpoint=CShift from Baseline=A to 24 Week Endpoint=DShift from Baseline=B to 24 Week Endpoint=AShift from Baseline=B to 24 Week Endpoint=BShift from Baseline=B to 24 Week Endpoint=CShift from Baseline=B to 24 Week Endpoint=DShift from Baseline=C to 24 Week Endpoint=AShift from Baseline=C to 24 Week Endpoint=BShift from Baseline=C to 24 Week Endpoint=CShift from Baseline=C to 24 Week Endpoint=DShift from Baseline=D to 24 Week Endpoint=AShift from Baseline=D to 24 Week Endpoint=BShift from Baseline=D to 24 Week Endpoint=CShift from Baseline=D to 24 Week Endpoint=D
Intravenous Cyclophosphamide27.134.824.99.40.01.11.70.00.00.60.00.00.00.00.60.0
Mycophenolate Mofetil17.139.233.15.50.01.72.21.10.00.00.00.00.00.00.00.0

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Induction Phase: Number of Participants Achieving Complete Remission

Number of participants achieving complete remission as defined by return to normal serum creatinine, proteinuria ≤500 mg/24 hours and an inactive urinary sediment (absence of red blood cells, white blood cells or cellular or granular casts) after 24 weeks. (NCT00377637)
Timeframe: 24 weeks

,
Interventionparticipants (Number)
Complete Remission - YesComplete Remission - No
Intravenous Cyclophosphamide15170
Mycophenolate Mofetil16169

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Induction Phase: Number of Patients Showing Treatment Response

Treatment response was adjudicated by a blinded clinical endpoints committee (CEC) and defined as: a) Decrease in proteinuria, defined as a decrease in the urine protein to creatinine ratio (UPCr) to <3 in subjects with baseline proteinuria ≥3 UPCr or a decrease in the UPCr by ≥50% in subjects with proteinuria <3 UPCr at Baseline, and b) Stabilization of serum creatinine or improvement. UPCr were derived from the 24 hour urine collection. Patients who did not show a treatment response at Week 24 or who withdrew earlier than Week 24 were considered non-responders. (NCT00377637)
Timeframe: 24 weeks

,
Interventionparticipants (Number)
ResponderNon-responder
Intravenous Cyclophosphamide9887
Mycophenolate Mofetil10481

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Maintenance Phase: Events Contributing to the Primary Endpoint: Kaplan-Meier Estimates of Percentage of Participants Not Receiving Rescue Therapy

The primary efficacy parameter was the time to treatment failure, adjudicated by the Clinical Endpoints Committee (CEC), defined as any of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or requirement for rescue therapy to treat deterioration or exacerbation of Lupus nephritis. Kaplan-Meier survival curves were estimated from the observed time to rescue treatment for each patient. The data presented are the percentage of participants who were rescue treatment free at each time interval as estimated by Kaplan-Meier. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

,
InterventionPercentage of participants (Number)
Start of Maintenance Phase to Month 3Month 3 to Month 6Month 6 to Month 9Month 9 to Month 12Month 12 to Month 15Month 15 to Month 18Month 18 to Month 21Month 21 to Month 24Month 24 to Month 27Month 27 to Month 30Month 30 to Month 33Month 33 to Month 36
Azathioprine99.195.193.091.988.487.183.183.181.780.378.875.9
Mycophenolate Mofetil10098.297.294.294.294.293.191.990.890.890.890.8

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Maintenance Phase: Events Contributing to the Primary Endpoint: Kaplan-Meier Estimates of Percentage of Participants Renal Flare Free, by Time Interval

A proteinuric flare is defined as a doubling of the urine protein:creatinine ratio, and proteinuria ≥1 g/24 h in patients with urine protein ≤0.5 g/24 h at the end of the induction phase, or proteinuria ≥2 g/24 h if urine protein was >0.5 g/24 h at the end of the induction phase. A nephritic flare is defined as a 25% increase in serum creatinine accompanied by 1 or more of the following: (a) simultaneous doubling of the proteinuria reaching a minimum of 2 g/24 h (b) new/increased hematuria or (c) the appearance of cellular casts. All flares were adjudicated by a clinical endpoints committee. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

,
InterventionPercentage of participants (Number)
Start of Maintenance Phase to Month 3Month 3 to Month 6Month 6 to Month 9Month 9 to Month 12Month 12 to Month 15Month 15 to Month 18Month 18 to Month 21Month 21 to Month 24Month 24 to Month 27Month 27 to Month 30Month 30 to Month 33Month 33 to Month 36
Azathioprine97.290.387.285.082.879.278.075.574.274.272.970.1
Mycophenolate Mofetil98.294.690.887.887.886.886.886.886.886.885.685.6

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Maintenance Phase: Kaplan-Meier Estimates of Percentage of Participants Treatment Failure Free, by Time Interval

Treatment Failure was adjudicated by a clinical endpoints committee and was defined as the time to the earliest occurrence of any one of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or a requirement for rescue therapy for exacerbation or deterioration of Lupus nephritis. Kaplan-Meier survival curves were estimated from the observed time to treatment failure for each patient. The data presented are the percentage of participants who were treatment-failure free at each time interval as estimated by Kaplan-Meier. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36

,
InterventionPercentage of participants (Number)
Start of Maintenance Phase to Month 3Month 3 to Month 6Month 6 to Month 9Month 9 to Month 12Month 12 to Month 15Month 15 to Month 18Month 18 to Month 21Month 21 to Month 24Month 24 to Month 27Month 27 to Month 30Month 30 to Month 33Month 33 to Month 36
Azathioprine97.289.386.283.077.574.170.768.367.165.963.458.6
Mycophenolate Mofetil98.293.789.986.086.084.984.983.982.882.881.781.7

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Survival

The number of participants who survived treatment (NCT00282412)
Timeframe: up to 5 years

InterventionParticipants (Count of Participants)
Hematopoietic Stem Cell Transplantation2

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Percentage of Participants With BCAR at Month 12 Assessed by Local Review

"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 2005 Banff criteria. All biopsies (T-cell and/or antibody mediated) of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Day 365 was used for the analyses at 12 months. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 12 months

Interventionpercentage of participants (Number)
Tacrolimus/MMF/Basiliximab15.4
Alefacept QW/Tacrolimus/MMF29.0
Alefacept QW/Tacrolimus20.2
Alefacept QOW/Tacrolimus/MMF18.1

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Percentage of Participants With Biopsy-confirmed Acute Rejection (BCAR) at Month 6 Assessed by Local Review

"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 2005 Banff criteria. All biopsies (T-cell and/or antibody mediated) of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Day 182 was used for the analyses at 6 months. Lost to follow-up or patients with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Tacrolimus/MMF/Basiliximab12.7
Alefacept QW/Tacrolimus/MMF26.3
Alefacept QW/Tacrolimus18.8
Alefacept QOW/Tacrolimus/MMF16.7

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Time to First BCAR Assessed by Central Review

The time to first BCAR (central review) was calculated as the first biopsy date in which the central reviewer confirmed an acute rejection minus the date of skin closure +1. Only participants with a BCAR are included in the analysis. (NCT00543569)
Timeframe: 12 months

Interventiondays (Median)
Tacrolimus/MMF/Basiliximab19
Alefacept QW/Tacrolimus/MMF10
Alefacept QW/Tacrolimus12
Alefacept QOW/Tacrolimus/MMF11.5

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Time to First BCAR Assessed by Local Review

The time to first BCAR (local review) was calculated as the first biopsy date in which the local reviewer confirmed an acute rejection minus the date of skin closure +1. Only participants with a BCAR are included in the analysis. (NCT00543569)
Timeframe: 12 months

Interventiondays (Median)
Tacrolimus/MMF/Basiliximab9
Alefacept QW/Tacrolimus/MMF12
Alefacept QW/Tacrolimus19
Alefacept QOW/Tacrolimus/MMF12.5

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Time to First T-cell Mediated BCAR Assessed by Central Review

The time to first T-cell mediated BCAR (central review) was calculated as the first biopsy date in which the central reviewer confirmed an acute rejection minus the date of skin closure +1. Only participants with a T-cell mediated BCAR are included in the analysis. (NCT00543569)
Timeframe: 12 months

Interventiondays (Median)
Tacrolimus/MMF/Basiliximab19
Alefacept QW/Tacrolimus/MMF10
Alefacept QW/Tacrolimus12
Alefacept QOW/Tacrolimus/MMF11.5

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Time to First T-cell Mediated BCAR Assessed by Local Review

The time to first T-cell mediated BCAR (local review) was calculated as the first biopsy date in which the local reviewer confirmed an acute rejection minus the date of skin closure +1. (NCT00543569)
Timeframe: 12 months

Interventiondays (Median)
Tacrolimus/MMF/Basiliximab9
Alefacept QW/Tacrolimus/MMF12
Alefacept QW/Tacrolimus18
Alefacept QOW/Tacrolimus/MMF12.5

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Change From Week 4 in GFR by Iothalamate Clearance at Month 6

The glomerular filtration rate was measured directly using iothalamate clearance. (NCT00543569)
Timeframe: Week 4 and Month 6

,,,
InterventionmL/min per 1.73 m^2 (Mean)
Week 4Change at Month 6 (N=48, 45, 45, 47)
Alefacept QOW/Tacrolimus/MMF52.096.60
Alefacept QW/Tacrolimus44.363.56
Alefacept QW/Tacrolimus/MMF56.513.47
Tacrolimus/MMF/Basiliximab48.005.81

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Change From Week 4 in Glomerular Filtration Rate Estimated by the MDRD Method at Month 6 and Month 12

The glomerular filtration rate (GFR) was calculated using the Modification of Diet in Renal Disease (MDRD) method. (NCT00543569)
Timeframe: Week 4, Month 6 and Month 12

,,,
InterventionmL/min per 1.73 m^2 (Mean)
Week 4Change at Month 6 (N=65, 66, 67, 66)Change at Month 12 (n=66, 64, 64, 66)
Alefacept QOW/Tacrolimus/MMF58.02.52.7
Alefacept QW/Tacrolimus51.68.39.1
Alefacept QW/Tacrolimus/MMF59.33.23.3
Tacrolimus/MMF/Basiliximab54.75.78.9

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Change From Week 4 in Serum Creatinine at Month 6 and 12

(NCT00543569)
Timeframe: Week 4 and Month 6 and 12

,,,
Interventionmg/dL (Mean)
Week 4Change at Month 6 (N=69, 69, 68, 70)Change at Month 12 (N=67, 67, 65, 68)
Alefacept QOW/Tacrolimus/MMF1.5-0.00.1
Alefacept QW/Tacrolimus1.6-0.3-0.2
Alefacept QW/Tacrolimus/MMF1.6-0.2-0.2
Tacrolimus/MMF/Basiliximab1.5-0.0-0.1

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Gastrointestinal Quality of Life Index Score Over Time

The impact of gastrointestinal (GI) symptoms on health-related quality of life was assessed using the Gastrointestinal Quality of Life Index (GIQLI) symptom severity score. The GIQLI is a 36-item self-administered questionnaire that assesses the impact of gastrointestinal symptoms during the past 2 weeks on a scale from 0 (all of the time) to 4 (never). Possible overall scores ranged from 0 to 4, with higher scores indicating a better quality of life according to the different symptomatic criteria. (NCT00543569)
Timeframe: Months 1, 3, 6, and 12

,,,
Interventionunits on a scale (Mean)
Month 1 (N=63, 60, 59, 58)Month 3 (N=53, 52, 53, 54)Month 6 (N=58, 61, 57, 59)Month 12 (N=62, 60, 52, 58)
Alefacept QOW/Tacrolimus/MMF2.983.163.173.19
Alefacept QW/Tacrolimus3.003.113.243.26
Alefacept QW/Tacrolimus/MMF2.973.293.263.18
Tacrolimus/MMF/Basiliximab2.852.963.053.13

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Gastrointestinal Symptom Rating Scale Scores Over Time

The impact of gastrointestinal (GI) symptoms on health-related quality of life was assessed using the Gastrointestinal Symptom Rating Scale Scores (GSRS). The GSRS a 15-item self-administered questionnaire that assesses the impact of gastrointestinal symptoms during the past week on a scale from 1 (no discomfort at all) to 7 (very severe discomfort). Possible overall scores range from 1 to 7, with lower scores indicating a better quality of life with respect to gastrointestinal symptoms. (NCT00543569)
Timeframe: Months 1, 3, 6, and 12

,,,
Interventionunits on a scale (Mean)
Month 1 (N=70, 61, 61, 64)Month 3 (N=58, 55, 53, 56)Month 6 (N=63, 67, 62, 63)Month 12 (N=63, 65, 53, 65)
Alefacept QOW/Tacrolimus/MMF1.481.341.421.57
Alefacept QW/Tacrolimus1.591.471.491.52
Alefacept QW/Tacrolimus/MMF1.431.421.411.42
Tacrolimus/MMF/Basiliximab1.721.521.631.63

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Graft Survival at Month 6 and Month 12

"Graft survival was defined as any participant who was known to have a functioning graft (i.e., not graft loss) at 6 months and 12 months after the skin closure date. Graft loss was defined as patient death, retransplant, permanent return to dialysis (dialysis greater than 30 days) or transplant nephrectomy.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF92.385.8
Alefacept QW/Tacrolimus96.086.5
Alefacept QW/Tacrolimus/MMF93.590.9
Tacrolimus/MMF/Basiliximab96.287.3

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Maximum Grade of T-cell Mediated Rejection as Assessed by Central Review

"The grade of acute T-cell mediated rejection was classified as IA, IB, IIA, IIB and III according to Banff 2005 criteria. If a patient had more than 1 T-cell mediated rejection, the episode with the most severe grade was used in the analysis.~Grade IA: Cases with significant interstitial infiltration (> 25% of parenchyma affected) and foci of moderate tubulitis; Grade IB: Cases with significant interstitial infiltration (> 25% of parenchyma affected) and foci of severe tubulitis; Grade IIA: Cases with mild to moderate intimal arteritis; Grade IIB: Cases with severe intimal arteritis comprising >25% of the luminal area; Grade III: Cases with transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionparticipants (Number)
Month 6 - Grade IAMonth 6 - Grade IBMonth 6 - Grade IIAMonth 6 - Grade IIBMonth 6 - Grade IIIMonth 12 - Grade IAMonth 12 - Grade IBMonth 12 - Grade IIAMonth 12 - Grade IIBMonth 12 - Grade III
Alefacept QOW/Tacrolimus/MMF1154012540
Alefacept QW/Tacrolimus2133021330
Alefacept QW/Tacrolimus/MMF1362114621
Tacrolimus/MMF/Basiliximab1050010500

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Maximum Grade of T-cell Mediated Rejection Assessed by Local Review

"The grade of acute T-cell mediated rejection was classified as IA, IB, IIA, IIB and III according to Banff 2005 criteria. If a patient had more than 1 T-cell mediated rejection, the episode with the most severe grade was used in the analysis.~Grade IA: Cases with significant interstitial infiltration (> 25% of parenchyma affected) and foci of moderate tubulitis; Grade IB: Cases with significant interstitial infiltration (> 25% of parenchyma affected) and foci of severe tubulitis; Grade IIA: Cases with mild to moderate intimal arteritis; Grade IIB: Cases with severe intimal arteritis comprising >25% of the luminal area; Grade III: Cases with transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionparticipants (Number)
Month 6 - Grade IAMonth 6 - Grade IBMonth 6 - Grade IIAMonth 6 - Grade IIBMonth 6 - Grade IIIMonth 12 - Grade IAMonth 12 - Grade IBMonth 12 - Grade IIAMonth 12 - Grade IIBMonth 12 - Grade III
Alefacept QOW/Tacrolimus/MMF2434034340
Alefacept QW/Tacrolimus8330083300
Alefacept QW/Tacrolimus/MMF64900651000
Tacrolimus/MMF/Basiliximab4330053300

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Patient Survival at Month 6 and Month 12

"Patient survival is any participant who is known to be alive 6 months and 12 months after the skin closure date.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF93.689.7
Alefacept QW/Tacrolimus97.387.8
Alefacept QW/Tacrolimus/MMF94.892.2
Tacrolimus/MMF/Basiliximab96.287.3

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Percentage of Participants With Anti-lymphocyte-treated Rejection at Months 6 and 12

"Participants with histologically proved Banff Grade II or III rejection could receive anti-rejection therapy with anti-lymphocyte antibodies per institutional protocol.~The use of anti-lymphocyte antibody therapy at any time during a suspected or proven rejection episode for the treatment of acute rejection was considered an event." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF7.77.7
Alefacept QW/Tacrolimus12.012.0
Alefacept QW/Tacrolimus/MMF20.820.8
Tacrolimus/MMF/Basiliximab6.36.3

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Percentage of Participants With BCAR at Month 6 and 12 Assessed by Central Review

"Rejection episodes were confirmed by biopsy by a central reviewer. Biopsies were graded according to the 2005 Banff criteria. All biopsies (T-cell and/or antibody mediated) of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF14.215.5
Alefacept QW/Tacrolimus12.112.1
Alefacept QW/Tacrolimus/MMF18.319.7
Tacrolimus/MMF/Basiliximab7.77.7

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Percentage of Participants With Clinically Treated Acute Rejection at Month 6 and Month 12

Patients who received immunosuppressive medications for the treatment of suspected or BCAR were considered to have a clinically-treated acute rejection. (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF23.123.1
Alefacept QW/Tacrolimus29.330.7
Alefacept QW/Tacrolimus/MMF33.835.1
Tacrolimus/MMF/Basiliximab19.020.3

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Percentage of Participants With Efficacy Failure at 6 and 12 Months Assessed by Central Review

Efficacy failure is defined as death, graft failure (permanent return to dialysis [>30 days] or retransplant), BCAR according to central review, or lost to follow-up. (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF20.525.6
Alefacept QW/Tacrolimus16.021.3
Alefacept QW/Tacrolimus/MMF22.126.0
Tacrolimus/MMF/Basiliximab11.419.0

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Percentage of Participants With Efficacy Failure at 6 and 12 Months Assessed by Local Review

Efficacy failure is defined as death, graft failure (permanent return to dialysis [>30 days] or retransplant), BCAR according to local review, or lost to follow-up. (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF23.129.5
Alefacept QW/Tacrolimus22.729.3
Alefacept QW/Tacrolimus/MMF29.933.8
Tacrolimus/MMF/Basiliximab15.225.3

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Percentage of Participants With Multiple Rejection Episodes at Months 6 and 12

All participants were evaluated for the incidence of multiple rejection episodes (clinically treated and/or BCAR as assessed by the local reviewer) through 6 months and 12 months. (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF2.63.8
Alefacept QW/Tacrolimus4.04.0
Alefacept QW/Tacrolimus/MMF3.97.8
Tacrolimus/MMF/Basiliximab1.31.3

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Percentage of Participants With T-cell Mediated BCAR at Month 6 and 12 Assessed by Central Review

"Rejection episodes were confirmed by biopsy by the central reviewer. Biopsies were graded according to the 2005 Banff criteria. All biopsies of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF14.215.5
Alefacept QW/Tacrolimus12.112.1
Alefacept QW/Tacrolimus/MMF17.018.4
Tacrolimus/MMF/Basiliximab7.77.7

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Percentage of Participants With T-cell Mediated BCAR at Month 6 and 12 Assessed by Local Review

"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 2005 Banff criteria. All biopsies of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF16.718.1
Alefacept QW/Tacrolimus18.818.8
Alefacept QW/Tacrolimus/MMF25.027.7
Tacrolimus/MMF/Basiliximab12.714.0

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Percentage of Participants With Treatment Failure at Month 6 and 12

Treatment failure was defined as death, graft loss, BCAR (local review), lost to follow-up or early discontinuation of treatment regimen. The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit. (NCT00543569)
Timeframe: 6 months and 12 months

,,,
Interventionpercentage of participants (Number)
Month 6Month 12
Alefacept QOW/Tacrolimus/MMF29.534.7
Alefacept QW/Tacrolimus38.445.2
Alefacept QW/Tacrolimus/MMF37.745.5
Tacrolimus/MMF/Basiliximab26.635.5

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Achieving Full Donor Chimerism

Achieving full donor chimerism (donor T cells >95%): Blood was sent for donor cell percentage measured by short tandem repeat (STR) at post-transplant Day 30; Day 60; Day 90; Day 120; Day 180; Day 270; and Day 360. Full donor chimerism is defined as donor CD3+ cells > 95%. (NCT00481832)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
T & B Cell Mobilization Auto & Allo HCT10

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Event-free Survival (EFS)

"Event-free survival (EFS) as determined for participants who receive both planned transplants, for a minimum of 3 years.~Events are defined as disease progression/relapse and death of all causes." (NCT00481832)
Timeframe: 3 years

Interventionpercentage of participants (Number)
T & B Cell Mobilization Auto & Allo HCT35

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Incidence of Acute Graft Versus Host Disease (GvHD)

The development of GvHD in vaccinated patients of any grade and at 6 months. (NCT00481832)
Timeframe: 6 Months

InterventionParticipants (Count of Participants)
T & B Cell Mobilization Auto & Allo HCT3

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Incidence of Chemotherapy-associated Pneumonitis

Interstitial pneumonitis (IP) is a risk associated with high-dose carmustine (BCNU) or other chemotherapy drugs used for transplantation. IP is diagnosed by 1) a decrease of >25% in DLCO compared with pre-transplant PFT DLCO values or 2) a drop of 7% or more in oxygen saturation after exertion. (NCT00481832)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
T & B Cell Mobilization Auto & Allo HCT16

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Incidence of Chronic Graft Versus Host Disease (GvHD)

The development of GvHD in vaccinated patients of any grade at 6 months. (NCT00481832)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
T & B Cell Mobilization Auto & Allo HCT3

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Median Time to Neutrophile Engraftment

Complete blood counts were measured daily after allogeneic transplant. Time to neutrophil engraftment is defined as the number of days it takes to reach an absolute neutrophils count (ANC) >500, counting from the day of transplant. (NCT00481832)
Timeframe: up to 45 days

InterventionDays (Median)
T & B Cell Mobilization Auto & Allo HCT17

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Median Time to Platelet Engraftment

Complete blood counts were measured daily after allogeneic transplant. Time to platelet engraftment is defined as the number of days it takes to reach platelet count >20,000, counting from the day of transplant. (NCT00481832)
Timeframe: Up to 45 days

InterventionDays (Median)
T & B Cell Mobilization Auto & Allo HCT11

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Overall Mortality Rate

Overall mortality is determined by Kaplan-Meier estimation. The overall morality rate is expressed as the percentage of patients who died for any reason, including disease-related death. (NCT00481832)
Timeframe: 3 years

Interventionpercentage of participants (Number)
T & B Cell Mobilization Auto & Allo HCT56

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Overall Survival (OS)

Overall Survival (OS) 3 years after transplant, for participants who received both transplants, as determined by Kaplan-Meier estimation. (NCT00481832)
Timeframe: 3 years

Interventionpercentage of participants (Number)
T & B Cell Mobilization Auto & Allo HCT57

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Relapse Rate

Relapse rate (disease recurrence) 3 years after transplant, for participants who received both transplants, as determined by Kaplan-Meier estimation. (NCT00481832)
Timeframe: 3 years

Interventionpercentage of participants (Number)
T & B Cell Mobilization Auto & Allo HCT27

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Area Under the Curve (AUC) From Time Zero to 12 Hours Post-Dose [AUC (0-12)] of Mycophenolic Acid (MPA)

AUC (0-12) = Area under the plasma concentration versus time curve from time zero (pre-dose) to 12 hours post-dose, measured in microgram-hours per milliliter (mcg*h/mL) (NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionmcg*h/mL (Mean)
Myfortic - Fed State56.5
Myfortic - Fasting State63.9

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Area Under the Curve From Time Zero to 12 Hours Post-Dose [AUC (0-12)] of Mycophenolic Acid Glucuronide (MPAG)

AUC (0-12) = Area under the plasma concentration versus time curve from time zero (pre-dose) to 12 hours post-dose, measured in microgram-hours per milliliter (mcg*h/mL) (NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionmcg*h/mL (Mean)
Myfortic - Fed State967.5
Myfortic - Fasting State962.2

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Maximum Observed Plasma Concentration (Cmax) of Mycophenolic Acid (MPA)

(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionmicrograms per milliliter (Mean)
Myfortic - Fed State12.8
Myfortic - Fasting State18.7

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Maximum Observed Plasma Concentration (Cmax) of Mycophenolic Acid Glucuronide (MPAG)

(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionmicrograms per milliliter (Mean)
Myfortic - Fed State104.9
Myfortic - Fasting State113.0

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Minimum Observed Plasma Concentration (Cmin) of Mycophenolic Acid (MPA)

(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionmicrograms per milliliter (Mean)
Myfortic - Fed State2.2
Myfortic - Fasting State1.7

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Minimum Observed Plasma Concentration (Cmin) of Mycophenolic Acid Glucuronide (MPAG)

(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionmicrograms per milliliter (Mean)
Myfortic - Fed State59.4
Myfortic - Fasting State57.0

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Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mycophenolic Acid (MPA)

(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionhours (Median)
Myfortic - Fed State5
Myfortic - Fasting State3

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Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mycophenolic Acid Glucuronide (MPAG)

(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose

Interventionhours (Median)
Myfortic - Fed State3
Myfortic - Fasting State3

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GI Mucosal Lesions Change and Clinical Symptoms Using The Gastrointestinal Symptom Rating Scale (GSRS) Score

"The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~A higher GSRS indicate worse symptoms and a difference between D30 and last SBCE scores greater or equal to 0.3 can be considered as a clinically significant improvement in the symptoms." (NCT00652834)
Timeframe: one month

InterventionGSRS score (Mean)
baselineday 30
Kidney Transplant Recipients With GI Symptoms2.992.19

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Calculated Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula at Month 18

Calculated Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula at Month 18 cGFR (in mL/min/1.73 m2) = 186.3*(C-1.154)*(A-0.203)*G*R where C = the serum concentration of creatinine (mg/dL), A = age (years), G = 0.742 when gender is female, otherwise G = 1, R = 1.21 when race is black, otherwise R = 1. (NCT00862979)
Timeframe: Month 18

InterventionmL/min (Mean)
CNI-regimen54.2
CNI-free-regimen66.9

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Reciprocal Creatinine Slope Between Month 6 and Month 18

Reciprocal Creatinine Slope is an indication of renal function over time with a higher slope value indicating an improvement in renal function. (NCT00862979)
Timeframe: Between Month 6 and Month 18

Intervention1/(μmol/L)/(hour) (Mean)
CNI-regimen0.045
CNI-free-regimen0.403

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Calculated Glomerular Filtration Rate (cGFR) According to Cockcroft-Gault at Month 12 and 18

Calculated Glomerular Filtration Rate (cGFR) according to Cockcroft-Gault at Month 12 and 18. For men: GFR=(140-Age) x Body weight (kg) / 72 x Serum Creatinine (mg/dl) For women: GFR=0.85 (140 -Age) x Body weight(kg)/ 72 x Serum Creatinine (mg/dl) (NCT00862979)
Timeframe: Month 12 and 18

,
InterventionmL/min (Mean)
Month 12Month 18
CNI-free-regimen69.866.9
CNI-regimen54.254.2

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Occurrence of Major Cardiac Events (MACE) From Month 6 to 18

Major cardiac events (MACE) was defined as one of the following: any death, myocardial infarction, coronary artery bypass grafting (NCT00862979)
Timeframe: Month 6 to Month 18

,
InterventionOccurences (Number)
Myocardial infarctionDeath
CNI-free-regimen01
CNI-regimen10

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Occurrence of Treatment Failures From Month 6 to 9 and Month 9 to 18

Treatment failure was defined as composite endpoint of biopsy proven acute rejection of ISHLT 1990 grade ≥ 3A resp. ISHLT 2004 grade ≥ 2R, acute rejection episodes associated with hemodynamic compromise, graft loss / re-transplant, death, loss to follow up (at least one condition must be present). If participant had an occurrence in each period it was counted for each period. (NCT00862979)
Timeframe: Month 6 to Month 9; Month 9 to Month 18

,
InterventionOccurences (Number)
Month 6 to Month 9 Treatment failure - all reasonsMonth 9 to Month 18 Treatment failure-all reasons
CNI-free-regimen415
CNI-regimen13

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Serum Creatinine at Month 6, 8, 9, 10 12 and 18

Serum Creatinine is an indicator of renal function measured in the blood (NCT00862979)
Timeframe: Month 6, 8, 9, 10 12 and 18

,
Interventionμmol/L (Mean)
Month 6Month 8Month 9Month 10Month 12Month 18
CNI-free-regimen1.491.271.241.201.221.27
CNI-regimen1.531.441.581.531.531.50

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The Number of Subjects Who Experience a Change From Baseline in Their Panel of Reactive Antibody (PRA) Titers at 12 Months Post Rituximab Infusion.

(NCT00446251)
Timeframe: Month 12 from start of study

InterventionParticipants (Number)
Rituximab Infusion and Mycophenolate Mofetil Group6

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The Number of Subjects Who Experience a Decrease in Their Panel of Reactive Antibodies (PRA) at 6 Months Post Rituximab Infusion.

the number of subjects who experience a decrease in their Panel of Reactive Antibodies (PRA) at 6 months and 12 months post Rituximab infusion (NCT00446251)
Timeframe: Month 6 from start of study

InterventionParticipants (Number)
Rituximab Infusion and Mycophenolate Mofetil Group6

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The Number of Subjects With a Negative Crossmatch at the Time of Transplant.

(NCT00446251)
Timeframe: Month 12 from start of study

InterventionParticipants (Number)
Rituximab Infusion and Mycophenolate Mofetil Group0

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Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments

Assessed donor engraftment in very high risk pediatric patients. (NCT00795132)
Timeframe: 24 months

Interventionparticipants (Number)
Related BM PBSC16
Unrelated BM PBSC18
Unrelated Cord Blood10

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Two Year Overall Survival

The probability that a given patient will be alive two years after transplantation. (NCT00795132)
Timeframe: 24 months

Interventionprobability (Mean)
Related BM PBSC0.501
Unrelated BM PBSC0.395
Unrelated Cord Blood0.438

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Change From Baseline in Corrected Creatinine Clearance (mL/Min) at Week 52

Corrected creatinine clearance was calculated using the Cockcroft and Gault formula: For adult males, creatinine clearance in mL/min = [(140 - age in years) * (weight in kg] divided by [72 * serum creatinine in mg/dL]. For adult females, creatinine clearance in mL/min = 0.85 * [(140 - age in years) * (weight in kg)] divided by (72 * serum creatinine in mg/dL). (NCT00717314)
Timeframe: Week 52

InterventionmL/min (Mean)
MMF, 50% CNI6.551
MMF, 75% CNI6.442

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Percentage of Participants Experiencing Acute Rejection, Graft Loss, Death, or a Decrease From BL in Creatinine Clearance of ≥20% at Week 52

The percentage of participants who experienced at least 1 of the following: a ≥20% decrease from BL in creatinine clearance, acute rejection, graft loss, or death 1 year after randomization. (NCT00717314)
Timeframe: Week 52

Interventionpercentage of participants (Number)
MMF, 50% CNI8.1
MMF, 75% CNI8.8

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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) at Week 52

BPAR was graded according to Banff criteria. (NCT00717314)
Timeframe: Week 52

Interventionpercentage of participants (Number)
MMF, 50% CNI0.0
MMF, 75% CNI5.9

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Percentage of Participants With Decrease in Glomerular Filtration Rate (GFR) of Greater Than 20%

The percentage of participants with a greater than 20% decrease of GFR during the 1-year period following regimen adjustment. Cockcroft and Gault formula was used for calculated creatinine clearance. (NCT00717314)
Timeframe: Week 52

Interventionpercentage of participants (Number)
MMF, 50% CNI5.4
MMF, 75% CNI0.0

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Changes From Baseline in Creatinine Clearance (Milliliters Per Minute [mL/Min])

Creatinine clearance calculated using the Cockcroft and Gault formula: For adult males, creatinine clearance in mL/min equaled (=) [(140 minus (-) age in years) multiplied by (*) (weight in kilograms (kg)] divided by [72 * serum creatinine in milligrams per deciliter (mg/dL)]. For adult females, creatinine clearance in mL/min = 0.85 * [(140 - age in years) * (weight in kg)] divided by (72 * serum creatinine in mg/dL). (NCT00717314)
Timeframe: Baseline and Weeks 16, 28, and 40

,
InterventionmL/min (Mean)
Baseline (n=36,31)Change at Week 16 (n=37,33)Change at Week 28 (n=34,33)Change at Week 40 (n=35,27)
MMF, 50% CNI1.3854.8845.3336.131
MMF, 75% CNI2.2505.6294.5604.125

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Percentage Change in Creatinine Clearance From Baseline

Creatinine clearance was calculated using the Cockcroft and Gault formula. (NCT00717314)
Timeframe: Weeks 16, 28, 40, and 52

,
Interventionpercentage change from baseline (Mean)
Week 16 (n=37,33)Week 28 (n=34,33)Week 40 (n=35,27)Week 52 (n=37,34)
MMF, 50% CNI10.3811.4512.3710.17
MMF, 75% CNI8.727.517.0311.51

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Percentage of Participants With Graft Loss or Death at Week 52

Graft loss was defined for this protocol as re-transplantion or death. (NCT00717314)
Timeframe: Week 52

,
Interventionpercentage of participants (Number)
Graft lossDeath
MMF, 50% CNI0.00.0
MMF, 75% CNI0.00.0

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Event-free Survival (EFS) Per Protocol

Event-free survival (EFS) through 4 years, as assessed in participants with poor-risk recurrent or primary refractory DLBCL treated with TLI and ATG followed by matched allogeneic hematopoietic cell transplantation as a consolidation to HCT. Event is defined as tumor progression or death. (NCT00482053)
Timeframe: 48 months

Interventionmonths (Median)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL48

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Incidence of Chronic Graft vs Host Disease (GvHD)

The incidence of chronic graft vs host disease (GvHD) is reported as any events within 3 years. Note that GvHD was assessed per investigator judgement. There was no protocol-specified criteria of GvHD. (NCT00482053)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL0

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Median Time to Neutrophil Engraftment After Allogeneic Transplant

Reported as neutrophil engraftment after allogeneic transplant, defined as absolute neutrophil count (ANC) > 500/µL, counting from the day of transplant. (NCT00482053)
Timeframe: within 1 month

InterventionDays (Median)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL10.5

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Median Time to Neutrophil Engraftment After Autologous Transplant

Reported as neutrophil engraftment after autologous transplant, defined as absolute neutrophil count (ANC) > 500/µL, counting from the day of transplant. (NCT00482053)
Timeframe: within 1 month

InterventionDays (Median)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL11

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Median Time to Platelet Engraftment After Allogeneic Transplant

Reported as platelet engraftment after allogeneic transplant, defined as platelet count > 20,000/µL, counting from the day of transplant. (NCT00482053)
Timeframe: within 1 month

InterventionDays (Median)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL10

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Median Time to Platelet Engraftment After Autologous Transplant

Reported as platelet engraftment after autologous transplant, defined as platelet count > 20,000/µL, counting from the day of transplant. (NCT00482053)
Timeframe: within 1 month

InterventionDays (Median)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL19

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Overall Survival (OS)

To evaluate the overall and transplant related mortality rate, reported as the number of subjects remaining alive 3 years after transplant. (NCT00482053)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL2

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Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)

"Assessed as the incidence of grade 2 to 4 acute graft-vs-host disease (GvHD) at Day 100 post-transplant.~Stage of Acute GvHD was assessed as follows.~Stage 1: Skin: rash < 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea > 500 mL/day or persistent nausea with positive biopsy for GvHD~Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea >1000 mL/day.~Stage 3: Skin: rash > 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea > 1500 mL/day.~Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin > 15 mg/dL. Gut: severe abdominal pain with or without ileus~Grade of Acute GvHD was determined as follows.~Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage~Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut~Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut~Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage" (NCT01220297)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo0
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo1

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Acute GvHD (Grade 3 to 4)

"Assessed as the incidence of grade 3 to 4 acute GvHD at Day 100 post-transplant.~Stage of Acute GvHD was assessed as follows.~Stage 1: Skin: rash < 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea > 500 mL/day or persistent nausea with positive biopsy for GvHD~Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea >1000 mL/day.~Stage 3: Skin: rash > 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea > 1500 mL/day.~Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin > 15 mg/dL. Gut: severe abdominal pain with or without ileus~Grade of Acute GvHD was determined as follows.~Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage~Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut~Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut~Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage" (NCT01220297)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo0
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo1

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Disease-free Survival (DFS)

Assessed as survival without recurrence of disease (NCT01220297)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo0
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo0

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Overall Survival

Overall survival is defined as time from enrollment to time of death or last follow-up, within 2 years. (NCT01220297)
Timeframe: 2 years

InterventionDays (Median)
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo405

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Veno-occlusive Disease (VoD)

Assessed as the incidence of veno-occlusive disease (VoD) at 100 days post-transplant. (NCT01220297)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo0
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo1

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cGVHD

Chronic graft vs host disease (NCT01010217)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Haplo Arm11
1AgMM Related/Unrelated Donors7
MUD Donor0
Second Transplant0
Myelofibrosis0

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Disease Free Survival

Participants who have survived without their original disease. (NCT01010217)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Haplo Arm50
1AgMM Related/Unrelated Donors24
MUD Donor12
Second Transplant0
Myelofibrosis0

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Engraftments

(NCT01010217)
Timeframe: Day 28

InterventionParticipants (Count of Participants)
Haplo Arm80
1AgMM Related/Unrelated Donors50
MUD Donor21
Second Transplant0
Myelofibrosis0

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Grade III-IV aGVHD

Acute Graft vs host disease (NCT01010217)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Haplo Arm11
1AgMM Related/Unrelated Donors7
MUD Donor1
Second Transplant0
Myelofibrosis0

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Number of Participants With Non-relapse Mortality (NRM)

Non-relapse mortality (NRM) is defined as death from any cause other than relapse disease. Bayesian monitoring scheme described in Thall, Simon, and Estey (1996) employed to perform interim monitoring of the data during the course of the trial separately within each group. (NCT01010217)
Timeframe: At 100 days

InterventionParticipants (Count of Participants)
Haplo Arm8
1AgMM Related/Unrelated Donors7
MUD Donor2
Second Transplant0
Myelofibrosis0

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Blood Pressure

Diastolic pressure (mmHg) (NCT01002339)
Timeframe: 1 year

InterventionmmHg (Mean)
Tacrolimus With Rapid Steroid Withdrawal76.67
Tacrolimus With Steroids Minimization74.59
CsA With Steroid Minimization76.64

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Blood Pressure

Systolic pressure (mmHg) (NCT01002339)
Timeframe: 1 year

InterventionmmHg (Mean)
Tacrolimus With Rapid Steroid Withdrawal135.36
Tacrolimus With Steroids Minimization133.97
CsA With Steroid Minimization136.28

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Changes of Carotid Intima-media Thickness Over Time

absolute difference between carotid intima-media thickness at study end versus baseline. (NCT01002339)
Timeframe: 1 year

Interventionmm (Mean)
Tacrolimus With Rapid Steroid Withdrawal0.12
Tacrolimus With Steroids Minimization0.04
CsA With Steroid Minimization0.01

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Lipidic Profile (Cholesterol)

Lipidic Profile (total cholesterol) (NCT01002339)
Timeframe: 1 year

Interventionmg/dl (Mean)
Tacrolimus With Rapid Steroid Withdrawal169.05
Tacrolimus With Steroids Minimization178.24
CsA With Steroid Minimization168.89

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Lipidic Profile (HDL-c)

(NCT01002339)
Timeframe: 1 year

Interventionmg/dl (Mean)
Tacrolimus With Rapid Steroid Withdrawal44.84
Tacrolimus With Steroids Minimization49.29
CsA With Steroid Minimization48.35

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Lipidic Profile (LDL-c)

(NCT01002339)
Timeframe: 1 year

Interventionmg/dl (Mean)
Tacrolimus With Rapid Steroid Withdrawal94.00
Tacrolimus With Steroids Minimization95.43
CsA With Steroid Minimization88.65

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Lipidic Profile (Triglycerides)

(NCT01002339)
Timeframe: 1 year

Interventionmg/dl (Mean)
Tacrolimus With Rapid Steroid Withdrawal159.44
Tacrolimus With Steroids Minimization145.59
CsA With Steroid Minimization160.78

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Number of Antihypertensive Drugs Patients Reported Taking.

(NCT01002339)
Timeframe: 1 year

Interventionnumber of antihypertensive drugs (Median)
Tacrolimus With Rapid Steroid Withdrawal2
Tacrolimus With Steroids Minimization2
CsA With Steroid Minimization2

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Patients Treated With Insulin or Oral Antidiabetic Drugs

(NCT01002339)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Tacrolimus With Rapid Steroid Withdrawal20
Tacrolimus With Steroids Minimization15.4
CsA With Steroid Minimization2.6

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Percentage of Patients Using Acetylsalicylic Acid (ASA)

(NCT01002339)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Tacrolimus With Rapid Steroid Withdrawal53.9
Tacrolimus With Steroids Minimization48.7
CsA With Steroid Minimization52.8

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Percentage of Patients Using Statins

(NCT01002339)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Tacrolimus With Rapid Steroid Withdrawal56.
Tacrolimus With Steroids Minimization61.5
CsA With Steroid Minimization73.7

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Primary Outcome Measure (Glucose Intolerance)

Glycemia >=140 and <200 mg/dl, 2 hours after a standard oral glucose tolerance test. Measured values: glucose intolerance at 1 year defined by ADA criteria. (NCT01002339)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Tacrolimus With Rapid Steroid Withdrawal26.9
Tacrolimus With Steroids Minimization31.0
CsA With Steroid Minimization33.3

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Proteinuria

(NCT01002339)
Timeframe: 1 year

Interventionmg/day (Mean)
Tacrolimus With Rapid Steroid Withdrawal208
Tacrolimus With Steroids Minimization241
CsA With Steroid Minimization343.2

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Rejection

Biopsy proven acute rejection. Measured variable: Rate of Biopsy proven acute rejection. (NCT01002339)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Tacrolimus With Rapid Steroid Withdrawal11.4
Tacrolimus With Steroids Minimization4.8
CsA With Steroid Minimization21.4

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Renal Function

Estimated Glomerular Filtration Rate (ml/min/1.73 m^2) (NCT01002339)
Timeframe: 1 year

Interventionml/min/1.73 m^2 (Mean)
Tacrolimus With Rapid Steroid Withdrawal51.9
Tacrolimus With Steroids Minimization47.4
CsA With Steroid Minimization44.6

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"Primary Outcome Measure New Onset Diabetes After Renal Transplantation (NODAT)"

American Diabetes Association criteria (ADA) including an oral glucose tolerance test. (NCT01002339)
Timeframe: 1 year

,,
Interventionpercentage of participants (Number)
% of patients with NODAT% of patients without NODAT
CsA With Steroid Minimization7.992.1
Tacrolimus With Rapid Steroid Withdrawal34.165.9
Tacrolimus With Steroids Minimization23.176.9

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Fibrosis Score, as Measured by Thoracic High Resolution Computerized Tomography (HRCT)

Imaging of the whole lung (WL) is performed using a volumetric high resolution computerized tomography (HRCT) scan, which is then analyzed using a computer algorithm to determine the percentage of overall pixels exhibiting features characteristic for quantitative lung fibrosis (QLF). Higher percentages for QLF-WL therefore represent greater involvement by lung fibrosis. (NCT00883129)
Timeframe: Measured at baseline and Month 24

,
Intervention% of lung exhibiting QLF (Mean)
BaselineMonth 24
Cyclophosphamide Arm8.918.48
Mycophenolate Arm8.257.99

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Forced Vital Capacity (FVC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value

The primary outcome is the course over time from baseline to 24 months for the FVC %-predicted. The FVC %-predicted represents the adjusted volume of air (adjusted as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity) that can be forcibly exhaled from the lungs after taking the deepest breath possible. The FVC %-predicted is reduced in patients with interstitial lung disease and is used as a measure of lung involvement and disease severity. (NCT00883129)
Timeframe: Measured at study Baseline and Months 3, 6, 12, 15, 18, 21, and 24

,
InterventionFVC %-pred (Mean)
BaselineMonth 3Month 6Month 9Month 12Month 15Month 18Month 21Month 24
Cyclophosphamide Arm66.5267.0367.8669.4269.8671.9472.5772.5570.15
Mycophenolate Arm66.5266.2268.0268.1168.4369.8470.5770.8769.65

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Single-breath Diffusing Capacity for Carbon Monoxide (DLCO), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value

The DLCO is a pulmonary function test that measures the capacity for the lung to carry out gas exchange between the inhaled breath and the pulmonary capillary blood vessels and the DLCO %-predicted represents the DLCO expressed as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity. The DLCO %-predicted is reduced in patients with interstitial lung disease and is used as a measure of disease severity. (NCT00883129)
Timeframe: Measured at study entry and Months 3, 6, 12, 15, 18, 21, and 24

,
InterventionDLCO %-pred (Mean)
BaselineMonth 3Month 6Month 9Month 12Month 15Month 18Month 21Month 24
Cyclophosphamide Arm54.0551.9250.8751.5553.1253.6255.954.2652.90
Mycophenolate Arm53.9953.3854.8654.1355.3257.7756.6255.4755.31

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Skin Involvement, as Measured by the Modified Rodnam Skin Thickness Scores (mRSS)

Skin thickness is quantified using the modified Rodnan measurement method (mRSS), with a scale that ranges from 0 (no skin involvement) to a maximum of 51. The reported skin score is determined by a clinical assessment of skin thickness, which is performed by a trained reader, and represents the sum of individual assessments that are made in each of 17 body areas. Each area is given a score in the range of 0-3 (0 = normal; 1= mild thickness; 2 = moderate; 3 = severe thickness). A higher score represents more severe skin involvement. (NCT00883129)
Timeframe: Measured at baseline and Months 3, 6, 9, 12, 15, 18, 21, and 24

,
InterventionmRSS score (Mean)
BaselineMonth 3Month 6Month 9Month 12Month 15Month 18Month 21Month 24
Cyclophosphamide Arm14.0412.8511.9510.619.479.809.878.507.87
Mycophenolate Arm15.3216.0314.3714.3312.4512.4311.9811.2211.40

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Tolerability, as Assessed by the Time to Withdrawal From the Study Drug or Meeting Protocol-defined Criteria for Treatment Failure.

The number of participants who remained in the study at the listed time points are reported (NCT00883129)
Timeframe: Continuous assessment from randomization to 24 months

,
InterventionParticipants (Count of Participants)
BaselineMonth 3Month 6Month 9Month 12Month 15Month 18Month 21Month 24
Cyclophosphamide Arm736456514644423938
Mycophenolate Arm696658555252494949

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Total Lung Capacity (TLC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value

The TLC represents the total volume of air within the lung after taking the deepest breath possible and the TLC %-predicted represents the TLC expressed as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity. The TLC %-predicted is reduced in patients with interstitial lung disease and is used as a measure of disease severity. (NCT00883129)
Timeframe: Measured at study entry and Months 6, 12, 18, and 24

,
InterventionTLC %-pred (Mean)
BaselineMonth 6Month 12Month 18Month 24
Cyclophosphamide Arm65.4967.3968.2569.6366.97
Mycophenolate Arm66.1667.8467.3168.5068.24

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Toxicity, as Measured by Adverse Events, Serious Adverse Events, and Death

(NCT00883129)
Timeframe: Measured throughout the 2-year study

,
InterventionParticipants (Count of Participants)
Leukopenia (<2.5x10^3 WBC/microliter)Neutropenia (<1.0x10^3 neutrophils/microliter)Anemia (Hgb <10 g/dl)Thrombocytopenia (<100x10^3 platelets/microliter)Hematuria (>10 RBC/high power field)PneumoniaSAE-TotalSAE-related to treatmentDeaths
Cyclophosphamide Arm3071342422711
Mycophenolate Arm4380352735

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Transitional Dyspnea Index Score

Change in breathlessness was assessed using the Transitional Dyspnea Index, which compares current symptoms to those at baseline. Total score ranges from - 9 to + 9. The lower the score, the more deterioration in severity of dyspnea. (NCT00883129)
Timeframe: Measured at Months 6, 12, 18, and 24

,
InterventionTransitional Dyspnea Index Score (Mean)
Month 6Month 12Month 18Month 24
Cyclophosphamide Arm0.311.231.782.09
Mycophenolate Arm0.741.170.911.86

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Number of Participants With Cytomegalovirus Infection or Disease

(NCT00336895)
Timeframe: 12 weeks

Interventionparticipants (Number)
Liver Transplant Subjects0

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Gastrointestinal Side Effects and Quality of Life (-Subscales of GSRS)

"The GSRS contains 15 items, each rated on a seven- point likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items breakdown into the following five scales: abdominal ( Abdominal pain, hunger pains and nausea): reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome ( borborygmus, abdominal distention, eructation and increased flatus) and constipation syndrome (constipation, hard stools and feeling of incomplete evacuation) The range of the scale for abdominal pain was 3 to 21, reflux 2 to 14, diarrhea 3 to 21, indigestion 4 to 28 and constipation 3 to 21.~Higher values represent more severe discomfort." (NCT00336895)
Timeframe: 12 weeks

Interventionunits on a scale (Mean)
Abdominal PainRefluxIndigestionDiarrheaConstipation
Liver Transplant Subjects10.782.647.085.444.56

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Gastrointestinal Side Effects and Quality of Life (Total Score of GSRS)

"The gastrointestinal Symptom Rating Scale (GSRS) is a validated scale, the items range from 1= No discomfort at all to 7= Very severe discomfort.~The scale ranges from a minimal value of 15 ( No discomfort at all) to a maximum of 105 ( Very severe discomfort)~The GSRS contains 15 items, each rated on a seven- point likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items breakdown into the following five scales: abdominal ( Abdominal pain, hunger pains and nausea): reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome ( borborygmus, abdominal distention, eructation and increased flatus) and constipation syndrome (constipation, hard stools and feeling of incomplete evacuation)" (NCT00336895)
Timeframe: screening, 2, 6 and 12 weeks

Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
Liver Transplant Subjects46.00029.040026.280025.3600

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Comparison by Treatment Assignment in the Number of Biopsy-Proven Acute Rejections Within 12 Months Post Kidney Transplantation

"Biopsy-proven acute renal (kidney) rejection [1, 2].~Diagnosis of acute rejection was made by renal biopsy using the Banff 97 criteria. The Banff 97 diagnostic category for renal allograft biopsies is an international standardized histopathological classification. Acute rejection is defined by a renal biopsy demonstrating a Banff 97 classification of Grade IA or greater, with higher scores indicating more severe rejection[2]~Ref: Racusen LC et al. The Banff 97 working classification of renal allograft pathology. Kidney Int, 55: 713-723, 1999" (NCT00141037)
Timeframe: Up to one year post kidney transplantation procedure

InterventionRejection Events (Number)
Steroid-Free Immunosuppression18
Steroid-Based Immunosuppression19

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The Difference in Linear Growth by Treatment Assignment at 1 Year Post Kidney Transplantation

Standardized Z-scores were computed following a formula using an age- and gender-specific calculation provided by the NHANES III 2000 Growth Data set. The Z-score system expresses anthropometric values of height as several standard deviations (SDs) below (e.g., a negative value) or above (a positive value) the reference mean or median value. In this study the measure was used to test whether there is a difference in the change in height between the treatment groups: Steroid-Based versus Steroid-Free (NCT00141037)
Timeframe: One year post kidney transplantation procedure

InterventionStandard Deviation Score (SDS) (Mean)
Steroid-Free Immunosuppression0.37
Steroid-Based Immunosuppression0.35

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Number of Participants Who Experienced Adverse Events and Death

Participants were monitored for adverse events, serious adverse events and deaths thorughout the prospective and follow-up phases of the study. (NCT00332839)
Timeframe: 12 months

,
InterventionParticipants (Number)
Adverse events (serious and non-serious)Serious adverse eventsDeaths
Calcineurin Inhibitor (CNI) Group44111
Certican Group44121

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Disease Free

Number of participants that were disease free (NCT01093586)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I4

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Disease Free

Number of participants that were disease free (NCT01093586)
Timeframe: At 2 years

InterventionParticipants (Count of Participants)
Arm I4

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Hematologic Engraftment

Number of participants that were able to complete engraftment by day 42. (NCT01093586)
Timeframe: On day +42

InterventionParticipants (Count of Participants)
Arm I10

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Incidence of Acute Graft-versus-host Disease (GVHD)

Number of participants that had acute GVHD (NCT01093586)
Timeframe: At 100 days

InterventionParticipants (Count of Participants)
Arm I11

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Incidence of Chronic GVHD

Number of participants that have chronic GVHD. Chronic GVHD will be diagnosed and graded on clinical and histological criteria from the Center for International Blood and Marrow Transplant Research (CIBMTR) (NCT01093586)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I1

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Occurrence of Serious Infections

Number of participants that had infections (NCT01093586)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Arm I13

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Overall Survival

Number of participants alive at 180 days post engraftment. (NCT01093586)
Timeframe: On day +180

InterventionParticipants (Count of Participants)
Arm I8

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Overall Survival

Number of participants that were alive. (NCT01093586)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I6

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Overall Survival

Number of participants that were alive. (NCT01093586)
Timeframe: At 2 years

InterventionParticipants (Count of Participants)
Arm I4

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Recurrence or Relapse

Number of subjects that had disease recurrence (NCT01093586)
Timeframe: one year in patients post UCBT

InterventionParticipants (Count of Participants)
Arm I2

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Recurrence or Relapse

Number of subjects that had disease recurrence (NCT01093586)
Timeframe: two years post transplant

InterventionParticipants (Count of Participants)
Arm I2

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Complete Response Rate (CRR)

"Complete response rate (CRR) was assessed as all of:~Negative immunoflixation on the serum and urine~Disappearance of any soft tissue plasmacytomas~< 5% plasma cells in bone marrow" (NCT00899847)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant3

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Event-free Survival (EFS)

To evaluate the graft versus myeloma effect by monitoring rate of event-free survival (EFS) (NCT00899847)
Timeframe: 2 years after the last participant is enrolled

Interventionpercentage of participants (Number)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant44

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Incidence of Graft Versus Host Disease (GvHD)

To evaluate the incidence acute GvHD of this tandem autologous/allogeneic transplant setting (NCT00899847)
Timeframe: 2 years after the last participant is enrolled.

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant1

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Overall Response Rate (ORR)

Overall response rate (ORR) = Complete Response Rate (CRR) + Partial Response Rate (PRR) (NCT00899847)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Peripheral Blood Stem Cell Transplant7

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Overall Survival (OS)

To evaluate the graft versus myeloma effect by monitoring rate of overall survival (OS) (NCT00899847)
Timeframe: 2 years after the last participant is enrolled

Interventionpercentage of participants (Number)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant67

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Partial Response Rate (PRR)

"Partial response rate (PRR) was assessed as~> 50% reduction in serum M-protein plus urine M-protein reduction by 90% or < 200 mg/24 hr~If serum M-protein is not measurable, then > 50% reduction in the involved serum free light chain~If involved serum free light chain is not measurable, then > 50% reduction in the bone marrow plasma cell percentage + > 50% reduction in the size of any soft tissue plasmacytoma." (NCT00899847)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Peripheral Blood Stem Cell Transplant4

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Median Time to Engraftment After Allo-PBSC Transplant

"Engraftment is assessed as:~Neutrophil engraftment is > 0.5 x 10⁹/L after cytopenia~Platelet engraftment is > 20 x 10⁹/L after cytopenia" (NCT00899847)
Timeframe: 1 month

InterventionDays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Autologous-Allogeneic Hematopoietic Stem Cell Transplant2410

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Median Time to Engraftment After Auto-PBSC Transplant

"Engraftment is assessed as:~Neutrophil engraftment is > 0.5 x 10⁹/L after cytopenia~Platelet engraftment is > 20 x 10⁹/L after cytopenia" (NCT00899847)
Timeframe: 1 month

InterventionDays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Autologous-Allogeneic Hematopoietic Stem Cell Transplant1115

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Number of Patient Who Gained Remission From the Nephrotic Syndrome

Efficacy of mycophenolate in preventing relapse of nephrotic syndrome secondary to membranous glomerulonephritis on withdrawal of tacrolimus therapy. (NCT00843856)
Timeframe: 10-109 weeks

InterventionParticipants (Count of Participants)
Tacrolimus8
Tacrolimus and Mycophenolate Mofetil8

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Number of Patients Achieved Remission

The degree of remission of proteinuria obtained (complete or partial) The rate of decline of renal function measured by the Modification of Diet in Renal Disease equation for glomerular filtration rate. (NCT00843856)
Timeframe: 6-12 months

InterventionParticipants (Count of Participants)
Tacrolimus16
Tacrolimus and Mycophenolate Mofetil19

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Core Study: Number Participants With Combined Graft Loss, Death or Loss to Follow-up

"The allograft was presumed to be lost on the day the patient starts dialysis and was not able to stop dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was considered as the day of graft loss.~A loss to follow-up in graft loss, death or loss to follow-up is a patient who did not experience graft loss or death and whose last day of contact was prior to Day 316, i.e. prior to the Month 12 visit window." (NCT00658320)
Timeframe: 12 months

InterventionParticipants (Number)
Everolimus + Reduced Dose of Cyclosporine5
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine3

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Core Study: Renal Function Measured by Calculated Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula

"Modification of Diet in Renal Disease (MDRD) formula is:~Calculated GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where~C is the serum concentration of creatinine [mg/dL],~A is patient age at sample collection date [years],~G=0.742 when gender is female, otherwise G=1,~R=1.21 when race is black, otherwise R=1" (NCT00658320)
Timeframe: Month 12

InterventionmL/min/1.73m^2 (Median)
Everolimus + Reduced Dose of Cyclosporine58.00
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine55.25

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Extension Study: Renal Function Measured by Calculated Glomerular Filtration Rate (cGFR) Using the Modification of Diet in Renal Disease (MDRD) Formula

"Renal function was assessed by glomerular filtration rate (GFR) using the MDRD formula:~GFR [mL/min/1.73m2] = 186.3*(C-1.154)*(A-0.203)*G*R C is the serum concentration of creatinine [mg/dL] A is age [years] G = 0.742 when gender is female, otherwise G=1 R = 1.21 when race is black, otherwise R=1" (NCT00658320)
Timeframe: Month 48

InterventionmL/min/1.73m^2 (Median)
Everolimus + Reduced Dose of Cyclosporine59.80

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Extension Study: Renal Function Measured by Calculated Glomerular Filtration Rate (cGFR) Using the Modification of Diet in Renal Disease (MDRD) Formula

"Renal function was assessed by glomerular filtration rate (GFR) using the MDRD formula:~GFR [mL/min/1.73m2] = 186.3*(C-1.154)*(A-0.203)*G*R C is the serum concentration of creatinine [mg/dL] A is age [years] G = 0.742 when gender is female, otherwise G=1 R = 1.21 when race is black, otherwise R=1~Loss to follow up (in In Primary Core Outcome Measure) is composite efficacy failure and contains incidence of treated BPAR, graft loss, death or loss to follow-up" (NCT00658320)
Timeframe: Month 24

InterventionmL/min/1.73m^2 (Median)
Everolimus + Reduced Dose of Cyclosporine58.90
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine54.95

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Core Study: Number of Patients With Composite Efficacy Endpoint

"The composite efficacy endpoint consisted of treated biopsy proven acute rejection (BPAR) episodes, graft loss, death or loss to follow-up. A treated BPAR was defined as a biopsy graded IA, IB, IIA, IIB, or III and which was treated with anti-rejection therapy. The allograft was presumed to be lost on the day the patient starts dialysis and was not able to stop dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was considered as the day of graft loss.~For the individual components (including loss of follow-up)of the composite endpoint, patients are counted for the first event to occur." (NCT00658320)
Timeframe: 12 months

,
InterventionParticipants (Number)
Composite Efficacy EndpointTreated BPARGraft LossDeathLoss to follow up (see caveats)
Everolimus + Reduced Dose of Cyclosporine73004
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine75002

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Extension Study: Cyclosporine Trough Levels

Blood was collected at all visits after Day 3 for trough (collected 5 minutes before study drug dose) cyclosporine levels and was analyzed at a central laboratory using immunoassay. (NCT00658320)
Timeframe: Month 24, Month 48

,
Interventionng/mL (Mean)
Month 24Month 48 (n=7,0)
Everolimus + Reduced Dose of Cyclosporine54.134.2
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine105.5NA

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Extension Study: Everolimus Trough Levels

Blood was collected at all visits after Day 3 for trough (collected 5 minutes before study drug dose) everolimus levels and was analyzed at a central laboratory using liquid chromatography mass spectrometry. (NCT00658320)
Timeframe: Month 24, Month 48

Interventionng/mL (Mean)
Month 24Month 48 (n=8)
Everolimus + Reduced Dose of Cyclosporine5.2584.408

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Extension Study: Number of Participants With Adverse Events and Serious Adverse Events

Additional information about Adverse Events can be found in the Adverse Event Section. (NCT00658320)
Timeframe: 24 Months

,
InterventionParticipants (Number)
Adverse EventsSerious Adverse Events
Everolimus + Reduced Dose of Cyclosporine5030
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine5030

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Extension Study: Number of Participants With Combined Efficacy Endpoint: Graft Loss, Death, Loss to Follow-up and/or Treated Biopsy Proven Acute Rejection (BPAR)

"Graft loss was defined as the day the patient started dialysis and was not able to subsequently be removed from dialysis or re-transplant.~Loss-to-follow was a patient who did not experience a treated BPAR, graft loss or death and whose last day of contact was prior to Month 24.~A Graft Biopsy was done within 48 hours of suspect rejection. Biopsies were read by the local pathologist according to the updated Banff '97 criteria.~Treated BPAR was based on local laboratory biopsy results and was defined as a biopsy Banff criteria graded IA to III that was treated with anti-rejection therapy." (NCT00658320)
Timeframe: 24 Months

,
InterventionParticipants (Number)
Combined Efficacy EndpointTreated BPARGraft LossDeathLoss to follow-up
Everolimus + Reduced Dose of Cyclosporine43001
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine55000

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Extension Study: Renal Function Measured by Calculated Glomerular Filtration Rate (GFR) Using the Nankivell Formula

"The Nankivell formula was used to calculate GFR at Month 24:~GFR[mL/min]=6.7/C + W/4 - UREA/2 - 100/H^2 + 35 (25 for females) W= body weight [kg] H= height [m] C= serum creatinine [mmol/L] UREA= serum urea [mmolL]" (NCT00658320)
Timeframe: Month 24, Month 48

,
InterventionmL/min (Mean)
Month 24Month 48 (9,0)
Everolimus + Reduced Dose of Cyclosporine63.4960.16
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine59.24NA

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Annual Change Rate in Total Plaque Volume (TPV) From Pre-conversion Baseline to 12 Months Post-transplant

Within-subject annual change rate in TPV in the left and right distal common carotid arteries from the pre-conversion baseline to 12 months post kidney transplant as determined by ultrasound. Annual change rate equals (=) (TPV at month 12 post-transplant minus [-] TPV at pre-conversion baseline) divided (/) by imaging interval in years. TPV is the sum of assessment in left and right distal common carotid arteries. (NCT00311311)
Timeframe: Pre-conversion baseline and 12 months post-transplant

Interventionmillimeter cube/year (mmˆ3/year) (Mean)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone-49.31
Tacrolimus With Mycophenolate/Prednisone0.66

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CIMT at Pre-conversion Baseline

Mean CIMT=average of left CIMT and right CIMT. (NCT00311311)
Timeframe: Pre-conversion baseline

Interventionmm (Mean)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.735
Tacrolimus With Mycophenolate/Prednisone0.773

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TPV at Pre-conversion Baseline

TPV is the sum of the assessment in left and right distal common carotid arteries. (NCT00311311)
Timeframe: Pre-conversion baseline

Interventionmmˆ3 (Mean)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone68.68
Tacrolimus With Mycophenolate/Prednisone48.57

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Annual Change Rate in Carotid Intima Media Thickness (CIMT) From Pre-conversion Baseline at 12, 18, 24 and 36 Months Post-transplant

Within-subject annual change rate in CIMT as determined by ultrasound. Mean CIMT=average of left CIMT and right CIMT. Annual CIMT Change Rate (mm/year) = (CIMT at Month x Post-transplant Visit - CIMT at Conversion Baseline) / Imaging interval in years. (NCT00311311)
Timeframe: Pre-conversion baseline, and 12, 18, 24 and 36 months post-transplant

,
Interventionmillimeter/year (mm/year) (Mean)
Annual Change Rate at 12 months (n=16,28)Annual Change Rate at 18 months (n=15,26)Annual Change Rate at 24 months (n=15,23)Annual Change Rate at 36 months (n=8,13)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.0480.0410.0230.028
Tacrolimus With Mycophenolate/Prednisone0.012-0.00020.015-0.007

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Annual Rate of Change in TPV From Pre-conversion Baseline to 18, 24 and 36 Months Post Transplant

Within-subject annual change rate in TPV in the left and right distal common carotid arteries from the pre-conversion baseline to 18, 24 and 36 months post kidney transplant as determined by ultrasound. Annual change rate equals (=) (TPV at month 18, 24 and 36 post-transplant minus [-] TPV at pre-conversion baseline) divided (/) by imaging interval in years. TPV is the sum of assessment in left and right distal common carotid arteries. (NCT00311311)
Timeframe: Pre-conversion baseline, and 18, 24 and 36 months post-transplant

,
Interventionmmˆ3/year (Mean)
Month 18 post-transplant (n=12,12)Month 24 post-transplant (n=11,14)Month 36 post-transplant (n=7,11)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone17.1525.286.62
Tacrolimus With Mycophenolate/Prednisone19.719.108.98

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Change From Pre-conversion Baseline in Adiponectin at Months 12, 24 and 36 Post-transplant

Adiponectin is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates less risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionmicrogram per milliliter (µg/mL) (Mean)
Change at 12 Months post-transplant (n=23,33)Change at 24 Months post-transplant (n=18,31)Change at 36 Months post-transplant (n=15,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone5.3522.2441.487
Tacrolimus With Mycophenolate/Prednisone-2.205-0.267-1.413

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Change From Pre-conversion Baseline in Endothelin-1 at Months 12, 24 and 36 Post-transplant

Endothelin-1 is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionpg/mL (Mean)
Change at 12 Months post-transplant (n=22,30)Change at 24 Months post-transplant (n=18,30)Change at 36 Months post-transplant (n=14,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.2940.4260.411
Tacrolimus With Mycophenolate/Prednisone0.0280.138-0.104

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Change From Pre-conversion Baseline in Fasting Lipid Parameters at 12, 18, 24 and 36 Months Post-transplant

Total Cholesterol (TC), Low Density Lipoprotein (LDL), High Density Lipoprotein (HDL) and Triglyceride (Tg) blood concentrations. Higher levels of TC, LDL and Tg are less desirable. Lower levels of HDL are less desirable. Change for each parameter = value at 12, 18, 24 and 36 months post-transplant - value at pre-conversion baseline. (NCT00311311)
Timeframe: Pre-conversion baseline, and 12, 18, 24 and 36 months post-transplant

,
Interventionmillimole/liter (mmol/L) (Mean)
Change in TC 12 Months post-transplant (n=23,30)Change in LDL 12 Months post-transplant (n=21,30)Change in HDL 12 Months post-transplant (n=23,30)Change in Tg 12 Months post-transplant (n=23,30)Change in TC 18 Months post-transplant (n=21,29)Change in LDL 18 Months post-transplant (n=20,29)Change in HDL 18 Months post-transplant (n=21,29)Change in Tg 18 Months post-transplant (n=21,29)Change in TC 24 Months post-transplant (n=19,27)Change in LDL 24 Months post-transplant (n=16,27)Change in HDL 24 Months post-transplant (n=19,27)Change in Tg 24 Months post-transplant (n=19,27)Change in TC 36 Months post-transplant (n=15,21)Change in LDL 36 Months post-transplant (n=13,21)Change in HDL 36 Months post-transplant (n=15,21)Change in Tg 36 Months post-transplant (n=15,21)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.5100.2770.0590.6170.3610.2220.1060.3590.2400.0670.0670.5470.3860.0270.0980.527
Tacrolimus With Mycophenolate/Prednisone-0.164-0.056-0.053-0.1170.0820.138-0.030-0.057-0.0080.054-0.019-0.088-0.323-0.2570.111-0.385

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Change From Pre-conversion Baseline in Fibrinogen at Months 12, 24 and 36 Post-transplant

Fibrinogen is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventiongram per liter (g/L) (Mean)
Change at 12 Months post-transplant (n=22,31)Change at 24 Months post-transplant (n=19,29)Change at 36 Months post-transplant (n=14,20)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.620.450.40
Tacrolimus With Mycophenolate/Prednisone0.200.330.21

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Change From Pre-conversion Baseline in Glucose at Months 12, 24 and 36 Post-transplant

Fasting plasma glucose. Change = value at month x post-transplant - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionmmol/L (Mean)
Change at 12 Months post-transplant (n=23,33)Change at 24 Months post-transplant (n=19,31)Change at 36 Months post-transplant (n=14,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.6060.8320.686
Tacrolimus With Mycophenolate/Prednisone-0.083-0.076-0.214

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Change From Pre-conversion Baseline in Glycosylated Hemoglobin(HbA1C) at Months 12, 24, and 36 Post-transplant

HbA1C, change = value at month x post-transplant - pre-conversion baseline. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionpercentage of glucose (Mean)
Change at 12 Months post-transplant (n=23,26)Change at 24 Months post-transplant (n=17,25)Change at 36 Months post-transplant (n=14,16)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.0080.0090.012
Tacrolimus With Mycophenolate/Prednisone0.0010.006-0.001

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Change From Pre-conversion Baseline in High Sensitivity C-Reactive Protein (hsCRP) at Months 12, 24 and 36 Post-transplant.

hsCRP is a biomarker of cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionmg/L (Mean)
Change at 12 Months post-transplant (n=23,33)Change at 24 Months post-transplant (n=18,31)Change at 36 Months post-transplant (n=15,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone1.611-0.5630.336
Tacrolimus With Mycophenolate/Prednisone1.3791.7233.164

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Change From Pre-conversion Baseline in Homocysteine at Months 12, 24 and 36 Post-transplant

Homocysteine is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionmicromole/liter (µmol/L) (Mean)
Change at 12 Months post-transplant (n=23,33)Change at 24 Months post-transplant (n=19,31)Change at 36 Months post-transplant (n=15,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone1.2781.3161.087
Tacrolimus With Mycophenolate/Prednisone0.5302.1582.459

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Change From Pre-conversion Baseline in Insulin at Months 12, 24, and 36 Post-transplant

Fasting insulin. Change = value at month x post-transplant - pre-conversion baseline. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionpicomole/liter (pmol/L) (Mean)
Change at 12 Months post-transplant (n=16,23)Change at 24 Months post-transplant (n=13,23)Change at 36 Months post-transplant (n=10,14)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone-4.549-6.120-22.759
Tacrolimus With Mycophenolate/Prednisone17.95332.180-2.731

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Change From Pre-conversion Baseline in Interleukin-6 (IL-6) at Months 12, 24 and 36 Post-transplant

IL-6 is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionpg/mL (Mean)
Change at 12 Months post-transplant (n=23,32)Change at 24 Months post-transplant (n=18,29)Change at 36 Months post-transplant (n=15,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone-0.2390.128-0.800
Tacrolimus With Mycophenolate/Prednisone0.4630.7830.164

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Change From Pre-conversion Baseline in Lipoprotein(a) at Months 12, 24 and 36 Post-transplant

Lipoprotein(a) is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionmilligram per deciliter (mg/dL) (Mean)
Change at 12 Months post-transplant (n=23,30)Change at 24 Months post-transplant (n=18,26)Change at 36 Months post-transplant (n=13,17)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone13.49612.51711.885
Tacrolimus With Mycophenolate/Prednisone-11.6806.0736.547

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Change From Pre-conversion Baseline in Tumor Necrosis Factor Alpha (TNF-alpha) at Months 12, 24 and 36 Post-transplant

TNF-alpha is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionpg/mL (Mean)
Change at 12 Months post-transplant (n=23,32)Change at 24 Months post-transplant (n=18,30)Change at 36 Months post-transplant (n=15,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone0.991-0.367-0.441
Tacrolimus With Mycophenolate/Prednisone0.0001.8940.000

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Change From Pre-conversion Baseline in Uric Acid at Months 12, 24 and 36 Post-transplant

Uric Acid is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionµmol/L (Mean)
Change at 12 Months post-transplant (n=23,33)Change at 24 Months post-transplant (n=19,31)Change at 36 Months post-transplant (n=15,22)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone-51.53-29.02-19.03
Tacrolimus With Mycophenolate/Prednisone11.103.89-0.58

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Change From Pre-conversion Baseline in Vitamin B12 at Months 12, 24 and 36 Post-transplant

Vitamin B12 is a biomarker for cardiovascular disease and atherosclerosis risk. A lower level indicates a greater risk. Change = month x post-transplant values - pre-conversion values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant

,
Interventionpmol/L (Mean)
Change at 12 Months post-transplant (n=21,33)Change at 24 Months post-transplant (n=19,29)Change at 36 Months post-transplant (n=14,21)
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone-10.21-36.23-40.00
Tacrolimus With Mycophenolate/Prednisone-11.38-27.46-46.39

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Number of Participants Who Used Anti-hypertensive Medications

"Participants who reported yes for taking anti-hypertensive medications as concomitant medication." (NCT00311311)
Timeframe: From consent to conversion, from conversion to Month 12, from Months 12 to 24, and from Months 24 to 36 post-transplant

,
Interventionparticipants (Number)
From consent to conversionFrom conversion to Month 12From Months 12 to Month 24From Months 24 to Month 36
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone19212118
Tacrolimus With Mycophenolate/Prednisone30303028

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Number of Participants Who Used Lipid Lowering Therapies

"Participants who reported yes for taking lipid lowering therapies as concomitant medication." (NCT00311311)
Timeframe: From consent to conversion, from conversion to Month 12, from Months 12 to 24, and from Months 24 to 36 post-transplant

,
Interventionparticipants (Number)
From consent to conversionFrom conversion to Month 12From Months 12 to Month 24From Months 24 to Month 36
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone13222318
Tacrolimus With Mycophenolate/Prednisone24292827

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GFR Measured by Iothalamate Clearance at Month 6

GFR measured using the iothalamate clearance method and determined by a central laboratory. (NCT00617604)
Timeframe: Month 6

InterventionmL/minute (Mean)
Placebo59.6029
Alefacept55.1613

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Graft Survival

"Graft survival was defined as any participant who was known to have a functioning graft (i.e., not graft loss) at 6 months. Graft loss is defined as re-transplantation, nephrectomy, death or as dialysis ongoing at end of study or at discontinuation of the participant unless superseded by follow-up information.~The Kaplan-Meier estimate of graft survival within the first 6 months following transplantation is reported. Participants lost to follow-up were censored at the time of last assessment." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo90.6
Alefacept95.2

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Patient Survival

Patient survival is any participant known to be alive at Month 6. The Kaplan-Meier estimate of patient survival within the first 6 months following transplantation is reported. Participants lost to follow-up were censored at the time of last assessment. (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo97.1
Alefacept99.0

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Percentage of Participants With Acute Rejection Diagnosed by Signs and Symptoms at Month 6

Acute rejection diagnosed by signs and symptoms, including biopsy-confirmed or suspected (not confirmed by biopsy - i.e. no biopsy was performed or biopsy did not confirm an acute T-cell mediated rejection). The Kaplan-Meier estimate of acute rejection diagnosed by signs and symptoms within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit. (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo27.4
Alefacept22.3

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Percentage of Participants With Anti-Lymphocyte Antibody Therapy for Treatment of Rejection at Month 6

The Kaplan-Meier estimate of anti-lymphocyte antibody therapy for acute rejection (clinically-treated or biopsy-confirmed) within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow-up visit. (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo4.7
Alefacept6.7

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Percentage of Participants With Biopsy Confirmed Acute Mixed T-Cell Mediated and Antibody-Mediated Rejection at Month 6

"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification. A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed acute mixed T-cell mediated and antibody-mediated rejections within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo0.0
Alefacept1.0

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Percentage of Participants With Biopsy Confirmed Acute Rejection (T-Cell Mediated or Antibody Mediated) at Month 6

"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification. A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed acute T-cell mediated or antibody-mediated rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo9.3
Alefacept12.4

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Percentage of Participants With Biopsy Confirmed Antibody-Mediated Acute Rejection at Month 6

"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification:~Acute antibody-mediated rejection - documented anti-donor antibody ('suspicious for' if antibody not demonstrated):~Grade I: acute tubular necrosis-like - complement split product positive (C4d+), minimal inflammation;~Grade II: capillary-margination and/or thromboses, C4d+~Grade III: arterial - v3, C4d+.~A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed antibody-mediated acute rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo2.9
Alefacept3.8

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Percentage of Participants With Biopsy-Confirmed Acute T-cell Mediated Rejection as Assessed by Central Review at Month 6

"Biopsies were graded by the central reviewer according to the Banff 97/05 updated histological classification. A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed acute T-cell mediated rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo9.6
Alefacept7.7

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Percentage of Participants With Biopsy-confirmed Acute T-cell Mediated Rejection at Month 6 Assessed by Local Review

"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification:~Grade IA: significant interstitial infiltration (>25% parenchyma affected) and foci of moderate tubulitis;~Grade IB: significant interstitial infiltration (>25% parenchyma affected) and foci of severe tubulitis;~Grade IIA: mild to moderate intimal arteritis;~Grade IIB: severe intimal arteritis comprising >25% of the luminal area;~Grade III: transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocyte inflammation.~A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed acute T-cell mediated rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo6.7
Alefacept10.6

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Percentage of Participants With Clinically Treated Acute Rejection at Month 6

Patients who received immunosuppressive medications for the treatment of suspected or biopsy-confirmed acute rejections were considered to have a clinically-treated acute rejection. The Kaplan-Meier estimate of clinically treated acute rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow-up visit. (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo24.8
Alefacept15.4

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Percentage of Participants With Delayed Graft Function

Delayed graft function was defined as the requirement for dialysis within the first week post-transplant. (NCT00617604)
Timeframe: 1 week

Interventionpercentage of participants (Number)
Placebo12.1
Alefacept7.6

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Percentage of Participants With Efficacy Failure at Month 6

"Efficacy failure is defined as death, graft loss, biopsy-confirmed acute T-cell mediated rejection assessed by local reading or lost to follow-up.~The Kaplan-Meier estimate of efficacy failure within the first 6 months following transplantation is reported." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo15.0
Alefacept21.0

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Percentage of Participants With Steroid-resistant Acute Rejection at Month 6

"A steroid-resistant acute rejection is defined as a rejection episode which did not resolve following treatment with corticosteroids. In the case that a rejection episode was not treated with corticosteroids first but only with antibodies, it was included in this category.~The Kaplan-Meier estimate of steroid-resistant acute rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo7.6
Alefacept5.7

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Percentage of Participants With Treatment Failure at Month 6

Treatment failure is defined as efficacy failure (death, graft loss, biopsy-confirmed acute T-cell mediated rejection assessed by local reading, lost to follow-up) or early discontinuation of alefacept/placebo at any time (during the 12-week administration period) for any reason. The Kaplan-Meier estimate of treatment failure within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow-up visit. (NCT00617604)
Timeframe: 6 months

Interventionpercentage of participants (Number)
Placebo20.6
Alefacept25.7

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Change From Month 1 in Creatinine Clearance

The creatinine clearance was calculated according to the Cockcroft-Gault formula. (NCT00617604)
Timeframe: Month 1, 3, and 6

,
InterventionmL/minute (Mean)
Change From Month 1 to Month 3 (n=81, 79)Change From Month 1 to Month 6 (n=77, 73)
Alefacept-0.58493.0227
Placebo3.03364.5388

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Change From Month 1 in Glomerular Filtration Rate (GFR)

The GFR was calculated using the Modification of Diet in Renal Disease (MDRD) formula. (NCT00617604)
Timeframe: Month 1, 3, and 6

,
InterventionmL/min/1.73 m² (Mean)
Change From Month 1 to Month 3 (n=93, 87)Change From Month 1 to Month 6 (n=83, 78)
Alefacept0.28892.5444
Placebo3.15482.4275

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Change From Month 1 in Serum Creatinine

(NCT00617604)
Timeframe: Month 1, 3, and 6

,
Interventionµmol/L (Mean)
Change From Month 1 to Month 3 (n=94, 88)Change From Month 1 to Month 6 (n=86, 81)
Alefacept-5.0830-11.7212
Placebo-5.0125-6.1777

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Maximum Histological Grade of All Biopsies After Local Review

"The grade of acute rejection was classified according to Banff 97/05 updated version. If a patient had more than 1 rejection episode, the episode with the most severe grade was used.~Acute T-cell mediated rejection:~Grade IA: significant interstitial infiltration (>25% parenchyma affected) and foci of moderate tubulitis;~Grade IB: significant interstitial infiltration (>25% parenchyma affected) and foci of severe tubulitis;~Grade IIA: mild to moderate intimal arteritis;~Grade IIB: severe intimal arteritis comprising >25% of the luminal area;~Grade III: transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocyte inflammation.~Acute antibody-mediated rejection:~Grade I: acute tubular necrosis-like - complement split product positive (C4d+), minimal inflammation;~Grade II: capillary-margination and/or thromboses, C4d+~Grade III: arterial - v3, C4d+." (NCT00617604)
Timeframe: 6 months

,
Interventionpercentage of participants (Number)
T-Cell Mediated Rejection - No EventT-Cell Mediated Rejection - Grade IAT-Cell Mediated Rejection - Grade IBT-Cell Mediated Rejection - Grade IIAT-Cell Mediated Rejection - Grade IIBT-Cell Mediated Rejection - Grade IIIAntibody Mediated Rejection - No EventAntibody Mediated Rejection - Grade IAntibody Mediated Rejection - Grade IIAntibody Mediated Rejection - Grade III
Alefacept89.51.91.04.82.90.096.21.91.90.0
Placebo93.52.80.01.91.90.097.22.80.00.0

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Number of Participants With Adverse Events

"Causally related was defined as adverse events (AEs) assessed by the Investigator as possibly or probably related to study drug or records where the relationship was missing.~A serious adverse event (SAE) was any untoward medical occurrence that, at any dose:~Resulted in death.~Was life-threatening.~Resulted in persistent or significant disability/incapacity.~Resulted in congenital anomaly or birth defect.~Required patient hospitalization or led to prolongation of hospitalization~Was considered a medically important event.~All rejections and any BK virus, Epstein Barr virus and/or cytomegalovirus infection had to be reported as an SAE" (NCT00617604)
Timeframe: 6 Months

,
Interventionparticipants (Number)
Any adverse eventAE causally related to alefacept/placeboAE causally related to MMFAE causally related to tacrolimusAE causally related to steroidsSerious adverse eventsSAE causally related to alefacept/placeboSAE causally related to MMFSAE causally related to tacrolimusSAE causally related to steroidsAE leading to discontinuation of alefacept/placeboAE leading to discontinuation of MMFAE leading to discontinuation of tacrolimusAE leading to discontinuation of steroidsDeaths
Alefacept101415649465716212015106321
Placebo10236564946621919141178813

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Mycophenolic Acid (MPA) Maintenance Treatment

The primary assessment was based on the percentage of patients who were maintained at week 13 on a dose at least one dose equivalent greater than at baseline (visit 2/week 1). A dose equivalent was defined as EC-MPS 180 mg/day or MMF 250 mg/day. (NCT00239005)
Timeframe: at week 13 (last visit)

InterventionPercentage of Patients (Number)
Enteric-Coated Mycophenolate Sodium (EC-MPS)47.06
Mycophenolate Mofetil (MMF)16.39

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Changes in Gastrointestinal (GI) Symptoms as Measured by the Gastrointestinal Symptom Rating Scale (GSRS).

The GSRS is a 15-item instrument designed to assess the impact of upper and lower GI symptoms. There are five subscales: reflux, diarrhea, constipation, abdominal pain, and indigestion-each of which produces a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort). A higher score represents greater impairment of quality of life due to GI symptoms (range from 1 to 7). (NCT00239005)
Timeframe: At week 3 and week 13 (last visit)

,
InterventionUnits on a scale (Least Squares Mean)
Week 3: change in GSRS Total Score (N= 61, 59)Week 13: change in GSRS Total Score (N= 60, 56)
Enteric-Coated Mycophenolate Sodium (EC-MPS)-0.63-0.44
Mycophenolate Mofetil (MMF)-0.32-0.25

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Changes in Gastrointestinal Symptoms as Measured by the Gastrointestinal Quality of Life Index (GIQLI).

Health-related quality of life (HRQoL)was assessed by the Gastrointestinal Quality of Life Index (GIQLI). The GIQLI is a 36-item questionnaire to assess the impact of GI disease on daily life. The GIQLI also has five different subscales (GI symptoms, emotional status, physical and social functions, and stress of medical treatment) producing a total score of the 36 items. Lower scores represent more dysfunction. A higher score represents a better quality of life (range from 0 to 144). (NCT00239005)
Timeframe: At week 3 and week 13 (last visit)

,
InterventionUnits on a scale (Least Squares Mean)
Week 3: change in GIQLI Total Score (N= 61, 58)Week 13: change in GIQLI Total Score (N= 60, 56)
Enteric-Coated Mycophenolate Sodium (EC-MPS)11.654.84
Mycophenolate Mofetil (MMF)6.081.77

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Freedom From Acute Rejection or HCV Recurrence or Treatment Failure

"Freedom from acute rejection (Banff>grade 2 with RAI score>4) or freedom from HCV recurrence (Batts/Ludwig>Stage 2, or >Grade 3) that requires HCV antiviral therapy or treatment failure (patient death, graft loss, premature withdrawal from study regimen or treatment with more than 1 dose of corticosteroids for presumptive rejection without a biopsy to confirm the rejection; reported values represent the Number of participants with Freedom From Acute Rejection or HCV Recurrence or Treatment Failure" (NCT00163657)
Timeframe: 12 months

Interventionparticipants (Number)
Treatment Arm 169
Treatment Arm 270
Treatment Arm 3133

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Freedom From HCV Recurrence Within First Year That Requires HCV Antiviral Therapy and Freedom From Treatment Failure

Participants would have their blood drawn and tested for the HCV virus to determine if they had recurrence (NCT00163657)
Timeframe: 12 month post transplant

Interventionparticipants (Number)
Treatment Arm 139
Treatment Arm 239
Treatment Arm 372

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Incidence of Chronic Graft-versus-host-disease (GVHD) at 1 Year

"Number of participants with chronic graft-versus-host-disease (GVHD) at 1 year.~Clinical Limited cGVHD~Oral abnormalities consistent with cGVHD, a positive skin or lip biopsy, and no other manifestations of cGVHD.~Mild liver test abnormalities (alkaline phosphatase <2 x upper limit of normal, AST or ALT <3 x upper limit of normal and total bilirubin <1.6) with positive skin or lip biopsy, and no other manifestations of cGVHD.~Less than 6 papulosquamous plaques, macular-papular or lichenoid rash involving <20% of body surface area (BSA), dyspigmentation involving <20% BSA, or erythema involving <50% BSA, positive skin biopsy, and no other manifestations of cGVHD.~Ocular sicca (Schirmer's test <5mm with no more than minimal ocular symptoms), positive skin or lip biopsy, and no other manifestations of cGVHD.~Vaginal or vulvar abnormalities with positive biopsy, and no other manifestations of cGVHD." (NCT00719849)
Timeframe: 1 year post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI0
Cyclophosphamide/Fludarabine/TBI/ATG3

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Incidence of Clinically Significant Infections at 1 Year

Number of participants with clinically significant infections at 1 year (NCT00719849)
Timeframe: 1 year post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI3
Cyclophosphamide/Fludarabine/TBI/ATG3

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Incidence of Clinically Significant Infections at 2 Years

Number of participants with clinically significant infections at 2 years (NCT00719849)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI3
Cyclophosphamide/Fludarabine/TBI/ATG3

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Incidence of Clinically Significant Infections at 6 Months

Number of participants with clinically significant infections at 6 months (NCT00719849)
Timeframe: 6 months post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI3
Cyclophosphamide/Fludarabine/TBI/ATG3

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Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD) at Day 100

"Number of participants with Grade II-IV acute graft-versus-host-disease (GVHD) at day 100.~Acute GVHD Staging and Grading:~Overall grade 1: stage 1-2 skin, no liver or gut Overall grade 2: stage 3 skin or stage 1 liver or stage 1 gut Overall grade 3: stage 4 skin or stage 2-4 liver or stage 2-4 gut (without GVHD as a major contributing cause of death) Overall grade 4: stage 4 skin or stage 2-4 liver or stage 2-3 gut (with GVHD as a major contributing cause of death)" (NCT00719849)
Timeframe: Day 100 post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI3
Cyclophosphamide/Fludarabine/TBI/ATG6

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Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD) at Day 100

"Number of participants with Grade III-IV acute graft-versus-host-disease (GVHD) at day 100.~Acute GVHD Staging and Grading:~Overall grade 1: stage 1-2 skin, no liver or gut Overall grade 2: stage 3 skin or stage 1 liver or stage 1 gut Overall grade 3: stage 4 skin or stage 2-4 liver or stage 2-4 gut (without GVHD as a major contributing cause of death) Overall grade 4: stage 4 skin or stage 2-4 liver or stage 2-3 gut (with GVHD as a major contributing cause of death)" (NCT00719849)
Timeframe: Day 100 post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI2
Cyclophosphamide/Fludarabine/TBI/ATG1

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Incidence of Neutrophil Engraftment at Day 42

Number of participants with neutrophil engraftment at day 42 (NCT00719849)
Timeframe: Day 42 post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI3
Cyclophosphamide/Fludarabine/TBI/ATG7

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Incidence of Non-relapse Mortality at 6 Months

Number of Participants with Non-relapse Mortality at 6 Months (NCT00719849)
Timeframe: 6 months post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI2
Cyclophosphamide/Fludarabine/TBI/ATG2

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Incidence of Platelet Engraftment at 6 Months

Number of participants with platelet engraftment at 6 months (NCT00719849)
Timeframe: 6 months post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI2
Cyclophosphamide/Fludarabine/TBI/ATG2

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Incidence of Relapse at 1 Year

"Number of participants with relapse at 1 year.~Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated." (NCT00719849)
Timeframe: 1 year post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI1
Cyclophosphamide/Fludarabine/TBI/ATG4

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Incidence of Relapse at 2 Years

"Number of participants with relapse at 2 years.~Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated." (NCT00719849)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Cyclophosphamide/Fludarabine/TBI1
Cyclophosphamide/Fludarabine/TBI/ATG5

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Probability of Progression-free Survival at 1 Year

Kaplan-Meier estimate of the probability of progression-free survival at 1 year (NCT00719849)
Timeframe: 1 year post transplant

Interventionprogression free survival probability (Number)
Cyclophosphamide/Fludarabine/TBI0.25
Cyclophosphamide/Fludarabine/TBI/ATG0.38

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Probability of Progression-free Survival at 2 Years

Kaplan-Meier estimate of the probability of progression-free survival at 2 years (NCT00719849)
Timeframe: 2 years post transplant

Interventionprogression free survival probability (Number)
Cyclophosphamide/Fludarabine/TBI0.25
Cyclophosphamide/Fludarabine/TBI/ATG0.25

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Probability of Survival at 1 Year

Kaplan-Meier estimate of the probability of survival at 1 year (NCT00719849)
Timeframe: 1 year post transplant

Interventionsurvival probability (Number)
Cyclophosphamide/Fludarabine/TBI0.25
Cyclophosphamide/Fludarabine/TBI/ATG0.50

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Probability of Survival at 2 Years

Kaplan-Meier estimate of the probability of survival at 2 years (NCT00719849)
Timeframe: 2 years post transplant

Interventionsurvival probability (Number)
Cyclophosphamide/Fludarabine/TBI0.25
Cyclophosphamide/Fludarabine/TBI/ATG0.38

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Chimerism

Count of participants who experienced dominance of one cord blood unit (defined by >or= 95% contribution of one cord blood unit to BM and all PB fractions -- CD3+, CD33+, CD56+, and CD19+) at 7 days, 14 days, 21 days, 28 days, 56 days, and 80 days, 6 months, 1 and 2 years post transplant. (NCT00719849)
Timeframe: 7 days, 14 days, 21 days, 28 days, 56 days, and 80 days, 6 months, 1 and 2 years post transplant

,
InterventionParticipants (Count of Participants)
Day 7Day 14Day 21Day 28Day 56Day 806 months1 year2 years
Cyclophosphamide/Fludarabine/TBI001111222
Cyclophosphamide/Fludarabine/TBI/ATG001556777

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The Proportion of Participants With Graft Loss or Death Within 12 Months Post Kidney Transplantation

Graft loss is defined as the need for dialysis for more than 30 days duration, allograft nephrectomy, or the decision to withdraw immunosuppression due to graft failure. (NCT00240994)
Timeframe: Up to one year post kidney transplantation procedure

InterventionProportion of participants (Number)
Alemtuzumab (Campath)0.057

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Duration of Exposure to Study Medication

The duration of exposure was calculated as the date of the last Mycophenolate sodium dose minus the date of the last Mycophenolate sodium dose +1. (NCT00423098)
Timeframe: 24 weeks

Interventiondays (Mean)
Standard Dose164.5
Low Dose157.7

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Change From Baseline in Overall Disease Activity With British Isles Lupus Assessment Group (BILAG)

BILAG (British Isles Lupus Assessment Group) index divides lupus activity into 8 organs/systems and was based on the principle of the physician's intention to treat, assessing activity in the previous one month. Each organ or system was given a score of A to E, where A = disease that is sufficiently active to require disease modifying treatment; a B = problems requiring symptomatic treatment; C = stable mild disease; D = previously affected but currently inactive system; and E = the system or organ has never been involved. [A=9, B=3, C=1, D/E=0 the score range for each patient will be 0-72]. (NCT00423098)
Timeframe: From Baseline to week 4, week 12 and week 24

,
InterventionUnits on a scale (Mean)
Change from baseline Week 4: (N= 40, 37)Change from baseline Week 12: (N= 41, 35)Change from baseline Week 24: (N= 40, 34)
Low Dose-5.5-8.7-9.4
Standard Dose-4.8-8.6-8.6

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Change From Baseline in Overall Disease Activity With Systematic Lupus Erythematosus Disease Activity Index (SLEDAI)

SLEDAI stands for Systemic Lupus Erythematosus Disease Activity Index and was a well established global score index based on assessment of 24 items measuring a disease activity in the 10-day period prior to the assessment. SLEDAI item weights range from 1 for fever to 8 for seizures. A maximum theoretical score is 105. Total score range from 1 to 105. A flare has been defined as a SLEDAI score increase of 3 or more to a level of 8 or higher. During flares SLEDAI scores of 25 to 30 are common. (NCT00423098)
Timeframe: From Baseline to week 4, week 12 and week 24

,
InterventionUnits on a scale (Mean)
Change from Baseline to Week 4: (N= 39, 37)Change from Baseline to Week 12: (N= 41, 35)Change from Baseline to Week 24: (N= 39, 34)
Low Dose-7.7-10.3-9.8
Standard Dose-7.4-9.7-10.3

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Cumulative Dose of Prednisone Equivalent Corticosteroids (CS)

Corticosteroid use was measured as cumulative dose until 12 and 24 weeks of treatment as well as daily doses at baseline, 12 and 24 weeks. (NCT00423098)
Timeframe: 12 Weeks and 24 Weeks

,
Interventionmg/kg (Mean)
Week 12Week 24
Low Dose68.273.0
Standard Dose106.1114.2

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Number of Patients With Adverse Events and Infections

Safety assessments included collecting all adverse events (AEs), serious adverse events (SAEs), with their severity and relationship to study drug. According to FDA 21CFR 314.80, a serious adverse event (SAE) is described as any adverse event that leads to death, is life threatening ( NIH criteria Grade 4), causes or prolongs hospitalization, results in a congenital anomaly, or any other important medical event not described above. (NCT00423098)
Timeframe: 24 weeks

,
Interventionparticipants (Number)
At least one adverse eventAny severe adverse eventAny drug related adverse eventAny serious adverse eventAny infectionAny severe infectionAny drug related infectionAny serious infection
Low Dose30316417161
Standard Dose357188253104

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Number of Patients With Complete Remission

Complete remission was defined as urine protein/urine creatinine ratio < 0.5 gram urine protein per gram urine creatinine, urine sediment normalized (no cellular casts, < 5 red cells per high power field), and serum creatinine within 10% of normal range according to local lab. (NCT00423098)
Timeframe: 24 Weeks

,
InterventionParticipants (Number)
YesNo
Low Dose831
Standard Dose834

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Number of Patients With Complete Remission

Complete remission was defined as urine protein/urine creatinine ratio < 0.5 gram urine protein per gram urine creatinine, urine sediment normalized (no cellular casts, < 5 red cells per high power field), and serum creatinine within 10% of normal value. (NCT00423098)
Timeframe: 12 Weeks

,
Interventionparticipants (Number)
YesNo
Low Dose534
Standard Dose933

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Number of Patients With Moderate to Severe Flares

A moderate to severe flare was defined as the occurrence of increased lupus activity after partial or complete remission, based on the presence of 1 BILAG A score or >=3 BILAG B scores. British Isles Lupus Assessment Group (BILAG) index divides lupus activity in 8 organs/systems which are each given a score of A to E. A=disease sufficiently active to need disease modifying treatment; B=problems requiring symptomatic treatment; C=mild stable disease; D=previously affected but currently inactive system; E=the system or organ has never been involved. BILAG score: A=9, B=3, C=1, D/E=0; range(0-72) (NCT00423098)
Timeframe: 12 and 24 weeks

,
Interventionparticipants (Number)
At week 12 - YesAt week 12 - NoAt week 24 - YesAt week 24 - No
Low Dose039039
Standard Dose042141

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Number of Patients With Partial Remission

Partial remission was defined as urine protein/creatinine ratio reduced by at least 50% from baseline and stable serum creatinine within 10% of baseline value) or improved. (NCT00423098)
Timeframe: Baseline to 12 and 24 weeks

,
InterventionParticipants (Number)
At 12 weeks - YesAt 12 weeks - NoAt 24 weeks - YesAt 24 weeks - No
Low Dose11281425
Standard Dose16262022

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Number of Patients With Treatment Failure

Treatment failure was defined as no therapeutic response (without complete or partial remission) or premature discontinuation during the first 24 weeks from study medication or the study for any reason except complete or partial remission. (NCT00423098)
Timeframe: 12 Weeks and 24 Weeks

,
Interventionparticipants (Number)
At 12 weeks - YesAt 12 weeks - NoAt 24 weeks - YesAt 24 weeks - No
Low Dose25142217
Standard Dose23192121

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Calculated Glomerular Filtration Rate (cGFR) After Kidney Transplant to Evaluate Kidney Function (12 Months Analysis)

The glomerular filtration rate (GFR) was calculated by the Nankivell formula: GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)^ 2 + C where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. (NCT00371826)
Timeframe: At Month 12

InterventionmL/min per 1.73 m^2 (Mean)
Calcineurin Inhibitor (CNI) Withdrawal65.2
CNI+MPA+ Steroid69.3
Steroid Withdrawal66.9

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Creatinine Clearance (CrCl) Calculated by the Cockcroft-Gault Formula (12 Months Analysis)

"Creatinine clearance were calculated according to the Cockcroft-Gault formula:~CrCl (males) = (140-A) × BW/(72 × Cr) CrCl (females) = CrCl (males) × 0.85 where A is age [years], BW is body weight [kg], and Cr is the serum concentration of creatinine [mg/dL].~The Cockcroft-Gault formula estimates creatinine clearance based on serum creatinine level, body weight, and age." (NCT00371826)
Timeframe: At Month 12

InterventionmL/min (Mean)
Calcineurin Inhibitor (CNI) Withdrawal66.1
CNI+MPA+ Steroid67.8
Steroid Withdrawal63.2

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Mean Serum Creatinine (12 Months Analysis)

(NCT00371826)
Timeframe: At Month 12

Interventionumol/L (Mean)
Calcineurin Inhibitor (CNI) Withdrawal118.9
CNI+MPA+ Steroid161.0
Steroid Withdrawal148.6

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Mean Urine Albumin/Creatinine Ratio (ACR) as Measurement of Proteinuria (12 Months Analysis)

Proteinuria is measured by spot morning urine Albumin/Creatinine Ratio [ACR]. When the ACR is more than or equal to 30 mg/mmol then it is known as proteinuria. (NCT00371826)
Timeframe: At Month 12

Interventionmg/mg (Mean)
Calcineurin Inhibitor (CNI) Withdrawal0.2
CNI+MPA+ Steroid0.1
Steroid Withdrawal0.1

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Number of Participants Hospitalized for Reasons Other Than Primary Transplantation (12 Months Analysis)

This analysis is reporting number of participants hospitalized for reasons (such as acute rejection, infection, gastrointestinal (GI) events, cardiovascular event, metabolic disorder and Other) other than primary transplantation. (NCT00371826)
Timeframe: Month 12

InterventionParticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal34
CNI+MPA+ Steroid31
Steroid Withdrawal13

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Number of Participants Hospitalized for Reasons Other Than Primary Transplantation (36 Months Analysis)

This analysis is reporting number of participants hospitalized for reasons (such as acute rejection, infection, gastrointestinal (GI) events, cardiovascular event, metabolic disorder and Other) other than primary transplantation. (NCT00371826)
Timeframe: Month 36

InterventionParticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal16
CNI+MPA+ Steroid30
Steroid Withdrawal1

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Number of Participants With Antibody-mediated Rejection Per Treatment Group (12 Months Analysis)

(NCT00371826)
Timeframe: At Month 12

InterventionParticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal5
CNI+MPA+ Steroid2
Steroid Withdrawal2

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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Per Treatment Group (12 Months Analysis)

A biopsy-proven acute rejection is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification. (NCT00371826)
Timeframe: At Month 12

InterventionParticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal15
CNI+MPA+ Steroid6
Steroid Withdrawal5

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Number of Participants With Composite Endpoint of Treatment Failure (12 Months Analysis)

"Composite endpoint of treatment failure includes biopsy-proven acute rejection (BPAR), graft loss, death and loss-to-follow-up. A BPAR is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification.~The allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss." (NCT00371826)
Timeframe: Month 12

InterventionParticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal16
CNI+MPA+ Steroid8
Steroid Withdrawal11

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Number of Participants With Erythropoietin Usage (12 Months Analysis)

(NCT00371826)
Timeframe: Month 12

Interventionparticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal20
CNI+MPA+ Steroid10
Steroid Withdrawal8

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Number of Participants With Erythropoietin Usage (36 Months Analysis)

(NCT00371826)
Timeframe: Month 36

Interventionparticipants (Number)
Calcineurin Inhibitor (CNI) Withdrawal5
CNI+MPA+ Steroid7
Steroid Withdrawal2

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Calculated Glomerular Filtration Rate (cGFR) After Kidney Transplant to Evaluate Kidney Function (36 Months Analysis)

The glomerular filtration rate (GFR) was calculated by the Nankivell formula: GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)^ 2 + C where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. (NCT00371826)
Timeframe: At Month 24 and 36

,,
InterventionmL/min per 1.73 m^2 (Mean)
Month 24 (n= 23, 36, 4)Month 36 (n= 19, 35, 3)
Calcineurin Inhibitor (CNI) Withdrawal69.571.6
CNI+MPA+ Steroid71.869.1
Steroid Withdrawal67.061.0

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Change in Bone Mineral Density Between Week 2 and Month 24 (36 Months Analysis)

Measurements of bone mineral density (BMD) by Dual Energy X-ray Absorptiometry (DEXA) were done at Week 2 and Month 24. Change in BMD between week 2 and Month 24 were done for neck of femur and lumbar spine. (NCT00371826)
Timeframe: Week 2, Month 24

,,
Interventiong/cm^2 (Mean)
Neck of Femur (Month 24- Week 2)Lumbar Spine (Month 24 - Week 2)
Calcineurin Inhibitor (CNI) Withdrawal0.2-0.0
CNI+MPA+ Steroid-0.1-0.1
Steroid Withdrawal-0.1-0.0

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Creatinine Clearance Calculated by the Cockcroft-Gault Formula (36 Months Analysis)

"Creatinine clearance were calculated according to the Cockcroft-Gault formula:~CrCl (males) = (140-A) × BW/(72 × Cr) CrCl (females) = CrCl (males) × 0.85 where A is age [years], BW is body weight [kg], and Cr is the serum concentration of creatinine [mg/dL].~The Cockcroft-Gault formula estimates creatinine clearance based on serum creatinine level, body weight, and age." (NCT00371826)
Timeframe: At Month 12, 24 and 36

,,
InterventionmL/min (Mean)
At Month 12 (n= 23, 39, 4)At Month 18 (n=22, 36, 4)At Month 24 (n= 23, 36, 4)At Month 36 (n= 23, 39, 4)
Calcineurin Inhibitor (CNI) Withdrawal69.869.965.666.3
CNI+MPA+ Steroid71.773.772.867.5
Steroid Withdrawal63.766.263.762.7

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Mean Serum Creatinine (36 Months Analysis)

(NCT00371826)
Timeframe: At Month 12, 18, 24 and 36

,,
Interventionumol/L (Mean)
At Month 12 (n= 23, 39, 4)At Month 18 (n= 22, 36, 4)At Month 24 (n = 23, 36, 4)At Month 36 (n= 19, 35, 3)
Calcineurin Inhibitor (CNI) Withdrawal112.7113.6119.9119.4
CNI+MPA+ Steroid123.0121.4123.7131.8
Steroid Withdrawal146.8146.3146.5176.0

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Mean Short-form 36 Health Survey (SF-36) Score as a Measure of Quality of Life Assessment (12 Months Analysis)

"SF-36 measures impact of disease on overall quality of life (QoL). 36-item survey has 8 subscales. The 8 subscales are: Physical functioning (PF), Role-physical (RP), Bodily pain (BP), General health (GH), Vitality (VT), Social functioning (SF), Role-emotional (RE) and Mental health (MH).~Score for eash sub-scale has been standardized with the use of norm-based methods based on assessment of the general U.S. population free of chronic conditions. Scores range from 1-100 with a mean=50 and a standard deviation=10. A higher score indicates less impact on QoL." (NCT00371826)
Timeframe: At Month 12

,,
Interventionunits on a scale (Mean)
Physical functioning (PF)Role-physical (RP)Bodily pain (BP)General health (GH)Vitality (VT)Social Functioning (SF)Role-emotional (RE)Mental health (MH)
Calcineurin Inhibitor (CNI) Withdrawal78.772.973.667.162.774.572.273.3
CNI+MPA+ Steroid76.978.281.571.272.481.181.179.1
Steroid Withdrawal82.0100.096.886.885.097.5100.092.8

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Mean Short-form 36 Health Survey (SF-36) Score as a Measure of Quality of Life Assessment (36 Months Analysis)

"SF-36 measures impact of disease on overall quality of life (QoL). 36-item survey has 8 subscales. The 8 subscales are : Physical functioning (PF), Role-physical (RP), Bodily pain (BP), General health (GH), Vitality (VT), Social functioning (SF), Role-emotional (RE) and Mental health (MH).~Score for each sub-scale has been standardized with the use of norm-based methods based on assessment of the general U.S. population free of chronic conditions. Scores range from 1-100 with a mean=50 and a standard deviation=10. A higher score indicates less impact on QoL." (NCT00371826)
Timeframe: At Month 24

,,
Interventionunits on a scale (Mean)
Physical functioning (PF)Role-physical (RP)Bodily pain (BP)General health (GH)Vitality (VT)Social Functioning (SF)Role-emotional (RE)Mental health (MH)
Calcineurin Inhibitor (CNI) Withdrawal84.375.081.671.670.085.972.977.5
CNI+MPA+ Steroid87.677.485.069.872.780.683.977.9
Steroid Withdrawal90.0100.084.067.060.068.833.362.0

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Mean Urine Albumin/Creatinine Ratio [ACR] as Measurement of Proteinuria (36 Months Analysis)

Proteinuria is measured by spot morning urine Albumin/Creatinine Ratio [ACR]. When the ACR is more than or equal to 30 mg/mmol then it is known as proteinuria. (NCT00371826)
Timeframe: At Month 12, 18, 24 and 36

,,
Interventionmg/mmol (Mean)
At 12 Month (n = 18, 32, 2)At 18 Month (n= 17, 31, 2)At 24 Month ( n= 16, 29, 1)At 36 Month ( n= 15, 25, 0)
Calcineurin Inhibitor (CNI) Withdrawal23.528.230.030.6
CNI+MPA+ Steroid8.18.16.623.4
Steroid Withdrawal4.85.37.0NA

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Number of Participants With Antibody-mediated Rejection Per Treatment Group (36 Months Analysis)

(NCT00371826)
Timeframe: At Month 12, 24 and 36

,,
InterventionParticipants (Number)
At Month 12At Month 24At Month 36
Calcineurin Inhibitor (CNI) Withdrawal111
CNI+MPA+ Steroid002
Steroid Withdrawal000

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Number of Participants With Any Wound Problems (36 Months Analysis)

Patients with any wound healing problem such as infection related to kidney surgery, dehiscence, lymphocele, hernia, seroma, hematoma, ureteral anastomotic complication and other were reported in this analysis. (NCT00371826)
Timeframe: At Month 12, 24 and 36

,,
InterventionParticipants (Number)
At Month 12At Month 24At Month 36
Calcineurin Inhibitor (CNI) Withdrawal6910
CNI+MPA+ Steroid111313
Steroid Withdrawal222

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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Influenced by Demographic Characteristics and Morbidities (12 Months Analysis)

The influence of demographic characteristics and comorbidities on incidence of BPAR were analyzed in the following way: Demographic characteristics were age (<55 years, ≥55 years), Expanded criteria Donor (ECD) organ (donor age >60 years or donor non heart-beating and donor age >50), gender, living vs. deceased donor, Body Mass Index (BMI) classes (underweight <18.5, normal 18.5 - <25.0, overweight 25.0 - <30.0, obesity 30.0 and above), years on dialysis before transplantation (<1, 1-5, >5 years). Comorbidities were diabetes, hypertension, cardiovascular diseases/events, nephrosclerosis, glomerulonephritis/glomerular disease, polycystic disease, and Cytomegalovirus status. (NCT00371826)
Timeframe: At Month 12

,,
InterventionParticipants (Number)
Age < 55 yearsAge > = 55 yearsECD OrganNO ECD OrganMaleFemaleLiving donorDeceased donorBMI < 18.5BMI 18.5 - <25BMI 25 - <30BMI >= 30Years on dialysis before transplantation: None< 1 Year on dialysis before transplantation1 - 5 Years on dialysis before transplantation> 5 Years on dialysis before transplantationDiabetes mellitus: NoDiabetes mellitus: YesHypertension: NoHypertension: YesCardiovascular disease : NoCardiovascular disease: YesNephrosclerosis: NoNephrosclerosis: YesGlomerulonephritis/glomerular disease : NoGlomerulonephritis/glomerular disease: YesCytomegalovirus : NegativeCytomegalovirus : Positive
Calcineurin Inhibitor (CNI) Withdrawal114312114960582138310521341115087213
CNI+MPA+ Steroid5106606012300312421533605124
Steroid Withdrawal4123412303110122410523413205

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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Influenced by Demographic Characteristics and Morbidities (36 Months Analysis)

The influence of demographic characteristics and comorbidities on incidence of BPAR were analyzed in the following way: Demographic characteristics were age (<55 years, ≥55 years), Expanded Criteria Donor [ECD] organ (donor age >60 years or donor non heart-beating and donor age >50), gender, living vs. deceased donor, Body Mass Index (BMI) classes (underweight <18.5, normal 18.5 - <25.0, overweight 25.0 - <30.0, obesity 30.0 and above), years on dialysis before transplantation (<1, 1-5, >5 years). Comorbidities were diabetes, hypertension, cardiovascular diseases/events, nephrosclerosis, glomerulonephritis/glomerular disease, polycystic disease, and Cytomegalovirus status. (NCT00371826)
Timeframe: At Month 36

,,
InterventionParticipants (Number)
Age < 55 yearsAge > = 55 yearsECD OrganNO ECD OrganMaleFemaleLiving donorDeceased donorBMI < 18.5BMI 18.5 - <25BMI 25 - <30BMI >= 30Years on dialysis before transplantation: None< 1 Year on dialysis before transplantation1 - 5 Years on dialysis before transplantation> 5 Years on dialysis before transplantationDiabetes mellitus: NoDiabetes mellitus: YesHypertension: NoHypertension: YesCardiovascular disease : NoCardiovascular disease: YesNephrosclerosis: NoNephrosclerosis: YesGlomerulonephritis/glomerular disease : NoGlomerulonephritis/glomerular disease: YesCytomegalovirus : NegativeCytomegalovirus : Positive
Calcineurin Inhibitor (CNI) Withdrawal6006425111400321331533604215
CNI+MPA+ Steroid7108716203411421621753717144
Steroid Withdrawal0000000000000000000000000000

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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Per Treatment Group (36 Months Analysis)

A biopsy-proven acute rejection is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification. (NCT00371826)
Timeframe: At Month 12, 24 and 36

,,
InterventionParticipants (Number)
At Month 12At Month 24At Month 36
Calcineurin Inhibitor (CNI) Withdrawal456
CNI+MPA+ Steroid458
Steroid Withdrawal000

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Number of Participants With Composite Endpoint of Treatment Failure (36 Months Analysis)

"Composite endpoint of treatment failure includes biopsy-proven acute rejection (BPAR), graft loss, death and loss-to-follow-up. A BPAR is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification.~The allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss." (NCT00371826)
Timeframe: At Month 12, 24 and 36

,,
InterventionParticipants (Number)
At Month 12At Month 24At Month 36
Calcineurin Inhibitor (CNI) Withdrawal467
CNI+MPA+ Steroid4610
Steroid Withdrawal000

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Number of Participants With Employment Status (12 Months Analysis)

"The various employment status reported are:~Employed/self employed full time~Employed part time~Unemployed~Homemaker~Volunteer~Permanently disabled~Non-permanently disable~Retired~Other" (NCT00371826)
Timeframe: At screening (at day 0 +/- 7 days ), At Month 12

,,
InterventionParticipants (Number)
Screening visit: Employed/self employed full timeMonth 12: Employed/self employed full timeScreening visit: Employed part timeMonth 12: Employed part timeScreening visit: UnemployedMonth 12: UnemployedScreening visit: HomemakerMonth 12 : HomemakerScreening visit: VolunteerMonth 12: VolunteerScreening visit: Permanently disabledMonth 12: Permanently disabledScreening visit: Non-permanently disabledMonth 12: Non-permanently disabledScreening visit: RetiredMonth 12: RetiredScreening visit: OtherMonth 12: Other
Calcineurin Inhibitor (CNI) Withdrawal18693106321121114111
CNI+MPA+ Steroid21147581550022202200
Steroid Withdrawal9240106520000001010

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Number of Participants With Employment Status (36 Months Analysis)

"The various employment status reported are:~Employed/self employed full time~Employed part time~Unemployed~Homemaker~Volunteer~Permanently disabled~Non-permanently disable~Retired~Other" (NCT00371826)
Timeframe: At screening (at day 0 +/- 7 days ), At Month 36

,,
InterventionParticipants (Number)
Screening visit: Employed/self employed full timeMonth 36: Employed/self employed full timeScreening visit: Employed part timeMonth 36: Employed part timeScreening visit: UnemployedMonth 36: UnemployedScreening visit: HomemakerMonth 36 : HomemakerScreening visit: Permanently disabledMonth 36: Permanently disabledScreening visit: Non-permanently disabledMonth 36: Non-permanently disabledScreening visit: RetiredMonth 36: RetiredScreening visit: OtherMonth 36: Other
Calcineurin Inhibitor (CNI) Withdrawal5443533300000011
CNI+MPA+ Steroid101074763311112200
Steroid Withdrawal1100101100000000

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Number of Participants With Histological Evidence Chronic Allograft Nephropathy (CAN) (12 Months Analysis)

"A per-protocol biopsy was performed at Baseline and Month 12 and read by an independent blinded pathologist in order to assess chronic allograft nephropathy. Chronic rejection is characterized by a slow progressive decline in renal function and is typically preceded by the histological picture of chronic allograft nephropathy. The presence of biopsy confirmed Grade I, II or III chronic allograft nephropathy by Banff 97 criteria was assessed on all optional biopsies obtained for clinical suspicion of chronic rejection.~Data summarized by 3 categories. Yes - Patients with histological evidence of CAN ; No - Patients with histological evidence of CAN and Not Done - Central protocol defined kidney allograft biopsies were not done." (NCT00371826)
Timeframe: At Month 12

,,
InterventionParticipants (Number)
YESNONot Done
Calcineurin Inhibitor (CNI) Withdrawal8157
CNI+MPA+ Steroid6268
Steroid Withdrawal253

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Number of Participants With Histological Evidence Chronic Allograft Nephropathy (CAN) (36 Months Analysis)

"Chronic rejection is characterized by a slow progressive decline in renal function and is typically preceded by the histological picture of chronic allograft nephropathy. The presence of biopsy confirmed Grade I, II or III chronic allograft nephropathy by Banff 97 criteria was assessed on all optional biopsies obtained for clinical suspicion of chronic rejection.~Data summarized by 3 categories. Yes - Patients with histological evidence of CAN ; No - Patients with histological evidence of CAN and Not Done - Central protocol defined kidney allograft biopsies were not done." (NCT00371826)
Timeframe: At Month 36

,,
InterventionParticipants (Number)
YESNONot Done
Calcineurin Inhibitor (CNI) Withdrawal288
CNI+MPA+ Steroid11614
Steroid Withdrawal111

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Number of Participants With New Onset Diabetes Mellitus After Transplantation (NODAT) and Impaired Fasting Glucose (36 Months Analysis)

"The symptoms of new onset diabetes mellitus after transplantation (NODAT) and impaired fasting glucose are defined as any of the following conditions:~Patients receiving glucose lowering treatment~2 fasting plasma glucose (FPG) values >= 126 mg/dL or 2 random plasma glucose (RPG) values >= 200 mg/dL or FPG value >= 126 mg/dL and 1 RPG value >= 200 mg/dL~Diabetes reported as treatment emergent AE with end date > Day 15" (NCT00371826)
Timeframe: At Month 36

,,
InterventionParticipants (Number)
Glucose lowering treatmentFPG or RPGDiabetes as treatment emergent AEAny of the above symptoms
Calcineurin Inhibitor (CNI) Withdrawal5356
CNI+MPA+ Steroid102411
Steroid Withdrawal2122

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Number of Participants With Notable Abnormal Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) as Measurement of Effect of Treatment on Cardiovascular Health (12 Months Analysis)

"Notable abnormal systolic blood pressure is defined as :~Either an increase of >=30 that results in >=180 or >200 (mm/Hg)~OR a decrease of >=30 that results in <=90 or <75 (mm/Hg)from baseline~Notable abnormal diastolic blood pressure is defined as :~Either an increase of >=20 that results in >=105 or >115 (mm/Hg)~OR a decrease of >=20 that results in <=50 or <40 (mm/Hg) from baseline" (NCT00371826)
Timeframe: Baseline, Overall post-baseline up to 12 month

,,
InterventionParticipants (Number)
SBP: >=180 mm/Hg or 200 mm/HgSBP: < = 90 mm/Hg or < 200 mm/HgDBP : >=105 mm/Hg or >115 mm/HgDBP: <=50 mm/Hg or <40 mm/Hg
Calcineurin Inhibitor (CNI) Withdrawal5131
CNI+MPA+ Steroid5064
Steroid Withdrawal7050

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Number of Participants With Notable Abnormal Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) as Measurement of Effect of Treatment on Cardiovascular Health (36 Months Analysis)

"Notable abnormal systolic blood pressure is defined as :~Either an increase of >=30 that results in >=180 or >200 (mm/Hg)~OR a decrease of >=30 that results in <=90 or <75 (mm/Hg) from baseline~Notable abnormal diastolic blood pressure is defined as :~Either an increase of >=20 that results in >=105 or >115 (mm/Hg)~OR a decrease of >=20 that results in <=50 or <40 (mm/Hg) from baseline" (NCT00371826)
Timeframe: Baseline, Overall post baseline up to Month 36

,,
InterventionParticipants (Number)
SBP: >=180 mm/Hg or 200 mm/HgSBP: < = 90 mm/Hg or < 200 mm/HgDBP : >=105 mm/Hg or >115 mm/HgDBP: <=50 mm/Hg or <40 mm/Hg
Calcineurin Inhibitor (CNI) Withdrawal3121
CNI+MPA+ Steroid4054
Steroid Withdrawal1010

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Number of Participants With Notable Abnormalities in Total Cholesterol and Triglycerides as Measurement of Effect of Treatment on Cardiovascular Health (12 Months Analysis)

"Notable abnormal total cholesterol is defined as : High: >= 9.1 mmol/L , normal range is 0.00 - 5.17 mmol/L~Notable abnormal triglycerides is defined as : High: >= 8.5 mmol/L, normal range is 0.30 - 2.00 mmol/L" (NCT00371826)
Timeframe: Overall post baseline up to month 12

,,
InterventionParticipants (Number)
Total Cholesterol: High (n = 48, 47,30)Triglycerides : High (n= 48, 46, 30)
Calcineurin Inhibitor (CNI) Withdrawal61
CNI+MPA+ Steroid00
Steroid Withdrawal30

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Number of Participants With Notable Abnormalities in Total Cholesterol and Triglycerides as Measurement of Effect of Treatment on Cardiovascular Health (36 Months Analysis)

"Notable abnormal total cholesterol is defined as : High: >= 9.1 mmol/L , normal range is 0.00 - 5.17 mmol/L~Notable abnormal triglycerides is defined as : High: >= 8.5 mmol/L, normal range is 0.30 - 2.00 mmol/L" (NCT00371826)
Timeframe: Overall Post Baseline up to month 36

,,
InterventionParticipants (Number)
Total Cholesterol: HighTriglycerides : High
Calcineurin Inhibitor (CNI) Withdrawal60
CNI+MPA+ Steroid01
Steroid Withdrawal10

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Number of Participants With Post Transplant Diabetes Mellitus (PTDM) and Impaired Fasting Glucose (12 Months Analysis)

"The symptoms of post transplant diabetes mellitus (PTDM) and impaired fasting glucose are defined as any of the following conditions:~Patients receiving glucose lowering treatment~Fasting plasma glucose (FPG) >= 126 mg/dL on 2 separate occasions~Hemoglobin subtype A1c (HbA1c) > 6.5%~Diabetes reported as treatment emergent AE with end date > Day 15" (NCT00371826)
Timeframe: At Month 12

,,
InterventionParticipants (Number)
Glucose lowering treatmentFPG >= 126 mg/dL on 2 separate occasionsHbA1c > 6.5%Diabetes as treatment emergent AEAny of the above symptoms
Calcineurin Inhibitor (CNI) Withdrawal10612819
CNI+MPA+ Steroid903210
Steroid Withdrawal55538

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Number of Participants With Sub Clinical Acute Rejection (12 Months Analysis)

"Based on Banff 97 criteria, sub clinical acute rejection can be:~GRADE IA - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>4 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IB - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>10 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IIA - Cases with significant interstitial infiltration and mild to moderate intimal arteritis (v1).~GRADE IIB - Cases with moderate to severe intimal arteritis comprising >25% of the luminal area (v2).~GRADE III - Cases with transmural arteritis or fibrinoid change and necrosis of medial smooth muscle cells (v3).~Borderline category is used when no intimal arteritis is present, but there are foci of mild tubulitis (1 to 4 mononuclear cells/tubular cross section)." (NCT00371826)
Timeframe: At Month 12

,,
InterventionParticipants (Number)
NOBorderlineGrade IAGrade IBGrade IIAGrade IIBGrade IIINot Done
Calcineurin Inhibitor (CNI) Withdrawal202100007
CNI+MPA+ Steroid292010008
Steroid Withdrawal52000003

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Number of Participants With Sub Clinical Acute Rejection (36 Months Analysis)

"Based on Banff 97 criteria, sub clinical acute rejection can be:~GRADE IA - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>4 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IB - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>10 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IIA - Cases with significant interstitial infiltration and mild to moderate intimal arteritis (v1).~GRADE IIB - Cases with moderate to severe intimal arteritis comprising >25% of the luminal area (v2).~GRADE III - Cases with transmural arteritis or fibrinoid change and necrosis of medial smooth muscle cells (v3).~Borderline' category is used when no intimal arteritis is present, but there are foci of mild tubulitis (1 to 4 mononuclear cells/tubular cross section)." (NCT00371826)
Timeframe: At Month 36

,,
InterventionParticipants (Number)
Month 36: NOMonth 36: BorderlineMonth 36: Grade IAMonth 36: Grade IBMonth 36: Grade IIAMonth 36: Grade IIBMonth 36: Grade IIIMonth 36: Not Done
Calcineurin Inhibitor (CNI) Withdrawal91000008
CNI+MPA+ Steroid1511000014
Steroid Withdrawal20000001

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Number of Participants With Wound Problems(12 Months Analysis)

Patients with any wound healing problem such as infection related to kidney surgery, dehiscence, lymphocele, hernia, seroma, hematoma, ureteral anastomotic complication and other were reported in this analysis. (NCT00371826)
Timeframe: At Month 12

,,
InterventionParticipants (Number)
Any wound healing problemInfection related to kidney surgeryDehiscenceLymphoceleHerniaSeromaHematomaUreteral anastomotic complicationOther
Calcineurin Inhibitor (CNI) Withdrawal1623334423
CNI+MPA+ Steroid1542429011
Steroid Withdrawal932313210

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Number of Patient Survival and Graft Survival (12 Months Analysis)

(NCT00371826)
Timeframe: At Month 12

,,
InterventionParticipants (Number)
Patient SurvivalGraft Survival
Calcineurin Inhibitor (CNI) Withdrawal4949
CNI+MPA+ Steroid4645
Steroid Withdrawal3030

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Number of Patient Survival and Graft Survival (36 Months Analysis)

(NCT00371826)
Timeframe: At Month 12, 24 and 36

,,
InterventionParticipants (Number)
Month 12: Patient SurvivalMonth 12: Graft SurvivalMonth 24: Patient SurvivalMonth 24: Graft SurvivalMonth 36: Patient SurvivalMonth 36: Graft Survival
Calcineurin Inhibitor (CNI) Withdrawal232323232323
CNI+MPA+ Steroid393939393939
Steroid Withdrawal444444

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Change From Baseline in Lower and Upper GI Symptom Burden Measured by GI Symptom Rating Scale Score

This is reflected by the total score. The total score incorporates lower and upper GI elements. GSRS overall score is the mean of 15 individual GI symptom scores, each rated on a 7- point scale: 1 = no discomfort, 2= minor discomfort, 3 = mild discomfort, 4 = moderate discomfort, 5 = moderately sever discomfort, 6 = severe discomfort and 7 = very severe discomfort. Change from Baseline was calculated using ANCOVA, model includes GSRS, center and treatment group. (NCT00400400)
Timeframe: Baseline, Day 30

Interventionchange in score on a scale (Mean)
Enteric-coated Mycophenolate Sodium-0.6
Mycophenolate Mofetil-0.5

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Change From Baseline (BL) to Day 30 in the Gastrointestinal Quality of Life Index (GIQLI) Total Score and Subscale Scores

The GIQLI is a 36-item questionnaire to assess the impact of GI disease on daily life. The GIQLI has 5 different subscales (GI symptoms, emotional status, physical and social functions, and stress of medical treatment) that are rated on a 5-point scale from 0 to 4. The individual scores are summed to produce a total score of the 36 items for a total possible score of 0 to 144. Lower scores represent greater dysfunction. (NCT00400400)
Timeframe: Baseline, Day 30

,
InterventionScore on a scale (Mean)
Overall Total Score: BL ( n= 197,197)Overall Total Score : Day 30 (n= 195,192)Overall Total Score : Change from BL (n=193,192)GI Symptom: BL (n= 197, 197)GI Symptom: Day 30 (n= 195, 192)GI Symptom: Change from BL (n= 193, 192)Emotional Status: BL (n= 196, 197)Emotional Status: Day 30 (n= 195, 192)Emotional Status: Change from BL (n= 192, 192)Physical Function: BL (n= 196, 197)Physical Function: Day 30 (n= 195, 192)Physical Function: Change from BL (n= 192, 192)Social Function: BL (n= 197, 197)Social Function: Day 30 (n= 195, 192)Social Function: Change from BL (n= 193, 192)Medical treatment: BL (n= 197, 197)Medical treatment: Day 30 (n= 194, 192)Medical treatment: Change from BL (n= 192, 192)
Enteric-coated Mycophenolate Sodium94.3105.010.746.752.65.911.913.21.321.723.72.110.711.91.23.33.60.2
Mycophenolate Mofetil93.4103.510.246.651.95.411.613.11.521.523.31.910.411.61.23.43.60.3

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Change From Baseline to Day 30 in the Severity of Gastrointestinal Symptoms Overall Total Score

The Severity Score for each GI symptom for each participant was calculated based on the physician's evaluation of current GI symptoms recorded at Baseline and Day 30. For each of the 16 individual GI symptoms the severity score ranged from 0 (absent) to 3 (severe). The Overall Total Score is the Mean of severity ratings of the 16 individual symptoms. (NCT00400400)
Timeframe: Baseline, Day 30

,
InterventionScore on a scale (Mean)
Baseline (n= 198,195)Day 30 (n=193, 191)Change from Baseline to Day 30 (n=193,189)
Enteric-coated Mycophenolate Sodium0.70.4-0.3
Mycophenolate Mofetil0.60.4-0.2

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Change in Gastrointestinal Symptom Rating Scale Subscale Scores After 30 Days of Treatment

The GSRS has five subscales (reflux, diarrhea, constipation, abdominal pain, indigestion) producing a mean subscale score ranging from 1 (=no discomfort at all) to 7 (very severe discomfort). The mean score at baseline (BL), the mean score at Day 30 and the mean Change from BL to Day 30 is presented for each of the five subscales. (NCT00400400)
Timeframe: Baseline to Day 30

,
InterventionScore on a scale (Mean)
Diarrhea: Baseline (BL)Diarrhea: Day 30Diarrhea: Change from BL to Day 30Indigestion : BaselineIndigestion: Day 30Indigestion: Change from BL to Day 30Constipation: BaselineConstipation: Day 30Constipation: Change from BL to Day 30Abdominal pain: BaselineAbdominal pain: Day 30Abdominal pain: Change from BL to Day 30Reflux : BaselineReflux : Day 30Reflux : Change from BL o Day 30
Enteric-coated Mycophenolate Sodium3.32.4-0.92.82.1-0.72.21.7-0.42.31.8-0.52.21.7-0.5
Mycophenolate Mofetil3.32.5-0.82.82.4-0.52.11.8-0.32.52.0-0.52.21.7-0.5

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Number of Participants With Biopsy-proven Acute Rejection (BPAR) and Treated Acute Rejection (TAR)

"TAR was defined as an episode of acute rejection that was suspected on clinical grounds and was treated and confirmed by the investigator according to the patient's response to therapy.~BPAR was defined a treated acute rejection that was confirmed by biopsy. A graft core biopsy was performed before or within 24 hours of initiation of anti-rejection therapy and was assessed by the pathologist at the center according to the BANFF 1997 criteria." (NCT00400400)
Timeframe: 30 days

,
InterventionParticipants (Number)
BPARTAR
Enteric-coated Mycophenolate Sodium00
Mycophenolate Mofetil11

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Number of Participants With Reported Dose Changes or Interruption of Study Medication During the 30 Days of Treatment

The number of participants with reported dose changes or interruption of study medication during the 30 days of treatment.The most common dose adjustments were dose increases back to baseline levels following a decrease or interruption and decreases due to abnormal laboratory value Adverse Events (leucopenia, thrombocytopenia, neutropenia, or anemia). (NCT00400400)
Timeframe: 30 days

,
InterventionParticipants (Number)
Study drug change or interruption: YESStudy drug change or interruption: NO
Enteric-coated Mycophenolate Sodium11188
Mycophenolate Mofetil12185

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The Number of Participants Who Responded to the Conversion to Mycophenolate Sodium (EC-MPS) Therapy

Response assessed using the Gastrointestinal Symptom Rating Scale (GSRS), designed to assess common symptoms with gastrointestinal (GI) disorders. The GSRS has 5 subscales (reflux, diarrhea, constipation, abdominal pain and indigestion) producing a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort). The total score is an average of scores across all 15 items; a higher score indicates more GI symptoms. Response was defined as Day 30 improvement in the GSRS Total Score (change from baseline) of greater than or equal to 0.3. Minimum score is 1; maximum score is 7. (NCT00400400)
Timeframe: Baseline, Day 30

,
InterventionParticipants (Number)
ResponseNo Response
Enteric-coated Mycophenolate Sodium12374
Mycophenolate Mofetil10988

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Acute Graft-vs-Host-Disease (aGvHD)

Development of acute graft-vs-host-disease (aGvHD) within 6 months, for participants receiving Allo-HCT. (NCT00185614)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Auto- Then Allo-HCT7

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Event-free Survival (EFS)

"Event-free survival (EFS) as determined for all participants who received the initial Auto-HCT treatment. Event was defined as any of the following within 3 years of the participant's last infusion of Auto-HCT or Allo-HCT: relapse; death; or last follow-up if there is no data to document the participant remained alive at 3 years." (NCT00185614)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Auto-HCT onlyAuto-HCT then Allo-HCTAll Participants
Auto- Then Allo-HCT02424

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Overall Survival (OS)

Overall Survival (OS) as determined for all participants who received the initial Auto-HCT treatment, as assessed from the date of the last transplant. (NCT00185614)
Timeframe: 3 years

Interventionparticipants (Number)
Auto-HCT onlyAuto-HCT then Allo-HCTAll Participants
Auto- Then Allo-HCT14142

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Relapse Rate

Relapse rate as determined for all participants who received the initial Auto-HCT treatment. Relapse was protocol-specified as progressive disease, indicated by an increase as compared to pre-Auto-HCT baseline, of serum or urine monoclonal protein >25%; bone marrow plasmacytosis >25%; or bone lesions on skeletal survey (any increase). (NCT00185614)
Timeframe: 3 years

Interventionparticipants (Number)
Auto-HCT onlyAuto-HCT then Allo-HCTAll Participants
Auto- Then Allo-HCT22931

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Number of Participants With Any Treatment Failure

Treatment failures were defined as a composite endpoint of biopsy proven acute rejection (BPAR), graft loss, and death, loss to follow up and discontinuations from study drug treatment due to lack of efficacy or toxicity (at least one condition must be present) during the first 6 months or until final assessment. Any participants who were suspected of having acute rejection episodes had biopsies performed to prove whether a rejection had occurred. Graft loss was considered as the day the patient started dialysis and was not able to subsequently be removed or the day of graft nephrectomy. (NCT00369278)
Timeframe: 6 months

InterventionParticipants (Number)
Intensified Mycophenolate Sodium19
Standard Mycophenolate Sodium24

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Time to First Occurrence of a Mycophenolic Acid (MPA) Plasma Concentration of ≥ 40 mg*h/L

Non-compartmental MPA pharmacokinetic parameters were derived from individual plasma concentration-time profiles using WinNonLin 5.2 software. The areas under the curve were calculated by means of the linear trapezoidal rule. (NCT00369278)
Timeframe: Assessed on day 3, 10, 21, 42, 56 and 84

InterventionDays (Median)
Intensified Mycophenolate Sodium7.00
Standard Mycophenolate Sodium43.00

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Number of Participants With Single Treatment Failures

"Rates for all individual components of the primary endpoint 'treatment failure' until day 180:~Acute rejection diagnosed by biopsy (BPAR)~graft loss~death~loss to follow up~discontinuation from study drug due to lack of efficacy or toxicity (adverse events, every adverse event had to be interpreted as toxicity)~conversion to another dosing regimen (conversion to tacrolimus, prograf, etc.)" (NCT00369278)
Timeframe: 6 months

,
InterventionParticipants (Number)
Biopsy-proven acute rejectionGraft lossDeathLoss to follow-upDiscontinuation due to lack of efficacy / toxicityConversion of dosesTreated rejections
Intensified Mycophenolate Sodium210117813
Standard Mycophenolate Sodium11200151024

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Renal Function as Measured by Glomerular Filtration Rate (GFR)

"The Glomerular Filtration Rate (GFR) was calculated using the following formulas:~Cockcroft-Gault formula: calculation using the participant's age, gender, weight, and serum creatinine levels.~MDRD formula: calculation using the participant's age, gender, serum creatinine, urea nitrogen, and albumin levels." (NCT00369278)
Timeframe: 6 months

,
Interventionml/min (Mean)
MDRD formula: Baseline [n=44, 56]MDRD formula: End of study [n=61, 64]Cockcroft-Gault formula: Baseline [n=61, 65]Cockcroft-Gault formula: End of study [n=63, 65]
Intensified Mycophenolate Sodium9.041.112.152.4
Standard Mycophenolate Sodium9.440.612.047.9

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Renal Function as Measured by Serum Creatinine

(NCT00369278)
Timeframe: 6 months

,
Interventionmg/dL (Mean)
BaselineEnd of study
Intensified Mycophenolate Sodium8.02.2
Standard Mycophenolate Sodium7.82.6

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Change in Proteinuria - Uprotein/Creatinine Ratio

Urine protein/creatinine ratio after 6 months treatment with MMF or placebo. (NCT00318474)
Timeframe: Plan was to measure uprotein/creatinine ratio for 12 months on MMF or placebo, and then 12 months post-treatment. Data given after 6 months MMF/placebo.

Interventionratio (Mean)
Mycophenolate Mofetil (MMF)1.40
Placebo1.58

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Number of Participants Who Died Due to Transplant

Determine the incidence of transplant-related mortality at 6 months after UCBT (NCT00309842)
Timeframe: At Month 6

InterventionParticipants (Count of Participants)
Unrelated UCBT for Blood Cancers58

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Number of Participants Who Were Alive at 1 Year Transplant Overall Survival

Number of patients alive at 1 year after transplant. (NCT00309842)
Timeframe: at 1 year

InterventionParticipants (Count of Participants)
Unrelated UCBT for Blood Cancers130

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Number of Participants With Chronic Graft-Versus-Host Disease

"Determine the incidence of chronic GVHD at 1 year after UCBT. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria.~Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level." (NCT00309842)
Timeframe: Year 1

InterventionParticipants (Count of Participants)
Unrelated UCBT for Blood Cancers39

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Number of Participants With Neutrophil Engraftment

Determine the incidence of neutrophil engraftment at day 42 after UCBT Patients diagnosed with graft failure (failure of absolute neutrophil count ANC > 5 x 10^8/L of donor origin by day +42) (NCT00309842)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Unrelated UCBT for Blood Cancers21

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Number of Participants With Platelet Engraftment

Determine the incidence of platelet engraftment (platelet recovery >50,000/uL) at 6 months after UCBT. (NCT00309842)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Unrelated UCBT for Blood Cancers159

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Percentage Chimerism at 1 Year

Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: 1 Year

Interventionpercentage of donor cells (Mean)
Unrelated UCBT for Blood Cancers99.1

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Percentage Chimerism at 2 Years

Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: 2 Years

Interventionpercentage of donor cells (Mean)
Unrelated UCBT for Blood Cancers100

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Percentage Chimerism at 6 Months

Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: Month 6

Interventionpercentage of donor cells (Mean)
Unrelated UCBT for Blood Cancers98.1

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Percentage Chimerism on Day 100

Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: Day 100

Interventionpercentage of donor cells (Mean)
Unrelated UCBT for Blood Cancers98.1

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Percentage Chimerism on Day 21

Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: Day 21

Interventionpercentage of donor cells (Mean)
Unrelated UCBT for Blood Cancers92.6

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Number of Participants With Acute Graft-Versus-Host Disease

"Determine the incidence of grade II-IV and grade III-IV acute graft-versus-host disease (GVHD) at day 100 after UCBT. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria following Przepiorka et al, 1995, Consensus Clinical Stage and Grade of Acute GVHD.~Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level." (NCT00309842)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Grade II-IVGrade III-IV
Unrelated UCBT for Blood Cancers10649

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12 Month Disease Free Survival Probability

The percentage of patients who experience death or disease relapse by one year will be calculated and a corresponding 95% confidence interval will be constructed using the normal approximation for binomial proportions. The survival function will be estimated and plotted using the method of Kaplan and Meier. (NCT02248597)
Timeframe: At 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)51.8

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Overall Survival

The survival function will be estimated and plotted using the method of Kaplan and Meier. (NCT02248597)
Timeframe: At 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)66.7

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Progression Free Survival

Kaplan-Meier estimation of the percentage of patients who are alive without progressive disease at one year. (NCT02248597)
Timeframe: At 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)51.8

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Rate of Acute GvHD

Kaplan-Meier estimation of the rate of acute GvHD in the study population at one year with a 95% confidence interval. (NCT02248597)
Timeframe: 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)51.8

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Relapse-free Mortality

Non-relapse mortality will be defined as time from registration to death due to anything other than relapse of hematological malignancy. Patients who relapse will be treated as a competing risk. (NCT02248597)
Timeframe: At 12 months

Interventiondays (Mean)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)86.8

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Number of Participants With Graft and Patient Survivals at 6 Months

Graft survival was defined as the number of patients with no graft loss. The allograft was presumed lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient went through a graft nephrectomy, then the day of nephrectomy was the day of graft loss. Patient survival was defined as the number of patients alive with or without a functioning graft. (NCT00284934)
Timeframe: 6 months

,
InterventionParticipants (Number)
Graft survivalPatient survival
High EC-MPS4545
Standard Dose EC-MPS4747

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Number of Participants With Treatment Failure Parameters (Biopsy-Proven Acute Rejection (BPAR), Graft Loss, Death, or Loss to Follow-up) at 6 Months

A biopsy-proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB, or III based on the Banff 1997 classification.The allograft was presumed lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient went through a graft nephrectomy, then the day of nephrectomy was the day of graft loss. (NCT00284934)
Timeframe: 6 months

,
InterventionParticipants (Number)
Biopsy proven acute rejectionTreated acute rejectionGraft lossDeath
High EC-MPS0000
Standard Dose EC-MPS0000

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Renal Function Assessed by Change in Estimated Glomerular Filtration Rate(eGFR)

Change in estimated glomerular filtration rate from baseline to Month 6 calculated by using abbreviated Modification of Diet in Renal Disease (MDRD) formula. Modification of Diet in Renal Disease (MDRD) formula is: GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where -C is the serum concentration of creatinine [mg/dL], -A is patient age at sample collection date [years], -G=0.742 when gender is female, otherwise G=1, -R=1.21 when race is black, otherwise R=1. (NCT00284934)
Timeframe: Baseline and Month 6

,
InterventionmL/min/1.73m^2 (Mean)
Baseline (n= 47, 45)Month 6 (n= 45, 43)Change from Baseline - Month 6 (n= 45, 43)
High EC-MPS56.449.12.4
Standard Dose EC-MPS45.344.7-0.4

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Renal Function at 3 Months Assessed by Change in Estimated Glomerular Filtration Rate (eGFR)

Change in estimated glomerular filtration rate from baseline to Month 3 calculated by using abbreviated MDRD formula. Modification of Diet in Renal Disease (MDRD) formula is: GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where -C is the serum concentration of creatinine [mg/dL], -A is patient age at sample collection date [years], -G=0.742 when gender is female, otherwise G=1, -R=1.21 when race is black, otherwise R=1. (NCT00284934)
Timeframe: Baseline and 3 months

,
InterventionmL/min/1.73m^2 (Mean)
Baseline (n= 47, 45)Month 3 (n= 44, 43)Change from Baseline to Month 3 (n= 44, 43)
High EC-MPS46.448.62.1
Standard Dose EC-MPS45.344.6-0.4

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Acute Rejection Rate

Biopsy proven acute rejection defined by biochemical and histological changes as well as the need for temporary steroid use occurred in 1 patient in each group both of which were steroid responsive (NCT00296244)
Timeframe: 6 months post-transplant

InterventionPercentage of participants (Number)
Control Group5
Study Group5

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Incidence and Severity of HCV Recurrence Post-OLT

The incidence and severity of HCV recurrence based on Hepatitis C PCR levels and protocol liver biopsy findings were found to be similar between the 2 groups. (NCT00296244)
Timeframe: 6 months post-transplant

InterventionPercentage of participants (Number)
Control Group27
Study Group29

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Infection as an Adverse Effect of Steroids

Incidence of bacterial infection was similar in the control group as well as study group, 4 patients in both groups had infection (NCT00296244)
Timeframe: 3 months post-transplant

InterventionPercentage of participants (Number)
Control Group20
Study Group21

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New-onset Diabetes Mellitus (NODM) as Secondary Outcome

The incidence of new-onset Diabetes mellitus (NODM, based on percentage of previously non-diabetic patients who developed DM post-transplantation, was similar between the 2 groups. (NCT00296244)
Timeframe: 6 months

InterventionPercentage of participants (Number)
Control Group40
Study Group42

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Graft Survival Rate

Percentage of recipients whose liver grafts are still working at the end of 1 and 2 years. (NCT00296244)
Timeframe: 1 and 2 years

,
Interventionpercentage of participants (Number)
1-year graft survival rate2-year graft survival rate
Control Group10090
Study Group94.784

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Patient Survival Rate

Percentage of recipients who are still alive at the end of 1 and 2 years. (NCT00296244)
Timeframe: 1 and 2 years

,
InterventionPercentage of participants (Number)
1-year patient survival rate2-year patient survival rate
Control Group10090
Study Group94.784

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Gastrointestinal Symptoms Under MMF-based Immunosuppressive Therapy

Assessed by GI complications at baseline. (NCT00267150)
Timeframe: week 0

InterventionParticipants (Number)
Any complicationDiarrheaDyspepsiaNauseaAbdominal pain/bloating/fullnessOther
Enteric Coated Mycophenolate-sodium191243111

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Non-inferiority Analysis of Renal Function, Calculated by Glomerular Filtration Rate (cGFR) Using Modification of Diet in Renal Disease (MDRD) Formula

"Modification of Diet in Renal Disease (MDRD) formula is:~GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where~C is the serum concentration of creatinine [mg/dL],~A is patient age at sample collection date [years],~G=0.742 when gender is female, otherwise G=1,~R=1.21 when race is black, otherwise R=1" (NCT00251004)
Timeframe: at 12 months

InterventionmL/min/1.73m^2 (Number)
Low-dose Everolimus Group54.66
High-dose Everolimus Group51.41
Control Group52.24

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Non-inferiority Analysis on Percentage of Participants With Composite Efficacy Endpoints

The primary efficacy endpoint was the 12 month analysis of primary efficacy failure defined as a composite endpoint including treated biopsy proven acute rejection (BPAR), graft loss, death or loss to follow-up. In the definition of composite efficacy failure, loss to follow-up includes patients who did not experience treated BPAR, graft loss or death on or after day 1 and whose last day of contact was prior to day 316, the start day of the 12 month visit window. (NCT00251004)
Timeframe: 12 months

InterventionPercentage of Participants (Number)
Low-dose Everolimus Group27.1
High-dose Everolimus Group21.5
Control Group25.3

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Percentage of Participants With the Composite Incidence of Graft Loss, Death or Loss to Follow up at 12 Months Post-transplantation

"Graft loss was defined as graft loss (the allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis) and re-transplant.~A loss to follow-up patient in the composite endpoint of graft loss, death or loss to follow-up (the main secondary efficacy endpoint) was a patient who did not experience graft loss or death from day 1 and whose last day of contact was prior to study day 316." (NCT00251004)
Timeframe: 12 months

InterventionPercentage of participants (Number)
Low-dose Everolimus Group11.6
High-dose Everolimus Group9.7
Control Group9.4

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Number of Participants With Composite Efficacy Endpoints - 12 Month Analysis

The primary efficacy endpoint was the 12 month analysis of primary efficacy failure defined as a composite endpoint including treated biopsy proven acute rejection (BPAR), graft loss, death or loss to follow-up. A treated BPAR episode was defined as a biopsy graded IA, IB, IIA, IIB, or III that was treated with anti-rejection therapy. Graft loss was defined as the allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis as well as re-transplant. (NCT00251004)
Timeframe: 12 months

,,
InterventionParticipants (Number)
Composite Endpoint (n)--Death--Graft Loss--Treated BPAR--Lost to follow up
Control Group7069509
High-dose Everolimus Group60913386
Low-dose Everolimus Group758134812

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The Number of Kidney Transplant up to 12 Months.

The number of kidney transplants up to month 12. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for infection over 12 months or until they seperated from the study. (NCT00446459)
Timeframe: Enrollment to month 8 or month 12 post enrollment.

InterventionParticipants (Number)
Mycophenolate Mofetil (MMF) Single Arm Study3

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The Number of Pariticpants With a White Blood Cell Count Below 2.0 Thousand (Low) or Total IgG/IgM Titers Below Range (620-1490 mg/dL).

The number of subjects with adverse hematologic effects with MMF while on-study. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for hematologic effects up to 12 months. (NCT00446459)
Timeframe: Enrollment to month 12.

InterventionParticipant (Number)
Mycophenolate Mofetil (MMF) Single Arm Study0

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The Number of Subjects With a 10% Decrease in PRA Level at Month 8.

(NCT00446459)
Timeframe: Enrollment to month 8

InterventionParticipant (Number)
Mycophenolate Mofetil (MMF) Single Arm Study9

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The Number of Subjects With Significant Infections up to Month 12.

The number of infections while on-study up to month 12. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for infection over 12 months or until they seperated from the study. (NCT00446459)
Timeframe: From enrollment to month 12.

InterventionParticipants (Number)
Mycophenolate Mofetil (MMF) Single Arm Study4

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The Number of Transplants With a Negative Crossmatch at Transplant.

The number negative crossmatch transplants up to month 12. Positivie crossmatch transplant carries a higher risk for rejection. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for negative crossmatch transplants to 12 months. (NCT00446459)
Timeframe: Number of Transplants with a Negative Crossmatch.

InterventionParticipant (Number)
Mycophenolate Mofetil (MMF) Single Arm Study0

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Number of Biopsy Proven Rejections at 12 Months

assessed by liver biopsy using Banff International Consensus Schema (NCT00206076)
Timeframe: 12 months

Interventionparticipants (Number)
MMF, CNI Discontinued0
MMF; CNI Decreased0

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Number of Participants With Adverse Events Including Infections at 12 Months

(NCT00206076)
Timeframe: 12 months

Interventionparticipants (Number)
MMF, CNI Discontinued2
MMF; CNI Decreased1

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Patient and Graft Survival at 12 Months

(NCT00206076)
Timeframe: 12 months

Interventionparticipants (Number)
CNI Discontinued6
CNI Reduction9

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Number of Participants Who Experienced Adverse Events, Serious Adverse Events and Death

Participants with adverse events (serious plus non-serious), serious adverse events and death were reported. (NCT00956293)
Timeframe: Months 6, 12, 24, 36, 48 and 60

,
InterventionParticipants (Number)
Adverse events (serious and non-serious)Serious adverse eventsDeaths
Control Group21110
Everolimus Group50280

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Assessment of GFR by the Cockcroft-Gault Method (LOCF)

the GFR was also calculated using the Cockcroft-Gault method (Cockcroft and Gault 1976) and the Modification of Diet in Renal Disease (MDRD) method (Levey et al., 1999, Rodrigo et al., 2003; Pierrat et al., 2003).Cockcroft-Gault formula For men: GFR= (140-Age)X Body Weight[kg]/72X Serum Creatinine[mg/dl] For women: GFR= 0,85x(140-Age) x Body Weight[kg]/72x Serum Creatinine [mg/dl] The Last Observation Carried Forward (LOCF) imputation technique was used for this analysis. (NCT00965094)
Timeframe: 9 months

InterventionmL/min (Mean)
Everolimus72.6
Reference Therapy72.7

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Renal Function Assessed as Glomerula Filtration Rate (GFR) - Nankivell Method - 9 Months After Renal Transplantation (LOCF)

The glomerular filtration rate (GFR) is the best clinical estimate of renal function in health and disease, and correlates well with the clinical severity of renal function disturbances. The GFR, calculated according to the Nankivell formula, was used as the primary outcome measure in this study. This equation has been validated in renal transplant patients against the true GFR measured by a radionuclide method and has been confirmed as a very accurate method to calculate the GFR in this specific population (Gaspari et al., 2004)GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)² + C Scr = serum creatinine concentration expressed in mmol/L. BW = body weight in kg, Surea = serum urea in mmol/L and Height is expressed in meters.The Last Observation Carried Forward (LOCF) imputation technique was used for this analysis. (NCT00965094)
Timeframe: 9 months

InterventionmL/min/1.73m^2 (Mean)
Everolimus15.0
Reference Therapy16.6

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Change in Renal Function (Creatinine Slope)

X(slope)=(1/value of creatinine). (NCT00965094)
Timeframe: 3 months, 5 months, 7 months, 9 months

,
Interventionmg/dl per month (Mean)
(ITT) Baseline 2: Month 3 N=15, 15(ITT) Month 3 (1st week) N=15, 4(ITT) Month 3 (2nd week) N=15, 4(ITT) Month 3 (3rd week) N=13, 2(ITT) Month 3 (4th week) N=12, 0*(ITT) Month 5 N=14, 15(ITT) Month 7 N=14, 14(ITT) Month 9 N=11, 15(PP) Baseline 2: Month 3 N=11, 15(PP) Month 3 (first week) N=11, 4(PP) Month 3 (second week) N=11, 4(PP) Month 3 (third week) N=10, 2(PP) Month 3 (fourth week) N=9, 0*(PP) Month 5 N=11, 15(PP) Month 7 N=11, 14(PP) Month 9 N=11, 15
Everolimus0.70.70.80.70.80.80.70.80.70.70.80.70.80.80.80.8
Reference Therapy0.70.80.90.9NA0.70.70.80.70.80.90.9NA0.70.70.8

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Participants Who Had Occurrence of Biopsy Proven Acute Rejection, Graft Loss or Death.

Biopsy-proven acute rejection was defined as a biopsy gradeed IA, IB, IIA, IIB, or III. The allograft was presumed to be lost if the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss. (NCT00965094)
Timeframe: 9 months

,
InterventionParticipants (Number)
NoYes
Everolimus141
Reference Therapy141

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Participants Who Had Occurrence of Treatment Failure.

Treatment failure was defined as a composite endpoint of biopsy-proven acute rejection, graft loss, death, loss to follow up, discontinuation due to lack of efficacy or toxicity or conversion to another regimen. (NCT00965094)
Timeframe: 9 months

,
InterventionParticipants (Number)
NoYes
Everolimus105
Reference Therapy141

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Mean Creatinine Clearance Rate

Creatinine clearance is a measure of kidney function. Creatinine clearance rate is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Creatinine clearance can be measured directly or estimated using established formulas. For this study, the creatinine clearance rate was calculated using the Nankivell formula. Normal values for healthy, young males are in the range of 100-135 ml/min and for females 90-125 ml/min. A low creatinine clearance indicates poor kidney function. (NCT00195273)
Timeframe: 12 months

Interventionml/min (Mean)
Sirolimus56.800
Cyclosporine (CsA)53.449

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Mean Creatinine Clearance Rate - 3 Months

Creatinine clearance is a measure of kidney function. Creatinine clearance rate is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Creatinine clearance can be measured directly or estimated using established formulas. For this study, the creatinine clearance rate was calculated using the Nankivell formula. Normal values for healthy, young males are in the range of 100-135 ml/min and for females 90-125 ml/min. A low creatinine clearance indicates poor kidney function. (NCT00195273)
Timeframe: 3 months

Interventionml/min (Mean)
Sirolimus55.200
Cyclosporine (CsA)51.878

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Number of Patients With Acute Rejection

The diagnosis of acute rejection was made via kidney biopsy (Banff criteria). The Banff criteria are standardized diagnostic categories based on histological assessments (e.g., cell types and distributions). Biopsy was performed before initiation of anti-rejection therapy, or at least within 24 hours of the start of therapy. (NCT00195273)
Timeframe: 3 and 12 months

,
Interventionpatients (Number)
3 months12 months
Cyclosporine (CsA)910
Sirolimus35

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Patient and Graft Survival

Graft survival is measured by graft loss which is defined as removal of the transplant. (NCT00195273)
Timeframe: 12 months

,
Interventionpatients (Number)
Patient survivalGraft loss
Cyclosporine (CsA)293
Sirolimus310

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Number of Participants With Adverse Events (AEs)

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. (NCT01085097)
Timeframe: Baseline up to Week 28

Interventionparticipants (Number)
Placebo14
Laquinimod 0.5 mg15
Laquinimod 1 mg15

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Percent Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 24

Estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease (MDRD) formula. (NCT01085097)
Timeframe: Baseline, Week 24

Interventionpercent change (Mean)
Placebo12.1
Laquinimod 0.5 mg18.0
Laquinimod 1 mg24.3

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Incidence of Hepatotoxicity as Measured by Bilirubin

100-day incidence of hepatotoxicity will be compared using a Gray test. Median and range of largest total bilirubin (mg/dL), (NCT01951885)
Timeframe: Up to day +100

Interventionmg/dL (Median)
Group A (Tacrolimus, Methotrexate)1.5
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)1.1

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Incidence of Hepatotoxicity as Measured by Veno-occlusive Disease (VOD)

100-day incidence of hepatotoxicity will be compared using the Gray test. Percentage of patients with VOD and 95% confidence interval (NCT01951885)
Timeframe: Up to day +100

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)8
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)2

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Incidence of Infection

100-day incidence of infection will be compared using a the Gray test. (NCT01951885)
Timeframe: Up to day +100

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)63
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)53

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Incidence of Pulmonary Toxicity Measured by Pulmonary Edema

180-day incidence of pulmonary toxicity will be compared using the Gray test. Percentage of patients with pulmonary edema and 95% confidence interval (NCT01951885)
Timeframe: Up to day +180

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)14
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)4

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Incidence of Pulmonary Toxicity Measured by Pulmonary Hemorrhage

180-day incidence of pulmonary toxicity will be compared using the Gray test. Percentage of patients with pulmonary hemorrhage and 95% confidence interval (NCT01951885)
Timeframe: Up to day +180

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)2
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)2

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Incidence of Pulmonary Toxicity Measured by Respiratory Failure

180-day incidence of pulmonary toxicity will be compared using the Gray test. Percentage of patients with respiratory failure and 95% confidence interval (NCT01951885)
Timeframe: Up to day +180

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)9
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)6

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Length of Hospitalization

Hospital stay will be compared between patients in groups A and B using the Wilcoxon rank sum test. (NCT01951885)
Timeframe: Date of transplant to date of discharge, assessed up to 1 year

Interventiondays (Median)
Group A (Tacrolimus, Methotrexate)31
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)27

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Length of Time on Continuous Infusion Narcotics

Start and stop dates for the need of continuous IV infusion of narcotics for severe mucositis pain will be recorded. Time on IV infusion will be compared between patients in groups A and B using the Wilcoxon rank sum test. (NCT01951885)
Timeframe: up to +28 day

Interventiondays (Median)
Group A (Tacrolimus, Methotrexate)10
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)9

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Overall Survival

Estimated using the Kaplan-Meier method and compared between the 2 groups using the log-rank test. (NCT01951885)
Timeframe: Up to 1 year

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)71
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)72

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Percentage of Participants Using Total Parenteral Nutrition (TPN) Within 100 Days

TPN use will be compared using the Chi-square test. (NCT01951885)
Timeframe: Up to day 100

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)41
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)38

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Percentage of Severe (Grade 3-4) Mucositis Graded According to the World Health Organization (WHO) Grading Scale

"Participants (percentage of) will be graded three times per week through day 28 of the study. Incidence will be compared through Wilcoxon rank sum tests.~The WHO grading scale for mucositis is scaled depending on the severity of mucositis symptoms, with a lower stage associated with a better outcome and greater stages associated with worse outcomes. The staging is as follows:~Stage 0: None Stage 1 (mild): Oral soreness, erythema Stage 2 (moderate): Oral erythema, ulcers, solid diet tolerated Stage 3 (severe): Oral ulcers, liquid diet only Stage 4 (life-threatening): Oral alimentation impossible" (NCT01951885)
Timeframe: Up to day 28

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)81.6
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)57.4

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Progression-free Survival

Estimated using the Kaplan-Meier method and compared between the 2 groups using the log-rank test. (NCT01951885)
Timeframe: Up to 1 year

Interventionpercentage of participants (Number)
Group A (Tacrolimus, Methotrexate)59
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)68

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Time to Neutrophil Engraftment

The number of days to reach a neutrophil count of greater than or equal to 500/ul for three consecutive laboratory values obtained on different days. The day of engraftment will be the first day of the three consecutive laboratory values. (NCT01951885)
Timeframe: Up to 28 days

Interventiondays (Median)
Group A (Tacrolimus, Methotrexate)17
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)15

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Time to Platelet Engraftment

The number of days to reach a platelet count of greater than or equal to 20,000/ul for three consecutive laboratory values obtained on different days, independent of platelet transfusions the prior 7 days. The day of engraftment will be the first day of the three consecutive laboratory values. For cases of delayed engraftment beyond 28 days, results will be recorded once the participant engrafts. (NCT01951885)
Timeframe: The date the participant engrafts, up to 28 days

Interventiondays (Median)
Group A (Tacrolimus, Methotrexate)27
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)23

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Cumulative Incidence of Participants With Acute GVHD

"Acute GVHD by grade will be estimated using cumulative incidence methods and compared using the Gray test.~A lower clinical grade and/or stage of GVHD corresponds to a better participant outcome and a higher grade corresponds to a worse participant outcome. Grading is as follows:~Grade 0: No stage 1-4 of any organ Grade 1: Stage 1-2 skin without liver, upper GI, or lower GI involvement. Grade 2: Stage 3 rash and/or stage 1 liver and/or stage 1 upper GI and/or stage 1 lower GI.~Grade 3: Stage 2-3 liver and/or stage 2-3 lower GI, with stage 0-3 skin and/or stage 0-1 upper GI.~Grade 4: Stage 4 skin, liver, or lower GI involvement, with stage 0-1 upper GI.~A greater stage of GVHD involves worsening end-organ involvement and worse participant outcomes based on the skin, liver, lower GI, and upper GI." (NCT01951885)
Timeframe: Day 7- Day 100

,
Interventionpercentage of participants (Number)
Grade 1-4Grade 2-4Grade 3-4
Group A (Tacrolimus, Methotrexate)37274
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)472813

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Incidence of Chronic GVHD

"Chronic GVHD will be graded as mild, moderate, or severe based on the National Institutes of Health Consensus Development Project. The type and duration of immunosuppressive treatment given for cGVHD will be recorded. Mild grades are associated with a better participant outcome and severe corresponds to a worse participant outcome. Grading is as follows:~Mild: 1 or 2 organs involved with no more than score 1 plus Lung score 0~Moderate: 3 or more organs involved with no more than score 1 OR At least 1 organ (not lung) with a score of 2 OR Lung score 1~Severe: At least 1 organ with a score of 3 OR Lung score of 2 or 3~Organ scores can range from 0 to 3, with 0 being no symptoms on the target organ and a better participant outcome while a score of 3 correlates to severe symptoms on the target organ and worse participant outcomes.~Potential target organs include: skin, mouth, eyes, GI tract, liver, lungs, joint /fascia, and genital tract" (NCT01951885)
Timeframe: at 12 months

,
Interventionpercentage of participants (Number)
any chronic GVHDmoderate-severe chronic GVHD
Group A (Tacrolimus, Methotrexate)2520
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)3623

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Incidence of Chronic GVHD

"Chronic GVHD will be graded as mild, moderate, or severe based on the National Institutes of Health Consensus Development Project. The type and duration of immunosuppressive treatment given for cGVHD will be recorded. Mild grades are associated with a better participant outcome and severe corresponds to a worse participant outcome. Grading is as follows:~Mild: 1 or 2 organs involved with no more than score 1 plus Lung score 0~Moderate: 3 or more organs involved with no more than score 1 OR At least 1 organ (not lung) with a score of 2 OR Lung score 1~Severe: At least 1 organ with a score of 3 OR Lung score of 2 or 3~Organ scores can range from 0 to 3, with 0 being no symptoms on the target organ and a better participant outcome while a score of 3 correlates to severe symptoms on the target organ and worse participant outcomes.~Potential target organs include: skin, mouth, eyes, GI tract, liver, lungs, joint /fascia, and genital tract" (NCT01951885)
Timeframe: at 6 months

,
Interventionpercentage of participants (Number)
any chronic GVHDmoderate-severe chronic GVHD
Group A (Tacrolimus, Methotrexate)1612
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)1511

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Incidence of Hepatotoxicity as Measured by Elevated Liver Enzymes

100-day incidence of hepatotoxicity will be compared using a Gray test. Percentage of patients with elevated Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), and alkaline phosphatase and a 95% confidence interval (NCT01951885)
Timeframe: Up to day +100

,
Interventionpercentage of participants (Number)
ASTALTAlkaline Phosphatase
Group A (Tacrolimus, Methotrexate)29332
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)15282

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Incidence of Nephrotoxicity

100-day incidence of nephrotoxicity will be compared using a Chi-squared test. Percentage of patients requiring dialysis and those with elevated creatinine using a 95% confidence interval (NCT01951885)
Timeframe: Up to day +100

,
Interventionpercentage of participants (Number)
Participants requiring dialysisParticipants with elevated creatinine
Group A (Tacrolimus, Methotrexate)1227
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil)42

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Change From Baseline in Left Ventricular Mass Index (LVMI)

Left ventricular hypertrophy grade was assessed by echocardiogram where the left ventricular mass index was calculated. The presence of LVM was defined as > 49.2 g/m^2.7 in men and >46.7 g/m^2.7 in women. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, Month 24

Interventiong/m^2.7 (Mean)
Tacrolimus-6.071
Everolimus-4.008

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Percentage of Participants With Major Cardiovascular Events (MACE)

The percentage of participants who experienced MACE were reported. MACE included acute myocardial infarction, insertion or replacement of implantable defibrillator, peripheral vascular disorders, congestive heart failure, coronary artery bypass, other events, percutaneous coronary intervention and stroke. (NCT01169701)
Timeframe: Month 24

InterventionPercentage of participants (Number)
Tacrolimus0.00
Everolimus0.00

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Change From Baseline in Cardiovascular Biomarkers, C-reactive Protein (CRP)

Blood samples were collected to analyze CRP. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24

,
Interventionmg/dl (Mean)
Month 6 (n=27,30)Month 24 (n=24,24)
Everolimus0.326-0.040
Tacrolimus0.5120.100

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Change From Baseline in Cardiovascular Biomarkers: Troponin I and Collagen Type 1 C-telopeptide (ICTP)

Blood samples were collected to analyze Troponin I and collagen type 1 C-telopeptide (ICTP). A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24

,
Interventionng/ml (Mean)
Troponin 1, Month 6 (n=27,30)Troponin 1, Month 24 (n=24,24)ICTP, Month 6 (n=27,30)ICTP, Month 24 (n=24,24)
Everolimus-0.006-0.007-0.195-0.125
Tacrolimus0.0000.0030.049-0.035

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Change From Baseline in Mean 24 Hour Systolic and Diastolic Blood Pressure

Blood pressure was measured using ambulatory blood pressure monitoring (ABPM). A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, Month 6, month 12, month 24

,
InterventionmmHg (Mean)
Month 6 (n=31,29)Month 12 (n=28,24)Month 24 (n=29,24)
Everolimus3.22.72.0
Tacrolimus-0.62.12.2

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Change From Baseline in the Cardiovascular Biomarker, Glycosylated Hemoglobin (HbA1c)

Blood samples were collected to analyze HbA1c. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24

,
InterventionPercentage of HbA1c (Mean)
Month 6 (n=20,22)Month12 (n=22,20)
Everolimus0.1590.185
Tacrolimus0.0150.045

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Change From Baseline in the Cardiovascular Biomarker, Myeloperoxidase (MPO)

Blood samples were collected to analyze MPO. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24

,
InterventionU/mL (Mean)
Month 6 (n=27,30)Month 24 (n=24,24)
Everolimus-0.093-0.642
Tacrolimus0.433-0.329

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Change From Baseline in the Cardiovascular Biomarker, N-terminal Pro-brain Natriuretic Peptide Fraction (NT-proBNP)

Blood samples were collected to analyze NT-proBNP. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24

,
Interventionpg/mL (Mean)
Month 6 (n=27,30)Month 24 (n=24,24)
Everolimus-79.10-193.3
Tacrolimus21.604-80.20

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Change From Baseline in the Cardiovascular Biomarker, Type 1 Procollagen Amino-terminal-propeptide (PINP)

Blood samples were collected to analyze PCR. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24

,
Interventionug/l (Mean)
Month 6 (n=27,30)Month 24 (n=24,24)
Everolimus-33.36-28.17
Tacrolimus-13.82-13.65

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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR), Graft Loss, Death and Lost to Follow up

The incidence of BPAR, graft loss, death and lost to follow-up events was calculated using relative frequency. (NCT01169701)
Timeframe: Month 24

,
InterventionPercentage of participants (Number)
BPARGraft lossDeathsLost to follow-up
Everolimus0.000.000.000.00
Tacrolimus0.000.000.003.13

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Pulse Wave Velocity (PWV)

Utilizing the SphygmoCor Device, ECG leads placed at the carotid and femoral arteries provided the measure of the pulse wave at that particular arterial location. The distance between the two vascular beds divided by the pulse wave time shift provided a measure of the pulse wave velocity. (NCT01169701)
Timeframe: Month 6, month 24

,
Interventionm/sec (Mean)
Month 6 (n=31,30)Month 24 (n=28,25)
Everolimus7.407.06
Tacrolimus7.017.58

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Renal Function as Measured by Creatinine Clearance

Creatinine clearance was calculated using the Cockroft-Gault formula. (NCT01169701)
Timeframe: Month 6, month 12, month 24

,
Interventionmg/min (Mean)
Month 6 (n=29,25)Month 12 (n=28,25)Month 24 (n=28,24)
Everolimus76.61873.36372.910
Tacrolimus64.84165.03766.933

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Renal Function as Measured by Estimated Glomerular Filtration Rate (eGFR)

Estimated GFR was caluclated using the modification of diet in renal disease (MDRD) formula. (NCT01169701)
Timeframe: Month 6, month 12, month 24

,
InterventionmL/min/1.73m^2 (Mean)
Month 6 (n=33,29)Month 12 (n=32,28)Month 24 (n=31,26)
Everolimus63.78161.22560.779
Tacrolimus55.64857.75757.727

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Renal Function Measured by Serum Creatinine

Serum samples were collected to analyze serum creatinine. (NCT01169701)
Timeframe: Month 6, month 12, month 24

,
Interventionmg/dl (Mean)
Month 6 (n=33,29)Month 12 (n=32,28)Month 24 (n= 31,26)
Everolimus1.2341.2561.260
Tacrolimus1.2321.2311.217

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6-month Acute Antibody-mediated Rejection Rate (AMR)

A diagnosis of AMR was based on the 2013 international Banff Classification Criteria and is defined as the presence of circulating donor-specific antibody (DSA) and either: 1) peritubular capillary staining of C4d and at least one of the following: peritubular capillaritis (ptc) score>0, glomerulitis (g) score>0, acute thrombotic microangiopathy (TMA) in the absence of any other cause, or other features consistent with AMR (endothelial injury, fibrin thrombi, microinfarctions, interstitial hemorrhage), or 2) absence of capillary staining of C4d and the presence of ptc>0 and g>0 or ptc>0 or g>0 and acute TMA, in the absence of any other cause of TMA. (NCT00275509)
Timeframe: Up to 6 months

InterventionParticipants (Count of Participants)
Tymoglobulin17
Daclizumab16

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6-month Acute Cellular-mediated Rejection Rate (CMR)

Per 2007 international Banff Classification Criteria, CMR 1A was diagnosed on biopsies displaying significant interstitial infiltration (>25% of parenchyma affected, i2 or i3) and foci of moderate tubulitis (t2). CMR IB was diagnosed in cases with significant interstitial infiltration (>25% of parenchyma affected, i2 or i3) and foci of severe tubulitis (t3). CMR IIA were cases with mild-to-moderate intimal arteritis (v1), while CMR IIB were those with severe intimal arteritis comprising >25% of the luminal area (v2). CMR III were those cases with transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation (v3). (NCT00275509)
Timeframe: Up to 6 months

InterventionParticipants (Count of Participants)
Tymoglobulin14
Daclizumab20

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6-month Cumulative Rejection Incidence (Either CMR, AMR or Both)

Biopsy shows evidence of either AMR or CMR or evidence both. (NCT00275509)
Timeframe: Up to 6 months

InterventionParticipants (Count of Participants)
Tymoglobulin21
Daclizumab23

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Arthritis Complete Response

Complete response at three months (This is defined as NCT00594932)
Timeframe: 3 months

Interventionparticipants (Number)
Patients Who Received MMF for the First 3 Months4
Patients Who Received Placebo for the First 3 Months0

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Major and Partial Clinical Response

Those meeting the Criteria for Major or Complete Clinical Response as listed in the preceding endpoints or who meet criteria for partial response defined as at least a 25% decrease in tender and swollen joint count and improvement of at least one severity rating for arthritis as at least one level on the British Isles Lupus Assessment Group Index. (NCT00594932)
Timeframe: 3 Months

Interventionparticipants (Number)
Patients Who Received MMF for the First 3 Months9
Patients Who Received Placebo for the First 3 Months5

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Major Arthritis Response

This is defined as at least a 50% reduction in tender + swollen joint counts and severity rated as mild as defined by the British Isles Lupus Assessment Group Index (NCT00594932)
Timeframe: 3 months

Interventionparticipants (Number)
Patients Who Received MMF for the First 3 Months5
Patients Who Received Placebo for the First 3 Months0

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Area Under the Plasma Concentration-time Curve (AUC(0-6h)) of Mycophenolate Mofetil

Area under the plasma concentration-time curve during a dosage interval at steady state (calculated using the trapezoidal rule, from t = 0 to t = 6 hours). (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration

Interventionhour* µg /ml (Mean)
CellCept33.523
Myfenax31.100

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Area Under the Plasma Concentration-time Curve (AUC(0-tau)) of Mycophenolate Mofetil

For participants with a 0-12h profile: Area under the plasma concentration-time curve during a dosage interval at steady state (calculated using the trapezoidal rule, from t = 0 to t = 12 hours). For participants with a 0-6h profile: AUC(0-tau) was calculated based on AUC(0-6h) using the extrapolation formula according to Fleming. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration

Interventionhour* µg /ml (Mean)
CellCept49.846
Myfenax48.255

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Degree of Fluctuation of the Concentration Levels of Mycophenolate Mofetil Over One Dosing Interval (PTF)

PTF was calculated as: (Cmax-Cmin)/(AUCt/t)*100 (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration

Interventionpercentage of AUC for a dosing interval (Mean)
CellCept351.05
Myfenax323.67

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Maximum Observed Plasma Concentration (Cmax) of Mycophenolate Mofetil

Cmax was directly obtained from measured values of plasma concentrations. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration

Interventionµg /ml (Mean)
CellCept16.189
Myfenax14.308

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Minimum Observed Plasma Concentration (Cmin) of Mycophenolate Mofetil

Cmin was directly obtained from measured values of plasma concentrations. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration

Interventionµg /ml (Mean)
CellCept1.584
Myfenax1.567

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Plasma Concentrations of Mycophenolate Mofetil in Pre-Administration Samples (Cpd)

Cpd was directly obtained from measured values of plasma concentrations. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration

Interventionµg /ml (Mean)
CellCept2.693
Myfenax3.001

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Time Corresponding to Occurrence of Cmax (Tmax) of Mycophenolate Mofetil

Tmax was directly obtained from measured values. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration

Interventionhours (Mean)
CellCept1.119
Myfenax1.344

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Summary of Participants With Adverse Events

"Summary of adverse events across three study time periods. The on-treatment time frame spanned the time during which study drug was administered. Relation to study drug was assessed by the investigator.~The Adverse Event count includes serious and non-serious AEs. A serious AE (SAE) was any event that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity or congenital anomaly/birth defect or was an important medical event could have jeopardized the patient's safety or required medical or surgical intervention to prevent one of the outcomes listed above.~Severity was measured on a three-point scale: mild, moderate, severe." (NCT00991510)
Timeframe: Day 1 up to Day 112

,,
Interventionparticipants (Number)
Adverse EventsRelated adverse eventsSevere adverse eventsAdverse events leading to discontinuationSerious adverse eventsAdverse events leading to death
CellCept1530210
Myfenax1770110
Overall2690310

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Number of Non-Relapse Mortalities (NRM)

Number of patients who expired without disease progression/relapse. (NCT01252667)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)1
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)0
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)1

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Number of Participants Surviving Overall

Number of patients surviving overall post-transplant. (NCT01252667)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)2
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)1
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)22

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Number of Participants Surviving Progression-free.

Number of patients surviving without progressive disease post-transplant. Progression is defined as the presence of >5% aberrant blasts by morphology on a marrow aspirate or the presence of circulating blasts in the peripheral blood. (NCT01252667)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)2
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)1
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)21

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Number of Participants Who Graft Rejected.

Number of patients who graft rejected post-transplant. Graft rejection is defined as <5% donor peripheral blood T cells (CD3+). (NCT01252667)
Timeframe: 1 Year post-transplant.

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)0
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)0

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Number of Participants With Minimal Residual/Recurring Disease (MRD) Post-transplant

Number of patients with MRD post-transplant, detected in the bone marrow as cytogenetic abnormalities or <5% monoclonal blasts by flow cytometry. (NCT01252667)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)2
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)0
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)6

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Part 1: Number of Participants With Dose Limiting Toxicities (DLT)

"The primary objective of part 1 is to determined the highest dose of clofarabine that can be tolerated safely in conjunction with nonmyeloablative transplant. If three patients successfully transplant without DLT, dose escalation occurs. If one of those three patients experiences DLT an additional three patients will be treated using the same dose. If a second DLT is observed in the first 3-6 patients, or if three patients are successfully transplanted without DLT at the highest dose the study will proceed to Part 2 using the maximum tolerated dose.~A Clofarabine related dose-limiting toxicity is defined as a grade 4 toxicity involving the lungs, heart, liver (not resolving in 48 hours), kidneys (not resolving in 48 hours), gastrointestinal tract, and/or central nervous system." (NCT01252667)
Timeframe: 14 days post-transplant

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)0
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)0

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Part 2: Number of Participants With Relapsed Disease

Number of high risk patients with relapsed disease after receiving the maximum dose of clofarabine. Relapse is defined as the presence of >5% aberrant blasts by morphology on a marrow aspirate or the presence of circulating blasts in the peripheral blood. (NCT01252667)
Timeframe: 6 months post-transplant

InterventionParticipants (Count of Participants)
Part 1 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)0
Part 2 - Dose 1 (30 mg/m^2 Clofarabine)0
Part 2 - Dose 2 (40 mg/m^2 Clofarabine)0
Part 2 - Dose 3 (50 mg/m^2 Clofarabine)3

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Pharmacokinetics (PK) of Clofarabine

Pharmacokinetic measurement of the volume of plasma from which clofarabine is completely removed per unit time. Clearance normalized to actual body weight. Pharmacokinetic analyses were performed on only a subset of patients and no aggregate calculations were made using the data. (NCT01252667)
Timeframe: Blood was drawn on day -6 before the first clofarabine dosing, at the end of the infusion, and at 3, 4, 5, 6 and 24 hours after the start of infusion. Only pre-dose samples were drawn on days -5, -4, and -3.

InterventionL/h/kg (Number)
ID 1ID 2
Part 1 - Dose 2 (40 mg/m^2 Clofarabine)0.530.37

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Pharmacokinetics (PK) of Clofarabine

Pharmacokinetic measurement of the volume of plasma from which clofarabine is completely removed per unit time. Clearance normalized to actual body weight. Pharmacokinetic analyses were performed on only a subset of patients and no aggregate calculations were made using the data. (NCT01252667)
Timeframe: Blood was drawn on day -6 before the first clofarabine dosing, at the end of the infusion, and at 3, 4, 5, 6 and 24 hours after the start of infusion. Only pre-dose samples were drawn on days -5, -4, and -3.

InterventionL/h/kg (Number)
ID 4ID 5ID 6
Part 1 - Dose 3 (50 mg/m^2 Clofarabine)0.290.400.50

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Estimated Glomerular Filtration Rate (eGFR)

Renal function was assessed by estimated Glomerular Filtration Rate (eGFR) using the Modification of Diet in Renal Disease (MDRD) formula. MDRD formula: GFR [mL/min/1.73m˄2] = 186.3*(C˄-1.154)*(A˄-0.203)*G*R. DEFINITIONS: C = serum concentration of creatinine [mg/dL]; A = age [years]; G = 0.742 when gender is female, otherwise G = 1; R = 1.21 when race is black, otherwise R = 1 (NCT01025817)
Timeframe: 12 Months

InterventionmL/min/1.73m˄2 (Mean)
Everolimus and Low Dose Tacrolimus63.14
Mycophenolate Mofetil and Standard Dose Tacrolimus63.06

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Number of Participants With Incidence of Adverse Events, Serious Adverse Events, and Tacrolimus-associated Adverse Events

Incidence of adverse events, serious adverse events, and tacrolimus-associated adverse events by System Organ Class (NCT01025817)
Timeframe: 12 Months

,
InterventionParticipants (Number)
Any system organ classMetabolism and nutrition disordersGastrointestinal disordersInjury, poisoning and procedural complicationsGeneral disorders &administration site conditionsInfections and infestationsInvestigationsRenal and urinary disordersVascular disordersBlood and lymphatic system disordersNervous system disordersRespiratory, thoracic and mediastinal disordersMusculoskeletal and connective tissue disordersSkin and subcutaneous tissue disordersPsychiatric disordersReproductive system and breast disordersCardiac disordersEye disordersImmune system disordersEndocrine disordersNeoplasms benign,malignant,other incl cysts/polypsEar and labyrinth disordersHepatobiliary disordersSurgical and medical proceduresCongenital, familial and genetic disordersSocial circumstances
Everolimus and Low Dose Tacrolimus3032662332231991841501411311301251221101099656512613121076200
Mycophenolate Mofetil and Standard Dose Tacrolimus30226324720217719614316012116315013411410810640473611815113061

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Number of Participants With Incidence of CMV (Viremia, Syndrome and Disease)

Participants with incidence of CMV (viremia, syndrome and disease). CMV is cytomegalovirus. (NCT01025817)
Timeframe: 12 Months

,
InterventionParticipants (Number)
CMV syndrome eventLab evidence of CMV ViremiaCMV Disease
Everolimus and Low Dose Tacrolimus972
Mycophenolate Mofetil and Standard Dose Tacrolimus13108

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Number of Participants With Incidence of Composite Efficacy Failure

Efficacy failure rate used the composite endpoint of: (1) treated biopsy-proven acute rejection (BPAR)*, (2) graft loss**, (3) participant death or(4) loss to follow-up. *A treated BPAR was defined as a biopsy graded IA, IB, IIA, IIB, or III and which was treated with anti-rejection therapy. **Graft loss is defined as when the allograft was presumed lost on the day the participant started dialysis and was not able to subsequently be removed from dialysis. (NCT01025817)
Timeframe: 12 Months

,
InterventionParticipants (Number)
Composite EndpointTreated Biopsy-proven Acute rejection (BPAR)Graft LossDeathLoss to follow up
Everolimus and Low Dose Tacrolimus7659469
Mycophenolate Mofetil and Standard Dose Tacrolimus623412517

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Number of Participants With Incidence of New Onset of Diabetes Mellitus

Incidence of new onset diabetes mellitus defined as non-diabetic patients before transplantation, who are receiving glucose lowering treatment for more than 30 days post-transplant, or with a random plasma glucose ≥200 mg dL (11.1 mmol/L) with 2 fasting plasma glucose values ≥126 mg/dL (7 mmol/L) (NCT01025817)
Timeframe: 12 Months

,
InterventionParticipants (Number)
Any New Onset DiabetesrandomGlucose≥200mg/dL w/2 fastingGlucose≥126mg/dLConcomitant Diabetes medicine for 30 days or more
Everolimus and Low Dose Tacrolimus251513
Mycophenolate Mofetil and Standard Dose Tacrolimus221214

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Number of Participants With Incidence of Proteinuria Events

Number of participants with Incidence of proteinuria events indicating chronic kidney disease (NCT01025817)
Timeframe: Baseline and 12 Months

,
InterventionParticipants (Number)
Baseline: Proteinuria (>=300 mg/g)Month 12, Day 316-450: Proteinuria (>=300 mg/g)
Everolimus and Low Dose Tacrolimus24336
Mycophenolate Mofetil and Standard Dose Tacrolimus25035

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Number of Participants With Incidence Rates of BKV Viremia, BKV Viruria, or BKV Nephropathy

Participants with Incidence of BKV (viremia, viruria, or nephropathy). BKV is Polyomavirus type BK. (NCT01025817)
Timeframe: 12 Months

,
InterventionParticipants (Number)
Lab evidence of BKV ViremiaLab evidence of BKV ViruriaBKV Disease (Nephropathy)
Everolimus and Low Dose Tacrolimus19195
Mycophenolate Mofetil and Standard Dose Tacrolimus27155

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To Compare 2-year Overall Survival of Patients With Lymphoma Undergoing RIC SCT Between Those Receiving Tacrolimus/Sirolimus/Methotrexate and Those Receiving Tacrolimus/Methotrexate or Cyclosporine/Mycophenolate Mofetil

(NCT00928018)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Sirolimus-Containing Regimen70
Sirolimus-Free Regimen68

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To Compare 2-year Progression-free Survival Between the Two Treatment Arms

(NCT00928018)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Sirolimus-Containing Regimen61
Sirolimus-Free Regimen58

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To Compare the 2-year Cumulative Incidence of Chronic GVHD Between the Two Treatment Arms.

(NCT00928018)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Sirolimus-Containing Regimen59
Sirolimus-Free Regimen63

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To Compare the 180-day Cumulative Incidence of Grades II-IV and Grades III-IV Acute GVHD Between the Two Treatment Arms

(NCT00928018)
Timeframe: 6 months

,
Interventionpercentage of participants (Number)
Grade II-IV aGVHDGrade III-IV aGVHD
Sirolimus-Containing Regimen93
Sirolimus-Free Regimen254

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To Compare the 2-year Cumulative Incidences of Disease Progression and of Non-relapse Mortality Between the Two Treatment Arms

(NCT00928018)
Timeframe: 2 years

,
Interventionpercentage of participants (Number)
Cumulative incidence of relapse/progressionNon-relapse mortality
Sirolimus-Containing Regimen2614
Sirolimus-Free Regimen3012

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To Compare the 2-year of Overall Survival, Progression-free Survival, Cumulative Incidences of Progression and Non-relapse Mortality Between the Treatment Arms for Each Histology Studied.

(NCT00928018)
Timeframe: 2 years

,,,
Interventionpercentage of participants (Number)
Overall SurvivalProgression Free SurvivalCumulative Incidence of ProgressionNon-relapse mortality
Aggressive Group: Sirolimus-Containing Regimen54463221
Aggressive Group: Sirolimus-Free Regimen7664279
Indolent Group: Sirolimus-Containing Regimen8271218
Indolent Group: Sirolimus-Free Regimen63533315

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AUC0-inf

Bioequivalence based on AUC0-inf - Area under concentration-time curve from time zero to infinity (extrapolated) (NCT00910663)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*h/mL (Mean)
Mycophenolate Mofetil (Test)14.6569
CellCept® (Reference)14.2048

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AUC0-t

Bioequivalence based on AUC0-t - Area under concentration-time curve from time zero to time of last non-zero concentration (NCT00910663)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*h/mL (Mean)
Mycophenolate Mofetil (Test)13.8388
CellCept® (Reference)13.4016

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Cmax

Bioequivalence based on Cmax - Maximum Drug Concentration (NCT00910663)
Timeframe: Blood samples collected over 72 hour period

Interventionµg/mL (Mean)
Mycophenolate Mofetil (Test)2.8358
CellCept® (Reference)2.9768

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Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60)

Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D6018

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Number of Number of Participants Who Experience Graft Failure, Day 360 (D60)

Number of participants who experience graft failure by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D602

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Number of Number of Participants Who Experience Graft Failure, Day 360 (D90)

Number of participants who experience graft failure by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D900

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Number of Number of Participants With Severe Chronic GVHD, Day 360 (D60)

Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D605

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Number of Number of Participants With Severe Chronic GVHD, Day 360 (D90)

Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D907

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Number of Participants Who Experience Disease Relapse, Days 60-180 (D60)

Number of participants who experience disease relapse between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable. (NCT02556931)
Timeframe: Between Day 60 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D6010

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Number of Participants Who Experience Disease Relapse, Days 90-180 (D90)

Number of participants who experience disease relapse between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable. (NCT02556931)
Timeframe: Between Day 90 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D9014

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Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D90)

Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D9016

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Number of Participants Who Experience Graft Failure, Days 60-180 (D60)

Number of participants who experience graft failure between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable. (NCT02556931)
Timeframe: Between Day 60 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D601

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Number of Participants Who Experience Graft Failure, Days 90-180 (D90)

Number of participants who experience graft failure between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable. (NCT02556931)
Timeframe: Between Day 90 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D900

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Number of Participants Who Experience Non-relapse Mortality, Day 360 (D60)

Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D604

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Number of Participants Who Experience Non-relapse Mortality, Day 360 (D90)

Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D904

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Number of Participants Who Experience Non-relapse Mortality, Days 60-180 (D60)

Number of participants who die for any reason other than disease relapse between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable. (NCT02556931)
Timeframe: Between Day 60 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D602

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Number of Participants Who Experience Non-relapse Mortality, Days 90-180 (D90)

Number of participants who die for any reason other than disease relapse between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable. (NCT02556931)
Timeframe: Between Day 90 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D901

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Number of Participants Who Experience Relapse, Day 360 (D60)

Number of participants who experience disease relapse by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D6010

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Number of Participants Who Experience Relapse, Day 360 (D90)

Number of participants who experience disease relapse by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360

InterventionParticipants (Count of Participants)
PBSCT D9014

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Number of Participants With Chronic GVHD, Days 60-180 (D60)

Number of participants who experience chronic GVHD requiring additional immunosuppressive therapy between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable. (NCT02556931)
Timeframe: Between Day 60 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D601

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Number of Participants With Chronic GVHD, Days 90-180 (D90)

Number of participants who experience chronic GVHD requiring additional immunosuppressive therapy between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable. (NCT02556931)
Timeframe: Between Day 90 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D902

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Number of Participants With Grades III-IV Acute GVHD, Days 60-180 (D60)

Number of participants who experience grade III or IV acute GVHD between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable. (NCT02556931)
Timeframe: Between Day 60 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D603

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Number of Participants With Grades III-IV Acute GVHD, Days 90-180 (D90)

Number of participants who experience grade III or IV acute GVHD between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable. (NCT02556931)
Timeframe: Between Day 90 and Day 180

InterventionParticipants (Count of Participants)
PBSCT D901

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Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D60 Cohort)

This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 60. (NCT02556931)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
PBSCT D6042

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Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D90 Cohort)

This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 90. (NCT02556931)
Timeframe: Day 90

InterventionParticipants (Count of Participants)
PBSCT D9033

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Event-free Survival

Estimated using Kaplan-Meier estimator. (NCT01044745)
Timeframe: From the date of transplant with relapse/progression or death as censored events, up to 2 years

Interventionmonths (Median)
Treatment (Rituximab and Allogeneic HCT Transplant)12

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Number of Participants With Grades II-IV Acute GVHD

Determined with death as a competing risk. Defined and staged using the 1994 consensus conference modifications of the Glucksberg criteria. (NCT01044745)
Timeframe: At day 100

InterventionParticipants (Count of Participants)
Treatment (Rituximab and Allogeneic HCT Transplant)16

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Overall Survival

Estimated using Kaplan-Meier estimator. (NCT01044745)
Timeframe: From the date of transplant with death from any cause as a censored event, up to 2 years

Interventionmonths (Median)
Treatment (Rituximab and Allogeneic HCT Transplant)12

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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 30 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR (polymerase chain reaction) for STR/VNTR (short tandme repeat/variable number tandem repeat) will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 30

Interventionparticipants (Number)
Haploidentical Transplant18

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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 60 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 60

Interventionparticipants (Number)
Haploidentical Transplant18

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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 90 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 90

Interventionparticipants (Number)
Haploidentical Transplant13

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Disease Free Survival at 12 Months

Disease free survival refers to patients with no evidence of disease after transplant, at the 12 month time point. (NCT00782379)
Timeframe: 12 months

Interventionparticipants (Number)
Haploidentical Transplant7

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Disease Free Survival at Day 100

Disease free survival refers to patients with no evidence of disease after transplant, at the Day 100 time point. (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant16

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Incidence of Graft Rejection for Patients at Day 100

Number of patients who experienced graft rejection by Day 100 (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant0

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Non-relapse Mortality at 1 Year After Peripheral Blood Stem Cell Transplantation (PBSCT)

Non-relapse mortality refers to whether a patient dies of causes related to something other than the primary disease. (NCT00782379)
Timeframe: 1 year

Interventionparticipants (Number)
Haploidentical Transplant2

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Non-relapse Mortality at Day 100 After Peripheral Blood Stem Cell Transplantation (PBSCT)

(NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant2

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Number of Patients Who Experienced Severe Graft-versus-host Disease (GVHD)(Grade 3 or 4)

Number of patients who experienced post-transplant complication (GVHD) as seen by clinical evidence (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant2

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Overall Survival at 12 Months

Overall survival, defined as a patient being alive after transplant, is without regard to disease status. (NCT00782379)
Timeframe: 12 months

Interventionparticipants (Number)
Haploidentical Transplant14

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Overall Survival at Day 100

Overall survival is assessed, without regard to disease status, post-transplant, at Day 100. (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant17

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Average Level of Protenuria at Week 52

Proteinuria is measured as the ratio of albumin/creatinine mg/mmol. Measurements were taken from participants urine samples. (NCT01266148)
Timeframe: 52 weeks

Interventionmg/mmol (Mean)
Everolimus7.2
Control1.2

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Measured Glomerular Filtration Rate (mGFR), 12 Months After Heart Transplantation

Measured Glomerular Filtration Rate (mGFR) describes the flow rate of filtered fluid through the kidney. GFR is equal to the clearance rate when any solute is freely filtered and is neither reabsorbed nor secreted by the kidneys. The rate therefore measured is the quantity of the substance in the urine that originated from a calculable volume of blood. Participants' urine was used for this assessment at week 52 after heart transplant. (NCT01266148)
Timeframe: Week 52

InterventionmGFR ml/min (Mean)
Everolimus79.8
Control61.5

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Calculated Glomerular Filtration Rate From Pre-Transplantation to Week 52

Calculated Glomerular Filtration Rate from pre-transplantation to week 52 was calculated according to the Modification of Diet in Renal Disease (MDRD) method. Measurements were taken prior to transplant (day 1), between weeks 7 to 11 and end of week 52. (NCT01266148)
Timeframe: Day 1, weeks 7 to 11 and of week 52

,
InterventionmGFR mL/min (Mean)
Day 1 (n= 46, 48)Weeks 7 to 11 (n=50, 51)Week 52 (n= 45, 55)
Control66.169.669.3
Everolimus65.484.9104.5

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Change in Calculated Glomerular Filtration Rate From Pre-transplantation to Week 52

Change in calculated glomerular filtration rate from pre-transplantation to week 52 was calculated according to the Modification of Diet in Renal Disease (MDRD) method. Measurements were taken prior to transplant (day 1), between weeks 7 to 11 and end week 52. (NCT01266148)
Timeframe: Day 1, weeks 7 to 11(baseline) and of week 52

,
InterventionmGFR mL/min (Mean)
Day 1 (n= 43, 47)Weeks 7 to 11 (n= 46, 49)Week 52 (n= 42, 53)
Control-12.6-6.8-8.0
Everolimus-13.07.427.8

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Change in Quality of Life - Euro Quality of Life 5D (EQ-5D)

Change in Quality of Life was assessed via the EQ-5D questionnaire which consists of: EQ-5D-5L descriptive system and EQ Visual Analogue scale (EQ VAS). The EQ-5D-5L comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 5 levels (no problems, slight problems, moderate problems, severe problems, and extreme problems). The patient indicates his/her health state by checking the most appropriate statement. This decision results in a 1-digit number expressing the level selected for that dimension. The digits for 5 dimensions are combined in a 5-digit number describing the respondent's health state. The possible score is 1 to 5 where a lower number indicates improvement. The EQ VAS records the patient's self-rated health on a 20 cm vertical, visual analogue scale with endpoints labelled 'the best health you can imagine' and 'the worst health you can imagine'. The score is 0 to 100 where a higher score represents improvement. (NCT01266148)
Timeframe: Pre transplant and 52 weeks

,
InterventionUnits on a scale (Mean)
EQ-5D-5L pre- transplant (n=45,49)EQ-5D-5L at week 52 (n=41,44)EQ VAS pre- transplant (n=41,48)EQ VAS at week 52 (n=41,42)
Control0.50690.836738.979.4
Everolimus0.57500.832946.080.0

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Change in Quality of Life Assessed by SF-36 (Minnesota Living With Heart Failure Questionnaire ([MLHF)]) From Pre-transplant to Week 52 of Treatment

Change in Quality of Life was assessed via the SF-36 (Minnesota Living with Heart Failure questionnaire ([MLHF)]) before transplant surgery and at week 52 of treatment. The SF-36 is a validated, self-administered questionnaire. The questionnaire, which includes 36 questions measures 8 dimensions of health: physical function, role-physical, bodily pain, general health, vitality, social function, role-emotional, and mental health. Scores can be summarized in 2 summary components assessing physical and mental health. Items in each dimension are coded, aggregated, summed, and transformed into a scale ranging from 0 (worse health) to 100 (best health). (NCT01266148)
Timeframe: Pre transplant and 52 weeks

,
InterventionUnits on a scale (Mean)
Physical Health pre- transplantPhysical Health at week 52Mental Health pre- transplantMental Health at week 52
Control32.948.838.753.9
Everolimus30.848.846.251.5

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Lipid Profile at 12 Months

Total Cholesterol, LDL-Chol, HDL-Chol and TG at week 52. Measurements were taken via participants blood samples. (NCT01266148)
Timeframe: 52 weeks

,
Interventionmmol/L (Mean)
Total cholesterolLDL-CHDL-C
Control5.12.81.6
Everolimus5.32.91.6

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Number of Rejections Leading to Hemodynamic Compromise

Number of all rejections were recorded through the duration of the study with the intent to identify rejections leading to hemodynamic compromise. (NCT01266148)
Timeframe: 52 weeks

,
Interventionrejections (Number)
Total rejectionTreated rejectionRejections with hemodynamic compromise
Control128170
Everolimus185430

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Occurrence of Treatment Failures up to 12 Months After Transplant

Treatment failure was defined as the number of participants who died or lost their graft at any timepoint througout the duration of the study. (NCT01266148)
Timeframe: 52 weeks

,
Interventionparticipants (Number)
Treatment failure (death)Graff lossTotal treatment failures (graft loss + death
Control303
Everolimus202

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Progression of Chronic Allograft Vasculopathy (CAV) Based on Incidence of Chronic Allograft Vasculopathy (CAV) From Baseline to Week 52

the progression of chronic allograft vasculopathy (CAV) assessed by intravascular ultrasound (IVUS) examinations, measured the incidence of CAV (in percent of patients) at baseline and at week 52. Incidence of CAV represents percent of patients having a MIT (maximal intima thickness) > 0.5 mm. (NCT01266148)
Timeframe: Baseline and week 52

,
InterventionPercentage of patients (Number)
No CAV at baseline (week 7)CAV at baseline (week 7)No CAV at week 52CAV at week 52
Control47.952.135.464.6
Everolimus43.556.55050

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Progression of Chronic Allograft Vasculopathy (CAV) Based on Maximal Intimal Thickness (MIT) From Baseline to Week 52

The progression of chronic allograft vasculopathy (CAV) was assessed by intravascular ultrasound (IVUS) examinations and measured Maximal Intimal Thickness (MIT)(in mm). A major coronary epicardial artery (preferentially the left-anterior descending coronary artery) was imaged, and the MIT parameters were recorded at baseline and at week 52. (NCT01266148)
Timeframe: Baseline and week 52

,
Interventionmm (Mean)
Baseline (Week 7)Week 52
Control0.560.65
Everolimus0.520.55

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Overall Survival

Number of participants alive 1 year post transplant. (NCT03096782)
Timeframe: Up to 12 months after transplant

InterventionParticipants (Count of Participants)
Chemotherapy Plus Cord Blood Transplant4

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Disease-free Survival

Number of participants that were in remission post transplant. (NCT03096782)
Timeframe: Up to12 months

InterventionParticipants (Count of Participants)
Complete Remission at 30 daysComplete Remission at 12 months
Chemotherapy Plus Cord Blood Transplant64

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Time to Engraftment

Number of days from transplant when participants achieved engraftment measure by ANC of 0.5 for three consecutive days. (NCT03096782)
Timeframe: Up to 12 months after transplant

InterventionParticipants (Count of Participants)
Twenty Two DaysTwenty Nine DaysThirty DaysNine DaysThirty Two Days
Chemotherapy Plus Cord Blood Transplant21111

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Percent Change in Mean Fluorescence Index (MFI) of Donor Specific Antibodies (DSA) With Increasing Doses of Enteric-Coated Mycophenolate Sodium (EC-MPS)

(NCT01044303)
Timeframe: 24 months

Interventionpercent of MFI change (Mean)
Mycophenolic Acid (MPA) Escalation43.1

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To Assess the Rate of Rejection, Infection and Renal Function as Mycophenolic Acid Dose is Increased.

(NCT01044303)
Timeframe: 24 months

Interventionparticipants (Number)
Rate of Infection2
Rate of Rejection2
Renal Function29

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Disease Response at One Year Following Hematopoietic Cell Transplantation

Number of patients with no evidence of disease at one year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)78
Regimen B (UBCT)8

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Immune Reconstitution Following Hematopoietic Cell Transplantation

Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)39
Regimen B (UBCT)4

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Non-relapse Mortality

Number of patients who experienced non-relapse mortality by 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)2
Regimen B (UBCT)5

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Number of Participants With Infections

Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant (NCT00919503)
Timeframe: 100 days post transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)57
Regimen B (UBCT)9

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Number of Patients With Grade II-IV Acute Graft-versus-host Disease

Number of patients diagnosed with overall grade II-IV acute GVHD by Day 100 post transplant (NCT00919503)
Timeframe: Day 100 post transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)44
Regimen B (UBCT)8

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Number of Patients With of Chronic Graft-versus-host Disease

Number of patients diagnosed with chronic GVHD and requiring systemic immunosuppression within 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)29
Regimen B (UBCT)5

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Overall Survival

Number of patients alive at 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)80
Regimen B (UBCT)9

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Preliminary Efficacy

Number of patients engrafted (>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)83
Regimen B (UBCT)10

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Donor Chimerism CD3 at 100 Days Post Transplant

Number of patients with peripheral blood donor chimerism for CD3 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. (NCT00919503)
Timeframe: Day 100 post transplant

,
InterventionParticipants (Count of Participants)
Greater than equal to 95%50 - 94%5-49%Less than 5%
Regimen A (PBSCT and BMT)492960
Regimen B (UBCT)7211

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Donor Chimerism CD33 at Day 100 Post Transplant

Number of patients with peripheral blood donor chimerism for CD33 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. (NCT00919503)
Timeframe: 100 days post transplant

,
InterventionParticipants (Count of Participants)
Greater than equal to 95%50-94%5-49%Less than 5%
Regimen A (PBSCT and BMT)72840
Regimen B (UBCT)7221

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Graft Failure

Percentage of participants who failed to engraft. (NCT00946023)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Transplant2

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Incidence of Chronic GVHD

Percentage of participants who experienced chronic GVHD. Chronic GVHD is graded using NIH consensus criteria and Seattle criteria. (NCT00946023)
Timeframe: 1 year post intervention

Interventionpercentage of participants (Number)
Transplant11

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Incidence of Grades II-IV Acute Graft-versus-Host-Disease (GVHD)

Percentage of participants who experienced grade II, III, or IV acute GVHD. Acute GVHD is graded using the Przepiorka criteria. (NCT00946023)
Timeframe: 1 year post intervention

Interventionpercentage of participants (Number)
Transplant41

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Incidence of Grades III-IV Acute GVHD

Percentage of participants who experienced grade II, III, or IV acute GVHD. Acute GVHD is graded using the Przepiorka criteria. (NCT00946023)
Timeframe: 1 year post intervention

Interventionpercentage of participants (Number)
Transplant5

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Non-relapse Mortality

Percentage of participants who died due to BMT-related reasons. (NCT00946023)
Timeframe: 1 year post intervention

Interventionpercentage of participants (Number)
Transplant8

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Engraftment

Percentage of patients who engrafted neutrophils and platelets. (NCT00946023)
Timeframe: Day 60

Interventionpercentage of participants (Number)
Neutrophil engraftmentPlatelet engraftment
Transplant9898

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Overall Survival

Percentage of participants alive. (NCT00946023)
Timeframe: 1 year post intervention

Interventionpercentage of participants (Number)
All participantsHaploidentical recipientsVV donorVF donorFF donor
Transplant8683948678

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Overall Survival

Percentage of participants alive. (NCT00946023)
Timeframe: 2 years post intervention

Interventionpercentage of participants (Number)
All participantsHaploidentical recipientsVV donorVF donorFF donor
Transplant7673877669

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Progression-free Survival

Percentage of participants alive and without relapse or disease progression. (NCT00946023)
Timeframe: 1 year post-intervention

Interventionpercentage of participants (Number)
All participantsHaploidentical recipientsVV donorVF donorFF donor
Transplant7170827065

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Progression-free Survival

Percentage of participants alive with and without relapse. (NCT00946023)
Timeframe: 2 years post-intervention

Interventionpercentage of participants (Number)
All participantsHaploidentical recipientsVV donorVF donorFF donor
Transplant6063685956

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Relapse

Percentage of participants alive with relapse or disease progression. (NCT00946023)
Timeframe: 1 year post intervention

Interventionpercentage of participants (Number)
All participantsHaploidentical recipientsVV donorVF donorFF donor
Transplant2020182317

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Relapse

Percentage of participants alive with relapse or disease progression. (NCT00946023)
Timeframe: 2 years post intervention

Interventionpercentage of participants (Number)
All participantsHaploidentical recipientsVV donorVF donorFF donor
Transplant2723253122

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Change From Baseline in the Euro Quality of Life 5D

"Change from baseline in Euro Quality of Life-5D from 3 Year Follow-Up to 5 to 7 Year Follow-Up Baseline Visit 1 (ITT Set)~Euro Quality of Life 5D (EQ-5D): is a descriptive system of healthrelated quality of life states consisting of five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression) each of which can be assessed as one of three levels of severity (no problems/some or moderate problems/extreme problems). A Visual Analogue Scale (VAS)-scale is also included in the EQ-5D questionnaire.~The EQ-5D index is calculated based on the United Kingdom Time Trade-Off (TTO) N3 value set which converts the five dimensions scores into a single measure with a possible range from -0.163 (worst possible health state) to +1 (perfect health). A positive change from baseline indicates an improvement in Quality of Life." (NCT02864706)
Timeframe: Baseline, 5-7 year visit

Interventionscores on the scale (Mean)
Everolimus0.2323
Control0.2982

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Change From Baseline in Visual Analog Scale (VAS)

"Change in visual analog scale (VAS) from baseline to the 5 to 7 Year follow up visit.~0 is no pain; and 10 is the worst possible pain" (NCT02864706)
Timeframe: baseline, at the 5-7 year visit

Interventionmm (Mean)
Everolimus35.6
Control34.0

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Measured Glomerular Filtration Rate (mGFR)

Renal function as assessed by measured Glomerular Filtration Rate (mGFR) (Cr-EDTA or iohexol clearance). Baseline Visit 1 and Patient 4252 excluded from the intent treat analysis set. (NCT02864706)
Timeframe: at the 5-7 year follow-up visit

InterventionmL/min/1.73m2 (Least Squares Mean)
Everolimus74.7
Control62.4

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Percent of Participants With Incidence of Coronary Allograft Vasculopathy (CAV)

"Cardiac Allograft Vasculopathy (CAV) was defined as mean maximal intimal thickness (MIT)~≥0.5 mm, measured for the entire matched pullback recording by intravascular ultrasound (IVUS). The incidence of CAV at 5-7 years was compared between groups using the Cochran-Mantel-Haenszel test with stratification according to baseline distribution of CAV incidence." (NCT02864706)
Timeframe: at the 5-7 year follow-up

Interventionpercent of participants (Number)
Everolimus53
Control74

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Progression of Cardiac Allograft Vasculopathy (CAV) Recorded by Intravascular Ultrasound (IVUS)

"Cardiac Allograft Vasculopathy (CAV) was defined as mean maximal intimal thickness (MIT)~≥0.5 mm, measured for the entire matched pullback recording by intravascular ultrasound (IVUS). The incidence of CAV at 5-7 years was compared between groups using the Cochran-Mantel-Haenszel test with stratification according to baseline distribution of CAV incidence." (NCT02864706)
Timeframe: within 5-7 years

Interventionmm (Mean)
Everolimus0.13
Control0.23

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Myocardial Structure and Function

Myocardial structure and function by echocardiography assessment measured by ventricular end systolic diameter. (NCT02864706)
Timeframe: within 5-7 years

,
Interventioncm (Mean)
LVESD (left ventricular end systolic diameter)LVEDD (left ventricular end diastolic diameter)
Control3.14.9
Everolimus3.14.7

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Number of Participants With Beck Depression Inventory (BDI)

Beck Depression Inventory (BDI) Score has the following categories of depression. Normal, Mild, Moderate Severe and Missing. (NCT02864706)
Timeframe: at the 5-7 year visit

,
InterventionParticipants (Count of Participants)
NormalMildModerateSevereMissing
Control134505
Everolimus156213

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Quality of Life by SF-36 Change From Pre-transplantation to 5-7 Year Follow-up

This Quality of life Short Form Survey with 36 items (Minnesota Living with Heart Failure Questionnaire)was administered to patients pre-transplantation and after transplantation at the 5-7 year visit. This data represents the change. The survey consist of scores on a scale. Each form is scaled from 0 t 100. 0 = maximum disability and 100 equals no disability. (NCT02864706)
Timeframe: at the 5-7 year visit

,
Interventionscores on a scale (Mean)
Physical Health SummaryMental Health SummaryPhysical FunctioningRole PhysicalBodily PainGeneral HealthVitalitySocial FunctioningRole EmotionalMental Health
Control13.215.340.855.17.223.428.943.942.814.4
Everolimus16.810.436.750.210.325.730.039.923.88.6

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Disease-free Survival at Five Years Post-transplant

Kaplan-Meier estimate of an event of relapse or death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of transplant to five years post-transplant

Interventionpercentage of survival probability (Number)
Busulfan, FTBI and VP1640

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Overall Survival at 5 Years Post-Transplant.

Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of Transplant to Five Years post-Transplant

Interventionpercentage of survival probability (Number)
Busulfan, FTBI and VP1640

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Overall Survival Comparing Diagnosis Groups

Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of Transplant to Five Years post-Transplant

Interventionpercentage of survival probability (Number)
AML/1CR67
AML/R150
AML/IF30
Active ALL33

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Immune Reconstruction/CD4+ Count at 1 Year

(NCT00425802)
Timeframe: 1 year

Interventioncells/microliter (Median)
Treatment333

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Immune Reconstruction/CD4+ Count at 3 Months

(NCT00425802)
Timeframe: 3 months

Interventioncells/microliters (Median)
Treatment253

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Immune Reconstruction/CD4+ Count at 6 Months

(NCT00425802)
Timeframe: 6 months

Interventioncells/microliter (Median)
Treatment312

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Incidence of Chronic GVHD at 1 Year

(NCT00425802)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Treatment14

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Incidence of Moderate to Severe Grades II to IV Graft Versus Host Disease (GVHD) at 100 Days

(NCT00425802)
Timeframe: 100 days

Interventionpercentage of patients (Number)
Treatment18

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Overall Survival at 1 Year

(NCT00425802)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Treatment90

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Time to Neutrophil Engraftment

(NCT00425802)
Timeframe: 2 years

Interventiondays (Median)
Treatment15

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Time to Platelet Engraftment

(NCT00425802)
Timeframe: 1 year

Interventiondays (Median)
Treatment12

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Event-Specific Survival Comparisons

Freedom from biopsy-proven acute rejection of the kidney allograft; Freedom from biopsy-proven acute rejection of the pancreas allograft; Death-censored kidney graft survival; Death-censored pancreas graft survival; Death-uncensored graft (kidney and pancreas) survival; and Patient survival. (NCT00533442)
Timeframe: over 1-10 years post-transplant

,
InterventionParticipants (Count of Participants)
Biopsy-Proven Acute Rejection of the KidneyBiopsy-Proven Acute Rejection of the PancreasDeath-Censored Kidney Graft FailureDeath-Censored Pancreas Graft FailureDeath-Uncensored Graft (Kidney&Pancreas) SurvivalPatient Death
Tacrolimus Plus MMF Plus Steroids2291872616
Tacrolimus Plus Rapamycin Plus Steroids811022515

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Overall Kidney Transplant Function at 12, 36, and 60 Months Post-transplant.

Comparisons of renal function (eGFR, measured in mL/min/1.73 m^2) at 12, 36, and 60 months post-transplant. (NCT00533442)
Timeframe: at 1-5 years post-transplant

,
InterventionmL/min/1.73m^2 (Mean)
Mean eGFR at 12 monthsMean eGFR at 36 monthsMean eGFR at 60 months
Tacrolimus Plus MMF Plus Steroids67.261.561.9
Tacrolimus Plus Rapamycin Plus Steroids71.263.056.5

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Overall Pancreas Transplant Function at 12, 36, and 60 Months Post-transplant.

Comparisons of pancreas function (C-peptide in ng/mL) at 12, 36, and 60 months post-transplant. (NCT00533442)
Timeframe: at 1-5 years post-transplant

,
Interventionng/mL (Mean)
Mean Log {C-peptide at 12 months}Mean Log {C-peptide at 36 months}Mean Log {C-peptide at 60 months}
Tacrolimus Plus MMF Plus Steroids1.150.9901.17
Tacrolimus Plus Rapamycin Plus Steroids1.080.9861.22

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Clinical Significant Infection, Requiring Treatment, Within 100 Days Post Transplant

Number of patients who experienced a clinical significant infection, requiring treatment, within 100 days post transplant. (NCT00553098)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)21

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Disease Response by 1 Year Post Transplant

Number of patients at 1 year with disease response (defined as no clinical evidence of active disease and/or sufficient level of donor chimerisms to prevent disease recurrence) (NCT00553098)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)15

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Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant

Number of Patients Who Achieve Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant (NCT00553098)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)16

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Greater Than 50% CD33+ Donor Chimerisms at 1 Year Post Transplant

Number of patients who achieve greater than 50% CD33+ donor chimerisms at 1 year post transplant. (NCT00553098)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)8

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Immune Reconstitution by 1 Year Post Transplant

Number of patients with normal range CD3 at 1 year post transplant (NCT00553098)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)7

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Number of Patients Diagnosed With Acute GVHD

Number of patients diagnosed with acute GVHD by Day 100 post transplant (NCT00553098)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)18

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Number of Patients Diagnosed With Chronic GVHD

Number of patients diagnosed with chronic GVHD within 1 year post transplant (NCT00553098)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)8

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Number of Patients Diagnosed With Overall Grade 1 or Grade 2 Acute GVHD

Number of patients diagnosed with overall grade I or grade II acute GVHD by Day 100 post transplant (NCT00553098)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)12

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Number of Patients Diagnosed With Overall Grade III or Grade IV Acute GVHD

Number of patients diagnosed with overall Grade III or Grade IV Acute GVHD by Day 100 post transplant (NCT00553098)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)6

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Number of Patients Who Achieve Greater Than 50% Donor T-cell Chimerism

The study will be considered a success and the protocol worthy of further study if there is sufficient evidence that this rate is greater than the 50% rate observed in the most recently transplanted patients with nonmalignant disorders. Analyses will be carried out separately for the alemtuzumab recipients and the TBI recipients. We will be 80% confidence of success if a one-sided 80% confidence interval for the proportion of patients with successful chimerism exceeds 50%. Cumulative incidence will be used to evaluate the probability of chimerism. (NCT00553098)
Timeframe: At 1 year post transplant

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)13

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Overall Survival

Number of patients alive at 1 year (NCT00553098)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Low Dose Radiation)19

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Graft Failure

Percentage of participants who failed to engraft. (NCT01135329)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Transplant8

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Number of Patients Surviving Overall

Number of subjects surviving two years post autologous transplant. (NCT00793572)
Timeframe: At 2 years after the autograft

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy21

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Number of Patients Surviving Progression-free

"Number of subjects surviving without progressive disease post-transplant.~Progressive disease criteria:~Greater than 25% increase in serum (absolute increase must be ≥0.5 g/dL) or urine (absolute increase must be ≥200 mg/24h) M proteins compared to best response status after autologous transplant.~Appearance of new lytic bone lesions or plasmacytomas." (NCT00793572)
Timeframe: At 2 years after the autograft

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy14

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Number of Patients With Chronic GVHD

Number of subjects with classic chronic or chronic extensive GVHD post allogeneic transplant. (NCT00793572)
Timeframe: 1 year post allo

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy10

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Number of Patients With Grade II-IV Acute GVHD

"Number of patients who developed acute GVHD post allogeneic transplant.~aGVHD Stages~Skin:~- a maculopapular eruption involving < 25% BSA~- a maculopapular eruption involving 25 - 50% BSA~- generalized erythroderma~- generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~- bilirubin 2.0 - 3.0 mg/100 mL~- bilirubin 3 - 5.9 mg/100 mL~- bilirubin 6 - 14.9 mg/100 mL~- bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT00793572)
Timeframe: 100 days post allo transplant

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy14

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Number of Patients With Non-relapse Mortality

Number of subjects with non-relapse mortalities post allogeneic transplant. (NCT00793572)
Timeframe: 200 and 365 days after allo

InterventionParticipants (Count of Participants)
200 days post allo1 Year post allo
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy11

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Event-Free Survival (EFS)

Number of patients surviving without relapsed/progressive disease (NCT01008462)
Timeframe: 1 Year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)9

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Number of Patients Who Engrafted

Number of patients with donor engraftment. (NCT01008462)
Timeframe: Day 84 post-allograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)13

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Number of Patients Who Had Infections

Number of patients who had infections. (NCT01008462)
Timeframe: 1 Year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)16

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Number of Patients With Relapsed/Progressive Disease

"Relapse/Progression defined as:~Nodes, liver, and/or spleen ≥50% increased or new by physical exam / imaging studies.~Circulating lymphocytes ≥50% increased by morphology and/or flow cytometry. Richter's transformation by lymph node biopsy ." (NCT01008462)
Timeframe: 1 year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)6

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Overall Survival

Number of patients surviving one year post-autograft (NCT01008462)
Timeframe: 1 year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)10

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Non-relapse Mortality (NRM)

Number of patients with non-relapse mortalities. (NCT01008462)
Timeframe: 200 days and 1 Year post-allograft

InterventionParticipants (Count of Participants)
200 days1 Year
Treatment (Autologous HCT, Donor HCT)01

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Number of Patients With Grade II-IV Acute Graft-versus-Host-Disease and/or Chronic Extensive Graft-versus-Host-Disease

"aGVHD The diagnosis of aGVHD is identified through various stages and grading of the disease related to Skin (Rash), Gut (Diarrhea, Nausea/vomiting and/or anorexia) and the liver (Bilirubin) assessed by severity and grading scale outlined in the section Grafts vs Hosts by Sullivan (1999).~GVHD Grades Grade I: 1-2 Skin Rash; No gut or liver involvement Grade II: Stage 1-3 Skin rash; Stage 1 gut and/or stage 1 liver involvement Grade III: Stage 2-4 gut involvement and/or stage 2-4 liver involvement with or without rash Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death~CGVHD The diagnosis of cGVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD." (NCT01008462)
Timeframe: 1 year post-allograft,

InterventionParticipants (Count of Participants)
Acute Graft-versus-Host-DiseaseChronic extensive Graft-versus-Host-Disease
Treatment (Autologous HCT, Donor HCT)81

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: 6 months

Interventionpercentage of donor cells (Mean)
Transplant Patients94

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 100

Interventionpercentage of donor cells (Mean)
Transplant Patients90

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 28

Interventionpercentage of donor cells (Mean)
Transplant Patients95

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 42

Interventionpercentage of donor cells (Mean)
Transplant Patients93

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: One year

Interventionpercentage of donor cells (Mean)
Transplant Patients99

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Number of Patients Who Died Peri-Transplant

Peri-transplant is defined as within 100 days of transplant. (NCT01043640)
Timeframe: By Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients2

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Number of Patients With Donor Derived Engraftment

Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells. (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients42

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Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients32

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Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients2

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Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients5

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Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients2

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Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients5

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Difference in Renal Function

"Difference in renal function between groups at listed time points assessed by mean serum creatinine. Increased serum creatinine could indicate worsening renal function. A normal serum creatinine range for the transplant population varies by patient, but a typical range for Scr would be 1-2 mg/dL." (NCT01336296)
Timeframe: Difference at 1 month, 3 months, 6 months, 1 year

,
Interventionmg/L (Mean)
1 month3 months6 months1 year
Myfortic Preload1.411.451.431.36
Myfortic Standard1.431.391.401.5

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Incidence of Biopsy-confirmed Acute Rejection by Banff '97 Criteria (Updated 2007) 3, 6 and 12 Months Post Transplant

"Incidence of biopsy-confirmed acute rejection by Banff '97 Criteria (updated 2007) post transplant. The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~As with humoral rejection, there are both acute & chronic forms:~The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~Class IA: there is at least 25% of parenchymal showing interstitial infiltration and foci of moderate tubulitis (defined as a certain number of immune cells present in tubular cross-sections).~Class IB: just like Class IA except there is more severe tubulitis.~Class IIA: there is mild-to-moderate intimal arteritis.~Class IIB: there is severe intimal arteritis comprising at least 25% of the lumenal area.~Class III: there is transmural (e.g. the full vessel wall thickness) arteritis." (NCT01336296)
Timeframe: 3, 6 and 12 months post transplant

,
Interventionparticipants (Number)
3 months6 months12 months
Myfortic Preload778
Myfortic Standard334

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Incidence of Chronic Alloantibody Rejection or Chronic Allograft Arteriopathy by Banff '97

The Banff features suggestive of chronic rejection were: a) chronic transplant glomerulopathy: Glomerular basement membrane duplication and mesangial cell proliferation, and b) vasculopathy: Fibrous intimal thickening often with fragmentation of internal elastic lamina. Chronic changes in the interstitium (ci), tubules (ct), vessels (cv), and glomerulus (cg) were likewise graded into 0, 1, 2, and 3. The severity of interstitial fibrosis and tubular atrophy, as also chronic transplant glomerulopathy and vasculopathy were used to grade chronic allograft changes. (NCT01336296)
Timeframe: 1 year

,
Interventionparticipants (Number)
mild Chronic allograft nephropathy (CAN)Moderate Chronic allograft nephropathy (CAN)
Myfortic Preload90
Myfortic Standard31

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Number of Patients Requiring Anti-lymphocyte Therapy for Acute Rejection

(NCT01336296)
Timeframe: 1 year

,
Interventionparticipants (Number)
Pulse methylprednisoloneThymoglobulinPlasmapheresisIntravenous immunoglobulin (IVIg)Rituximab
Myfortic Preload56000
Myfortic Standard30201

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Severity of Acute Rejection by Banff '97 Criteria

"Severity of biopsy-confirmed acute rejection by Banff '97 Criteria (updated 2007) at 1 year. The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~As with humoral rejection, there are both acute & chronic forms:~The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~Class IA: there is at least 25% of parenchymal showing interstitial infiltration and foci of moderate tubulitis (defined as a certain number of immune cells present in tubular cross-sections).~Class IB: just like Class IA except there is more severe tubulitis.~Class IIA: there is mild-to-moderate intimal arteritis.~Class IIB: there is severe intimal arteritis comprising at least 25% of the lumenal area.~Class III: there is transmural (e.g. the full vessel wall thickness) arteritis." (NCT01336296)
Timeframe: Severity 1 year post transplant

,
Interventionparticipants (Number)
Grade IAGrade IBGrade IIAGrade IIBAntibody Mediated RejectionMixed Acute Rejection
Myfortic Preload341000
Myfortic Standard110011

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Acute GvHD

overall grade II-IV acute GvHD (NCT00053989)
Timeframe: Day +100

InterventionParticipants (Count of Participants)
All Patients16

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Day 100 TRM

treatment related mortality within 100 days from hematopoietic stem cell (HSC) infusion on day 0 (NCT00053989)
Timeframe: from start or conditioning (day -6 or -5) through day +100 after HSC infusion

InterventionParticipants (Count of Participants)
All Patients4

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OS

Overall survival with events defined as death due to any cause and censored patients are alive as of 1 year post HSC infusion (NCT00053989)
Timeframe: 1 year

Interventionpercentage of participants (Number)
All Patients44

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PFS

Progression free survival defined as time from HSC infusion (day 0) until progression of disease or death due to any cause. Patients are censored if alive without disease progression through 1 year after HSC infusion (NCT00053989)
Timeframe: 1 year

Interventionpercentage of participants (Number)
All Patients27

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Percentage of Participants Discontinuing Immunosuppressants (MMF) for More Than 14 Consecutive Days or 30 Cumulative Days Within 24 Weeks of Transplantation

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
MMF + CsA13.89

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Percentage of Participants Lost To Follow Up Within 24 Weeks of Transplantation

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
MMF + CsA0.0

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Percentage of Participants Requiring Use of Additional Immunosuppressants Not Specified in the Protocol Within 24 Weeks of Transplantation

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
MMF + CsA0.0

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Percentage of Participants With Graft Loss Within 24 Weeks of Transplantation

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
MMF + CsA0.0

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Percentage of Participants With Abnormal Serum Creatinine and BUN Values at Baseline and During Treatment

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
Serum creatinineBUN
MMF + CsA13.8972.22

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Percentage of Participants With Abnormal Serum Creatinine and BUN Values at Baseline and Normal Values During Treatment

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
Serum creatinineBUN
MMF + CsA16.672.78

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Percentage of Participants With Biopsy Proven Acute Rejection (BPAR) by Week

Percentage of participants with BPAR of greater than or equal to (≥) International Society of Heart and Lung Transplant (ISHLT) Grade III. The ISHLT graded symptoms on a scale of Grade 0 through VI. Grade 0 equals (=) no rejection. Grade IA = regional (perivascular or interstitial) infiltration and no necrosis, and grade IB = dissemination but little infiltration and no necrosis. Grade II = 1 focus of invasive infiltration with or without (+/-) associated cardiomyocyte necrosis. Grade IIIA = 2 or more foci of invasive infiltration +/- associated cardiomyocyte necrosis, and grade IIIB = diffuse inflammatory pathological changes associated with cardiomyocyte necrosis. Grade IV = diffuse, infiltrative multi-foci +/- edema; +/- hemorrhage; and +/-vasculitis. (NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
Day 1Week 1Week 2Week 4Week 8Week 12Week 16Week 20Week 24
MMF + CsA0.00.02.782.782.782.782.782.782.78

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Percentage of Participants With Normal Serum Creatinine and Blood Urea Nitrogen (BUN) Values At Baseline (BL) And During Treatment

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
Serum creatinineBUN
MMF + CsA47.228.33

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Percentage of Participants With Normal Serum Creatinine and BUN Values at Baseline and Abnormal Values During Treatment

(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24

Interventionpercentage of participants (Number)
Serum creatinineBUN
MMF + CsA22.2216.67

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Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)

AEs with a start date on or after the first dose date in study 211LE202. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the subject at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the subject or may require intervention to prevent one of the other outcomes listed above. (NCT01930890)
Timeframe: Up to Week 108

,,,
Interventionparticipants (Number)
Any eventModerate or severe eventSevere eventEvent related to dose-blinded treatmentEvent related to MMFSerious eventSerious event related to dose-blinded treatmentSerious event related to MMFFatal event
BIIB023 20 mg/kg (211LE201) to BIIB023 20 mg/kg (211LE202)1963493121
BIIB023 3 mg/kg (211LE201) to BIIB023 3 mg/kg (211LE202)23124587230
Placebo (211LE201) to BIIB023 20 mg/kg (211LE202)731124110
Placebo (211LE201) to BIIB023 3 mg/kg (211LE202)420121000

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Number of Participants Who Discontinued Study Treatment or Withdrew From Study Due to an AE

AEs with a start date on or after the first dose date in study 211LE202. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the subject at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the subject or may require intervention to prevent one of the other outcomes listed above. (NCT01930890)
Timeframe: Up to Week 108

,,,
Interventionparticipants (Number)
Discontinued treatment due to an AEWithdrew from study due to an AE
BIIB023 20 mg/kg (211LE201) to BIIB023 20 mg/kg (211LE202)01
BIIB023 3 mg/kg (211LE201) to BIIB023 3 mg/kg (211LE202)02
Placebo (211LE201) to BIIB023 20 mg/kg (211LE202)00
Placebo (211LE201) to BIIB023 3 mg/kg (211LE202)00

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Full Donor Chimerism (FDC)

Proportion of patients achieving full donor T-cell chimerism (FDC) by on or before Day 90 post non-myeloablative allogeneic transplant with allogeneic cytokine-induced killer (CIK) cells will be determined. FDC is defined as the attainment of >95% donor type CD3+ cells. The outcome will be reported as number of participants who achieved full donor chimerism, a number without dispersion. (NCT01392989)
Timeframe: 90 days

InterventionParticipants (Count of Participants)
Allogeneic Cytokine Induced Killer Cells (CIK)6

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Overall Survival (OS)

Overall survival (OS) is an expression of the number of participants that remain alive 2 years after cytokine-induced killer (CIK) infusion. The outcome will be reported as the number of participants alive 2 years after CIK infusion, a number without dispersion. (NCT01392989)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Allogeneic Cytokine Induced Killer Cells (CIK)16

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Event-free Survival (EFS) Rate

Event-free Survival (EFS) rate will be assessed on all enrolled participants and is defined as the duration of time after cytokine-induced killer (CIK) cell infusion that the participants remain alive with experiencing relapse, Grade 3 to 4 acute graft vs host disease (aGVHD), or death. The outcome will be reported as the number of participants, stratified by receipt of CIK cells, that did not experience a specified event, a number without dispersion. (NCT01392989)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
CIK-infused participantsNon-CIK-infused participants
Allogeneic Cytokine Induced Killer Cells (CIK)144

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Number of Participants That Experience Grade 2 to 4 aGvHD Within 100 Days and 1 Year

"Acute graft vs host disease (aGvHD) Grade 2 to 4 was staged & graded using modified Keystone criteria, as below. The outcome is reported as the number of participants that experience Grade 2 to 4 aGvHD within 100 days and 1 year.~Stage 1: Skin: rash < 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea 500 to 1000 mL/day or persistent nausea with positive biopsy for GvHD~Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea 1000 to 1500 mL/day.~Stage 3: Skin: rash > 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea > 1500 mL/day.~Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin > 15 mg/dL. Gut: severe abdominal pain with or without ileus Grade of aGvHD was determined as follows.~Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage~Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut~Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut~Grade 4: Stage 4 Skin + or Stage 2" (NCT01392989)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
All participants within 100 daysAll participants within 1 yearCIK-infused participants within 100 daysCIK-infused participants within 1 yearNon-CIK-infused participants within 100 daysNon-CIK-infused participants within 1 year
Allogeneic Cytokine Induced Killer Cells (CIK)9113566

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Drug Discontinuation Due to Side Effects

Drug discontinuation due to drug side effects regarding Cellcept and Myfortic. (NCT00336817)
Timeframe: 12 weeks

InterventionParticipants (Count of Participants)
Myfortic Group1
CellCept Group2

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Incidence of Biopsy-proven Acute Cellular Rejection During the Study Period

number of patients with ACR (NCT00336817)
Timeframe: 12 weeks

Interventionparticipants (Number)
Myfortic Group2
CellCept Group1

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Incidence of Cytomegalovirus Infection or Disease During the Study Period

number of participants (NCT00336817)
Timeframe: 12 weeks

Interventionparticipants (Number)
Myfortic Group0
CellCept Group0

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Incidence of Graft Loss or Death During the Study Period

number of patients (NCT00336817)
Timeframe: 12 weeks

Interventionparticipants (Number)
Myfortic Group0
CellCept Group0

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Number of Participants With Bone Marrow Suppression

Number of participants with: Thrombocytopenia (<50,000 mm3), Leukopenia (< 2000 mm3), absolute neutrophils count ( <1000 mm3) or hemoglobin ( < 7.0 g/dL) (NCT00336817)
Timeframe: 12 weeks

Interventionparticipants (Number)
Myfortic Group0
CellCept Group0

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Number of Participants With Clinically Significant Decrease in Serum Creatinine From Baseline Through Week 12

Creatinine levels (NCT00336817)
Timeframe: 12 weeks

Interventionparticipants (Number)
Myfortic Group0
CellCept Group0

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Number of Participants With Neurotoxicity

(NCT00336817)
Timeframe: 12 weeks

Interventionparticipants (Number)
Myfortic Group0
CellCept Group0

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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil

"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms. The total GSRS range of scores is 15 to 105 with higher scores meaning the worst of symptoms." (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks

,
Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
CellCept Group23.0722.1426.0725.21
Myfortic Group21.8024.9028.9021.80

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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale)

"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~Constipation subscale range is 3 to 21 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks

,
Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
CellCept Group4.074.214.794.54
Myfortic Group4.504.606.504.50

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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale)

"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~The Diarrhea subscale range is 3 to 21 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks

,
Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
CellCept Group4.794.364.505.29
Myfortic Group4.005.806.603.70

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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale)

"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~The Indigestion subscale range is 4 to 28 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks

,
Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
CellCept Group5.866.437.506.86
Myfortic Group5.405.806.505.60

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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale)

"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~The abdominal Pain subscale range is 3 to 21 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks

,
Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
CellCept Group5.294.795.935.36
Myfortic Group5.205.806.405.30

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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale)

"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~Reflux subscale range is 2 to 14 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks

,
Interventionunits on a scale (Mean)
screening2 weeks6 weeks12 weeks
CellCept Group3.072.363.363.07
Myfortic Group2.702.902.902.70

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Mean Change From Baseline in Forced Vital Capacity (FVC)

compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration. (NCT00333437)
Timeframe: Baseline, 12 months

InterventionLiters (Mean)
Treatment0.1786

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Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)

DLCO was measured before beginning and after completion of study therapy (NCT00333437)
Timeframe: 12 months

InterventionLiters (Mean)
Treatment1.86

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Mean Change in Six Minute Walk Distance

Comparison of 6-minute walk distance before beginning and after completing study therapy (NCT00333437)
Timeframe: 12 months

InterventionFeet (Mean)
Treatment264.3

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Change in Shortness of Breath (Self-reported)

Participants reported frequency of shortness of breath experienced with exertion (NCT00333437)
Timeframe: Baseline, 12 months

Interventionparticipants (Number)
Patient-reported less shortness of breathPatient-reported no change in shortness of breathPatient-reported increased shortness of breath
Treatment610

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Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils)

BAL samples were colleected from the affected lobe (as determined by lung CT scans) before beginning and after completing study therapy. (NCT00333437)
Timeframe: Baseline, 12 months

InterventionCells/uL (Mean)
Mean change in neutrophil countMean change in eosinophil count
Treatment-3-6.3

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Number of Patients Who Engrafted

Continued engraftment will be defined as the detection of donor T-cells (CD3+) as a proportion of the total T-cell of greater than 5%. (NCT00397813)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Arm A - Dose Level 135
Arm A - Dose Level 20
Arm A - Dose Level 30
Arm B - Dose Level 112
Arm B - Dose Level 24
Arm B - Dose Level 324

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Number of Patients Who Had Infections

Number of patients who experienced bacterial, fungal, or viral infections. (NCT00397813)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Arm A - Dose Level 124
Arm A - Dose Level 20
Arm A - Dose Level 30
Arm B - Dose Level 112
Arm B - Dose Level 24
Arm B - Dose Level 324

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Number of Patients With HCT Failure.

HCT failure will be defined as graft rejection (defined as < 5% donor T-cell chimerism) or disease progression within 200 days of transplant. (NCT00397813)
Timeframe: 200 days

InterventionParticipants (Count of Participants)
Arm A - Dose Level 14
Arm A - Dose Level 20
Arm A - Dose Level 30
Arm B - Dose Level 15
Arm B - Dose Level 23
Arm B - Dose Level 32

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Number of Patients With Progression-free Survival

Evidence of disease progression will be an indication for therapeutic intervention. Defined as any evidence by morphologic or flow cytometric evaluation of the bone marrow aspirate of an incremental increase in 5% blasts in MDS/CMML; defined as any evidence of blastic transformation in agnogenic myeloid metaplasia/atypical CML; and defined as progressive erythrocytosis, thrombocytosis, or evidence of leukemic transformation in polycythemia vera and essential thrombocythemia. (NCT00397813)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Arm A - Dose Level 124
Arm A - Dose Level 20
Arm A - Dose Level 30
Arm B - Dose Level 11
Arm B - Dose Level 22
Arm B - Dose Level 311

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Number of Patients With Relapse/Progression

Evidence of disease progression will be an indication for therapeutic intervention. Defined as any evidence by morphologic or flow cytometric evaluation of the bone marrow aspirate of an incremental increase in 5% blasts in MDS/CMML; defined as any evidence of blastic transformation in agnogenic myeloid metaplasia/atypical CML; and defined as progressive erythrocytosis, thrombocytosis, or evidence of leukemic transformation in polycythemia vera and essential thrombocythemia. (NCT00397813)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Arm A - Dose Level 16
Arm A - Dose Level 20
Arm A - Dose Level 30
Arm B - Dose Level 16
Arm B - Dose Level 23
Arm B - Dose Level 36

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Graft Failure

Number of patients with graft failure (grade IV thrombocytopenia and neutropenia after day 21 that lasts > 2 weeks andn is refractory to growth factor support). (NCT00358657)
Timeframe: Day 84

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)0

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Graft Rejection

Number of patients with graft rejection (CD3 donor chimerisms <5%). (NCT00358657)
Timeframe: Day 84

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)1

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Immune Reconstitution

Number of patients with normal range CD3 @ 1 year post transplant (NCT00358657)
Timeframe: 1 year post transplant

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)4

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Incidence of Chronic GVHD

Number of patients diagnosed with chronic GVHD by 1 year post transplant (NCT00358657)
Timeframe: 1 year post transplant

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)11

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Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)

Number of patients diagnosed with overall GI/G2 acute GVHD by Day 100 (NCT00358657)
Timeframe: Day 100 post transplant

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)7

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Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)

Number of patients diagnosed with overall GIII/IV acute GVHD by Day 100 (NCT00358657)
Timeframe: Day 100 post transplant

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)5

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Number of Patients With Infections

Number of patients with clinically significant infections requiring treatment within 200 days after HCT (NCT00358657)
Timeframe: Through day 200 after HCT

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)11

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Proportion of Patients Who Achieve Greater Than 5% Donor T-cell Chimerism

Number of patients who achieve greater than 5% donor T-cell (CD3+) chimerisms (NCT00358657)
Timeframe: By day 84

InterventionParticipants (Count of Participants)
Treatment (Chemo, Total-body Irradiation, Transplant)13

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Incidence of Acute Graft-vs-host Disease (aGVHD)

incidence of aGVHD (grades 2 - 4) 100 days post allogeneic hematopoietic cell transplantation (NCT00360685)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
Tacrolimus And Methotrexate45
Tacrolimus And Mycophenolate Mofetil33

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Incidence of Severe Mucositis

Mucositis was assessed prospectively daily while the patient was hospitalized and graded retrospectively based on nurse and clinician assessments according to the clinical criteria set forth in the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 3.0). Severe mucositis as defined as grade 3 or grade 4. (NCT00360685)
Timeframe: 2 year

Interventionparticipants (Number)
Tacrolimus And Methotrexate25
Tacrolimus And Mycophenolate Mofetil14

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Overall Survival

number of participants alive at one year (NCT00360685)
Timeframe: 1 year

Interventionparticipants (Number)
Tacrolimus And Methotrexate28
Tacrolimus And Mycophenolate Mofetil27

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GI Toxicities

Hospitalizations due to Gastrointestinal (GI) toxicities of mycophenolic acid enteric coated (Myfortic) (NCT00374803)
Timeframe: 12 months

Interventionparticipants (Number)
Mycophenolic Acid Loading Group5
Mycophenolic Acid No Load Group4

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Incidence of All Biopsy Proven Acute Rejection.

Treatment efficacy, defined as the incidence of all biopsy proven acute rejection. Biopsy was proven with tissue samples collected on patients with elevated serum creatinine (NCT00374803)
Timeframe: 12 months

InterventionParticipants (Number)
Mycophenolic Acid Loading Group1
Mycophenolic Acid No Load Group4

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Incidence of Post Transplant Infections

Incidence of post transplant infections that resulted in hospitalization (NCT00374803)
Timeframe: 12 months

Interventionparticipants (Number)
Mycophenolic Acid Loading Group4
Mycophenolic Acid No Load Group4

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Renal Function at 12 Months

Renal function measured by serum creatinine (SCr) at 12 months post-transplant (NCT00374803)
Timeframe: 12 months

Interventionmg/dL (Mean)
Mycophenolic Acid Loading Group1.36
Mycophenolic Acid No Load Group1.5

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Patient and Allograft Survival 12 Months

Patient and allograft survival at 12 months post-transplant. Allograft survival is different from rejection. An allograft can have rejection, but the allograft can still have survival. If an allograft fails and is no longer functioning this would be considered allograft failure and non-survival. (NCT00374803)
Timeframe: 12 months

,
Interventionparticipants (Number)
Patient SurvivalAllograft Survival
Mycophenolic Acid (Myfortic) Preload2222
Mycophenolic Acid (Myfortic) Standard2322

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Incidence of Biopsy Confirmed Acute Rejection at 12 Months.

(NCT00374231)
Timeframe: 12 months

Interventionparticipants (Number)
Corticosteroid Withdrawa.5

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Patient Survival.

(NCT00374231)
Timeframe: 12 months

Interventionparticipants (Number)
Corticosteroid Withdrawal39

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Time Post Transplant Corticosteroid Withdrawal

The mean days from post transplant corticosteroid withdrawal. (NCT00374231)
Timeframe: 12 months

Interventiondays (Mean)
Corticosteroid Withdrawal257

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Survival at 1 Year After Transplantation

The number of patients survival status 1 year after transplantation (NCT00387959)
Timeframe: 1 Year after transplant

Interventionparticipants (Number)
Alive at 1 Year Post TransplantDied Prior to 1 Year Post Transplant
Unrelated Donor Umbilical Cord Transplant106

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Engraftment Rates

To assess hematopoietic engraftment rates. (NCT00429143)
Timeframe: 6 months

Interventionparticipants (Number)
Haploidentical Allogeneic Transplantation23

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Incidence of Grades III-IV GVHD

"To determine the incidence and severity of GVHD in these patients using a combination of cyclophosphamide, tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis.'~Severity was graded using CTCAE 3.0 (1=mild, 2=moderate, 3=severe, 4=life threatening/disabling, 5=death)" (NCT00429143)
Timeframe: 6 months

Interventionparticipants (Number)
Haploidentical Allogeneic Transplantation2

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Lymphoid Recovery

To assess the pace of lymphoid recovery in this patient population. (NCT00429143)
Timeframe: 6 months

Interventionparticipants (Number)
Haploidentical Allogeneic Transplantation23

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Optimal Dose of CD3+ Donor Lymphocytes (T-cells) for Consistent Engraftment Without GVHD

"To determine the optimal dose of CD3+ donor lymphocytes required for consistent engraftment without the development of grade III/IV GVHD.~Measured as CD3+ donor lymphocytes given as n x 10^8/kg.~n was found to be 2 and was found to be the optimal dose and was the only dose given." (NCT00429143)
Timeframe: 6 months

Interventionlymphocytes x 10^8/kg (Number)
Haploidentical Allogeneic Transplantation2

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Overall Survival of Participants

To determine overall survival at 6 months post-transplant. (NCT00429143)
Timeframe: 6 months

Interventionparticipants (Number)
Haploidentical Allogeneic Transplantation13

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Complete Donor Hematopoietic Cell Chimerism

Complete donor hematopoietic cell chimerism was evaluated in transplant recipients. Complete donor chimerism will be assessed as the presence of > 95% donor T-cells (CD3+) in the blood. The outcome is reported as the percentage of participants that achieve complete donor chimerism, a number without dispersion. (NCT00568633)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Allo-HSCT + TLI + ATG56

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Disease-free Survival (DFS)

Disease-free survival is defined as the time interval between the date of attaining a first complete remission (CR) and the date of relapse. Disease free survival (DFS) will compared to conventional therapy vs Non-myeloablative Host Conditioning (NMA HCT). The outcome is reported as the number of participants which never experienced disease relapse (without dispersion). (NCT00568633)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Allo-HSCT + TLI + ATG5
Best Standard Care9

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Early Graft Loss

Early graft loss means a failure to achieve donor T-cell chimerism of > 5% at any time after transplant. The outcome is reported as the percentage of participants that experience early graft loss, a number without dispersion. (NCT00568633)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Allo-HSCT + TLI + ATG4

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Non-relapse Mortality

Non-relapse mortality is defined as death that occurs after therapy, from any cause except a cause associated with relapse. This will be reported as the number of participants experiencing non-relapse mortality (a number without dispersion). (NCT00568633)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Allo-HSCT + TLI + ATG1
Best Standard Care2

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Overall Survival (OS)

Overall survival defined as the time interval between the date of attaining a first complete remission (CR) and the date of death from any cause. The outcome is reported as the number of participants alive (without dispersion). (NCT00568633)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Allo-HSCT + TLI + ATG9
Best Standard Care13

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Patients Completing the Intended Therapy in Both Arms

The assessment for completion of the intended therapy (in both arms) will be reported as the percentage of participants, a number without dispersion (NCT00568633)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Allo-HSCT + TLI + ATG100
Best Standard Care81.8

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Relapse Rate

Relapse will be determined as ≥ 5% blast cells in the bone marrow, not secondary to regeneration after myelosuppressive therapy; OR emergence of extramedullary leukemia; OR the re-emergence of blasts in the peripheral blood. The outcome will be reported as the number and percentage of participants that meet these criteria (a number without dispersion). (NCT00568633)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Allo-HSCT + TLI + ATG20
Best Standard Care24

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Donor Chimerism at 1 Year

Number of participants with full (95-100%), mixed (5-94%), and no (0-4%) donor cells. Chimerism is reported for unsorted whole blood and T cells. (NCT00489281)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Whole blood72092237Whole blood72092236T cells72092237T cells72092236
95-100%Unknown or not measured5-94%0-4%
Transplant - 200 cGy6
Transplant - 400 cGy9
Transplant - 200 cGy9
Transplant - 400 cGy3
Transplant - 200 cGy1
Transplant - 400 cGy1
Transplant - 200 cGy7
Transplant - 400 cGy10
Transplant - 400 cGy2
Transplant - 200 cGy0
Transplant - 400 cGy0
Transplant - 200 cGy13

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Donor Chimerism at 30 Days

Number of participants with full (95-100%), mixed (5-94%), and no (0-4%) donor cells. Chimerism is reported for unsorted whole blood and T cells. (NCT00489281)
Timeframe: 30 days

InterventionParticipants (Count of Participants)
Whole blood72092236Whole blood72092237T cells72092236T cells72092237
95-100%5-94%0-4%Unknown or not measured
Transplant - 200 cGy12
Transplant - 400 cGy8
Transplant - 200 cGy15
Transplant - 400 cGy3
Transplant - 200 cGy1
Transplant - 400 cGy1
Transplant - 200 cGy4
Transplant - 400 cGy4
Transplant - 200 cGy18
Transplant - 200 cGy5
Transplant - 400 cGy0
Transplant - 200 cGy2

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Belatacept Pharmacokinetic (PK) Parameter: Maximum Serum Concentration

Maximum Plasma Concentration (Cmax) is the maximum observed serum drug concentration. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

Interventionµg/mL (Geometric Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF221.3
Group 2: Belatacept (MI) + MMF227.6
Group 3: Belatacept (LI) + MMF205.4

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Belatacept Pharmacokinetic (PK) Parameter: Time to Achieve the Maximum Plasma Concentration

Maximum Plasma Concentration (Tmax) is the time taken to reach the maximum observed plasma concentration. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

Interventionhour (Median)
Group 1: Basiliximab+Belatacept (MI) + MMF1.00
Group 2: Belatacept (MI) + MMF1.08
Group 3: Belatacept (LI) + MMF1.00

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Belatacept PK Parameter: Amount Excreted in Ascites Fluid Over Days 1 to 14

Amount Excreted in Ascites (Ae,asc) was estimated from the ascites drug concentrations and volumes within a dosing interval. (NCT00555321)
Timeframe: Days 1 to 14

Interventionµg (Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF46654
Group 2: Belatacept (MI) + MMF31425
Group 3: Belatacept (LI) + MMF81451

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Belatacept PK Parameter: Area Under the Serum Concentration-time Curve to the End of the Dosing Period (AUCtau)

Area under the plasma concentration-time curve for each dosing interval is determined using the linear trapezoidal rule. The AUC(TAU) of belatacept from the MI regimens and LI regimens were calculated over 2 and 4 weeks respectively. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

Interventionµg*h/mL (Geometric Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF19865
Group 2: Belatacept (MI) + MMF21526
Group 3: Belatacept (LI) + MMF19730

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Belatacept PK Parameter: Minimum Plasma Concentration

Minimum Plasma Concentration (Cmin) is the minimum observed serum drug concentration. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

Interventionµg/mL (Geometric Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF23.63
Group 2: Belatacept (MI) + MMF27.20
Group 3: Belatacept (LI) + MMF5.91

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Belatacept PK Parameter: Terminal Half-life

Terminal Half-life (T 1/2) is the time a drug takes for the concentration levels to fall to 50% of their value. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

Interventionhour (Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF240.80
Group 2: Belatacept (MI) + MMF227.74
Group 3: Belatacept (LI) + MMF207.88

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Belatacept PK Parameter: Total Body Clearance

Total body clearance is the rate and extent at which the drug is eliminated from the body. The clearance of a drug is used to understand the processes involved in drug elimination, distribution and metabolism. CLT was estimated from AUC (TAU) between Weeks 12 and 16, assuming steady state. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

InterventionmL/h/kg (Geometric Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF0.45
Group 2: Belatacept (MI) + MMF0.41
Group 3: Belatacept (LI) + MMF0.45

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Belatacept PK Parameter: Volume of Distribution

Volume of distribution (Vss) is the volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration. . Vss was estimated from AUC (TAU) between Weeks 12 and 16, assuming steady state. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.

InterventionL/kg (Mean)
Group 1: Basiliximab+Belatacept (MI) + MMF0.09
Group 2: Belatacept (MI) + MMF0.08
Group 3: Belatacept (LI) + MMF0.11

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Percentage of Participants Surviving With Functional Graft by End of Study (Includes LTE Data)

For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)

Interventionpercentage of participants (Number)
Group 1: Basiliximab+Belatacept (MI) + MMF93.3
Group 2: Belatacept (MI) + MMF85.2
Group 3: Belatacept (LI) + MMF95.8
Group 4: Tacrolimus + MMF92.1
Group 5: Tacrolimus96.2

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Percentage of Participants Who Experienced at Least One Episode of Acute Rejection (AR), Graft Loss, or Death by 6 Months Post-transplant

Any AR that was clinically suspected and biopsy proven (by central pathologist) was included in this triple composite end point. All biopsies for suspected AR were assessed by a blinded central histopathologist using Banff grading schema. Graft loss was defined as impairment of liver function to such a degree that the participant died or underwent re-transplantation. For 95% (confidence interval) CI within each group, normal approximation is used if N>=5, otherwise exact method is used. (NCT00555321)
Timeframe: At 6 months posttransplant

Interventionpercentage of participants (Number)
Group 1: Basiliximab+Belatacept (MI) + MMF48.0
Group 2: Belatacept (MI) + MMF41.7
Group 3: Belatacept (LI) + MMF46.9
Group 4: Tacrolimus + MMF15.1
Group 5: Tacrolimus38.0

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Percentage of Participants Who Experienced at Least One Episode of Acute Rejection, Graft Loss, or Death by 12 Months

Any AR that was clinically suspected and biopsy proven (by central pathologist) was included in this triple composite end point. All biopsies for suspected AR were assessed by a blinded central histopathologist using Banff grading. Graft loss was defined as impairment of liver function to such a degree that the participant died or underwent re-transplantation. For 95% CI within each group, normal approximation is used if N>=5. Otherwise exact method is used. For 95% CI of difference, adjustment is made for randomization strata if N >= 5 in each treatment arm. (NCT00555321)
Timeframe: At 12 months posttransplant

Interventionpercentage of participants (Number)
Group 1: Basiliximab+Belatacept (MI) + MMF52.0
Group 2: Belatacept (MI) + MMF47.9
Group 3: Belatacept (LI) + MMF53.1
Group 4: Tacrolimus + MMF18.9
Group 5: Tacrolimus40.0

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Percentage of Participants Who Experienced at Least One Episode of Acute Rejection, Graft Loss, or Death by End of Study (Includes LTE Data)

Any AR that was clinically suspected and biopsy proven (by central pathologist) was included in this triple composite end point. All biopsies for suspected AR were assessed by a central histopathologist using Banff criteria. For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)

Interventionpercentage of participants (Number)
Group 1: Basiliximab+Belatacept (MI) + MMF36.7
Group 2: Belatacept (MI) + MMF37.0
Group 3: Belatacept (LI) + MMF25.0
Group 4: Tacrolimus + MMF21.1
Group 5: Tacrolimus23.1

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Belatacept Trough Concentration Before Each Infusion During the LTE

Minimum Plasma Concentration (Cmin) is the minimum observed serum drug concentration. (NCT00555321)
Timeframe: Samples were collected predose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105, 532, 728.

,,
Interventionµg/mL (Geometric Mean)
Day 5: (n=40, 43, 41)Day 14: (n=41, 43, 38)Day 28: (n=42, 38, 37)Day 56: (n=12, 13, 12)Day 84: (n=34, 31, 34 )Day 112: (n=29, 26, 29)Day 168: (n=30, 29, 29)Day 252: (n=28, 28, 27)Day 336: (n=26, 23, 21)Day 364: (n=28, 28, 22 )Day 532: (n=22, 24, 14)Day 728: (n=10, 12, 14)
Group 1: Basiliximab+Belatacept (MI) + MMF79.1532.3923.5118.9127.1110.127.303.653.222.522.714.75
Group 2: Belatacept (MI) + MMF79.4031.1622.2318.8427.329.648.013.743.384.014.684.20
Group 3: Belatacept (LI) + MMF70.8829.2121.726.576.606.753.573.594.373.624.914.22

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Glycosylated Hemoglobin (HbA1C) Values: 12-month Treatment Phase

The HbA1c test is important in diabetes as a long-term measure of control over blood glucose, where the glucose bound to hemoglobin during the past 3-4 months is measured. A baseline diabetes participant was one who had a medical history of diabetes or being under anti-diabetic medication at the time of the transplantation. BL = baseline, DM = Diabetes mellitus. (NCT00555321)
Timeframe: 6, 12 months (mth) posttransplant

,,,,
InterventionPercentage of glycosylated hemoglobin (Mean)
6 mo: All participants (n=39, 34, 38, 47, 35)6 mo: DM at BL (n=15, 12, 10, 12, 10)6 mo: DM at BL/DM at 6m (n=23, 14, 14, 19, 18)12 mo: All participants (n=40, 35, 35, 44, 37)12 mo: DM at BL (n=15, 11, 7, 11, 12)12 mo: DM at BL/DM at 12 mth (n=23,13 ,11,18,20)
Group 1: Basiliximab+Belatacept (MI) + MMF5.86.56.16.06.96.3
Group 2: Belatacept (MI) + MMF5.86.66.45.86.76.6
Group 3: Belatacept Less Intensive (LI) + MMF5.66.15.95.76.76.2
Group 4: Tacrolimus + MMF5.45.85.55.56.26.0
Group 5: Tacrolimus5.45.45.45.86.56.3

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Mean Change From Baseline in Calculated GFR During the LTE

GFR is a measure of the rate at which blood is filtered by the kidney. MDRD is an equation (calculation) used to estimate GFR in participants with impaired renal function based on serum creatinine (ScR), age, race, gender, blood urea nitrogen (BUN), and albumin (Alb). GFR (mL/min/1.73 m^2) = 170*(Scr)^-0.999*(Age)^-0.176*(0.762 if female)*(1.180 if African American)*(BUN)^-0.170*(Alb)^+0.318. ). BL= baseline, mL= milliliters; min= minute; m^2= meters squared. (NCT00555321)
Timeframe: Baseline (pretransplant time point), 1, 2, 3, 6,12, 18, 24, 30, 36 months posttransplant

,,,,
InterventionmL/min/1.73 m^2 (Mean)
Baseline (BL); (n=27,24,20,33,23)1 month; (n=28,27,23,36,25)Change from BL to 1 month; (n=26,24,20,31,23)2 months; (n=21,21,15,19,19)Change from BL to 2 months; (n=20,19,14,17,18)3 months; (n=27, 26, 24, 35, 24)Change from BL to 3 months; (n=24, 24, 20, 30, 21)6 months; (n=29, 26, 24, 24, 38, 23)Change from BL to 6 months; (n=27, 23, 20, 33, 20)12 months; (n=29, 25, 24, 36, 24)Change from BL to 12 months; (n=26, 23, 20, 31,21)18 months; (n=24, 25, 21, 33, 21)Change from BL to 18 months; (n=21, 23, 17, 28,18)24 months; (n=13, 15, 15, 22, 15)Change from BL to 24 months; (n=10, 13, 11, 19,13)At 30 months; (n=6, 6, 6, 12, 7)Change from BL to 30 months; (n=5, 5, 3, 8, 6)
Group 1: Basiliximab+Belatacept (MI) + MMF64.488.226.386.226.087.023.387.624.088.122.684.822.584.827.9102.941.4
Group 2: Belatacept (MI) + MMF85.6103.120.299.412.697.013.793.18.6101.319.495.011.287.69.994.440.4
Group 3: Belatacept (LI) + MMF78.095.116.0102.330.797.017.092.312.294.816.591.37.496.315.290.417.1
Group 4: Tacrolimus + MMF77.968.6-7.671.2-11.666.5-8.164.6-10.967.8-9.369.0-6.873.3-2.264.1-15.0
Group 5: Tacrolimus83.059.1-22.664.4-17.457.0-23.556.7-23.361.7-20.263.2-6.166.32.359.5-10.7

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Mean Change From Baseline in Calculated GFR, by Modification of Diet in Renal Disease (MDRD) Equation: 12-month Treatment Phase

GFR is a measure of the rate at which blood is filtered by the kidney. MDRD is an equation (calculation) used to estimate GFR in participants with impaired renal function based on serum creatinine (ScR), age, race, gender, blood urea nitrogen (BUN), and albumin (Alb). GFR (mL/min/1.73 m^2) = 170*(Scr)^-0.999*(Age)^-0.176*(0.762 if female)*(1.180 if African American)*(BUN)^-0.170*(Alb)^+0.318. ). BL= baseline, mL= milliliters; min= minute; m^2= meters squared. (NCT00555321)
Timeframe: Baseline [BL] (pretransplant time point), 1, 2, 3, 6 and 12 months posttransplant

,,,,
InterventionmL/min/1.73 m^2 (Mean)
BL; (n=47, 42, 42, 45, 45)1 month; (n=45, 44, 42, 49, 49)Change from BL to 1 month; (n=43, 38, 37, 41, 45)2 months; (n=29, 30, 23, 25, 27)Change from BL to 2 months; (n=28, 26, 20, 22, 25)3 months; (n=36, 36, 35, 46, 37)Change from BL to 3 months; (n=33, 34, 30, 40, 33)6 months; (n=39, 34, 38, 49, 36)Change from BL to 6 months; (n=37, 30, 33, 42, 31)12 months; (n=40, 33, 35, 46, 38)Change from BL to 12 month; (n=37, 29, 30, 41, 33)
Group 1: Basiliximab+Belatacept (MI) + MMF66.385.621.086.125.386.522.082.218.683.818.2
Group 2: Belatacept (MI) + MMF77.493.115.396.618.391.713.390.37.697.719.2
Group 3: Belatacept (LI) + MMF76.689.611.4105.831.596.618.386.05.685.64.2
Group 4: Tacrolimus + MMF73.764.9-8.776.3-1.565.0-8.461.9-11.768.4-6.3
Group 5: Tacrolimus80.262.2-17.766.9-14.060.4-20.259.8-22.763.8-17.1

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Mean Change From Baseline in Measured Glomerular Filtration Rate (GFR): 12-month Treatment Phase

"GFR was assessed using a true measure of glomerular filtration via iothalamate clearance test. The month 2 time point was selected as the baseline time point with respect to measured GFR due to logistical difficulty in obtaining measured GFR at the time of liver transplant and post-transplant renal function largely stabilizing by 2 months. All Measured GFR > 200 were truncated at 200." (NCT00555321)
Timeframe: Baseline (2 month), 12 months posttransplant

,,,,
InterventionmL/min/1.73m^2 (Mean)
2 months (n=37, 36, 35, 41, 37)12 months (n=39, 35, 29, 40, 32)Change from 2 to 12 months (n=31, 29, 26, 36, 29)
Group 1: Basiliximab+Belatacept (MI) + MMF72.488.914.2
Group 2: Belatacept (MI) + MMF86.693.15.8
Group 3: Belatacept (LI) + MMF98.673.1-19.6
Group 4: Tacrolimus + MMF65.975.25.3
Group 5: Tacrolimus58.570.57.3

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Mean Change From Baseline Serum Creatinine at Months 1, 2, 3, 6 and 12

Measurement of SCr is commonly used as an indicator of renal function. High creatinine blood level is an indicator of deficient filtering by the kidney. SCr was determined at baseline and various post-baseline time points. (NCT00555321)
Timeframe: Baseline (pretransplant time point), 1, 2, 3, 6 and 12 months posttransplant

,,,,
Interventionmg/dL (Mean)
Baseline (BL); (n=47, 42, 42, 45, 45)At 1 months; (n=45, 44, 42, 49, 49)Change from BL to 1 months; (n=43, 38, 37, 41, 45)At 2 months; (n=29, 30, 23, 25, 27)Change from BL to 2 months; (n=28, 26, 20, 22, 25)At 3 months; (n=36, 36, 35, 46, 37)Change from BL to 3 months; (n=33, 34, 30, 40, 33)At 6 months; (n=39, 34, 38, 49, 36)Change from BL to 6 months; (n=37, 30, 33, 42, 31)At 12 months; (n=40, 33, 35, 46, 38)Change from BL to 12 months; (n=37, 29, 30, 41,33)
Group 1: Basiliximab+Belatacept (MI) + MMF1.40.9-0.50.9-0.70.9-0.61.0-0.51.0-0.5
Group 2: Belatacept (MI) + MMF1.00.8-0.10.8-0.20.9-0.10.90.00.8-0.2
Group 3: Belatacept (LI) + MMF0.90.9-0.10.8-0.20.8-0.10.90.11.00.1
Group 4: Tacrolimus + MMF1.11.20.11.00.01.20.11.30.21.20.1
Group 5: Tacrolimus1.01.30.31.20.21.30.31.30.41.20.3

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Mean Change in Baseline Values of Cystatin C at 2 and 12 Months

Cystatin C is a protein encoded by the CST3 gene, which is mainly used as a biomarker of kidney function. If kidney function and glomerular filtration rate decline, the blood levels of cystatin C rise. (NCT00555321)
Timeframe: Baseline (pretransplant), 2, and 12 months posttransplant

,,,,
Interventionmg/L (Mean)
Baseline (BL); (n=44, 47, 48, 49, 43)2 months; (n=40, 39, 39, 48, 43)Change from BL to 2 months; (n=35, 38, 38, 44,37)12 months; (n=40, 36, 36, 49, 40)Change from BL to 12 months; (n=35, 35, 35,45,34)
Group 1: Basiliximab+Belatacept (MI) + MMF1.21.1-0.21.1-0.2
Group 2: Belatacept (MI) + MMF1.21.1-0.10.9-0.2
Group 3: Belatacept (LI) + MMF1.11.0-0.11.20.1
Group 4: Tacrolimus + MMF1.41.40.11.3-0.1
Group 5: Tacrolimus1.21.50.31.30.1

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Number of Participants (Who Were HCV Positive at Baseline) With HCV Ribonucleic Acid (RNA) Levels >2.4*10^6 U/mL and >4.7*10^6 U/mL: 12-month Treatment Phase

Recurrent hepatitis C infection of the allograft following liver transplantation can be detected by monitoring HCV RNA levels. In HCV positive participants, quantitative HCV RNA levels > 2.4 * 10^6 U/mL and > 4.7 * 10^6 U/mL were descriptively summarized by treatment group. BL=baseline (NCT00555321)
Timeframe: Baseline (pretransplant), 6 and 12 months (mo) posttransplant

,,,,
Interventionparticipants (Number)
BL; HCV RNA > 2.4*10^6 U/mL (n=21,22,19,22,23)BL; HCV RNA > 4.7 *10^6 U/mL (n=21,22,19,22,23)6 mo; HCV RNA > 2.4*10^6 U/mL (n=16,15,15,21,14)6 mo; HCV RNA > 4.7*10^6 U/mL (n=16,15,15,21,14)12 mo; HCV RNA > 2.4*10^6 U/mL (n=15,13,14,20,13)12 mo; HCV RNA > 4.7*10^6 U/mL (n=15,13,14,20,13)
Group 1: Basiliximab+Belatacept (MI) + MMF209776
Group 2: Belatacept (MI) + MMF417776
Group 3: Belatacept Less Intensive (LI) + MMF218775
Group 4: Tacrolimus + MMF3014111311
Group 5: Tacrolimus10111077

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Number of Participants (Who Were HCV Positive at Baseline) With HCV RNA Levels >2.4 * 10^6 U/mL and >4.7 * 10^6 U/mL During the LTE

Recurrent hepatitis C infection of the allograft following liver transplantation can be detected by monitoring HCV RNA levels. In HCV positive participants, quantitative HCV RNA levels > 2.4 x 10^6 U/mL and > 4.7 x 10^6 U/mL were descriptively summarized by treatment group. BL = baseline (NCT00555321)
Timeframe: BL (pretransplant), 12, 18, 24, 30 months (mo) posttransplant

,,,,
Interventionparticipants (Number)
BL; HCV RNA > 2.4*10^6 U/mL (n=10,11,6,15,10)12 mo; HCV RNA > 2.4*10^6 U/mL (n=11,10,8,16,10)12 mo; HCV RNA > 4.7*10^6 U/mL (n=11,10,8,16,10)18 mo; HCV RNA > 2.4*10^6 U/mL (n=7,10,3,12,9)18 mo; HCV RNA > 4.7*10^6 U/mL (n=7,10,3,12,9)24 mo; HCV RNA > 2.4*10^6 U/mL (n=4,3,4,8,4)24 mo; HCV RNA > 4.7*10^6 U/mL (n=4,3,4,8,4)30 mo; HCV RNA > 2.4*10^6 U/mL (n=1,1,1,2,2)30 mo; HCV RNA > 4.7*10^6 U/mL (n=1,1,1,2,2)
Group 1: Basiliximab+Belatacept (MI) + MMF066322211
Group 2: Belatacept (MI) + MMF154221000
Group 3: Belatacept Less Intensive (LI) + MMF043221000
Group 4: Tacrolimus + MMF2108766511
Group 5: Tacrolimus144321111

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Number of Participants at Risk of First Acute Rejection as Determined by Kaplan-Meier Method by 12 Months

The time from transplantation to the first AR episode in each treatment arm was summarized using Kaplan-Meier curves. Acute Rejections were clinically suspected and biopsy proven by central pathologist. (NCT00555321)
Timeframe: 3, 6, 9 and 12 months posttransplant

,,,,
Interventionparticipants (Number)
By 3 monthsBy 6 monthsBy 9 monthsBy 12 months
Group 1: Basiliximab+Belatacept (MI) + MMF30272523
Group 2: Belatacept (MI) + MMF31292925
Group 3: Belatacept (LI) + MMF30282723
Group 4: Tacrolimus + MMF48474742
Group 5: Tacrolimus34323228

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Number of Participants Having Acute Rejection by Rejection Activity Index During the LTE

Acute Rejections were clinically suspected and biopsy proven by central pathologist. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI). An overall score of 0-2 is considered indeterminate, score of 3-4 is mild, score of 5-6 is moderate, and score of 7-9 is severe. Only the episode with the highest total RAI score for each participant was counted. (NCT00555321)
Timeframe: Day 1 (randomization) through End of study (database lock of 20-June-2011)

,,,,
Interventionparticipants (Number)
By 12 months; Indeterminate scoreBy 12 months; Mild ScoreBy 12 months; Moderate ScoreBy 12 months; Severe ScoreAt end of study; Indeterminate scoreAt end of study; Mild ScoreAt end of study; Moderate ScoreAt end of study; Severe Score
Group 1: Basiliximab+Belatacept (MI) + MMF08100810
Group 2: Belatacept (MI) + MMF05100510
Group 3: Belatacept (LI) + MMF02400240
Group 4: Tacrolimus + MMF05100510
Group 5: Tacrolimus04020402

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Number of Participants Having Acute Rejections During the LTE

Acute rejections were clinically suspected and biopsy proven by central pathologist. The number of episodes of AR was counted. (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)

,,,,
Interventionparticipants (Number)
By 12 months (m); OverallBy 12 m ; 1 episodeBy 12 m ; 2 episodesBy 12 m; >2 episodesBy Database lock; OverallBy Database lock ; 1 episodeBy Database lock ; 2 episodesBy Database lock ; >2 episodes
Group 1: Basiliximab+Belatacept (MI) + MMF98109810
Group 2: Belatacept (MI) + MMF66006600
Group 3: Belatacept (LI) + MMF65106510
Group 4: Tacrolimus + MMF65106510
Group 5: Tacrolimus65106501

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Number of Participants Having Acute Rejections: 12-month Treatment Phase

Acute rejections were clinically suspected and biopsy proven by central pathologist. The number of episodes of AR was counted. (NCT00555321)
Timeframe: 3 , 6, and 12 months

,,,,
Interventionparticipants (Number)
By 3 months ; OverallBy 3 months ; 1 episodeBy 3 months ; 2 episodesBy 3 months ; >2 episodesBy 6 months ; OverallBy 6 months ; 1 episodeBy 6 months ; 2 episodesBy 6 months ; >2 episodesBy 12 months ; OverallBy 12 months ; 1 episodeBy 12 months ; 2 episodesBy 12 months ; >2 episodes
Group 1: Basiliximab+Belatacept (MI) + MMF171511201811221831
Group 2: Belatacept (MI) + MMF151410151230161330
Group 3: Belatacept (LI) + MMF141400151410161420
Group 4: Tacrolimus + MMF440054107610
Group 5: Tacrolimus131210151320151320

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Number of Participants Who Had Acute Rejection by Banff Grade by 12 Months

Acute Rejections (AR) were clinically suspected and biopsy proven by central pathologist. The Banff grading is a classification of renal allograft pathology and AR. Grade I: AR requiring moderate (>25%) to severe mononuclear cell interstitial infiltrate and moderate tubulitis; Grade II: AR requiring severe tubulitis and/or intimal arteritis; Grade III: AR requiring transmural arteritis. Only the episode with highest Banff grade for each participant was counted. (NCT00555321)
Timeframe: 3, 6 and 12 months posttransplant

,,,,
Interventionparticipants (Number)
By 3 months: Grade 1By 3 months: Grade 2By 3 months: Grade 3By 6 months: Grade 1By 6 months: Grade 2By 6 months: Grade 3By 12 months: Grade 1By 12 months: Grade 2By 12 months: Grade 3
Group 1: Basiliximab+Belatacept (MI) + MMF143016401570
Group 2: Belatacept (MI) + MMF861681781
Group 3: Belatacept (LI) + MMF761771781
Group 4: Tacrolimus + MMF310410610
Group 5: Tacrolimus652762762

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Number of Participants Who Had Adverse Events (AEs), Death, Serious AEs (SAEs) or Were Discontinued Due to AEs (Includes Long Term Extension [LTE] Data)

AE=any new untoward medical occurrence or worsening of a pre-existing medical condition in a subject administered an investigational product and that does not necessarily have a causal relationship with this treatment. SAE=any untoward medical occurrence that results in death, is life-threatening, requires or prolongs inpatient hospitalization (including elective surgery), results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. (NCT00555321)
Timeframe: Day 1 (randomization) to Week 104 + within 56 Days after the last infusion/dose, Deaths were monitored up to database lock (20-June-2011)

,,,,
Interventionparticipants (Number)
SAEsRelated SAEsDiscontinued due to SAEsAEsRelated AEsDiscontinued due to AEsDeaths (due to AE)Deaths (not due to AE)
Group 1: Basiliximab+Belatacept (MI) + MMF18523027220
Group 2: Belatacept (MI) + MMF22942721531
Group 3: Belatacept (LI) + MMF17912320110
Group 4: Tacrolimus + MMF291213833130
Group 5: Tacrolimus21902623000

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Number of Participants Who Had Adverse Events of Special Interest During 12-month Treatment Phase

AE of of special interest included malignancies (including skin carcinomas), infections (viral, cytomegalovirus, herpes, fungal, and bacterial), serious infections (NCT00555321)
Timeframe: Day 1 (randomization) to 12 months or ≤ 56 days after discontinuation of study medication

,,,,
Interventionparticipants (Number)
MalignanciesPost-transplant lymphoproliferative disorderAll InfectionsBacterial infectionsFungal infectionsViral infectionsCytomegalovirus infectionsPolyoma virus infectionsHerpes infectionsHepatitis C virus recurrenceSerious infectionsAutoimmune eventsAcute peri-infusional eventsThrombotic and embolic events
Group 1: Basiliximab+Belatacept (MI) + MMF103256105031411053
Group 2: Belatacept (MI) + MMF003911911413712013
Group 3: Belatacept (LI) + MMF21301114141004613107
Group 4: Tacrolimus + MMF203166940313120NA10
Group 5: Tacrolimus202913571029122NA5

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Number of Participants Who Had AEs of Special Interest During the LTE

AE of special interest included malignancies (including skin carcinomas), infections (viral, cytomegalovirus, herpes, fungal, and bacterial). (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)

,,,,
Interventionparticipants (Number)
MalignanciesInfections and Infestations
Group 1: Basiliximab+Belatacept (MI) + MMF18
Group 2: Belatacept (MI) + MMF46
Group 3: Belatacept (LI) + MMF39
Group 4: Tacrolimus + MMF811
Group 5: Tacrolimus38

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Number of Participants Who Had AEs, Death, SAEs or Were Discontinued Due to AEs: 12-month Treatment Phase

AE=any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant administered an investigational product and that does not necessarily have a causal relationship with this treatment. SAE=any untoward medical occurrence that results in death, is life-threatening, requires or prolongs inpatient hospitalization (including elective surgery), results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event (NCT00555321)
Timeframe: Day 1 (randomization) to 12 m + 8 week follow-up or ≤ 56 days after discontinuation of study medication

,,,,
Interventionparticipants (Number)
DeathSAEsRelated SAEsDiscontinued due to SAEsAEsRelated AEsDiscontinued due to AEs
Group 1: Basiliximab+Belatacept (MI) + MMF42812750457
Group 2: Belatacept (MI) + MMF42911648347
Group 3: Belatacept (LI) + MMF9371411483312
Group 4: Tacrolimus + MMF14016453427
Group 5: Tacrolimus4351913504218

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Number of Participants Who Received Anti-hypertensive Therapy at Month 12

(NCT00555321)
Timeframe: 12 months posttransplant

,,,,
Interventionparticipants (Number)
Received at least 1 medicationReceived 1 medicationReceived 2 medicationsReceived 3 medicationsReceived 4 medications
Group 1: Basiliximab+Belatacept (MI) + MMF2013611
Group 2: Belatacept (MI) + MMF1916210
Group 3: Belatacept Less Intensive (LI) + MMF148240
Group 4: Tacrolimus + MMF2617531
Group 5: Tacrolimus2112531

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Number of Participants With Acute Rejections by Rejection Activity Index (RAI) by 12 Months

Acute Rejections were clinically suspected and biopsy proven by central pathologist. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI). An overall score of 0-2 is considered indeterminate, score of 3-4 is mild, score of 5-6 is moderate, and score of 7-9 is severe. Only the episode with the highest total RAI score for each participant was counted. (NCT00555321)
Timeframe: 3, 6 and 12 months posttransplant

,,,,
Interventionparticipants (Number)
By 3 months; Indeterminate scoreBy 3 months; Mild ScoreBy 3 months; Moderate ScoreBy 3 months; Severe ScoreBy 6 months; Indeterminate scoreBy 6 months; Mild ScoreBy 6 months; Moderate ScoreBy 6 months; Severe ScoreBy 12 months; Indeterminate scoreBy 12 months; Mild ScoreBy 12 months; Moderate ScoreBy 12 months; Severe Score
Group 1: Basiliximab+Belatacept (MI) + MMF014300173001741
Group 2: Belatacept (MI) + MMF086106810781
Group 3: Belatacept (LI) + MMF077007800790
Group 4: Tacrolimus + MMF031004100610
Group 5: Tacrolimus072408340834

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Number of Participants With Central Biopsy Proven Acute Rejection by Treatment Type by 12 Months

Acute rejections were clinically suspected and biopsy proven by central pathologist. Corticosteroid-resistant rejection = Continued rejection, as documented by liver biopsy, after the completion of 2 days of corticosteroids and requiring use of T-cell depleting agent. Refractory rejection=Continued rejection, as documented by liver biopsy, after use of corticosteroids and T cell depletion therapy. Increase in the dose of TAC was monitored in participants who were assigned to one of the TAC-based regimens. TRT= treatment (NCT00555321)
Timeframe: 3, 6 and 12 months posttransplant

,,,,
Interventionparticipants (Number)
By 3 months: Acute rejectionsBy 3 months: Treated participantsBy 3 months: Corticosteroid treatment OnlyBy 3 months: Corticosteroid resistantBy 3 months: RefractoryBy3 months: Initial lymphocyte depleting TRTBy 3 months: Increase in dose of TacrolimusBy 3 months: Other / not availableBy 6 months: Acute rejectionsBy 6 months: Treated participantsBy 6 months: Corticosteroid treatment OnlyBy 6 months: Corticosteroid resistantBy 6 months: RefractoryBy 6 months: Initial lymphocyte depleting TRTBy 6 months: Increase in dose of TacrolimusBy 6 months: Other / not availableBy 12 months: Acute rejectionsBy 12 months: Treated participantsBy 12 months: Corticosteroid treatment OnlyBy 12 months: Corticosteroid resistantBy 12 months: RefractoryB12 months: Initial lymphocyte depleting TRT12 months: Other / not available
Group 1: Basiliximab+Belatacept (MI) + MMF7101000000201212000002212120000
Group 2: Belatacept (MI) + MMF15117112001511611300161271130
Group 3: Belatacept (LI) + MMF14770000015880000016880000
Group 4: Tacrolimus + MMF43200010543000107540000
Group 5: Tacrolimus131090001015121000011152100001

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Number of Participants With Central Biopsy Proven Acute Rejection by Treatment Type During the LTE

Acute rejections were clinically suspected and biopsy proven by central pathologist. Corticosteroid-resistant rejection = Continued rejection, as documented by liver biopsy, after the completion of 2 days of corticosteroids and requiring use of T-cell depleting agent. Refractory rejection=Continued rejection, as documented by liver biopsy, after use of corticosteroids and T cell depletion therapy. Increase in the dose of TAC was monitored in participants who were assigned to one of the TAC-based regimens. DBL=database lock, TRT=treatment (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)

,,,,
Interventionparticipants (Number)
By 12 months: Acute rejectionsBy 12 months: Treated participantsBy 12 months: Corticosteroid treatment OnlyBy 12 months: Corticosteroid resistantBy 12 months: RefractoryBy 12 months: Initial lymphocyte depleting TRTBy 12 months: Other / not availableBy 12 months: Increase in dose of TACBy DBL: Acute rejectionsBy DBL: Treated participantsBy DBL: Corticosteroid treatment OnlyBy DBL: Corticosteroid resistantBy DBL: RefractoryBy DBL: Initial lymphocyte depleting treatmentBy DBL: Other/ Not availableBy DBL: Increase in dose of TAC
Group 1: Basiliximab+Belatacept (MI) + MMF9440000094400000
Group 2: Belatacept (MI) + MMF6540010065400100
Group 3: Belatacept (LI) + MMF6440000064400000
Group 4: Tacrolimus + MMF6540000165400001
Group 5: Tacrolimus6650000066500001

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Number of Participants With Marked Electrolytes, Protein and Metabolic Test Abnormalities During the LTE

Low Serum Potassium: <3.0 meq/L; High serum potassium:>6.0 mEq/L; Low serum magnesium:<0.8 mEq/L; Low serum sodium: <130 mEq/L; High serum sodium: >155 mEq/L; Low inorganic phosphorus: <2.0 mg/dL; High uric acid: >10 mg/dL (NCT00555321)
Timeframe: Every 4 weeks from Week 53 to Week 104.

,,,,
Interventionparticipants (Number)
Low Serum PotassiumHigh Serum PotassiumLow Serum SodiumHigh Serum SodiumLow Inorganic PhosphorusHigh Uric Acid
Group 1: Basiliximab+Belatacept (MI) + MMF011010
Group 2: Belatacept (MI) + MMF012001
Group 3: Belatacept (LI) + MMF001110
Group 4: Tacrolimus + MMF001016
Group 5: Tacrolimus000006

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Number of Participants With Marked Electrolytes, Protein and Metabolic Test Abnormalities: 12-month Treatment Phase

Low total calcium: <7 mg/dL; High total calcium: >12.5 mg/dL ; Low bicarbonate: <11 mEq/L; Low serum potassium: <3.0 mEq/L; High serum potassium:>6.0 mEq/L; High serum magnesium: >2.46 mEq/L; Low serum magnesium:<0.8 mEq/L; Low serum sodium: <130 mEq/L; High serum sodium: >155 mEq/L; Low inorganic phosphorus: <2.0 mg/dL; Low albumin: <2 g/dL; High uric acid: >10 mg/dL (NCT00555321)
Timeframe: Baseline (pretransplant), Weeks 4, 12, 24, and 52

,,,,
Interventionparticipants (Number)
Low Total Calcium (n=48, 47, 44, 53, 50)High Total Calcium (n=48, 47, 44, 53, 50)Low Bicarbonate (n=47, 47, 44, 53, 50)Low Serum Potassium (n=48, 47, 44, 53, 50)High Serum Potassium (n=48, 47, 44, 53, 50)High Serum Magnesium (n=48, 47, 44, 53, 50)Low Serum Magnesium (n=48, 47, 44, 53, 50)Low Serum Sodium (n=48, 47, 44, 53, 50)High Serum Sodium (n=48, 47, 44, 53, 50)Low Inorganic Phosphorus (n=48, 47, 44, 53, 50)Low Albumin (n=47, 47, 45, 53, 50)High Uric Acid (n=48, 47, 44, 53, 50)
Group 1: Basiliximab+Belatacept (MI) + MMF000010230022
Group 2: Belatacept (MI) + MMF100200020211
Group 3: Belatacept (LI) + MMF100110220102
Group 4: Tacrolimus + MMF101120020205
Group 5: Tacrolimus000010120005

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Number of Participants With Marked Hematology Abnormalities During the LTE

Low platelet count: <50*10^9 c/µl; Low leukocytes: <2.0*10^3 c/µl; Low lymphocytes (absolute): <0.5*10^3 c/µl; Low neutrophils (absolute): <1.0*10^3 c/µl. (NCT00555321)
Timeframe: Every 4 weeks from Week 53 to Week 104.

,,,,
Interventionparticipants (Number)
Low Platelets: (n=29, 26, 22, 37, 25)Low Leukocytes : (n=29, 27, 22, 37, 25)Low Absolute Lymphocyte: (n=29, 27, 22, 36, 25)Low Absolute Neutrophils: (n=29, 27, 22, 36, 25)
Group 1: Basiliximab+Belatacept (MI) + MMF1180
Group 2: Belatacept (MI) + MMF2232
Group 3: Belatacept (LI) + MMF0031
Group 4: Tacrolimus + MMF2352
Group 5: Tacrolimus0030

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Number of Participants With Marked Hematology Abnormalities: 12-month Treatment Phase

Low hemoglobin: <8 g/dL; Low platelet count: <50*10^9 C/L; Low leukocytes: <2.0 *10^3 c/µL; Low lymphocytes (absolute): <0.5*10^3 c/µL; Low neutrophils (absolute): <1.0*10^3 Cc/µL. (NCT00555321)
Timeframe: Baseline (pretransplant), 2, 4, 8, 12 weeks, and every 4 weeks for week 16 to 52

,,,,
Interventionparticipants (Number)
Low Hemoglobin: (n=48, 46, 44, 52, 50)Low Platelets: (n=48, 46, 43, 52, 50)Low Leukocytes : (n=48, 46, 44, 52, 50)Low Absolute Lymphocyte: (n=48, 46, 44, 52, 50)Low Absolute Neutrophils : (n=48, 46, 44, 52, 50)
Group 1: Basiliximab+Belatacept (MI) + MMF206296
Group 2: Belatacept (MI) + MMF616184
Group 3: Belatacept (LI) + MMF245264
Group 4: Tacrolimus + MMF024178
Group 5: Tacrolimus623161

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Number of Participants With Marked Liver and Kidney Function Abnormalities During the LTE

ULN= upper limit of normal; Normal ranges are provided by the Central Laboratory and may vary according to sex and age. High alanine aminotransferase (ALT): >5.0*ULN U/L; High aspartate aminotransferase (AST): >5.0*ULN U/L; High direct bilirubin: >3.0*ULN mg/dL; High g-glutamyl transferase (GGT): >5.0*ULN U/L; High total bilirubin: >3.0*ULN mg/dL; High creatinine: > 3.0*ULN mg/dL (NCT00555321)
Timeframe: Every 4 weeks from Week 53 to Week 104.

,,,,
Interventionparticipants (Number)
High ALTHigh ASTHigh Direct BilirubinHigh GGTHigh Total BilirubinHigh Creatinine
Group 1: Basiliximab+Belatacept (MI) + MMF001210
Group 2: Belatacept (MI) + MMF234630
Group 3: Belatacept (LI) + MMF121610
Group 4: Tacrolimus + MMF4431321
Group 5: Tacrolimus332800

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Number of Participants With Marked Liver and Kidney Function Abnormalities: 12-month Treatment Phase

ULN= upper limit of normal; Normal ranges are provided by the central laboratory and may vary according to sex and age. High alkaline phosphatase (ALP): >5.0*ULN U/L; High alanine aminotransferase (ALT): >5.0*ULN U/L; High aspartate aminotransferase (AST): >5.0*ULN U/L; High direct bilirubin: >3.0 * ULN mg/dL; High g-glutamyl transferase (GGT): >5.0*ULN U/L; High total bilirubin: >3.0*ULN mg/dL; High creatinine: > 3.0*ULN mg/dL (NCT00555321)
Timeframe: Baseline (pretransplant), 4, 12, 24, 52 weeks

,,,,
Interventionparticipants (Number)
High ALP: (n=48, 47, 44, 53, 50)High ALT: (n=48, 47, 44, 53, 50)High AST: (n=48, 47, 44, 53, 50)High Direct Bilirubin: (n=48, 47, 44, 53, 50)High GGT: (n=48, 47, 44, 53, 50)High Total Bilirubin: (n=48, 47, 44, 53, 50)High Creatinine: (n=47, 44, 44, 53, 50)
Group 1: Basiliximab+Belatacept (MI) + MMF5169152590
Group 2: Belatacept (MI) + MMF618141824130
Group 3: Belatacept (LI) + MMF619161924160
Group 4: Tacrolimus + MMF41682027120
Group 5: Tacrolimus6921622100

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Percentage of Participants (Who Were Hepatitis C Virus [HCV] Positive at Baseline) With HCV Recurrence (Assessed by Central Pathologist) by 12 Months

HCV Recurrence is defined as Histological confirmation on liver biopsy by the Ishak (modified Knodell) system and required both a score >= 5 out of 18 on modified Histological Activity Index grading and a fibrosis Score >= 2 out of 6 on modified staging. All biopsies, including Week 52 biopsies, were considered. Only the first HCV recurrence episode for each participant was counted. For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. For 95% CI of difference, normal approximation was used if N>=5 in both arms, otherwise exact method was used. (NCT00555321)
Timeframe: 6 and 12 months posttransplant

,,,,
Interventionpercentage of participants (Number)
6 months12 months
Group 1: Basiliximab+Belatacept (MI) + MMF39.160.9
Group 2: Belatacept (MI) + MMF21.730.4
Group 3: Belatacept Less Intensive (LI) + MMF23.828.6
Group 4: Tacrolimus + MMF20.052.0
Group 5: Tacrolimus33.337.5

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Percentage of Participants (Who Were Hepatitis C Virus [HCV] Positive at Baseline) With HCV Recurrence (Assessed by Central Pathologist) During the LTE

HCV Recurrence is defined as Histological confirmation on liver biopsy by the Ishak (modified Knodell) system and required both a score >= 5 out of 18 on modified Histological Activity Index grading and a fibrosis Score >= 2 out of 6 on modified staging. All biopsies, including Week 52 biopsies, were considered. Only the first HCV recurrence episode for each participant was counted. For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. For 95% CI of difference, normal approximation was used if N>=5 in both arms, otherwise exact method was used. (NCT00555321)
Timeframe: 12 months posttransplant, end of study (database lock, 20-June-2011)

,,,,
Interventionpercentage of participants (Number)
At 12 monthsAt end of study
Group 1: Basiliximab+Belatacept (MI) + MMF50.066.7
Group 2: Belatacept (MI) + MMF41.750.0
Group 3: Belatacept Less Intensive (LI) + MMF022.2
Group 4: Tacrolimus + MMF58.864.7
Group 5: Tacrolimus72.772.7

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Percentage of Participants Meeting the Definition of Dyslipidemia, Hypertriglyceridemia or Hypercholesterolemia at Any Given Time: 12-month Treatment Phase

Percentage of participants at any given time (at Month 6 and Month 12) who met the definition of dyslipidemia.Dyslipidemia is defined as hypertriglyceridemia (TGs ≥ 500 mg/dL [5.65 mmol/L]), hypercholesterolemia (LDL ≥ 100 mg/dL [2.59 mmol/L]), or elevated non-HDL (non-HDL ≥ 130 mg/dL [3.36 mmol/L]) in the presence of high TGs (TGs ≥ 200 mg/dL [2.26 mmol/L]). (NCT00555321)
Timeframe: 6 and 12 months posttransplant

,,,,
Interventionpercentage of participants (Number)
6 months: Dyslipidemia6 months: Hypertriglyceridemia6 months: Hypercholesterolemia12 months: Dyslipidemia12 months: Hypertriglyceridemia12 months: Hypercholesterolemia
Group 1: Basiliximab+Belatacept (MI) + MMF36.06.030.040.04.030.0
Group 2: Belatacept (MI) + MMF31.3029.233.3029.2
Group 3: Belatacept Less Intensive (LI) + MMF30.6024.544.92.040.8
Group 4: Tacrolimus + MMF32.1030.234.01.930.2
Group 5: Tacrolimus26.0022.042.02.036.0

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Percentage of Participants Surviving With Functional Graft: 12-month Treatment Phase

For 95% CI within each group, normal approximation was used if N>=5. Otherwise exact method was used. (NCT00555321)
Timeframe: At 6 and 12 months

,,,,
Interventionpercentage of participants (Number)
At 6 monthsAt 12 months
Group 1: Basiliximab+Belatacept (MI) + MMF90.090.0
Group 2: Belatacept (MI) + MMF89.683.3
Group 3: Belatacept (LI) + MMF77.667.3
Group 4: Tacrolimus + MMF92.592.5
Group 5: Tacrolimus90.088.0

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Percentage of Participants Who Develop Dyslipidemia, Hypertriglyceridemia and Hypercholesterolemia After Randomization and Transplantation: 12-month Treatment Phase

Percentage of participants who develop dyslipidemia, defined as hypertriglyceridemia (triglycerides [TGs] ≥ 500 mg/dL [5.65 mmol/L]), hypercholesterolemia (Low density lipoprotein [LDL] ≥ 100 mg/dL [2.59 mmol/L]), or elevated non-high density lipoprotein (non- high density lipoprotein [HDL] ≥ 130 mg/dL [3.36 mmol/L]) in the presence of high TGs (TGs ≥ 200 mg/dL [2.26 mmol/L]). (NCT00555321)
Timeframe: 6 and 12 months posttransplant

,,,,
Interventionpercentage of participants (Number)
By 6 month: DyslipidemiaBy 6 month: HypertriglyceridemiaBy 6 month: HypercholesterolemiaBy 12 month: DyslipidemiaBy 12 month: HypertriglyceridemiaBy 12 month: Hypercholesterolemia
Group 1: Basiliximab+Belatacept (MI) + MMF50.02.442.959.54.854.8
Group 2: Belatacept (MI) + MMF54.3048.657.1054.3
Group 3: Belatacept Less Intensive (LI) + MMF45.7039.158.72.252.2
Group 4: Tacrolimus + MMF33.3028.650.02.442.9
Group 5: Tacrolimus59.5051.470.3062.2

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Percentage of Participants Who Developed Hypertension in 12-month Treatment Phase

Percentage of participants who develop hypertension after randomization and transplantation. Transient post-operative increases in BP were not to be counted as new onset hypertension. Hypertension was to be assessed only at or after the Week 4 visit. Participants were considered to have hypertension when either of the following criteria were met: (1) SBP ≥ 130 mm Hg or DBP ≥ 80 mm Hg or (2) participant received an antihypertensive medication(s) for the indication of hypertension or due to medical history of hypertension. (NCT00555321)
Timeframe: 6 and 12 months posttransplant

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Interventionpercentage of participants (Number)
By 6 monthsBy 12 months
Group 1: Basiliximab+Belatacept (MI) + MMF94.1100
Group 2: Belatacept (MI) + MMF93.393.3
Group 3: Belatacept Less Intensive (LI) + MMF93.893.8
Group 4: Tacrolimus + MMF100.0100.0
Group 5: Tacrolimus100100.0

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Percentage of Participants Who Have Hypertension at Any Given Time During the 12-month Treatment Phase

Percentage of participants at any given time who meet the definition of hypertension. Participants were considered to have hypertension when either of the following criteria were met: (1) SBP ≥ 130 mm Hg or DBP ≥ 80 mm Hg or (2) participant received an antihypertensive medication(s) for the indication of hypertension or due to medical history of hypertension. (NCT00555321)
Timeframe: 6 and 12 months posttransplant

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Interventionpercentage of participants (Number)
By 6 monthsBy 12 months
Group 1: Basiliximab+Belatacept (MI) + MMF64.072.0
Group 2: Belatacept (MI) + MMF77.168.8
Group 3: Belatacept Less Intensive (LI) + MMF69.459.2
Group 4: Tacrolimus + MMF71.779.2
Group 5: Tacrolimus70.070.0

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Percentage of Participants With New Onset Diabetes Mellitus (NODM): 12-month Treatment Phase

A participant who did not have diabetes prior to randomization was determined to have NODM if(i) the participant received an antidiabetic medication for a duration of at least 30 days or(ii) at least two fasting plasma glucose (FPG) tests indicate that FPG is>=126 mg/dL (7.0 mmol/L). For 95% CI within each group, normal approximation is used if N>=5. For 95% CI of difference, adjustment is made for randomization strata (HCV-Infection status at baseline) if N >= 5 in each treatment arm. (NCT00555321)
Timeframe: 6 and 12 months posttransplant

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Interventionpercentage of participants (Number)
6 months12 months
Group 1: Basiliximab+Belatacept (MI) + MMF35.535.5
Group 2: Belatacept (MI) + MMF15.615.6
Group 3: Belatacept (LI) + MMF13.913.9
Group 4: Tacrolimus + MMF21.123.7
Group 5: Tacrolimus35.137.8

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Summary Statistics for Diastolic Blood Pressure: 12-month Treatment Phase

Participants were considered to have hypertension if they had Diastolic Blood Pressure (SBP) ≥ 80 mmHg. (NCT00555321)
Timeframe: BL (pretransplant), 1, 3, 6, 9, 12 months posttransplant

,,,,
Interventionmm Hg (Mean)
BL (n=50, 48, 49, 53, 49)1 month (n=50, 48, 49, 52, 48)Change from BL to 1 month (n=50, 48, 49, 52, 47)3 months (n=44, 46, 41, 48, 42)Change from BL to 3 months (n=44, 46, 41, 48, 41)6 months (n=43, 40, 36, 47, 37)Change from BL to 6 months (n=43, 40, 36, 47, 36)9 months (n=34, 30, 29, 41, 31)Change from BL to 9 months (n=34, 30, 29, 41, 31)12 months (n=42, 37, 36, 46, 38)Change from BL to 12 months (n=42, 37, 36, 46, 37)
Group 1: Basiliximab+Belatacept (MI) + MMF64.574.410.077.411.574.48.875.410.076.510.26
Group 2: Belatacept (MI) + MMF63.174.711.778.315.279.417.078.515.278.516.3
Group 3: Belatacept Less Intensive (LI) + MMF62.972.69.776.112.076.312.577.613.274.510.6
Group 4: Tacrolimus + MMF67.577.810.781.714.477.810.779.511.280.310.21
Group 5: Tacrolimus68.674.65.878.29.976.68.679.511.279.510.8

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Summary Statistics for Lipid Parameters - Serum Triglyceride: 12-month Treatment Phase

(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant

,,,,
Interventionmg/dL (Mean)
BL (n=29, 30, 25, 34, 32)1 month (n=29, 33, 29, 41, 43)Change from BL to 1 month (n=16, 20, 17, 27, 30)6 months (n=26, 29, 25, 41, 30)Change from BL to 6 months (n=15, 19, 18, 28, 22)12 months (n=38, 29, 32, 42, 34)Change from BL to 12 months (n=22, 19, 20, 25, 21)
Group 1: Basiliximab+Belatacept (MI) + MMF94.4153.971.2234.3199.9255.1237.2
Group 2: Belatacept (MI) + MMF110.1162.945.7157.118.3159.020.3
Group 3: Belatacept Less Intensive (LI) + MMF80.2149.085.8162.788.7158.291.6
Group 4: Tacrolimus + MMF84.4149.168.2148.160.8156.472.3
Group 5: Tacrolimus89.2163.571.8167.284.3190.592.6

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Summary Statistics for Lipid Parameters- Serum Cholesterol: 12-month Treatment Phase

(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant

,,,,
Interventionmg/dL (Mean)
BL (n=45, 42, 44, 49, 47)1 month (n=46, 47, 42, 49, 49)Change from BL to 1 month (n=42, 41, 39, 45, 46)6 months (n=39, 34, 38, 48, 36)Change from BL to 6 months (n=35, 30, 35, 44, 33)12 months (n=40, 33, 35, 45, 37)Change from BL to 12 months (n=35, 29,32, 42, 34)
Group 1: Basiliximab+Belatacept (MI) + MMF112.9166.357.0180.868.6186.879.4
Group 2: Belatacept (MI) + MMF119.7184.967.7177.351.3178.957.5
Group 3: Belatacept Less Intensive (LI) + MMF121.3190.661.3182.256.6186.460.7
Group 4: Tacrolimus + MMF111.0162.954.0170.658.9163.858.2
Group 5: Tacrolimus106.4164.153.4177.960.8175.257.3

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Summary Statistics for Lipid Parameters- Serum Low Density Lipoprotein Cholesterol (LDL): 12-month Treatment Phase

(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant

,,,,
Interventionmg/dL (Mean)
BL (n=29, 30, 25, 34, 32)1 month (n=30, 34, 29, 41, 42)Change from BL to 1 month (n=17, 21, 17, 27, 30)6 months (n=27, 29, 27, 41, 30)Change from BL to 6 months (n=16, 19, 18, 28, 22)12 months (n=38, 29, 32, 42, 35)Change from BL to 12 months (n=22, 19, 20, 25, 22)
Group 1: Basiliximab+Belatacept (MI) + MMF52.395.242.799.241.293.037.8
Group 2: Belatacept (MI) + MMF58.693.234.3107.545.9107.245.4
Group 3: Belatacept Less Intensive (LI) + MMF58.9115.757.198.248.7103.237.2
Group 4: Tacrolimus + MMF63.689.826.196.728.989.023.7
Group 5: Tacrolimus59.998.441.592.826.193.813.7

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Summary Statistics for Lipid Parameters-Serum Total Non-High Density Lipoprotein (Non-HDL) Cholesterol: 12-month Treatment Phase

(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant

,,,,
Interventionmg/dL (Mean)
BL (n=45, 42, 44, 49, 46)1 months (n=46, 47, 42, 49, 49)Change from BL to 1 months (n=42, 41, 39, 45, 45)6 months (n=39, 34, 38, 48, 36)Change from BL to 6 months (n=35, 30, 35, 44, 32)12 months (n=40, 33, 35, 45, 37)Change from BL to 12 months (n=35, 29, 32, 42, 33)
Group 1: Basiliximab+Belatacept (MI) + MMF80.4125.147.2139.359.5143.266.7
Group 2: Belatacept (MI) + MMF83.5147.966.0134.444.0137.449.2
Group 3: Belatacept Less Intensive (LI) + MMF84.8148.054.1138.948.2142.253.4
Group 4: Tacrolimus + MMF77.7121.747.0125.148.1118.247.4
Group 5: Tacrolimus76.8130.047.5135.448.6131.944.1

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Summary Statistics for Lipid Parameters; Serum HDL Cholesterol: 12-month Treatment Phase

(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant

,,,,
Interventionmg/dL (Mean)
BL (n=46, 42, 44, 49, 46)1 month (n=46, 47, 42, 49, 49)Change from BL to 1month (n=43, 41, 39, 45, 45)6 months (n=39, 34, 38, 48, 36)Change from BL to 6 months (n=36, 30, 35, 44, 32)12 months (n=40, 33, 35, 45, 37)Change from BL to12 months (n=36, 29, 32, 42, 33)
Group 1: Basiliximab+Belatacept (MI) + MMF32.141.39.941.59.243.612.7
Group 2: Belatacept (MI) + MMF36.236.91.742.87.341.58.3
Group 3: Belatacept Less Intensive (LI) + MMF36.542.57.244.29.444.27.3
Group 4: Tacrolimus + MMF33.341.27.145.510.845.610.9
Group 5: Tacrolimus31.134.13.442.69.543.310.0

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Summary Statistics for Mean Arterial Pressure: 12-month Treatment Phase

(NCT00555321)
Timeframe: BL (pretransplant), 1, 3, 6, 9, 12 months posttransplant

,,,,
Interventionmm Hg (Mean)
BL (n=50, 48, 49, 53, 49)1 month (n=50, 48, 49, 52, 48)Change from BL to 1 month (n=50, 48, 49, 52, 47)3 months (n=44, 46, 41, 48, 41)Change from BL to 3 months (n=44, 46, 41, 48, 41)6 months (n=43, 40, 36, 47, 37)Change from BL to 6 months (n=43, 40, 36, 47, 36)9 months (n=34, 30, 29, 41, 31)Change from BL to 9 months (n=34, 30, 29, 41, 31)12 months (n=42, 37, 36, 46, 38)Change from BL to 12 months (n=42, 37, 36, 46, 37)
Group 1: Basiliximab+Belatacept (MI) + MMF81.490.79.393.911.391.08.792.110.193.010.1
Group 2: Belatacept (MI) + MMF80.591.911.494.514.095.114.494.313.194.614.3
Group 3: Belatacept Less Intensive (LI) + MMF79.188.79.692.212.292.412.693.013.290.110.6
Group 4: Tacrolimus + MMF85.495.010.0100.115.195.410.598.212.399.214.3
Group 5: Tacrolimus87.591.34.196.59.695.08.597.711.399.012.1

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Summary Statistics for Systolic Blood Pressure: 12-month Treatment Phase

(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 3, 6, 9, 12 months posttransplant

,,,,
Interventionmm Hg (Mean)
BL (n=50, 48, 49, 53, 49)1 month (n=50, 48, 49, 52, 48)Change from BL to 1 month (n=50, 48, 49, 52, 47)3 months (n=44, 46, 41, 48, 42)Change from BL to 3 months (n=44, 46, 41, 48, 41)6 months (n=43, 40, 36, 47, 37)Change from BL to 6 months (n=43, 40, 36, 47, 36)9 months (n=34, 30, 29, 41, 31)Change from BL to 9 months (n=34, 30, 29, 41, 31)12 months (n=42, 37, 36, 46, 38)Change from BL to 12 months (n=42, 37, 36, 46, 37)
Group 1: Basiliximab+Belatacept (MI) + MMF115.3123.38.0127.010.9124.08.4125.510.4125.89.2
Group 2: Belatacept (MI) + MMF115.4126.310.9126.911.7126.59.3125.88.8127.010.2
Group 3: Belatacept Less Intensive (LI) + MMF111.4120.99.4124.412.7124.612.8123.913.3121.210.6
Group 4: Tacrolimus + MMF121.2129.28.5136.916.3130.610.0135.614.4137.015.9
Group 5: Tacrolimus125.3124.60.6133.09.1132.08.3133.911.6138.014.6

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Number of Participants Experiencing Graft Failure

graft failure = absolute neutrophil count (ANC) <5 x 10^8/L and an acellular bone marrow aspirate/biopsy (NCT00630253)
Timeframe: From Day 1 to event, assessed up to100 days

InterventionParticipants (Count of Participants)
Marrow Isolex0
UCB Arm0
Marrow Clinimax0

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Number of Participants Experiencing Overall Survival

"The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.~Overall survival will be defined as time from enrollment to date of death or censored at the date of last documented contact for patients still alive." (NCT00630253)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Marrow Isolex15
UCB Arm8
Marrow Clinimax5

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Number of Participants With Acute Graft-Versus-Host Disease (GVHD)

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00630253)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Marrow Isolex0
UCB Arm0
Marrow Clinimax0

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Number of Participants With Chronic Graft-Versus-Host Disease (GVHD)

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. (NCT00630253)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Marrow Isolex2
UCB Arm0
Marrow Clinimax0

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EFS

event-free survival after umbilical cord blood transplant (NCT00622895)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
Treatment: Allogeneic UCB After Reduced Intensity Conditioning1

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Event-free Survival (EFS)

The events will be defined as any one of the following: death; respiratory failure; renal failure, as defined by chronic dialysis > or = 6 months or kidney transplantation; occurrence of cardiomyopathy, confirmed by clinical CHF (New York Class III or IV) or LVEF < 30% by echocardiogram, sustained for at least 3 months despite therapy; organ dysfunction specific events must be documented on at least two occasions > or = 3 months apart, or sustained for a 3-month period (documented from the first occurrence). (NCT00622895)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Treatment: Allogeneic HCT After Reduced Intensity Conditioning1

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Incidence of Graft Rejection

Engraftment is defined as achieving > 5% donor peripheral blood T cell chimerism by Day 56 after HCT. Primary graft failure is defined as a donor peripheral blood T cell chimerism peak of < 5% by Day 56 post-HCT. Methodological requirements for chimerism are as defined by institutional standard of practice. Secondary Graft Failure is defined as documented engraftment followed by loss of the graft with donor peripheral blood T cell chimerism < 5% as demonstrated by a chimerism assay (NCT00622895)
Timeframe: Up to day +56

InterventionParticipants (Count of Participants)
Treatment: Allogeneic HCT After Reduced Intensity Conditioning0

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Overall Survival

Event is defined as death due to any cause. (NCT00622895)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment: Allogeneic HCT After Reduced Intensity Conditioning1

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The Percent of Participants With Definite and Probable Viral, Fungal, and Bacterial Infections

The percent of participants with definite and probable viral, fungal, and bacterial infections after transplant (NCT00622895)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment: Allogeneic HCT After Reduced Intensity Conditioning2

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Incidence and Severity of Graft-versus-host Disease (GVHD)

The grading of acute and chronic GVHD will follow previously published guidelines and according to institutional standard of practice but will also include capture of symptoms and characterization of alternative causes. The highest level of organ abnormalities, the etiologies contributing to the abnormalities and biopsy results pertaining to GVHD will be identified. Since both GVHD and SSc involve the skin and the gastrointestinal tract, all diagnostic biopsies of these organs will be centrally reviewed by a study pathologist. (NCT00622895)
Timeframe: Up to 5 years post-transplant

Interventionunits on a scale (Number)
acute GVHD severity maximum gradechronic GVHD maximum grade
Treatment: Allogeneic HCT After Reduced Intensity Conditioning22

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Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36)

The Medical Outcome Short Form (36) Health Survey instrument (SF-36) is a general assessment of health quality of life with eight components: physical functioning, role limitations due to physical health, pain index, general health perceptions, vitality, social functioning, role limitations due to emotional problems and Mental Health Index. Each domain is positively scored, indicating that higher scores are associated with positive outcome. (NCT00622895)
Timeframe: Up to 5 years

Interventionunits on a scale (Number)
SF-36 pretransplant overall scorepretransplant limitations due to physical healthpretransplant limitations due to emotional health
Treatment: Allogeneic HCT After Reduced Intensity Conditioning49.35050

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Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ)

The questionnaire includes measure of quality of life and measure of the scale of skin tightness, activity level and function specifically designed for patients with systemic sclerosis (NCT00622895)
Timeframe: Up to 5 years

Interventionunits on a scale (Number)
pre-transplant SHAQ5 year post transplantpre-transplant Raynaud symptoms5 year post transplant Raynaud symptomspre-transplant Finger ulcer symptoms5 year post-transplant Finger ulcer symptomspre-transplant Overall health symptoms5 year post-transplant overall health symptoms
Treatment: Allogeneic HCT After Reduced Intensity Conditioning1.1250.12530.5302.50.2

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Skin Score

The skin score measure is a scale: the name of the scale is the modified Rodnan skin score (mRSS). Total score of mRSS is from 0 to 51. Higher values represents worse skin score. Highest value is 51, represents very hidebound tight thick skin. Lowest value is 0, represent normal skin, no tightness. (NCT00622895)
Timeframe: Up to 5 years post-transplant

Interventionunits on a scale (mRSS) (Number)
Pre-transplant (baseline) skin score5 year post-transplant skin score
Treatment: Allogeneic HCT After Reduced Intensity Conditioning174

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Stimulation of Growth After 12 Months (Delta Z-score)

(NCT00707759)
Timeframe: 12 months

Interventionunits on a scale (Mean)
A: Withdrawal Steroids1.2
B: Control Steroids0.6

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Count of Participants With Acute Cellular Rejection Grade Equal to or Greater Than IA, by the Banff 2007 Criteria

Acute cellular rejection is when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade ≥ IA by Banff 2007 criteria. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus0
Induction: Alemtuzumab, Maintenance: MMF + Belatacept2
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac4

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Count of Participants With Antibody Mediated Rejection

Antibody mediated rejection (AMR) is defined as diffusely positive staining for C4d, presence of circulating anti-donor antibodies and morphologic evidence of acute tissue injury. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus0
Induction: Alemtuzumab, Maintenance: MMF + Belatacept1
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac0

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Count of Participants With Biopsy Proven Acute Rejection at Any Time Post-Transplant

Biopsy proven acute rejection was defined as histologic evidence of borderline or higher cellular rejection per local pathologist. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus3
Induction: Alemtuzumab, Maintenance: MMF + Belatacept2
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac5

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Count of Participants With CAN/IFTA Grade I, II or III at Any Time Post-transplant

CAN/IFTA grades were determined per local pathology interpretations of biopsy tissue. These grades reflect the severity of interstitial fibrosis and tubular atrophy present in the tissue obtained during a kidney biopsy. Higher grades indicate greater severity in interstitial fibrosis and tubular atrophy present the kidney biopsy tissue. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus1
Induction: Alemtuzumab, Maintenance: MMF + Belatacept3
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac5

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Count of Participants With de Novo Anti-donor HLA Antibodies at Wk 52

The presence of antibodies reactive to Histocompatibility Antigen (HLA) molecules expressed on the renal allograft have been associated with both acute and chronic injury to the transplanted kidney. The development of de novo anti- donor HLA antibodies may mean a person is more likely to reject the graft. (NCT01436305)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus0
Induction: Alemtuzumab, Maintenance: MMF + Belatacept0
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac0

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Count of Participants With Delayed Graft Function Post-Transplant

Delayed graft function is defined as dialysis in the first week on one or more occasions for any indication other than the treatment of acute hyperkalemia in the setting of otherwise acceptable renal function (NCT01436305)
Timeframe: Any time within the first week post-transplant

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus0
Induction: Alemtuzumab, Maintenance: MMF + Belatacept2
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac0

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Count of Participants With EBV Infection as Reported on the Case Report Form as Adverse Events

"Viral infections following renal transplantation is a significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during this study using participant blood samples.~Acronym: Epstein-Barr virus (EBV)" (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus0
Induction: Alemtuzumab, Maintenance: MMF + Belatacept0
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac0

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Count of Participants With Infections Requiring Hospitalization or Systemic Therapy Reported as Serious Adverse Events

Infections of certain types (i.e., excluding those identified in the protocol as occurring commonly in this study population) were required to be reported as a serious adverse event if they required either inpatient hospitalization of prolongation of a current hospitalization. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus2
Induction: Alemtuzumab, Maintenance: MMF + Belatacept2
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac1

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Count of Participants With Rejection

The number of participants who were treated by their local physician for any type of rejection including, but not limited to cellular rejection and antibody- mediated rejection of the transplanted kidney regardless of the presence of a biopsy. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus1
Induction: Alemtuzumab, Maintenance: MMF + Belatacept3
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac5

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Count of Participants With Treated Diabetes Between Day 14 and Wk 52

Treated diabetes is defined as the receipt of oral medication or insulin for >14 days between 14 days and 52 weeks post-transplant (NCT01436305)
Timeframe: Day 14 to Week 52

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus1
Induction: Alemtuzumab, Maintenance: MMF + Belatacept0
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac1

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Count of Participants With Use of Anti-hypertensive Medications at Wk 52

Anti-hypertensive medications are a class of drugs that are used to treat hypertension. The medications seek to prevent the complications of high blood pressure, such as stoke and myocardial infarction. (NCT01436305)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus3
Induction: Alemtuzumab, Maintenance: MMF + Belatacept3
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac7

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Mean Glomerular Filtration Rate (GFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52

GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI). A score of ≥ 90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. (NCT01436305)
Timeframe: Week 52

InterventionmL/min/1.73m^2 (Mean)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus55.9
Induction: Alemtuzumab, Maintenance: MMF + Belatacept51.6
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac58.3

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Number of Events of Death or Graft Loss

This measure counts deaths and graft loss occurring at any point post transplantation. Graft loss is defined as need for dialysis for greater than 30 days duration, allograft nephrectomy, or retransplantation. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

InterventionEvents (Number)
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus2
Induction: Alemtuzumab, Maintenance: MMF + Belatacept3
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac0

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Count of Participants by Chronic Kidney Disease (CKD) Stage Post-Transplant

"The stages of Chronic Kidney Disease are defined using the participant's GFR value as indicated below:~Stage 1 if GFR value is ≥90; Stage 2 if GFR value is ≥60 and < 90; Stage 3A if 45 ≤GFR < 60; Stage 3B if 30 ≤ GFR < 45; Stage 4 if 15 ≤GFR < 30;l Stage 5 if GFR < 15.~Stage 1 means kidney function is normal. Stage 2 indicates mildly reduced kidney function, pointing to kidney disease. Stages 3A and 3B indicate moderately reduced kidney function. Stage 4 indicates severely reduced kidney function. Stage 5 indicates very severe or end stage kidney failure." (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156

,,
InterventionParticipants (Count of Participants)
Week 52 - Stage 1Week 52 - Stage 2Week 52 - Stage 3AWeek 52 - Stage 3BWeek 52 - Stage 4Week 104 - Stage 1Week 104 - Stage 2Week 104 - Stage 3AWeek 104 - Stage 3BWeek 104 - Stage 4Week 156 - Stage 1Week 156 - Stage 2Week 156 - Stage 3AWeek 156 - Stage 3BWeek 156 - Stage 4
Induction: Alemtuzumab, Maintenance: MMF + Belatacept011011010001100
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus013000111001110
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac033101321005110

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Count of Participants by Severity of First Acute Cellular Rejection by Wk 52

"Acute cellular rejection is when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade ≥ IA by Banff 2007 criteria. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection. Originally, this endpoint was worded as The severity of first and highest acute cellular rejection within the first 52 weeks. But since the highest grade for each subject coincided with the first ACR episode for each subject, only a summary of severity of the first episode is presented here." (NCT01436305)
Timeframe: Transplantation through Week 52

,,
InterventionParticipants (Count of Participants)
Grade IAGrade IBGrade IIAGrade IIBGrade III
Induction: Alemtuzumab, Maintenance: MMF + Belatacept10010
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus00000
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac10210

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Count of Participants With BKV and CMV Viremia (Local Center Monitoring) Reported as Adverse Events

"Viral infections following renal transplantation is significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during this study using participant blood samples.~Acronyms: BK Polyoma Virus (BKV); Cytomegalovirus (CMV)." (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

,,
InterventionParticipants (Count of Participants)
BKVCMV
Induction: Alemtuzumab, Maintenance: MMF + Belatacept00
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus01
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac20

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Count of Participants With CKD Stage 4 or 5

"The stages of Chronic Kidney Disease are defined using the participant's GFR value as indicated below.~Stage 1 if GFR value is ≥90; Stage 2 if 60 ≤ GFR < 90; Stage 3A if 45 ≤ GFR < 60; Stage 3B if 30 ≤ GFR < 45; Stage 4 if 15 ≤ GFR < 30; Stage 5 if GFR < 15.~Stage 1 means kidney function is normal. Stage 2 indicates mildly reduced kidney function, pointing to kidney disease. Stages 3A abd 3B indicate moderately reduced kidney function. Stage 4 indicates severely reduced kidney function. Stage 5 indicates very severe or end stage kidney failure." (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156

,,
InterventionParticipants (Count of Participants)
Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept100
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus000
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac000

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Count of Participants With Either New Onset Diabetes After Transplant (NODAT) or Impaired Fasting Glucose (IFG) at Wk 52 Based on Criteria Specified by the ADA and WHO

"New onset diabetes is the development of diabetes post-kidney transplant. It was identified by the clinical sites caring for each participant and reported directly in the clinical database. Impaired fasting glucose (IFG) is a determination made by referencing glucose measurements obtained from a standard chemistry panel. Any fasting glucose measure that is between 110 and 125 mg/dL is classified as IFG.~Acronyms: American Diabetes Association (ADA); World Health Organization (WHO)." (NCT01436305)
Timeframe: Week 52

,,
InterventionParticipants (Count of Participants)
New onset diabetes during first 52 weeksImpaired fasting glucose at week 52
Induction: Alemtuzumab, Maintenance: MMF + Belatacept00
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus01
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac00

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Count of Participants With Estimated Glomerular Filtration Rate (GFR) < 60 mL/Min/1.73 m^2 by CKD EPI

GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI). A score of ≥90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. This measure specifically looked at participants with scores less than 60. (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156

,,
InterventionParticipants (Count of Participants)
Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept211
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus322
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac432

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Count of Participants With Fever > 39 Degrees Celsius and Blood Pressure < 90mm Hg Within 24 Hours of Onset of Transplant Procedure

Temperature of >39 degrees Celsius would be an indication of fever most often in response to an infection or illness. Systolic blood pressure <90mm Hg would be an indication of low blood pressure. (NCT01436305)
Timeframe: 24 hours after transplantation

,,
InterventionParticipants (Count of Participants)
Fever >39 degreesSystolic BP <90
Induction: Alemtuzumab, Maintenance: MMF + Belatacept01
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus00
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac00

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Count of Participants With Use of Lipid Lowering Medications at Baseline and Wks 24, 52, 104 and 156

Lipid lowering medications are used in the treatment of high levels of fats (lipids), such as cholesterol in blood (NCT01436305)
Timeframe: Baseline, Week 24, Week 52, Week 104, Week 156

,,
InterventionParticipants (Count of Participants)
BaselineWeek 24Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept11111
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus54333
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac22233

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Fasting Lipid Profile (Total Cholesterol, Non-HDL Cholesterol, LDL, HDL, and Triglyceride) at Baseline and Wks 24, 52, 104 and 156

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are detailed below.~Total cholesterol: 75-169 mg/dL if age ≤ 20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease" (NCT01436305)
Timeframe: Baseline, Week 24, Week 52, Week 104, Week 156

,,
Interventionmg/dL (Mean)
Tot. Chol. BaselineTot. Chol. W24Tot. Chol. W52Tot. Chol. W104Tot. Chol. W156Non-HDL BaselineNon-HDL W24Non-HDL W52Non-HDL W104Non-HDL W156LDL BaselineLDL W24LDL W52LDL W104LDL W156HDL BaselineHDL W24HDL W52HDL W104HDL W156Triglyc. BaselineTriglyc. W24Triglyc. W52Triglyc. W104Triglyc. W156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept160.2159.0187.0142.5133.5118.8129.3151.0110.5102.586.686.6114.058.055.541.329.736.032.031.0307.8249.7187.0220.0228.0
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus141.2171.6156.0170.5183.5108.2128.0117.5129.5138.576.776.769.5100.5116.033.043.638.541.045.0158.7161.0319.3146.0115.0
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac165.6185.6157.5189.0181.7122.3129.061.0135.6136.083.483.449.0101.4106.043.356.659.053.445.7206.9115.958.0172.8156.5

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HbA1c Measured at Days 28 & 84, and Weeks 24, 36, 52, 72, 104 and 156

Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control. A value below 6.0% reflects normal levels, 6.0% to 6.4% reflects prediabetes, and a value of ≥ 6.5% reflects diabetes. (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156

,
Interventionpercent (Mean)
Day 28Day 84Week 24Week 36Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept5.15.05.15.34.85.25.2
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus5.35.76.96.77.05.75.6

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HbA1c Measured at Days 28 & 84, and Weeks 24, 36, 52, 72, 104 and 156

Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control. A value below 6.0% reflects normal levels, 6.0% to 6.4% reflects prediabetes, and a value of ≥ 6.5% reflects diabetes. (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156

Interventionpercent (Mean)
Day 28Day 84Week 24Week 36Week 52Week 72Week 104Week 156
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac5.85.96.57.27.68.16.67.8

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Mean Calculated eGFR Using MDRD 4 Variable Model

The estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease equation (MDRD). A score of ≥90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156

,,
InterventionmL/min/1.73m^2 (Mean)
Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept47.869.365.5
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus52.454.249.0
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac55.760.461.5

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Number of All Adverse Events (AEs) and Serious Adverse Events (SAEs)

Adverse events were collected systematically from enrollment through last study visit. Displayed below are counts of all adverse events per treatment group (including both serious and non-serious adverse events). Separately counts of all adverse events determined to be serious are displayed per treatment group. More detail about adverse events for this trial is displayed in the 'Adverse Event' section. (NCT01436305)
Timeframe: Enrollment through last study visit (up to week 156)

,,
InterventionEvents (Number)
All Adverse EventsSerious Adverse Events
Induction: Alemtuzumab, Maintenance: MMF + Belatacept2511
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus216
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac407

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Standardized Blood Pressure Measurement at Wk 52

A blood pressure measurement consists of two numbers: the systolic and diastolic pressures. Systolic pressure measures the pressure in blood vessels when the heart beats. Diastolic pressure measures the pressure in blood vessels between beats of the heart. Systolic measures of <120 and diastolic measures of <80 are considered normal. Systolic measures of 120-139 and diastolic measures of 80-89 are considered at risk (or pre-hypertension). Systolic measures of ≥140 and diastolic measures of ≥90 are considered high. (NCT01436305)
Timeframe: Week 52

,,
InterventionmmHg (Mean)
Systolic Blood Pressure at Week 52Diastolic Blood Pressure at Week 52
Induction: Alemtuzumab, Maintenance: MMF + Belatacept146.792.7
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus147.580.8
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac139.979.3

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The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine

The estimated Glomerular Filtration Rate (eGFR) was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI). A score of ≥90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. An estimate of the slope, or change over time, in eGFR was produced using standard statistical linear modeling procedures. The estimate was then re-scaled so that it can be interpreted as a change in eGFR per month. Positive numbers indicate increasing kidney function. Larger numbers indicate greater change in kidney function. (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156

,,
InterventionChange in eGFR (mL/min/1.73m^2) by month (Mean)
Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept0.620.620.69
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus1.291.271.33
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac1.070.680.48

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Total Daily Prescribed Pill Number at Days 28 and 84, and Wks 24, 36, 52, 72, 104 and 156

This is a measure of the total number of pills a participant was prescribed on a given day (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156

,
InterventionNumber of pills (Mean)
Day 28Day 84Week 24Week 36Week 52Week 104Week 156
Induction: Alemtuzumab, Maintenance: MMF + Belatacept15.813.58.314.014.315.515.0
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus28.822.214.813.014.615.314.0

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Total Daily Prescribed Pill Number at Days 28 and 84, and Wks 24, 36, 52, 72, 104 and 156

This is a measure of the total number of pills a participant was prescribed on a given day (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156

InterventionNumber of pills (Mean)
Day 28Day 84Week 24Week 36Week 52Week 72Week 104Week 156
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac27.321.316.617.017.816.014.812.7

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Type of Treatment of Rejection

"Upon having a biopsy performed, persons often receive treatment for rejection based on the results of the biopsy, which may or may not have shown signs of rejection. Details of biopsy findings and corresponding treatment are presented here for each instance of treatment for rejection. Acronyms and abbreviations are defined below.~ACR=Acute Cellular Rejection ATG=Anti-thymocyte globulin therapy Chr. AMR=Chronic Antibody Mediated Rejection Gd.=Grade IFTA=Interstitial Fibrosis and Tubular Atrophy IVIG=Intravenous Immunoglobulin therapy.~Only 'for cause' biopsies were performed post-transplant; thus, it is possible for a participant to be included in the analysis population and not have a biopsy for this outcome measure." (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)

,,
InterventionBiopsy (Number)
Borderline rejection; IVIG and plasmapheresisACR Gd. IA + Chr. AMR + IFTA Gd. I; Pulse SteroidsACR Gd. IA + IFTA Gd. II; Pulse SteroidsACR Gd. IIB; ATG and Pulse SteroidsBorderline + IFTA Gd. I; with Pulse SteroidsACR Gd. IA + IFTA Gd. I; Pulse SteroidsACR Gd. IIA; Pulse SteroidsACR Gd. IIA + IFTA Gd. I; ATG and Pulse SteroidsACR Gd. IIB + IFTA Gd. I; ATG and Pulse Steroids
Induction: Alemtuzumab, Maintenance: MMF + Belatacept011100000
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus100000000
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac000011121

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Change of Lung Function Measured by Forced Vital Capacity (FVC) Between Baseline and 24 Weeks

Forced vital capacity, or FVC, is the amount in liters of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It measures the effect that lung disease has on a person's ability to inhale and exhale. Higher values are better, and decreases over time can indicate disease progression. (NCT02370693)
Timeframe: 24 weeks

InterventionPercentage of change in FVC %predicted (Mean)
Bortezomib Plus Mycophenolate Mofetil0.51
Placebo Plus Mycophenolate Mofetil-0.69

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Change of Skin Fibrosis Measured by the Rodnan Skin Score Between Baseline and 24 Weeks

The Modified Rodnan Skin Score (mRSS) is a measure of skin thickness that sums individual scores of skin thickness (0 - No Thickening, 1 - Mild Thickening, 2 - Moderate Thickening, 3 - Severe Thickening) measured at 17 body sites to reach a total score (Range: 0 to 51, higher value representing thicker skin). The Score is used as a surrogate for disease activity and severity, and a worsening score over time is associated with worse outcomes. (NCT02370693)
Timeframe: 24 weeks

InterventionScore on a Scale (Mean)
Bortezomib Plus Mycophenolate Mofetil-3.00
Placebo Plus Mycophenolate Mofetil-0.33

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Number of Participants With Serious Adverse Events

To assess the safety and tolerability of bortezomib with mycophenolate mofetil assessed by the incidence of serious adverse events. (NCT02370693)
Timeframe: First dosing day to last study visit day: Mean duration 8 months.

InterventionNumber of Participants with Serious Adve (Mean)
Bortezomib Plus Mycophenolate Mofetil0.33
Placebo Plus Mycophenolate Mofetil0.25

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Number of Patients That Engrafted Blood Counts by 30 Days After Transplant

Number of patients whose Absolute Neutrophil Count (ANC) recovered to >500 x10^3/uL for at least 3 consecutive days after transplant (NCT00827099)
Timeframe: Day 30

Interventionparticipants (Number)
Umbilical Cord Blood Transplant5

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Acute Graft Versus Host Disease Among Patients Who Received Early Transplant

Acute graft versus host disease (graded II, III, or IV) among patients who received early allogeneic HCT on study. (NCT02756572)
Timeframe: At day 100

InterventionParticipants (Count of Participants)
Received Allogeneic HCT on Study0

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Demonstrate the Feasibility of Collecting Resource Utilization Data for Trial Participants

The amount of days of hospitalization (the major driver of costs within the first year) will be collected for resource utilization. (NCT02756572)
Timeframe: Within the first year of induction chemotherapy on study

Interventiondays (Median)
Treatment (Chemotherapy, HCT)49

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Event-free Survival (EFS) Among Patients Who Received Early Transplant

Event-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. Events included death, relapse, and grade 3-4 acute graft vs host disease. (NCT02756572)
Timeframe: Up to 100 days post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study91

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Event-free Survival (EFS) Among Patients Who Received Early Transplant

Event-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. Events included death, relapse, and grade 3-4 acute graft vs host disease. (NCT02756572)
Timeframe: Up to 6 months post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study82

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Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - AGE

Patients who receive early transplant will be compared to those that don't using the Wilcoxon rank sum test for the quantitative variable of age. (NCT02756572)
Timeframe: From time of subject's study enrollment to time of subject's feasibility success assessment (i.e. when subject received transplant within 60 days or when it was determined subject would not proceed with transplant on study)

Interventionyears (Median)
Received Allogeneic HCT on Study55
Did Not Receive Allogeneic HCT on Study57

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Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant

Overall survival among patients who did not receive early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 100 days after induction day 1

InterventionPercentage of participants (Number)
Did Not Receive Allogeneic HCT on Study75

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Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant

Overall survival among patients who did not receive early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 6 months after induction day 1

InterventionPercentage of participants (Number)
Did Not Receive Allogeneic HCT on Study62

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Overall Survival (OS) Among Patients Who Received Early Transplant.

Overall survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 100 days post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study91

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Overall Survival (OS) Among Patients Who Received Early Transplant.

Overall survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 6 months post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study82

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Relapse-free Survival (RFS) Among Patients Who Received Early Transplant

Relapse-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 100 days post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study91

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Relapse-free Survival (RFS) Among Patients Who Received Early Transplant

Relapse-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 6 months post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study82

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Demonstrate the Feasibility of Collecting Patient-reported Outcomes for Trial Participants

The amount of returned patient-reported outcome questionnaires will be summarized for each collection timepoint using percent collection from surviving patients for PRO timepoints. (NCT02756572)
Timeframe: Up to 12 months post-HCT

InterventionParticipants (Count of Participants)
Enrollment PROs returnedPost G-CLAM PROs returnedPre-HCT PROs returned6 months post-HCT PROs returned12 months post-HCT PROs returned
Treatment (Chemotherapy, HCT)2723843

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Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - GENDER

Patients who receive early transplant will be compared to those that don't using the Fisher's exact test for the categorical variables of gender. (NCT02756572)
Timeframe: From time of subject's study enrollment to time of subject's feasibility success assessment (i.e. when subject received transplant within 60 days or when it was determined subject would not proceed with transplant on study)

,
InterventionParticipants (Count of Participants)
FemaleMale
Did Not Receive Allogeneic HCT on Study1011
Received Allogeneic HCT on Study81

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Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Enrollment and Incidence of Early Transplant

Success will be defined as enrollment of at least 30 patients with transplants occurring in 15 of these 30 (50%) within 60 days of enrollment or start of induction chemotherapy with G-CLAM, whichever is earlier. (NCT02756572)
Timeframe: Up to 60 days after start of chemotherapy

InterventionParticipants (Count of Participants)
Received allogeneic HCT on study within 60 days (feasibility success)Did not receive allogeneic HCT on study within 60 days (feasibility failure)
Treatment (Chemotherapy, HCT)921

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Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Relapsed-free Survival 6 Months After Transplant

Success will be defined as observing a 40% of higher 6-month relapse-free survival among patients who received early transplant on study. (NCT02756572)
Timeframe: 6 months after early allogeneic HCT on study

InterventionParticipants (Count of Participants)
No relapse within 6 months post-HCT (feasibility success)Relapse within 6 months post-HCT (feasibility failure)
Received Early Allogeneic HCT on Study62

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Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 28

"Complete Remission (CR), defined as <5% blasts in bone marrow with hematologic recovery (ANC>1000/ul and platelets >100,000/ml).~CRi, defined as complete remission with insufficient hematologic recovery (ANC<1000/ul or platelets<100,000/ul).~CRp, defined as complete remission but platelets <100,000/ul. Minimal residual disease (MRD), defined as any detectable disease by flow cytometry, cytogenetics, FISH or PCR in a patient otherwise fulfilling remission criteria.~Morphologic leukemia-free state (MLFS), defined as <5% blasts in bone marrow with no hematologic recovery.~Relapse, defined as >5% blasts in bone marrow, flow cytometry, or manual differential; OR treatment for active relapsed disease." (NCT02756572)
Timeframe: Approximately 28 days after early allogeneic HCT

InterventionParticipants (Count of Participants)
CR with MRDCR without MRDCRi with MRDCRi without MRDMLFS with MRDMLFS without MRDRelapse
Received Allogeneic HCT on Study0701000

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Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 84

"Complete Remission (CR), defined as <5% blasts in bone marrow with hematologic recovery (ANC>1000/ul and platelets >100,000/ml).~CRi, defined as complete remission with insufficient hematologic recovery (ANC<1000/ul or platelets<100,000/ul).~CRp, defined as complete remission but platelets <100,000/ul. Minimal residual disease (MRD), defined as any detectable disease by flow cytometry, cytogenetics, FISH or PCR in a patient otherwise fulfilling remission criteria.~Morphologic leukemia-free state (MLFS), defined as <5% blasts in bone marrow with no hematologic recovery.~Relapse, defined as >5% blasts in bone marrow, flow cytometry, or manual differential; OR treatment for active relapsed disease." (NCT02756572)
Timeframe: Approximately 84 days after early allogeneic HCT

InterventionParticipants (Count of Participants)
CR with MRDCR without MRDCRi with MRDCRi without MRDMLFS with MRDMLFS without MRDRelapse
Received Allogeneic HCT on Study0402002

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# of Patients Developing Denovo Donor Specific Antibody (DSA) Post-transplant

Number of patients (%) with development of denovo DSA after transplant (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A5
Group B1
Group C5

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# Patients Experiencing a First Biopsy-proven ACR With Severity Banff > or = 2a Grade

Number of patients with their First BPACR with a Banff grade >= Banff 2a using the Banff 2007 classification system for biopsy grading. Banff grade 2a and above is considered severe cellular rejection and includes grades of Banff 2a, Banff 2b, and Banff 3. (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A5
Group B12
Group C0

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# Patients Experiencing a Graft Loss But Not Including Patients Who Died With Functioning Graft (Death-censored Graft Loss)

Number of patient who experienced Graft loss, not including (censored) patients who lost graft due to death (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A0
Group B1
Group C1

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# Patients With Composite Endpoint of Either Experiencing Death, Graft Loss, or eGFR < 45ml/Min at 24 Months

Composite Endpoint of number of patients who experienced either patient death, allograft loss, or had an eGFR < 45 ml/min/1.73m2 (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A11
Group B13
Group C21

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# Patients With Composite Endpoint of Experiencing Either Death, Graft Loss, or eGFR < 45ml/Min

Number of Patients that experienced patient Death or Graft Loss or had an estimated GFR (eGFR) (MDRD) < 45 mL/min (NCT01729494)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
Group A9
Group B15
Group C14

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Biopsy Proven Acute Antibody Mediated Rejection

Incidence of patients experiencing a Biopsy proven acute antibody mediated rejection (BPAMR) (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A2
Group B2
Group C3

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Biopsy Proven Acute Cellular Rejection

Biopsy proven acute cellular rejection (BPACR) (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A14
Group B22
Group C2

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Biopsy Proven Acute Rejection

Incidence of all biopsy proven acute rejection whether clinically relevant or clinically silent. (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A20
Group B26
Group C7

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Biopsy Proven Mixed Acute Rejection

Incidence of patients experiencing a Biopsy proven acute cellular rejection with either DSA positive or C4d staining positive indicating antibody rejection (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A4
Group B2
Group C2

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Delayed Graft Function

Number of patients experiencing Delayed graft function (DGF) within first week after transplant. DGF is defined as need for dialysis within the first week after transplant. (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A3
Group B1
Group C5

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Discontinuation of Mycophenolate

Number of patients who were discontinued from mycophenolate treatment at 2 years (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A11
Group B9
Group C13

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Discontinuation of Study Treatment (Belatacept or Tacrolimus)

Number of patients who had to Discontinue study treatment (belatacept or tacrolimus) at 2 years (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A11
Group B9
Group C5

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eGFR (MRDRD) < 45 ml/Min/1.73m2

Patients with reduced Renal function measured by estimated GFR MDRD < 45 ml/min at 24 months (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A9
Group B8
Group C20

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Leukopenia (WBC < 2000/mm3)

Number of patients developing leukopenia defined as WBC < 2000/mm3 (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A22
Group B14
Group C15

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Mean eGFR (MDRD) (ml/Min/1.73m2)

Mean eGFR (MDRD) (ml/min/1.73m2) measured for all patients reaching 2 year endpoint (NCT01729494)
Timeframe: 24 months

Interventionml/min/1.73m2 (Mean)
Group A65.5
Group B65.3
Group C63.4

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New Onset Diabetes After Transplantation (NODAT)

Number of patients developing New Onset Diabetes Mellitus after Transplant by one of 5 definitions; only patients without preexisting diabetes were included (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A11
Group B5
Group C12

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Patient Death

Number of Patients who experienced death, all causes (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A2
Group B4
Group C1

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Proteinuria UPC Ratio > 0.8

Number of Patients with a Urine protein/creatinine (UPC) ratio > 0.8 (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A11
Group B5
Group C21

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Requirement of T-cell Depleting Therapy for Biopsy Proven Acute Rejection (BPAR)

Number of patients requiring anti-lymphocyte therapy for the treatment of BPAR rejection (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A8
Group B15
Group C0

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Steroid Therapy

Number of patients on treatment with corticosteroids at 2 years (NCT01729494)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Group A16
Group B14
Group C9

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Time to First BPAR

Mean Time to first episode of BPAR (days) (NCT01729494)
Timeframe: 24 months

Interventiondays (Mean)
Group A229
Group B131.6
Group C159.6

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Duration of Prednisone Maintenance Dosing

The duration of prednisone maintenance dosing was defined as the number of days that subjects maintained a prednisone dose of not more than 10 mg/day in the absence of new persistent lesions. (NCT00683930)
Timeframe: 52 weeks

InterventionDays (Median)
Placebo136.5
MMF 2 g/Day and MMF 3 g/Day Groups Combined186.0
Mycophenolate Mofetil 2 g/Day185.0
Mycophenolate Mofetil 3 g/Day187.0

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Percentage of Patients Achieving Responder Status at Week 52

The proportion of subjects achieving responder status (defined as no new persistent lesions and prednisone dose of not more than 10 mg/day from Week 48 until study termination at Week 52) in the two active treatment groups combined (2 g/day and 3 g/day mycophenolate mofetil) compared with the placebo group (NCT00683930)
Timeframe: 52 weeks

InterventionPercentage of Participants (Number)
Placebo63.9
MMF 2 g/Day and MMF 3 g/Day Groups Combined69.0

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Time to Initial Response

Time to initial response defined as the time that the subject first demonstrated responder status (defined as no new persistent lesions and prednisone dose of not more than 10 mg/day from Week 48 until study termination at Week 52) (NCT00683930)
Timeframe: up to 52 weeks

InterventionWeeks (Median)
Placebo31.3
MMF 2 g/Day and MMF 3 g/Day Groups Combined24.1

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Time to Sustained Response

Time to sustained response is defined as the week the subject first demonstrates both of the conditions of responder status provided the conditions are maintained through to study termination at Week 52. If a subject does not have a sustained response, time to sustained response is censored on the last day of the study. (NCT00683930)
Timeframe: up to 52 weeks

InterventionWeeks (Median)
Placebo46.0
MMF 2 g/Day and MMF 3 g/Day Groups Combined32.1

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Number of Non-relapse Participant Mortalities

Defined as death in any patient for whom there has not been a diagnosis of relapse or disease progression. (NCT00789776)
Timeframe: Day 200

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)0
Dose 2 (5.0 x 10^6/kg NK Cells)0

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Number of Participants Who Experienced Chronic Extensive GVHD

Number of patients who developed chronic extensive GVHD post-transplant. The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD. (NCT00789776)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)2
Dose 2 (5.0 x 10^6/kg NK Cells)3

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Number of Participants Who Experienced Graft Failure

Graft failure is defined as grade IV thrombocytopenia and neutropenia after Day +21 that lasts >2 weeks and is refractory to growth factor support. (NCT00789776)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)0
Dose 2 (5.0 x 10^6/kg NK Cells)4

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Number of Participants With Dose Limiting Toxicities

Defined as having at least one of the following adverse events, independent of the attribution to the Natural Killer cell infusion: grade IV infusional toxicity (based on the Adapted Common Toxicity Criteria); grade IV regimen-related toxicity (based on Adapted Common Toxicity Criteria); grade IV acute Graft-Versus-Host Disease; non-relapse mortality. (NCT00789776)
Timeframe: Day 35 (28 days after NK cell infusion)

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)0
Dose 2 (5.0 x 10^6/kg NK Cells)0

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Number of Participants With Grades III-IV Acute GVHD

"Number of patients who developed acute GVHD post-transplant. aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00789776)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)1
Dose 2 (5.0 x 10^6/kg NK Cells)0

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Number of Participants With Relapsed Disease

"CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever greater than 38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~AML, ALL >5% marrow blasts by morphologic or flow cytometric, or appearance of extramedullary disease.~CLL ≥1 of: Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or number of plasmacytomas or lytic bone lesions." (NCT00789776)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)1
Dose 2 (5.0 x 10^6/kg NK Cells)10

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Number of Subjects Surviving Post-transplant.

Number of subjects surviving post-transplant. (NCT00789776)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Dose 1 (2.5 x 10^6/kg NK Cells)5
Dose 2 (5.0 x 10^6/kg NK Cells)25

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Immune Reconstitution Efficacy - Naive CD4 T Cells

The development of total naive CD4 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI

Interventioncells/mm3 (Median)
Cultured Thymus Tissue Implantation With Immunosuppression156

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Immune Reconstitution Efficacy - Naive CD8 T Cells

The development of total naive CD8 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI

Interventioncells/mm3 (Median)
Cultured Thymus Tissue Implantation With Immunosuppression37

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Immune Reconstitution Efficacy - Response to Mitogens

Measurement of the T cell proliferative response to the mitogen phytohemagglutin (PHA). (NCT00579527)
Timeframe: 1 year post-CTTI

Interventioncounts per minute (cpm) (Median)
Cultured Thymus Tissue Implantation With Immunosuppression139189

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Immune Reconstitution Efficacy - Total CD3 T Cells

The development of total CD3 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI

Interventioncells/mm3 (Median)
Cultured Thymus Tissue Implantation With Immunosuppression726

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Immune Reconstitution Efficacy - Total CD4 T Cells

The development of total CD4 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI

Interventioncells/mm3 (Median)
Cultured Thymus Tissue Implantation With Immunosuppression593

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Immune Reconstitution Efficacy - Total CD8 T Cells

The development of total CD8 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI

Interventioncells/mm3 (Median)
Cultured Thymus Tissue Implantation With Immunosuppression145

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Survival at 1 Year Post-CTTI

Survival at 1 year post cultured thymus tissue implantation was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time. (NCT00579527)
Timeframe: 1 year post-CTTI

Intervention% of participants who survive to 1 year (Number)
Cultured Thymus Tissue With Immunosuppression71

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Survival at 2 Years Post-CTTI

Survival at 2 years post cultured thymus tissue implantation was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time. (NCT00579527)
Timeframe: 2 years post-CTTI

Intervention% of participants who survive to 2 years (Number)
Cultured Thymus Tissue Implantation With Immunosuppression71

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Adjusted Mean Change From Baseline in Disease Activity as Measured by BILAG 2004 Over Time During Year 1 of the Double-blind Period

"Adjusted mean change from baseline in British Isles Lupus Assessment Group (BILAG) score over time during Year 1 of the double-blind period based on a repeated measure mixed model and presented at each visit in the first 12-month of the double-blind period. BILAG index measures disease activity in different organs/systems separately. BILAG score is calculated for each of 9 systems depending on the clinical features present and whether they are new (4 points), worse (3 points), the same (2 points), improving (1 point) or not present (0 points) in the last 4 weeks compared with previously. BILAG A represents the presence of serious features of lupus. BILAG B represents more moderate features of the disease. BILAG C includes only mild symptomatic features. BILAG D represents prior activity with no current symptoms due to active lupus. BILAG E represents an organ that has never been involved. Overall BILAG score ranges from 0-108, with higher scores reflecting a worse outcome." (NCT01714817)
Timeframe: Day 1 to Day 365

InterventionScores on a Scale (Mean)
Abatacept IV-8.22
Placebo IV-7.60

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Adjusted Mean Change From Baseline in UPCR at Day 365 of the Double-blind Period in Overall Population

Adjusted Mean Change from Baseline in Urine protein/creatinine ratio (UPCR) at Day 365 of the double-blind period in the overall population (NCT01714817)
Timeframe: Day 1 and Day 365

InterventionUPCR (mg/mg) (Mean)
Abatacept IV-2.99
Placebo IV-2.90

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Adjusted Mean Change From Baseline in Urine Protein/Creatinine Ratio (UPCR) at Day 365 of the Double-blind Period in Nephrotic Participants

Adjusted Mean Change from Baseline in UPCR at Day 365 of the double-blind period in nephrotic participants (NCT01714817)
Timeframe: Baseline and Day 365

InterventionUPCR (mg/mg) (Mean)
Abatacept IV-5.01
Placebo IV-4.84

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AUC (TAU): Area Under the Serum Concentration Time Curve Over a Dosing Interval

AUC (TAU): Area under the serum concentration time curve over a dosing interval between Days 337 to 365. (NCT01714817)
Timeframe: Days 337 to 365

Interventionug*h/mL (Geometric Mean)
Abatacept IV36480.24

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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (Percentage of Blood)

"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729

InterventionPercentage (Mean)
Abatacept IV0.0328
Placebo IV0.0325

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Percentage of Nephrotic Participants in Complete Renal Response of Lupus Glomerulonephritis at Day 365 of the Double-blind Period

Number of participants achieving CR was divided by total participants in that arm, expressed as a percentage. CR is defined the following criteria: eGFR is normal or no <85% of the baseline value; eGFR is based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equivalent for at least 28 days prior. Subjects with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use, race and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 365

InterventionPercentage (Number)
Abatacept IV27
Placebo IV29.5

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Percentage of Nephrotic Participants in Complete Renal Response of Lupus Glomerulonephritis at Day 729 of the Double-blind Period

Number of participants achieving CR was divided by total participants in that arm, expressed as a percentage. CR is defined the following criteria: eGFR is normal or no <85% of the baseline value; eGFR is based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equivalent for at least 28 days prior. Subjects with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use, race and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 729

InterventionPercentage (Number)
Abatacept IV50.0
Placebo IV49.0

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Percentage of Participants in Complete Renal Response (CR) of Lupus Glomerulonephritis at Day 365 of the Double-blind Period

Number of participants achieving CR was divided by the total number of participants in that arm and expressed as a percentage. CR defined as: eGFR is normal or no <85% of the baseline; eGFR based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equiv. for at least 28 days prior to assessment. Participants with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as having achieved CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use (Yes/No), race (Asian/ Black/Caucasian/Other) and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 365

InterventionPercentage (Number)
Abatacept IV35.1
Placebo IV33.5

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Percentage of Participants in Overall Population in Complete Renal Response of Lupus Glomerulonephritis at Day 729 of the Double-blind Period

Number of participants achieving CR was divided by total participants in that arm, expressed as a percentage. CR is defined the following criteria: eGFR is normal or no <85% of the baseline value; eGFR is based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equivalent for at least 28 days prior. Subjects with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use, race and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 729

InterventionPercentage (Number)
Abatacept IV61.9
Placebo IV52.7

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Adjusted Mean Change From Baseline in Disease Activity as Measured by BILAG 2004 Over Time During the Double-blind Period

"BILAG index measures and reports disease activity in different organs/systems separately. The BILAG score is calculated for each of nine systems depending on the clinical features present and whether they are new (4 points), worse (3 points), the same (2 points), improving (1 point) or not present (0 points) in the last 4 weeks compared with previously. A BILAG A represents the presence of one or more serious features of lupus. A BILAG B represents more moderate features of the disease. A BILAG C includes only mild symptomatic features. A BILAG D represents only prior activity with no current symptoms due to active lupus. A BILAG E represents an organ that has never been involved. Overall BILAG score ranges from 0-108, with higher scores reflecting a worse outcome." (NCT01714817)
Timeframe: Day 1 to Day 729; Day 365 to Day 729

,
InterventionScores on a Scale (Mean)
Day 1 to Day 729Day 365 to Day 729
Abatacept IV-9.31-0.95
Placebo IV-8.53-0.40

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Adjusted Mean Change From Baseline in eGFR Over Time

Estimated glomerular filtration rate(eGFR), will be calculated by the CKD-EPI formula shown below.50 eGFR is expressed as mL/min per 1.73m2. For the purpose of this study lower limit of normal eGFR is defined as 90mL/min per 1.73m2 eGFR = 141 X min (Scr/k, 1)α X max (Scr/k, 1)-1.209 X 0.993Age X (1.018 [if female]) X (1.159 [if black]) Where Scr is serum creatinine (mg/dL), k is 0.7 for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/k or 1, and max indicates the maximum of Scr/k or 1, age in years. (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionmL/min per 1.73m2 (Mean)
Day 365Day 729
Abatacept IV6.857.20
Placebo IV5.857.91

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Adjusted Mean Change From Baseline in UPCR Over Time

A repeated measure mixed model that included the baseline UPCR value, randomization stratification factors, time, and time by treatment interaction as fixed effects and subject as a random effect was used. (NCT01714817)
Timeframe: Day 365; Day 729, includes data up to July 1st 2017 when double-blind therapy ended

,
InterventionUPCR (mg/mg) (Mean)
Day 365Day 729
Abatacept IV-2.95-3.13
Placebo IV-2.68-2.72

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Cmax: Maximum Observed Serum Concentration Following Participants Receiving Active Abatacept IV

Cmax: Maximum observed serum concentration following participants receiving active abatacept IV (NCT01714817)
Timeframe: at 1 hour post Day 1 dose and 30 minutes post Day 337 dose

Interventionug/mL (Geometric Mean)
Day 1Day 337
Abatacept IV527.43203.51

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Cmin (ug/mL): Trough Level Serum Concentration of Abatacept Prior to the Administration of the IV Infusion

Trough level serum concentration of abatacept prior to the administration of the IV infusion on Days 1 to 365 (NCT01714817)
Timeframe: Days 1 to 365

Interventionug/mL (Geometric Mean)
Day 15Day 29Day 57Day 85Day 113Day 169Day 281Day 337Day 365
Abatacept IV69.9790.4636.4334.4616.4213.9814.4414.9913.62

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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (g/L)

"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729

,
Interventiong/L (Mean)
ALBUMIN (g/L)HEMOGLOBIN (g/L)PROTEIN, TOTAL (g/L)
Abatacept IV9.28.89.9
Placebo IV8.19.010.1

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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (mmol/L)

"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729

,
Interventionmmol/L (Mean)
BLOOD UREA NITROGEN (mmol/L)CALCIUM, TOTAL (mmol/L)CHLORIDE, SERUM (mmol/L)GLUCOSE, SERUM (mmol/L)PHOSPHORUS, INORGANIC (mmol/L)POTASSIUM, SERUM (mmol/L)SODIUM, SERUM (mmol/L)
Abatacept IV-2.310.097-1.1-0.23-0.077-0.02-0.2
Placebo IV-2.250.108-0.5-0.58-0.037-0.10-0.5

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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (U/L)

"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729

,
InterventionU/L (Mean)
ALANINE AMINOTRANSFERASE (ALT) (U/L)ALKALINE PHOSPHATASE (ALP) (U/L)ASPARTATE AMINOTRANSFERASE (AST) (U/L)G-GLUTAMYL TRANSFERASE (GGT) (U/L)
Abatacept IV-2.28.20.3-5.3
Placebo IV-3.411.70.3-4.1

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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (Umol/L)

"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729

,
Interventionumol/L (Mean)
BILIRUBIN, TOTAL (umol/L)CREATININE (umol/L)
Abatacept IV1.77-5.6
Placebo IV1.00-6.2

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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (x10^9 Cells/L)

"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729

,
Interventionx10^9 cells/L (Mean)
EOSINOPHILS (ABSOLUTE) (x10^9 cells/L)LYMPHOCYTES (ABSOLUTE) (x10^9 cells/L)MONOCYTES (ABSOLUTE) (x10^9 cells/L)NEUTROPHILS (ABSOLUTE) (x10^9 cells/L)PLATELET COUNT (x10^9 cells/L)
Abatacept IV0.0340.141-0.018-2.259-4.9
Placebo IV0.010-0.149-0.050-2.289-9.1

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Median Percent Change From Baseline in UPCR Over Time

"A repeated measure mixed model that included the baseline UPCR value, randomization stratification factors, time, and time by treatment interaction as fixed effects and subject as a random effect was used.~% Change from Baseline = (post baseline - baseline value) / baseline value x 100" (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionPercent (Median)
Day 365Day 729
Abatacept IV-83.77-89.83
Placebo IV-84.12-87.28

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Median Time to Complete Renal Response During the Double-blind Period in All Participants

The estimate of median time to Complete Renal Response is based on Kaplan-Meier analysis. Complete renal response (CR): defined as meeting ALL of the following criteria: eGFR normal OR no less than 85% of the baseline value; Urine protein/creatinine ratio (UPCR) < 0.5; Urine sediment: No cellular casts; Daily corticosteroid dose must be no greater than 10 mg prednisone or equivalent for at least 28 days prior to assessment. (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionDays (Median)
Day 365Day 729
Abatacept IV280.0170.0
Placebo IV309.0282.0

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Median Time to Complete Renal Response During the Double-blind Period in Nephrotic Participants

The estimate of median time to Complete Renal Response in nephrotic participants is based on Kaplan-Meier analysis. Complete renal response (CR): defined as meeting ALL of the following criteria: eGFR normal OR no less than 85% of the baseline value; Urine protein/creatinine ratio (UPCR) < 0.5; Urine sediment: No cellular casts; Daily corticosteroid dose must be no greater than 10 mg prednisone or equivalent for at least 28 days prior to assessment. (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionDays (Median)
Day 365Day 729
Abatacept IV366.0365.0
Placebo IV368.0368.0

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Median Time to First Sustained Change to No Response During the Double-blind Period

"Sustained response defined as response present at 2 consecutive visits approximately 4 weeks apart. No renal response (NR): defined as not meeting criteria for CR or PR or withdrawn~The estimate of median time is based on Kaplan-Meier analysis" (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionDays (Median)
Day 365Day 729
Abatacept IVNANA
Placebo IVNANA

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Median Time to First Treatment Failure and Overall Treatment Failure During the Double-blind Period

"First treatment failure (or Lupus treatment failure) is defined as any of the following: Death, unless due to physical trauma or violence; Renal Flare; sustained doubling of creatinine from baseline (greater of Screening or Study Day 1 value); initiation of rescue therapy for treatment of active lupus nephritis after Study Week 20.~Overall treatment failure is defined as lupus treatment failure plus discontinuation of study drug for any reason except death due to physical trauma or violence, pregnancy or administrative decision by Sponsor.~The hazard ratio is estimated using the Cox proportional hazards model which includes treatment group, stratification variables (baseline ACEis/ARBs use, RACE) and baseline UPCR.~The estimate of median time is based on Kaplan-Meier analysis" (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionDays (Median)
First treatment failure (FTF) - Day 365Overall treatment failure (OTF) - Day 365FTF - Day 729OTF - Day 729
Abatacept IVNANANANA
Placebo IVNANANANA

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Median Time to Partial Renal Response During the Double-blind Period in All Participants

The estimate of median time to Partial Response (PR) is based on Kaplan-Meier analysis. Partial renal response (PR): defined as meeting ALL of the following criteria: Participant does not meet criteria for CR; eGFR no less than 85% of the lesser of the values at screening or randomization (Day 1); UPCR < 0.5 OR 50% reduced from baseline and < 1 if baseline value was < 3, OR 50% reduced from baseline and < 3 if baseline value was 3; Urine sediment: no cellular casts; daily corticosteroid dose no greater than 10 mg/day prednisone or prednisone equivalent for at least 28 days prior to assessment (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionDays (Median)
Day 365Day 729
Abatacept IV226.059.0
Placebo IV253.058.0

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Median Time to Partial Renal Response During the Double-blind Period in Nephrotic Participants

The estimate of median time to Partial Response (PR) in nephrotic participants is based on Kaplan-Meier analysis. Partial renal response (PR): defined as meeting ALL of the following criteria: Participant does not meet criteria for CR; eGFR no less than 85% of the lesser of the values at screening or randomization (Day 1); UPCR < 0.5 OR 50% reduced from baseline and < 1 if baseline value was < 3, OR 50% reduced from baseline and < 3 if baseline value was 3; Urine sediment: no cellular casts; daily corticosteroid dose no greater than 10 mg/day prednisone or prednisone equivalent for at least 28 days prior to assessment (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionDays (Median)
Day 365Day 729
Abatacept IV225.058.5
Placebo IV196.056.0

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Number of Participants With Abatacept Induced Antibody Response Over Time in the Double-blind Period

Participants who experienced a positive antibody response relative to baseline (ECL Assay) (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionParticipants (Number)
Day 365, overall
Abatacept IV7
Placebo IV9

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Number of Participants With Any Adverse Events (AEs) During Year 1 of the Double-blind Period

All AEs were coded and grouped into preferred terms (PT) by system organ class (SOC), using the Medical Dictionary for Regulatory Activities (MedDRA, version 21.0). Investigators determined the intensity of each AE as mild, moderate, severe, or very severe and assessed the relationship to study drug. (NCT01714817)
Timeframe: From Day 1 up to 56 days post last dose in Year 1 of the double-blind period

,
InterventionParticipants (Number)
Participants with Adverse EventsParticipants with Serious Adverse EventsParticipants with infection Adverse EventsParticipants with malignanciesParticipants with autoimmune eventsParticipants with peri-infusional Adverse EventsParticipants with acute infusional Adverse Events
Abatacept IV1884915021072
Placebo IV194391471994

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Number of Participants With Any Adverse Events (AEs) During Year 2 of the Double-blind Period and Long-term Extension

All AEs were coded and grouped into preferred terms (PT) by system organ class (SOC), using the Medical Dictionary for Regulatory Activities (MedDRA, version 21.0). Investigators determined the intensity of each AE as mild, moderate, severe, or very severe and assessed the relationship to study drug. (NCT01714817)
Timeframe: From the first dose in Year 2 of the double-blind period up to 56 days post last dose

,
InterventionParticipants (Number)
Participants with Adverse EventsParticipants with Serious Adverse EventsParticipants with infection Adverse EventsParticipants with malignanciesParticipants with autoimmune events
Abatacept IV1271510007
Placebo IV13725107111

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Number of Participants With Marked Electrolyte Laboratory Abnormalities During Year 1 of the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value SODIUM, SERUM mmol/L 4.0 NA <0.95X LLN OR >1.05X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.05X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier

,
Interventionparticipants (Number)
SODIUM, SERUM, lowSODIUM, SERUM, highPOTASSIUM, SERUM, lowPOTASSIUM, SERUM, highCHLORIDE, SERUM, lowCHLORIDE, SERUM, highCALCIUM, TOTAL, lowCALCIUM, TOTAL, highPHOSPHORUS, INORGANIC, lowPHOSPHORUS, INORGANIC, high
Abatacept IV12370012913
Placebo IV12571010713

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Number of Participants With Marked Electrolyte Laboratory Abnormalities in the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value SODIUM, SERUM mmol/L 4.0 NA <0.95X LLN OR >1.05X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.05X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR NCT01714817)
Timeframe: Day 1 to Day 729

,
Interventionparticipants (Number)
SODIUM, SERUM, lowSODIUM, SERUM, highPOTASSIUM, SERUM, lowPOTASSIUM, SERUM, highCHLORIDE, SERUM, lowCHLORIDE, SERUM, highCALCIUM, TOTAL, lowCALCIUM, TOTAL, highPHOSPHORUS, INORGANIC, lowPHOSPHORUS, INORGANIC, high
Abatacept IV0023000136
Placebo IV0122100043

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Number of Participants With Marked Hematology Laboratory Abnormalities During Year 1 of the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value HEMOGLOBIN g/L 4.0 HB >3 G/DL DECREASE FROM PRE RX HEMATOCRIT vol 6.3 HCT <0.75X PRE RX ERYTHROCYTES x10*12 c/L 5.2 RBC <0.75X PRE RX PLATELET COUNT x10*9 c/L 5.0 PLAT <0.67X LLN OR >1.5X ULN, OR IF PRE RX1.25X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.2X PRE RX OR .750 X10*3 c/uL BASOPHILS (ABSOLUTE) x10*9 c/L 8.3 BASOA IF VALUE > 400/MM3 MONOCYTES (ABSOLUTE) x10*9 c/L 8.3 MONOA IF VALUE > 2000/MM3 LYMPHOCYTES (ABSOLUTE) x10*9 c/L 8.3 LYMPA IF VALUE < .750 X10*3 c/uL OR IF VALUE > 7.50 X10*3 c/uL~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier

,
Interventionparticipants (Number)
HEMOGLOBIN, lowHEMOGLOBIN, highHEMATOCRIT, lowHEMATOCRIT, highERYTHROCYTES, lowERYTHROCYTES, highPLATELET COUNT, lowPLATELET COUNT, highLEUKOCYTES, lowLEUKOCYTES, highEOSINOPHILS (ABSOLUTE), lowEOSINOPHILS (ABSOLUTE), highBASOPHILS (ABSOLUTE), lowBASOPHILS (ABSOLUTE), highMONOCYTES (ABSOLUTE), lowMONOCYTES (ABSOLUTE), highLYMPHOCYTES (ABSOLUTE), lowLYMPHOCYTES (ABSOLUTE), high
Abatacept IV6NA12NA7NA403529NA2NA1NA0811
Placebo IV10NA12NA10NA302125NA6NA1NA11042

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Number of Participants With Marked Hematology Laboratory Abnormalities in the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value HEMOGLOBIN g/L 4.0 HB >3 G/DL DECREASE FROM PRE RX HEMATOCRIT vol 6.3 HCT <0.75X PRE RX ERYTHROCYTES x10*12 c/L 5.2 RBC <0.75X PRE RX PLATELET COUNT x10*9 c/L 5.0 PLAT <0.67X LLN OR >1.5X ULN, OR IF PRE RX1.25X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.2X PRE RX OR .750 X10*3 c/uL BASOPHILS (ABSOLUTE) x10*9 c/L 8.3 BASOA IF VALUE > 400/MM3 MONOCYTES (ABSOLUTE) x10*9 c/L 8.3 MONOA IF VALUE > 2000/MM3 LYMPHOCYTES (ABSOLUTE) x10*9 c/L 8.3 LYMPA IF VALUE < .750 X10*3 c/uL OR IF VALUE > 7.50 X10*3 c/uL~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 729

,
Interventionparticipants (Number)
HEMOGLOBIN, lowHEMOGLOBIN, highHEMATOCRIT, lowHEMATOCRIT, highERYTHROCYTES, lowERYTHROCYTES, highPLATELET COUNT, lowPLATELET COUNT, highLEUKOCYTES, lowLEUKOCYTES, highEOSINOPHILS (ABSOLUTE), lowEOSINOPHILS (ABSOLUTE), highBASOPHILS (ABSOLUTE), lowBASOPHILS (ABSOLUTE), highMONOCYTES (ABSOLUTE), lowMONOCYTES (ABSOLUTE), highLYMPHOCYTES (ABSOLUTE), lowLYMPHOCYTES (ABSOLUTE), high
Abatacept IV8NA6NA4NA21193NA11NA0NA0430
Placebo IV9NA2NA2NA40245NA6NA0NA0620

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Number of Participants With Marked Liver and Kidney Function Laboratory Abnormalities During Year 1 of the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value ALKALINE PHOSPHATASE (ALP) U/L 5.0 ALP >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX ASPARTATE AMINOTRANSFERASE (AST) U/L 5.0 AST >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX ALANINE AMINOTRANSFERASE (ALT) U/L 5.0 ALT >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX G-GLUTAMYL TRANSFERASE (GGT) U/L 5.0 GGT >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX BILIRUBIN, TOTAL umol/L 5.1 TBILI >2X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX BILIRUBIN, DIRECT umol/L 5.1 DBILI >1.5X ULN, OR IF PRE RX>ULN THEN USE >2X PRE RX BLOOD UREA NITROGEN mmol/L 5.1 BUN >2X PRE RX CREATININE umol/L 5.0 CREAT >1.5X PRE RX~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier

,
Interventionparticipants (Number)
ALKALINE PHOSPHATASE (ALP), lowALKALINE PHOSPHATASE (ALP), highASPARTATE AMINOTRANSFERASE (AST), lowASPARTATE AMINOTRANSFERASE (AST), highALANINE AMINOTRANSFERASE (ALT), lowALANINE AMINOTRANSFERASE (ALT), highG-GLUTAMYL TRANSFERASE (GGT), lowG-GLUTAMYL TRANSFERASE (GGT), highBILIRUBIN, TOTAL, lowBILIRUBIN, TOTAL, highBILIRUBIN, DIRECT, lowBILIRUBIN, DIRECT, highBLOOD UREA NITROGEN, lowBLOOD UREA NITROGEN, highCREATININE, lowCREATININE, high
Abatacept IVNA1NA5NA8NA17NA0NA0NA20NA24
Placebo IVNA1NA0NA2NA15NA0NA0NA12NA20

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Number of Participants With Marked Liver and Kidney Function Laboratory Abnormalities in the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value ALKALINE PHOSPHATASE (ALP) U/L 5.0 ALP >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX ASPARTATE AMINOTRANSFERASE (AST) U/L 5.0 AST >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX ALANINE AMINOTRANSFERASE (ALT) U/L 5.0 ALT >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX G-GLUTAMYL TRANSFERASE (GGT) U/L 5.0 GGT >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX BILIRUBIN, TOTAL umol/L 5.1 TBILI >2X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX BILIRUBIN, DIRECT umol/L 5.1 DBILI >1.5X ULN, OR IF PRE RX>ULN THEN USE >2X PRE RX BLOOD UREA NITROGEN mmol/L 5.1 BUN >2X PRE RX CREATININE umol/L 5.0 CREAT >1.5X PRE RX~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 729

,
Interventionparticipants (Number)
ALKALINE PHOSPHATASE (ALP), lowALKALINE PHOSPHATASE (ALP), highASPARTATE AMINOTRANSFERASE (AST), lowASPARTATE AMINOTRANSFERASE (AST), highALANINE AMINOTRANSFERASE (ALT), lowALANINE AMINOTRANSFERASE (ALT), highG-GLUTAMYL TRANSFERASE (GGT), lowG-GLUTAMYL TRANSFERASE (GGT), highBILIRUBIN, TOTAL, lowBILIRUBIN, TOTAL, highBILIRUBIN, DIRECT, lowBILIRUBIN, DIRECT, highBLOOD UREA NITROGEN, lowBLOOD UREA NITROGEN, highCREATININE, lowCREATININE, high
Abatacept IVNA4NA2NA4NA17NA0NA1NA10NA17
Placebo IVNA0NA3NA3NA11NA0NA0NA9NA16

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Number of Participants With Marked Urinalysis Laboratory Abnormalities During Year 1 of the Double Blind Period

LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value PROTEIN, URINE Unknown UPRO IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 GLUCOSE, URINE N/A UGLU IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 BLOOD, URINE N/A UBLD IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 RBC, URINE hpf 5.0 URBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 WBC, URINE hpf 5.0 UWBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier

,
Interventionparticipants (Number)
PROTEIN, URINE, lowPROTEIN, URINE, highGLUCOSE, URINE, lowGLUCOSE, URINE, highBLOOD, URINE, lowBLOOD, URINE, highRed blood cells (RBC), URINE, lowRed blood cells (RBC), URINE, highWhite blood cells (WBC), URINE, lowWhite blood cells (WBC), URINE, high
Abatacept IVNA0NA0NA0NA93NA91
Placebo IVNA0NA0NA0NA103NA98

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Number of Participants With Marked Urinalysis Laboratory Abnormalities in the Double Blind Period

LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value PROTEIN, URINE Unknown UPRO IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 GLUCOSE, URINE N/A UGLU IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 BLOOD, URINE N/A UBLD IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 RBC, URINE hpf 5.0 URBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 WBC, URINE hpf 5.0 UWBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 (NCT01714817)
Timeframe: Day 1 to Day 729

,
Interventionparticipants (Number)
PROTEIN, URINE, lowPROTEIN, URINE, highGLUCOSE, URINE, lowGLUCOSE, URINE, highBLOOD, URINE, lowBLOOD, URINE, highRed blood cells (RBC), URINE, lowRed blood cells (RBC), URINE, highWhite blood cells (WBC), URINE, lowWhite blood cells (WBC), URINE, high
Abatacept IVNA0NA0NA0NA58NA46
Placebo IVNA0NA0NA0NA55NA59

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Number of Participants With Other Marked Chemistry Laboratory Abnormalities During Year 1 of the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value CALCIUM, TOTAL mmol/L 5.2 CA <0.8X LLN OR >1.2X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.25X PRE RX OR 1.25X ULN, OR IF PRE RXULN GLUCOSE, SERUM mmol/L 4.1 GLUC <65 mg/dL, OR >220 mg/dL PROTEIN, TOTAL g/L 5.0 TPRO <0.9X LLN OR >1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE 1.1X PRE RX OR 2X PRE R~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier

,
Interventionparticipants (Number)
GLUCOSE, SERUM, lowGLUCOSE, SERUM, highPROTEIN, TOTAL, lowPROTEIN, TOTAL, highALBUMIN, lowALBUMIN, high
Abatacept IV331044010NA
Placebo IV29526111NA

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Number of Participants With Other Marked Chemistry Laboratory Abnormalities in the Double Blind Period

"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value CALCIUM, TOTAL mmol/L 5.2 CA <0.8X LLN OR >1.2X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.25X PRE RX OR 1.25X ULN, OR IF PRE RXULN GLUCOSE, SERUM mmol/L 4.1 GLUC <65 mg/dL, OR >220 mg/dL PROTEIN, TOTAL g/L 5.0 TPRO <0.9X LLN OR >1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE 1.1X PRE RX OR 2X PRE R~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 729

,
Interventionparticipants (Number)
GLUCOSE, SERUM, lowGLUCOSE, SERUM, highGLUCOSE, FASTING SERUM, lowGLUCOSE, FASTING SERUM, highPROTEIN, TOTAL, lowPROTEIN, TOTAL, highALBUMIN, lowALBUMIN, high
Abatacept IV153331024NA
Placebo IV24211735NA

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Number of Participants With Sustained Change From Higher Level of Response to no Response During the Double-blind Period

Sustained change to no response is defined as going from CR (or PR) to NR and remaining in NR for at least 2 consecutive visits; visits should be approximately 4 weeks apart. This analysis will be based on time from response CR (or PR) to the first visit in which the no response (NR) was achieved and sustained to the next visit. (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionParticipants (Number)
Day 365Day 729
Abatacept IV552
Placebo IV356

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Percentage of Participants in Treatment Failure Over Time During the Double-blind Period

"Lupus treatment failure is defined as any of the following: Death, unless due to physical trauma or violence; Renal Flare; sustained doubling of creatinine from baseline (greater of Screening or Study Day 1 value); initiation of rescue therapy for treatment of active lupus nephritis after Study Week 20.~Overall treatment failure is defined as lupus treatment failure plus discontinuation of study drug for any reason except death due to physical trauma or violence, pregnancy or administrative decision by Sponsor." (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionPercentage (Number)
Lupus treatment failure (LTF) - Day 365Overall treatment failure (OTF) - Day 365LTF - Day 729OTF - Day 729
Abatacept IV3.54.54.55.2
Placebo IV4.44.95.38.4

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Percentage of Participants With Ranked Outcome of Complete Renal Response, Partial Renal Response (PR), and No Renal Response (NR) During the Double-blind Period

Complete Renal Response or Complete Response (CR): defined as meeting ALL of the following criteria: eGFR normal OR no less than 85% of the baseline value; UPCR < 0.5; Urine sediment: No cellular casts; Daily corticosteroid dose must be no greater than 10 mg prednisone or equivalent for at least 28 days prior to assessment. Partial Renal Response or Partial Response (PR): defined as meeting ALL of the following criteria: Participant does not meet criteria for CR; eGFR no less than 85% of the lesser of the values at screening or randomization (Day 1); UPCR < 0.5 OR 50% reduced from baseline and < 1 if baseline value was < 3, OR 50% reduced from baseline and < 3 if baseline value was greater than or equal to 3; Urine sediment: no cellular casts; daily corticosteroid dose no greater than 10 mg/day prednisone or prednisone equivalent for at least 28 days prior to assessment. No Renal Response or No Response (NR): defined as not meeting criteria for CR or PR or withdrawn (NCT01714817)
Timeframe: Day 365, Day 729

,
InterventionPercentage (Number)
CR - Day 365PR - Day 365NR - Day 365CR - Day 729PR - Day 729NR - Day 729
Abatacept IV35.120.844.160.725.913.4
Placebo IV33.521.744.853.622.723.6

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Summary Statistics for Diastolic Blood Pressure

Summary statistics for diastolic blood pressure (NCT01714817)
Timeframe: Day 1 to Day 729

,
InterventionmmHg (Mean)
Day 1, end of observationDay 365, end of observationDay 729, end of observation
Abatacept IV76.573.567.5
Placebo IV77.077.572.7

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Summary Statistics for Heart Rate

Summary statistics for Heart Rate (NCT01714817)
Timeframe: Day 1 to Day 729

,
Interventionbeats per minute (Mean)
Day 1, end of observationDay 365, end of observationDay 729, end of observation
Abatacept IV80.678.576.2
Placebo IV81.470.076.7

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Summary Statistics for Systolic Blood Pressure

Summary statistics for systolic blood pressure (NCT01714817)
Timeframe: Day 1 to Day 729

,
InterventionmmHg (Mean)
Day 1, end of observationDay 365, end of observationDay 729, end of observation
Abatacept IV122.0112.3108.6
Placebo IV122.6115.0114.2

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AUC0-inf - Area Under the Concentration-time Curve From Time Zero to Infinity (Extrapolated)

Bioequivalence based on AUC0-inf (NCT00907907)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*hr/mL (Mean)
Mycophenolate Mofetil26.6752
Cellcept®26.7354

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AUC0-t - Area Under the Concentration-time Curve From Time Zero to Time of Last Non-zero Concentration (Per Participant)

Bioequivalence based on AUC0-t (NCT00907907)
Timeframe: Blood samples collected over 72 hour period

Interventionµg*hr/mL (Mean)
Mycophenolate Mofetil25.2539
Cellcept®25.3253

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Cmax - Maximum Observed Concentration

Bioequivalence based on Cmax (NCT00907907)
Timeframe: Blood samples collected over 72 hour period

Interventionµg/mL (Mean)
Mycophenolate Mofetil11.0939
Cellcept®11.1715

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Number of Non-Relapse Mortalities

Number of subjects expired without disease progression/relapse. (NCT01231412)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Arm I (MMF and CSP)12
Arm II (MMF, CSP, and Sirolimus)4
Arm 0 (CSP and Sirolimus)0

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Number of of Participants Surviving Overall

Number of subjects surviving overall post-transplant. (NCT01231412)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Arm I (MMF and CSP)53
Arm II (MMF, CSP, and Sirolimus)75
Arm 0 (CSP and Sirolimus)6

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Number of Participants With Relapse/Progression

"Relapse/Progression criteria:~CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever >38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~AML, ALL, MDS >5% blasts by morphologic or flow cytometric evaluation of the BMA or appearance of extramedullary disease CLL ≥1 of: Physical exam/imaging studies ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or numb" (NCT01231412)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Arm I (MMF and CSP)16
Arm II (MMF, CSP, and Sirolimus)16
Arm 0 (CSP and Sirolimus)1

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Number of Patients With Chronic Extensive GVHD

Number of patients who developed chronic extensive GVHD post-transplant. The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD. (NCT01231412)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Arm I (MMF and CSP)38
Arm II (MMF, CSP, and Sirolimus)43
Arm 0 (CSP and Sirolimus)3

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Number of Patients With Grades II-IV Acute GVHD

"Number of patients with grades II-IV acute GVHD~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT01231412)
Timeframe: At day 100 post-transplant

InterventionParticipants (Count of Participants)
Arm I (MMF and CSP)39
Arm II (MMF, CSP, and Sirolimus)22
Arm 0 (CSP and Sirolimus)3

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Number of Patients With Grades III-IV Acute GVHD

"Number of patients with grades III-IV acute GVHD~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT01231412)
Timeframe: Up to 100 days

InterventionParticipants (Count of Participants)
Arm I (MMF and CSP)8
Arm II (MMF, CSP, and Sirolimus)2
Arm 0 (CSP and Sirolimus)0

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Number of Participants With Disease-free Survival (DFS)

Disease free survival (DFS), defined as the time to death, relapse or disease progression. (NCT01871441)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Haploidentical Allogeneic HSCT)1

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Number of Participants With Relapse of Disease

Relapse of Disease is defined as the return of a disease or the signs and symptoms of a disease after a period of improvement. Relapse is almost always associated with the immunological failure of the donor immune system to recognize and/or respond to reemergence of a tumor. The number of participants with relapse of disease will be collected. (NCT01871441)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Treatment (Haploidentical Allogeneic HSCT)2

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Percentage of Participants With at Least One Adverse Event (AE)

An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events. (NCT00551291)
Timeframe: Up to approximately 2 years

Interventionpercentage of participants (Number)
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta90.00

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Mean Number of Blood Transfusions Per Visit

(NCT00551291)
Timeframe: Up to approximately 2 years

Interventiontransfusions/visit (Mean)
Baseline (n=8)Week 12 (n=6)Week 18 (n=5)End of Study (n=3)
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta4.135.832.802.33

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Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement

International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) <11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment. (NCT00551291)
Timeframe: Up to approximately 2 years

Interventionpercentage of participants (Number)
Week 12 (n=4)Week 18 (n=7)End of study (n=3)
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta50.0071.43100.00

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Donor (Allogeneic) Hematopoietic Engraftment

Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT. (NCT01626092)
Timeframe: Day 100 Following Hematopoietic Cell Transplant (HCT)

Interventionparticipants (Number)
Intent-To-Treat Patients1

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Event-free Survival

The one-year event free survival is defined by the patient who has neither experienced relapse nor death within one year after post transplantation. And the rate is calculated by computing the ratio between total number of one year event free survival patients and the total number of patients. (NCT01807611)
Timeframe: One year post-transplantation

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients49

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Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD)

Acute and chronic graft-vs.-host disease will be evaluated using the standard grading criteria. The estimate will be the number of recipients who experienced GVHD divided by the total number of patients considered in this group. (NCT01807611)
Timeframe: 100 days post-transplant for acute GVHD; one year post-transplant for chronic GVHD .

InterventionParticipants (Count of Participants)
Number of Transplant Recipients With Acute Graft Versus Host Disease (aGVHD)24
Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD)16

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Number of Transplant Recipients With Malignant Relapse

Bone marrow studies for disease status evaluation will be performed at 1-year post-transplant. Testing will include a research evaluation for minimal residual disease. (NCT01807611)
Timeframe: One-year post-transplantation

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients18

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Number of Transplant Recipients With Successful Engraftment

Neutrophil engraftment will be determined using the parameters put forth by the Center for International Blood and Marrow Registry. Assessments will be made upon review of daily complete blood count and serial chimerism studies. Successful engraftment for the purposes of this objective will be patients who do not experience graft failure. (NCT01807611)
Timeframe: 42 days post engraftment

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients70

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Overall Survival

The one-year survival is defined by the patient who has not died within one year after post transplantation. And the rate is calculated by computing the ratio between total number of one year survival patients and the total number of patients. (NCT01807611)
Timeframe: one year post-transplantation

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients59

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Disease-Free Survival (DFS)

Estimate the DFS at one-year post-transplantation. The event is defined as relapse or death due to relapse. The number of participants who did not relapse up to one year post transplant is reported. (NCT01621477)
Timeframe: one year post transplant

Interventionparticipants (Number)
Treatment7

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Event-Free Survival (EFS)

Estimate the EFS at one-year post-transplantation. The event is defined as relapse or death due to any cause. The number of participants who were alive without relapse at one year post-transplant is reported. (NCT01621477)
Timeframe: one year post transplant

Interventionparticipants (Number)
Treatment4

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Incidence of Malignant Relapse

Estimate the incidence of malignant relapse at one year post-transplant. The number of participants with malignant relapse or progressive disease is given. Relapse was evaluated using standard WHO criteria for each disease. (NCT01621477)
Timeframe: One year post transplantation.

Interventionparticipants (Number)
Treatment10

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One-year Survival (OS)

Evaluate the number of participants alive at 1 year. The number of participants surviving to one-year post-transplantation is given. (NCT01621477)
Timeframe: One year post transplant

Interventionparticipants (Number)
Treatment7

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Incidence and Severity of Acute Graft Versus Host Disease (GVHD)

The number of participants with acute GVHD is given, organized by grade. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe. (NCT01621477)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
No Acute GVHDGrade IGrade IIGrade IIIGrade IV
Treatment93131

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Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)

The severity of chronic GVHD will be described. Chronic GVHD was evaluated using NIH Consensus Global Severity Scoring. The number of participants with chronic GVHD is given, organized by severity. (NCT01621477)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
No Chronic GVHDMildModerateSevere
Treatment15020

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Neutrophil Engraftment - The Days Till ANC Recovery

The primary engraftment endpoint, neutrophil engraftment, is defined as the first of three consecutive days on which the absolute neutrophil count is > 500/µL. The duration and extent of neutrophil engraftment is the time from transplant to neutrophil engraftment. (NCT00544115)
Timeframe: Up to 180 days post transplant

Interventiondays (Median)
Regimen I17
Regimen II16
Regimen III15
Regimen IV14
Regimen V18
Regimen VI16

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Two-year Overall Survival

Overall survival (OS) was measured from peripheral stem cell infusion to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. Participants were followed up to 2 years after transplant and Kaplan-Meier survival analysis was used to generate the two-year Overall Survival estimate presented. (NCT00544115)
Timeframe: Up to 2 years post transplant

Interventionpercentage of survival probability (Number)
Regimen I58
Regimen II50
Regimen III54
Regimen IV50
Regimen V38
Regimen VI50

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Change in EMT Score

"Change (M12 - M3) in EMT Score. Progression in EMT score is defined as an increase by >=1 of EMT score from M3 to M12.~EMT score 0 (the best): <1 EMT score 1 (the better) : 1-10% of tubular atrophy EMT score 2 : 10-25% of tubular atrophy EMT score 3 : 25-50% of tubular atrophy EMT score 4 (the worst): >50% of tubular atrophy" (NCT01079143)
Timeframe: M3 and M12 post transplantation

Interventionscores on a scale (Mean)
Certican EMT+-0.3
Certican EMT-0.9
Neoral EMT+-0.3
Neoral EMT-0.6

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Change in Urine Protein/Creatinine Ratio (Without Imputation)

One of the factors associated with EMT progression between M3 and M12 according to univariate analysis (ITT biopsies M3 & M12). (NCT01079143)
Timeframe: Month 3 (baseline), Month 12

Interventionmg/mmol (Mean)
Certican EMT+44.4
Certican EMT-3.5
Neoral EMT+16.0
Neoral EMT-29.8

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Change From Baseline (M3) in Estimated Glomerular Filtration Rate (eGFR)

"eGFR was calculated according to the abbreviated Modification of Diet in Renal Disease (MDRD) Formula and creatinine clearance according to the Cockcroft-Gault formula (mL/min/1.73m²).~LOCF = Last observation carried forward" (NCT01079143)
Timeframe: M3 (baseline) to M12 post transplantation

,
InterventionmL/min/1.73m^2 (Least Squares Mean)
without imputationimputation by LOCF(96, 97)
Certican6.995.96
Neoral2.542.15

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Change in Estimated Glomerular Filtration Rate (eGFR) at M12 From Baseline (M3) - ANCOVA Model

The average patient renal function was evaluated on the basis of eGFR was calculated according to the abbreviated MDRD formula and creatinine clearance according to the Cockcroft-Gault formula (mL/min/1.73m²). (NCT01079143)
Timeframe: Baseline (M3), M12

,,,
InterventionmL/min/1.73m² (Mean)
without imputationimputation by LOCF (36, 60, 39, 58)
Certican EMT-5.627.3
Certican EMT+8.6-11.9
Neoral EMT-3.8-4.9
Neoral EMT+1.55.1

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Change in Interstitial Fibrosis/Tabular Atrophy (IF/TA) Grade

Difference (M12 - M3) in IF/TA grade. IF/TA grade was assessed during centralized reading according to the Banff 2005/2007 classification comprising grade I (<25%), grade II (25-50%) and grade III (>50% of lesions). (NCT01079143)
Timeframe: M3 and M12 post transplantation

,,,
InterventionNumber of Participants (Number)
Difference in IF/TA grade -1Difference in IF/TA grade 0Difference in IF/TA grade 1Difference in IF/TA grade 2Difference in IF/TA grade 3
Certican EMT-121183NA
Certican EMT+59732
Neoral EMT-233135NA
Neoral EMT+791150

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Change in Percentage of Interstitial Fibrosis (IF) by Numerical Quantification

Percentage of IF measured by numerical quantification. The IF percentage was transposed in grade according to the following rule: grade 0 for an IF % ≤5%, grade I for an IF % ranging from >5% to < 25%, grade II for an IF % ranging from 25 to 50%, grade III for an IF % >50%. Interstitial graft fibrosis has been identified as the primary cause of graft loss following death with a functional graft [3-5]. (NCT01079143)
Timeframe: M3 and M12 post transplantation

,,,
InterventionPercentage of IF (Mean)
Percentage of IF at M3 (n=26,43,32,53)Percentage of IF at M12 (n=24,42,32,53)Change in Percentage of IF (n=24,42,32,53)
Certican EMT-15.920.94.9
Certican EMT+22.827.65.1
Neoral EMT-17.820.42.7
Neoral EMT+23.427.43.9

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Incidence (Number) of BPAR

A biopsy proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB or III as per 2005/2007 Banff histological classification system. (NCT01079143)
Timeframe: M6 and M12 post transplantation

,,,
InterventionNumber of participants (Number)
M6 - NoM6 - YesM12 - NoM12 - Yes
Certican EMT-5284317
Certican EMT+333297
Neoral EMT-590563
Neoral EMT+390372

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Incidence (Number) of Participants With Graft Losses

If a participant underwent a graft nephrectomy, then the day of nephrectomy was considered as the day of graft loss. (NCT01079143)
Timeframe: M6 and M12 post transplantation

,,,
InterventionParticipants (Number)
M6 - NoM6 - YesM12 - NoM12 - Yes
Certican EMT-600564
Certican EMT+360351
Neoral EMT-590590
Neoral EMT+390381

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Incidence (Number) of Subclinical Rejections and Borderline Lesions

"Incidence of subclinical rejections and borderline lesions with regards to histological evidence of rejection with clinical findings.~Subclinical rejections: rejection without clinical symptoms which is diagnosed by chance when a graft biopsy is performed.~Clinically suspected BPAR: rejection suspected because of the presence of clinical symptoms (fever, pain, increase of creatinine) and then confirmed by the graft biopsy.~Borderline lesions: suspicious for acute T-cell mediated rejection. This category is used when no intimal arteritis is present, but there are foci of tubulitis with minor interstitial infiltration or interstitial infiltration with mild tubulitis." (NCT01079143)
Timeframe: M3

,
InterventionParticipants (Number)
Subclinical rejections-No (n=68, 84)Subclinical rejections- Yes (n=68, 84)Subclinical rejections- missing (n=68, 84)Clinically suspected BPAR - No (N=68, 84)Clinically suspected BPAR - Yes (N=68, 84)Clinically suspected BPAR - Missing (N=68, 84)Borderline lesions - No (n=68,84)Borderline lesions - Yes (n=68,84)Borderline lesions - Missing (n=68,84)
Certican671168016261
Neoral8401840171131

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Interstitial Fibrosis/Tabular Atrophy (IF/TA)

Incidence and severity of IF/TA according to 2005/2007 Banff classification system grades (grades l to lll). IF/TA grade was assessed during centralized reading according to the Banff 2005/2007 classification comprising grade I (<25%), grade II (25-50%) and grade III (>50% of lesions). (NCT01079143)
Timeframe: M3 and M12 post transplantation

,,,
InterventionNumber of participants (Number)
Interstitial Fibrosis/Tubular Atrophy (IF/TA)IF/TA grade at M3 at grade 1IFTA grade at M3 at grade IIIF/TA grade at M3 at grade IIIIF/TA grade at M12 at grade 0IT/TA grade at M12 at grade IIF/TA grade at M12 at grade IIIF/TA grade at M12 at grade III
Certican EMT-38500201850
Certican EMT+1311209863
Neoral EMT-44900311561
Neoral EMT+13171171591

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Number of Participants With Epithelial-mesenchymal Transition (EMT) Score

"Incidence and severity of EMT score. The intensity of EMT markers expression present and detectable in the renal graft at an early stage following transplantation is known as EMT score.~EMT score 0 (the best): <1 EMT score 1 (the better): 1-10% of tubular atrophy EMT score 2: 10-25% of tubular atrophy EMT score 3: 25-50% of tubular atrophy EMT score 4 (the worst): >50% of tubular atrophy" (NCT01079143)
Timeframe: M3 and M12 post transplantation

,,,
Interventionparticipants (Number)
EMT Score 0 at M3 (n= 26,43,32, 53)EMT Score 1 at M3 (n= 26,43,32, 53)EMT Score 2 at M3 (n= 26,43,32, 53)EMT Score 3 at M3 (n= 26,43,32, 53)EMT Score 4 at M3 (n= 26,43,32, 53)EMT Score 0 at M12 (n= 25,41,32, 53)EMT Score 1 at M12 (n= 25,41,32, 53)EMT Score 2 at M12 (n= 25,41,32, 53)EMT Score 3 at M12 (n= 25,41,32, 53)EMT Score 4 at M12 (n= 25,41,32, 53)EMT Missing Score at M12 (n= 25,41,32, 53)
Certican EMT-2617000131111422
Certican EMT+0017901710431
Neoral EMT-2825000191712410
Neoral EMT+0020840910940

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Number of Participants With Epithelial-mesenchymal Transition (EMT) Status

Incidence and severity of EMT status. EMT status was determined centrally using the graft biopsy taken at 3 months. The result was quickly obtained (within 7 to 15 days) and sent to the company in charge of randomization in order to allocate each patient to a treatment group and to provide the investigator with this information without confirming EMT status. (NCT01079143)
Timeframe: M3 and M12 post transplantation

,,,
InterventionParticipants (Number)
EMT Status at M3-Negative (n=26,43,32,53)EMT Status at M3- Positive (n=26,43,32,53)EMT Status at M3 - Not done (n=26,43,32,53)EMT Status at M12- Negative (n=25,41,32,53)EMT Status at M12- Positive (n=25,41,32,53)EMT Status at M12- Not done (n=25,41,32,53)
Certican EMT-430024170
Certican EMT+02608170
Neoral EMT-530036170
Neoral EMT+03209230

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Number of Participants With Progression of Renal Fibrosis Using Numerical Quantification

Comparisons according to epithelial-mesenchymal transition (EMT) profile and immunosuppressive treatment (NCT01079143)
Timeframe: M3 to M12 post transplantation

,,,
InterventionParticipants (Number)
Fibrosis Progression - No (n=24, 42, 31, 53)Fibrosis progression - Yes (n=24, 42,31,43)Fibrosis progression - Missing (n=24, 42, 31, 53)
Certican EMT-30121
Certican EMT+1862
Neoral EMT-42110
Neoral EMT+19120

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Number of Participants With Progression of Renal Graft Fibrosis (Primary Comparison - ITT Population

"Progression of Interstitial Fibrosis/Tabular Atrophy (IF/TA) is the percentage (%) of participants with an increase >= 1 in IF/TA grade according to Banff (2005 - 2007)according to Epithelial-mesenchymal transition (EMT) profile and by treatment groups.~Grade I (the better): mild interstitial fibrosis and tubular atrophy (<25% of cortical area) Grade II : moderate interstitial fibrosis and tubular atrophy (26-50% of cortical area) Grade III (the worse) : severe interstitial fibrosis and tubular atrophy (>50% of cortical area)" (NCT01079143)
Timeframe: Month 3 (M3) and Month 12 (M12) post transplantation

,
InterventionParticipants (Number)
Participants with an IF/TA grade <= II at M3Participants with Fibrosis progression, M3 to M12
Certican EMT+2612
Neoral EMT+3116

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Risk Factors of IF/TA Progression

"Composite factors regarding fibrosis progression at 12 months using a logistic regression model; the parameters initially considered concerned the demographic characteristics of both recipient and donor, transplantation characteristics, hypertension, diabetes, study treatments, immunosuppression, EMT, IF/TA, function at M3, the onset of acute rejection, the presence of anti-donor antibodies at M12, infections and BK virus viremia.~Arteriolar hyaline thickening is thickening of the walls of arterioles by the deposition of homogeneous pink hyaline material.~BPAR is biopsy proven acute rejection. TEM progression is the increase ≥ 1 of TEM score between Month 3 and Month 12" (NCT01079143)
Timeframe: M12 post transplantation

,
InterventionParticipants (Number)
Donor Sex - MaleDonor Sex - FemaleDonor Age - <=50 yearsDonor Age - >50 yearsExpanded Criteria donor: NOExpanded Criteria Donor: YesDelayed graft function: NoDelayed graft function: YesCC at M3 <50 mL/min/1.73m^2CC at M3 >=50 mL/min/1.73m^2 (n=67, 85)Mesangial matrix increase at M3 - 0 (n=64, 85)Mesangial matrix (mm) increase at M3: 1 (n=64, 85)Mesangial matrix increase at M3: 2 (n=64, 85)Interstitial fibrosis (ci) at M3: 0 (n=66, 85)Interstitial fibrosis (ci) at M3: 1 (n=66, 85)Interstitial fibrosis (ci) at M3: 2 (n=66, 85)Arteriolar hyaline thickening (ah) at M3: 0Arteriolar hyaline thickening (ah) at M3: 1Arteriolar hyaline thickening (ah) at M3: 2Arteriolar hyaline thickening (ah) at M3: 3BPAR - NoBPAR - YesTEM Progression fron M3-M12: No (n=67, 83)TEM Progression fron M3-M12: Yes (n=67, 83)TEM Progression fron M3-M12: Missing (n=67, 83)
No- No Fibrosis Progression4838483863237610355084105527356218179757263
Yes- Fibrosis Progression452228394027521540275923521403615106551226410

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Severity of BPAR

"A biopsy proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB or III as per 2005/2007 Banff histological classification system.~Biopsy graded IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IIA: Mild to moderate intimal arteritis. Biopsy graded IIB: Severe intimal arteritis comprising > 25% of the lumenal area.~Biopsy graded III: Transmural (full vessel wall thickness) arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (with accompanying lymphocytic inflammation)." (NCT01079143)
Timeframe: M6 and M12 post transplantation

,,,
InterventionParticipants (Number)
M6: Banff type Grade IAM6: Banff type Grade IBM6: Banff type Grade IIAM6: Banff type Grade IIBM6: Banff type Grade IIIM12: Banff type Grade IAM12: Banff type Grade IBM12: Banff type Grade IIAM12: Banff type Grade IIBM12: Banff type Grade III
Certican EMT-2401075110
Certican EMT+0010022100
Neoral EMT-0000000100
Neoral EMT+0000010000

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Treatment Failures

A treatment failure is defined as biopsy proven acute rejection (BPAR), a graft loss, a death or a loss to follow up. It was assessed between randomization and 6 and 12 months post-transplantation. (NCT01079143)
Timeframe: M6 and M12 post transplantation

,,,
InterventionNumber of Participants (Number)
M6: Treatment Failure- NoM6: Treatment Failure- YesM12: Treatment Failure- NoM12: Treatment Failure- YesM6: BPAR - NoM6: BPAR -YesM12: BPAR - NoM12: BPAR - YesM6: Graft Loss - NoM6: Graft Loss - YesM12: Graft Loss - NoM12: Graft Loss - YesM6: Death - NoM6: Death - YesM12: Death - NoM12: Death - YesM6: Loss to follow-up - NoM6: Loss to follow-up - YesM12: Loss to follow-up - NoM12: Loss to follow-up - Yes
Certican EMT-52842185284317600564600600600591
Certican EMT+333297333297360351360360360360
Neoral EMT-590563590563590590590590590590
Neoral EMT+390363390372390381390390390390

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Type of Biopsy Proven Acute Rejection (BPAR)

"A biopsy proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB or III as per 2005/2007 Banff histological classification system.~Biopsy graded IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IIA: Mild to moderate intimal arteritis. Biopsy graded IIB: Severe intimal arteritis comprising > 25% of the lumenal area.~Biopsy graded III: Transmural (full vessel wall thickness) arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (with accompanying lymphocytic inflammation)." (NCT01079143)
Timeframe: M6 and M12 post transplantation

,,,
InterventionParticipants (Number)
Cellular AR - NoCellular AR - Yes
Certican EMT-4614
Certican EMT+306
Neoral EMT-581
Neoral EMT+381

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Allograft Rejection Rates at 30 Days

Acute Allograft Rejection (NCT00970073)
Timeframe: 30 days

InterventionParticipants (Count of Participants)
Delayed CNI Group 13
Delayed CNI Group 21
Early CNI / Control Arm0

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Estimated Glomerular Filtration Rate (eGFR) at 12 Months Post-surgery

Postoperative acute kidney injury is measured as reduced (eGFR) within 12 months post-surgery. (NCT00970073)
Timeframe: 12 Months

InterventionmL/min (Median)
Delayed CNI Group 172
Delayed CNI Group 287.8
Early CNI / Control Arm51.0

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Graft Survival

90% graft survival, related to the deaths of 3 patients during the study period. (NCT00970073)
Timeframe: 12 months post transplant

InterventionParticipants (Count of Participants)
Delayed CNI Group 110
Delayed CNI Group 29
Early CNI / Control Arm8

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Patient Survival

(NCT00970073)
Timeframe: 12 months post-transplant

InterventionParticipants (Count of Participants)
Delayed CNI Group 110
Delayed CNI Group 29
Early CNI / Control Arm8

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Number of Participants That Experience One Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplant (HSCT)

To assess relapse free survival in participants undergoing Hematopoietic Stem Cell Transplant (HSCT) using the Thomas Jefferson University 2 step approach with an extra day inserted between the donor lymphocyte infusion (DLI) and administration of cyclophosphamide. (NCT01341301)
Timeframe: 1 year after undergoing hematopoietic stem cell transplant

InterventionParticipants (Count of Participants)
Allogeneic HSCT5

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Number of Participants With Grade II-III and III-IV Acute GVHD and Chronic GVHD

"Number of patients who developed acute GVHD post-transplant. Grade I +1 to +2 skin rash, No gut or liver involvement Grade II +1 to +3 skin rash, +1 gastrointestinal involvement and/or +1 liver involvement Grade III +2 to +4 gastrointestinal involvement and/or +2 to +4 liver involvement with or without a rash Grade IV Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death~The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD." (NCT00104858)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy and Rituximab Followed by HCT)46

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Number of Participants With Relapse/Progression

"Relapse/Progression criteria for CLL~Progressive disease:~≥1 of: Physical exam/imaging studies ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~Relapsed disease:~Criteria of progression occurring 6 months after achievement of complete or partial remission." (NCT00104858)
Timeframe: Day 84

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy and Rituximab Followed by HCT)1

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Number of Patients Achieving Complete Response and Partial Response (Overall Response Rate)

"Complete Remission (CR):~Imaging studies (Xray, CT, MRI) (nodes, liver, and spleen): Normal Peripheral blood by flow cytometry: No clonal lymphocytes Bone marrow by morphology: No nodules; or if present, nodules are free from CLL cells by immunohistochemistry Duration: ≥2 months~CR with minimal residual disease Peripheral blood or bone marrow by flow cytometry: >0 - <1 CLL cells/1000 leukocytes (0.1%)~Partial Remission (PR):~Both criteria:~Absolute lymphocyte count in peripheral blood: ≥50% decrease Physical exam/Imaging studies (nodes, liver, and/or spleen): ≥50% decrease Duration: ≥2 months" (NCT00104858)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy and Rituximab Followed by HCT)35

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Overall Survival

Number of participants surviving post-transplant (NCT00104858)
Timeframe: At 18 months

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy and Rituximab Followed by HCT)34

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Donor and Host Polymorphisms of the FCgammaRIIIa Receptor and CD32 and Their Impact on Disease Response and Relapse

Number of participants without progressive disease and surviving at one year. Participants with FCgammaRIIIa receptor vs participants without FCgammaRIIIa receptor. (NCT00104858)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Surviving participants w/ FCgammaRIIIa receptorSurviving participants w/o FCgammaRIIIa receptorw/ FCgammaRIIIa receptor w/o progressive diseasew/o FCgammaRIIIa receptor w/o progressive disease
Treatment (Chemotherapy and Rituximab Followed by HCT)221219

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Rituxan Concentration

Median rituxan level at days 60, 84, 180, and 1 year. (NCT00104858)
Timeframe: Days 60, 84, 180, and 1 year

Interventionug/ml (Median)
Day 60Day 84Day 1801 year
Treatment (Chemotherapy and Rituximab Followed by HCT)109511.3.03

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Disease Progression/Relapse

"CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever greater than 38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~AML, ALL >5% marrow blasts by morphologic or flow cytometric, or appearance of extramedullary disease.~CLL ≥1 of: Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or number of plasmacytomas or lytic bone lesions." (NCT00118352)
Timeframe: Up to 5 years

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)25

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Incidence of Grade III-IV Acute GVHD

"Severity of Individual Organ Involvement~Liver:~Stage 2 - bilirubin (3-5.9mg/100ml) Stage 3 - bilirubin (6-14.9mg/100ml) Stage 4 - bilirubin > 15mg/100ml~Gut:~Diarrhea is graded stage 1 to stage 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as stage 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall~Severity of GVHD~Grade III - Stage 2 to 4 gastrointestinal involvement and/or Stage 2 to 4 liver involvement with or without a rash Grade IV - Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00118352)
Timeframe: 100 days after transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)25

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Incidence of Graft Rejection

Percentage patients that experienced graft rejection. (NCT00118352)
Timeframe: 84 days after transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)0

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Incidence of High-dose Corticosteroid Utilization.

Percentage patients requiring steroids greater than 1 mg/kg. (NCT00118352)
Timeframe: 100 days after transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)83.3

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Incidence of Infection

Percentage patients that experienced infection(s). (NCT00118352)
Timeframe: Up to 5 years post-transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)100

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Incidence of Non-relapse Mortality

Percentage patient deaths due to non-relapse mortality (NCT00118352)
Timeframe: 100 days after transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)8.3

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Change From Baseline in Glomerular Filtration Rate (GFR) at 12 Months Posttransplant

Mean percent change from baseline in estimated glomerular filtration rate (GFR) calculated by modification of diet in renal disease (MDRD)-6 variable equation at 12 months posttransplantation. MDRD-6 variables: serum creatinine, albumin and urea nitrogen, gender, age and ethnicity. (NCT00118742)
Timeframe: 12 months posttransplant

InterventionPercent change in GFR (mL/min) (Mean)
CellCept + CNI (Tacrolimus or Cyclosporine)1.2
CellCept + Sirolimus19.7

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Change From Baseline in Glomerular Filtration Rate (GFR) at 24 Months Posttransplant

Mean percent change from baseline in estimated GFR calculated by modification of diet in renal disease (MDRD)-6 variable equation at 6 and 24 months posttransplantation. MDRD-6 variables: serum creatinine, albumin and urea nitrogen, gender, age and ethnicity. (NCT00118742)
Timeframe: 24 months posttransplant

InterventionMean percent change in GFR (mL/min) (Mean)
CellCept + CNI (Tacrolimus or Cyclosporine)-8.6
CellCept + Sirolimus13.5

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Change From Baseline in Glomerular Filtration Rate (GFR) at 6 Months Posttransplant

Mean percent change from baseline in estimated GFR calculated by modification of diet in renal disease (MDRD)-6 variable equation at 6 and 24 months posttransplantation. MDRD-6 variables: serum creatinine, albumin and urea nitrogen, gender, age and ethnicity. (NCT00118742)
Timeframe: 6 months posttransplant

InterventionPercent change in GFR (mL/min) (Mean)
CellCept + CNI (Tacrolimus or Cyclosporine)1.1
CellCept + Sirolimus25.5

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Change From Baseline in Creatinine Clearance

Mean percent change from baseline in calculated creatinine clearance (CL) at 6, 12, and 24 months posttransplantation (NCT00118742)
Timeframe: 6, 12, and 24 months posttransplantation

,
InterventionPercent change in creatinine CL (mL/min) (Mean)
6 months12 months24 months
CellCept + CNI (Tacrolimus or Cyclosporine)-1.3-3.0-12.8
CellCept + Sirolimus18.514.07.9

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Number of Donors Discontinuing Atorvastatin Due to Toxicity

"The number of donors who prematurely discontinue atorvastatin therapy due to toxicity. Donors will be assessed for the following events:~Musculoskeletal and connective tissue disorders: grade 2-5~Hepatobiliary disorders: grade 2-5~Other unexpected events thought related to the use of atorvastatin; grade 2-5~In cases where the NCI criteria do not apply, intensity will be defined as:~Mild: awareness of symptom or sign, but easily tolerated~Moderate: discomfort is enough to cause interference with normal activities~Severe: inability to perform normal daily activities~Life threatening: immediate risk of death from the reaction as it occurred" (NCT01527045)
Timeframe: Prior to stem cell collection

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)2
Primary - Reg B (TBI Alone)0
Adjunct1

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Number of Non-relapse Mortalities

Number of patients who died without relapsed/progressive disease. (NCT01527045)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)4
Primary - Reg B (TBI Alone)1
Adjunct0

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Number of Patients Requiring Secondary Systemic Immunosuppressive Therapy

Number of patients requiring systemic immunosuppressive therapy other than those used for prophylaxis and initial therapy. (NCT01527045)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)23
Primary - Reg B (TBI Alone)3
Adjunct4

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Number of Patients Surviving Overall

Number of patients surviving overall post-transplant (NCT01527045)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)21
Primary - Reg B (TBI Alone)3
Adjunct9

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Number of Patients With Chronic Extensive GVHD

Number of patients who developed chronic extensive GVHD post-transplant. The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD. Patients were evaluated as described in the National Institutes of Health (NIH) consensus project guidelines. (NCT01527045)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)8
Primary - Reg B (TBI Alone)1
Adjunct5

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Number of Patients With Grade III-IV Acute Graft-versus-host Disease (GVHD) Post-transplant

"Number of patients who developed acute GVHD post allogeneic transplant.~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT01527045)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)2
Primary - Reg B (TBI Alone)0
Adjunct0

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Number of Patients With Grades II-IV Acute Graft-versus-host-disease (GVHD)

"Number of patients who developed acute GVHD post allogeneic transplant.~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT01527045)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)15
Primary - Reg B (TBI Alone)2
Adjunct6

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Number of Patients With Recurrent or Progressive Malignancy

"CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever greater than 38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~AML, ALL, MDS >5% marrow blasts by morphologic or flow cytometric, or appearance of extramedullary disease.~CLL ≥1 of: Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or number of plasmacytomas or lytic bone lesions." (NCT01527045)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Primary - Reg A (Flu/TBI)7
Primary - Reg B (TBI Alone)4
Adjunct3

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Bronchiolitis Obliterans

Development of bronchiolitis obliterans during treatment (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil5
Placebo4

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Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy

Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy (NCT00089141)
Timeframe: 2 years

Interventionparticipants (Number)
Mycophenolate Mofetil11
Placebo10

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Death

Death from any cause after enrollment in the study (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil19
Placebo10

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Death or Recurrent Malignancy

Death due to any cause or development of recurrent malignancy at any time after enrollment (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil25
Placebo15

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Definitive Absence of Efficacy Success

Administration of secondary systemic therapy for chronic GVHD, death during primary therapy, or onset of recurrent malignancy or bronchiolitis obliterans during primary therapy (NCT00089141)
Timeframe: 2 years

Interventionparticipants (Number)
Mycophenolate Mofetil45
Placebo40

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End of Systemic Treatment

Withdrawal of all immunosuppressive treatment without recurrent malignancy (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil15
Placebo15

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Non-relapse Mortality

Death without prior development of recurrent malignancy (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil8
Placebo5

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Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy

Administration of any systemic therapy other than the immunosuppressive agents used for initial treatment, because of persistent or progressive chronic graft-versus-host disease (NCT00089141)
Timeframe: 2 years

Interventionparticipants (Number)
Mycophenolate Mofetil24
Placebo25

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Recurrent Malignancy

Development of recurrent malignancy after enrollment in the study (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil17
Placebo10

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Withdrawal of Prednisone

Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD (NCT00089141)
Timeframe: within 4 years

Interventionparticipants (Number)
Mycophenolate Mofetil30
Placebo33

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Incidence of Chronic Extensive GVHD

Number of patients who developed chronic extensive GVHD post-transplant. The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD. (NCT00089011)
Timeframe: Day 180 post-transplantation

InterventionParticipants (Count of Participants)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)15
Arm II (Nonmyeloablative Conditioning With TBI)6

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Incidence of Grade III/IV GVHD

"Number of patients who developed acute/chronic GVHD post-transplant. aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00089011)
Timeframe: Day 180 post-transplantation

InterventionParticipants (Count of Participants)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)2
Arm II (Nonmyeloablative Conditioning With TBI)4

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Incidences of Grades II-IV Acute GVHD

"Number of patients who developed acute/chronic GVHD post-transplant. aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 3 skin and/or stage 1 gut involvement and/or stage 1 liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00089011)
Timeframe: Day 180 post-transplantation

InterventionParticipants (Count of Participants)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)26
Arm II (Nonmyeloablative Conditioning With TBI)14

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Incidences of Graft Rejection

Number of patients who rejected their graft. Rejection is defined as the inability to detect or loss of detection of greater than 5% donor T cells (CD3+) as a proportion of the total T cell population, respectively, after nonmyeloablative HCT. (NCT00089011)
Timeframe: Day 180 post-transplantation

InterventionParticipants (Count of Participants)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)0
Arm II (Nonmyeloablative Conditioning With TBI)0

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Overall Survival

Number of patients surviving post-transplant. (NCT00089011)
Timeframe: At 1 year after conditioning

InterventionParticipants (Count of Participants)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)85
Arm II (Nonmyeloablative Conditioning With TBI)39

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Rate and Duration of Steroid Use for the Treatment of Chronic GVHD

Number of patients that received prednisone treatment of chronic GVHD, and number of days for which they received prednisone. (NCT00089011)
Timeframe: Up to 5 years

Interventiondays (Median)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)78
Arm II (Nonmyeloablative Conditioning With TBI)89

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Rates of Disease Progression

"Relapse/Progression criteria:~CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever >38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~CMML, AML, ALL >30% BM blasts w/ deteriorating performance status, or worsening of anemia, neutropenia, or thrombocytopenia.~CLL ≥1 of: Physical exam/imaging studies ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or number of plasmacytomas or lytic bone lesions." (NCT00089011)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI)41
Arm II (Nonmyeloablative Conditioning With TBI)33

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Number of Non-Relapse Mortalities

"Percentage of NRM as estimated by cumulative incidence methods with competing risks.~Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref) Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198" (NCT00105001)
Timeframe: 200 days after transplant

InterventionParticipants (Count of Participants)
Arm I (MMF and Tacrolimus)3
Arm II (MMF and Tacrolimus Alternate Schedule)6
Arm III (MMF, Tacrolimus, and Sirolimus)2

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Number of Participants Surviving Overall

"Number of patients surviving, estimated by cumulative incidence methods~Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref) Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198" (NCT00105001)
Timeframe: 1 Year post-transplant

InterventionParticipants (Count of Participants)
Arm I (MMF and Tacrolimus)48
Arm II (MMF and Tacrolimus Alternate Schedule)47
Arm III (MMF, Tacrolimus, and Sirolimus)40

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Number of Participants Surviving Without Progression

"Number of patients with progression-free survival, estimated by cumulative incidence methods~Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref) Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198" (NCT00105001)
Timeframe: 2 Years post-transplant

InterventionParticipants (Count of Participants)
Arm I (MMF and Tacrolimus)28
Arm II (MMF and Tacrolimus Alternate Schedule)27
Arm III (MMF, Tacrolimus, and Sirolimus)26

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Number of Participants Utilizing High-Dose Corticosteroids

"Number of patients utilizing high-dose corticosteroids (as a surrogate marker for reduction of acute GVHD), estimated by cumulative incidence methods.~Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref) Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198" (NCT00105001)
Timeframe: 150 days after transplant

InterventionParticipants (Count of Participants)
Arm I (MMF and Tacrolimus)38
Arm II (MMF and Tacrolimus Alternate Schedule)35
Arm III (MMF, Tacrolimus, and Sirolimus)22

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Number of Participants With Grades II-IV Acute GVHD

"Number of patients with grades II-IV acute GVHD~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT00105001)
Timeframe: 150 days after transplant

InterventionParticipants (Count of Participants)
Arm I (MMF and Tacrolimus)44
Arm II (MMF and Tacrolimus Alternate Schedule)34
Arm III (MMF, Tacrolimus, and Sirolimus)32

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Engraftment

Number of subjects who engrafted post-transplant. Engraftment will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor12
Unrelated Donor4

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Incidence of Acute GVHD (Grades III-IV)

Number of patients with Grade III-IV acute GVHD post-transplant. Severe GVHD will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor2
Unrelated Donor0

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Incidence of Chronic (Extensive) GVHD

Number of subjects with chronic extensive GVHD post-transplant. Severe GVHD will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor4
Unrelated Donor1

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Non-relapse Mortality

Early NRM will be monitored in a sequential fashion. (NCT00054353)
Timeframe: At day 100

InterventionParticipants (Count of Participants)
Related Donor1
Unrelated Donor1

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PFS

PFS will be calculated for all patients from the date of transplant until the time of progression, relapse, death, or the date the patient was last known to be in remission. Progressive disease is defined as greater than 25% increase in serum or urine M proteins compared to best response status after autologous transplant and/or appearance of new lytic bone lesions or plasmocytomas. (NCT00054353)
Timeframe: At 1 year post-transplant

InterventionParticipants (Count of Participants)
Related Donor4
Unrelated Donor1

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Relapse Rate

Number of subjects who relapsed after achieving CR post-transplant. Relapse rate will be summarized using cumulative incidence estimates. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor1
Unrelated Donor1

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Response Rate

Number of subjects who achieved CR post-transplant. Response rate will be summarized using cumulative incidence estimates. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor2
Unrelated Donor1

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OS

Number of subjects surviving. OS will be estimated by the method of Kaplan and Meier. Confidence intervals will be estimated. (NCT00054353)
Timeframe: At 6 months and then every year thereafter, up to 5 years

,
InterventionParticipants (Count of Participants)
6 Months1 Year2 Years3 Years4 Years5 Years
Related Donor854333
Unrelated Donor320000

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Disease-free Survival-incidence of Survival Without Relapse

Sufficient evidence will be taken to be an observed rate of DFS at one year after transplant that corresponds to a one-sided 95% confidence interval with an upper limit lower than 35%. (NCT00045435)
Timeframe: By 1 year after transplant

Interventionpercentage of participants (Number)
Treatment (Nonmyeloablative Donor PBSC Transplant)47

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Incidence of Rejection

Percent patients who developed infections post-transplant. (NCT00045435)
Timeframe: By 1 year after transplant

Interventionpercentage of participants (Number)
Treatment (Nonmyeloablative Donor PBSC Transplant)0

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Incidence of Relapse

Percent patients with relapsed disease post-transplant. (NCT00045435)
Timeframe: By 1 year after transplant

Interventionpercentage of participants (Number)
Treatment (Nonmyeloablative Donor PBSC Transplant)41.2

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Nonrelapse Mortality (NRM)-Incidence of Nonrelapse Death

Defined as death without morphologic evidence of disease. Sufficient evidence will be taken to be an observed rate of NRM within 200 days of transplant that corresponds to a one-sided 80% confidence interval with a lower limit greater than 15%. (NCT00045435)
Timeframe: 200 days after transplant

Interventionpercentage of participants (Number)
Treatment (Nonmyeloablative Donor PBSC Transplant)6

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Overall Survival

Percent patients surviving. (NCT00045435)
Timeframe: By 1 year after transplant

Interventionpercentage of participants (Number)
Treatment (Nonmyeloablative Donor PBSC Transplant)70.6

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Incidence of Acute and Chronic GVHD

Percent patients with acute/chronic GVHD (NCT00045435)
Timeframe: aGVHD: 100 days after transplant; cGVHD: 1 Year after transplant.

Interventionpercentage of participants (Number)
Grade II-IV aGVHDcGVHD
Treatment (Nonmyeloablative Donor PBSC Transplant)35.335.3

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Incidence of Grade IV Acute GVHD

"Clinical Stage of acute GVHD according to Organ System~Skin:~- Maculopapular rash <25% of body surface~- Maculopapular rash 25-50% of body surface~- Maculopapular rash >50% body surface area or generalized erythroderma~- Generalized erythroderma with bullous formation and desquamation~Liver:~- Bilirubin 2-3 mg/dl~- Bilirubin 3.1-6 mg/dl~- Bilirubin 6.1-15 mg/dl~- Bilirubin >15 mg/dl~Gut:~- >500-1000 mL diarrhea per day or (nausea, anorexia or vomiting with biopsy (EGD) confirmation of upper GI GVHD~- >1000 -1500 mL diarrhea per day~- >1500 mL diarrhea per day~- >1500 mL diarrhea per day plus severe abdominal pain with or without ileus~Overall Clinical Grading of Severity of acute GVHD Grade IV: 0-4 Skin, 2-4 Liver, and/or 2-4 GI" (NCT00096161)
Timeframe: Within 100 days after the last DLI

Interventionpercentage of participants (Number)
Group 1A (Pentostatin, DLI Dose Level 1)5
Group 1B (Pentostatin, DLI Dose Level 2)0
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS)0

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Incidence of Infections

(NCT00096161)
Timeframe: 100 days after DLI

Interventionpercentage of participants (Number)
Group 1A (Pentostatin, DLI Dose Level 1)80
Group 1B (Pentostatin, DLI Dose Level 2)70
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS)33.3

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Incidence of Relapse/Progression

"CML Acquisition of a new cytogenetic abnormality and/or development of accelerated phase or blast crisis. Criteria for accelerated phase: unexplained fever >38.3° C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, BM blasts and promyelocytes >20%.~AML, ALL, CMML >30% BM blasts w/ deteriorating performance status, or worsening of anemia, neutropenia, or thrombocytopenia.~CLL Progressive disease: ≥1 of: physical exam/imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ tx; or definite increase in the size/number of plasmacytomas or lytic bone lesions." (NCT00096161)
Timeframe: 1 year after DLI

Interventionpercentage of participants (Number)
Group 1A (Pentostatin, DLI Dose Level 1)45
Group 1B (Pentostatin, DLI Dose Level 2)20
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS)33.3

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Percentage Patients With an Increase of at Least 10 Percentage Points in Donor T-cell Chimerism

"A regimen will be considered successful if 20 patients are enrolled, at least 13 demonstrate improved chimerism. If fewer than 5 patients have shown improvement in chimerism then it can be at least 75% confident that the true rate of improvement is less than 0.53. Enrollment to the regimen will stop and the next regimen will be opened. Enrollment to a regimen may also be stopped at any time it becomes impossible to achieve 5 of 10 or 13 of 20 successful improvements.~Chimerism in hematopoietic cell transplant derives from this idea of a mixed entity, referring to someone who has received a transplant of genetically different tissue. A test for chimerism after a hematopoietic cell transplant involves identifying the genetic profiles of the recipient and of the donor and then evaluating the extent of mixture in the recipient's blood cells or marrow cells." (NCT00096161)
Timeframe: From the time of enrollment maintained to day 56 after the last DLI, up to Day 112

Interventionpercentage of participants (Number)
Group 1A (Pentostatin, DLI Dose Level 1)60
Group 1B (Pentostatin, DLI Dose Level 2)30
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS)33.3

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Survival

Percentage patients surviving. (NCT00096161)
Timeframe: 1 year after DLI

Interventionpercentage of participants (Number)
Group 1A (Pentostatin, DLI Dose Level 1)60
Group 1B (Pentostatin, DLI Dose Level 2)90
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS)66.7

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Incidence of GVHD

"Percentage patients with acute or chronic GVHD.~The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD." (NCT00096161)
Timeframe: 1 year after DLI

,,
Interventionpercentage of participants (Number)
aGVHDcGVHD
Group 1A (Pentostatin, DLI Dose Level 1)2050
Group 1B (Pentostatin, DLI Dose Level 2)020
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS)016.7

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Mean International Society for Heart and Lung Transplantation Biopsy Score Over the First 6 Months Post-transplantation

Mean ISHLT biopsy score Biopsies of the heart may be various grades and each is assigned a numerical score. Grade 0 is 0 points, 1A = 1, 1B = 2, grade 2 = 3, grade 3A = 4, Grade 3B = 5, and grade 4 = 6 units. The mean biopsy score is the numeric average of the biopsy scores for the first 6 post-transplant months. Best value is 0, worst score is 6. (NCT00299221)
Timeframe: 6 months

Interventionunits on a scale (Mean)
Monotherapy0.7
Combination Therapy0.65

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Mean ISHLT Biopsy Score Over First Year Post-transplant

Mean ISHLT biopsy score Biopsies of the heart may be various grades and each is assigned a numerical score. Grade 0 is 0 points, 1A = 1, 1B = 2, grade 2 = 3, grade 3A = 4, Grade 3B = 5, and grade 4 = 6 units. The mean biopsy score is the numeric average of the biopsy scores for the first 6 post-transplant months. Best value is 0, worst score is 6. (NCT00299221)
Timeframe: 1 year

Interventionunits on a scale (Mean)
Monotherapy0.67
Combination Therapy0.62

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Number of Patients With Allograft Vasculopathy (CAD) at One Year Post Transplant

Number of patients diagnosed with allograft vasculopathy / coronary artery disease (CAD) at one year post transplant (NCT00299221)
Timeframe: 1 year

Interventionpatients (Number)
Monotherapy0
Combination Therapy0

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Number of Patients With Cytomegalovirus (CMV) at One Year Post-transplant

Number of patients developing cytomegalovirus disease by 1 year post-transplant (NCT00299221)
Timeframe: 1 year

Interventionparticipants (Number)
Monotherapy2
Combination Therapy2

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Percent of Patients Alive at One Year Post-transplant

Percent of patients alive at one year post-transplant. In other words, all cause mortality over time (NCT00299221)
Timeframe: 1 year

Interventionpercent of participants (Number)
Monotherapy98
Combination Therapy98

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Best Overall Response (BOR) at Cycle 7 Day 1

BOR was defined as the percentage of participants who achieved an overall response (CR+PR) based on cGvHD disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response at any time point (up to Cycle 7 Day 1 or the start of additional systemic therapy for cGvHD). Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. This analysis was based on the primary cutoff date (May 2020). (NCT03112603)
Timeframe: up to Cycle 7 Day 1 (each cycle was comprised of 4 weeks)

Interventionpercentage of participants (Number)
Ruxolitinib76.4
Best Available Therapy60.4

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BOR During Cross-over Treatment With Ruxolitinib

BOR was defined as the percentage of participants who achieved an overall response (CR+PR) based on cGvHD disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response at any time point (up to Cycle 7 Day 1 or the start of additional systemic therapy for cGvHD). Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. (NCT03112603)
Timeframe: from Crossover Cycle 1 Day 1 to any time point up to and including Crossover Cycle 7 Day 1 (each cycle was comprised of 4 weeks)

Interventionpercentage of participants (Number)
Ruxolitinib Cross-Over Period81.4

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Duration of Response Through Study Completion

DOR was defined as the time from first response until cGvHD progression, death, or the date of change/addition of systemic therapies for cGvHD and as assessed for responders only. Response was based on cGvHD disease assessments (National Institutes of Health consensus criteria). Duration of response was evaluated in participants who achieved a CR or PR at or before Cycle 7 Day 1. The analysis included a larger number of participants than the number of participants who achieved CR or PR at Cycle 7 Day 1 (82 ruxolitinib and 42 BAT) because the analysis took into account not only those participants who achieved CR or PR at Cycle 7 Day 1, but also participants who achieved CR or PR before Cycle 7 Day 1 but who may have lost their response at Cycle 7 Day 1. For this reason, the number of participants in this analysis does not align with the best overall response (BOR) at Cycle 7 Day 1. This analysis was based on the cutoff date upon study completion (December 2022). (NCT03112603)
Timeframe: from first response to LPLV (approximately 5 years)

InterventionMonths (Median)
RuxolitinibNA
Best Available Therapy6.4

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Efficacy of Ruxolitinib Versus Investigator's Choice Best Available Therapy (BAT) in Participants With Moderate or Severe Steroid Refractory Chronic Graft Versus Host Disease (SR-cGvHD) Assessed by Overall Response Rate (ORR) at the Cycle 7 Day 1 Visit

ORR was defined as the percentage of participants in each arm demonstrating a complete response (CR) or partial response (PR) based on chronic GvHD (cGvHD) disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response. Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. (NCT03112603)
Timeframe: Cycle 7 Day 1 (each cycle was comprised of 4 weeks)

Interventionpercentage of participants (Number)
Ruxolitinib49.7
Best Available Therapy25.6

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Number of Participants With Any Treatment-emergent Adverse Event (TEAE)

Adverse events were defined as the appearance of (or worsening of any pre-existing) undesirable signs, symptoms, or medical conditions that occurred after the participant's signed informed consent was obtained. Abnormal laboratory values or test results occurring after informed consent constituted adverse events only if they induced clinical signs or symptoms, were considered clinically significant, required therapy (e.g., hematologic abnormality that required transfusion or hematological stem cell support), or required changes in study medication(s). TEAEs were defined as those AEs that started or worsened during the on-treatment period (either randomized or cross-over period). (NCT03112603)
Timeframe: from Baseline to LPLV (approximately 5 years)

InterventionParticipants (Count of Participants)
Ruxolitinib165
Best Available Therapy148
Ruxolitinib Cross-Over Period70

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ORR at the End of Cycle 3

ORR was defined as the percentage of participants in each arm demonstrating a CR or PR based on cGvHD disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response. Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. (NCT03112603)
Timeframe: Cycle 4 Day 1 (each cycle was comprised of 4 weeks)

Interventionpercentage of participants (Number)
Ruxolitinib54.5
Best Available Therapy31.1

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Overall Survival (OS)

Overall survival was defined as the time from the date of randomization to the date of death due to any cause. (NCT03112603)
Timeframe: from the date of randomization to the date of death due to any cause up to LPLV (approximately 5 years)

Interventionmonths (Median)
RuxolitinibNA
Best Available TherapyNA

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Rate of Failure-free Survival (FFS)

Composite time to event endpoint incorporating the following FFS events: (i) relapse or recurrence of underlying disease or death due to underlying disease, (ii) nonrelapse mortality, or (iii) addition or initiation of another systemic therapy for cGvHD. (NCT03112603)
Timeframe: Baseline to when the last participant reached Cycle 7 Day 1 (each cycle was comprised of 4 weeks)

Interventionmonths (Median)
RuxolitinibNA
Best Available Therapy5.7

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Rate of FFS at Study Completion

Composite time to event endpoint incorporating the following FFS events: (i) relapse or recurrence of underlying disease or death due to underlying disease, (ii) nonrelapse mortality, or (iii) addition or initiation of another systemic therapy for cGvHD. (NCT03112603)
Timeframe: From Baseline to Last Participant Last Visit (LPLV) (approximately 5 years)

Interventionmonths (Median)
Ruxolitinib38.4
Best Available Therapy5.7

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Rate of Participants With Clinically Relevant Improvement of the Modified Lee cGvHD Symptom Scale Score

"To assess improvement of symptoms based on the total symptom score (TSS); a responder was defined as having achieved a clinically relevant reduction from Baseline of the TSS. The scale consists of 30 items in 7 subscales (skin, eye, mouth, lung, nutrition, energy, and psychological). Participants reported their level of symptom bother over the previous month on a 5-point likert scale: not at all, slightly, moderately, quite a bit, or extremely. Subscale scores and the summary score range from 0 to 100, with a higher score indicating worse symptoms." (NCT03112603)
Timeframe: Baseline; Cycle 7 Day 1 (each cycle was comprised of 4 weeks)

Interventionpercentage of participants (Number)
Ruxolitinib24.2
Best Available Therapy11.0

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Utilization of Medical Resources

The percentage of participants with at least one submission to healthcare encounter was assessed. (NCT03112603)
Timeframe: from Baseline to LPLV (approximately 5 years)

Interventionpercentage of participants (Number)
Ruxolitinib57.0
Best Available Therapy65.8

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AUCinf of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"AUCinf was defined as the area under the concentration-time curve from time 0 to infinity. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

Interventionng*hour/mL (Geometric Mean)
Day 1
Ruxolitinib642

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AUClast of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"AUClast was defined as the area under the concentration-time curve up to the last measurable concentration of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

Interventionng*hour/mL (Geometric Mean)
Day 1Day 15
Ruxolitinib636945

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Change From Baseline in EQ-5D-5L

The EQ-5D-5L is a descriptive classification consisting of five dimensions of health: mobility, self-care, usual activities, anxiety/depression, and pain/discomfort. The five-level version (no problems, slight problems, moderate problems, severe problems, and extreme problems) uses a 5-point Likert scale, with 1 being no problems and 5 being extreme problems. (NCT03112603)
Timeframe: Baseline; up to Cycle 39 Day 1 (each cycle was comprised of 4 weeks)

,
Interventionscores on a scale (Mean)
Cycle 2 Day 1Cycle 3 Day 1Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 9 Day 1Cycle 12 Day 1Cycle 15 Day 1Cycle 18 Day 1Cycle 21 Day 1Cycle 24 Day 1Cycle 27 Day 1Cycle 30 Day 1Cycle 33 Day 1Cycle 36 Day 1Cycle 39 Day 1
Best Available Therapy-0.01-0.04-0.01-0.030.01-0.00-0.020.020.030.02-0.000.010.030.010.050.040.01
Ruxolitinib0.030.030.040.020.050.070.070.070.070.070.080.070.060.050.000.060.06

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Change From Baseline in Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT)

"Change from Baseline was calculated as the post-Baseline value minus the Baseline value. The FACT-BMT is a 50-item self-report questionnaire that measures the effect of a therapy on domains including physical, functional, social/family, and emotional well-being, together with additional concerns relevant for bone marrow transplantation participants. The questions were based on a 5-point Likert scale, where 0 corresponds to not at all and 4 corresponds to very much. The higher the final score, the better the quality of life. The FACT-BMT total score ranges from 0 to 148." (NCT03112603)
Timeframe: Baseline; up to Cycle 39 Day 1 (each cycle was comprised of 4 weeks)

,
Interventionscores on a scale (Mean)
Cycle 2 Day 1Cycle 3 Day 1Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 9 Day 1Cycle 12 Day 1Cycle 15 Day 1Cycle 18 Day 1Cycle 21 Day 1Cycle 24 Day 1Cycle 27 Day 1Cycle 30 Day 1Cycle 33 Day 1Cycle 36 Day 1Cycle 39 Day 1
Best Available Therapy-0.22-1.82-1.05-0.232.410.851.203.747.588.193.687.845.105.755.674.776.64
Ruxolitinib2.320.621.981.252.914.145.327.265.074.105.245.906.237.535.178.728.65

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CL/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"CL/F was defined as the oral dose clearance of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

InterventionLiters/hour (Geometric Mean)
Day 1Day 15
Ruxolitinib15.615.2

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Cmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"Cmax was defined as the maximum observed plasma concentration of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

Interventionnanograms per milliliter (ng/mL) (Geometric Mean)
Day 1Day 15
Ruxolitinib167215

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Cumulative Incidence of Malignancy Relapse/Recurrence (MR)

Defined as the cumulative incidence rate from competing risk analysis of MR from the date of randomization to hematologic malignancy relapse/recurrence. (NCT03112603)
Timeframe: Months 3, 6, 12, 18, 24, 30, and 36

,
Interventionpercentage of participants (Number)
0 to < 3 months3 to < 6 months6 to < 12 months12 to < 18 months18 to < 24 months24 to <30 months30 to <36 months
Best Available Therapy1.312.656.086.086.086.787.50
Ruxolitinib1.923.225.187.828.488.488.48

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Cumulative Incidence of Non-relapse Mortality (NRM)

Defined as the cumulative incidence rate from competing risk analysis for NRM from the date of randomization to the date of death not preceded by underlying disease relapse/recurrence. (NCT03112603)
Timeframe: Months 3, 6, 12, 18, 24, 30, and 36

,
Interventionpercentage of participants (Number)
Month 3Month 6Month 12Month 18Month 24Month 30Month 36
Best Available Therapy4.446.4315.1216.4819.2219.2222.0
Ruxolitinib5.459.1315.3015.9317.8317.8317.83

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Percentage of Participants Successfully Tapered Off of All Corticosteroids

All corticosteroid dosages prescribed to the participant and all dose changes during the study were to be recorded for assessment of participants who successfully tapered off of all corticosteroids. Participants who completely tapered off corticosteroids refer to those who permanently discontinued steroids as per the dose administration panel and who did not restart steroids in the same interval. Participants who were tapered off and continued follow-up were also counted as being tapered off with 0 dose in subsequent intervals until they discontinued from the main treatment period or restarted steroid treatment. (NCT03112603)
Timeframe: up to Day 179

,
Interventionpercentage of participants (Number)
Day 1 to ≤ Day 28Day 29 to ≤ Day 56Day 57 to ≤ Day 84Day 85 to ≤ Day 112Day 113 to ≤ Day 140Day 141 to ≤ Day 168Day 169 to ≤ Day 179
Best Available Therapy2.65.48.510.312.416.815.9
Ruxolitinib2.59.614.016.319.724.224.1

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Percentage of Participants With a ≥ 50% Reduction in Daily Corticosteroid Dose

All corticosteroid dosages prescribed to the participant and all dose changes during the study were to be recorded for assessment of participants with a ≥ 50% reduction in daily corticosteroid dose. (NCT03112603)
Timeframe: from Day 15 up to Day 182

,
Interventionpercentage of participants (Number)
Day 15 to ≤ Day 28Day 29 to ≤ Day 42Day 43 to ≤ Day 56Day 57 to ≤ Day 70Day 71 to ≤ Day 84Day 85 to ≤ Day 98Day 99 to ≤ Day 112Day 113 to ≤ Day 126Day 127 to ≤ Day 140Day 141 to ≤ Day 154Day 155 to ≤ Day 168Day 169 to ≤ Day 182
Best Available Therapy13.233.141.147.951.454.060.466.268.368.371.688.8
Ruxolitinib12.735.048.458.762.369.571.273.272.870.074.681.9

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t1/2 of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"t1/2 was defined as the apparent terminal phase disposition half-life of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

Interventionhours (Geometric Mean)
Day 1Day 15
Ruxolitinib2.402.32

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Tmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"tmax was defined as the time to reach the maximum plasma concentration of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

Interventionhours (Median)
Day 1Day 15
Ruxolitinib0.8331.00

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Vz/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses

"Vz/F was defined as the apparent oral dose volume of distribution of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose

InterventionLiters (Geometric Mean)
Day 1Day 15
Ruxolitinib54.050.9

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Area Under the Plasma Concentration Time Curve From 0-12 Hours for Mycophenolic Acid and Mycophenolic Acid Glucuronide Phenolic Glucuronide of Mycophenolic Acid

The area under the plasma concentration-time curve from time zero to twelve hours (AUC [0-12h]) is area under the plasma concentration-time curve from time zero through 12 hours. AUC (0-12) hours was computed using the linear trapezoidal rule. For the calculations of AUC (0-12h), concentrations below the limit of quantification were assigned a value of zero if they occurred at the beginning of a profile. When such values appeared at the end of a profile they were assigned as missing data. AUC was reported in microgram hour per milliliter (mcg*h/mL). (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants greater than (>) 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants less than (<) 24 months

Interventionmcg*h/mL (Mean)
MPA RawMPAG RawMPAG (MPA Equivalents)
Drug MMF29487289

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Area Under the Plasma Concentration-Time Curve From 0 to 12 Hours of Mycophenolic Acid Normalized for Dose And for Body Surface Area

The area under the plasma concentration-time curve from time zero to twelve hours (AUC [0-12h]) is area under the plasma concentration-time curve from time zero through 12 hours. AUC (0-12) hours was computed using the linear trapezoidal rule. For the calculations of AUC (0-12h), concentrations below the limit of quantification were assigned a value of zero if they occurred at the beginning of a profile. When such values appeared at the end of a profile they were assigned as missing data. AUC0-12h was normalized to 600 milligram per square meter (mg/m^2) and 1.5 gram. AUC was reported in microgram hour per milliliter (mcg*h/mL). (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants greater than (>) 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants less than (<) 24 months

Interventionmcg*h/mL (Mean)
Normalized to 600mg/m^2Normalized to 1.5g
Drug MMF65.6363

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Maximum Plasma Concentration for Mycophenolic Acid and Mycophenolic Acid Glucuronide

The Plasma Concentration (Cmax) is defined as maximum observed analyte concentration. Cmax was obtained directly from the measured plasma concentration-time curves. (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants > 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants < 24 months

Interventionmcg/mL (Mean)
MPA RawMPAG RawMPAG (MPA Equivalents)
Drug MMF7.9854.632.4

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Number of Participants With Adverse Events and Serious Adverse Events

An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is defined as any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is a significant medical event. (NCT02630563)
Timeframe: Up to Day 32

Interventionparticipants (Number)
Participants with AEParticipants with SAE
Drug MMF10

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Plasma Concentration of Mycophenolic Acid and Its Metabolite Mycophenolic Acid Glucuronide at Each Time Point

Mycophenolic Acid Glucuronide (MPAG) is an active metabolite of Mycophenolic Acid (MPA). (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants greater than (>) 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants less than (<) 24 months

Interventionmcg/mL (Mean)
Raw MPA at 0.0Raw MPA at 0.50Raw MPA at 0.75Raw MPA at 1.00Raw MPA at 1.50Raw MPA at 2.00Raw MPA at 4.00Raw MPA at 8.00Raw MPA at 12.00Raw MPAG at 0.00Raw MPAG at 0.50Raw MPAG at 0.75Raw MPAG at 1.00Raw MPAG at 1.50Raw MPAG at 2.00Raw MPAG at 4.00Raw MPAG at 8.00Raw MPAG at 12.00
Drug MMF0.7869.035.675.846.166.231.891.380.67626.226.545.837.243.251.046.319.214.6

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Time to Maximum Plasma Concentration for Mycophenolic Acid and Mycophenolic Acid Glucuronide

Tmax is the amount of time after dosing to when the maximum concentration of MPA and MPAG was achieved. (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants > 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants < 24 months

Interventionhour (Median)
MPA RawMPAG Raw
Drug MMF1.351.99

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Acute Graft Versus Host Disease (aGVHD) of Grades 2-4 According to the Consensus Grading

Acute graft versus host disease is graded according to the 1994 Keystone Consensus Grading. aGVHD grade was evaluated from day 0 through 100 days post-transplant. The first day of acute GVHD onset at grades 2-4 was used to calculate the cumulative incidence. Relapse/death prior to onset was considered competing events. (NCT03128359)
Timeframe: Up to 100 days post-stem cell infusion

Interventionpercentage of probability (Number)
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis)47
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis)53

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Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS) at 1 Year

Estimates will be calculated using the Kaplan-Meier method, Greenwood formula will be used to calculate standard error (SE), and log-log transformation method will be used to construct 95% confidence intervals. Graft versus host disease (GVHD, acute and chronic), disease status and vital status will be monitored per clinical standard operating procedure. For this endpoint failure is defined as the first occurrence of grade 3 or 4 acute GVHD, or moderate/severe chronic GVHD, or disease relapse (for patients in complete remission at the start of conditioning) or disease progression (for patients with active disease at the start of conditioning) or death (from any cause). Patients not experiencing any of these will be censored at his/her date of last contact. (NCT03128359)
Timeframe: From stem cell infusion to grade 3-4 acute graft versus host disease (GVHD), moderate-severe chronic GVHD, relapse, progression or death (from any cause), whichever occurs first, assessed for up to 1 year.

Interventionpercentage of survival probability (Number)
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis)53
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis)84

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Overall Survival (OS) at 1 Year

Estimates was calculated using the Kaplan-Meier method, Greenwood formula was used to calculate SE, and log-log transformation method was used to construct 95% confidence intervals. Each patient's vital status was monitored per clinical standard operating procedure. For this endpoint failure is defined as death (from any cause). Patients not experiencing a death event was censored at his/her date of last contact. (NCT03128359)
Timeframe: From start of transplant to death, or last follow up, whichever occurs first, assessed for up to 1 year.

Interventionpercentage of probability (Number)
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis)68
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis)100

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Measured Glomerular Filtration Rate

Progression of renal function measured by mean mGFR at 36 months after renal TX. The mGFR was measured using Iohexol or Cr-EDTA clearance according to local practice. (NCT00634920)
Timeframe: Month 36

InterventionmL/min/1.73m^2 (Mean)
Everolimus (CNI-free)48.2
Control (CsA)46.1

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Measured Glomerular Filtration Rate

To compare the efficacy between treatment regimens by assessing the difference in renal function evaluated by mean measured glomerular filtration rate (mGFR) 12 months after renal transplantation (TX). The mGFR was measured using Iohexol or Cr-EDTA clearance according to local practice. (NCT00634920)
Timeframe: Month 12

InterventionmL/min/1.73m^2 (Mean)
Everolimus (CNI-free)51.5
Control (CsA)47.8

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Time to First Malignancy

This is the time to first diagnosed malignancy. Malignancies (skin- or solid cancer) were listed whether they reoccurred in situ, were metastatic or de novo. This is shown as mean time. (NCT00634920)
Timeframe: Months 12, 24, 36

InterventionMonths (Mean)
Everolimus (CNI-free)35.5
Control (CsA)35.1

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Time to Treatment Failure

Treatment failure was defined as graft loss or death.Time to treatment failure is shown as mean time to treatment failure. (NCT00634920)
Timeframe: Months 12, 24, 36

InterventionDays (Mean)
Everolimus (CNI-free)972.7
Control (CsA)959.5

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Calculated Glomerular Filtration Rate

The GFR was calculated according to the Modification of Diet in Renal Disease Study Group (MDRD) method, the Cockcroft-Gault method, and the Nankivell formula. cGFR was calculated from blood samples collected at predefined time points. (NCT00634920)
Timeframe: Months 12, 36

,
InterventionmL/min/1.73m^2 (Mean)
MDRD M12MDRD M36Cockcroft-Gault M12Cockcroft-Gault M36Nankivel M12Nankivel M36
Control (CsA)60.157.445.642.161.858.9
Everolimus (CNI-free)65.059.445.443.166.361.8

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Lipid Profile for Apolipoprotein

Blood lipid levels of patients in both groups for Apolipoprotein (Apo) A1 and B. (NCT00634920)
Timeframe: Months 12, 24, 36

,
Interventiong/L (Mean)
Month 12: Apolipoprotein A1Month 24: Apolipoprotein A1Month 36: Apolipoprotein A1Month 12: Apolipoprotein BMonth 24: Apolipoprotein B (Month 36: Apolipoprotein B
Control (CsA)1.461.361.560.9231.0580.934
Everolimus (CNI-free)1.591.551.700.9351.1780.984

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Lipid Profile for HDL-C, LDL-C,Total Cholesterol, and Triglycerides

Blood lipid levels of patients in both groups: HDL-C, LDL-C,Total cholesterol, and triglycerides. (NCT00634920)
Timeframe: Months 12, 24, 36

,
Interventionmmol/L (Mean)
Month 12: HDL CholesterolMonth 24: HDL CholesterolMonth 36: HDL CholesterolMonth 12: LDL CholesterolMonth 24: LDL CholesterolMonth 36: LDL CholesterolMonth 12: Total CholesterolMonth 24: Total CholesterolMonth 36: Total CholesterolMonth 12: TriglyceridesMonth 24: TriglyceridesMonth 36: Triglycerides
Control (CsA)1.4191.4091.5293.1302.9252.8225.3185.1124.8301.8681.7571.580
Everolimus (CNI-free)1.4861.4771.4953.5693.3813.2066.0915.8235.5952.4612.2882.164

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Number of Antihypertensive Drugs Taken

(NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionNumber of antihypertensive dugs (Mean)
Month 12Month 24Month 36
Control (CsA)2.52.42.2
Everolimus (CNI-free)2.52.52.0

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Number of Lipid-lowering Drugs Taken

(NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionNumber of lipid-lowering drugs (Mean)
Month 12Month 24Month 36
Control (CsA)0.80.90.8
Everolimus (CNI-free)0.91.00.9

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Percentage of Participants on Antihypertensive Drugs

(NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionPercentage of participants (Number)
Month 12: No antihypertensive drugsMonth 12: Has antihypertensive drugsMonth 24: No antihypertensive drugsMonth 24: Has antihypertensive drugsMonth 36: No antihypertensive drugsMonth 36: Has antihypertensive drugs
Control (CsA)3.396.75.394.712.887.2
Everolimus (CNI-free)9.290.84.295.815.684.4

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Percentage of Participants on Lipid-lowering Drugs

(NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionPercentage of participants (Number)
Month 12Month 24Month 36
Control (CsA)60.065.063.0
Everolimus (CNI-free)75.078.073.0

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Percentage of Participants Who Developed CAN (Chronic Allograft Nephropathy)

Assessed by protocol biopsies findings (Banff 1997 lesion scores and morphometry of the interstitial space) (NCT00634920)
Timeframe: Month 12, Month 36

,
InterventionPercentage of participants (Number)
Month 12Month 36
Control (CsA)1.064.0
Everolimus (CNI-free)1.059.0

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Percentage of Participants Who Had Donor Specific Antibodies (DSA)

Venous blood was drawn for donor specific (DSA) measurements prior to transplantation and at the final visit (36 months). The blood sample was first screened for the presence of PRA i.e. donor specific Immunoglobulin-G antibodies against specific HLA antigens. If PRA antibodies were detected, the blood sample was tested for specific DSAs on single antigen Luminex beads (coated with single HLA class I or II molecules). In this way, the specificity of these antibodies could be determined. (NCT00634920)
Timeframe: Month 36

,
InterventionPercentage of participants (Number)
ND (not done)NegativePositive
Control (CsA)9.070.021.0
Everolimus (CNI-free)7.078.015.0

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Percentage of Participants With Biopsy Proven Acute Rejection (BPAR)

A BPAR was defined as a biopsy graded IA, IB, IIA, IIB, or III (Banff 97 classification). Biopsy graded IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells). Biopsy grade IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells). Biopsy grade IIA: Mild to moderate intimal arteritis. Biopsy graded IIB: Severe intimal arteritis comprising > 25% of the lumenal area. (NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionPercentage of participants (Number)
Month 12: lAMonth 12: lBMonth 12: llAMonth 12: llBMonth 24: lAMonth 24: lBMonth 36: lAMonth 36: lB
Control (CsA)4.40.02.21.14.43.31.10.0
Everolimus (CNI-free)19.610.92.22.25.41.12.21.1

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Percentage of Participants With Graft Loss or Death

The allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, the day of nephrectomy was the day of graft loss. Graft loss was considered an SAE (serious adverse event). (NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionPercentage of participants (Number)
Month 12: Event First YearMonth 12: No Event First YearMonth 24: Event Second YearMonth 24: No Event Second YearMonth 36: Event Third YearMonth 36: No Event Third Year
Control (CsA)0.0100.01.198.92.297.8
Everolimus (CNI-free)0.0100.01.198.90.0100.0

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Percentage of Participants With Treatment Failures

Treatment failure was defined as graft loss or death. (NCT00634920)
Timeframe: Months 12, 24, 36

,
InterventionPercentage of participants (Number)
Month 12: No FailureMonth 12: FailureMonth 24: No FailureMonth 24: FailureMonth 36: No FailureMonth 36: Failure
Control (CsA)100.00.098.81.296.73.3
Everolimus (CNI-free)100.00.098.81.298.81.2

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Progression of Measured Glomerular Filtration Rate

Change in renal progression measured by mean mGFR from week 7 to Month 36 (NCT00634920)
Timeframe: Week 7, Week 52, Month 36

,
InterventionmL/min/1.73m^2 (Mean)
Week 7Week 52Change from week 7 to Week 52Month 36Change from week 7 to Month 36
Control (CsA)47.847.80.046.1-1.7
Everolimus (CNI-free)46.351.55.648.21.3

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Proteinuria (Measured as Urine Albumin/Creatinine Ratio (mg/mmol))

"Proteinuria is when a large amount of protein, that should remain circulating in a person's blood, is spilled into their urine and eliminated from the body." (NCT00634920)
Timeframe: Months 12, 24, 36

,
Interventionmg/mmol (Mean)
Month 12Month 24Month 36
Control (CsA)11.2724.5580.73
Everolimus (CNI-free)17.3162.8378.78

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Number of Infectious Complications

The number of infections complications occurring among study participants is presented here. (NCT01921218)
Timeframe: Baseline up to Month 36

Interventioncomplications (Number)
Belatacept Treatment Group12
Control Group18

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Number of Participants With Donor-specific Antibody Formation

The number of participants in each group with donor-specific antibody formation at 36 months following randomization. (NCT01921218)
Timeframe: Month 36

InterventionParticipants (Count of Participants)
Belatacept Treatment Group3
Control Group4

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Time to Initiation of Dialysis

Time to dialysis is measured as the time of randomization to initiation of dialysis. Participants already requiring dialysis at the time of enrollment were excluded from this endpoint analysis. (NCT01921218)
Timeframe: Up to Year 2

Interventionmonths (Mean)
Belatacept Treatment Group11.75
Control Group10.5

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Glomerular Filtration Rate (GFR)

The glomerular filtration rate (GFR) assesses kidney function. GFR uses values for serum creatinine (SCr) measured in mg/dL, age in years, blood urea nitrogen (BUN) measures in mg/dL, and serum albumin (Alb) measured in g/dL. GFR is calculated as 170 x (SCr/0.95)^(-0.999) x (Age)^(-0.176) x (0.762 if the patient is female) x (1.180 if the patient is black) x (BUN)^(-0.170) x (Alb)^(0.318). A value of 90 or above is considered normal while values between 15 and 29 indicate severely decreased kidney function and values below 15 indicate kidney failure. The GFR in participants who do not require dialysis will be followed for two years. (NCT01921218)
Timeframe: Baseline up to Month 24

InterventionmL/min/1.73 m^2 (Mean)
BaselineMonth 1Month 2Month 3Month 6Month 9Month 12
Control Group14.51110.516.514.513.520

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Glomerular Filtration Rate (GFR)

The glomerular filtration rate (GFR) assesses kidney function. GFR uses values for serum creatinine (SCr) measured in mg/dL, age in years, blood urea nitrogen (BUN) measures in mg/dL, and serum albumin (Alb) measured in g/dL. GFR is calculated as 170 x (SCr/0.95)^(-0.999) x (Age)^(-0.176) x (0.762 if the patient is female) x (1.180 if the patient is black) x (BUN)^(-0.170) x (Alb)^(0.318). A value of 90 or above is considered normal while values between 15 and 29 indicate severely decreased kidney function and values below 15 indicate kidney failure. The GFR in participants who do not require dialysis will be followed for two years. (NCT01921218)
Timeframe: Baseline up to Month 24

InterventionmL/min/1.73 m^2 (Mean)
BaselineMonth 1Month 2Month 3Month 6Month 9Month 12Month 18
Belatacept Treatment Group14.2514.2519.3316.3314.6714.6713.677

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Number of Participants With Anti-human Leukocyte Antigen (HLA) Alloantibodies

The presence of anti-HLA Class I and Class II alloantibodies is categorized as being negative (absent for both classes of alloantibodies), positive for Class I, positive for Class II, and positive for both Class I and Class II alloantibodies. (NCT01921218)
Timeframe: Baseline up to Month 36

,
InterventionParticipants (Count of Participants)
Baseline - NegativeBaseline - Positive Class IBaseline - Positive Class IIBaseline - Positive Class I and IIMonth 12 - NegativeMonth 12 - Positive Class IMonth 12 - Positive Class IIMonth 12 - Positive Class I and IIMonth 24 - NegativeMonth 24 - Positive Class IMonth 24 - Positive Class IIMonth 24 - Positive Class I and IIMonth 36 - NegativeMonth 36 - Positive Class IMonth 36 - Positive Class IIMonth 36 - Positive Class I and II
Belatacept Treatment Group6000410010201020
Control Group5200243312111211

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Delta Alanine Aminotransferase

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch9

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Delta Alkaline Phosphatase

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch13.8

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Delta Bilirubin

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch-6.0

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Delta Cholesterol Fasting Level

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch1.2

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Delta Hemoglobin

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch-2.7

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Delta Hepatitis C Viral Load

Percent change in HCV load determined 3 months after switch from MMF to SRL. (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch15

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Delta Platelet Count

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch-8.5

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Delta Tacrolimus Trough Level

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch23

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Delta Triglyceride Fasting Level

Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch23

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Final Hepatitis C Viral Load

Percent change in HCV load determined 3 months after switch from SRL to MMF (NCT01134952)
Timeframe: 3 month

Interventionpercent change (Mean)
MMF SRL Switch-47

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Sirolimus Trough Level

(NCT01134952)
Timeframe: 3 month

Interventionng/ml (Mean)
MMF SRL Switch7.2

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Relapse Free Survival

Number of patients with relapsed disease within 1 Year post-transplant. Relapse is defined as the detection of > 5% blasts after a documented complete remission. (NCT00036738)
Timeframe: Assessed up to 1 year

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Nonmyeloablative HSCT)3

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Leukemia-free Survival

Number of patients surviving in CR up to five years post-transplant. (NCT00036738)
Timeframe: Assessed up to 5 years

InterventionParticipants (Count of Participants)
6 Months1 Year2 Years3 Years4 Years5 Years
Treatment (Allogeneic Nonmyeloablative HSCT)191713111110

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Overall Survival

Number of patients surviving up to five years post-transplant. (NCT00036738)
Timeframe: Assessed up to 5 years

InterventionParticipants (Count of Participants)
6 Months1 Year2 Years3 Years4 Years5 Years
Treatment (Allogeneic Nonmyeloablative HSCT)262419171613

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Number of Participants Who Developed Acute Graft Versus Host Disease

(NCT00255684)
Timeframe: 3 months

Interventionparticipants (Number)
Conditioning Therapy Followed by TBI0

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Number of Participants Who Survived 100 Days or Longer

(NCT00255684)
Timeframe: 100 days

Interventionparticipants (Number)
Conditioning Therapy Followed by TBI13

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Graft Failure Rate

Percentage of participants who experienced failure to engraft (also called graft failure or graft rejection). Failure to engraft is defined as <5% donor chimerism and absence of relapse or any other reason for that chimerism value. All participants who met this criterion were included in this outcome measure. (NCT00134004)
Timeframe: Cumulative incidence for the entire study, up to 11 years

Interventionpercentage of participants (Number)
Mini-haplo BMT13

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Hematologic and Non-hematologic Toxicities as Measured by NCI Common Toxicity Criteria for Adverse Events, v 3.0 Weekly Until 1 Year After Transplantation

Percentage of study participants who experienced a serious adverse event (SAE) within 1 year of bone marrow transplant. Complete data is provided in the Adverse Event tables. (NCT00134004)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Mini-haplo BMT9.5

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Progression-free Survival

Percentage of participants who do not experience disease relapse, disease progression, or death. (NCT00134004)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Mini-haplo BMT34

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Relapse Rate

Percentage of participants who experience disease relapse. (NCT00134004)
Timeframe: Cumulative incidence for the entire study, up to 11 years

Interventionpercentage of participants (Number)
Mini-haplo BMT55

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Number of Participants Experiencing Progression-free Survival

Incidence of Progression-free survival - Number of patients who were alive and did not have disease progression. Patients with leukemia and lymphoma involving the bone marrow (BM) and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT00305682)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant43
Arm 2 - No Prior Autologous Transplant32
Arm 3 - Refractory Leukemia/Lymphoma1
Arm 4: MT2006-01 Coenrolling Patients21
Arm 5 - Previous Autologous Transplant16
Arm 6 - No Prior Autologous Transplant24

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Number of Participants Experiencing Progression-free Survival at 2 Years

Incidence of Progression-free survival - Number of patients who were alive and did not have disease progression (NCT00305682)
Timeframe: 2 Years

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant36
Arm 2 - No Prior Autologous Transplant25
Arm 3 - Refractory Leukemia/Lymphoma1
Arm 4: MT2006-01 Coenrolling Patients17
Arm 5 - Previous Autologous Transplant16
Arm 6 - No Prior Autologous Transplant20

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Number of Participants Experiencing Relapse (Incidence of Relapse)

Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT00305682)
Timeframe: Year 1

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant43
Arm 2 - No Prior Autologous Transplant14
Arm 3 - Refractory Leukemia/Lymphoma4
Arm 4: MT2006-01 Coenrolling Patients7
Arm 5 - Previous Autologous Transplant20
Arm 6 - No Prior Autologous Transplant2

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Number of Participants Experiencing Relapse (Incidence of Relapse) at 2 Years

Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT00305682)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant49
Arm 2 - No Prior Autologous Transplant19
Arm 3 - Refractory Leukemia/Lymphoma4
Arm 4: MT2006-01 Coenrolling Patients11
Arm 5 - Previous Autologous Transplant20
Arm 6 - No Prior Autologous Transplant4

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Number of Participants Who Were Alive at 1 Year Post Transplant

Overall Survival - Number of patients alive at 1 year post transplant (NCT00305682)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant59
Arm 2 - No Prior Autologous Transplant40
Arm 3 - Refractory Leukemia/Lymphoma1
Arm 4: MT2006-01 Coenrolling Patients26
Arm 5 - Previous Autologous Transplant26
Arm 6 - No Prior Autologous Transplant25

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Number of Participants Who Were Alive at 2 Years Post Transplant

Overall Survival - Number of patients alive at 2 years post transplant (NCT00305682)
Timeframe: 2 Years

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant50
Arm 2 - No Prior Autologous Transplant31
Arm 3 - Refractory Leukemia/Lymphoma1
Arm 4: MT2006-01 Coenrolling Patients20
Arm 5 - Previous Autologous Transplant21
Arm 6 - No Prior Autologous Transplant23

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Number of Participants Who Were Dead at 6 Months After Study Completion

Incidence of Non-relapse mortality - Number of Patients Dead at 6 Months after study completion (NCT00305682)
Timeframe: Month 6

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant10
Arm 2 - No Prior Autologous Transplant20
Arm 3 - Refractory Leukemia/Lymphoma2
Arm 4: MT2006-01 Coenrolling Patients5
Arm 5 - Previous Autologous Transplant3
Arm 6 - No Prior Autologous Transplant6

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Number of Participants With Acute Graft-versus-host Disease (GVHD)

"Determine the incidence of grade II-IV and grade III-IV acute graft-versus-host disease (GVHD) at day 100 post transplant. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging.~Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level." (NCT00305682)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant45
Arm 2 - No Prior Autologous Transplant24
Arm 3 - Refractory Leukemia/Lymphoma1
Arm 4: MT2006-01 Coenrolling Patients12
Arm 5 - Previous Autologous Transplant13
Arm 6 - No Prior Autologous Transplant13

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Number of Participants With Chronic Graft-Versus-Host Disease

Determine the incidence of chronic GVHD at 1 year after transplant. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. (NCT00305682)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant18
Arm 2 - No Prior Autologous Transplant20
Arm 3 - Refractory Leukemia/Lymphoma0
Arm 4: MT2006-01 Coenrolling Patients3
Arm 5 - Previous Autologous Transplant3
Arm 6 - No Prior Autologous Transplant3

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Number of Participants With Neutrophil Engraftment

Time to 1st 3 consecutive days with absolute neutrophil count (ANC) > 5 x 10^8/L and percentage of patients with neutrophil recovery by day 42 (Cumulative incidence). (NCT00305682)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant93
Arm 2 - No Prior Autologous Transplant65
Arm 3 - Refractory Leukemia/Lymphoma6
Arm 4: MT2006-01 Coenrolling Patients32
Arm 5 - Previous Autologous Transplant32
Arm 6 - No Prior Autologous Transplant29

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Number of Participants With Platelet Engraftment

Time to platelets > 20,000 (first of 3 consecutive days) with no platelet transfusions for seven days and percentage of patients with platelet engraftment >50,000 by day 100. (NCT00305682)
Timeframe: Day 180

InterventionParticipants (Count of Participants)
Arm 1-Previous Autologous Transplant75
Arm 2 - No Prior Autologous Transplant47
Arm 3 - Refractory Leukemia/Lymphoma3
Arm 4: MT2006-01 Coenrolling Patients28
Arm 5 - Previous Autologous Transplant34
Arm 6 - No Prior Autologous Transplant25

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Percentage of Donor Chimerism at 100 Days

Chimerism studies will be performed on the blood and bone marrow (BM). BM chimerism days 21 and 100, at 6 months and 1 year to determine the relative contribution of donor and recipient hematopoiesis. (NCT00305682)
Timeframe: 100 days

Interventionpercentage of donor cells (Mean)
Arm 1-Previous Autologous Transplant94
Arm 2 - No Prior Autologous Transplant94
Arm 3 - Refractory Leukemia/Lymphoma100
Arm 4: MT2006-01 Coenrolling Patients93
Arm 5 - Previous Autologous Transplant85
Arm 6 - No Prior Autologous Transplant86

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Percentage of Donor Chimerism at 180 Days

Chimerism studies will be performed on the blood and bone marrow (BM). BM chimerism days 21 and 100, at 6 months and 1 year to determine the relative contribution of donor and recipient hematopoiesis. (NCT00305682)
Timeframe: 180 Days

Interventionpercentage of donor cells (Mean)
Arm 1-Previous Autologous Transplant96
Arm 2 - No Prior Autologous Transplant98
Arm 3 - Refractory Leukemia/Lymphoma88
Arm 4: MT2006-01 Coenrolling Patients94
Arm 5 - Previous Autologous Transplant91
Arm 6 - No Prior Autologous Transplant98

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Percentage of Donor Chimerism at 21 Days

Chimerism studies will be performed on the blood and bone marrow (BM). BM chimerism days 21 and 100, at 6 months and 1 year to determine the relative contribution of donor and recipient hematopoiesis. (NCT00305682)
Timeframe: 21 days

Interventionpercentage of donor cells (Mean)
Arm 1-Previous Autologous Transplant77
Arm 2 - No Prior Autologous Transplant73
Arm 3 - Refractory Leukemia/Lymphoma57
Arm 4: MT2006-01 Coenrolling Patients77
Arm 5 - Previous Autologous Transplant69
Arm 6 - No Prior Autologous Transplant68

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Percentage of Donor Chimerism at 365 Days

Chimerism studies will be performed on the blood and bone marrow (BM). BM chimerism days 21 and 100, at 6 months and 1 year to determine the relative contribution of donor and recipient hematopoiesis. (NCT00305682)
Timeframe: 365 days

Interventionpercentage of donor cells (Mean)
Arm 1-Previous Autologous Transplant99
Arm 2 - No Prior Autologous Transplant98
Arm 4: MT2006-01 Coenrolling Patients99
Arm 5 - Previous Autologous Transplant87
Arm 6 - No Prior Autologous Transplant100

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Modification of Diet in Renal Disease (MDRD) Estimation of Glomerular Filtration Rate (GFR)

"This outcome measure is used to determine if the reduction of calcineurin inhibitor immunosuppression leads to improved native kidney function. Native kidney function is assessed using the Modification of Diet in Renal Disease (MDRD) estimation of glomerular filtration rate (GFR) from serum or plasma creatinine samples at the reported time points.~Reference intervals include:~Healthy 18 years and up: 60-120 mL/min/1.73 sqm Chronic kidney disease: GFR < 60 mL/min/1.73 sqm Kidney failure: GFR < 15 mL/min/1.73 sqm" (NCT01230502)
Timeframe: 12 months post enrollment/randomization

InterventionmL/min/1.73 sqm (Mean)
Group 3: Donor Specific Regulation (DSR) -, Standard of Care82.4

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Modification of Diet in Renal Disease (MDRD) Estimation of Glomerular Filtration Rate (GFR)

"This outcome measure is used to determine if the reduction of calcineurin inhibitor immunosuppression leads to improved native kidney function. Native kidney function is assessed using the Modification of Diet in Renal Disease (MDRD) estimation of glomerular filtration rate (GFR) from serum or plasma creatinine samples at the reported time points.~Reference intervals include:~Healthy 18 years and up: 60-120 mL/min/1.73 sqm Chronic kidney disease: GFR < 60 mL/min/1.73 sqm Kidney failure: GFR < 15 mL/min/1.73 sqm" (NCT01230502)
Timeframe: 6 months post enrollment/randomization

InterventionmL/min/1.73 sqm (Mean)
Group 3: DSR (-), Standard of Care60.14

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Change in Best Spectacle-corrected Visual Acuity (BSCVA)

Change in best spectacle-corrected visual acuity (BSCVA) from baseline. Analysis on eye level (NCT01232920)
Timeframe: 6 months

InterventionLogMAR (Mean)
Methotrexate-0.26
Mycophenolate Mofetil-0.19

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Number of Participants Achieving Treatment Success

"TREATMENT SUCCESS is defined as controlled ocular inflammation in both eyes with less than or equal to 10 mg/day of prednisone and/or 2 topical steroid drops/day sustained for 2 visits separated by at least 28 days (control of inflammation and prednisone dose must be achieved by 5-month visit and sustained until 6-month visit).~Discontinuation of study medication at any time due to efficacy, tolerability, or safety may result in a declaration of TREATMENT FAILURE. Note that all patients will be classified as either a treatment success or failure." (NCT01232920)
Timeframe: 6 months

Interventionparticipants (Number)
Methotrexate24
Mycophenolate Mofetil15

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Time to Control of Inflammation

(NCT01232920)
Timeframe: 6 months

Interventiondays (Median)
Methotrexate139
Mycophenolate Mofetil124

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Number of Eyes With Resolution of Macular Edema

(NCT01232920)
Timeframe: 6 months

,
InterventionEyes (Number)
Eyes with Macular Edema at BaselineEyes with Resolved Macular Edema
Methotrexate2217
Mycophenolate Mofetil137

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Absolute Neutrophil Count (ANC) Engraftment

The median time in days to achieve ANC recovery defined as ANC>500uL. (NCT01434472)
Timeframe: Up to day 100

InterventionDays (Median)
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT)16

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Hematopoietic Toxicity

Clinical sequelae due to hematopoietic toxicity as defined by incidences of neutropenic fever and bleeding. (NCT01434472)
Timeframe: Up to day 100

InterventionIncidences (Number)
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT)5

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Overall Survival

(NCT01434472)
Timeframe: Up to 2 years post transplant

InterventionParticipants (Count of Participants)
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT)14

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Platelet Engraftment

The median time in days to achieve platelet recovery as defined as platelet >20,000uL. (NCT01434472)
Timeframe: Up to day 100

InterventionDays (Median)
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT)9

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Progression-free Survival

Based on a one-sample chi-square test with one-sided significance level of five percent. This study will be deemed successful if the 1 year progression-free survival of this highest-risk group of patients is 54% or greater. (NCT01434472)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT)11

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Response Rates

Response is defined as having achieved a Partial Response or better. (NCT01434472)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT)16

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BANFF Grades of First AMR.

Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR within 6 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, or C4d staining positive. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes. (NCT02495077)
Timeframe: 6 months post-transplantation

InterventionParticipants (Count of Participants)
Experimental0
Control0

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Change in eGFR Between 3 Months and 24 Months as Measured by CKD-EPI

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 3 and 24 was calculated as the month 24 eGFR minus the month 3 eGFR for each participant. A window of +/- 14 days was used for month 3 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 3 months and 24 months post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental2.7
Control4.4

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Change in eGFR Between 3 Months and 24 Months as Measured by MDRD

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 3 and 24 was calculated as the month 24 eGFR minus the month 3 eGFR for each participant. A window of +/- 14 days was used for month 3 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 3 months and 24 months post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental2.8
Control4.8

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Change in eGFR Between 6 Months and 24 Months as Measured by CKD-EPI

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 6 and 24 was calculated as the month 24 eGFR minus the month 6 eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental0.8
Control4.8

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Change in eGFR Between 6 Months and 24 Months as Measured by MDRD

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 6 and 24 was calculated as the month 24 eGFR minus the month 6 eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental1.0
Control5.2

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Change in eGFR Between Post-transplant Nadir and 24 Months as Measured by CKD-EPI

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. Post-transplant nadir was defined as the lowest value of eGFR from the first 6 months post-transplant. The change in eGFR between nadir and month 24 was calculated as the month 24 eGFR minus the nadir eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental8.5
Control12.0

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Change in eGFR Between Post-transplant Nadir and 24 Months as Measured by MDRD

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. Post-transplant nadir was defined as the lowest value of eGFR from the first 6 months post-transplant. The change in eGFR between nadir and month 24 was calculated as the month 24 eGFR minus the nadir eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental8.1
Control11.6

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Creatinine Reduction Ratio (CRR), Defined as the First Creatinine on Day 2 Divided by he First Creatinine After Surgery

Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 2 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. (NCT02495077)
Timeframe: Day 2 post-transplantation

InterventionPercentage (Mean)
Experimental24.28
Control20.97

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Creatinine Reduction Ratio (CRR), Defined as the First Creatinine on Day 5 Divided by the First Creatinine After Surgery.

Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 5 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. (NCT02495077)
Timeframe: Day 5 post-transplantation

InterventionPercentage (Mean)
Experimental47.06
Control43.37

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Days From Transplantation Until Event (ACR, AMR, or Hospitalization for Infection and/or Malignancy)

Participants are considered to have met this endpoint if they experienced biopsy-proven T-cell mediated rejection (ACR) or antibody mediated rejection (AMR) based on central pathology reading or were hospitalized for infection and/or malignancy. For participants who met one or more of these three components, the earliest event date of the three components was used as the time of meeting the endpoint. Participants who did not meet any of the three components were censored at their last date of follow-up. Event (or censor) day was calculated as event (or censor) date minus transplant date. (NCT02495077)
Timeframe: 24 months post-transplantation

InterventionDays to event (Median)
Experimental642
Control613

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Duration of Delayed Graft Function (DGF), Defined as Time From Transplantation to the Last Required Dialysis Treatment.

Participants are considered to have had DGF if they had at least one dialysis treatment in the first week post-transplant. For this endpoint, duration was calculated as the date of last post-transplant dialysis treatment minus the date of the first post-transplant dialysis treatment. (NCT02495077)
Timeframe: First post-transplant dialysis treatment to last post-transplant dialysis treatment

InterventionDays (Mean)
Experimental13.27
Control15.74

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eGFR Values as Measured by CKD-EPI

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Day 7 post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental41.01
Control41.85

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eGFR Values as Measured by MDRD

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Day 7 post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental38.93
Control39.96

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Number of Dialysis Sessions.

The number of dialysis sessions a person had during their first 8 weeks post-transplant was used for this endpoint. (NCT02495077)
Timeframe: 8 weeks post-transplantation

InterventionDialysis sessions (Mean)
Experimental0.14
Control0.26

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Percent of Participants That Required at Least One Dialysis Treatment.

Dialysis within the first week post-transplant is used in the setting of delayed graft function (DGF). Participants are considered to have had DGF if they had at least one dialysis treatment in the first week post-transplant. (NCT02495077)
Timeframe: 1 week post-transplantation

Interventionpercentage of participants (Number)
Experimental31.0
Control35.7

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Percent of Participants With Any Infection Requiring Hospitalization or Resulting in Death.

Participants were considered to have met this endpoint if they had an infection that required hospitalization or resulted in death. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental43.0
Control39.3

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Percent of Participants With BANFF Chronicity Scores > or Equal 2 on the 24 Month Biopsy.

The Banff 2013 classification involves scoring numerous characteristics of renal biopsy specimens. The ci (interstitial fibrosis) and ct (tubular atrophy) scores are two such characteristics. The scores can take values of 0, 1, 2, or 3 for each characteristic (ci and ct), indicating increasing severity of disease as the scores increase. Participants are considered to have met this endpoint if their ci + ct score on the 24 month biopsy summed to be > or equal to 2. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental73.1
Control36.4

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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection (AMR)

Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR within 6 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, or C4d staining positive.Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes. (NCT02495077)
Timeframe: 6 months post-transplantation

InterventionParticipants (Count of Participants)
Experimental0.0
Control0.0

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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection (AMR).

Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR within 24 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, or C4d staining positive. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participant (Number)
Experimental1.3
Control0.0

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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection AMR or Suspicious for AMR

Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR or suspicious for AMR within 6 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, C4d staining positive, or suspicious. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes; suspicious-when 2 of 3 factors for acute/active are present. (NCT02495077)
Timeframe: 6 months post-transplantation

Interventionpercentage of participants (Number)
Experimental2.8
Control0.0

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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection AMR or Suspicious for AMR.

Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR or suspicious for AMR within 24 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, C4d staining positive, or suspicious. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes; suspicious-when 2 of 3 factors for acute/active are present (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental3.8
Control1.4

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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR)

Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of greater than or equal to IA with or without clinical symptoms within 6 months of transplant were determined to have met the endpoint. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection.Criteria include: IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 6 month post-transplantation

Interventionpercentage of participants (Number)
Experimental4.2
Control3.0

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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR) or Borderline Rejection

Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of borderline or greater than or equal to IA with or without clinical symptoms within 24 months of transplant were determined to have met the endpoint. Severity is graded as Borderline, IA, IB, IIA, IIB, or III, with borderline representing possible cellular rejection, IA being the mildest form of cellular rejection, and III being the most severe form of cellular rejection. Criteria include: Borderline-no intimal arteritis is present but foci of mild tubulitis; IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental12.8
Control7

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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR) or Borderline Rejection.

Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of borderline or greater than or equal to IA with or without clinical symptoms within 6 months of transplant were determined to have met the endpoint. Severity is graded as Borderline, IA, IB, IIA, IIB, or III, with borderline representing possible cellular rejection, IA being the mildest form of cellular rejection, and III being the most severe form of cellular rejection.Criteria include: Borderline-no intimal arteritis is present but foci of mild tubulitis; IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 6 months post-transplantation

Interventionpercentage of participants (Number)
Experimental8.5
Control6.1

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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR).

Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of greater than or equal to IA with or without clinical symptoms within 24 months of transplant were determined to have met the endpoint. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection. Criteria include: IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental5.1
Control4.2

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Percent of Participants With BK Viremia That Require a Change in Immunosuppression or Anti-viral Treatment as Per Standard of Care at the Site.

Participants were considered to have met this endpoint if they had a reported case of BK viremia that required a change in their existing immunosuppression or the use of anti-viral therapy. (NCT02495077)
Timeframe: 24 months post-transplantation

InterventionPercent of participants (Number)
Experimental28.9
Control13.4

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Percent of Participants With CMV Viremia That Require a Change in Immunosuppression or Anti-viral Treatment as Per Standard of Care at the Site

Participants were considered to have met this endpoint if they had a reported case of CMV viremia that required a change in their existing immunosuppression or the use of anti-viral therapy. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental18.4
Control11.6

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Percent of Participants With de Novo DSA.

Donor specific antibody (DSA) can be formed post-transplant as part of the recipient's alloimmune response to the transplanted organ. DSA was determined by a central laboratory. Participants with newly developed DSA (i.e., de novo) following transplant were considered to have met this endpoint. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental8.0
Control3.6

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Percent of Participants With Death or Graft Failure.

Participants who died or experienced graft failure were considered to have met this endpoint. Graft failure was defined as the need for post-transplant dialysis for more than 56 days. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental5.3
Control7.1

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Percent of Participants With Impaired Wound Healing Manifested by Wound Dehiscence, Wound Infection, or Hernia at the Site of the Transplant Incision

Participants were considered to have met this endpoint if they had a reported case of impaired wound healing at the site of the transplant incision manifested by one wound dehiscence, wound infection, or hernia. (NCT02495077)
Timeframe: 24 months post-transplantation

InterventionPercent of participants (Number)
Experimental7.9
Control11.6

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Percent of Participants With Locally Treated Rejection, Defined as Treatment Administered for Rejection Based on Clinical Signs or Biopsy Findings.

Biopsies were read by the local pathologist at the hospital where the participant was a patient. These local reads informed clinical care for the participant, which may or may not include prescribing/administering medication to the participant to help with clinical concerns or findings noted on a biopsy. Participants were considered to have met this endpoint if they have a report of receiving treatment for clinical or biopsy-proven rejection during the 24 month post-transplant follow-up. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental16.2
Control27

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Percent of Participants With Locally Treated Rejection, Defined as Treatment Administered for Rejection Based on Clinical Signs or Biopsy Findings.

Biopsies were read by the local pathologist at the hospital where the participant was a patient. These local reads informed clinical care for the participant, which may or may not include prescribing/administering medication to the participant to help with clinical concerns or findings noted on a biopsy. Participants were considered to have met this endpoint if they have a report of receiving treatment for clinical or biopsy-proven rejection during the first 6 months post-transplant. (NCT02495077)
Timeframe: 6 months post-transplantation

Interventionpercentage of participants (Number)
Experimental12.6
Control20.5

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Percent of Participants With Malignancy.

Participants were considered to have met this endpoint if they had a reported case of malignancy. (NCT02495077)
Timeframe: 24 months post-transplantation

InterventionPercent of Participants (Number)
Experimental1.8
Control0.9

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Percent of Participants With Mycobacterial or Fungal Infections

Participants were considered to have met this endpoint if they had at least one mycobacterial of fungal infection. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental6.1
Control6.3

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Percent of Participants With Only Graft Failure.

Participants who experienced graft failure were considered to have met this endpoint. Graft failure was defined as the need for post-transplant dialysis for more than 56 days. (NCT02495077)
Timeframe: 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental2.7
Control2.7

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Percent of Participants With Primary Non-Function (PNF), Defined as Dialysis-dependency for More Than 3 Months.

Post-transplant dialysis is sometimes required in the setting of kidney transplant. If such dialysis continues for more than 3 months, the participant is considered to have PNF and, as such, meets this endpoint definition. (NCT02495077)
Timeframe: Transplantation through at least month 3 up to month 24

InterventionPercent of Participants (Number)
Experimental2.8
Control0.9

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The Difference Between the Mean eGFR (Modified MDRD) in the Experimental vs. Control Groups.

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the month 24 eGFR for each treatment group. (NCT02495077)
Timeframe: 24-Month post-transplantation

InterventionmL/min/1.73m2 (Mean)
Experimental52.45
Control57.35

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The Percent of Participants Who Need Dialysis After Week 1.

Participants who needed dialysis after the first week post-transplant were considered to have met this endpoint. (NCT02495077)
Timeframe: 1 week to 24 months post-transplantation

Interventionpercentage of participants (Number)
Experimental9.0
Control2.8

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The Percent of Participants Whose Day 2 Serum CRR Was Less Than 30%.

Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 2 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. A participant was considered to have met this endpoint if their day 2 serum CRR was less than 30%. (NCT02495077)
Timeframe: Day 2 post-transplantation

Interventionpercentage of participants (Number)
Experimental57.1
Control68.6

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The Percent of Participants Whose Day 5 Serum CRR Was Less Than 70%.

Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 5 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. A participant was considered to have met this endpoint if their day 5 serum CRR was less than 70%. (NCT02495077)
Timeframe: Day 5 post-transplantation

Interventionpercentage of participants (Number)
Experimental74.4
Control88.4

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The Percent of Participants With a Serum Creatinine of More Than 3 mg/dL.

Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. This endpoint is ascertaining slow graft function in the immediate days post-transplant. A participant was considered to have met this endpoint if their day 5 serum creatinine was greater than 3 mg/dL. (NCT02495077)
Timeframe: Day 5 post-transplantation

Interventionpercentage of participants (Number)
Experimental47.4
Control42.9

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Change From Baseline (Immediately After Surgery) in Serum Creatinine.

Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. eGFR values from 24, 48, and 72 hours post-transplant (i.e., days 1, 2, and 3) were used to generate an estimate of the serum creatinine at each time point of interest for each treatment group. (NCT02495077)
Timeframe: 24, 48 and 72 hours post-transplantation

,
Interventionmg/dL (Mean)
24 Hours48 Hours72 Hours
Control6.855.875.21
Experimental7.356.245.77

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eGFR Values as Measured by CKD-EPI

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Days 30, 90, and 180 post-transplantation

,
InterventionmL/min/1.73m2 (Mean)
Day 30Day 90Day 180
Control50.6351.4452.65
Experimental49.2949.8050.56

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eGFR Values as Measured by MDRD

Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Days 30, 60, and 180 post-transplantation

,
InterventionmL/min/1.73m2 (Mean)
Day 30Day 90Day 180
Control48.2048.9950.16
Experimental46.6447.1447.89

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Acute Rejection

Number of subjects who experience acute rejection of the renal allograft. (NCT01653847)
Timeframe: 12 months post transplant

InterventionParticipants (Count of Participants)
Group 1: Tacrolimus With MMF.4
Group 2: Tacrolimus With Everolimus0

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Patient Survival

The number of patients who were alive at 2 years post transplant (NCT01653847)
Timeframe: baseline - 24 months post transplant

InterventionParticipants (Count of Participants)
Group 1: Tacrolimus With MMF.20
Group 2: Tacrolimus With Everolimus20

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Renal Allograft Survival

The number of subjects with renal allograft survival. (NCT01653847)
Timeframe: 12 months post-transplant

InterventionParticipants (Count of Participants)
Group 1: Tacrolimus With MMF.20
Group 2: Tacrolimus With Everolimus20

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Change in Glomerular Filtration Rate (GFR)

Evaluate the change in graft function (as measured by GFR) at 12 months post-transplant from baseline. (NCT01653847)
Timeframe: 3 months, 6 months, and 12 months post-transplant

,
Interventionml/minutes per 1.73 meters^2 (Mean)
3 months6 months12 months
Group 1: Tacrolimus With MMF.636465
Group 2: Tacrolimus With Everolimus666472

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Change in T Cell & B Cell Generation

Evaluate the change in regulatory T cell generation and review the relationship of the newly generated T cells with their function in the two maintenance immunosuppressive regimens at baseline, 3 and 12 months post-transplant. (NCT01653847)
Timeframe: Baseline, 3 months, and 12 months post-transplant

,
InterventionMean % of Treg cells in peripheral blood (Mean)
Baseline3 Months12 Months
Group 1: Tacrolimus With MMF.1.050.80.81
Group 2: Tacrolimus With Everolimus0.931.121.18

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Percent of Participants With Adverse Events (AEs) Attributable to the Donor Alloantigen Reactive Tregs (darTregs) Infusion

"AEs classified by the site investigator/clinician as possibly or definitely related to the study treatment, the Donor Alloantigen Reactive Tregs (darTregs) infusion. These AEs include:~infusion reaction~Grade 3 or higher cytokine release syndrome (Reference: National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 (4/28/2009) grading criteria~malignant cellular transformation." (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation

InterventionPercent of Participants (Number)
Cohort 20

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Percent of Participants With Grade 3 or Higher Infectious Adverse Event(s)

"The severity of infectious adverse events (AEs) was classified into grades as follows:~Grade 1 = asymptomatic; clinical or diagnostic observation only; intervention with oral antibiotic, antifungal, or antiviral agent only; no invasive intervention required~Grade 2 = symptomatic; intervention with intravenous antibiotic, antifungal, or antiviral agent; invasive intervention may be required~Grade 3 = any infection associated with hemodynamic compromise requiring pressors; any infection necessitating intensive care unit level of care; any infection necessitating operative intervention; any infection involving the central nervous system; any infection with a positive fungal blood culture; any proven or probable aspergillus infection; any tissue invasive fungal infection; any pneumocystis jiroveci infection~Grade 4 = life-threatening infection~Grade 5 = death resulting from infection" (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation

InterventionPercent of Participants (Number)
Cohort 10
Cohort 211.1

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Percent of Participants With Grade 3 or Higher Wound Complication(s) Adverse Event(s)

"The severity of adverse events (AEs) was classified into grades using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 (4/28/2009):~Grade 3 wound complications are defined as Hernia without evidence of strangulation; fascial disruption/dehiscence; primary wound closure or revision by operative intervention indicated~Grade 4 complications are defined as Hernia with evidence of strangulation; major reconstruction flap, grafting, resection, or amputation indicated" (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation

InterventionPercent of Participants (Number)
Cohort 10
Cohort 20

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Percent of Participants With Biopsy-Proven Acute and/or Chronic Rejection

"Biopsy-proven acute rejection graded as Mild, Moderate or Severe, per 1997 Banff classification. Chronic Rejection graded using Banff 2000 classification.~References: 1.) Banff Schema for Grading Liver Allograft Rejection: An International Consensus Document developed by an international panel of experts in liver transplantation pathology, hepatology, and surgery (Hepatology 1997; 25(3): 658-663). 2.) Update of the International Banff Schema for Liver Allograft Rejection: Working Recommendations for the Histopathologic Staging and Reporting of Chronic Rejection (Hepatology 2000; 31(3): 792-799)." (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation

,
InterventionPercent of Participants (Number)
Mild Acute RejectionModerate Acute RejectionSevere Acute RejectionChronic Rejection
Cohort 10000
Cohort 211.1000

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Percent of Participants With Grade 2 or Higher Hematologic Adverse Events (AEs) of Anemia, Neutropenia, and/or Thrombocytopenia

"The severity of adverse events (AEs) was classified into grades using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 (4/28/2009):~Grade 1 = mild AE~Grade 2 = moderate AE~Grade 3 = severe and undesirable AE~Grade 4 = life-threatening or disabling AE~Grade 5 = death" (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation

,
InterventionPercent of Participants (Number)
AnemiaNeutropeniaThrombocytopenia
Cohort 166.716.716.7
Cohort 222.200

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Duration in Days of Adverse Events (AEs)- Including Infection, Wound Complications, Post-transplant Diabetes, Hemorrhagic Cystitis and Malignancy

AEs reported as an infection, wound complication, post-transplant diabetes, hemorrhagic cystitis and/or malignancy. Time (in days) from the start date of the AE until the end date of the AE. Two events contributed to this calculation. (NCT02029638)
Timeframe: First Dose of Study Medication to End of Study (Up to 25 Months After Enrollment)

InterventionDays (Mean)
Enrolled, Transplanted9.5

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Duration in Days of Graft-versus-Host Disease in Transplanted Participants

Graft-versus-host disease (GVHD) is a medical complication that can occur after a person receives transplanted tissue, most commonly occurring after a bone marrow transplant. The white blood cells from the donated tissue recognize the tissue recipient's cells as foreign. These donor cells then attack the recipient's cells. Duration (in days) is measured as the time from the start of the GVHD event to the end of the GVHD event. (NCT02029638)
Timeframe: Transplant to Two Years Post-Transplant

InterventionDays (Mean)
Enrolled, Transplanted7

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Number of Days From Neutrophil Nadir to Absolute Neutrophil Recovery

Time (in days) from neutrophil nadir, the first day post-transplant on which the absolute neutrophil count (ANC) is below 500 per µL, to the first day after three consecutive daily ANCs ≥ 500 per µL. ANC is a measure of the number of neutrophils present in the blood. Neutrophils are a type of white blood cell that fight against infection. A healthy person has an ANC between 2,500 and 6,000 per µL. A value below 500 per µL means the risk of infection is higher. (NCT02029638)
Timeframe: Post-Transplant Neutrophil Nadir to Neutrophil Recover

InterventionDays (Mean)
Enrolled, Transplanted15

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Number of Days From Transplant to Platelet Count Recovery

Time (in days) from transplant to the first day of a platelet count of ≥20,000 per μL without a prior platelet transfusion in the preceding seven days. Low platelet numbers is associated with increased risk of bleeding and bruising. A healthy person has a platelet count ranging from 150,000 to 450,000 platelets per microliter of blood. (NCT02029638)
Timeframe: Transplant to Platelet Count Recovery

InterventionDays (Mean)
Enrolled, Transplanted36

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Number of Days Post-Transplant to the First Episode of Acute Rejection Requiring Treatment

Number of days post-transplant to the first episode of acute rejection that required treatment. This includes acute rejection episodes requiring treatment that were not biopsy proven. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months After Enrollment)

InterventionDays (Mean)
Enrolled, Transplanted23

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Number of Participants Experiencing an Incidence of Graft-versus-Host Disease Post-Transplant

Graft-versus-host disease (GVHD) is a medical complication that can occur after a person receives transplanted tissue, most commonly occurring after a bone marrow transplant. The white blood cells from the donated tissue recognize the tissue recipient's cells as foreign. These donor cells then attack the recipient's cells. (NCT02029638)
Timeframe: Transplant to Two Years Post-Transplant

InterventionParticipants (Count of Participants)
Enrolled, Transplanted1

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Number of Participants Free From Return to Immunosuppression for the Duration of the Study

Participants who were able to withdrawal successfully from all immunosuppression medication and remained off all immunosuppression medication for the remainder of the study. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 Months)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted0

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Number of Transplanted Participants Who Developed Donor- Specific Antibody After Initiation of Immunosuppression Withdrawal

Donor-specific antibodies are directed against antigens expressed on donor organs. These antibodies can result in an immune attack on the transplanted organ, increasing risk of graft loss and/or rejection. (NCT02029638)
Timeframe: Initiation of Immunosuppression Withdrawal to End of Study (to 25 months)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted0

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Number of Transplanted Participants Who Developed Donor-Specific Antibody During Study Participation

Donor-specific antibodies are directed against antigens expressed on donor organs. These antibodies can result in an immune attack on the transplanted organ, increasing risk of graft loss and/or rejection. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted0

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Number of Transplanted Participants Who Died

Number of participant deaths after receiving a transplant per protocol. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months After Enrollment)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted1

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Number of Transplanted Participants Who Remained Off Immunosuppression for at Least 52 Weeks, Including Those in Whom the 52 Week Biopsy Was Not Performed

Number of transplanted participants who remained off immunosuppression for ≥52 weeks, including those in whom the 52 week biopsy was not performed. This outcome included participants who were able to withdrawal successfully from all immunosuppression medication and remain off all immunosuppression for 52 weeks after the completion of withdrawal. (NCT02029638)
Timeframe: Transplant to 52 Weeks after Discontinuation of All Immunosuppression

InterventionParticipants (Count of Participants)
Enrolled, Transplanted0

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Number of Transplanted Participants With Acute Renal Allograft Rejection

Acute renal allograft rejection demonstrated either by biopsy or clinically (when a biopsy could not be performed). This measure includes participants with biopsy proven acute renal allograft rejection and those that have creatinine values 25% or greater relative to baseline for over 72 hours. Baseline serum creatinine is defined as the average of the lowest three serum creatinine values during 2 to 4 weeks post-transplant, excluding days on dialysis. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months After Enrollment)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted1

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Number of Transplanted Participants With Chronic T Cell-Mediated or Antibody-Mediated Rejection

Outcome includes participants who experienced chronic T cell-mediated rejection, antibody-mediated rejection and progressive interstitial fibrosis/tubular atrophy (IF/TA), transplant glomerulopathy or chronic obliterative arteriopathy, without an alternative, non-rejection related cause. Reference: Banff 2007 Classification Renal Allograft Pathology definition of terms. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months After Enrollment)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted1

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Number of Transplanted Participants With Engraftment Syndrome

Engraftment syndrome is a complication that can occur following bone marrow transplant. The presence of engraftment syndrome is diagnosed by monitoring the common symptoms, which include: fever, rash, fluid in the lungs, and serum creatinine values above 4 mg/dL occurring within a week of absolute neutrophil recovery (e.g., first day after three consecutive daily absolute neutrophil counts ≥ 500 per µL), without apparent other cause. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months After Enrollment)

InterventionParticipants (Count of Participants)
Enrolled, Transplanted0

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Percent of Participants Who Achieved Operational Tolerance

Operational tolerance is defined as remaining off all immunosuppression 52 weeks after completion of immunosuppression withdrawal, with no evidence of biopsy-proven allograft rejection and, with acceptable renal function defined as a serum creatinine that has increased no more than 25% above baseline at the primary endpoint visit. Baseline creatinine is defined as the average of the lowest three creatinine values during 2 to 4 weeks post-transplant, excluding days on dialysis. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT02029638)
Timeframe: Transplantation through 52 Weeks after Discontinuation of All Immunosuppression

InterventionPercent of participants (Number)
Enrolled, Transplanted0

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Histological Severity of Biopsies Demonstrating Acute Rejection as Defined by Banff 2007 Classification Renal Allograft Pathology

This outcome includes results from biopsies with proven acute renal allograft rejection according to the 2007 Banff Classification Renal Allograft Pathology. A Banff result of indeterminate is not classified as rejection. (NCT02029638)
Timeframe: Transplant to End of Study (Up to 25 months After Enrollment)

Interventionparticipants (Number)
Acute Cellular Rejection Banff Grade IAAcute Cellular Rejection Banff Grade IBAcute Cellular Rejection Banff Grade IIAAcute Cellular Rejection Banff Grade IIBAcute Cellular Rejection Banff Grade IIIAcute Antibody Mediated Rejection
Enrolled, Transplanted000000

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Number of Adverse Events (AEs) by Severity- Including Infection, Wound Complications, Post-Transplant Diabetes, Hemorrhagic Cystitis and Malignancy

AEs reported as an infection, wound complication, post-transplant diabetes, hemorrhagic cystitis and/or malignancy. Grades are based on National Cancer Institute--Common Terminology Criteria (NCI-CTCAE) Version 4.0. (NCT02029638)
Timeframe: First Dose of Study Medication to End of Study (Up to 25 Months After Enrollment)

,
InterventionEvents (Number)
Grade 1Grade 2Grade 3Grade 4Grade 5
Enrolled, Not Transplanted00000
Enrolled, Transplanted00200

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Number of Adverse Events (AEs)- Including Infection, Wound Complications, Post-transplant Diabetes, Hemorrhagic Cystitis and Malignancy

AEs reported as an infection, wound complication, post-transplant diabetes, hemorrhagic cystitis and/or malignancy. (NCT02029638)
Timeframe: First Dose of Study Medication to End of Study (Up to 25 Months After Enrollment)

,
InterventionEvents (Number)
InfectionWound complicationsPost-transplant diabetesHemorrhagic cystitisMalignancy
Enrolled, Not Transplanted00000
Enrolled, Transplanted20000

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Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach

(NCT01982682)
Timeframe: Up to 1 year after HSCT

InterventionParticipants (Count of Participants)
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT)27

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Count of Participants That Experienced Death as a Result of Graft-versus-host Disease (GVHD)

"Graft versus host disease was clinically characterized based on 4 stages of progression for three major body areas, skin, liver, gut. Subjects who experienced life threatening reactions in their skin, liver and gut ultimately experienced functional impairment and expired.~Life threatening reactions in the Skin were characterized by desquamation (the shedding of the outer layers of skin) and bullae (a bubblelike cavity filled with air or fluid, in particular).~Life threatening reactions in the Liver were characterized by Bilirubin, > 15 mg/dl Life threatening reactions in the Gut were characterized by Pain +/- ileus (a painful obstruction of the ileum or other part of the intestine.)" (NCT01982682)
Timeframe: Up to 1 year after HSCT

InterventionParticipants (Count of Participants)
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT)2

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Number of Participants With Successful Engraftment

Will be reported descriptively. Successful engraftment is defined as ANC (absolute neutrophil count, the number of white blood cells (WBCs) that are neutrophils) ≥ 0.5x109/L for at least 30 days and Platelet engraftment > 20,000 with no transfusion x 7 days. (NCT01982682)
Timeframe: Up to 1 year after HSCT

InterventionParticipants (Count of Participants)
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT)37

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Median Pace of T Cell Immune Recovery at 28 Days Post Hematopoietic Stem Cell Transplantation (HSCT)

An excess amount of residual T-lymphocytes in the Peripheral Blood Stem Cell Collection (PBSC) product may increase the risk of GVHD. The ideal amount of T-cells left in the PBSC product is no greater than 5x104/kg. Every effort will be made to keep T-cell amounts to below this threshold. The median pace of T-cell immune recovery at 28 days will be monitored by collecting cluster of differentiation (CD24) and (CD38). (NCT01982682)
Timeframe: At 28 days post HSCT

Interventioncells/ul (Median)
Median CD3/4 count at d+28Median cluster of diff. 38 (CD3/8) count at d+28
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT)41.348

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Median Pace of T Cell Immune Recovery at 90 Days Post Hematopoietic Stem Cell Transplantation (HSCT)

An excess amount of residual T-lymphocytes in the Peripheral Blood Stem Cell Collection (PBSC) product may increase the risk of GVHD. The ideal amount of T-cells left in the PBSC product is no greater than 5x104/kg. Every effort will be made to keep T-cell amounts to below this threshold. The median pace of T-cell immune recovery at 90 days will be monitored by collecting cluster of differentiation (CD24) and (CD38). (NCT01982682)
Timeframe: 90 days post HSCT

Interventioncells/ul (Median)
Median CD3/4 count at d+90Median CD3/8 count at d+90
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT)141384

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Cumulative Oral Corticosteroid Dose

(NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)

Interventionmilligram (mg) (Median)
Rituximab (RTX)2775.00
Mycophenolate Mofetil (MMF)4005.00

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Percentage of Participants (Excluding Telemedicine [TM] Participants) Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score

(NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to clinical cut-off date (CCOD) of 28 November 2018)

InterventionPercentage of Participants (Number)
Rituximab (RTX)40.3
Mycophenolate Mofetil (MMF)9.5

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Percentage of Participants With Anti-Drug Antibodies (ADA)

Participants with treatment-induced and treatment-enhanced anti-drug antibodies. The clinical relevance of anti-rituximab antibody formation in RITUXAN treated pemphigus vulgaris (PV) participants is unclear. (NCT02383589)
Timeframe: Baseline up to 52 Weeks (up to CCOD of 28 November 2018)

InterventionPercentage of Participants (Number)
Rituximab (RTX)31.7

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Time to Initial Sustained Complete Remission

(NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)

InterventionWeeks (Median)
Rituximab (RTX)NA
Mycophenolate Mofetil (MMF)NA

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Time to Protocol Defined Disease Flare

Disease flare is defined as the appearance of three or more new lesions a month that do not heal spontaneously within 1 week or by the extension of established lesions in a participant who has achieved disease control. (NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)

InterventionWeeks (Median)
Rituximab (RTX)NA
Mycophenolate Mofetil (MMF)NA

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Total Number of Protocol Defined Disease Flares

Disease flare is defined as appearance of three or more new lesions a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who has achieved disease control. (NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)

InterventionNumber of Flares (Number)
Rituximab (RTX)6
Mycophenolate Mofetil (MMF)44

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Percentage of Participants With Immunoglobulin (Ig) Levels Below Lower Limit of Normal (LLN)

(NCT02383589)
Timeframe: Baseline; Weeks 16, 24, 40 and 52; (end of treatment: up to Week 52) (up to CCOD of 28 November 2018)

,
InterventionPercentage of Participants (Number)
Baseline (IgA)Week 16 (IgA)Week 24 (IgA)Week 40 (IgA)Week 52 (IgA)Baseline (IgG)Week 16 (IgG)Week 24 (IgG)Week 40 (IgG)Week 52 (IgG)Baseline (IgM)Week 16 (IgM)Week 24 (IgM)Week 40 (IgM)Week 52 (IgM)
Mycophenolate Mofetil (MMF)01.82.22.73.66.01.82.20011.923.228.324.328.6
Rituximab (RTX)001.7006.19.83.43.54.37.624.627.129.829.8

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Recipient and Donor Genotyping for Selected Variants of CYP3A5, ABCB1 (MDR1), and CYP4A Genes

A blood sample was obtained from recipients and donors to measure gene polymorphism effects on metabolism of calcineurin inhibitor and everolimus. The polymorphisms are represented as the number of SNP occurrences for the CYP3A5, ABCB1 (MDR1) gene, and CYP4A4*22 genes. (NCT01936519)
Timeframe: 2 years

,
InterventionParticipants (Count of Participants)
Recipient Genotypes : rs776746 (CYP3A5 gene)Recipient Genotypes : rs1045642 (ABCB1 gene)Recipient Genotypes : rs1128503 (ABCB1 gene)Recipient Genotypes : rs2032582 (ABCB1 gene)Recipient Genotypes : rs35599367 (CYP4A4*22 gene)Donor Genotypes : rs776746 (CYP3A5 gene)Donor Genotypes : rs1045642 (ABCB1 gene)Donor Genotypes : rs1128503 (ABCB1 gene)Donor Genotypes : rs2032582 (ABCB1 gene)Donor Genotypes : rs35599367 (Cyp4A4*22)
Calcineurin Inhibitor and Mycophenolic Acid11671131000
Everolimus and Mycophenolic Acid11680105001

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Renal Function as Measured by 24 Hour Urine Creatinine Clearance

Renal Function was assessed by 24 hr urine collection creatinine clearance measured (mL/min). 24 Hr urine collection was assessed at baseline, 6 months, 1 year, and 2 years post transplant. (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years

,
InterventionmL/min (Mean)
24hr Urine Creatinine Clearance at 6 months24hr Urine Creatinine Clearance at 1 year24hr Urine Creatinine Clearance at 2 years
Calcineurin Inhibitor With Mycophenolic Acid68.0968.0961.54
Everolimus With Mycophenolic Acid70.7586.890.63

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Renal Function as Measured by Cockcroft Gault Creatinine Clearance

The Cockcroft-Gault formula for estimating creatinine clearance was determined at 6 months, 1 year, and 2 years post transplant (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years

,
InterventionmL/min/1.73m2 (Mean)
Cockcroft Gault Creatinine Clearance at 6 monthsCockcroft Gault Creatinine Clearance at 1 yearCockcroft Gault Creatinine Clearance at 2 years
Calcineurin Inhibitor and Mycophenolic Acid79.8486.8480.85
Everolimus and Mycophenolic Acid100.17113.47108.16

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Renal Function as Measured by Iothalamate Clearance

Iothalamate Clearance was assessed at 6 months, 1 year, and 2 years post transplant. (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years

,
InterventionmL/min/1.73m2 (Mean)
Iothalamate Clearance at 6 monthsIothalamate Clearance at 1 yearIothalamate Clearance at 2 years
Calcineurin Inhibitor and Mycophenolic Acid67.9966.6557.19
Everolimus and Mycophenolic Acid74.23104.0179.41

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Renal Function as Measured by Modification of Diet in Renal Disease (MDRD) Estimated Glomerular Filtration Rate (eGFR)

Modification of Diet in Renal Disease (MDRD) estimated Glomerular Filtration Rate (eGFR) was assessed at 6 months, 1 year, and 2 years post transplant. (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years

,
InterventionmL/min/1.73 m2 (Mean)
MDRD eGFR at 6 monthsMDRD eGFR at 1 yearMDRD eGFR at 2 years
Calcineurin Inhibitor and Mycophenolic Acid62.1860.6353.29
Everolimus and Mycophenolic Acid81.2788.0187.37

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Renal Function as Measured by Serum Creatinine Level

Serum creatinine levels were assessed at 6 months, 1 year, and 2 years post transplant (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years

,
Interventionmg/dL (Mean)
Serum Creatinine at 6 monthsSerum Creatinine at 1 yearSerum Creatinine at 2 years
Calcineurin Inhibitor and Mycophenolic Acid1.291.291.51
Everolimus and Mycophenolic Acid1.020.950.95

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Anti-CFZ533 Antibodies - Part I

To evaluate the immunogenicity of CFZ533 via the quantitative analysis of anti-CFZ533 antibodies (NCT02217410)
Timeframe: Baseline to end of study

Interventionanti-CFZ533 antibodies (Number)
CFZ533 + TAC + MMF (Part 1)0

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Mean AUClast Pharmacokinetic Parameter - Part I

Quantify pharmacokinetics of CFZ533 in combination with MMF, CS, and tacrolimus in de novo renal transplant patients during the treatment and follow-up periods. (NCT02217410)
Timeframe: Day 1

Interventionday*ug/mL (Mean)
CFZ533 + TAC + MMF (Part 1)367

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Mean Cmax Pharmacokinetic Parameter- Part I

Pharmacokinetics as defined by the systemic concentrations and Cmax of certain immunosuppressant medications used in Part I (NCT02217410)
Timeframe: Day 1

Interventionug/mL (Mean)
CFZ533 + TAC + MMF (Part 1)66.3

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Mean Tmax Pharmacokinetic Parameter - Part I

Quantify pharmacokinetics of CFZ533 in combination with MMF, CS, and tacrolimus in de novo renal transplant patients during the treatment and follow-up periods. (NCT02217410)
Timeframe: Day 1

Interventionday (Median)
CFZ533 + TAC + MMF (Part 1)0.237

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Anti-CFZ533 Antibodies - Part II

To evaluate the immunogenicity of CFZ533 via the quantitative analysis of anti-CFZ533 antibodies (NCT02217410)
Timeframe: Baseline to end of study (screening, baseline, Day 141, Day 225, Day 309, Study Completion)

Interventionanti-CFZ533 antibodies (Number)
ScreeningDay 141Day 225Day 309Study Completion
Tac + MMF (Part 2)00000

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Anti-CFZ533 Antibodies - Part II

To evaluate the immunogenicity of CFZ533 via the quantitative analysis of anti-CFZ533 antibodies (NCT02217410)
Timeframe: Baseline to end of study (screening, baseline, Day 141, Day 225, Day 309, Study Completion)

Interventionanti-CFZ533 antibodies (Number)
ScreeningBaselineDay 141Day 225Day 309Study Completion
CFZ533 + MMF (Part 2)000000

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CFZ533 Plasma PK Concentrations - Part II

Quantify the systemic concentrations of CFZ533 in combination with MMF, CS, and tacrolimus in de novo renal transplant patients during the treatment and follow-up periods. A full pharmacokinetic analysis can be performed on the concentration-time data to evaluate the impact of renal transplantation on the various medications used in the treatment regimen. (NCT02217410)
Timeframe: throughout study period (day 84 to day 336)

Interventionug/mL (Mean)
Day 84Day 112Day 140Day 168Day 196Day 224Day 252Day 280Day 308Day 336End of study
CFZ533 + MMF (Part 2)247211178157148147151160132156133

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Efficacy as Defined by the Frequency and Severity (Banff Classification) of Treated Biopsy Proven Acute Rejection (tBPAR) Adjudicated Data - Part II

"To assess the activity of the investigational arm as compared to the standard of care control arm in de novo renal transplant patients as measured by the frequency and severity of tBPAR as measured on the Banff classification scale.~An adjudication was performed on all on cause renal biopsies by an independent expert committee blinded to therapy." (NCT02217410)
Timeframe: 3, 6, 9, and 12 months

,
Interventionevents (Number)
Month 3Month 6Month 9Month 12
CFZ533 + MMF (Part 2)6777
Tac + MMF (Part 2)2333

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eGFR - Part II

"Renal function as assessed by MDRD (Modification of Diet in Renal Disease) formula.~eGFR: Estimated glomerular filtration rate" (NCT02217410)
Timeframe: Day 1, Day 29, Day 337,

,
Interventionml/min (Mean)
Day 1Day 29Day 337
CFZ533 + MMF (Part 2)9.855.658.2
Tac + MMF (Part 2)9.744.344.2

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Free CD40 and Total CD40 on B Cells - Part II

The magnitude and duration of peripheral blood CD40 occupancy. MESF: molecules of equivalent soluble fluorochrome (NCT02217410)
Timeframe: Baseline to end of study (Day 1/predose)

,
InterventionMESF (Mean)
CFZ553 + MMF (Baseline)CFZ553 + MMF (D1 - 6h post dose)CFZ553 + MMF (D15)CFZ553 + MMF (D 29)CFZ553 + MMF (D 57)CFZ553 + MMF (D 85)CFZ553 + MMF (D 197)CFZ553 + MMF (253)CFZ553 + MMF (EoS)Tac + MMF (Baseline)Tac + MMF (D1 - 6h post dose)Tac + MMF (D15)Tac + MMF (D29)Tac + MMF (D57)Tac + MMF (D85)Tac + MMF (D197)Tac + MMF (D253)Tac + MMF (EoS)
Free CD40 on Whole Blood B Ceels30836.001623.81799.62817.63597.13635.473699.02667.38176.1731508.3326437.6524441.6727840.0027994.2925044.0021360.0015752.7521200.00
Total CD40 on Whole Blood B Cells12778.8013806.9015160.3813299.6012234.389330.862820.721427.141069.6714581.4313715.0013707.1412698.7512583.858701.542067.601750.506276.17

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Total sCD40 Plasma Concentrations - Part II

To quantify the change from baseline and recovery of peripheral blood total soluble CD40 (NCT02217410)
Timeframe: 12 months

,
Interventionng/mL (Mean)
BaselineDay 1Day 4Day 15Day 29Day 57Day 85Day 113Day 141Day 169Day 197Day 225Day 253Day 281Day 309Day 337End of Study
CFZ533 + MMF (Part 2)3.026.9524.669.6101140189215237238253258236273286298303
Tac + MMF (Part 2)3.671.161.160.8690.3620.4380.4290.3910.4530.5370.4230.4520.4570.4550.4540.4110.959

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Total Soluble CD40 and Total Soluble CD154 Concentrations in Plasma - Part 1

To quantify the change from baseline and recovery of peripheral blood total soluble CD40 and total soluble CD154 (NCT02217410)
Timeframe: Baseline to end of study (Day 1, Day 29, Day 337)

,
Interventionng/ml (Mean)
BaselineDay 1Day 2Day 3Day 4Day 8Day 15Day 22Day 29Day 36Day 43Day 50Day 57Day 64Day 71Day 85Day 99Day 113Day 127EoS
sCD1540.1250.05850.1390.1570.2410.3990.08790.05000.2250.1160.01930.047800.03160.01480.013900.06680.04880.0184
sCD40 (Part I)4.038.8616.724.831.354.084.110212012814515616116315616815585.712.20.918

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GVHD-free Relapse-free Survival (GRFS)

Number of participants alive, without relapse, and without GVHD at 1 year. (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant19

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Number of Participants Who Experience Chronic GVHD

Number of participants who experience chronic GVHD by two years after BMT. (NCT02833805)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Bone Marrow Transplant1

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Number of Participants Who Experience Grades II-IV Acute GVHD

Number of participants who experience grade II, III, or IV acute GVHD by Day 100. (NCT02833805)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Bone Marrow Transplant4

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Number of Participants Who Experience Grades III-IV Acute GVHD

Number of participants who experience grade III or IV acute GVHD by Day 100. (NCT02833805)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Bone Marrow Transplant2

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Number of Participants Who Experience Primary Graft Failure

Number of participants who experience primary graft failure by one year after BMT. (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant21

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Number of Participants Who Experience Secondary Graft Failure

Number of participants who experience secondary graft failure by one year after BMT. (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant19

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Number of Participants With Full Donor Chimerism

Number of participants with full donor chimerism at Day 60. (NCT02833805)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Bone Marrow Transplant21

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Overall Survival and Engraftment at One Year

Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT). (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant19

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Overall Survival at One Year

Number of participants alive at one year after BMT. (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant19

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Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts

Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (>500 ANC). (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant21

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Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts

Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year. (NCT02833805)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant20

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100-day Overall Survival

Proportion of patients who survived 100 days or more after enrolled on the study (NCT00057954)
Timeframe: Assessed at least twice a week for the first 60 days and weekly until day 100.

InterventionProportion of participants (Number)
Transplant0.83

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Progression-free Survival

Progression-free survival was defined as time from enrollment to disease progression or death from any cause, whichever occurred first. Patients who did not have progression-free survival events were censored at last date of disease assessment. (NCT00057954)
Timeframe: Assessed day 100 post transplant and every 3 months during year 1, every 6 months during years 2-3, then every 12 months during years 4-5 or through diagnosis of disease progression

Interventiondays (Median)
Transplant104

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Proportion of Participants With Successful Engraftment

(NCT00057954)
Timeframe: Assessed daily during inpatient stay

InterventionProportion of participants (Number)
Transplant1

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Complete Response Rate

"Completed response is defined as:~Bone marrow evaluation: Repeat bone marrow showing < 5% myeloblasts with normal maturation of all cell lines, with no evidence for dysplasia (see dysplasia qualifier under peripheral blood evaluation).~Peripheral blood evaluation [absolute values must last at least 2 months] Hemoglobin >11 g/dl (untransfused, not on erythropoietin) Neutrophils (1500/mm3 (not on a myeloid growth factor)) Platelets (100,000/mm3 (not on a thrombopoetic agent)) Blasts - 0% No dysplasia. No detectable cytogenetic abnormality, if preexisting abnormality was present" (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.

Interventionpercentage of participants (Number)
Arm I35.3

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Number of Patients Who Developed Disease Progression After Achieving Complete Response

Disease free survival (DFS) was listed as a secondary endpoint in the study protocol, which would be assessed in patients who achieved complete response (CR). It was defined to be time from CR to documented progression or to death without progression. Patients without documented progression or death reported were censored at the time of last disease evaluation. However, due to the small number of patients with CR, the number of patients who developed disease progression was reported here. (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.

Interventionparticipants (Number)
Arm I1

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Overall Survival

Overall survival (OS) is defined to be the time from registration to death from any cause, with follow-up censored at the date of last contact. Kaplan-Meier method was used to estimate the distribution of OS. (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.

Interventionyears (Median)
Arm I1.2

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Proportion of Graft Versus Host Disease

Proportion of Graft versus Host Disease is calculated as number of patients with Graft versus Host Disease divided by all eligible and treated patients (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.

Interventionproportion of participants (Number)
Arm I0.412

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Time to Engraftment for Neutrophil

Time to neutrophil engraftment is defined from date of infusion to date of neutrophil engraftment. Neutrophil engraftment is defined as ANC > 500/mm3 on two consecutive measurements. The date of engraftment is the date of the first ANC > 500/mm3. (NCT00045305)
Timeframe: Daily while hospitalized and then at least 1x/week for the first 50 days and then at least every other week until day 100.

Interventiondays (Median)
Arm I18

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Time to Engraftment for Platelet

Time to platelet engraftment is defined from date of infusion to date of platelet engraftment. The platelet engraftment is defined as platelets > 20,000 on two consecutive measurements, at least seven days apart, without platelet transfusions in between and for at least three days before the first measurement that is over 20,000. The date of engraftment is the date of the first measurement that is over 20,000. (NCT00045305)
Timeframe: Daily while hospitalized and then at least 1x/week for the first 50 days and then at least every other week until day 100.

Interventiondays (Median)
Arm I18

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Two-Year Disease-free Survival of Study Participants Who Completed the Study Regimen

"Survival and complete resolution of all signs of leukemia 2 years after transplant with all of the following:~1, Normal bone marrow with blasts <5% with normal cellularity, normal megakaryopoiesis, more than 15% erythropoiesis, and more than 25% granulocytopoiesis.~2. Normalization of blood counts (no basts, platelets >100,000/mm3, granulocytes >1,500/mm3) 3. No extramedullary disease." (NCT00589316)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
Dose Level 1: 12 Gy Iodine-131 + BC8 Monoclonal Antibody0
Dose Level 2: 14 Gy Iodine-131 + BC8 Monoclonal Antibody1
Dose Level 3: 16 Gy Iodine-131 + BC8 Monoclonal Antibody1
Dose Level 4: 18 Gy Iodine-131 + BC8 Monoclonal Antibody1
Dose Level 5: 20 Gy Iodine-131 + BC8 Monoclonal Antibody1
Dose Level 6: 22 Gy Iodine-131 + BC8 Monoclonal Antibody0
Dose Level 7: 24 Gy Iodine-131 + BC8 Monoclonal Antibody0
Dose Level 8: 26 Gy Iodine-131 + BC8 Monoclonal Antibody1

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Chronic Graft vs Host Disease (GvHD)

The incidence of chronic graft vs host disease (cGvHD) following the infusion of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells will be assessed. The outcome is reported as the number of allogeneic CD8+ memory T-cell recipients who experience cGvHD more 30 days but within 1 year of the cellular infusion, a number without dispersion. (NCT02424968)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells2

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Disease Progression (TDP)

Whether or not the treated disease returns, known as disease progression or relapse, is a measure of treatment efficacy. Recipients of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells were monitored for disease progression through 1 year after the cellular infusion. The outcome is reported as the number of allogeneic CD8+ memory T-cells recipients that experienced disease progression within 12 months (1 year). (NCT02424968)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells4

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Event-free Survival (EFS

Event-free survival (EFS) is defined as the number of transplant recipients of allogeneic cluster of differentiation 8 (CD8+) memory T-cells that remain alive at 12 months after transplant without disease relapse. Relapse is defined as bone marrow blasts > 5% . The outcome is expressed as the number of allogeneic CD8+ memory T-cell recipients remaining alive at 1 year after transplant without disease relapse, a number without dispersion. (NCT02424968)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells15

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Full-dose Donor Chimerism (FDC)

A measure of success for the therapeutic infusion of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells is full-dose donor chimerism (FDC). This means to achieve ≥ 95% donor cells in either the CD3+ blood cell lineage or whole blood, within 90 days of the allogeneic CD8+ memory T-cell infusion. The outcome is reported as the number of participants that achieve FDC within 90 days, a number without dispersion. (NCT02424968)
Timeframe: 3 months

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells12

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Incidence of Acute Graft vs Host Disease (GvHD)

Occurrence of acute graft vs host disease (aGvHD) following the infusion of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells will be assessed. The outcome is reported as the number of allogeneic CD8+ memory T-cell recipients who experience aGvHD within 30 days of the cellular infusion, a number without dispersion. (NCT02424968)
Timeframe: Up to 30 days post-infusion

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells1

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LOWSKY Grade 3 or Higher Toxicities

Related adverse events, ie, toxicities, ≥ Grade 3 are significant considerations in the treatment of study participants receiving allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells. The outcome is reported as the number of allogeneic CD8+ memory T-cells transplant recipients who experienced ≥ Grade 3 toxicity within 60 days of infusion of the allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells, a number without dispersion. (NCT02424968)
Timeframe: Up to 60 days post-infusion

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells0

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Non-relapse Mortality (NRM)

Non-relapse mortality (NRM) is defined as death without known disease relapse or recurrence. The outcome is expressed as the number of allogeneic CD8+ memory T-cells tr. ansplant recipients whose cause of death was not disease relapse or recurrence, a number without dispersion (NCT02424968)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells1

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Overall Survival (OS)

Overall survival (OS) is defined as remaining alive 12 months after the infusion of allogeneic cluster of differentiation 8 (CD8+) memory T-cells. The outcome is reported as the number of allogeneic CD8+ memory T-cell transplant recipients remaining alive at 12 months after the cellular infusion, a number without dispersion (NCT02424968)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Infusion of Allogeneic CD8+ Memory T-cells15

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Incidence of Chronic GVHD

Cumulative incidence of chronic GVHD by 1 year. Chronic GVHD diagnosis follow National Institutes of Health (NIH) Consensus guidelines. (NCT03018223)
Timeframe: 1 year post HCT

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis20.0

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Incidence of Grade II-IV Acute Graft vs. Host Disease (GVHD)

Cumulative incidence of grade II-IV acute GVHD by day 100 after HCT. Acute GVHD organ staging and assessment of overall grade will use standard consensus criteria. The cumulative incidence of acute and chronic GVHD will be estimated, considering malignancy relapse and non-relapse death as competing risk events. (NCT03018223)
Timeframe: 100 days post hematopoietic cell transplant (HCT)

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis18.8

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Overall Survival (OS)

Overall survival is defined as time from transplant to death or last follow-up, and is reported as percentage of surviving participants. (NCT03018223)
Timeframe: Up to 1 year post HCT

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis70.2

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Progression Free Survival (PFS)

Progression-free survival defined by the time interval from transplant to relapse/recurrence, to death or to last follow-up. Reported as percentage of participants who are disease free one year post HCT. (NCT03018223)
Timeframe: Up to 1 year post HCT

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis56.6

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Percent of Participants Experiencing Acute Allograft Rejection

Estimate the composite of treatment failure rate, defined as acute allograft rejection with a Banff grade 1A or higher, graft loss, or death at six months post-conversion, in patients converted to a once-daily immunosuppressant regimen of Envarsus®, everolimus, and prednisone versus patients converted to a twice-daily regimen of Envarsus®, MMF, and prednisone. (NCT02954198)
Timeframe: Baseline to 6 months post conversion

InterventionParticipants (Count of Participants)
Control: Envarsus + MMF0
Intervention: Envarsus + Everoliumus0

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Percent of Participants Who Experienced Kidney Transplant Graft Loss

Measure and compare time-to-event analysis between the two arms graft loss (time to event analysis) (NCT02954198)
Timeframe: Baseline to 6 months post conversion

InterventionParticipants (Count of Participants)
Control: Envarsus + MMF0
Intervention: Envarsus + Everoliumus0

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Self-reported Medication Adherence From Baseline to 6 Months.

Percent of subjects reporting high medication adherence at baseline compared to 6 months post-conversion, using the Morisky Medication Adherence scale (MMAS). The MMAS rates medication adherence on a scale of 0 to 8. 0 is high adherence, 1-2 is medium adherence, and greater than or equal to 3 is low adherence. (NCT02954198)
Timeframe: 6 months post conversion

,
Intervention% of participants (Number)
Baseline6 months post-conversion
Control: Envarsus + MMF8059
Intervention: Envarsus + Everoliumus4547

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Subject Specific Change on Medication Side Effect Scale

Examine subject specific change on a validated Medication Side Effect Scale at the time of the conversion versus six months post-conversion, compared between the two arms. Side effect burden scale is from 0 to 180. A lower score is less side effect burden, a higher score is more side effect burden. (NCT02954198)
Timeframe: Baseline to 6 months post conversion

,
Interventionunits on a scale (Mean)
Baseline6 month post-conversion
Control: Envarsus + MMF7193
Intervention: Envarsus + Everoliumus3738

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Dose Modifications

Number of dose modifications from baseline to 3 months post-conversion. (NCT02953873)
Timeframe: Baseline to 3 months post conversion

Interventionnumber of dose modifications (Median)
Homozygous CYP3A5 1 expressorsHeterozygous CYP3A5 1 expressorsCYP3A5 1 non expressers
Conversion Arm124

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Dose-normalized Trough

Difference in dose-normalized trough at steady state before and after conversion from tacrolimus IR to Astagraf XL® (NCT02953873)
Timeframe: Baseline to 3 months post-conversion

Interventionng/dL (Median)
Pre-Conversion Dose TroughPost-Conversion Dose Trough
Conversion Arm0.590.44

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Number of Days to Reach Therapeutic Trough Goal

Days to reach therapeutic goal after conversion (NCT02953873)
Timeframe: Baseline to 3 months post conversion

Interventionnumber of days (Median)
Homozygous CYP3A5 1 expressorsHeterozygous CYP3A5 1 expressorsCYP3A5 1 non-expressors
Conversion Arm151015

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Total Daily Dose

Difference in Total Daily Dose necessary for steady state therapeutic goal (NCT02953873)
Timeframe: Baseline to 3 months post conversion

Interventionmg (Median)
Pre-Conversion Tacrolimus DosePost-Conversion Tacrolimus Dose
Conversion Arm1015

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Weight-Based Dose Requirement

Weight-based dose requirements to reach therapeutic goal pre- and post-conversion (NCT02953873)
Timeframe: Baseline to 3 months post conversion

Interventionmg/kg (Median)
Pre-ConversionPost-Conversion
Conversion Arm0.110.16

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Donor Cell Engraftment

Chimerism analysis was performed in patients who recieved nonmyeloablative tranplsnat. In this group the definition of engraftment was a CD3 count greater than 50%. In the myeloablative group, engraftment was defined as an absolute neutrophil count greater than 50%. (NCT00322101)
Timeframe: After stem cell infusion to day 28

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)11
Arm II (Myeloablative Regimen)11

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Incidence and Severity of Acute and Chronic Graft-vs-host Disease

(NCT00322101)
Timeframe: After transplantation

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)1
Arm II (Myeloablative Regimen)4

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Incidence of Disease Progression/Relapse

Disease progression/relapse was defined by IWG criteria (NCT00322101)
Timeframe: After stem cell infusion to date of last follow up.

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)7
Arm II (Myeloablative Regimen)2

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Non-relapse Mortality

(NCT00322101)
Timeframe: At 100 days

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)0
Arm II (Myeloablative Regimen)0

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Overall Survival

(NCT00322101)
Timeframe: At 2 years

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)6
Arm II (Myeloablative Regimen)6

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Progression-free Survival

IWG criteria was used to determine disease progression (NCT00322101)
Timeframe: After stem cell infusion to date of last follow up.

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)7
Arm II (Myeloablative Regimen)5

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Change From Baseline in Anti-Double Stranded Deoxyribonucleic Acid (Anti-dsDNA) Antibody Levels at Week 52

Anti-dsDNA antibodies are a group of anti-nuclear antibodies targeting double stranded DNA. (NCT02550652)
Timeframe: Baseline and Week 52

Interventionlog IU/mL (Mean)
OBINUTUZUMAB 1000MG and MMF-0.810
PLACEBO and MMF-0.080

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Change From Baseline in C4 Levels at Week 52

Complement C4 is a blood test that reflects activation of complement pathway associated with immune deposition in certain autoimmune diseases. (NCT02550652)
Timeframe: Baseline, Week 52

Interventiong/L (Mean)
OBINUTUZUMAB 1000MG and MMF0.101
PLACEBO and MMF0.003

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Change From Baseline in Complement Component 3 (C3) Levels at Week 52

Complement C3 is a blood test that reflects activation of complement pathway associated with immune deposition in certain autoimmune diseases. (NCT02550652)
Timeframe: Baseline and Week 52

Interventiong/L (Mean)
OBINUTUZUMAB 1000MG and MMF0.311
PLACEBO and MMF0.108

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Percentage of Participants Who Achieve Protocol Defined Complete Renal Response (CRR) at Week 52

Percentage of participants with normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 red blood cells (RBCs)/high-power field (HPF) and the absence of red cell casts) and urinary protein to creatinine ratio < 0.5. Normalization of serum creatinine is defined as serum creatinine ≤ the upper limit of normal (ULN) range of central laboratory values if baseline (Day 1) serum creatinine is above the ULN or serum creatinine ≤ 15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. (NCT02550652)
Timeframe: From baseline to Week 52

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF34.9
PLACEBO and MMF22.6

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Percentage of Participants Who Achieve Protocol Defined CRR at Week 24

CRR defined as normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 red blood cells (RBCs)/high-power field (HPF) and the absence of red cell casts) and urinary protein to creatinine ratio < 0.5. Normalization of serum creatinine is defined as serum creatinine ≤ the upper limit of normal (ULN) range of central laboratory values if baseline (Day 1) serum creatinine is above the ULN or serum creatinine ≤ 15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. (NCT02550652)
Timeframe: Week 24

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF23.8
PLACEBO and MMF24.2

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Percentage of Participants Who Achieve Protocol Defined Modified CRR (mCRR1) at Week 52

mCRR1 has got two components only, i.e. serum Creatinine and urinary protein to creatinine ratio. mCRR1 is defined by attainment of normalization of serum creatinine as evidenced by 1.) serum creatinine ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is above the ULN or serum creatinine ≤15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine ≤ the ULN range of central laboratory values and 2.) Urinary protein to creatinine ratio <0.5. (NCT02550652)
Timeframe: Week 52

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF39.7
PLACEBO and MMF25.8

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Percentage of Participants Who Achieve Protocol Defined Overall Response (OR) at Week 52

OR includes both CRR and partial renal response (PRR). CRR as defined in the primary outcome measure above. PRR defined as 50% improvement in urine protein:creatinine ratio, with one of following conditions met: 1. If baseline urine protein:creatinine ratio is ≤ 3.0, then urine protein:creatinine ratio of <1.0. 2. If baseline protein:creatinine ratio is > 3.0, then urine protein:creatinine ratio of <3.0, serum creatinine ≤15% above baseline value, and no urinary red cell casts and either RBCs/HPF ≤50% above baseline or <10 RBCs/HPF. (NCT02550652)
Timeframe: From baseline to Week 52

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF55.6
PLACEBO and MMF35.5

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Percentage of Participants Who Achieve Protocol Defined Partial Renal Response (PRR) at Week 52

PRR defined as serum creatinine ≤15% above baseline value, no urinary red cell casts and either RBCs/HPF ≤ 50% above baseline or < 10 RBCs/HPF, 50% improvement in urine protein:creatinine ratio, with one of following conditions met: 1. If baseline urine protein:creatinine ratio is ≤ 3.0, then a urine protein:creatinine ratio of < 1.0. 2. If baseline protein:creatinine ratio is > 3.0, then a urine protein:creatinine ratio of < 3.0. (NCT02550652)
Timeframe: From baseline to Week 52

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF55.6
PLACEBO and MMF33.9

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Percentage of Participants Who Achieve Protocol Defined Second mCRR (mCRR2) at Week 52

mCRR2 is defined by normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 RBCs/HPF and the absence of red cell casts), and urinary protein to creatinine ratio <0.5. Normalization of serum creatinine as evidenced by the following: Serum creatinine ≤15% above baseline if baseline (Day 1) serum creatinine is above the normal range of the central laboratory values or ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. (NCT02550652)
Timeframe: Week 52

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF44.4
PLACEBO and MMF33.9

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Percentage of Participants Who Achieve Protocol Defined Third mCRR (mCRR3) at Week 52

mCRR3 is defined by normalization of serum creatine as evidenced by serum creatinine ≤ the ULN range of central laboratory values and urinary protein to creatinine ratio < 0.5. (NCT02550652)
Timeframe: Week 52

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF46.0
PLACEBO and MMF38.7

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Percentage of Participants With Anti-Drug Antibody (ADA) to Obinutuzumab

Antibodies are a blood protein produced in response to and counteracting a specific antigen. (NCT02550652)
Timeframe: From baseline up to Week 104

Interventionpercentage of participants (Number)
OBINUTUZUMAB 1000MG and MMF9.38

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Area Under the Plasma Concentration Versus Time Curve (AUC) of Obinutuzumab

(NCT02550652)
Timeframe: Week 0, Week 24, Week 52

Interventionug/mL*day (Mean)
Week 0-24Week 24-52Week 0-52
OBINUTUZUMAB 1000MG and MMF105951581126406

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Change From Baseline of Participant's Global Assessment of Disease Activity Visual Analog Scale (VAS) Score

Each VAS had a range from 0-100 with higher scores indicating greater symptom impact on global health status. (NCT02550652)
Timeframe: Baseline (Day 1), Weeks 4, 12, 24, 36, 52

,
Interventionscore on scale (Mean)
BaselineWeek 4Week 12Week 24Week 36Week 52
OBINUTUZUMAB 1000MG and MMF41.3-14.4-19.9-25.0-24.8-25.4
PLACEBO and MMF39.4-8.7-11.6-20.8-19.6-23.3

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Maximum Observed Plasma Concentration (Cmax) of Obinutuzumab

(NCT02550652)
Timeframe: Week 0, Week 24, Week 52

Interventionug/mL (Mean)
Week 0-24Week 24-52Week 0-52
OBINUTUZUMAB 1000MG and MMF559605605

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Percent Change From Baseline in Circulating CD19-Positive B-Cell Levels

CD19+ B cell is a B-lymphocyte with a transmembrane protein that is encoded by the gene CD19. (NCT02550652)
Timeframe: Baseline, Week 2, Week 4, Week 12, Week 24, Week 52

,
InterventionPercent change of cells/uL (Mean)
Week 2Week 4Week 12Week 24Week 52
OBINUTUZUMAB 1000MG and MMF-97.469-98.777-97.045-96.628-98.620
PLACEBO and MMF39.293-5.1860.661-11.44637.695

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Percentage of Participants With Adverse Events (AEs)

An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. AEs, including AEs of Special Interest were reported based on the national cancer institute common terminology criteria for AEs, Version 4.0 (NCI-CTCAE, v4.0). Reported are the number of subjects with AEs, Grade 3-5 AEs, and Serious Adverse Events (SAEs), Infections and Serious infections. (NCT02550652)
Timeframe: From Baseline up to Week 104

,
Interventionpercentage of participants (Number)
Adverse EventsGrade 3 AEsGrade 4 AEsGrade 5 AEsSerious Adverse EventsInfectionsSerious infections
OBINUTUZUMAB 1000MG and MMF90.629.710.91.625.075.07.8
PLACEBO and MMF88.524.613.16.629.562.318.0

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Percentage of Participants With First Protocol Defined CRR Over the Course of 52 Weeks

CRR included normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 RBCs/HPF and the absence of red cell casts) and urinary protein to creatinine ratio < 0.5. Normalization of serum creatinine is defined as serum creatinine ≤ the ULN range of central laboratory values if baseline serum creatinine is above the ULN or serum creatinine ≤ 15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. Percentage of participants with response at various time points were measured using Kaplan Meier method. (NCT02550652)
Timeframe: From Baseline to Week 52

,
Interventionpercentage of participants (Number)
Week 12Week 24Week 36Week 52
OBINUTUZUMAB 1000MG and MMF10263650
PLACEBO and MMF10283540

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Percentage of Participants With First Protocol Defined Overall Response Over the Course of 52 Weeks

OR includes both CRR and partial renal response(PRR). CRR as defined in the primary outcome measure above. PRR defined as 50% improvement in upcr, with one of these conditions met: 1. If baseline upcr is ≤3.0, then upcr of <1.0. 2. If baseline pcr is > 3.0, then upcr of <3.0, serum creatinine ≤15% above baseline value, and no urinary red cell casts and either RBCs/HPF ≤50% above baseline or <10 RBCs/HPF. Percentage of participants with response at various time points were measured using Kaplan Meier method. (NCT02550652)
Timeframe: From baseline to Week 52

,
Interventionpercentage of participants (Number)
Week 12Week 24Week 36Week 52
OBINUTUZUMAB 1000MG and MMF26576375
PLACEBO and MMF19415158

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Systemic Clearance of Obinutuzumab

(NCT02550652)
Timeframe: Day 0, Week 24, Week 52

InterventionL/day (Mean)
Day 0Week 24Week 52
OBINUTUZUMAB 1000MG and MMF0.2550.1470.137

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Terminal Plasma Half-Life (t1/2) of Obinutuzumab

(NCT02550652)
Timeframe: Day 0, Week 24, Week 52

Interventionday (Mean)
Day 0Week 24Week 52
OBINUTUZUMAB 1000MG and MMF13.120.522.1

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Volume of Distribution Under Steady State (Vss) of Obinutuzumab

(NCT02550652)
Timeframe: Day 0, Week 24, Week 52

InterventionL (Mean)
Day 0Week 24Week 52
OBINUTUZUMAB 1000MG and MMF3.673.673.67

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Non-relapse Mortality

(NCT01690520)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Standard of Care)16
Arm II (Experimental)16

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Overall Survival

(NCT01690520)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Standard of Care)52
Arm II (Experimental)57

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Proportion of Participants With Chronic Graft Versus Host Disease

(NCT01690520)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Standard of Care)27
Arm II (Experimental)23

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Proportion of Patients With Severe Acute Graft Versus Host Disease

(NCT01690520)
Timeframe: Up to 100 days post-transplant

Interventionproportion of participants (Number)
Arm I (Standard of Care)0.14
Arm II (Experimental)0.16

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Time to Neutrophil Engraftment

First of two consecutive days with absolute neutrophil count (ANC) equal to or greater than 500 cell/microliter measured from day of transplant. (NCT01690520)
Timeframe: Up to 55 days post-transplant

Interventiondays (Median)
Arm I (Standard of Care)20.0
Arm II (Experimental)22.0

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Time to Platelet Engraftment (20k)

First day of seven consecutive days with platelet count equal to or greater than 20,000 cells/microliter without platelet transfusions measured from day of transplant. (NCT01690520)
Timeframe: Up to 100 days post-transplant

Interventiondays (Median)
Arm I (Standard of Care)40.0
Arm II (Experimental)38.0

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GI Tolerability as Measured by GSRS

"Side-effects using the GSRS (Gastrointestinal Symptoms Rating Scale)~GSRS has 15 items, each rated on a 7-point scale from 1 (no discomfort) to 7 (very severe discomfort). GSRS total minimum value is 15; maximum value is 105. Higher scores represent greater discomfort." (NCT00574197)
Timeframe: Baseline and 6 months

Interventionscore on a scale (Mean)
GSRS at baselineGSRS at 6 months
Enteric-coated Mycophenolate Sodium (Myfortic)55.6734.17

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Allograft Survival Rate

Allograft survival is defined as participants who did not need to be re-transplanted or placed on dialysis due to the failure of their allograft transplantation during the course of this study. (NCT01517984)
Timeframe: 6 to 18 months post-randomization

Interventionparticipants (Number)
Randomized to Tacrolimus Withdrawal14
Randomized to Control Group7

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Incidence of Acute Rejection

Acute renal allograft rejection is defined as histological reading of borderline or greater determined by the local pathology laboratory. Participants suspected of having a rejection episode on the basis of clinical signs, symptoms, or on the basis of laboratory tests, had a renal ultrasound and underwent a renal transplant biopsy. Any detection of acute cellular rejection or acute humoral rejection resulted in participants in the 'Randomized to Tacrolimus Withdrawal' group to be restarted on tacrolimus and followed per the reduced follow-up schedule of events. (NCT01517984)
Timeframe: 6 to 18 months post-randomization

Interventionparticipants (Number)
Randomized to Tacrolimus Withdrawal6
Randomized to Control Group0

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Participant Survival Rate

Number of participants who did not die within the course of this study. (NCT01517984)
Timeframe: 6 to 18 months post-transplantation

Interventionparticipants (Number)
Randomized to Tacrolimus Withdrawal14
Randomized to Control Group7

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Percentage of Participants in the Experimental Arm Off Tacrolimus

Participants in the 'Randomized to Tacrolimus Withdrawal' group were considered fully withdrawn once they no longer received any doses of tacrolimus. Participants met this endpoint if they did not resume taking tacrolimus as of 18 months post randomization with stable allograft function and without rejection of donor-specific antibodies. (NCT01517984)
Timeframe: 18 months post-randomization

Interventionpercentage of participants (Number)
Randomized to Tacrolimus Withdrawal43

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Percentage of Participants With New Donor Specific Antibodies (DSAs)

Donor specific antibodies are antibodies that are directed against antigens expressed on donor organs. These antibodies can result in an immune attack on the transplanted organ, increasing risk of graft loss and/or rejection. (NCT01517984)
Timeframe: 6 to 18 months post-randomization

Interventionpercentage of participants (Number)
Randomized to Tacrolimus Withdrawal36
Randomized to Control Group14

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Estimated GFR Using the Chronic Kidney Disease Epidemiology (CKD-EPI) Equation

Estimated glomerular filtration rate (eGFR) is a test to measure the level of kidney function. In this measure, the effects of tacrolimus withdrawal on long-term kidney function was assessed by comparing absolute 24 month eGFR (18 months post-randomization) and change in eGFR from 6 to 24 months (randomization to 18 months randomization). Lower numbers indicate poorer kidney function (NCT01517984)
Timeframe: 6 months post-transplantation, 24 months post-transplantation

,
InterventionmL/min (Mean)
6 Month eGFR24 Month eGFRChange in eGFR from 6 to 24 months
Randomized to Control Group62.368.66.3
Randomized to Tacrolimus Withdrawal56.261.75.5

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Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 100 Days

(NCT00352976)
Timeframe: by 100 days

Interventionmg/dL (Mean)
Treatment With TBI88.0

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Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 180 Days

(NCT00352976)
Timeframe: by 180 days

Interventionmg/dL (Mean)
Treatment With TBI98.7

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Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 365 Days

(NCT00352976)
Timeframe: by 365 days

Interventionmg/dL (Mean)
Treatment With TBI107.2

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Average IgG Levels as a Measure of Immune Reconstitution After Transplant by 365 Days

(NCT00352976)
Timeframe: by 365 days

Interventionmg/dL (Mean)
Treatment With TBI724.0

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Average IgG Levels as a Measure of Immune Reconstitution After Transplant, by 180 Days

(NCT00352976)
Timeframe: by 180 days

Interventionmg/dL (Mean)
Treatment With TBI652.9

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Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 100 Days

(NCT00352976)
Timeframe: by 100 days

Interventionmg/dL (Mean)
Treatment With TBI71.1

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Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 180 Days

(NCT00352976)
Timeframe: by 180 days

Interventionmg/dL (Mean)
Treatment With TBI77.0

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Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 365 Days

(NCT00352976)
Timeframe: by 365 days

Interventionmg/dL (Mean)
Treatment With TBI82.8

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Average Immunoglobulin G (IgG) Levels as a Measure of Immune Reconstitution After Transplant, by 100 Days

(NCT00352976)
Timeframe: by 100 days

Interventionmg/dL (Mean)
Treatment With TBI550.6

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Number of Participant With Neutrophil Recovery

Number of participant with neutrophil recovery. Neutrophil recovery is defined as absolute neutrophil count ≥500/µL for three consecutive days (NCT00352976)
Timeframe: by day 42

InterventionParticipants (Count of Participants)
Treatment With TBI78

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Number of Participants Experiencing Acute Graft-versus-host Disease (GVHD)

Number of participants experiencing acute GVHD (all grades) by day 100 (NCT00352976)
Timeframe: at 100 days

InterventionParticipants (Count of Participants)
Treatment With TBI15

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Number of Participants Experiencing Chronic GVHD

Number of participants experiencing chronic Graft Vs Host Disease by 1 year (NCT00352976)
Timeframe: at one year

InterventionParticipants (Count of Participants)
Treatment With TBI6

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Number of Participants Experiencing Infections by Day 100

(NCT00352976)
Timeframe: by day 100

InterventionParticipants (Count of Participants)
Treatment With TBI53

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Number of Participants Experiencing Infections by Day 180

(NCT00352976)
Timeframe: by day 180

InterventionParticipants (Count of Participants)
Treatment With TBI55

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Number of Participants Experiencing Infections by Day 365

(NCT00352976)
Timeframe: by day 365

InterventionParticipants (Count of Participants)
Treatment With TBI56

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Number of Participants Experiencing Overall Survival

Number of participants experiencing overall survival by 1 year (NCT00352976)
Timeframe: at one year

InterventionParticipants (Count of Participants)
Treatment With TBI70

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Number of Participants With Secondary Graft Failure at 100 Days

Secondary Graft Rejection by day 100 (NCT00352976)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Treatment With TBI4

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Number of Participants With BK Viral Load <600 Copies/mL

A Viral load of <600 copies/mL for at least 3 months indicates sustained clearance of BK viremia, confirmed by blood test (NCT01649609)
Timeframe: Up to 12 months from enrollment

InterventionParticipants (Count of Participants)
Standard Immunosuppression Reduction Arm13
mTOR Substitution Arm17

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Number of Participants With Incidence of BK Nephropathy

The number of people with incidence of BK Nephropathy in each of the two Arms (NCT01649609)
Timeframe: Up to 24 months from randomization

InterventionParticipants (Count of Participants)
Standard Immunosuppression Reduction Arm3
mTOR Substitution Arm1

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Count of Participants Who Achieved Neutrophil Engraftment

Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm^3 (0.5 x 10^9/L) or greater. (NCT01685411)
Timeframe: By Day 42

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant5

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Count of Participants With Disease Free Survival

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 5 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant1

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Count of Participants With Disease Free Survival

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 7 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant1

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Counts of Participants With Disease Free Survival

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 2 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant3

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Number of Participant Who Were Alive at 2 Years Post Transplant

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 2 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant4

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Number of Participant Who Were Alive at 5 Years Post Transplant

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 5 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant3

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Number of Participant Who Were Alive at 7 Years Post Transplant

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 7 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant1

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Percentage of Participants With Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: 6 Months

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant0

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Percentage of Participants With Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: 1 Year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant0

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Percentage of Participants With Engraftment Failure

Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets. (NCT01685411)
Timeframe: Day 42

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant0

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Percentage of Participants With Relapse

The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 1 Year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant40

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Percentage of Participants With Relapse

The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 2 Years

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant40

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Percentage of Participants With Acute Graft-Versus-Host Disease by Grade

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Grade 2-4Grade 3-4
Allogeneic Hematopoietic Stem Cell Transplant4020

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Number of Participants With Sustained Cell Engraftment of Donor Cells

Sustained stem cell engraftment of donor cells will be evaluated by chimerism (FISH fluorescence in situ hybridization OR VNTR (Variable Number of Tandem Repeats), based on recipient/donor gender, at 30 days, 100 days, 6 months and 1 year following the use of reduced intensity conditioning. (NCT02435901)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Reduced Intensity Regimen29

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Event Free Survival; Number of Participants Who Survived at 2 Years

29 participants will be evaluated for Event Free Survival. (NCT02435901)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Expired Secondary to SepsisExpired Secondary to GVHDSurvived Participants
Reduced Intensity Regimen1226

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Dose-normalized AUC of Mycophenolic Acid

"Bioavailability (12h AUC) of mycophenolic acid in renal transplant patients after administration of MMF+/-PAN and EC-MPS+/-PAN~For evaluation of pharmacokinetic and pharmacodynamic parameters blood will be collected before, 0.5h, 1h, 1.5h, 2h, 3h, 4h, 5h, 6h, 8h, 10h, 12h after drug intake." (NCT01801280)
Timeframe: Study duration for each patient: 2 months. After 10-14 days of drug intake blood samples for PK/PD analysis will be collected. On the next day new treatment starts. There are 4 study visits at the study center. Duration will be approximately 12hours

Interventionmg*h/L (Mean)
Mycophenolate Mofetil (MMF)41
MMF+PAN38
EC-MPS43
EC-MPS + PAN46

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Collection of Blood for Future Analysis of Peripheral Blood Mononuclear Cells (PBMCs)

Blood was drawn and banked for future use in order to characterize immune system markers and further the understanding of the immune factors that contribute to disease progression in ALS. (NCT01884571)
Timeframe: Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6), Post-Treatment Period (Months 8 and 12)

InterventionParticipants (Count of Participants)
Immunosuppression Regimen31

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Collection of Cerebrospinal Fluid for Future Analysis of Cytokine Levels

Lumbar punctures (LPs) were performed to collect cerebrospinal fluid (CSF). CSF is banked for future use to characterize immune system markers and to further the understanding of the immune factors that contribute to disease progression in ALS. Cytokines are markers of neuroinflammation and can be categorized as neurotoxic or neuroprotective. The role that cytokines play in in ALS progression is still not yet fully understood. (NCT01884571)
Timeframe: Pre-Treatment Period (two months prior to the start of treatment), Treatment Period (Months 2 and 6), Post-Treatment Period (Month 12)

InterventionParticipants (Count of Participants)
Immunosuppression Regimen29

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Mean Rate of Change in Grip Strength Treatment Compared to Pre-Treatment

Hand grip was measured using a study approved dynamometer to test the maximum isometric strength of the hand and forearm muscles. The grip strength of the left and right hands were analyzed together. Grip strength was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. Grip strength is a measurement of muscle strength and declines as ALS progresses. A positive value means that scores during treatment were higher than pre-treatment scores, indicating an increase in grip strength over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)

Interventionpounds change per month (Mean)
Immunosuppression Regimen1.38

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Mean Rate of Change of ALSFRS-R Scores During Treatment Compared to Pre-Treatment

The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability). ALSFRS-R was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in ability over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)

Interventionunits on a scale change per month (Mean)
Immunosuppression Regimen-0.24

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Mean Rate of Change of Hand-Held Dynamometry (HHD) During Treatment Compared to Pre-Treatment

Hand held dynamometry (HHD) is a measure of muscle strength and scores decrease as ALS progresses. Six proximal muscle groups were examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group are established from the initial values for each participant. Strength determinations were converted to Z scores and averaged to provide an HHD megascore. The Z-score indicates the number of standard deviations away from the mean of 0. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. HHD was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in strength over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)

Interventionz-score change per month (Mean)
Immunosuppression Regimen-0.05

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Mean Rate of Change of Slow Vital Capacity (SVC) During Treatment Compared to Pre-Treatment

Vital capacity (VC), percent of predicted normal, was determined using the slow VC method. SVC measures the amount of air exhaled following a deep breath. For this test, participants hold a mouthpiece in their mouth, breathe in deeply, and breathe out as much air as they can. The test was done seated in a chair and then repeated while lying on an exam table at the Screening Visit. For all other visits, this test was done while seated in a chair. This test takes 15-20 minutes. SVC was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. SVC is a way to measure respiratory insufficiency in persons with ALS and SVC decreases as ALS progresses. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)

Interventionpercent predicted change per month (Mean)
Immunosuppression Regimen-0.18

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Mean Rate of Change of T-cell Subsets in Blood Treatment Compared to Pre-Treatment

Blood was collected for ribonucleic acid (RNA) and the mean rate of decline of T-cells during the 6 month treatment period compared to the pre-treatment period was assessed (blood was collected twice during the 3-month long lead in period). The precise role that T-cells have in ALS is unknown and this study aims to further the understanding of how T-cells operate in persons with ALS. T-cell measurement is a ratio where the relative expression levels of FOXP3 messenger ribonucleic acid (mRNA) was calculated using the Comparative CT Method (ΔΔCT Method), normalizing to β-actin. Samples were obtained during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in T-cells over time. (NCT01884571)
Timeframe: Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6)

Interventionfold change per month (Mean)
Immunosuppression Regimen-0.04

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Number of Participants With an Average Increase in ALSFRS-R Score of One Point Per Month

The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score (best of 48) from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). A clinical response is defined as a rate of change of ALSFRS-R of +6 points over 6 months (mean of +1 point per month), where typically patients with ALS have a decline in ALSFRS-R by an average of -1/month. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)

InterventionParticipants (Count of Participants)
Immunosuppression Regimen0

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Number of Participants in Overall Survival.

(NCT01487577)
Timeframe: 1 year

Interventionparticipants (Number)
Mycophenolate Mofetil15

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Number of Participants Who Experienced Nonrelapse Mortality.

(NCT01487577)
Timeframe: 1 year

Interventionparticipants (Number)
Mycophenolate Mofetil0

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Number of Participants Who Experienced Relapse.

(NCT01487577)
Timeframe: 1 year

Interventionparticipants (Number)
Mycophenolate Mofetil4

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Number of Participants With Grade ≥3 Toxicities Scored According to the CTCAE Version 4.0.

(NCT01487577)
Timeframe: 100 days

Interventionparticipants (Number)
Mycophenolate Mofetil0

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Number of Participants With Acute Grade II-IV GVHD, Acute Grade III-IV GVHD, and Chronic GVHD.

Acute GVHD will be graded according to the Modified Glucksberg Staging Criteria. Chronic GVHD will be graded according to NIH Chronic GVHD Consensus Guidelines. (NCT01487577)
Timeframe: 1 year

Interventionparticipants (Number)
Acute grade II-IV GVHDAcute grade III-IV GVHDChronic GVHD
Mycophenolate Mofetil642

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Number of Participants With Neutrophil and Platelet Engraftment.

Neutrophil and platelet engraftment definitions as defined by the CIBMTR Data Management Manual. (NCT01487577)
Timeframe: 100 days

Interventionparticipants (Number)
Incidence of neutrophil engraftmentIncidence of platelet engraftment
Mycophenolate Mofetil1815

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Pharmacokinetic Analysis of MMF AUC to Evaluate MMF Dose Relationships to Drug Exposure.

Pharmacokinetic analysis includes, but is not limited to, area under the plasma concentration versus time curve (AUC). (NCT01487577)
Timeframe: 100 days

Interventionmcg*hr/mL (Median)
AUC, Intermittent IV dosingAUC, Continuous infusionAUC, Intermittent PO dosing
Mycophenolate Mofetil46.540.136.1

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Pharmacokinetic Analysis of MMF Clearance to Evaluate MMF Dose Relationships to Drug Exposure.

Pharmacokinetic analysis includes, but is not limited to, clearance. (NCT01487577)
Timeframe: 100 days

InterventionmL/min/kg (Median)
Clearance, Intermittent IV dosingClearance, Continuous infusionClearance, Intermittent PO dosing
Mycophenolate Mofetil10.215.110.9

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Pharmacokinetic Analysis of MMF Steady State Concentration to Evaluate MMF Dose Relationships to Drug Exposure.

Pharmacokinetic analysis includes, but is not limited to, steady-state concentrations. (NCT01487577)
Timeframe: 100 days

Interventionmcg/mL (Median)
Steady-state concentration, Intermittent IV dosingSteady-state concentration, Continuous infusionSteady-state concentration, Intermittent PO dosing
Mycophenolate Mofetil1.91.71.5

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Overall Survival

Number of patients surviving 18 months post-transplant. (NCT00060424)
Timeframe: At 18 months

InterventionParticipants (Count of Participants)
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)15

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Rate of Relapse

Number of patients with relapsed disease post-transplant. Relapse/progression is defined as 1) Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, 2) circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, or 3) lymph node Biopsy Richter's transformation. (NCT00060424)
Timeframe: 18 months

InterventionParticipants (Count of Participants)
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)8

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Acute Grade II-IV GVHD and Chronic (Extensive) GVHD

"Number of patients who developed acute/chronic GVHD post-transplant. aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 3 skin and/or stage 1 gut involvement and/or stage 1 liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00060424)
Timeframe: aGVHD: 100 days after transplant; cGVHD: 1 Year after transplant.

InterventionParticipants (Count of Participants)
Acute GVHDChronic extensive GVHD
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)1010

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Rate and Types of Infections

Number of infections patients experienced, by infection type. (NCT00060424)
Timeframe: 18 months

Interventioninfections (Number)
ViralFungalFever of unknown originBacterialOther
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)27136535

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Duration of Renal Response in Participants Who Achieve Partial or Complete Renal Response at Any Time During the Study

Number of days between first visit with response to last consecutive visit with partial or complete response. Complete renal response is defined as: (1) uPCR <0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline) (from a 24 hour urine collection); and (2) eGFR within normal range. Partial renal response is defined as: (1) ≥ 50% reduction in uPCR from Day 1 (Baseline; from a 24-hour urine collection) and, (2) with one of the following: (a) uPCR of < 1.0 mg/mg if the Day 1 (Baseline) was ≤ 3.0 mg/mg, or, (b) uPCR < 3.0 mg/mg if the Day 1 (Baseline) ratio was > 3.0 mg/mg; and stabilization of renal function (eGFR + or - 25% of Day 1 [Baseline] or serum creatinine within normal range). Estimated from the Kaplan-Meier Curve. (NCT01499355)
Timeframe: up to Week 52

Interventiondays (Mean)
Placebo48.3
BIIB023 3 mg/kg45.6
BIIB023 20 mg/kg52.1

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Percentage of Participants Who Achieve a Complete or Partial Renal Response at Week 52

Complete renal response is defined as: (1) urinary protein:creatinine ratio (uPCR) < 0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline; from a 24 hour urine collection); and (2) estimated glomerular filtration rate (eGFR) within normal range. Partial renal response is defined as: (1) ≥ 50% reduction in uPCR from Day 1 (Baseline; from a 24-hour urine collection) and, (2) with one of the following: (a) uPCR of < 1.0 mg/mg if the Day 1 (Baseline) was ≤ 3.0 mg/mg, or, (b) uPCR < 3.0 mg/mg if the Day 1 (Baseline) ratio was > 3.0 mg/mg; and stabilization of renal function (eGFR + or - 25% of Day 1 [Baseline] or serum creatinine within normal range). (NCT01499355)
Timeframe: Week 52

Interventionpercentage of participants (Number)
Placebo25
BIIB023 3 mg/kg16
BIIB023 20 mg/kg31

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Percentage of Participants Who Achieve Complete Renal Response at Week 52

Complete renal response is defined as uPCR < 0.5 mg/mg with ≥ 50% reduction of uPCR from Baseline (from a 24-hour urine collection) and eGFR within normal range. (NCT01499355)
Timeframe: Week 52

Interventionpercentage of participants (Number)
Placebo6
BIIB023 3 mg/kg8
BIIB023 20 mg/kg8

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Percentage of Participants With Active Urinary Sediment at Baseline Who Have Inactive Urinary Sediment at Week 52

Active urinary sediment is defined by 1 of the following (in the absence of a urinary tract infection or menses): > 5 red blood cell/high power field (RBC/HPF) or above the reference range for the laboratory, and > 5 white blood cell/high power field (WBC/HPF) or above the reference range for the laboratory, and presence of cellular casts (RBC or WBC). Inactive urinary sediment is defined as: < 5 RBC/HPF and < 5 WBC/HPF, or within the laboratory reference range, and no cellular casts (no RBC or WBC casts). (NCT01499355)
Timeframe: Baseline, Week 52

Interventionpercentage of participants (Number)
Placebo38
BIIB023 3 mg/kg5
BIIB023 20 mg/kg21

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Percentage of Participants With uPCR > 3.0 mg/mg at Baseline Who Achieve uPCR <1.0 mg/mg at Week 52

(NCT01499355)
Timeframe: Baseline (Day 1), Week 52

Interventionpercentage of participants (Number)
Placebo0
BIIB023 3 mg/kg22
BIIB023 20 mg/kg13

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Time to Renal Response (Partial or Complete) in Participants Who Achieve Renal Response at Week 52

Onset of renal response was calculated as weeks elapsed from baseline date to first visit where renal response was achieved. Complete renal response is defined as: (1) uPCR <0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline) (from a 24 hour urine collection); and (2) eGFR within normal range. Partial renal response is defined as: (1) ≥ 50% reduction in uPCR from Day 1 (Baseline; from a 24-hour urine collection) and, (2) with one of the following: (a) uPCR of < 1.0 mg/mg if the Day 1 (Baseline) was ≤ 3.0 mg/mg, or, (b) uPCR < 3.0 mg/mg if the Day 1 (Baseline) ratio was > 3.0 mg/mg; and stabilization of renal function (eGFR + or - 25% of Day 1 [Baseline] or serum creatinine within normal range). Estimated from the Kaplan-Meier Curve. (NCT01499355)
Timeframe: Baseline to Week 52

Interventionweeks (Median)
Placebo10.6
BIIB023 3 mg/kg5.2
BIIB023 20 mg/kg4.1

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Duration of Renal Response in Participants Who Achieve Complete Renal Response at Week 52

Duration of response was calculated as the days in between the date of Week 52 visit and the date when the participant last became complete renal responder on or before Week 52 visit. Complete renal response: (1) uPCR <0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline) (from a 24 hour urine collection); and (2) eGFR within normal range. (NCT01499355)
Timeframe: Week 52

,,
InterventionParticipants (Count of Participants)
1-day duration27-day duration78-day duration141-day duration169-day duration
BIIB023 20 mg/kg21001
BIIB023 3 mg/kg30100
Placebo20010

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Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Discontinuation During the Run-In Period

AEs that had an onset on or after dosing of MMF on run-in Day 1 up to the first double-blind dose, or any pre-existing condition that worsened. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed above. (NCT01499355)
Timeframe: Day 1 to Week 12

Interventionparticipants (Number)
Any EventModerate or Severe EventSevere EventRelated Event to MMFSerious EventRelated Serious Event to MMFFatal EventDiscontinued Treatment Due to EventWithdrew From Study Due to Event
Run-In: All Enrolled Participants20994189028122010

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Number of Participants With AEs, SAEs and AEs Leading to Study Discontinuation During the Double-Blind Period

AEs that had an onset on or after dosing of BIIB023 or placebo, or any pre-existing condition that worsened. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed above. (NCT01499355)
Timeframe: Week 12 to Week 56

,,
Interventionparticipants (Number)
Any eventModerate or severe eventSevere eventEvent related to double-blind treatmentEvent related to MMFSerious eventSerious event related to double-blind treatmentSerious event related to MMFFatal event
BIIB023 20 mg/kg53225112210230
BIIB023 3 mg/kg60326152411340
Placebo482645217361

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Acute Graft-versus-Host Disease (GVHD) Grade 2-4 Risk From Time of Transplant Until Day 90 Post-transplant

GVHD grading system goes from 0-4 where grade 4 is the most severe. Grade 0 and 1 do not require systemic treatment, Grade 2-4 require treatment. This trial evaluated the risk of developing acute GVHD grades 2-4 within 90 days of transplant. (NCT00185692)
Timeframe: 90 days

InterventionParticipants (Count of Participants)
Transplantation of CD34+ Cells1

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Engraftment of Haploidentical CD34+ Selected Blood Stem Cells in Older Patients or Those With Medical Co-morbidities Following Total Lymphoid Irradiation and Antithymocyte Globulin Transplant Conditioning

number achieving donor cell engraftment (>95%) by day 90 after transplant. (NCT00185692)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Transplantation of CD34+ Cells4

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Evaluate the Risk for Disease Progression and Relapse

Percentage patients who relapsed/progressed within 1 year post-transplant. (NCT00040846)
Timeframe: 1 year after transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)21.7

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Evaluate the Risk/Incidence of Infections

Percentage patients who experienced infections within 100 days post-transplant. (NCT00040846)
Timeframe: 100 days after transplant

Interventionpercentage of participants (Number)
Dose Level 1 (No Campath)91.7

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Determine Whether Engraftment Can be Maintained With a Single Dose Fludarabine, DLI and Continued MMF/CSP, Defined as Rejection Rate < 20%.

Mixed chimerism will be defined as the detection of donor T cells (CD3+) and granulocytes (CD 33+), as a proportion of the total T cell and granulocyte population, respectively, of greater than 5% and less than 95% in the peripheral blood. Full donor chimerism is defined as > 95% donor CD3+ T cells. (NCT00040846)
Timeframe: 100 days after transplant

Interventionpercentage of participants (Number)
CD3 - Graft rejectionCD3 - Mixed chimerismCD3 - Full donor chimerismCD3 - UnknownCD33 - Graft RejectionCD33 - Mixed chimerismCD33 - Full donor chimerismCD33 - Unknown
Dose Level 1 (No Campath)3.318.3708.331.73.38015

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Evaluate the Risk of Occurrence of Acute and Chronic GVHD

"Percentage patients who developed acute/chronic GVHD.~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade III: Stage 2 - 4 gastrointestinal involvement and/or +2 to +4 liver involvement, with or without a rash Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00040846)
Timeframe: 1 year after transplant

Interventionpercentage of participants (Number)
Grade III-IV aGVHDcGVHD
Dose Level 1 (No Campath)23.341.7

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Donor Engraftment (Chimerism)

Defined by the detection of at least 50% donor derived T-cells (CD3+), as a proportion of the total T-cell population (NCT00049504)
Timeframe: At day +84 after transplantation

InterventionParticipants (Count of Participants)
Treatment (Nonmyeloablative HSCT)34

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Incidence of Grades III-IV Acute GVHD

Grade III GVHD represents moderate severity. Grade IV GVHD represents extreme severity (NCT00049504)
Timeframe: At any time within 200 days after transplantation

InterventionParticipants (Count of Participants)
Treatment (Nonmyeloablative HSCT)4

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Response Rates

(NCT00119392)
Timeframe: Up to 8 years

InterventionParticipants (Count of Participants)
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant)25

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Engraftment and Hematopoietic Toxicity

Median number of days after transplantation to a neutrophil count less than 500 neutrophils per microliter and a platelet count less than 50,000 platelets per microliter. (NCT00119392)
Timeframe: At day +100

Interventiondays (Median)
NeutrophilsPlatelets
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant)1711

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Incidence and Severity of Acute Graft-versus-host Disease (GVHD) and Chronic GVHD.

(NCT00119392)
Timeframe: At day +84

InterventionParticipants (Count of Participants)
Acute GVHD: Grade 1-2Acute GVHD: Grade 3Chronic extensive GVHD
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant)2745

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Overall and Progression-free Survival

Kaplan-Meier estimates for overall survival (OS) and progression free survival (PFS) assessed at two years. (NCT00119392)
Timeframe: Up to 8 years

Interventionpercent (Number)
Overall survivalProgression free survival
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant)5431

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Mean Percent Change in Calculated Creatinine Clearance From Baseline to Months 6, 12, and 24

"Renal allograft function determined by mean percent change from baseline in calculated creatinine clearance (Cockroft and Gault method) by treatment group at 6, 12, and 24 months postrandomization.~percent change= [(calculated creatinine clearance at Month t - calculated creatinine clearance at baseline)/calculated creatinine clearance at baseline]*100 percent, where t=6, 12, and 24 months postrandomization" (NCT00121810)
Timeframe: baseline 6, 12, and 24 months

,
Interventionpercent change (Mean)
BaselinePercent Change from Baseline at Month 6Percent Change from Baseline at Month 12Percent Change from Baseline at Month 24
Mycophenolate Mofetil + Cyclosporine or Tacrolimus60.5-1.7-2.3-4.2
Mycophenolate Mofetil + Sirolimus59.77.04.44.7

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Mean Percent Change in Calculated Glomerular Filtration Rate From Baseline to Months 6, 12, and 24 (Nankivell Equation)

"Renal allograft function determined by mean percent change from baseline in calculated Glomerular Filtration Rate (Nankivell equation) by treatment group at 6, 12, and 24 months postrandomization.~percent change= [(calculated Glomerular Filtration Rate at Month t - calculated Glomerular Filtration Rate at baseline)/calculated Glomerular Filtration Rate at baseline]*100 percent, where t=6, 12, and 24 months postrandomization." (NCT00121810)
Timeframe: baseline, 6, 12, and 24 months

,
Interventionpercent change (Mean)
BaselinePercent Change from Baseline at Month 6Percent Change from Baseline at Month 12Percent Change from Baseline at Month 24
Mycophenolate Mofetil + Cyclosporine or Tacrolimus72.7-0.5-0.9-1.8
Mycophenolate Mofetil + Sirolimus71.38.35.26.5

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Mean Percent Change in Glomerular Filtration Rate From Baseline to Month 12

"The primary efficacy endpoint was mean percent change in renal function from baseline to 12 months postrandomization, as measured by Glomerular Filtration Rate utilizing renal clearance of cold iothalamate.~percent change= [(Glomerular Filtration Rate at Month 12-Glomerular Filtration Rate at baseline)/Glomerular Filtration Rate at baseline]*100 percent." (NCT00121810)
Timeframe: baseline to 12 months

,
Interventionpercent change (Mean)
BaselineMean Percent Change from Baseline at Month 12
Mycophenolate Mofetil + Cyclosporine or Tacrolimus58.85.2
Mycophenolate Mofetil + Sirolimus59.524.4

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Mean Percent Change in Glomerular Filtration Rate From Baseline to Month 24

"A secondary efficacy endpoint was mean percent change in renal function from baseline to 24 months postrandomization, as measured by Glomerular Filtration Rate utilizing renal clearance of cold iothalamate.~percent change= [(Glomerular Filtration Rate at Month 24-Glomerular Filtration Rate at baseline)/Glomerular Filtration Rate at baseline]*100 percent." (NCT00121810)
Timeframe: Baseline to 24 months

,
Interventionpercent change (Mean)
BaselinePercent Change from Baseline at Month 24
Mycophenolate Mofetil + Cyclosporine or Tacrolimus58.83.4
Mycophenolate Mofetil + Sirolimus59.58.6

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Mean Percent Change in Serum Creatinine From Baseline to Months 6, 12, and 24

"Renal allograft function determined by mean percent change from baseline in serum creatinine by treatment group at 6, 12, and 24 months postrandomization.~percent change= [(serum creatinine at Month t-serum creatinine at baseline)/serum creatinine at baseline]*100 percent, where t=6, 12, and 24 months postrandomization." (NCT00121810)
Timeframe: baseline, 6, 12, and 24 months

,
Interventionpercent change (Mean)
BaselinePercent Change from Baseline at Month 6Percent Change from Baseline at Month 12Percent Change from Baseline at Month 24
Mycophenolate Mofetil + Cyclosporine or Tacrolimus124.46.620.430.8
Mycophenolate Mofetil + Sirolimus121.1-3.76.06.1

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Overall Survival

measured from date of registration to study until death from any cause with patients still alive censored at date of last contact (NCT00053014)
Timeframe: 1 year

Interventionparticipants (Number)
Treatment1

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Serious Adverse Events

Twice a week for the first two months, one time a week during month 3, one time every two weeks for months 4-9. (NCT00053014)
Timeframe: 9 months

Interventionparticipants (Number)
Treatment0

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Count of Participants Diagnosed With Epstein-Barr Virus (EBV) Infection as an Adverse Event

Viral infections following renal transplantation is a significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during this study using participant blood samples. Displayed are counts of all participants diagnosed with EBV infection as an adverse event by EBV test(s), diagnosed by test results from the local clinical pathology laboratory. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational0
Control0

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Count of Participants Diagnosed With Malignancy as an Adverse Event

An increased risk/incidence of malignancy is a recognized complication of immunosuppression in recipients of organ transplants. In Phase 3 clinical trials, overall malignancy rates were similar across all treatment groups, with the exception of posttransplant lymphoproliferative disease (PTLD).Displayed are counts of all participants who experienced malignancy reported as an adverse event. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational0
Control0

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Count of Participants With an Infectious Disease Serious Adverse Event(s) Requiring Hospitalization or Systemic Therapy

Infections were required to be reported as a serious adverse event if they required either inpatient hospitalization or prolongation of a current hospitalization. Displayed are counts of all participants who experienced infection(s) as an adverse event, by treatment group. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational11
Control11

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Count of Participants With Defined CKD Stage 4 or 5 at Wk 52 Post-Transplant

"The stages of Chronic Kidney Disease (CKD) are defined using the participant's GFR value:~Stage 1 if GFR value is ≥ 90 (kidney function is normal)~Stage 2 if 60 ≤ GFR < 90 (mildly reduced kidney function, pointing to kidney disease)~Stage 3A if 45 <= GFR < 60*~Stage 3B if 30 <= GFR < 45*~Stage 4 if 15 ≤ GFR < 30 (severely reduced kidney function)~Stage 5 if GFR < 15 (severe or end stage kidney failure).~Stages 3A abd 3B indicate moderately reduced kidney function.*" (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational0
Control0

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Count of Participants With Delayed Graft Function at Wk 52 Post-Transplant

Delayed grafted function is defined as dialysis in the first week on one or more occasions for any indication other than the treatment of acute hyperkalemia in the setting of otherwise acceptable renal function. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational1
Control1

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Count of Participants With eGFR < 60 mL/Min/1.73 m^2 Measured by CKD-EPI at Wk 52 Post-Transplant

"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational5
Control5

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Count of Participants With Evidence of Pancreatic Loss at Week 52 Post-Transplant

C-peptide is a measure of pancreatic function. The definition of pancreatic loss: a C-peptide value of <0.3 ng/mL. (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational1
Control0

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Count of Participants With Evidence of Partial Pancreatic Graft Function at Week 52 Post-Transplant

C-peptide is a measure of pancreatic function. The definition of partial pancreatic graft function: a fasting C-peptide levels >0.3ng.mL (0.1nmol.L) plus the participant's continued requirement for exogenous insulin or oral hypoglycemic agent(s). (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational1
Control0

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Count of Participants With Full Pancreatic Graft Function (Insulin Independent) at Wk 52 Post-Transplant

Participants with full pancreatic graft functions are defined as those that no longer require exogenous insulin therapy. (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational19
Control21

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Count of Participants With Successful Discontinuation of Tacrolimus in Recipients Randomized to the Investigational Arm

Participants achieved successful discontinuation if they were able to discontinue (e.g., off tacrolimus therapy completely) over a 4-8 weeks after tacrolimus withdrawal was initiated at week 40. (NCT01790594)
Timeframe: Week 40 through week 48 Post-Transplant

InterventionParticipants (Count of Participants)
Investigational5

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Mean Calculated eGFR Using MDRD 4 Variable Model at Wk 52 Post-Transplant

"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease equation (MDRD):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate severe or endstage kidney failure." (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionmL/min/1.73m^2 (Mean)
Investigational68.7
Control67.0

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Mean Estimated Glomerular Filtration Rate (eGFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52 Post-Transplant

"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01790594)
Timeframe: Week 52 Post-Transplant

InterventionmL/min/1.73m^2 (Mean)
Investigational77.0
Control74.6

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The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine Post-Transplant

"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or endstage kidney failure.~An estimate of the slope, or change over time, in eGFR was produced using standard statistical linear modeling procedures. The estimate was then re-scaled so that it could be interpreted as a change in eGFR per month. Positive numbers indicate increasing kidney function.~Larger numbers indicate greater change in kidney function." (NCT01790594)
Timeframe: Day 28 through Week 52 Post-Transplant

InterventioneGFR change over time (by month) (Mean)
Investigational0.1
Control-0.1

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Count of Participant Diagnosed With BK Polyoma Virus (BKV) and Cytomegalovirus (CMV) Viremia As Adverse Events

"Viral infections following renal transplantation is a significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss.~Specific viruses were monitored during this study using participant blood samples. Displayed are counts of participants who experienced BKV and CMV viremia as adverse events, diagnosed by test results from the local clinical pathology laboratory." (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant

,
InterventionCount of Participants (Number)
BK ViremiaCMV Viremia
Control33
Investigational85

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Count of Participants With Acute Rejection (AR) of Kidney or Pancreatic Transplant During the First 52 Wks Post-Transplant

"Biopsy-proven acute rejection (AR) of the kidney (renal) or pancreas during the first 52 weeks post-transplant. AR grading using standard Banff* criteria. For both kidney and pancreas, AR is defined as a grade ≥1.~AR for the kidney: Banff 2007 criteria. Severity of AR is graded by as IA, IB, IIA, IIB, or III, with IA defined as the mildest form of AR and III being the most severe.~AR for the pancreas: Banff 2011 Criteria. Severity of AR is graded as I, II, or III, with I defined as the mildest form of AR and III being the most severe." (NCT01790594)
Timeframe: Transplant through Week 52

,
InterventionCount of Participants (Number)
KidneyPancreas
Control21
Investigational25

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Count of Participants With Biopsy-Proven Humoral Rejection During the First 52 Weeks Post-Transplant

"Humoral rejection (i.e., antibody mediated rejection) of:~the kidney as defined by diffusely positive staining for C4d, presence of circulating anti-donor antibodies, and morphologic evidence of acute tissue injury determined by local pathology and,~the pancreas as defined by the presence of circulating anti-donor antibodies, and histopathological data including morphologic evidence of microvascular tissue injury and C4d staining in interacinar capillaries determined by local pathology." (NCT01790594)
Timeframe: Transplant through Week 52

,
InterventionCount of Participants (Number)
KidneyPancreas
Control00
Investigational00

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Count of Participants With De Novo Anti-Donor Antibodies or Anti-Human Leukocyte Antigen (HLA) Antibodies During the First 52 Weeks Post-Transplant

"The de novo development of donor-specific antibody (DSA) is associated with an increased risk of graft rejection.~The presence of anti-Histocompatibility Antigen (HLA) antibodies (alloantibodies) is associated with increased risk of acute and chronic injury to the transplant allograft." (NCT01790594)
Timeframe: Transplant through Week 52

,
InterventionCount of Participants (Number)
De novo DSAAnti-HLA
Control01
Investigational02

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Count of Participants With Event of Death, Graft Loss, or Undetectable C-peptide

"This measure counts death, graft loss, or undetectable C-peptide value (e.g., C-peptide <0.3 ng/mL) occurring at any point post-transplant and independent of each other.~Kidney Graft Loss was defined as 90 consecutive days of dialysis dependency.~Pancreas graft loss was defined as returning to exogenous insulin therapy or initiation of oral hypoglycemic agents for greater than 30 days.~Factitious hypoglycemia due to surreptitious insulin administration results in elevated serum insulin levels and low or undetectable C-peptide levels." (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant

,
InterventionParticipants (Count of Participants)
DeathKidney Graft LossPancreas Graft LossUndetectable C-peptide
Control0000
Investigational1010

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Count of Participants With the Occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

"Adverse events were collected systematically. Counts of all participants who experienced at least one adverse event (AEs, SAEs) by assigned treatment group.~Refer to the Serious Adverse Events and Other Adverse Events tables for more detail." (NCT01790594)
Timeframe: From Enrollment (Pre-Transplant) to Week 52 Post-Transplant

,
InterventionCount of Participants (Number)
All Adverse EventsSerious Adverse Events
Control2119
Investigational2220

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Count of Participants With Use of Anti-hypertensive Medication From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant

Anti-hypertensive medications are a class of drugs that are used to treat hypertension. The medications seek to prevent the complications of high blood pressure, such as stoke and myocardial infarction. (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52

,
InterventionCount of Participants (Number)
BaselineDay 28Day 84Week 28Week 36Week 52
Control19107111111
Investigational181411131313

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Count of Participants With Use of Lipid Lowering Medications at Baseline, Wk 28 and Wk 52 Post-Transplant

Lipid lowering medications are used in the treatment of high levels of fats (lipids), such as cholesterol in blood (NCT01790594)
Timeframe: Baseline (Pre-Transplant), Week 28, and Week 52

,
Interventionparticipants (Number)
BaselineWeek 28Week 52
Control181616
Investigational181919

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Fasting Blood Sugar (FBS) From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant

"Fasting blood sugar (e.g., glucose) test is used to help diagnose diabetes, prediabetes, and gestational diabetes.~Reference fasting blood sugar (glucose) values:~70 to 99 mg/dL is normal~100 to 125 mg/dL is considered prediabetes~126 mg/dL or higher on two separate tests is considered diabetes." (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52

,
Interventionmg/dL (Mean)
BaselineDay 28Day 84Week 28Week 36Week 52
Control217.3100.989.796.287.291.5
Investigational183.3100.096.0106.596.098.6

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Fasting Lipid Profile at Baseline (Pre-Transplant)

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01790594)
Timeframe: Baseline (Pre-Transplant)

,
Interventionmg/dL (Mean)
Tot. Chol. BaselineNon-HDL BaselineLDL BaselineHDL BaselineTriglyc. Baseline
Control140.582.460.758.1107.5
Investigational142.891.066.551.8120.9

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Fasting Lipid Profile at Wk 28 Post-Transplant

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01790594)
Timeframe: Week 28 Post-Transplant

,
Interventionmg/dL (Mean)
Tot. Chol. Week 28Non-HDL Week 28LDL Week 28HDL Week 28Triglyc. Week 28
Control149.297.481.251.887.8
Investigational159.9112.693.147.393.2

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HbA1c at Baseline (Pre-Transplant) Through Wk 52 Post-Transplant

"Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control:~A value below 6.0% reflects normal levels,~6.0% to 6.4% reflects prediabetes, and~a value of ≥ 6.5% reflects diabetes." (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52

,
Interventionpercent (Mean)
BaselineDay 28Day 84Week 28Week 36Week 52
Control8.56.14.95.25.15.3
Investigational8.66.04.85.35.35.5

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Lipid Profile at Wk 52 Post-Transplant

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤ 20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01790594)
Timeframe: Week 52 Post-Transplant

,
Interventionmg/dL (Mean)
Tot. Chol. Week 52Non-HDL Week 52LDL Week 52HDL Week 52Triglyc. Week 52
Control162.8112.392.747.395.4
Investigational164.2115.796.948.588.6

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Severity Grade of First Biopsy-Proven Acute Rejection (AR) During the First 52 Weeks Post-Transplant

"AR grading using standard Banff* criteria. For both kidney and pancreas, AR is defined as a grade ≥1.~AR for the kidney: Banff 2007 criteria. Severity of AR is graded by as IA, IB, IIA, IIB, or III, with IA defined as the mildest form of AR and III being the most severe.~AR for the pancreas: Banff 2011 Criteria. Severity of AR is graded is I, II, or III, with I defined as the mildest form of AR and III being the most severe." (NCT01790594)
Timeframe: Transplant through Week 52

,
InterventionParticipants (Number)
Kidney First Grade IAKidney First Grade IBKidney First Grade IIAKidney First Grade IIBPancreas First Grade IPancreas First Grade II
Control101001
Investigational010141

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Standardized Blood Pressure Measurement From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant

"A blood pressure measurement consists of two numbers: the systolic and diastolic pressures. Systolic pressure measures the pressure in blood vessels when the heart beats. Diastolic pressure measures the pressure in blood vessels between beats of the heart.~Systolic measures of <120 and diastolic measures of <80 are considered normal.~Systolic measures of 120-139 and diastolic measures of 80-89 are considered at risk (or pre-hypertension).~Systolic measures of ≥140 and diastolic measures of ≥90 are considered high." (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52

,
InterventionmmHg (Mean)
Systolic BP at BaselineSystolic BP at Day 28Systolic BP at Day 84Systolic BP at Week 28Systolic BP at Week 36Systolic BP at Week 52Diastolic BP at BaselineDiastolic BP at Day 28Diastolic BP at Day 84Diastolic BP at Week 28Diastolic BP at Week 36Diastolic BP at Week 52
Control158.3112.0123.8126.2126.7127.085.265.473.473.376.877.0
Investigational161.3116.9126.3136.1133.5132.082.767.072.876.676.574.2

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Type of Treatment(s) Participants Received for Biopsy-Proven Pancreatic Allograft Rejection During the First 52 Weeks Post-Transplant

"Participants are stratified by kidney biopsy results/treatment received.~Upon having a for-cause biopsy performed, persons often receive treatment for rejection based on the biopsy results, which may or may not reveal signs of rejection. Details of biopsy findings and corresponding treatment are provided for each instance of treatment for rejection.~Results summary format: biopsy results; treatment.~Acronyms and abbreviations:~ACR=Acute Cellular Rejection~IFTA=Interstitial Fibrosis and Tubular Atrophy~ATG=Anti-thymocyte globulin therapy~IVIG=Intravenous Immunoglobulin therapy~Gd =Grade~PO=Orally~QD=Daily" (NCT01790594)
Timeframe: Transplant through Week 52

,
InterventionParticipants (Count of Participants)
ACR-Gd. I/ATGACR-Gd. I/ATG, Pulse SteroidsACR-Gd. I/ATG, Pulse Steroids, IVIGACR-Gd. I, IFTA-Gd. I/Pulse Steroids, Solumedrol,ACR-Gd. II/ATG, Pulse SteroidsNo grade reported/None
Control000010
Investigational111111

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Type of Treatment(s) Participants Received for Biopsy-Proven Renal Allograft Rejection During the First 52 Weeks Post-Transplant

"Participants are stratified by kidney biopsy results/treatment received.~In the event of a for cause renal (kidney) biopsy:~-The diagnosis of acute cellular rejection (ACR) using the Banff 2007 renal allograft pathology criteria. These criteria for renal allograft biopsies is an international histopathological classification standard. ACR is defined by a renal biopsy demonstrating a Banff 2007 classification of Grade IA or greater, with higher scores indicating more severe rejection. (Ref: Solez K, Colvin RB et al. Banff 07 classification of renal allograft pathology: updates and future directions. Am J Transplant 2008 8(4): 753-60).~Acronyms and abbreviations:~ACR=Acute Cellular Rejection*~Normal*~Borderline* (criteria for ACR not fulfilled)~Gd.=Grade*~IFTA=Interstitial Fibrosis and Tubular Atrophy*~ATG=Anti-thymocyte globulin therapy~IVIG=Intravenous Immunoglobulin therapy~PO=Orally~QD=Daily *Banff 2007 renal allograft pathology criteria" (NCT01790594)
Timeframe: Transplant through Week 52

,
InterventionParticipants (Count of Participants)
Borderline/NoneBorderline/Pulse SteroidsBorderline, IFTA-Gd. I/Pulse Steroids, IVIGACR-Gd. IA/ATG, Pulse SteroidsACR-Gd. IB/ATG, Pulse SteroidsACR-Gd. IIA/ATG, Pulse SteroidsACR-Gd. IIB/ATG, Pulse SteroidsIFTA-Gd. I/NoneIFTA-Gd. I/Potassium citrateNormal/Steroids QDNo grade reported/IVIGNo grade reported/NoneNormal/None
Control0111010001042
Investigational1000101120113

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Incidence of Adverse Events

Number of subjects who developed reportable AEs, assessed using adapted version of the Common Toxicity Criteria (NCT00453388)
Timeframe: Up to Day 100

InterventionParticipants (Count of Participants)
Arm II (2 vs 2.5 vs 3 vs 1 vs 0 Gy TBI De-escalation)3
Arm III (2 vs 2.5 vs 3 Gy TBI Dose-escalation)0

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Incidence of Grades III-IV Acute GVHD

"Number of subjects who developed maximum grade acute graft-vs-host disease~aGVHD Stages~Skin:~- a maculopapular eruption involving < 25% BSA~- a maculopapular eruption involving 25 - 50% BSA~- generalized erythroderma~- generalized erythroderma with bullous formation and often with desquamation~Liver:~- bilirubin 2.0 - 3.0 mg/100 mL~- bilirubin 3 - 5.9 mg/100 mL~- bilirubin 6 - 14.9 mg/100 mL~- bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade III: Stage 2 - 4 gastrointestinal involvement and/or +2 to +4 liver involvement, with or without a rash Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00453388)
Timeframe: Up to Day 100

InterventionParticipants (Count of Participants)
Arm II (2 vs 2.5 vs 3 vs 1 vs 0 Gy TBI De-escalation)1
Arm III (2 vs 2.5 vs 3 Gy TBI Dose-escalation)0

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Number of Patients Who Engraft at Each Dose of TBI Used

Number of subjects who engrafted. Engraftment defined as greater than 95% donor chimerism. (NCT00453388)
Timeframe: Up to Day 200

InterventionParticipants (Count of Participants)
Arm II (2 vs 2.5 vs 3 vs 1 vs 0 Gy TBI De-escalation)5
Arm III (2 vs 2.5 vs 3 Gy TBI Dose-escalation)1

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Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose

The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug. (NCT01399047)
Timeframe: Baseline to 8 weeks

InterventionParticipants (Count of Participants)
Mycophenolate17
Placebo19

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Unified Batten Disease Rating Scale Behavior Subscale Change

"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Mood and behavior severity was measured by the behavior subscale of the UBDRS with a range of 0-55 with zero indicating better outcome.~The overall treatment effect was determined - mean difference in behavior subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks

Interventionunits on a scale (Mean)
Mycophenolate-1.28
Placebo-0.37

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Unified Batten Disease Rating Scale Capability Subscale Change

"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Capability severity was measured by the capability subscale of the UBDRS with a range of 0-14 with higher scores indicating better outcome.~The overall treatment effect was determined - mean difference in capability subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks

Interventionunits on a scale (Mean)
Mycophenolate0.33
Placebo0.47

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Unified Batten Disease Rating Scale Physical Subscale Change

"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Motor impairment was measured by the physical subscale of the UBDRS with a range of 0-112 with zero indicating better outcome.~The overall treatment effect was determined - mean difference in physical subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks

Interventionunits on a scale (Mean)
Mycophenolate-1.18
Placebo2.17

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Unified Batten Disease Rating Scale Seizure Subscale Change

"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Seizure severity was measured by the seizure subscale of the UBDRS with a range of 0-54 with zero indicating better outcome.~The overall treatment effect was determined - mean difference in seizure subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks

Interventionunits on a scale (Mean)
Mycophenolate-0.24
Placebo-1.44

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Development of Infectious Complications

The incidence of definite and probable viral, fungal and bacterial infections will be tabulated for each patient. (NCT01570348)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)1

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Disease Activity

Evaluated using a standardized tool for evaluating CD (CDAI). (NCT01570348)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)0

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EFS

Described graphically using a Kaplan-Meier estimate. Generated with confidence intervals using Greenwood's formula to calculate the standard error. Estimated with exact 90% confidence intervals. (NCT01570348)
Timeframe: Up to 5 years post-transplant

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)1

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Event-free Survival (EFS)

Defined as alive and free of active CD. Described graphically using a Kaplan-Meier estimate. Generated with confidence intervals using Greenwood's formula to calculate the standard error. Estimated with exact 90% confidence intervals. (NCT01570348)
Timeframe: At 1 year post-transplant

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)1

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Incidence and Severity of GVHD

The grading of acute and chronic GVHD will follow previously published guidelines but will also include capture of symptoms and characterization of alternative causes. The highest level of organ abnormalities, the etiologies contributing to the abnormalities and biopsy results pertaining to GVHD will be identified. Since both GVHD and CD involve the gastrointestinal tract, all diagnostic biopsies of these organs will be reviewed by pathologists experienced in the diagnosis of GVHD and IBD, respectively. (NCT01570348)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)1

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Incidence of Disease-modifying Drugs for CD Initiated Post-transplant

Includes the administration of any therapy (drugs, biologics, or any other treatments) clearly given as immunomodulatory therapy for underlying CD. (NCT01570348)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)0

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Incidence of Graft Rejection

Engraftment is defined as achieving > 5% donor peripheral blood CD3 T cell chimerism by day 84 after HCT. Primary graft failure is defined as a donor peripheral blood CD3 T cell chimerism peak of < 5% by Day 84 post-HCT. Secondary graft failure is defined as documented engraftment followed by loss of the graft with donor peripheral blood CD3 T cell chimerism < 5% as demonstrated by a chimerism assay. (NCT01570348)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)0

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Overall Survival

Characterized by the event rates as functions of all patients enrolled and at risk of the event, with exact confidence intervals. (NCT01570348)
Timeframe: Time of treatment assignment until death due to any cause, assessed up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)1

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Quality of Life Measured Using the Previously Validated Short Inflammatory Bowel Disease Questionnaire

(NCT01570348)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic BMT)0

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Count of Participant Deaths or Graft Loss by Wk 52 Post-Transplant

This measure counts deaths and graft loss occurring at any point post transplantation. Graft loss is defined as 90 days of dialysis dependency. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac2
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept0
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0

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Count of Participants With Acute Cellular Rejection Grade ≥ IA Defined by Banff 2007 Criteria By Wk 52 Post-Transplant

Acute cellular rejection occurs when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade equal to or greater than IA by Banff 2007 criteria as determined by local pathology. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept10
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept4

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Count of Participants With Antibody Mediated Rejection by Wk 52 Post-Transplant

Antibody mediated rejection is defined by diffusely positive staining for C4d, presence of circulating anti-donor antibodies, and morphologic evidence of acute tissue injury and was determined by local pathology. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept1
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0

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Count of Participants With Biopsy Proven Acute Rejection By Wk 52 Post-Transplant

Biopsy proven acute rejection definition: histologic evidence of a Banff grade of ≥1A per local pathologist. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept10
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept4

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Count of Participants With Defined CKD Stage 4 or 5 at Wk 52 Post-Transplant

"The stages of Chronic Kidney Disease (CKD) are defined using the participant's GFR value:~Stage 1 if GFR value is ≥ 90 (kidney function is normal)~Stage 2 if 60 ≤ GFR < 90 (mildly reduced kidney function, pointing to kidney disease)~Stage 3A if 45 <= GFR < 60*~Stage 3B if 30 <= GFR < 45*~Stage 4 if 15 ≤ GFR < 30 (severely reduced kidney function)~Stage 5 if GFR < 15 (severe or end stage kidney failure).~Stages 3A abd 3B indicate moderately reduced kidney function.*" (NCT01856257)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac4
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept4
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0

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Count of Participants With Delayed Graft Function at Wk 52 Post-Transplant

Delayed grafted function is defined as dialysis in the first week on one or more occasions for any indication other than the treatment of acute hyperkalemia in the setting of otherwise acceptable renal function. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac6
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept9
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0

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Count of Participants With eGFR < 60 mL/Min/1.73 m^2 Measured by CKD-EPI at Wk 52 Post-Transplant

"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01856257)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac21
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept15
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept6

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Count of Participants With Epstein-Barr Virus (EBV) Infection as Reported on the Case Report Form as Adverse Events

Viral infections following renal transplantation, including but not limited to EBV infection, is a significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac0
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept0
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0

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Count of Participants With Graft Rejection by Wk 52 Post-Transplant

The number of participants who were treated by their local physician for any type of rejection including, but not limited to cellular rejection and antibody- mediated rejection of the transplanted kidney regardless of the presence of a biopsy. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac7
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept14
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept4

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Count of Participants With Infections Requiring Hospitalization or Systemic Therapy by Wk 52 Post-Transplant

Infections of certain types (i.e., excluding those identified in the protocol as occurring commonly in this study population) were required to be reported as a serious adverse event if they required either inpatient hospitalization or prolongation of a current hospitalization. (NCT01856257)
Timeframe: Transplantation through Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept8
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept2

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Count of Participants With Treated Diabetes Between Day 14 and Wk 52 Post-Transplant

Treated diabetes is defined as receipt of any oral medication or insulin for the treatment of diabetes for >14 days. (NCT01856257)
Timeframe: Day 14 through week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac3
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept2
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0

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Count of Participants With Use of Anti-hypertensive Medication at Wk 52 Post-Transplant

Anti-hypertensive medications are a class of drugs that are used to treat hypertension. The medications seek to prevent the complications of high blood pressure, such as stroke and myocardial infarction. (NCT01856257)
Timeframe: Week 52

InterventionParticipants (Count of Participants)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac18
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept23
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept8

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Mean Calculated eGFR Using MDRD 4 Variable Model at Wk 52 Post-Transplant

"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease equation (MDRD):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate severe or endstage kidney failure." (NCT01856257)
Timeframe: Week 52

InterventionmL/min/1.73m^2 (Mean)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac56.1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept57.0
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept58.2

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Mean Estimated Glomerular Filtration Rate (eGFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52 Post-Transplant

"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01856257)
Timeframe: Week 52

InterventionmL/min/1.73m^2 (Mean)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac59.2
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept61.5
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept63.0

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The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine Post-Transplant

"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or endstage kidney failure.~An estimate of the slope, or change over time, in eGFR was produced using standard statistical linear modeling procedures. The estimate was then re-scaled so that it could be interpreted as a change in eGFR per month. Positive numbers indicate increasing kidney function.~Larger numbers indicate greater change in kidney function." (NCT01856257)
Timeframe: Day 28 through Week 52 Post-Transplant

InterventioneGFR change over time (by month) (Mean)
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac0.3
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept1.3
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0.8

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Count of Participants by Severity of First Acute Cellular Rejection by Wk 52 Post-Transplant

Acute cellular rejection occurs when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade equal to or greater than IA by Banff 2007 criteria as determined by local pathology. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection. Originally it was 2 endpoints but all participants' highest grade was also their first grade so only reporting their first grade. (NCT01856257)
Timeframe: Transplantation through Week 52

,,
InterventionParticipants (Count of Participants)
IAIBIIAIIBIII
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept22000
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept31402
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac01000

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Count of Participants Experiencing ≥ 1 Adverse Event (AEs) or Serious Adverse Events (SAEs) by Wk 52

Adverse events were collected systematically from enrollment through Wk 52, the last study visit. Provided are numbers of participants with ≥ 1 adverse event (serious or non-serious adverse events) by treatment arm. (NCT01856257)
Timeframe: Enrollment through Week 52

,,
InterventionParticipants (Count of Participants)
Adverse EventsSerious Adverse Events
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept96
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept2821
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac2119

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Count of Participants With BK Polyoma Virus (BKV) and Cytomegalovirus (CMV) Viremia (Local Center Monitoring) as Adverse Events by Wk 52 Post-Transplant

Viral infections following renal transplantation is significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during the study, using participant blood samples. Displayed are counts of participants who experienced BKV and CMV viremia as adverse events by treatment arm. (NCT01856257)
Timeframe: Transplantation through Week 52

,,
InterventionParticipants (Count of Participants)
BKVCMV
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept11
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept46
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac01

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Count of Participants With Either New Onset Diabetes After Transplant (NODAT) or Impaired Fasting Glucose (IFG) at Week 52 Post-Transplant -Based on Criteria Specified by the ADA and WHO

"New onset diabetes is the development of diabetes post-kidney transplant. It was identified by the clinical sites caring for each participant and reported directly in the clinical database. Impaired fasting glucose (IFG) is a determination made by referencing glucose measurements obtained from a standard chemistry panel. Any fasting glucose measure that is between 110 and 125 mg/dL is classified as IFG.~Acronyms: American Diabetes Association (ADA); World Health Organization (WHO)." (NCT01856257)
Timeframe: Transplantation through Week 52

,,
InterventionParticipants (Count of Participants)
New onset diabetes during first 52 weeksImpaired fasting glucose at week 52
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept00
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept10
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac12

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Count of Participants With Fever > 39 Degrees Celsius and Blood Pressure < 90 mmHg Within 24 Hours of Onset of Transplant Procedure

Temperature of >39 degrees Celsius (e.g., 102.2 degrees Fahrenheit) would be an indication of fever most often in response to an infection or illness. Systolic blood pressure <90mm Hg would be an indication of low blood pressure. (NCT01856257)
Timeframe: Within 24 Hours of transplant procedure

,,
InterventionParticipants (Count of Participants)
Fever >39 CelsiusSystolic BP < 90 mmHg
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept00
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept00
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac00

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Count of Participants With Use of Lipid Lowering Medications at Baseline and Wk 28 and Wk 52 Post-Transplant

Lipid lowering medications are used in the treatment of high levels of fats (lipids), such as cholesterol in blood. (NCT01856257)
Timeframe: Baseline (Pre-Transplant), Week 28, and Week 52

,,
InterventionParticipants (Count of Participants)
BaselineWeek 28Week 52
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept345
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept799
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac1389

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Fasting Lipid Profile at Baseline (Pre-Transplant)

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01856257)
Timeframe: Baseline

,,
Interventionmg/dL (Mean)
Total cholesterolNon-HDLLDLHDLTriglyceride
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept167.6123.465.244.1227.1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept159.4116.183.143.3194.4
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac167.1122.091.145.1156.8

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Fasting Lipid Profile at Wk 28 Post-Transplant

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01856257)
Timeframe: Week 28

,,
Interventionmg/dL (Mean)
Total cholesterolNon-HDLLDLHDLTriglyceride
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept167.3115.082.752.3166.6
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept179.5133.5109.846.0153.3
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac169.6119.695.651.0126.4

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Fasting Lipid Profile at Wk 52 Post-Transplant

"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤ 20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01856257)
Timeframe: Week 52

,,
Interventionmg/dL (Mean)
Total cholesterolNon-HDLLDLHDLTriglyceride
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept174.8130.894.644.0182.8
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept163.7121.286.342.4170.0
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac177.1128.6102.948.5125.8

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Hemoglobin A1c (HbA1c) Measurements Over Time

"Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control:~A value below 6.0% reflects normal levels,~6.0% to 6.4% reflects prediabetes, and~a value of ≥ 6.5% reflects diabetes." (NCT01856257)
Timeframe: Baseline (Pre-Transplant) and Days 28 and -84, and Weeks 28, -36, and -52 Post-Transplant

,,
Interventionpercentage (Mean)
BaselineDay 28Day 84Week 28Week 36Week 52
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept5.95.45.45.65.55.8
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept5.75.65.56.05.96.7
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac5.75.45.66.26.77.8

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Standardized Blood Pressure Measurement at Wk 52 Post-Transplant

"A blood pressure measurement consists of two numbers: the systolic and diastolic pressures. Systolic pressure measures the pressure in blood vessels when the heart beats. Diastolic pressure measures the pressure in blood vessels between beats of the heart.~Systolic measures of <120 and diastolic measures of <80 are considered normal.~Systolic measures of 120-139 and diastolic measures of 80-89 are considered at risk (or pre-hypertension).~Systolic measures of ≥140 and diastolic measures of ≥90 are considered high." (NCT01856257)
Timeframe: Week 52

,,
InterventionmmHg (Mean)
Systolic BP at W52Diastolic BP at W52
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept132.075.7
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept133.779.1
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac135.077.7

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Total Daily Prescribed Pill Count

This is a measure of the total number of pills a participant was prescribed on a given day (NCT01856257)
Timeframe: Day 28, Day 84, Week 28, Week 36, and Week 52

,,
Interventionpills per day (Mean)
Day 28Day 84Week 28Week 36Week 52
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept23.521.016.613.913.7
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept18.917.215.515.415.7
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac25.822.219.719.124.4

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Type of Rejection Classified by Pathologist - For Cause Kidney Biopsies

"Upon having a biopsy performed, persons often receive treatment for rejection based on the results of the biopsy, which may or may not have shown signs of rejection. Details of local biopsy findings are presented here for rejection. Acronyms and abbreviations are defined as follows:~ACR= Acute T-Cell Mediated rejection~AMR= Acute Antibody-mediated rejection~Chr. AMR=Chronic Antibody Mediated Rejection~Gd.=Grade~IFTA=Interstitial Fibrosis and Tubular Atrophy" (NCT01856257)
Timeframe: Transplantation through Week 52

,,
InterventionBiopsy (Number)
BorderlineBorderline, AMR-Gd. I (ATN-Like)Borderline, AMR-Gd.I (ATN-Like), Chr.AMR, IFTA-Gd1Borderline, AMR-Gd. I (ATN-Like), IFTA-Gd. IBorderline, Chr.AMR, IFTA-Gd. IBorderline, IFTA-Gd. IBorderline, IFTA- Gd. IIACR-Gd. IAACR-Gd. IA, IFTA-Gd. IACR-Gd. IA, IFTA-Gd. IIACR-Gd. IBACR-Gd. IB, IFTA-Gd. IACR-Gd. IB, IFTA-Gd. IIACR-Gd. IB, IFTA-Gd. IIIACR-Gd. IIAACR-Gd. IIA, IFTA-Gd. IACR-Gd. IIIAMR-Gd. II (Capillary/Glomerular)IFTA-Gd. IIFTA-Gd. II
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept00000102000110000010
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept50010214100101222052
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac31101120011000000141

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Type of Treatment for Detected Graft Rejection

"Upon having a biopsy performed, persons often receive treatment for rejection based on the results of the biopsy, which may or may not have shown signs of rejection. Details of treatment are presented here for rejection. Acronyms and abbreviations are defined below.~ATG=Thymoglobulin" (NCT01856257)
Timeframe: Transplantation through Week 52

,,
InterventionBiopsy (Number)
ATGATG, Pulse SteroidsATG, Pulse Steroids, PrografAntibioticPlasmapheresisPlasmapheresis, Oral SteroidsPulse SteroidsPulse Steroids, LeflunomidePulse Steroids, Plasmapheresis, EculizumabPrednisone
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept0200003000
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept1510009102
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac0001116010

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Disease Relapse or Progression as Measured by CT Scan or PET

(NCT00574496)
Timeframe: 3 years

Interventionmonths (Median)
High-Risk or Relapsed Hodgkin Lymphoma34.3

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Overall Survival

(NCT00574496)
Timeframe: up to 8 years

Interventionmonths (Median)
High-Risk or Relapsed Hodgkin Lymphoma70.3

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Progression-free Survival at 1 Year

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions (NCT00574496)
Timeframe: 1 year

Interventionproportion of progression-free pts (Number)
High-Risk or Relapsed Hodgkin Lymphoma33.3

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Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)

Diffusing capacity of the lungs for carbon monoxide (DLCO) measures how much oxygen travels from the alveoli of the lungs to the blood stream. It is used to determine the severity of lung disease. DLCO for a given individual is compared to reference or predicted values. The reference values are based on healthy individuals with normal lung function and indicates values that would be expected for someone of the same sex, age and height. DLCO % predicted compares the patients DLCO values with the reference values. An individuals DLCO result that is at least 80% of the predicted value is considered normal. Change in DLCO % predicted is measured by median change (and interquartile range) from Baseline median to week 52 median. (NCT01670565)
Timeframe: Baseline and Week 52

Intervention% predicted (Median)
Mycophenolate Mofetil + Belimumab2.00
Mycophenolate Mofetil + Saline (Placebo)0.00

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Change in Forced Vital Capacity (FVC)

Forced vital capacity (FVC) is the amount of air that can be forcibly exhaled from the lungs after taking a deep breath. It is used to determine the severity of lung disease. FVC for a given individual is compared to reference or predicted values. The reference values are based on healthy individuals with normal lung function and indicates values that would be expected for someone of the same sex, age and height. FVC % predicted compares the patients FVC values with the reference values. Results are considered normal if FVC is 80 percent or more of the predicted value. Change in FVC % predicted is measured by median change (and interquartile range) from Baseline median to week 52 median. (NCT01670565)
Timeframe: Baseline and Week 52

Intervention% predicted (Median)
Mycophenolate Mofetil + Belimumab5.00
Mycophenolate Mofetil + Saline (Placebo)-2.00

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Change in in Short Form-36 (SF-36) Questionnaire:Mental Component Summary

The Short Form 36 (SF-36) is a validated 36 item questionnaire which measures quality of life across eight domains: physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, general health. The mental component score (MCS) is composed of a subset of the 8 health domains. Each component is directly transformed into a 0 to 100 scale on the assumption that each question carries equal weight. A score of 0 is equal to maximum disability, and a score of 100 is equivalent to no disability. The SF-36 mental component can be obtained by looking at the mean average of all the emotionally relevant items. Change in Short Form 36 mental (SF-36 MC) is measured by median change (and interquartile range) from Baseline to week 52. The median change can range from -100 to 100. A positive median change indicates indicates an improved outcome. (NCT01670565)
Timeframe: Baseline and at 52 weeks

Interventionscore on a scale (Median)
Mycophenolate Mofetil + Belimumab7.50
Mycophenolate Mofetil + Saline (Placebo)3.00

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Change in Modified Rodnan Skin Score (MRSS)

Change in MRSS is measured by median change (and interquartile range) from Baseline median to week 52 median. The efficacy of the drug will be measured as the change in the Modified Rodnan Skin Score (MRSS) at 52 weeks. The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. The skin score is 0 for uninvolved skin through 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas. The minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease (NCT01670565)
Timeframe: Baseline and at 52 weeks

Interventionunits on a scale (Median)
Mycophenolate Mofetil + Belimumab-10
Mycophenolate Mofetil + Saline (Placebo)-3.0

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Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI)

"The Scleroderma Health Assessment Questionnaire (SHAQ) consist of the Health Assessment Questionnaire (HAQ) and 8 other domains addressing scleroderma related manifestations that contribute to disability. It is a quality of life measure. Each question is scored from 0 (without difficulty) to 3 (unable to do). Some domains in the SHAQ are visual analog scales that are measured first and then changed to a 0-3 scale.~The maximum from each category is added together and divided by the number of categories completed. The total scale range is 0-3. A higher score indicates worse functionality.~Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ-DI) is measured as median change (and interquartile range) from Baseline median to week 52 median. The reported median change can range from -3 to 3. A negative median change indicates a better outcome." (NCT01670565)
Timeframe: Baseline and Week 52

Interventionscore on a scale (Median)
Mycophenolate Mofetil + Belimumab-0.25
Mycophenolate Mofetil + Saline (Placebo)0.00

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Change in Short Form-36 (SF-36) Questionnaire: Physical Component Summary

The Short Form 36 (SF-36) is a 36 item questionnaire which measures quality of life across eight domains: physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, general health. The physical component score is composed of a subset of the 8 health domains.The SF-36 physical component can be obtained by looking at the mean average of all the physically relevant items. Each component is directly transformed into a 0 to 100 scale on the assumption that each question carries equal weight. A score of 0 is equal to maximum disability, and a score of 100 is equivalent to no disability. Change in Short Form 36 physical component (SF-36 PC) is measured by median change (and interquartile range) from Baseline to week 52. The median change can range from -100 to 100. A positive median change indicates indicates an improved outcome. (NCT01670565)
Timeframe: Baseline and Week 52

Interventionscores on a scale, 0-100 (Median)
Mycophenolate Mofetil + Belimumab8.00
Mycophenolate Mofetil + Saline (Placebo)-3.00

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Number of Adverse Events and Serious Adverse Events

The safety and tolerability of belimumab in patients with systemic sclerosis will be as assessed by comparing the rates of adverse events (AEs) and serious adverse events (SAEs) between treatment and placebo groups. (NCT01670565)
Timeframe: At 52 weeks

,
Interventionnumber of AEs (Number)
Total number of AEsTotal number of infectious AEsSerious AEs
Mycophenolate Mofetil + Belimumab53180
Mycophenolate Mofetil + Saline (Placebo)56163

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Effectiveness and Safety of a Particular Drug Regimen to Prevent Kidney Rejection

Number of Participants with Kidney Rejections (NCT01005706)
Timeframe: 12 months

Interventionparticipants (Number)
Tacrolimus Withdrawal Arm4
Tacrolimus Minimization Arm1

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Dose-Normalized C0

"Dose normalized C0 was determined (in mg/L) from blood samples collected predose. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized C0 equals (=) C0 divided by (/) (actual dose taken/1000) For the EC-MPS group: Dose normalized C0 = C0 / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose

Interventionmg/L (Mean)
MMF/Prednisone2.962
EC-MPS/Prednisone4.658

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Dose-Normalized Cmax (mg/L)

"Dose-normalized Cmax in plasma was determined (in mg/L) from blood samples collected predose and postdose on Day 1. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized Cmax = Cmax / (actual dose taken/1000) For the EC-MPS group: Dose normalized Cmax = Cmax / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

Interventionmg/L (Mean)
MMF/Prednisone18.402
EC-MPS/Prednisone29.996

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Dose-Normalized Cmin

"Dose-normalized Cmin was determined (in mg/L) from blood samples collected predose and postdose. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized Cmin = Cmin/ (actual dose taken/1000) For the EC-MPS group: Dose normalized Cmin = Cmin / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

Interventionmg/L (Mean)
MMF/Prednisone1.702
EC-MPS/Prednisone2.613

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Dose-Normalized MPA AUC0-12

"Dose-normalized MPA AUC0-12 in plasma was determined (mg*h/L) from blood samples collected predose and postdose on Day 1. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized MPA AUC = MPA AUC / (actual dose taken/1000) For the EC-MPS group: Dose normalized MPA AUC = MPA AUC / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

Interventionmg*h/L (Mean)
MMF/Prednisone61.53862
EC-MPS/Prednisone94.65765

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Maximum Plasma Concentration (Cmax)

The mean maximum MPA concentration in plasma was determined (in mg/L) in blood samples collected predose and postdose on Day 1. (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

Interventionmg/L (Mean)
MMF/Prednisone15.385
EC-MPS/Prednisone17.827

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Minimum Plasma Concentration (Cmin)

The mean minimum MPA concentration in plasma was determined (in mg/L) from blood samples collected predose and postdose on Day 1. (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

Interventionmg/L (Mean)
MMF/Prednisone1.385
EC-MPS/Prednisone1.620

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MPA Area Under the Curve From 0 to 12 Hours (AUC0-12)

The mean MPA AUC0-12 in plasma was determined (in mg multiplied by hours, per Liter [mg*h/L]) from blood samples collected predose and postdose on Day 1. (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

Interventionmg*h/L (Mean)
MMF/Prednisone50.36348
EC-MPS/Prednisone57.06682

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Pre-dose Trough Concentration (C0)

The mean mycophenolic acid (MPA) concentration in plasma was determined (in milligrams per liter [mg/L]) from blood samples collected predose (immediately before receiving study treatment). (NCT01033864)
Timeframe: Day 1 predose

Interventionmg/L (Mean)
MMF/Prednisone2.387
EC-MPS/Prednisone2.944

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Percentage of Participants By Time to Maximum Plasma Concentration (Tmax)

(NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours

,
Interventionpercentage of participants (Number)
Tmax equals (=) 0.5333 hours (hrs)Tmax=0.6000 hrsTmax=0.7333 hrsTmax=0.7667 hrsTmax=1.0667 hrsTmax=1.0833 hrsTmax=1.1167 hrsTmax=1.2167 hrsTmax=2.0167 hrsTmax=2.0833 hrsTmax=2.1000 hrsTmax=2.1167 hrsTmax=2.1667 hrsTmax=3.1167 hrsTmax=3.1833 hrs
EC-MPS/Prednisone0.00.00.00.09.10.00.00.09.118.29.127.39.19.19.1
MMF/Prednisone16.716.716.78.38.316.78.38.30.00.00.00.00.00.00.0

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Regression Coefficients For Participants Receiving MMF

The estimated regression coefficients for participants who received MMF presented in milligrams per liter (mg/L). (NCT01033864)
Timeframe: Day 1 at 30 minutes and 1 and 2 hours postdose

Interventionmg/L (Number)
InterceptConcentration at 30 minutes (C0.5)Concentration at 1 hour (C1)Concentration at 2 hours (C2)
MMF/Prednisone2.171920.740311.893232.85923

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Creatinine Clearance at 1 Month After Transplantation

Creatinine clearance is an indicator of renal function. Creatinine clearance is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Normal values for healthy, young males are in the range of 100-135 milliliters per minute (mL/min) and for females, 90-125 mL/min. Creatinine clearance decreases with age. A low creatinine clearance rate indicates poor kidney function. (NCT01672957)
Timeframe: Month 1

InterventionmL/min (Mean)
Renal Transplant Participants65.82

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Creatinine Clearance at Month 12 After Transplantation

Creatinine clearance is an indicator of renal function. Creatinine clearance is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Normal values for healthy, young males are in the range of 100-135 mL/min and for females, 90-125 mL/min. Creatinine clearance decreases with age. A low creatinine clearance rate indicates poor kidney function. (NCT01672957)
Timeframe: Month 12

InterventionmL/min (Mean)
Renal Transplant Participants69.69

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Creatinine Clearance at Month 6 After Transplantation

Creatinine clearance is an indicator of renal function. Creatinine clearance is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Normal values for healthy, young males are in the range of 100-135 mL/min and for females, 90-125 mL/min. Creatinine clearance decreases with age. A low creatinine clearance rate indicates poor kidney function. (NCT01672957)
Timeframe: Month 6

InterventionmL/min (Mean)
Renal Transplant Participants73.38

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GFR at Month 12 After Transplantation

GFR is an index of kidney function. GFR describes the flow rate of filtered fluid through the kidney. A normal GFR is > 90 mL/min, although children and older people usually have a lower GFR. Lower values indicates poor kidney function. A GFR < 15 mL/min indicates kidney failure. (NCT01672957)
Timeframe: Month 12

InterventionmL/min (Mean)
Renal Transplant Participants58.03

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GFR at Month 6 After Transplantation

GFR is an index of kidney function. GFR describes the flow rate of filtered fluid through the kidney. A normal GFR is > 90 mL/min, although children and older people usually have a lower GFR. Lower values indicates poor kidney function. A GFR < 15 mL/min indicates kidney failure. (NCT01672957)
Timeframe: Month 6

InterventionmL/min (Mean)
Renal Transplant Participants56.78

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Glomerular Filtration Rate (GFR) at Month 1 After Transplantation

GFR is an index of kidney function. GFR describes the flow rate of filtered fluid through the kidney. A normal GFR is greater than (>) 90 mL/min, although children and older people usually have a lower GFR. Lower values indicates poor kidney function. A GFR less than (<) 15 mL/min indicates kidney failure. (NCT01672957)
Timeframe: Month 1

InterventionmL/min (Mean)
Renal Transplant Participants53.54

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Mean Dose of Mycophenolate Mofetil

(NCT01672957)
Timeframe: Baseline, Months 1, 6, and 12

Interventionmilligrams (mg) (Mean)
Baseline (n=121)Month 1 (n=119)Month 6 (n=88)Month 12 (n=69)
Renal Transplant Participants2033.061718.491457.391539.86

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Percentage of Participants Who Received Other Immunosuppressive Agents in Combination With Mycophenolate Mofetil

Participants could have received more than one other immunosuppressive agents, at the discretion of treating physician. Percentage of participants who received 1 other immunosuppressive agent, 2 other immunosuppressive agents, and 3 other immunosuppressive agents are reported. (NCT01672957)
Timeframe: Baseline, Months 1, 6, and 12

Interventionpercentage of participants (Number)
1 Other agent: at baseline (n=121)1 Other agent: at Month 1 (n=119)1 Other agent: at Month 6 (n=86)1 Other agent: at Month 12 (n=69)2 Other agents: at baseline (n=121)2 Other agents: at Month 1 (n=119)2 Other agents: at Month 6 (n=86)2 Other agents: at Month 12 (n=69)3 Other agents: at baseline (n=121)3 Other agents: at Month 1 (n=119)3 Other agents: at Month 6 (n=86)3 Other agents: at Month 12 (n=69)
Renal Transplant Participants0.80.82.37.290.999.297.792.88.3000

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Percentage of Participants With Acute Rejection

Percentage of participants who experienced acute rejection within 1 month of transplantation, Month 2 to Month 6 after transplantation, Month 7 to Month 12 after transplantation are reported. (NCT01672957)
Timeframe: Baseline to Month 1, Months 2 to 6, Months 7 to 12

Interventionpercentage of participants (Number)
Baseline to Month 1 (n=127)Months 2 to 6 (n=124)Months 7 to 12 (n=111)
Renal Transplant Participants16.50.80.9

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Percentage of Participants With Graft Survival

Graft survival was defined as those participants who did not experience graft loss. Graft loss defined as physical loss (nephrectomy), functional loss (necessitating maintenance dialysis for >8 weeks), re-transplant or death during the first 12 months after transplantation. (NCT01672957)
Timeframe: Months 1, 6, and 12

Interventionpercentage of participants (Number)
Month 1 (n=127)Month 6 (n=123)Month 12 (n=111)
Renal Transplant Participants10099.298.4

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All-Cause Mortality

All-cause mortality is defined as death from any cause occurring after randomization. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance0
MMF Withdrawal0

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Cumulative Excess Systemic Steroid Dose From Time of Clinically Significant Disease Reactivation to Return to Pre-Flare Dose or End of Trial Participation

"For each participant who experienced disease reactivation, excess systemic steroid dose was summed from the time of clinically significant disease reactivation until the dose returns to pre-flare levels or the end of study participation, whichever occurred first. Excess systemic steroid dose was defined as the total dose given for the flare minus (-) a participant's pre-flare steroid dose. Participants who do not have an increase in their steroid use due to the flare had their excess dose set to zero. Steroids include medications that code to a medication class which includes the terms glucocorticoid or corticosteroid. Systemic steroids will include any of these steroids that are taken by mouth (PO), intravenous (IV), or intramuscular (IM)." (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionSteroid dose (mg) (Mean)
MMF Maintenance812.8
MMF Withdrawal1750.7

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Cumulative Systemic Steroid Dose by Week 60

"Steroids include medications that code to a medication class which includes the terms glucocorticoid or corticosteroid. Systemic steroids will include any of these steroids that are taken by mouth (PO), intravenous (IV), or intramuscular (IM). Total cumulative systemic steroid dose, in milligrams, was summarized over the 60 week study period, or until early study termination, for each participant." (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

Interventionsteroid dose (mg) (Mean)
MMF Maintenance851
MMF Withdrawal912

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Number of Malignancies Reported as Adverse Events (AEs).

The number of malignancies reported as AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionAdverse Events (Number)
MMF Maintenance2
MMF Withdrawal3

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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance20
MMF Withdrawal25

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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup

"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance9
MMF Withdrawal12

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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF<2000 mg/Day Subgroup

"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance11
MMF Withdrawal13

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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance15
MMF Withdrawal19

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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance5
MMF Withdrawal6

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Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance4
MMF Withdrawal8

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Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance3
MMF Withdrawal7

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Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance1
MMF Withdrawal1

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Number of Participants Experiencing Any British Isles Lupus Assessment Group (BILAG) A Flare by Week 60

The BILAG assesses participants on a variety of disease activity criteria in nine body system categories (constitutional, mucocutaneous, neuropsychiatric, musculoskeletal, cardiorespiratory, gastrointestinal, ophthalmic, renal, and hematological). Each category is scored as an A, B, C, or D/E, where A indicates most severe disease activity and D/E indicates inactive/no disease activity. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance1
MMF Withdrawal4

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Number of Participants Experiencing Clinically Significant Disease Reactivation by Week 60

Disease reactivation requires:1) SELENA-SLEDAI mild/moderate or severe flare,and 2) Increased immunosuppressive therapy on a sustained basis,defined by one of the following criteria:a) Sustained activity:Significant prolonged SLE flare requiring steroid increase/burst to ≥15 mg/day prednisone (or equivalent) for >4 weeks.b) Frequent relapsing/remitting:Participant flares requiring an increase/burst of steroids and is successfully tapered to <15 mg/day within 4 weeks, but this occurs on >2 occasions, or IA, IM or IV steroids on more than1 occasion.c)Clinical activity of sufficient severity to warrant resumption of/increased dose of MMF or addition of other major immunosuppressive including AZA or MTX.Regardless of steroid use, if the investigator observes disease activity of sufficient severity to warrant resumption, addition or increase in dosage of major immunosuppressant in the setting of a SELENA-SLEDAI flare, participant has met the primary endpoint.Risk difference also included (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance5
MMF Withdrawal9

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Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF <2000 mg/Day Subgroup

"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance3
MMF Withdrawal6

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Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup

"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance1
MMF Withdrawal2

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Number of Participants in the Lupus Nephritis Subgroup Experiencing a British Isles Lupus Assessment Group (BILAG) A Renal Flare by Week 60

The BILAG assesses participants on a variety of disease activity criteria in nine body system categories (constitutional, mucocutaneous, neuropsychiatric, musculoskeletal, cardiorespiratory, gastrointestinal, ophthalmic, renal, and hematological). Each category is scored as an A, B, C, or D/E, where A indicates most severe disease activity and D/E indicates inactive/no disease activity. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance0
MMF Withdrawal2

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Number of Serious Adverse Events (SAEs).

The number of SAEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionSerious Adverse Events (Number)
MMF Maintenance12
MMF Withdrawal6

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The Addition of Aggressive Adjunctive Therapy to Mycophenolate Mofetil (MMF) or Change in MMF Therapy to Cytotoxic Drug Due to Flare by Week 60

The addition of aggressive adjunctive therapy could include intravenous (IV) immunoglobulin or rituximab at any point during the participant's study participation. A change in therapy to cytotoxic drug due to flare could include drugs such as cyclophosphamide, etc. A blinded list of study medications was reviewed to identify the addition of aggressive adjunctive therapy or cytotoxic drugs. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionParticipants (Count of Participants)
MMF Maintenance0
MMF Withdrawal1

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Time From Clinically Significant Disease Reactivation to Improvement in British Isles Lupus Assessment Group (BILAG) From Maximum Level During Flare

For each participant who experienced disease reactivation, time from clinically significant disease reactivation to improvement in BILAG from maximum level (at least an A or B) during the flare was calculated in study days as: date of clinically significant disease reactivation minus (-) date of BILAG improvement. If multiple body systems had a BILAG flare at the visit, then the body system with the most severe score was tracked for improvement; if multiple body systems had the same score (at least an A or B), then just one needed to show improvement. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionDays (Mean)
MMF Maintenance114.5
MMF Withdrawal40.5

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Time From Clinically Significant Disease Reactivation to Recovery to Baseline British Isles Lupus Assessment Group (BILAG) Score or BILAG C

For each participant who experienced disease reactivation, time from clinically significant disease reactivation to recovery to baseline BILAG scores or BILAG C, whichever is worse, was calculated in study days as: date of clinically significant disease reactivation minus (-) date of BILAG recovery. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionDays (Mean)
MMF Maintenance114.5
MMF Withdrawal77.7

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Time From Clinically Significant Disease Reactivation to Return to Pre-Flare Steroid Dose

For each participant who experienced disease reactivation, time from clinically significant disease reactivation to recovery to pre-flare steroid dose was calculated in study days as: date of clinically significant disease reactivation minus (-) date of return to pre-flare steroid dose. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionDays (Mean)
MMF MaintenanceNA
MMF Withdrawal37

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Time to Clinically Significant Disease Reactivation

The time to clinically significant disease reactivation was defined as the time from Baseline/Day 0 to the date of the first Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) assessment that met (or went on to meet) the criteria for clinically significant disease reactivation. Time to clinically significant disease reactivation was defined in study weeks as: date of SELENA-SLEDAI assessment that met reactivation criteria minus (-) baseline date. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionWeeks (Mean)
MMF Maintenance38.0
MMF Withdrawal38.5

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Time to First Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare

The time to first mild/moderate or severe SELENA-SLEDAI flare was defined as the time from Baseline/Day 0 to the date of the first mild/moderate or severe SELENA-SLEDAI flare. Time to SELENA-SLEDAI flare was defined in study weeks as: date of SELENA-SLEDAI flare minus (-) baseline date. The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionWeeks (Mean)
MMF Maintenance20.5
MMF Withdrawal27.5

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Time to First Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare

The time to first severe SELENA-SLEDAI flare was defined as the time from Baseline/Day 0 to the date of the first severe SELENA-SLEDAI flare. Time to severe SELENA-SLEDAI flare was defined in study weeks as: date of SELENA-SLEDAI flare minus (-) baseline date. The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

InterventionWeeks (Mean)
MMF Maintenance43.5
MMF Withdrawal41.5

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Change From Baseline in the Functional Assessment of Chronic Illness Therapy (FACIT-F) Fatigue Scale (FS): Total Score

FACIT-Fatigue scale (FS) is a 13-item questionnaire completed by the patient (participant), that provides a measure of fatigue/quality of life, with a 7-day recall period. The participant scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). The larger the participant's response to the questions (with the exception of 2 negatively stated), the greater the fatigue. For all questions, except for the 2 negatively stated ones, the code was reversed and a new score was calculated as 4 minus the participant's response. The sum of all responses resulted in the FACIT-FS score for a total possible score of 0 (worse score) to 52 (better score). A higher score reflected an improvement in the participant's health status. A decrease in the FACIT-FS score reflects worse fatigue/quality of life. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60

,
Interventionunits on a scale (Mean)
Week 24Week 48Week 60
MMF Maintenance-2.41-3.39-3.13
MMF Withdrawal-0.81-0.850.42

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Change From Baseline in the Lupus Quality of Life (QoL)Score

The Lupus QoL assessment is a 34 item questionnaire across 8 domains that is designed to find out how systemic lupus erythematosus (SLE) affects a participant's life over the preceding 4 weeks. Scores range from 0 (worst QoL) to 100 (best QoL). Domains include physical health, pain, planning, intimate relationships, burden to others, emotional health, body image, and fatigue. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60

,
InterventionScores on a Scale (Mean)
Change in Physical Health at Week 24Change in Physical Health at Week 48Change in Physical Health at Week 60Change in Pain at Week 24Change in Pain at Week 48Change in Pain at Week 60Change in Planning at Week 24Change in Planning at Week 48Change in Planning at Week 60Change in Intimate Relationships at Week 24Change in Intimate Relationships at Week 48Change in Intimate Relationships at Week 60Change in Burden to Others at Week 24Change in Burden to Others at Week 48Change in Burden to Others at Week 60Change in Emotional Health at Week 24Change in Emotional Health at Week 48Change in Emotional Health at Week 60Change in Body Image at Week 24Change in Body Image at Week 48Change in Body Image at Week 60Change in Fatigue at Week 24Change in Fatigue at Week 48Change in Fatigue at Week 60
MMF Maintenance-1.11-2.830.00-0.17-3.10-0.190.35-5.43-3.03-1.47-0.81-2.081.56-0.58-1.33-0.87-2.03-1.522.94-0.173.223.78-2.18-0.57
MMF Withdrawal1.040.461.561.771.601.423.90-0.180.184.29-1.102.860.18-4.43-3.550.09-1.22-0.87-1.09-0.56-4.45-1.17-1.56-1.95

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Change From Baseline in the Short Form Health Survey (SF-36) Physical Component Summary (PCS) Score

The SF-36 is a 36-item, patient-reported survey of patient health. Higher scores indicate better outcomes while lower scores indicate more disability. The Physical Component Score is comprised of the Physical Functioning Scale, the Role-Physical Scale, the Bodily Pain Scale, and the General Health Scale. It is scaled from 0 to 100 with a score of 0 equivalent to maximum disability and a score of 100 equivalent to no disability. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60

,
InterventionScores on a Scale (Mean)
Week 24Week 48Week 60
MMF Maintenance-0.75-1.92-1.51
MMF Withdrawal0.900.251.51

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Change From Baseline in the Short Form Health Survey (SF-36) Physical Functioning (PF) Score

The SF-36 is a 36-item, patient-reported survey of patient health. Higher scores indicate better outcomes while lower scores indicate more disability. The PF score is used to assess changes in physical functioning. It is scaled from 0 to 100 with a score of 0 equivalent to maximum disability and a score of 100 equivalent to no disability. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60

,
InterventionScores on a Scale (Mean)
Week 24Week 48Week 60
MMF Maintenance-0.33-0.49-0.19
MMF Withdrawal0.220.951.79

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Change From Baseline in the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Disease Damage Index for Systemic Lupus Erythematosus (SLICC/DI): Total Score

The SLICC/DI measures accumulated damage that has occurred since the onset of systemic lupus erythematosus (SLE), regardless of cause, in 12 organ systems. SLE damage is defined as an irreversible change in an organ or system that has been present for at least 6 months. The SLICC/DI includes 39 areas of damage in 12 domains, where each item is rated as present or absent; if evidence of damage is present for a particular item, it is given a score of 1. Some items are scored with 2 or 3 points in the case of recurring events or end stage renal disease. The SLICC/DI total score will be computed as the sum of all scores for items indicated as present; scores can range from 0 to 45. Higher scores indicate more damage. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60

,
InterventionScores on a Scale (Mean)
Week 24Week 48Week 60
MMF Maintenance0.00.00.0
MMF Withdrawal0.00.00.0

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Number of Grade 3, 4, or 5 Adverse Events (AEs)

The number of Grade 3, 4, or 5 AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

,
InterventionAdverse Events (Number)
Grade 3Grade 4Grade 5
MMF Maintenance1820
MMF Withdrawal1500

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Number of Grade 3, 4, or 5 Hematological Adverse Events (AEs).

The number of Grade 3, 4, or 5 hematological AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60

,
InterventionAdverse Events (Number)
Grade 3Grade 4Grade 5
MMF Maintenance300
MMF Withdrawal000

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Percentage of Participants With Chronic GVHD

Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. (NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib39
Tacrolimus/Methotrexate/Maraviroc43
Tacrolimus/MMF/Cyclophosphamide28

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Percentage of Participants With Chronic GVHD Requiring Immunosupressive Therapy

Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. This endpoint considers the occurrence of chronic GVHD that necessitated initiation of immunosuppressive therapy for treatment. (NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib29
Tacrolimus/Methotrexate/Maraviroc33
Tacrolimus/MMF/Cyclophosphamide22

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Percentage of Participants With Disease Relapse or Progression

Relapse is defined by either morphological or cytogenetic evidence of acute leukemia or MDS consistent with pretransplant features, or radiologic evidence of lymphoma. Progression of disease applies to patients with lymphoproliferative diseases (lymphoma or chronic lymphocytic leukemia) not in remission prior to transplantation and is defined as increase in size of prior sites of disease or evidence of new sites of disease. (NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib24
Tacrolimus/Methotrexate/Maraviroc31
Tacrolimus/MMF/Cyclophosphamide28

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Percentage of Participants With Disease-free Survival

Disease-free survival is defined as being alive and free of disease relapse or progression. (NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib58
Tacrolimus/Methotrexate/Maraviroc56
Tacrolimus/MMF/Cyclophosphamide60

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Percentage of Participants With Grade II-IV Acute GVHD

"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL~2-3 mg/dL~3.01-6 mg/dL~6.01-15.0 mg/dL~>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT02208037)
Timeframe: Day 180 Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib26
Tacrolimus/Methotrexate/Maraviroc32
Tacrolimus/MMF/Cyclophosphamide27

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Percentage of Participants With Grade III-IV Acute GVHD

"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL 1.2-3 mg/dL 2.3.01-6 mg/dL 3.6.01-15.0 mg/dL 4.>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT02208037)
Timeframe: Day 180 Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib8
Tacrolimus/Methotrexate/Maraviroc9
Tacrolimus/MMF/Cyclophosphamide2

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Percentage of Participants With GVHD-free Survival

GVHD-free survival is defined as being alive without previous onset of Grade III-IV acute GVHD or chronic GVHD requiring immunosuppressive therapy. (NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib43
Tacrolimus/Methotrexate/Maraviroc34
Tacrolimus/MMF/Cyclophosphamide53

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Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS)

GRFS is defined as being free of grade III-IV acute GVHD onset, chronic GVHD onset requiring systemic immunosuppressive therapy, disease relapse or progression, and death from any cause. (NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib35.5
Tacrolimus/Methotrexate/Maraviroc27.2
Tacrolimus/MMF/Cyclophosphamide44.1

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Percentage of Participants With Overall Survival

(NCT02208037)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Tacrolimus/Methotrexate/Bortezomib68
Tacrolimus/Methotrexate/Maraviroc66
Tacrolimus/MMF/Cyclophosphamide71

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Percentage of Participants With Neutrophil Recovery

Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ 500/mm^3 for three consecutive measurements on three different days. (NCT02208037)
Timeframe: Days 28 and 100 Post-transplant

,,
Interventionpercentage of participants (Number)
Day 28Day 100
Tacrolimus/Methotrexate/Bortezomib9496
Tacrolimus/Methotrexate/Maraviroc9395
Tacrolimus/MMF/Cyclophosphamide9598

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Percentage of Participants With Platelet Recovery

Platelet recovery is defined as the first day of a sustained platelet count >20,000/mm^3 with no platelet transfusion in the preceding seven days. (NCT02208037)
Timeframe: Days 60 and 100 Post-transplant

,,
Interventionpercentage of participants (Number)
Day 60Day 100
Tacrolimus/Methotrexate/Bortezomib9191
Tacrolimus/Methotrexate/Maraviroc9292
Tacrolimus/MMF/Cyclophosphamide9096

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Donor Cell Engraftment

Donor cell engraftment will be assessed with donor/recipient chimerism. Chimerism may be evaluated in bone marrow, whole blood, or CD3 fractions. Full donor chimerism is defined as the presence of ≥ 95% of donor cells as a proportion of total cells. Mixed chimerism is defined as the presence of donor cells, as a proportion of total cells, of < 95% but > 5% in the bone marrow or peripheral blood. Full and mixed chimerism will be evidence of donor cell engraftment. Donor cells of ≤ 5% will be considered as graft rejection. (NCT02208037)
Timeframe: Days 28 and 100 Post-transplant

InterventionParticipants (Count of Participants)
Day 2872130179Day 2872130181Day 2872130180Day 10072130180Day 10072130181Day 10072130179
Full ChimerismMixed ChimerismGraft RejectionDeadNo Assay Performed
Tacrolimus/Methotrexate/Bortezomib58
Tacrolimus/MMF/Cyclophosphamide64
Tacrolimus/Methotrexate/Bortezomib11
Tacrolimus/MMF/Cyclophosphamide8
Tacrolimus/Methotrexate/Bortezomib1
Tacrolimus/Methotrexate/Maraviroc1
Tacrolimus/MMF/Cyclophosphamide2
Tacrolimus/Methotrexate/Bortezomib2
Tacrolimus/Methotrexate/Maraviroc4
Tacrolimus/MMF/Cyclophosphamide0
Tacrolimus/Methotrexate/Bortezomib17
Tacrolimus/Methotrexate/Maraviroc21
Tacrolimus/MMF/Cyclophosphamide18
Tacrolimus/Methotrexate/Bortezomib63
Tacrolimus/Methotrexate/Maraviroc56
Tacrolimus/MMF/Cyclophosphamide62
Tacrolimus/Methotrexate/Bortezomib14
Tacrolimus/Methotrexate/Maraviroc17
Tacrolimus/MMF/Cyclophosphamide13
Tacrolimus/Methotrexate/Bortezomib5
Tacrolimus/Methotrexate/Maraviroc3
Tacrolimus/MMF/Cyclophosphamide3
Tacrolimus/Methotrexate/Bortezomib4
Tacrolimus/Methotrexate/Maraviroc10
Tacrolimus/MMF/Cyclophosphamide5
Tacrolimus/Methotrexate/Bortezomib3
Tacrolimus/Methotrexate/Maraviroc6
Tacrolimus/MMF/Cyclophosphamide9

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Clinical Response

"Patients will be followed according to response criteria as referenced in BMT SOP Standards of Therapy last updated 2008. Clinical Response = CR + PR.~Complete Response Requires all of the following:~Serum and urine negative for monoclonal proteins by immunofixation~Normal free light chain ratio~Plasma cells in marrow < 5%~Partial Response (PR) Requires any of the following:~- ≥ 50% reduction in current serum monoclonal protein levels > 0.5 g/dL or urine light chain levels > 100 mg/day with a visible peak or free light chain levels > 10mg/dL~Progressive Disease (PD) Requires any of the following:~If progressing from CR, any detectable monoclonal protein or abnormal free light chain ratio (light chain must double)~If progressive from PR or SD, ≥ 50% increase in the serum M protein to > 0.5 g/dL,or ≥ 50% increase in urine M protein to > 200mg/day with visible peak present.~Free light chain increase of ≥ 50% to" (NCT01529827)
Timeframe: In the first 100 days from day 0 of transplant

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic PBSCT)45

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Median Time to Neutrophil Engraftment

Median time to recovery of absolute neutrophil count >=500/uL for 3 consecutive days. Summarized using standard descriptive statistics along with corresponding 95% confidence intervals. (NCT01529827)
Timeframe: Day 100

Interventiondays (Median)
Treatment (Reduced Intensity Allogeneic PBSCT)17

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Progression Free Survival (PFS) at One Year

Assessed using Kaplan Meier and Proportional Hazards (NCT01529827)
Timeframe: day of transplant until progression up to 5 years

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic PBSCT)85

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Number of Days for Absolute Neutrophil Count to Recover

Average number of day per patient for absolute neutrophil count to recover(> 500/mm3 for 3 consecutive days). (NCT00594308)
Timeframe: From Day -1 (day before stem cell infusion) to Day+20 (20 days after stem cell infusion)

Interventiondays per patient (Mean)
Basiliximab 20 mg14.00

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Number of Patients Engrafting at Day +30 by Short Tandem Repeat (STR) on Peripheral Blood Mononuclear Cells (PBMC's).

(NCT00594308)
Timeframe: until 30 days after stem cell transplant

Interventionparticipants (Number)
Basiliximab 20 mg10

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Number of Patients With Acute Grade II-IV GVHD

Number of patients with Grade II-IV GVHD according to NMDP/CIBMTR GVHD severity scale. This scale measures the degree of GVHD involvement in the patient's skin (inflammatory skin disease), liver (bilirubin levels) and intestinal tract (amount of diarrhea) as well as the level of decline in a patient's activity and physical abilities. (NCT00594308)
Timeframe: until 30 days after stem cell transplant

Interventionparticipants (Number)
Basiliximab 20 mg10

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Time to Resolution of Cytopenias: Platelet Transfusion Independence

Average number of days per patient for resolution of cytopenias. (NCT00594308)
Timeframe: From Day -1 (day before stem cell infusion) to Day +20 (20 days after stem cell infusion)

Interventiondays per patient (Mean)
Basiliximab 20 mg15.33

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Percentage of Participants Alive at 1 Year After Transplant

One-year survival rate after transplant (NCT00763490)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Double Cord Blood Tranplant40

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Percentage of Patients Alive at the End of the Trial

Event Free Survival (EFS) was determined. Patients were followed up to 5 years (median time of 2.35 years). (NCT00763490)
Timeframe: 5 Years

Interventionpercentage of patients (Number)
Double Cord Blood Tranplant35

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Cumulative Incidence of Neutrophil and Platelet Engraftment

The failure to achieve a neutrophil count > 500/uL or a platelet count >30.0 x 10e9 /L within 35 days of the stem cell infusion will be defined as primary engraftment failure. (NCT00763490)
Timeframe: Day 35

Interventionpercentage of participants (Number)
Cumulative incidence of platlet engraftmentCumulative incidence of neutrophil engraftment
Double Cord Blood Tranplant7389

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Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)

"The percentage of patients with acute GVHD (Grade II-IV) was determined at 100 days. Patients were followed up to 5 years and the percentage of patients that developed chronic GVHD at the end of the study was tabulated.~Acute GVHD is staged and graded (grade 0-IV, where grade 0 is no involvement and involvement increases by grade) by the number and extent of organ involvement. Patients can have involvement of three organs: skin (rash/dermatitis), liver (hepatitis/jaundice), and gastrointestinal tract (abdominal pain/diarrhea)." (NCT00763490)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
Acute GVHD (Grades II-IV)Chronic GVHD
Double Cord Blood Tranplant4035

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Number of Non-Relapse Mortalities

Number of patients expired without disease progression/relapse. (NCT01251575)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Treatment (Fludarabine, Transplant, Immunosuppression)3

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Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD)

"Number of patients with grades II-IV acute GVHD~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT01251575)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Class I Mismatch15
Class II Mismatch12

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Number of Patients With Grade III-IV Acute GVHD

"Number of patients with grades III-IV acute GVHD~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT01251575)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Treatment (Fludarabine, Transplant, Immunosuppression)2

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Number of Participants With Event-free Survival (EFS)

"The Kaplan-Meier estimate of EFS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) - date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not experience any events defined above is provided." (NCT02199041)
Timeframe: One year after transplantation

InterventionParticipants (Count of Participants)
Treatment4

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Number of Participants With Malignant Relapse

"Relapse was evaluated using standard World Health Organization (WHO) criteria for each disease. The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Relapse defined as the recurrence of original disease. Death is the competing risk event. The analysis will be implemented using Statistical Analysis System (SAS) macro (bmacro252-Excel2007cin).~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced malignant relapse is provided" (NCT02199041)
Timeframe: One year after transplantation

InterventionParticipants (Count of Participants)
Treatment7

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Number of Participants With Neutrophil Engraftment

Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10^9/L (500/mm^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided. (NCT02199041)
Timeframe: Until day 42 post-transplant

InterventionParticipants (Count of Participants)
Treatment11

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Number of Participants With Overall Survival (OS)

"The Kaplan-Meier estimate of OS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where OS = min (date of last follow-up, date of death) - date of hematopoietic cell transplantation (HCT) and all participants surviving after 1 year post-transplant will be considered as censored.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not die at 1 year post-transplant is provided." (NCT02199041)
Timeframe: One year after transplantation

InterventionParticipants (Count of Participants)
Treatment6

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Number of Participants With Secondary Graft Failure

"The cumulative incidence of secondary graft failure will be estimated using the Kalbfleisch-Prentice method. Deaths due to toxicity and relapse before day 100 are the competing events.~Secondary graft failure or graft rejection will be defined as no evidence of donor chimerism by umbilical cord blood (UCB) and/or haploidentical donor (<10%), or too few cells to perform adequate chimerism analysis, in research participants with prior neutrophil engraftment.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced secondary graft failure is provided." (NCT02199041)
Timeframe: 100 days after transplantation

InterventionParticipants (Count of Participants)
Treatment0

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Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT

"Cumulative incidence of acute GVHD was estimated using Kalbfleisch-Prentice method. Death is the competing risk event. SAS macro (bmacro252-Excel2007cin) available at St. Jude was used for analysis. Severity of GVHD and stage were determined using the Clinical Oncology Group (COG) Stem Cell Committee Consensus Guidelines for establishing organ stage and overall grade of acute GVHD. Participants are graded on a scale from I to IV, with I being mild and IV being severe.~Overall Clinical Grade (based on the highest stage obtained):~Grade 0: No stage 1-4 of any organ. Grade I: Stage 1-2 skin and no liver or gut involvement. Grade II: Stage 3 skin, or Stage I liver involvement, or Stage 1 gastrointestinal (GI).~Grade III: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI. Grade IV: Stage 4 skin, liver or GI involvement.~Due to early close of study, a small number of patients were enrolled. The number of patients who experienced acute GVHD is provided." (NCT02199041)
Timeframe: 100 days after transplantation

InterventionParticipants (Count of Participants)
No Acute GVHDGrade IGrade IIGrade IIIGrade IV
Treatment80121

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Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT

"Cumulative incidence of acute and chronic GVHD was estimated using Kalbfleisch-Prentice method. Death is competing risk event. SAS macro (bmacro252-Excel2007cin) available St. Jude was used for such analysis. Severity of chronic GVHD was evaluated using National Institutes of Health (NIH) Consensus Global Severity Scoring. Mild is considered a better outcome with severe being the worst.~Criteria for grading chronic GVHD:~Mild: 1-2 organs/sites, maximum organ score of 1, and lung score of 1. Moderate: 3 or more organs/sites and maximum organ score of 1 and lung score of 1, OR at least 1 organ/site and maximum organ score of 2 and lung score of 1. Severe: At least 1 organ/site and maximum organ score of 3 and lung score of 2-3.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced chronic GVHD is provided." (NCT02199041)
Timeframe: 100 days after transplantation

InterventionParticipants (Count of Participants)
No Chronic GVHDMildModerateSevere
Treatment11001

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Disease-Free Survival (DFS)

1-year post-transplant disease free survival (DFS), defined as success if a patient is alive and disease free at 1-year post-transplant. (NCT01350245)
Timeframe: 1 year post-transplant

Interventionpercentage of patients (Number)
TJU 2 Step Regimen78.6

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Probability of Overall Survival at 15 Months Post-treatment

Probability of overall survival at 15 months post-treatment, defined as success if a patient is alive 1-year post-transplant. (NCT01350245)
Timeframe: 15 months

Interventionpercentage of probability (Number)
TJU 2 Step Regimen85

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Overall Survival (OS)

Percentage of participants alive at 3 years. (NCT01490723)
Timeframe: From date of treatment to date of relapse or death, up to 3 years

InterventionParticipants (Count of Participants)
Yttrium-90 Ibritumomab + Chemo14

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Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) (Calculated With Modified Diet in Renal Disease (MDRD)-4 Formula - ITT

Renal function was assessed by measuring serum creatinine and serum urea and by calculating creatinine clearance using the MDRD-4 formula. eGFR = 186.3*(serum creatinine [mg/dL])^-1.154 * (age at screening) -0.203 * (0.742 if female) * (1.21 if African American). A positive change from baseline indicates improvement. (NCT01410448)
Timeframe: baseline, 3 Months

InterventionmL/min (Mean)
Immediate Everolimus (IE)38.64
Delayed Everolimus (DE)39.13

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Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) (Calculated With Modified Diet in Renal Disease (MDRD)-4 Formula - Modified ITT

Renal function was assessed by measuring serum creatinine and serum urea and by calculating creatinine clearance using the MDRD-4 formula. eGFR = 186.3*(serum creatinine [mg/dL])^-1.154 * (age at screening) -0.203 * (0.742 if female) * (1.21 if African American). A positive change from baseline indicates improvement. (NCT01410448)
Timeframe: baseline, 12 months

InterventionmL/min (Mean)
Immediate Everolimus (IE)41.26
Delayed Everolimus (DE)41.56

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Change From Baseline in Serum Creatinine - ITT

Blood samples were collected to assess serum creatinine measurements. A negative change from baseline indicates improvement. (NCT01410448)
Timeframe: baseline, 3 months

Interventionmg/dL (Mean)
Immediate Everolimus (IE)-4.79
Delayed Everolimus (DE)-5.13

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Change From Baseline in Serum Creatinine - Modified ITT

Blood samples were collected to assess serum creatinine measurements. A negative change from baseline indicates improvement. (NCT01410448)
Timeframe: baseline, 12 months

Interventionmg/dL (Mean)
Immediate Everolimus (IE)-4.96
Delayed Everolimus (DE)-5.22

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Duration of DGF

The duration of DGF was defined as the elapsed time from first to last day of post-transplant dialysis. (NCT01410448)
Timeframe: 3 months

InterventionDays (Median)
Immediate Everolimus (IE)8.50
Delayed Everolimus (DE)5.50

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Participant/Graft Survival Rate: Percentage of Participants With Failure Events of Death or Graft Loss - Worst-case Scenario

The percentage of participants who experienced death or graft loss was assessed. In the worst-case scenario, failure, i.e. participants death or graft loss, was identified in one of the following cases: occurrence of at least one failure event or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)6.74
Delayed Everolimus (DE)18.42

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Patient Survival Rate: Percentage of Deaths - Worst-case Scenario

The percentage of deaths was assessed. In the worst-case scenario, failure, i.e. death, was identified in one of the following cases: participant's death or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 Months, 12 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)6.22
Delayed Everolimus (DE)18.42

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Percentage of Participants Who Experienced Treatment Failure - Worst-case Scenario

The percentage of participants who experienced treatment failure was assessed. Treatment failure was defined as the occurrence of at least one failure event among death, graft loss or biopsy-proven acute rejection (BPAR). In the worst-case scenario, treatment failure was identified in one of the following cases: occurrence of at least one treatment failure event or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)11.40
Delayed Everolimus (DE)21.05

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Percentage of Participants With a New Onset of Diabetes

The percentage of participants with a new onset of diabetes was assessed. (NCT01410448)
Timeframe: 12 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)3.14
Delayed Everolimus (DE)4.05

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Percentage of Participants With a New Onset of Malignancy

The percentage of participants with a new onset of malignancy was assessed. (NCT01410448)
Timeframe: 12 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)0
Delayed Everolimus (DE)0.68

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Percentage of Participants With Acute Rejection (AR)

AR was defined as an episode of increased serum creatinine >30% that was clinically diagnosed as an acute rejection but was not biopsy proven. (NCT01410448)
Timeframe: 12 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)12.44
Delayed Everolimus (DE)10.53

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Percentage of Participants With Delayed Graft Function (DGF) -

DGF was defined as the need for dialysis in the first week after transplant, excluding Renal Replacement Therapy within the first 24 hours after transplantation. (NCT01410448)
Timeframe: 3 Months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)23.83
Delayed Everolimus (DE)31.58

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Percentage of Participants Without Wound Healing Complications - Worst-case Scenario

The percentage of participants without wound healing complications was assessed. Wound healing complications consisted of lymphorrhea, fluid collections, wound dehiscence, wound infections and incisional hernia. In the worst-case scenario, failure, i.e. at least one healing complication occurrence, was identified in one of the following cases: wound complication occurrence, missing information about wound complication occurrence or study discontinuation due to any reason for participants who did not complete the 12 month follow-up visit. (NCT01410448)
Timeframe: 12 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)65.80
Delayed Everolimus (DE)59.47

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Percentage of Participants Without Wound Healing Complications - Worst-case Scenario

The percentage of participants without wound healing complications was assessed. Wound healing complications consisted of lymphorrhea, fluid collections, wound dehiscence, wound infections and incisional hernia. In the worst-case scenario, failure, i.e. at least one healing complication occurrence, was identified in one of the following cases: wound complication occurrence, missing information about wound complication occurrence, or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months

InterventionPercentage of participants (Number)
Immediate Everolimus (IE)68.39
Delayed Everolimus (DE)61.58

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Graft Survival Rate: Percentage of Participants With Graft Loss - Worst-case Scenario

The percentage of participants who experienced graft loss was assessed. In the worst-case scenario, failure, i.e. graft loss, was identified in one of the following cases: occurrence of graft loss or discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months, 12 months

,
InterventionPercentage of participants (Number)
3 months12 months
Delayed Everolimus (DE)18.4219.47
Immediate Everolimus (IE)6.747.25

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Percentage of Participants With BPAR - Worst-case Scenario

A biopsy-proven acute rejection was defined as a biopsy graded IA, IB, IIA, IIB or III. In the worst-case scenario, failure, i.e. BPAR, was identified in one of the following cases: occurrence of BPAR or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 Months, 12 months

,
InterventionPercentage of participants (Number)
3 monts12 months
Delayed Everolimus (DE)21.0524.74
Immediate Everolimus (IE)11.4015.54

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Percentage of Participants With Proteinuria

Incidence of proteinuria (>1,000 mg/day in urine collected in 24 hours or > 1.0 if measured on the urine protein/creatinine concentration ratio in a spot urine sample) was assessed. (NCT01410448)
Timeframe: 3 months

,
InterventionPercentage of participants (Number)
YesNoMissing
Delayed Everolimus (DE)4.2168.4227.37
Immediate Everolimus (IE)4.1568.9126.94

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Cholesterol

(NCT01624948)
Timeframe: 3 months post-randomization

Interventionmg/dL (Mean)
Everolimus+Tacrolimus/Prednisone212
Standard of Care: 50% Reduction of MPA170

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Evaluation for the Development of BK Virus Nephropathy or Doubling of BK Viremia Levels

A doubling of BK viremia levels or the development of BKV nephropathy in subjects enrolled in the experimental study arm will prompt a conversion to standard care therapy. We will closely monitor BKV levels in the urine and blood and assess renal function monthly, as per our usual standard of care. Based on BKV results as well as renal function, as assessed by serum Cr, biopsies may be done for cause. Patients will have a final visit at month 4 to monitor for adverse events. (NCT01624948)
Timeframe: 3 months post-randomization

Interventionparticipants (Number)
Everolimus+Tacrolimus/Prednisone2
Standard of Care: 50% Reduction of MPA2

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Evidence of Reduction of BK Viruria and/or Clearance of BK Viremia

composite outcome of a 50% or greater reduction in BKV urine levels and/or complete clearance of BKV viremia by 3 months after randomization (NCT01624948)
Timeframe: 3 months post-randomization

Interventionparticipants (Number)
Everolimus+Tacrolimus/Prednisone11
Standard of Care: 50% Reduction of MPA8

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Median Between the Calculated Mean Residual Expression of NFAT-regulated Genes

"Measurement of the expression of three NFAT-regulated genes IL-2, interferon gamma, and GM-CSF, to predict a rejection episode.~The residual gene expression after Tacrolimus intake was calculated as T1.5/T0*100, where T0 is the adjusted number of transcripts at Tacrolimus pre-dose level and T1.5 is the number of transcripts 1.5 hours after drug intake. For all three genes the residual expression was averaged and presented as MRE of NFAT-regulated genes." (NCT01624948)
Timeframe: 3 months post-randomization

Interventionpercent residual expression (Median)
Rejection46.35
No Rejection29.12

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p70S6 Kinase Phosphorylation

(NCT01624948)
Timeframe: 3 months post-randomization

InterventionMean Fluorescence Intensity (Median)
Reached Primary Endpoint5002
Failed to Reach Primary Endpoint4353

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Proteinuria

(NCT01624948)
Timeframe: 3 months post-randomization

Interventiong/g creatinine (Mean)
Everolimus+Tacrolimus/Prednisone0.16
Standard of Care: 50% Reduction of MPA0.23

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Complete Remission (CR) Rate at Day 30 Post HSCT

The CR rate at 30 days (Day +30) post stem cell transplant infusion (NCT04002115)
Timeframe: 30 days

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^2100

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Neutrophil Engraftment

Rates of engraftment, defined as the first day of Absolute Neutrophil Count (ANC) greater than 500 for the first of three consecutive days (NCT04002115)
Timeframe: 1 year

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Rate of Acute Graft-versus-host Disease (GVHD)

The rate of any grade (1-4) of acute GvHD as measured from day of transplantation to Day +100 using the Glucksberg criteria. (NCT04002115)
Timeframe: 100 days

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Rate of Chronic GVHD

The rate of any grade (1-4) of Chronic GvHD as measured from Day +100 to Year 1 post-transplantation using the Glucksberg criteria. (NCT04002115)
Timeframe: 1 year

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Severity of Acute Graft-versus-host Disease (GVHD)

The highest grade (1-4) of acute GvHD experienced by participants as measured from day of transplantation to Day +100 using the Glucksberg criteria (NCT04002115)
Timeframe: 100 days

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Severity of Chronic GVHD

The highest overall grade (1-4) of chronic GvHD experienced by participants as measured from Day +100 to Year 1 post-transplantation using the Glucksberg criteria (NCT04002115)
Timeframe: 1 year

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Number of Patients Reported Ascites

(NCT01903798)
Timeframe: Week 24

InterventionParticipants (Count of Participants)
Continue Prednisolone (Lille <0.45)0
Rilonacept + Prednisolone (Lille <0.45)0
Standard of Care (Lille ≥ 0.45)1
Mycophenolate + Prednisolone (Lille ≥ 0.45)0

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Survival at Day 29 of the Assigned Treatment

"To determine whether treatment with prednisolone + mycophenolate mofetil is better than standard of care treatment among patients with alcoholic hepatitis who fail to respond to 1 week of prednisolone (i.e., Lille score of ≥0.45). Primary outcome is survival at Day 29.~All study participants received the Standard of care (prednisolone) with or without experimental drug at Day 1 (based on randomization). Response to the treatment was determined at Day 8. Data was collected for both responders and non-responders." (NCT01903798)
Timeframe: Day 8 to Day 29

InterventionParticipants (Count of Participants)
Continue Prednisolone (Lille <0.45)2
Rilonacept + Prednisolone (Lille <0.45)0
Standard of Care (Lille ≥ 0.45)1
Mycophenolate + Prednisolone (Lille ≥ 0.45)1

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Incidence of Cytomegalovirus Infection or Disease

Incidence of CMV infection/disease in three study groups (SRL, EVR anda MPS). (NCT03468478)
Timeframe: 12 months follow up

InterventionParticipants (Count of Participants)
Sirolimus +Tacrolimus9
Everolimus +Tacrolimus7
Mycophenolate +Tacrolimus39

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BPAR (Biopsy-proven Acute Rejection) Incidence During the First 12 Months Post-transplant

BPAR (biopsy-proven acute rejection) incidence during the first 12 months post-transplant. Grading is determined using standard Banff criteria. (NCT01680861)
Timeframe: 1 year

Interventionparticipants (Number)
Tacrolimus/Everolimus1
Tacrolimus/EC-MPS3

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Discontinuance of Any Study Medication (Tacrolimus, Everolimus, or EC-MPS)

(NCT01680861)
Timeframe: during the first 12 months post-transplant

Interventionparticipants (Number)
Tacrolimus/Everolimus0
Tacrolimus/EC-MPS1

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eGFR (Calculated Glomerular Filtration Rate), i.e., Renal Function, at 1 Month Post-transplant.

using the abbreviated MDRD formula. (NCT01680861)
Timeframe: at 1 month post-transplant

Interventionml/min per 1.73 m2 (Mean)
Tacrolimus/Everolimus82.1
Tacrolimus/EC-MPS62.1

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eGFR (Renal Function) at 6 Months Post-transplant

using the abbreviated MDRD formula. (NCT01680861)
Timeframe: at 6 months post-transplant

Interventionml/min per 1.73 m2 (Mean)
Tacrolimus/Everolimus66.7
Tacrolimus/EC-MPS63.7

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eGFR (Renal Function) at Month 3 Post-transplant

Renal function as determined by the estimated glomerular filtration rate (eGFR) at 3 months post-transplant, using the abbreviated MDRD formula. (NCT01680861)
Timeframe: at 3 months post-transplant

Interventionml/min per 1.73 m^2 (Mean)
Tacrolimus/Everolimus75.7
Tacrolimus/EC-MPS65.6

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Graft Loss (Return to Permanent Dialysis or Death)

(NCT01680861)
Timeframe: during the first 12 months post-transplant

Interventionparticipants (Number)
Tacrolimus/Everolimus0
Tacrolimus/EC-MPS0

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Incidence of Chronic Allograft Nephropathy (CAI) at 12 Months Post-transplant

Incidence of (biopsy-proven) chronic allograft nephropathy (CAI) [interstitial fibrosis and tubular atrophy, using standard Banff criteria] at 12 months post-transplant. (NCT01680861)
Timeframe: 1 year

Interventionparticipants (Number)
Tacrolimus/Everolimus3
Tacrolimus/EC-MPS3

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Chronic Graft Versus Host Disease (GVHD)

"Percentage of participants who developed chronic GVHD as defined by the NIH consensus criteria. This system gives scores from 0 to 3 for Karnofsky performance score, skin, mouth, eyes, gastrointestinal, liver, lungs, joints, and genitals, as well as an overall severity (mild, moderate, or severe).~Mild chronic GVHD involves only 1 or 2 organs or sites (except the lung), with no clinically significant functional impairment (maximum of score 1 in all affected organs or sites).~Moderate chronic GVHD involves 1) at least 1 organ or site with clinically significant but no major disability (maximum score of 2 in any affected organ or site) OR 2) 3 or more organs or sites with no clinically significant functional impairment (maximum score of 1 in all affected organs or sites).~Severe chronic GVHD indicates major disability caused by chronic GVHD (score of 3 in any organ or site)." (NCT02080195)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Nonmyeloablative Conditioning and BMT0

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The Feasibility of the Conditioning Regimen and Post Transplantation Cyclophosphamide in Refractory SLE Patients With Donors Having Various Degrees of Matching

Number of participants who were alive at 1 year after transplant and who had not suffered graft rejection, acute or chronic GVHD, or Grade 3 or higher (CTCAE V4.0) adverse events. (NCT02080195)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Nonmyeloablative Conditioning and BMT1

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Acute Graft Versus Host Disease (GVHD)

Percentage of participants who developed grades II-IV and grades III-IV acute GVHD. Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade I (skin involvement stages 1+ to 2+, with no liver or GI involvement), Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+). (NCT02080195)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Grades II-IV acute GVHDGrades III-IV acute GVHD
Nonmyeloablative Conditioning and BMT00

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Graft Failure

Number of participants with primary and/or secondary graft failure. (NCT02080195)
Timeframe: 60 days

InterventionParticipants (Count of Participants)
Primary graft failureSecondary graft failure
Nonmyeloablative Conditioning and BMT00

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Survival

Number of patients alive and alive without relapse, respectively. (NCT02080195)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Overall SurvivalEvent Free Survival
Nonmyeloablative Conditioning and BMT11

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Median Time to ANC > 500

(NCT00723099)
Timeframe: By day 55

Interventiondays (Median)
Treatment (Chemotherapy, Transplant)18

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Number of Participants With Graft Failure/Rejection

descriptive (NCT00723099)
Timeframe: By day 55

Interventionparticipants (Number)
Treatment (Chemotherapy, Transplant)3

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Overall Survival

Kaplan-Meier and cumulative incidence estimates will be used. (NCT00723099)
Timeframe: At 1 year

Interventionpercent of patients (Number)
Treatment (Chemotherapy, Transplant)35

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Percent of Patients With Acute GVHD Grades III-IV

Fischer's exact test was used to determined percent of patients with acute grade III-IV GVHD by Glucksberg criteria (NCT00723099)
Timeframe: 100 days

Interventionpercent of patients (Number)
Treatment (Chemotherapy, Transplant)12

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Percent of Patients With Chronic GVHD

Kaplan-Meier and cumulative incidence estimates will be used to measure percent of patients with chronic GVHD by NIH consensus criteria. (NCT00723099)
Timeframe: At 2 years

Interventionpercent of patients (Number)
Treatment (Chemotherapy, Transplant)19

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Percent of Patients With Grade II-IV Acute Graft Versus Host Disease

Chi-square test was used to determine percent of grade II-IV GVHD using Glucksberg criteria (NCT00723099)
Timeframe: By day 100

Interventionpercent of patients (Number)
Treatment (Chemotherapy, Transplant)67

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Percent of Patients With Non-relapse Mortality

Kaplan-Meier and cumulative incidence estimates (NCT00723099)
Timeframe: 1 year

Interventionpercent of patients (Number)
Treatment (Chemotherapy, Transplant)38

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Percent of Patients With Non-relapse Mortality

Kaplan-Meier and cumulative incidence estimates (NCT00723099)
Timeframe: 6 months

Interventionpercent of patients (Number)
Treatment (Chemotherapy, Transplant)21

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Time to Platelet Engraftment of > 20,000 Cells Per mm3

median and range (NCT00723099)
Timeframe: By 6 months

Interventiondays (Median)
Treatment (Chemotherapy, Transplant)46

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Engraftment of HLA Identical PBSC Allografts

"Number of patients who engrafted by Day 56 post allogeneic transplant. Failure to engraft is defined as the absence of detectable donor cells in the marrow. The rates and accompanying confidence intervals associated with failure of engraftment at day +56 will be calculated after every 5th patient is enrolled on the study. If the lower limit to the appropriate one-sided 80% confidence interval exceeds 25%, this will be considered sufficient evidence of an excess failure rate and the study will be stopped. For these purposes, all patients will be evaluated together (patients with chemosensitive and chemoresistant disease)." (NCT00005803)
Timeframe: Day 56

InterventionParticipants (Count of Participants)
Treatment (Tandem Transplantation)53

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Non-Relapse Mortality

"The rates and accompanying confidence intervals associated with transplant-related mortality will be calculated after every 5th patient is enrolled on the study. If the lower limit to the appropriate one-sided 80% confidence interval exceeds 25%, this will be considered sufficient evidence of an excess failure rate and the study will be stopped. For these purposes, all patients will be evaluated together (patients with chemosensitive and chemoresistant disease)." (NCT00005803)
Timeframe: Day 100 post-non-myeloablative allografting following mobilization and high-dose chemotherapy with autografting

InterventionParticipants (Count of Participants)
Treatment (Tandem Transplantation)3

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Overall Survival (OS)

Number of patients surviving by interval. Chemosensitive and chemoresistant subjects will be analyzed separately. (NCT00005803)
Timeframe: From the date of autologous transplant until the time of death, assessed up to 3 years

,,
InterventionParticipants (Count of Participants)
6 Months1 Year1.5 Years2 Years3 Years
Chemoresistant Group1410988
Chemosensitive Group3931272623
Unknown Chemosensitivity Group10000

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Progression Free-survival (PFS)

Number of patients surviving without disease by interval. Chemosensitive and chemoresistant subjects will be analyzed separately. (NCT00005803)
Timeframe: From the date of autologous transplant until the time of progression, relapse, death, or the date the patient was last known to be in remission, assessed up to 3 years

,,
InterventionParticipants (Count of Participants)
6 Months1 Year1.5 Years2 Years3 Years
Chemoresistant Group128888
Chemosensitive Group3627252522
Unknown Chemosensitivity Group10000

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Acute Graft-Versus-Host Disease (aGVHD) Outcome

"Grading of Acute GVHD:~Severity of Individual Organ Involvement:~Skin~1 a maculopapular eruption involving less than 25% of the body surface~2 a maculopapular eruption involving 25-50% of the body surface~3 generalized erythroderma~4 generalized erythroderma with bullous formation and/or with desquamation Liver~1 bilirubin 2.0-3.0mg/100mL~2 bilirubin 3-5.9mg/100mL~3 bilirubin 6-14.9mg/100mL~4 bilirubin >15mg/100mL Gut Diarrhea is graded +1 to +4 in severity. Nausea/vomiting and/or anorexia caused by GVHD is assigned as +1 in severity Diarrhea~1 <1000mL of liquid stool/day~2 >1,000mL of stool/day~3 >1,500mL of stool/day~4 2,000mL of stool/day, severe abdominal pain, with or without ileus~Severity of GVHD:~Grade 1 +1 to +2 skin rash; No gut or liver involvement Grade 2 +1 to +3 skin rash;+1 GI involvement and/or +1 liver" (NCT00245037)
Timeframe: Day 100, Month 6

Interventionpercentage of analyzed participants (Number)
Day 100Month 6
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)5560

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Chronic Graft-Versus-Host Disease (cGVHD) Outcome

"Grading of Chronic GVHD:~Limited: Localized skin involvement and/or hepatic dysfunction due to chronic GVHD~Extensive:~One or more of the following:~Generalized skin involvement Liver histology showing chronic aggressive hepatitis, bridging necrosis or cirrhosis Involvement of the eye: Schirmer's test with <5 mm wetting Involvement of minor salivary glands or oral mucosa demonstrated on labial biopsy Involvement of any other target organ~Chronic GVHD Severity:~Mild: Signs and symptoms of cGVHD do not interfere substantially with function and do not progress once appropriately treated with local therapy or standard systemic therapy.~Moderate: Signs and symptoms of cGVHD interfere somewhat with function despite appropriate therapy or are progressive through first line systemic therapy.~Severe: Signs and symptoms of cGVHD limit function substantially despite appropriate therapy or are progressive through second line systemic therapy" (NCT00245037)
Timeframe: Years 1, 2 and 3

Interventionpercentage of analyzed participants (Number)
Year 1Year 2Year 3
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)64.66667.3

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Non-relapse Mortality

Percent of subjects with non-relapse mortality two years after conditioning with busulfan with fludarabine/200 cGy TBI in patients with hematologic malignancies at moderate to high risk for graft rejection and/or relapse of underlying disease. (NCT00245037)
Timeframe: Two years post-transplant

InterventionParticipants (Count of Participants)
Year 1Year 2
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)2733

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Overall Survival

The percentage of overall patient survival (out of 147 participants) for Years 1, 2, 3 and 5. (NCT00245037)
Timeframe: Years 1, 2, 3 and 5

Interventionpercentage of analyzed participants (Number)
Year 1Year 2Year 3Year 5
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)60484229

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Progression-Free Survival

"The percentage of progression-free patients (out of 147 participants) at Years 1, 2, 3, and 5.~Definition of Disease Progression:~MM/Plasma Cell: Increasing bone pain or increase in serum/urine monoclonal protein by 25%.~CLL/NHL/HD: New sites of lymphadenopathy; ≥ 25% increase in lymph node size; Blood or bone marrow involvement with clonal B-cells; Increase of ≥ 25% bone marrow involvement; ≥ 25% increase in blood involvement with clonal B-cells.~AML/ALL: Any incidence of relapse (>5% blasts) by evaluation of the bone marrow aspirate.~CML: Inability to control platelet or granulocyte counts; Increase in baseline number of metaphases demonstrating the Ph+ chromosome by >25%; Any other new cytogenetic abnormality; Transformation to accelerated phase or blast crisis.~MDS/MPD: Any evidence by morphologic or flow cytometric evaluation of the bone marrow aspirate of new blasts (>5%) or worsening cytopenia or cytogenetic evidence of recurrence." (NCT00245037)
Timeframe: Years 1, 2, 3, and 5

Interventionpercentage of analyzed participants (Number)
Year 1Year 2Year 3Year 5
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)48393529

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Relapse Mortality

The percentage of patients (out of 147 participants) who relapsed at Years 1 and 2. Relapse is defined as the presence of >5% blasts by morphology on a post-transplant bone marrow aspirate. (NCT00245037)
Timeframe: Years 1 and 2

Interventionpercentage of analyzed participants (Number)
Year 1Year 2
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)1320

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Participants Experiencing Acute Rejection Episode

(NCT02123108)
Timeframe: 12 months post liver transplant

InterventionParticipants (Count of Participants)
Basiliximab3
Tacrolimus Group4

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Participants Experiencing Adverse Event Attributable to Study Drug

(NCT02123108)
Timeframe: 12 months post liver transplantation

InterventionParticipants (Count of Participants)
Basiliximab0
Tacrolimus Group0

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Participants Experiencing Graft Failure

(NCT02123108)
Timeframe: 12 months post transplant

InterventionParticipants (Count of Participants)
Basiliximab2
Tacrolimus Group5

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Renal Recovery/ Function

Number of participants who experienced dialysis independence or improvement based upon kidney function labs as a measure of renal recovery/ function following OLT in patients after undergoing orthotopic liver transplant (NCT02123108)
Timeframe: 12 months post-transplant

InterventionParticipants (Count of Participants)
Basiliximab28
Tacrolimus Group26

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Survival

Participants at risk minus incidence of death within the first year post-transplant (NCT02123108)
Timeframe: 12 months post liver transplant

InterventionParticipants (Count of Participants)
Basiliximab28
Tacrolimus Group26

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Change From Baseline (Randomization) in Creatinine Clearance Estimated Using the Adjusted Cockcroft-Gault Formula

Creatinine clearance by the Cockcroft-Gault formula is computed in mL/min/1.73m^2 from the creatinine clearance in mL/min by multiplying it by 1.73 and dividing it by the body surface area Baseline was Day 28 visit. (NCT01625377)
Timeframe: Baseline, Week 24

InterventionmL/min/1.73m^2 (Mean)
Tacrolimus-9.0
Everolimus (RAD001)0.7

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Change From Baseline (Randomization) in Glomerular Filtration Rate Estimated by Abbreviated Modification of Diet in Renal Disease (MDRD) Formula

"Change in glomerular filtration rate was calculated using the MDRD abbreviated formula.~GFR in mL/min/1.73m^2 for men of non-black ethnicity: 186 * [C/88]^-1.154 * [A]^-0.023*G*R ; C = serum creatinine (in μmol/L); A = Age (in years). G = 0.742 when the patient is a women; Otherwise G=1 R= 1.21 when the patient was of black ethnicity; Otherwise R = 1 Baseline was Day 28 visit." (NCT01625377)
Timeframe: Baseline, Week 24

InterventionmL/min/1.73m^2 (Mean)
Tacrolimus-11.8
Everolimus (RAD001)0.1

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Change From Baseline (Randomization) in Glomerular Filtration Rate Estimated by CKD-EPI Formula

"GFR estimated by using the Chronic kidney disease- epidemiology (CKD-EPI) formula:~eGFR (mL/min/1.73m^2) = 141 * min(C/K,1)^ α * max(C/K,1)^-1.209 * 0.993^A * 1.1018 (if male) * 1.159 (if black) where C = serum creatinine (in mg/dL) ; A = Age (in years); K = 0.7 for women and 0.9 for men; α = -0.329 for women and -0.411 for men. Baseline was Day 28 visit." (NCT01625377)
Timeframe: Baseline, Week 24

InterventionmL/min/1.73m^2 (Mean)
Tacrolimus-6.9
Everolimus (RAD001)2.4

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Change From Baseline (Randomization) in Renal Function

"Change in renal function was measured by change in glomerular filtration rate (GFR). GFR calculated using the abbreviated modification of diet in renal disease (aMDRD) formula.~GFR in mL/min/1.73m^2 for men of non-black ethnicity: 186 * [C/88]^-1.154 * [A]^-0.023*G*R ; C = serum creatinine (in μmol/L); A = Age (in years). G = 0.742 when the patient is a women; Otherwise G=1 R= 1.21 when the patient was of black ethnicity; Otherwise R = 1 Baseline was Day 28 visit." (NCT01625377)
Timeframe: Baseline, Week 24

InterventionmL/min/1.73m^2 (Least Squares Mean)
Tacrolimus-13.29
Everolimus (RAD001)1.05

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Change From Baseline (Randomization) in Serum Creatinine

"Change in serum creatinine concentrations from baseline (randomization) to week 24 post-randomization was one of the efficacy assessments of renal function.~Baseline was Day 28 visit." (NCT01625377)
Timeframe: Baseline, Week 24

Interventionµmol/L (Mean)
Tacrolimus7.2
Everolimus (RAD001)-1.3

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Change From Baseline (Randomization) in Urine Protein/Creatinine Ratio

Change in urine protein/creatinine ratio from baseline (randomization) to week 24 post-randomization was one of the efficacy assessments of renal function. Baseline was Day 28 visit. (NCT01625377)
Timeframe: Baseline, week 24

Interventionmg/mmol (Mean)
Tacrolimus-2.3
Everolimus (RAD001)21.9

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Number of Patients in Different Stages of Chronic Kidney Diseases According to the K/DOQI Classification System

"Kidney disease outcomes quality initiative (K/DOQI) classification is based on glomerular filtration rate (GFR), abbreviated MDRD formula (mL/min/1.73m^2) :~Stage 1 : GFR >= 90; Stage 2 = GFR was between 60-89; Stage 3 = GFR was between 30-59 ; Stage 4 = GFR was between 15-29; Stage 5 = GFR was < 15 (or dialysis)" (NCT01625377)
Timeframe: At Week 24

,
InterventionPatients (Number)
Stage 1Stage 2Stage 3Stage 4Stage 5
Everolimus (RAD001)4129400
Tacrolimus24342800

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Number of Patients Reported With Different Categories of Severity of BPAR According to Banff Classification

"Biopsy proven acute rejection was defined as a clinically suspected acute rejection confirmed by biopsy.~The severity of BPAR was categorized as :~Mild (Banff grade I, RAI = 4 and 5) Moderate (Banff grade II, RAI = 6 and 7) Severe (Banff grade III, RAI = 8 and 9) Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI) ranging from 0 to 9. An overall score of 0-3 is considered indeterminate, score of 4-5 is mild acute, score of 6-7 is moderate acute , and score of 8-9 is severe acute. Only the episode with the highest total RAI score for each participant was counted." (NCT01625377)
Timeframe: at 12 week and 24 week

,
InterventionPatients (Number)
Week 12: MildWeek 12: ModerateWeek 12: SevereWeek 24: MildWeek 24: ModerateWeek 24: Sever
Everolimus (RAD001)110530
Tacrolimus110110

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Number of Patients With Any Adverse Events, Serious Adverse Events, Death and Premature Discontinuation

Baseline was Day 28 visit. This endpoint reports patients with total adverse events (any), serious adverse events, death and premature discontinuation. (NCT01625377)
Timeframe: Baseline to 24 weeks

,
InterventionPatients (Number)
Any Adverse eventsSerious Adverse eventsDeathAt least one AE led to premature discontinuation
Everolimus (RAD001)8142218
Tacrolimus852814

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Number of Patients With Death or Graft Loss

The graft was presumed to be lost on the day the patient was registered again on the waiting list, or the day he/she received a new graft. (NCT01625377)
Timeframe: at week 24

,
InterventionPatients (Number)
Graft LossDeath
Everolimus (RAD001)01
Tacrolimus11

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Number of Patients With Treated or Not Treated Biopsy Proven Acute Rejection (BPAR)

Biopsy proven acute rejection was defined as a clinically suspected acute rejection confirmed by biopsy. (NCT01625377)
Timeframe: at 12 week and 24 week

,
InterventionPatients (Number)
Week 12, Treated BPARWeek 12, Not treated BPARWeek 24, Treated BPARWeek 24, Not Treated BPAR
Everolimus (RAD001)2081
Tacrolimus2020

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Number of Patients With Treated or Untreated BPAR With RAI Score Greater Than 3

"Biopsy proven acute rejection (BPAR) was defined as a clinically suspected acute rejection confirmed by biopsy. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI) ranging from 0 to 9. An overall score of 0-3 is considered indeterminate, score of 4-5 is mild acute, score of 6-7 is moderate acute , and score of 8-9 is severe acute. Only the episode with the highest total RAI score for each participant was counted.~The patients with treated or untreated BPAR having RAI score > 3 were reported in this end point." (NCT01625377)
Timeframe: At 24 weeks

,
InterventionPatients (Number)
Not Treated BPAR: RAI score >3Not Treated BPAR: Missing RAI scoreTreated BPAR: RAI score >3
Everolimus (RAD001)018
Tacrolimus002

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Number of Patients With Treatment Failures

"Incidence of treatment failures, assessed with composite criterion including treated biopsy proven acute rejection (tBPAR) with a rejection activity index (RAI) according to Banff classification >3, graft loss or death at 6 months.~Biopsy proven acute rejection (BPAR) was defined as a clinically suspected acute rejection confirmed by biopsy. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI) ranging from 0 to 9. An overall score of 0-3 is considered indeterminate, score of 4-5 is mild acute, score of 6-7 is moderate acute , and score of 8-9 is severe acute. Only the episode with the highest total RAI score for each participant was counted.~The graft was presumed to be lost on the day the patient was registered again on the waiting list, or the day he/she received a new graft." (NCT01625377)
Timeframe: At week 12 and week 24

,
InterventionPatients (Number)
week 12, Treatment failures - NOweek 12, Treatment failures - YESweek 24, Treatment failures - NOweek 24, Treatment failures - YES
Everolimus (RAD001)882819
Tacrolimus912894

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Number of Participants Achieving Treatment Success After Switching to Other Medication (Phase II, 0-6 Months)

Controlled ocular inflammation (≤ 0.5+ anterior chamber cells, ≤ 0.5+ vitreous haze, no active retinal/choroidal lesions in both eyes) with 7.5 mg/day of oral prednisone and ≤ 2 drops/day of topical 1% prednisolone acetate for patients who crossed over to other medication following treatment failure at 6 months (or earlier). (NCT01829295)
Timeframe: 6 Months

InterventionParticipants (Count of Participants)
Switched Over to Methotrexate20
Switched Over to Mycophenolate Mofetil7

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Number of Participants Achieving Treatment Success at 12 Months on Same Medication (Phase I, 6-12 Months)

Controlled ocular inflammation (≤ 0.5+ anterior chamber cells, ≤ 0.5+ vitreous haze, no active retinal/choroidal lesions in both eyes) with 7.5 mg/day of oral prednisone and ≤ 2 drops/day of topical 1% prednisolone acetate in patients who were a treatment success at the primary outcome of 6 months. (NCT01829295)
Timeframe: 12 Months

InterventionParticipants (Count of Participants)
Methotrexate48
Mycophenolate Mofetil40

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Number of Participants Achieving Treatment Success at 6 Months (Phase I, 0-6 Months)

Controlled ocular inflammation (≤ 0.5+ anterior chamber cells, ≤ 0.5+ vitreous haze, no active retinal/choroidal lesions in both eyes) with 7.5 mg/day of oral prednisone and ≤ 2 drops/day of topical 1% prednisolone acetate. (NCT01829295)
Timeframe: 6 Months

InterventionParticipants (Count of Participants)
Methotrexate64
Mycophenolate Mofetil56

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Percent Probability of 18 Months-relapse Event Between Arms

Probability of patients relapsing at 18 months. A relapse event is defined in protocol section 10.2.3. Time to relapse/non-response is defined as time from transplant to when all criteria of section 10.2.3 are met. (NCT01824693)
Timeframe: From transplant up to 18 months

Interventionpercent probability (Number)
Arm I (Busulfan, Cyclophosphamide, Melphalan)17
Arm II (Busulfan, Fludarabine Phosphate)55

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Percent Probability of Event-free Survival (EFS)

Probability of Event-free Survival (EFS) for Patients after 18 months. An event is either treatment related mortality (TRM), primary or secondary graft failure, or relapse/non-response (as defined in protocol section 10). Time to event is time from transplant with patients who die between the start of the conditioning regimen and transplant given a time to event of zero. (NCT01824693)
Timeframe: From transplant up to 18 months

Interventionpercent probability (Number)
Arm I (Busulfan, Cyclophosphamide, Melphalan)83
Arm II (Busulfan, Fludarabine Phosphate)22

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Percentage of Participants Who Experience Primary Graft Failure Event Between Arms

Primary Graft failure is defined as the failure to achieve an ANC >= 500/uL after 42 days, determined by 3 consecutive measurements on different days; OR < 5% donor cells in blood or bone marrow by day +42 (as demonstrated by a chimerism assay), without evidence of Juvenile Myelomonocytic Leukemia (JMML). (NCT01824693)
Timeframe: Day 0 - day 540 (18 months) following completion of stem cell transplant

Interventionpercentage of patients (Number)
Arm I (Busulfan, Cyclophosphamide, Melphalan)0
Arm II (Busulfan, Fludarabine Phosphate)0

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Graft Loss - Percentage of Participants With an Event

Graft loss was defined as physical loss (nephrectomy), functional loss [necessitating maintenance dialysis for greater than (>)8 weeks], retransplant or death. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52

Interventionpercentage of participants (Number)
Mycophenolate Mofetil, Adapted Dose5.6
Mycophenolate Mofetil, Fixed Dose5.0

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Participant Survival

Participants survival was defined as the percentage of participants living with or without a functioning graft between Weeks 0 and 52. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52

Interventionpercentage of participants (Number)
Mycophenolate Mofetil, Adapted Dose98.4
Mycophenolate Mofetil, Fixed Dose98.3

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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) Before Week 12 or Acute Subclinical Rejection on Protocol Biopsy at Week 12

BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Subclinical acute rejection was defined as an increase of serum creatinine at Week 12 strictly less than 10 percent (%) compared to baseline (BL) values and BPAR of Grade greater than or equal to (≥) 1 according to Banff 1997 classification at Week 12. (NCT02005562)
Timeframe: Week 12

Interventionpercentage of participants (Number)
Mycophenolate Mofetil, Adapted Dose24.2
Mycophenolate Mofetil, Fixed Dose19.8

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Time to Graft Loss

The median time, in days, from randomization to graft loss event. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52

Interventiondays (Median)
Mycophenolate Mofetil, Adapted DoseNA
Mycophenolate Mofetil, Fixed DoseNA

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Time to Occurrence of First BPAR Between Day 0 and Week 52

BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Subclinical acute rejection at Week 12 was included in the analysis. Subclinical acute rejection was defined as an increase of serum creatinine at Week 12 strictly less than 10% compared to BL values and BPAR of Grade ≥1 according to Banff 1997 classification at Week 12. The occurrence of the first BPAR was defined as the time from randomization to the first recorded BPAR between Day 0 and Week 52. The results of protocol biopsies at Week 12 were taken into account. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52

Interventiondays (Median)
Mycophenolate Mofetil, Adapted DoseNA
Mycophenolate Mofetil, Fixed DoseNA

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Time to Occurrence of First BPAR Between Day 0 and Week 52 - Percentage of Participants With an Event

BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Subclinical acute rejection at Week 12 was included in the analysis. Subclinical acute rejection was defined as an increase of serum creatinine at Week 12 strictly less than 10% compared to BL values and BPAR of Grade ≥1 according to Banff 1997 classification at Week 12. The occurrence of the first BPAR was defined as the time from randomization to the first recorded BPAR between Day 0 and Week 52. The results of protocol biopsies at Week 12 were taken into account. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52

Interventionpercentage of participants (Number)
Mycophenolate Mofetil, Adapted Dose24.6
Mycophenolate Mofetil, Fixed Dose14.9

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Creatinine Clearance Values Estimated With the Cockcroft-Gault Equation (Milliliters Per Minute [mL/Min])

The mean creatinine clearance values at Weeks 2, 4, 6, 12, 16, 26, 39, and 52 estimated using the Cockcroft-Gault equation. (NCT02005562)
Timeframe: Weeks 2, 4, 6, 12, 16, 26, 39, and 52

,
InterventionmL/min (Mean)
Week 2 (n=120,114)Week 4 (n=119,113)Week 6 (n=119,113)Week 12 (n=117,112)Week 16 (n=116,112)Week 26 (n=115,112)Week 40 (n=114,108)Week 52 (n=115,110)
Mycophenolate Mofetil, Adapted Dose46.6851.0353.6656.5556.5560.2159.1758.29
Mycophenolate Mofetil, Fixed Dose45.2646.7349.7753.0153.4955.1856.8156.45

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Creatinine Clearance Values Estimated With the Modification of Diet in Renal Disease (MDRD) Simplified Equation

The mean creatinine clearance values at Weeks 2, 4, 6, 12, 16, 26, 39, and 52 estimated using the MDRD simplified equation. For males, the MDRD simplified equation was defined as MDRD (mL/min/1.73 square meters [m^2]) =186 multiplied by (*) serum creatinine in mg/L raised to the power of (^) -1.154 * age ^ -0.203. For females, the MDRD simplified equation was defined as MDRD (mL/min/1.73 m^2) = males formula * 0.742. (NCT02005562)
Timeframe: Weeks 2, 4, 6, 12, 16, 26, 39, and 52

,
InterventionmL/min/1.73 m^2 (Mean)
Week 2 (n=120,114)Week 4 (n=119,113)Week 6 (n=119,113)Week 12 (n=117,112)Week 16 (n=116,112)Week 26 (n=115,112)Week 40 (n=114,108)Week 52 (n=115,110)
Mycophenolate Mofetil, Adapted Dose40.6145.0248.1250.9350.9354.2252.2350.82
Mycophenolate Mofetil, Fixed Dose39.6041.3144.6947.6848.5249.4650.0148.88

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Graft Histology - Percentage of Participants With at Least One Borderline Lesion at Week 12 and Week 52

Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Weeks 12 and 52

,
Interventionpercentage of participants (Number)
Week 12Week 52
Mycophenolate Mofetil, Adapted Dose7.96.4
Mycophenolate Mofetil, Fixed Dose12.45.0

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Graft Histology - Percentage of Participants With at Least One Chronic Graft Nephropathy at Week 12 and Week 52

Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Weeks 12 and 52

,
Interventionpercentage of participants (Number)
Week 12Week 52
Mycophenolate Mofetil, Adapted Dose20.632.5
Mycophenolate Mofetil, Fixed Dose17.434.7

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Percentage of Participants With at Least One BPAR at Week 12 and Week 52

BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Weeks 12 and 52

,
Interventionpercentage of participants (Number)
Week 12Week 52
Mycophenolate Mofetil, Adapted Dose14.19.1
Mycophenolate Mofetil, Fixed Dose10.010.0

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Serum Creatinine Values [Micromoles Per Liter (µmol/L)]

The mean serum creatinine values at Weeks 2, 4, 6, 12, 16, 26, 39, and 52. (NCT02005562)
Timeframe: Weeks 2, 4, 6, 12, 16, 26, 39, and 52

,
Interventionµmol/L (Mean)
Week 2 (n=120,114)Week 4 (n=119,113)Week 6 (n=119,113)Week 12 (n=117,112)Week 16 (n=116,112)Week 26 (n=115,112)Week 40 (n=114,108)Week 52 (n=115,110)
Mycophenolate Mofetil, Adapted Dose202.41168.92155.74140.80145.48131.73136.24148.62
Mycophenolate Mofetil, Fixed Dose201.31174.86158.96147.70148.48143.73141.89144.72

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Incidence of Chronic Graft Versus Host Disease (cGVHD)

Chronic graft versus host disease (cGVHD) is a reaction which typically develops 3 to 6 months after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. (NCT00176865)
Timeframe: 6 months and 1 year

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor1
Arm 2 - Matched Unrelated Donor0
Arm 3 - Mismatched Double Cord Donors0

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Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD)

Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease. (NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor1
Arm 2 - Matched Unrelated Donor3
Arm 3 - Mismatched Double Cord Donors0

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Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD)

Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease. (NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor1
Arm 2 - Matched Unrelated Donor1
Arm 3 - Mismatched Double Cord Donors0

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Number of Subjects Alive at 100 Days

(NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor3
Arm 2 - Matched Unrelated Donor8
Arm 3 - Mismatched Double Cord Donors4

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Number of Subjects Alive at One Year

(NCT00176865)
Timeframe: Day 365

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor3
Arm 2 - Matched Unrelated Donor7
Arm 3 - Mismatched Double Cord Donors3

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Number of Subjects With Mixed Chimerism

>10% Donor Cells at Day 100 (NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor3
Arm 2 - Matched Unrelated Donor8
Arm 3 - Mismatched Double Cord Donors4

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Percentage of Donor Chimerism at 100 Days

The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 100

Interventionpercentage of donor cells (Mean)
Arm 1 - Matched Sibling Donor96.5
Arm 2 - Matched Unrelated Donor75.5
Arm 3 - Mismatched Double Cord Donors100

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Percentage of Donor Chimerism at 180 Days

The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 180

Interventionpercentage of donor cells (Mean)
Arm 1 - Matched Sibling Donor88.9
Arm 2 - Matched Unrelated Donor73.3
Arm 3 - Mismatched Double Cord Donors90.5

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Percentage of Donor Chimerism at 365 Days

The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 365

Interventionpercentage of donor cells (Mean)
Arm 1 - Matched Sibling Donor81.9
Arm 2 - Matched Unrelated Donor78.6
Arm 3 - Mismatched Double Cord Donors91.7

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Death From GVHD

(NCT00112593)
Timeframe: Within the first 360 days

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation)0

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Death From Regimen Toxicity or Opportunistic Infection

(NCT00112593)
Timeframe: Within the first 100 days

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation)0

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Overall Survival

Kaplan-Meier estimate assessed at 1 year. (NCT00112593)
Timeframe: Up to 1 year

Interventionsurvival probability (Number)
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation)0.40

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Progression of HIV

Count of participants with HIV progression. (NCT00112593)
Timeframe: Within 1 year

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation)0

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Reconstitution of HIV-specific Immunity

(NCT00112593)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation)2

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Successful Induction of Mixed Hematopoietic Chimerism as Assessed by the Percentage of Peripheral Blood T Cells That Are of Donor Origin

Determined by a DNA-based assay that compares the profile of amplified fragment length polymorphisms (ampFLP) of the patient and donor. (NCT00112593)
Timeframe: Up to day 80

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation)5

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Acute Rejection

Number of Participants with Acute Rejection (AR). AR is defined as allograft dysfunctions in the setting of recipient immune system engaging an allo-response against the kidney transplant. (NCT02213068)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Belatacept + MPA4
Belatacept + Low-Dose Tac0
Tacrolimus + MPA Standard Treatment Regimen2

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Change in eGFR (MDRD) at 2 Years Post-transplant Compared to Baseline at Month 3 (Conversion)

To assess change in renal function by calculated (MDRD) GFR of adult EBV seropositive renal transplant recipients of living or standard criteria donors converted from Tacrolimus to Belatacept or low dose Tacrolimus with Belatacept at three months post-operatively compared to renal transplant recipients randomized to remain on standard dose Tacrolimus and MPA for maintenance therapy at 2 years post-transplantation (NCT02213068)
Timeframe: 2 years

InterventionmL/min/1.73m2 (Mean)
Belatacept + MPA-5.44
Belatacept + Low-Dose Tac8.08
Tacrolimus + MPA Standard Treatment Regimen-0.38

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Graft Survival

Number of Subjects with a functioning Graft (NCT02213068)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Belatacept + MPA8
Belatacept + Low-Dose Tac8
Tacrolimus + MPA Standard Treatment Regimen10

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Patient Survival

Number of Subjects alive at the end of 24 months (NCT02213068)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Belatacept + MPA9
Belatacept + Low-Dose Tac7
Tacrolimus + MPA Standard Treatment Regimen9

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Cumulative Incidence of Chronic GVHD

Cumulative incidence (measured as a percentage) of chronic graft versus host disease (GVHD). (NCT02120157)
Timeframe: 2 years

Interventionpercent (Number)
Haploidentical BMT With PTCy for Acute Leukemias and MDS11

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Cumulative Incidence of Non-relapse Mortality

Cumulative incidence (measured as a percentage) of non-relapse mortality at 180 days following myeloablative, Human Leukocyte Antigen (HLA)-mismatched bone marrow transplant (BMT) for patients with high risk hematologic malignancies. (NCT02120157)
Timeframe: Day 180

Interventionpercent (Number)
Haploidentical BMT With PTCy for Acute Leukemias and MDS0

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Incidence of Donor Cell Engraftment

Incidence of donor cell engraftment measured as the percentage of donor cell engraftment. (NCT02120157)
Timeframe: 60 days

Interventionpercentage of donor cell engraftment (Number)
Haploidentical BMT With PTCy for Acute Leukemias and MDS84

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Number of Participants With Donor Cell Engraftment

Number of Participants with Donor Cell Engraftment at Day 60 following myeloablative, HLA-mismatched BMT. (NCT02120157)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Haploidentical BMT With PTCy for Acute Leukemias and MDS27

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Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4

Cumulative incidence (measured as a percentage) of acute GVHD grades 2-4 (overall) and grades 3-4 (severe). (NCT02120157)
Timeframe: 100 days

Interventionpercent (Number)
Grades 2-4Grades 3-4
Haploidentical BMT With PTCy for Acute Leukemias and MDS100

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Primary and Secondary Graft Failure

Incidence (measured as a percentage) of primary and secondary graft failure. (NCT02120157)
Timeframe: 2 years

Interventionpercentage of graft failure (Number)
PrimarySecondary
Haploidentical BMT With PTCy for Acute Leukemias and MDS160

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Steroid and Non-steroid Immunosuppressants

Number of participants who used steroid and non-steroid immunosuppressants to treat GVHD. (NCT02120157)
Timeframe: Two Years

InterventionParticipants (Count of Participants)
Steroid immunosuppressantsNon-steroid immunosuppressants
Haploidentical BMT With PTCy for Acute Leukemias and MDS42

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Steroid and Non-steroid Immunosuppressants Use Duration

Duration of use of steroid and non-steroid immunosuppressants (in months) to treat GVHD. (NCT02120157)
Timeframe: Two Years

Interventionmonths (Number)
Steroid immunosuppressantsNon-steroid immunosuppressants
Haploidentical BMT With PTCy for Acute Leukemias and MDS1819

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Survival

Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 1 year. Incidence as a percentage. (NCT02120157)
Timeframe: up to 1 years

Interventionpercent (Number)
overall survival (OS)progression-free survival (PFS)disease-free survival (DFS)event-free survivalRelapse-free GVHD-free survival (GRFS)
Haploidentical BMT With PTCy for Acute Leukemias and MDS7768686865

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Survival

Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 2 years. Incidence as a percentage. (NCT02120157)
Timeframe: up to 2 years

Interventionpercent (Number)
overall survival (OS)progression-free survival (PFS)disease-free survival (DFS)event-free survivalRelapse-free GVHD-free survival (GRFS)
Haploidentical BMT With PTCy for Acute Leukemias and MDS7364646452

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Time to Neutrophil and Platelet Recovery

Time to neutrophil and platelet recovery in median days (NCT02120157)
Timeframe: 100 days

Interventiondays (Median)
neutrophilplatelet
Haploidentical BMT With PTCy for Acute Leukemias and MDS2221

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Duration of Wound Healing

A wound will be considered healed if all the suture material and staples are removed and the wound is intact. Number of participants is based on all patients of the respective treatment group in the safety set, excluding patients with no answer (unknown). (NCT01843348)
Timeframe: Post transplant until individual reporting

Interventiondays (Mean)
TAC+MPA42.4
TAC+Certican54.1
CycA+Certican85.3

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Glomular Filtration Rate (GFR) mL/Min Via Cockcroft- Gault Method at Month 12 Post Transplant

Cockcroft-Gault formula: For men: GFR= ((140-age) × body weight in kg)∕(72 x serum creatinine in mg∕dl)For women: GFR= (0.85×(140-age) × body weight in kg)∕(72 x serum creatinine in mg/dl), ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT01843348)
Timeframe: Month 12 post transplant

InterventionmL/min per 1.73m² (Least Squares Mean)
TAC+MPA60.26
TAC+Certican52.25
CycA+Certican51.30

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Glomular Filtration Rate (GFR) Via Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Method at Month 12 Post Transplant

Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) method = GFR=141 x min(Scr/κ, 1)α x max(Scr/κ, 1)1.209 x 0.993Age x 1.018 [if female] x 1.159 [if black] where Scr is serum creatinine, κ is 0.7 for females and 0.9 for males, α is 0.329 for females and 0.411 for males, min indicates the minimum of Scr/κ or 1, and max indicates the maximum of Scr/κ or 1. last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT01843348)
Timeframe: Month 12 post transplant

InterventionmL/min per 1.73m² (Least Squares Mean)
TAC+MPA51.62
TAC+Certican44.42
CycA+Certican42.44

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Glomular Filtration Rate (GFR) Via Modification of Diet in Renal Disease (MDRD) Method at Month 12 Post Transplant

Modification of Diet in Renal Disease (MDRD) = For men: GFR = 170 x (serum creatinine -0,999) x (age-0,176) x (urea nitrogen -0,17) x (albumin0,318) For women: GFR = 170 x (serum creatinine -0,999) x (age-0,176) x (urea nitrogen -0,17) x albumin0,318) x 0.762 with urea nitrogen = urea / 2.144. last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT01843348)
Timeframe: Month 12 post transplant

InterventionmL/min per 1.73m² (Least Squares Mean)
TAC+MPA53.24
TAC+Certican45.72
CycA+Certican43.47

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Glomular Filtration Rate (GFR) mL/Min Via Nankivell Method at Month 12 - Standard Regimen vs Certican Regimens

"To demonstrate non-inferiority in renal function assessed by glomerular filtration rate (Nankivell formula) in at least one of the Certican® treatment regimens compared to the standard regimen group at month 12 post-transplantation in renal transplant patients. Nankivell formula:~GFR = 6.7/Scr + BW/4 - Surea/2 - 100/(height)² + C where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. The eGFR is expressed in mL/min per 1.73m². If a patient was on dialysis at the time of urea or creatinine assessment, the eGFR was set to 0. Analysis set = per protocol set" (NCT01843348)
Timeframe: One year post transplant

,,
InterventionmL/min per 1.73m² (Mean)
Month 1 - Day 1 to 60 (146,111,78)Month 3 - Day 61 to 136 (143,108,79)Month 6 - Day 137 to 228 (142,108,76)Month 9 - Day 229 to 319 (140,106,77)Month12 - Day 320 to 450 (147,111, 80)
CycA+Certican59.4762.2263.1762.8961.51
TAC+Certican60.5461.2162.7664.6863.34
TAC+MPA62.6266.3668.0569.4770.41

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Percent of Participants With Delayed Graft Function and Slow Graft Function

Delayed graft function (DGF) was defined as the need for dialysis within the first 7 days post-transplantation, excluding the first post-transplantation day. Slow graft function (SGF) was defined as a serum creatinine >3.0 mg/dL at Day 5 post-transplantation. Full analysis set (NCT01843348)
Timeframe: Post transplant to month 12

,,
InterventionPercent of participants (Number)
Delayed graft function (197,187,172)Slow graft function (195,187,171)
CycA+Certican22.149.7
TAC+Certican20.348.7
TAC+MPA17.846.2

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Percent of Participants With Delayed Graft Function by Day

Delayed graft function (DGF) was defined as the need for dialysis within the first 7 days post-transplantation, excluding the first post-transplantation day. (NCT01843348)
Timeframe: Post transplant up to day 7

,,
InterventionPercent of participants (Number)
day 1 (8,7,4)day 2 (2,2,2)day 3 (5,1,2)day 4 (4,2,4)day 5 (4,5,3)day 6 (1,1,2)day 7 (2,4,0)>7 days (9,16,21)
CycA+Certican10.55.35.310.57.95.30.055.3
TAC+Certican18.45.32.65.313.22.610.542.1
TAC+MPA22.95.714.311.411.42.95.725.7

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Percent of Participants With Viral Infections

Viral infections for BKV Virus Humane Polyomavirus 1 and Cytomegalovirus (NCT01843348)
Timeframe: Post transplant to month 12

,,
InterventionPercent of participants (Number)
Viral infections - CMVMissingViral infections - CMVAsymptomaticViral infections - CMVMildViral infections - CMVModerateViral infections - CMVSevereViral infections - BKVAsymptomaticViral infections - BKVMildViral infections - BKVModerateViral infections - BKVSevere
CycA+Certican1.01.01.01.00.05.03.01.00
TAC+Certican0.01.02.01.00.08.07.02.00
TAC+MPA1.07.05.06.01.010.05.07.00

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Percent of Participants With Wound Healing Complications During Study

Information collected to report wound healing process which included percentage of participants with complications, fluid collections detected and occurrence of lymphoceles (NCT01843348)
Timeframe: Post transplant until individual reporting

,,
InterventionPercent of participants (Number)
Wound healing complicationFluids detectedOccurrence of lymphoceles
CycA+Certican22.227.821.8
TAC+Certican19.126.818.2
TAC+MPA14.318.711.8

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Percentage of Participants With Composite Treatment Failure Endpoints - Difference Between Groups at Month 12

Combined endpoint included: biopsy proven acute rejection (BPAR) defined as a rejection which was acute and proven by biopsy, graft loss (GL) defined as: allograft was presumed to be lost on the day the patient starts dialysis and not able to be removed from dialysis or death. Patients who prematurely discontinued the study: if the patient did not suffer from an event before discontinuation and reason was not related to efficacy, the patient was assessed as having had no event, otherwise the patient was assessed as having had an event. Full analysis set (FAS) (NCT01843348)
Timeframe: Month 12 post transplant

,,,,
InterventionPercentage of participants (Number)
BPAR or graft loss or deathBPAR, graft loss, death, or loss of follow-up
CycA+Certican24.632.7
CycA+Certican -Tac+MPA - Difference Between Groups14.917.1
TAC+Certican13.022.6
Tac+Certican - Tac+MPA - Difference Between Groups3.27.0
TAC+MPA9.815.6

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Percentage of Participants With Treatment Failure Endpoints at Month 12

Treatment failure endpoints: biopsy proven acute rejection (BPAR) defined as a rejection which was acute and proven by biopsy, graft loss (GL) defined as: allograft was presumed to be lost on the day the patient starts dialysis and not able to be removed from dialysis or death. Patients who prematurely discontinued the study: if the patient did not suffer from an event before discontinuation and reason was not related to efficacy, the patient was assessed as having had no event, otherwise the patient was assessed as having had an event. Full analysis set (FAS) (NCT01843348)
Timeframe: Month 12 post transplant

,,
InterventionPercentage of participants (Number)
Biopsy proven acute rejection (BPAR)Treated BPAR (tBPAR)Graft lossDeath
CycA+Certican24.623.69.06.5
TAC+Certican12.011.56.36.3
TAC+MPA9.38.85.44.9

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Cumulative Incidence of Chronic GVHD According to BMTCTN

Will be summarized with a proportion and a 95% confidence interval. (NCT01707004)
Timeframe: Up to 1 year

Interventionpercentage (Number)
Decitabine + Bone Marrow Transplant40.7

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Cumulative Incidence of Grade III-IV Acute GVHD

Determined by the standard bone marrow transplant (BMT) Clinical Trials Network criteria (BMTCTN). Will be analyzed using KM method, a Graft versus Host Disease (GVHD) grade III-IV will be obtained from the KM estimates along with 95% confidence intervals. (NCT01707004)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Decitabine + Bone Marrow Transplant27.8

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Number of Participants With Complete Remission After Transplantation

(NCT01707004)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Decitabine + Bone Marrow Transplant14

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Number of Participants With Primary Graft Failure

Defined as less than 5% donor chimerism in the cluster of differentiation (CD)3 and CD33 selected cell populations at any time after transplantation. Will be analyzed using KM method. (NCT01707004)
Timeframe: Day 30

InterventionParticipants (Count of Participants)
Decitabine + Bone Marrow Transplant0

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Overall Survival (OS)

Will be analyzed using Kaplan-Meier (KM) method, and OS will be obtained from the KM estimates along with 95% confidence intervals. (NCT01707004)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Decitabine + Bone Marrow Transplant64.7

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Percentage of Participants With Platelet Recovery by Day 30

Platelet recovery is defined as the first day of a platelet count greater than 20,000/mm^3 with no platelet transfusions. Will be summarized with mean and standard deviation or median and interquartile range, and the change will be tested using a one-sample paired t-test at a two-tailed significance level of 0.05. (NCT01707004)
Timeframe: Up to day 30

Interventionpercentage with plt engraftment, day 30 (Number)
Decitabine + Bone Marrow Transplant58

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Progression Free Survival

(NCT01707004)
Timeframe: Up to 1 year

Interventiondays (Median)
Decitabine + Bone Marrow Transplant141

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Time to Neutrophil Recovery

Defined as achieving an absolute neutrophil count (ANC) greater than or equal to 500/ul for three consecutive measurements on different days. Will be summarized with mean and standard deviation or median and interquartile range, and the change will be tested using a one-sample paired t-test at a two-tailed significance level of 0.05. (NCT01707004)
Timeframe: Up to 1 year

Interventiondays (Mean)
Decitabine + Bone Marrow Transplant16

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Two-year Disease-free Survival of Study Participants Who Completed the Study Regimen

"Survival and complete resolution of all signs of leukemia for 2 years after transplant with all of the following:~Normal bone marrow with blasts <5% with normal cellularity, normal megakarypoiesis, more than 15% erythropoiesis and more than 25% granulocytopoiesis.~Normalization of blood counts (no blasts, platelets >100,000/mm3, granulocytes >1,500/mm3).~No extramedullary disease." (NCT00119366)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Dose Level 1: 12 Gy Iodine-131+ BC8 Monoclonal Antibody0
Dose Level 7: 22 Gy Iodine-131+ BC8 Monoclonal Antibody2
Dose Level 8: 24 Gy Iodine-131+ BC8 Monoclonal Antibody1
Dose Level 9: 26 Gy Iodine-131+ BC8 Monoclonal Antibody0
Dose Level 10: 28 Gy Iodine-131+ BC82

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Number of Participants With 100% Donor Chimerism at Day 28 and Day 84

Post-transplant bone marrow samples were collected on day 28 and day 84 after transplant for DNA Chimerism Analysis (NCT00119366)
Timeframe: Day 28 and Day 80 after transplant

,,,,
InterventionParticipants (Count of Participants)
Day 28 Donor ChimerismDay 84 Donor Chimerism
Dose Level 1: 12 Gy Iodine-131+ BC8 Monoclonal Antibody00
Dose Level 10: 28 Gy Iodine-131+ BC874
Dose Level 7: 22 Gy Iodine-131+ BC8 Monoclonal Antibody11
Dose Level 8: 24 Gy Iodine-131+ BC8 Monoclonal Antibody33
Dose Level 9: 26 Gy Iodine-131+ BC8 Monoclonal Antibody22

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Participant Disease Response Within 4 Weeks After Transplant

"The number of participants that are in complete remission (CR) or relapsed within 4 weeks after transplant.~Complete Remission is defined as complete resolution of all signs of leukemia for at least four weeks with all of the following:~Normal bone marrow with blasts <5% with normal cellularity, normal megakarypoiesis, more than 15% erythropoiesis and more than 25% granulocytopoiesis.~Normalization of blood counts (no blasts, platelets >100,000/mm3, granulocytes >1,500/mm3).~No extramedullary disease.~Relapse is measured as follows:~After CR: >5% blasts in the bone marrow and/or peripheral blood.~Confirmation of relapse by bone marrow analysis with more than 10% blasts.~Extramedullary disease confirmed cytologically or histologically." (NCT00119366)
Timeframe: 4 weeks after transplant

,,,,
InterventionParticipants (Count of Participants)
Number of participants that are in CR 4 weeks after transplantNumber of participants that relapsed 4 weeks after transplant
Dose Level 1: 12 Gy Iodine-131 + BC8 Monoclonal Antibody01
Dose Level 10: 28 Gy Iodine-131 + BC8 Monoclonal Antibody62
Dose Level 7: 22 Gy Iodine-131 + BC8 Monoclonal Antibody20
Dose Level 8: 24 Gy Iodine-131 + BC8 Monoclonal Antibody30
Dose Level 9: 26 Gy Iodine-131 + BC8 Monoclonal Antibody11

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Research Highlights

Safety/Toxicity (340)

ArticleYear
COVID-19 Vaccine in Immunosuppressed Adults with Autoimmune rheumatic Diseases (COVIAAD): safety, immunogenicity and antibody persistence at 12 months following Moderna Spikevax primary series.
RMD open, 11-29, Volume: 9, Issue: 4
2023
Efficacy and safety of immunosuppressive agents for adults with lupus nephritis: a systematic review and network meta-analysis.
Frontiers in immunology, Volume: 14
2023
Long-term efficacy and safety of different corticosteroid courses plus mycophenolate mofetil for autoimmune encephalitis with neuronal surface antibodies without tumor.
Frontiers in immunology, Volume: 14
2023
Efficacy and safety of multi-target therapy in children with lupus nephritis.
Pediatric research, Volume: 94, Issue: 6
2023
Effectiveness of tacrolimus in a case of immune checkpoint inhibitor-induced hepatotoxicity that was refractory to steroids and mycophenolate mofetil.
Clinical journal of gastroenterology, Volume: 16, Issue: 5
2023
A Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Tacrolimus and Corticosteroids in Combination With or Without Mycophenolate Mofetil in Liver Transplantation Recipients Infected With Hepatitis B Virus.
Transplantation proceedings, Volume: 55, Issue: 2
2023
Comparison of the efficacy and safety of leflunomide versus mycophenolate mofetil in treating IgG4-related disease: a retrospective cohort study.
Clinical rheumatology, Volume: 42, Issue: 7
2023
Synthetic Pharmacotherapy for Systemic Lupus Erythematosus: Potential Mechanisms of Action, Efficacy, and Safety.
Medicina (Kaunas, Lithuania), Dec-27, Volume: 59, Issue: 1
2022
Efficacy and safety of tacrolimus versus mycophenolate mofetil as induction treatment and low-dose tacrolimus as treatment for lupus nephritis: a meta-analysis.
Zeitschrift fur Rheumatologie, Volume: 82, Issue: 9
2023
Assessing the efficacy and safety of mycophenolate mofetil versus azathioprine in patients with autoimmune hepatitis (CAMARO trial): study protocol for a randomised controlled trial.
Trials, Dec-13, Volume: 23, Issue: 1
2022
Acute Calcineurin Inhibitor Nephrotoxicity Diagnosed Using Kidney Doppler Ultrasonography After Heart Transplant: A Case Report.
Transplantation proceedings, Volume: 54, Issue: 10
2022
Mycophenolate-Induced Hepatotoxicity Precipitates Tacrolimus Nephrotoxicity in a Kidney Transplant Recipient: A Case Report.
Transplantation proceedings, Volume: 54, Issue: 10
2022
Immunogenicity and safety of the mRNA-based BNT162b2 vaccine in systemic autoimmune rheumatic diseases patients.
Clinical rheumatology, Volume: 41, Issue: 12
2022
Update on the Efficacy and Safety Profile of Voclosporin: An Integrated Analysis of Clinical Trials in Lupus Nephritis.
Arthritis care & research, Volume: 75, Issue: 7
2023
Temporal shifts in safety and efficacy profile of mycophenolate mofetil 2 g versus 3 g daily early after heart transplantation.
Pharmacotherapy, Volume: 42, Issue: 9
2022
Efficacy and safety of mycophenolate mofetil in the treatment of moderate to severe Graves' orbitopathy: a meta-analysis.
Bioengineered, Volume: 13, Issue: 6
2022
Evaluation of nefrotoxicity by tacrolimus and micophenolate mofetil associated with kidney ischemia and reperfusion: experimental study in rats.
Revista do Colegio Brasileiro de Cirurgioes, Volume: 49
2022
Immunogenicity and safety of SARS-CoV-2 mRNA vaccine in patients with nephrotic syndrome receiving immunosuppressive agents.
Pediatric nephrology (Berlin, Germany), Volume: 38, Issue: 4
2023
The Efficacy and Safety of Mizoribine versus Mycophenolate Mofetil for the Treatment of Renal Transplantation: A Systematic Review and Meta-Analysis.
Computational intelligence and neuroscience, Volume: 2022
2022
Comparative Efficacy and Safety of Tacrolimus, Cyclosporin A, Mycophenolate Mofetil, Cyclophosphamide, and Corticosteroids as Induction Therapy for Membranous Lupus Nephritis: A Network Meta-Analysis.
Pharmacology, Volume: 107, Issue: 9-10
2022
The efficacy and safety of mycophenolate mofetil in Thai neuromyelitis optica spectrum disorder patients.
Multiple sclerosis and related disorders, Volume: 63
2022
A mechanistic target of rapamycin inhibitor, everolimus safely ameliorated lupus nephritis in a patient complicated with tuberous sclerosis.
Modern rheumatology case reports, 01-03, Volume: 7, Issue: 1
2023
Efficacy and Safety of Tacrolimus in the Treatment of Pediatric Henoch-Schönlein Purpura Nephritis.
Paediatric drugs, Volume: 24, Issue: 4
2022
Adverse Events in NMOSD Therapy.
International journal of molecular sciences, Apr-09, Volume: 23, Issue: 8
2022
Efficacy and safety of azathioprine, mycophenolate mofetil, and reduced dose of rituximab in neuromyelitis optica spectrum disorder.
European journal of neurology, Volume: 29, Issue: 8
2022
Schisandrin A alleviates mycophenolic acid-induced intestinal toxicity by regulating cell apoptosis and oxidative damage.
Toxicology mechanisms and methods, Volume: 32, Issue: 8
2022
Efficacy and safety of high-dose of mycophenolate mofetil compared with cyclophosphamide pulse therapy as induction therapy in Japanese patients with proliferative lupus nephritis.
Modern rheumatology, Oct-15, Volume: 32, Issue: 6
2022
Long-term safety and effectiveness of mycophenolate mofetil in adults with lupus nephritis: a real-world study in Japan.
Modern rheumatology, Jul-01, Volume: 32, Issue: 4
2022
Safety and efficacy of the mRNA BNT162b2 vaccine against SARS-CoV-2 in five groups of immunocompromised patients and healthy controls in a prospective open-label clinical trial.
EBioMedicine, Volume: 74
2021
Characterization of Mycophenolate Mofetil Gastrointestinal Toxicity and Risk Factors for Severe Disease and Poor Prognosis.
Inflammatory bowel diseases, 05-04, Volume: 28, Issue: 5
2022
A prospective multicenter study assessing humoral immunogenicity and safety of the mRNA SARS-CoV-2 vaccines in Greek patients with systemic autoimmune and autoinflammatory rheumatic diseases.
Journal of autoimmunity, Volume: 125
2021
A network meta-analysis of randomized controlled trials comparing the effectiveness and safety of voclosporin or tacrolimus plus mycophenolate mofetil as induction treatment for lupus nephritis.
Zeitschrift fur Rheumatologie, Volume: 82, Issue: 7
2023
A retrospective study of adverse effects of mycophenolate mofetil administration to dogs with immune-mediated disease.
Journal of veterinary internal medicine, Volume: 35, Issue: 5
2021
Mycophenolate Mofetil Hepatotoxicity Associated With Mitochondrial Abnormality in Liver Transplant Recipients and Mice.
Journal of pediatric gastroenterology and nutrition, 10-01, Volume: 73, Issue: 4
2021
Long-term Efficacy and Safety of Everolimus Versus Mycophenolate in Kidney Transplant Recipients Receiving Tacrolimus.
Transplantation, 02-01, Volume: 106, Issue: 2
2022
Efficacy and safety of voclosporin versus placebo for lupus nephritis (AURORA 1): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial.
Lancet (London, England), 05-29, Volume: 397, Issue: 10289
2021
[Efficacy and safety of mycophenolate mofetil versus cyclophosphamide in the treatment of Henoch-Schönlein purpura nephritis with nephrotic-range proteinuria in children: a prospective randomized controlled trial].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, Volume: 23, Issue: 4
2021
A systematic overview of the spermatotoxic and genotoxic effects of methotrexate, ganciclovir and mycophenolate mofetil.
Acta obstetricia et gynecologica Scandinavica, Volume: 100, Issue: 9
2021
Safety and effectiveness of mycophenolate mofetil associated with tacrolimus for liver transplantation immunosuppression: a systematic review and meta-analysis of randomized controlled trials.
Clinics (Sao Paulo, Brazil), Volume: 76
2021
Efficacy and safety of immunosuppressive therapies in the treatment of high-risk IgA nephropathy: A network meta-analysis.
Medicine, Feb-26, Volume: 100, Issue: 8
2021
Immune checkpoint inhibitor toxicity: A new indication for therapeutic plasma exchange?
Journal of clinical apheresis, Volume: 36, Issue: 4
2021
Real-World Experience of Safety of Mycophenolate Mofetil in 119 Japanese Patients with Systemic Lupus Erythematosus: A Retrospective Single-Center Study.
BioMed research international, Volume: 2021
2021
Impact of Pharmacogenetic Determinants of Tacrolimus and Mycophenolate on Adverse Events in Renal Transplant Patients.
Current drug metabolism, Volume: 22, Issue: 5
2021
Efficacy and safety of nintedanib in patients with systemic sclerosis-associated interstitial lung disease treated with mycophenolate: a subgroup analysis of the SENSCIS trial.
The Lancet. Respiratory medicine, Volume: 9, Issue: 1
2021
Preliminary evidence on abatacept safety and efficacy in refractory juvenile localized scleroderma.
Rheumatology (Oxford, England), 08-02, Volume: 60, Issue: 8
2021
Delayed Calcineurin Inhibitor Introduction Without Antibody Induction in Liver Transplantation Is Safe and Helps Preserve Kidney Function.
Transplantation proceedings, Volume: 53, Issue: 2
2021
Haploidentical Stem Cell Transplantation With Post-transplant Cyclophosphamide for Pediatric Acute Leukemia is Safe and Effective.
Journal of pediatric hematology/oncology, Oct-01, Volume: 43, Issue: 7
2021
Safety and efficacy of mycophenolate mofetil in treating neuromyelitis optica spectrum disorders: a protocol for systematic review and meta-analysis.
BMJ open, 11-30, Volume: 10, Issue: 11
2020
Randomized clinical trial to compare efficacy and safety of repeated courses of rituximab to single-course rituximab followed by maintenance mycophenolate-mofetil in children with steroid dependent nephrotic syndrome.
BMC nephrology, 11-30, Volume: 21, Issue: 1
2020
Efficacy and safety of mycophenolate mofetil therapy in neuromyelitis optica spectrum disorders: a systematic review and meta-analysis.
Scientific reports, 10-07, Volume: 10, Issue: 1
2020
Comparative efficacy and safety of mycophenolate mofetil and cyclophosphamide in the induction treatment of lupus nephritis: A systematic review and meta-analysis.
Medicine, Sep-18, Volume: 99, Issue: 38
2020
Suppression of cGMP-Dependent Photoreceptor Cytotoxicity With Mycophenolate Is Neuroprotective in Murine Models of Retinitis Pigmentosa.
Investigative ophthalmology & visual science, 08-03, Volume: 61, Issue: 10
2020
Evaluation of efficacy and safety of rituximab in combination with mycophenolate mofetil in patients with nonspecific interstitial pneumonia non-responding to a first-line immunosuppressive treatment (EVER-ILD): A double-blind placebo-controlled randomize
Respiratory medicine and research, Volume: 78
2020
Immunosuppressive Drug-Associated Adverse Event Profiles in De Novo Kidney Transplant Recipients Receiving Everolimus and Reduced Tacrolimus Doses.
Therapeutic drug monitoring, Volume: 42, Issue: 6
2020
Wound healing adverse events in kidney transplant recipients receiving everolimus with reduced calcineurin inhibitor exposure or current standard-of-care: insights from the 24-month TRANSFORM study.
Expert opinion on drug safety, Volume: 19, Issue: 10
2020
Comparative efficacy and safety of mycophenolate mofetil versus cyclophosphamide in patients with active antineutrophil cytoplasmic antibody-associated vasculitis: a meta-analysis of randomized trials.
Zeitschrift fur Rheumatologie, Volume: 80, Issue: 5
2021
The efficacy and safety of immunosuppressive therapies in the treatment of IgA nephropathy: A network meta-analysis.
Scientific reports, 04-08, Volume: 10, Issue: 1
2020
Phase II, multi-center, open-label, single-arm clinical trial evaluating the efficacy and safety of Mycophenolate Mofetil in patients with high-grade locally advanced or metastatic osteosarcoma (ESMMO): rationale and design of the ESMMO trial.
BMC cancer, Mar-30, Volume: 20, Issue: 1
2020
Long-Term Effects of the Replacement of Calcineurin Inhibitors With Everolimus and Mycophenolate in Patients With Calcineurin Inhibitor-Related Nephrotoxicity.
Transplantation proceedings, Volume: 52, Issue: 3
2020
Efficacy and Safety of a Quadruple Regimen Compared with Triple Regimens in Patients with Mycophenolic Acid-Related Gastrointestinal Complications After Renal Transplantation: A Short-Term Single-Center Study.
Annals of transplantation, Feb-28, Volume: 25
2020
Ranking Self-reported Gastrointestinal Side Effects of Pharmacotherapy in Sarcoidosis.
Lung, Volume: 198, Issue: 2
2020
Efficacy and safety of iguratimod on patients with relapsed or refractory IgG4-related disease.
Clinical rheumatology, Volume: 39, Issue: 2
2020
Systemic safety analysis of mycophenolate in Graves' orbitopathy.
Journal of endocrinological investigation, Volume: 43, Issue: 6
2020
Efficacy and safety of mycophenolate mofetil in patients with virus-negative lymphocytic myocarditis: A prospective cohort study.
Journal of autoimmunity, Volume: 106
2020
Vancomycin relieves mycophenolate mofetil-induced gastrointestinal toxicity by eliminating gut bacterial β-glucuronidase activity.
Science advances, Volume: 5, Issue: 8
2019
Accuracy, discriminative properties and reliability of a human ESC-based in vitro toxicity assay to distinguish teratogens responsible for neural tube defects.
Archives of toxicology, Volume: 93, Issue: 8
2019
Exposure-Toxicity Relationships of Mycophenolic Acid in Adult Kidney Transplant Patients.
Clinical pharmacokinetics, Volume: 58, Issue: 12
2019
Efficacy and Safety of Mizoribine Combined With Tacrolimus in Living Donor Kidney Transplant Recipients: 3-Year Results by a Chinese Single Center Study.
Transplantation proceedings, Volume: 51, Issue: 5
2019
Adverse effects of mycophenolic acid in renal transplant recipients: gender differences.
International journal of clinical pharmacy, Volume: 41, Issue: 3
2019
Genetic Variants Associated With Immunosuppressant Pharmacokinetics and Adverse Effects in the DeKAF Genomics Genome-wide Association Studies.
Transplantation, Volume: 103, Issue: 6
2019
Safety of Everolimus With Reduced Calcineurin Inhibitor Exposure in De Novo Kidney Transplants: An Analysis From the Randomized TRANSFORM Study.
Transplantation, Volume: 103, Issue: 9
2019
Predictable and Unusual Adverse Effects of Immunosuppression in Pediatric Liver Transplant Patients.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 17, Issue: Suppl 1
2019
A new analytic tool developed to assess safe use recommendations.
Pharmacoepidemiology and drug safety, Volume: 28, Issue: 5
2019
RELATE: Relationship of limited sampling strategy and adverse effects of mycophenolate mofetil in pediatric renal transplant patients.
Pediatric transplantation, Volume: 23, Issue: 2
2019
Efficacy and Safety of Delayed Prolonged-Release Tacrolimus Initiation in De Novo Hepatitis C Virus-Negative Orthotopic Liver Transplant Recipients: A Single-Center, Single-Arm, Prospective Study.
Annals of transplantation, Jan-18, Volume: 24
2019
A randomized, controlled double-blind study comparing the efficacy and safety of dose-ranging voclosporin with placebo in achieving remission in patients with active lupus nephritis.
Kidney international, Volume: 95, Issue: 1
2019
The impact of tacrolimus exposure on extrarenal adverse effects in adult renal transplant recipients.
British journal of clinical pharmacology, Volume: 85, Issue: 3
2019
Efficacy and Safety of Mycophenolate Mofetil Versus Intravenous Pulse Cyclophosphamide as Induction Therapy in Proliferative Lupus Nephritis.
Iranian journal of kidney diseases, Volume: 12, Issue: 5
2018
Efficacy and safety of mycophenolate mofetil in progressive multiple sclerosis patients.
Journal of neurology, Volume: 265, Issue: 11
2018
An intact microbiota is required for the gastrointestinal toxicity of the immunosuppressant mycophenolate mofetil.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, Volume: 37, Issue: 9
2018
Efficacy and safety of low dose Mycophenolate mofetil treatment for immunoglobulin G4-related disease: a randomized clinical trial.
Rheumatology (Oxford, England), 01-01, Volume: 58, Issue: 1
2019
Differential profiles of adverse events associated with mycophenolate mofetil between adult and pediatric renal transplant patients.
The Journal of international medical research, Volume: 46, Issue: 11
2018
Comparative efficacy and safety of low-dose and high-dose cyclophosphamide as induction therapy for lupus nephritis: a network meta-analysis.
Zeitschrift fur Rheumatologie, Volume: 78, Issue: 5
2019
Side effects and efficacy of renal sparing immunosuppression in pediatric liver transplantation-A single center matched cohort study.
Pediatric transplantation, Volume: 22, Issue: 5
2018
Steroid-free immunosuppression with low-dose tacrolimus is safe and significantly reduces the incidence of new-onset diabetes mellitus following liver transplantation.
Scandinavian journal of gastroenterology, Volume: 53, Issue: 6
2018
Systemic inflammatory response in a liver transplant recipient: a potential side effect of mycophenolate mofetil.
BMJ case reports, Mar-28, Volume: 2018
2018
Efficacy and safety of everolimus plus low-dose calcineurin inhibitor vs. mycophenolate mofetil plus standard-dose calcineurin inhibitor in renal transplant recipients: A systematic review and meta-analysis
.
Clinical nephrology, Volume: 89, Issue: 5
2018
Efficacy and Safety of Mycophenolate Mofetil in Patients With Autoimmune Hepatitis and Suboptimal Outcomes After Standard Therapy.
Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association, Volume: 16, Issue: 2
2018
Efficacy and safety of mycophenolate mofetil in patients with IgA nephropathy: an update meta-analysis.
BMC nephrology, Jul-19, Volume: 18, Issue: 1
2017
Efficacy and safety of multitarget therapy with cyclophosphamide and tacrolimus for lupus nephritis: a prospective, single-arm, single-centre, open label pilot study in Japan.
Lupus, Volume: 27, Issue: 2
2018
Retrospective evaluation of the efficacy and safety of belatacept with thymoglobulin induction and maintenance everolimus: A single-center clinical experience.
Clinical transplantation, Volume: 31, Issue: 9
2017
Efficacy and Safety of Mycophenolate Mofetil and Tacrolimus as Second-line Therapy for Patients With Autoimmune Hepatitis.
Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association, Volume: 15, Issue: 12
2017
Validation of the cell line LS180 as a model for study of the gastrointestinal toxicity of mycophenolic acid.
Xenobiotica; the fate of foreign compounds in biological systems, Volume: 48, Issue: 5
2018
Effectiveness and safety of mycophenolate mofetil in idiopathic pulmonary fibrosis.
PloS one, Volume: 12, Issue: 4
2017
Safety of oral and intravenous mycophenolate mofetil in healthy cats.
Journal of feline medicine and surgery, Volume: 20, Issue: 2
2018
Efficacy and safety of rituximab in comparison with common induction therapies in pediatric active lupus nephritis.
Pediatric nephrology (Berlin, Germany), Volume: 32, Issue: 6
2017
Mesenchymal Stem Cells Attenuate the Adverse Effects of Immunosuppressive Drugs on Distinct T Cell Subopulations.
Stem cell reviews and reports, Volume: 13, Issue: 1
2017
Cytokine Profile in Calcineurin Inhibitor-Induced Chronic Nephrotoxicity in Chinese Liver Transplant Recipients.
Transplantation proceedings, Volume: 48, Issue: 8
2016
Combination of tacrolimus and mycophenolate mofetil induces oxidative stress and genotoxicity in spleen and bone marrow of Wistar rats.
Mutation research. Genetic toxicology and environmental mutagenesis, Nov-01, Volume: 810
2016
Efficacy and Safety of Monotherapy With Mycophenolate Mofetil in Liver Transplantation Patients With Nephrotoxicity.
Transplantation proceedings, Volume: 48, Issue: 7
2016
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Long-term Use (72)

ArticleYear
The long-term effects of multidrug immunosuppressive protocols based on calcineurin inhibitors and conversion to rapamycin on the morphology, apoptosis, and proliferation of rat salivary glands.
Pharmacological reports : PR, Volume: 75, Issue: 5
2023
Evaluation of T-Cell Immune Status of Reduced-Dose Cyclosporine and Everolimus Combination Therapy in Kidney Transplant Patients.
Transplantation proceedings, Volume: 55, Issue: 4
2023
Mycophenolate mofetil and telmisartan for the treatment of proteinuria secondary to minimal change disease podocytopathy in a dog.
Journal of veterinary internal medicine, Volume: 36, Issue: 6
2022
Pemphigus Vulgaris Aggravated: Rifampicin Found at the Scene of the Crime.
Cutis, Volume: 109, Issue: 5
2022
Systemic treatment of children and adolescents with atopic dermatitis aged ≥2 years: a Delphi consensus project mapping expert opinion in Northern Europe.
Journal of the European Academy of Dermatology and Venereology : JEADV, Volume: 36, Issue: 11
2022
The Mycophenolate-based Immunosuppressive Regimen Is Associated With Increased Mortality in Kidney Transplant Patients With COVID-19.
Transplantation, 10-01, Volume: 106, Issue: 10
2022
Nodular Regenerative Hyperplasia of the liver in Juvenile Dermatomyositis.
Pediatric rheumatology online journal, Apr-20, Volume: 20, Issue: 1
2022
Mycophenolate Mofetil as a Rescue Therapy in Frequently Relapsing/Steroid-Dependent Nephrotic Syndrome in Children; Ability to Maintain Remission.
Iranian journal of kidney diseases, Volume: 15, Issue: 5
2021
Gastrointestinal complications after kidney transplantation.
World journal of gastroenterology, Oct-14, Volume: 26, Issue: 38
2020
Target-oriented delivery of self-assembled immunosuppressant cocktails prolongs allogeneic orthotopic liver transplant survival.
Journal of controlled release : official journal of the Controlled Release Society, 12-10, Volume: 328
2020
Scleroderma-related interstitial lung disease: principles of management.
Expert review of respiratory medicine, Volume: 13, Issue: 4
2019
Th1, Th2, Th17 cell subsets in two different immunosuppressive protocols in renal allograft recipients (Sirolimus vs mycophenolate mofetil): A cohort study.
International immunopharmacology, Volume: 67
2019
Study protocol: mycophenolate mofetil as maintenance therapy after rituximab treatment for childhood-onset, complicated, frequently-relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome: a multicenter double-blind, randomized, placebo-contr
BMC nephrology, 11-01, Volume: 19, Issue: 1
2018
It hasn't gone away: the problem of glucocorticoid use in lupus remains.
Rheumatology (Oxford, England), 04-01, Volume: 56, Issue: suppl_1
2017
Current State of Immunosuppression: Past, Present, and Future.
Critical reviews in eukaryotic gene expression, Volume: 25, Issue: 2
2015
Tacrolimus and mycophenolate mofetil associations: Induction of oxidative stress or antioxidant effect?
Human & experimental toxicology, Volume: 34, Issue: 11
2015
Tacrolimus-based, steroid-free regimens in renal transplantation: 3-year follow-up of the ATLAS trial.
Transplantation, Sep-15, Volume: 94, Issue: 5
2012
Mycophenolate mofetil and FK506 have different effects on kidney allograft fibrosis in rats that underwent chronic allograft nephropathy.
BMC nephrology, Jul-02, Volume: 13
2012
Conversion from cyclosporine to sirolimus in chronic renal allograft dysfunction: a 4-year prospective study.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 9, Issue: 1
2011
Steroid avoidance reduce the cost of morbidities after live-donor renal allotransplants: a prospective, randomized, controlled study.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 9, Issue: 2
2011
Pharmacokinetic evaluation of mycophenolate mofetil for pemphigus.
Expert opinion on drug metabolism & toxicology, Volume: 7, Issue: 2
2011
The effects of early rapid corticosteroid reduction on cell-mediated immunity in kidney transplant recipients.
Transplant immunology, Jan-15, Volume: 24, Issue: 2
2011
Combination therapy of mycophenolate mofetil and tacrolimus in lupus nephritis.
Lupus, Volume: 19, Issue: 8
2010
Cost-benefit of steroid avoidance in renal transplant patients: a prospective randomized study.
Scandinavian journal of urology and nephrology, Volume: 44, Issue: 3
2010
Impact of daclizumab, low-dose cyclosporine, mycophenolate mofetil and steroids on renal function after kidney transplantation.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Volume: 25, Issue: 1
2010
Oral beclomethasone dipropionate for the treatment of gastrointestinal chronic graft-versus-host disease.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 15, Issue: 10
2009
Minimization of maintenance immunosuppression early after renal transplantation: an interim analysis.
Transplantation, Aug-15, Volume: 88, Issue: 3
2009
Efficacy and safety of long-term mycophenolate sodium therapy in pemphigus vulgaris.
Journal of the European Academy of Dermatology and Venereology : JEADV, Volume: 23, Issue: 12
2009
[Prolonged complete clinical remission in patients with severe pemphigus vulgaris after cycles of intravenous cyclophosphamide].
Actas dermo-sifiliograficas, Volume: 100, Issue: 2
2009
Safe conversion of mycophenolate mofetil to azathioprine in kidney transplant recipients with sirolimus-based immunosuppression.
Nephrology (Carlton, Vic.), Volume: 14, Issue: 2
2009
Calcineurin inhibitor sparing in renal transplantation.
Transplantation, Sep-27, Volume: 86, Issue: 6
2008
Influence of lansoprazole and rabeprazole on mycophenolic acid pharmacokinetics one year after renal transplantation.
Therapeutic drug monitoring, Volume: 30, Issue: 1
2008
Steroid-avoidance immunosuppression regimen in live-donor renal allotransplant recipients: a prospective, randomized, controlled study.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 5, Issue: 2
2007
[Mycophenolate mofetil combined with steroids: new experiences in the treatment of idiopathic retroperitoneal fibrosis].
Vojnosanitetski pregled, Volume: 64, Issue: 6
2007
Monitoring of inosine monophosphate dehydrogenase activity as a biomarker for mycophenolic acid effect: potential clinical implications.
Therapeutic drug monitoring, Volume: 29, Issue: 2
2007
Mycophenolate mofetil promotes prolonged improvement of renal dysfunction after pediatric liver transplantation: experience of a single center.
Pediatric transplantation, Volume: 11, Issue: 1
2007
Impact of tacrolimus and mycophenolate mofetil combination on cardiovascular risk profile after kidney transplantation.
Journal of the American Society of Nephrology : JASN, Volume: 17, Issue: 12 Suppl 3
2006
A prospective study on the use of mycophenolate mofetil in children with cyclosporine-dependent nephrotic syndrome.
Pediatric nephrology (Berlin, Germany), Volume: 22, Issue: 1
2007
Impact of cyclosporine reduction with MMF: a randomized trial in chronic allograft dysfunction. The 'reference' study.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, Volume: 6, Issue: 11
2006
Long-term administration of enteric-coated mycophenolate sodium (EC-MPS; myfortic) is safe in kidney transplant patients.
Clinical nephrology, Volume: 66, Issue: 2
2006
Long-term safety and efficacy after conversion of maintenance renal transplant recipients from mycophenolate mofetil (MMF) to enteric-coated mycophenolate sodium (EC-MPA, myfortic).
Clinical nephrology, Volume: 66, Issue: 2
2006
Immune suppression attenuates hypertension and renal disease in the Dahl salt-sensitive rat.
Hypertension (Dallas, Tex. : 1979), Volume: 48, Issue: 1
2006
Toxicity-sparing protocols using mycophenolate mofetil in renal transplantation.
Transplantation, Oct-15, Volume: 80, Issue: 2 Suppl
2005
A pilot study using mycophenolate mofetil in relapsing or resistant ANCA small vessel vasculitis.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Volume: 20, Issue: 12
2005
Mycophenolate mofetil for renal dysfunction after pediatric liver transplantation.
Transplantation, Jun-15, Volume: 79, Issue: 11
2005
Long-term administration of enteric-coated mycophenolate sodium in kidney transplant patients.
Transplantation proceedings, Volume: 37, Issue: 2
2005
A large, prospective, randomized, open-label, multicentre study of corticosteroid withdrawal in SPK transplantation: a 3-year report.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Volume: 20 Suppl 2
2005
Mycophenolate mofetil monotherapy in liver transplant recipients: a single center experience.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 10, Issue: 9
2004
Time-related clinical determinants of long-term tacrolimus pharmacokinetics in combination therapy with mycophenolic acid and corticosteroids: a prospective study in one hundred de novo renal transplant recipients.
Clinical pharmacokinetics, Volume: 43, Issue: 11
2004
Analysis of the cardiovascular risk profile in stable kidney transplant recipients after 50% cyclosporine reduction.
Clinical transplantation, Volume: 18, Issue: 4
2004
Clinical efficacy and toxicity profile of tacrolimus and mycophenolic acid in relation to combined long-term pharmacokinetics in de novo renal allograft recipients.
Clinical pharmacology and therapeutics, Volume: 75, Issue: 5
2004
Long-term use of mycophenolate mofetil is associated with a reduction in the incidence and risk of late rejection.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, Volume: 3, Issue: 1
2003
Mycophenolate mofetil: a pharmacoeconomic review of its use in solid organ transplantation.
PharmacoEconomics, Volume: 20, Issue: 10
2002
Strategies to reduce toxicities and improve outcomes in renal transplant recipients.
Pharmacotherapy, Volume: 22, Issue: 3
2002
Reducing adverse effects of immunosuppressive agents in kidney transplant recipients.
Progress in transplantation (Aliso Viejo, Calif.), Volume: 11, Issue: 4
2001
Open randomized trial comparing early withdrawal of either cyclosporine or mycophenolate mofetil in stable renal transplant recipients initially treated with a triple drug regimen.
Journal of the American Society of Nephrology : JASN, Volume: 13, Issue: 2
2002
A pilot study of steroid-free immunosuppression in the prevention of acute rejection in renal allograft recipients.
Transplantation, Sep-15, Volume: 72, Issue: 5
2001
Systemic mycophenolate mofetil in comparison with systemic cyclosporin A in high-risk keratoplasty patients: 3 years' results of a randomized prospective clinical trial.
Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie, Volume: 239, Issue: 5
2001
Multicenter trial exploring calcineurin inhibitors avoidance in renal transplantation.
Transplantation, May-15, Volume: 71, Issue: 9
2001
[Treatment of uveitis with immunosuppressives: antimetabolites and alkylating agents].
Bulletin de la Societe belge d'ophtalmologie, Issue: 279
2001
Mycophenolate (CellCept) treatment of myasthenia gravis, chronic inflammatory polyneuropathy and inclusion body myositis.
Journal of the neurological sciences, Apr-01, Volume: 185, Issue: 2
2001
The use of intravenous tacrolimus and mycophenolate mofetil as induction and maintenance immunosuppression in simultaneous pancreas--kidney recipients with previous transplants.
Clinical transplantation, Volume: 15, Issue: 2
2001
Effect of mycophenolate mofetil therapy on inosine monophosphate dehydrogenase induction in red blood cells of heart transplant recipients.
Clinical pharmacology and therapeutics, Volume: 69, Issue: 3
2001
Randomized study on the conversion of treatment with cyclosporine to azathioprine or mycophenolate mofetil followed by dose reduction.
Transplantation, Jul-15, Volume: 70, Issue: 1
2000
Mycophenolate mofetil in cadaveric renal transplantation. US Renal Transplant Mycophenolate Mofetil Study Group.
American journal of kidney diseases : the official journal of the National Kidney Foundation, Volume: 34, Issue: 2
1999
Mycophenolic acid for psoriasis. A review of pharmacology, long-term efficacy, and safety.
Journal of the American Academy of Dermatology, Volume: 17, Issue: 6
1987
RS-61443--a new, potent immunosuppressive agent.
Transplantation, Volume: 51, Issue: 1
1991
Mycophenolic acid in the treatment of psoriasis: long-term administration.
Archives of dermatology, Volume: 113, Issue: 7
1977
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Pharmacokinetics (402)

ArticleYear
Analytical and Clinical Validation of Assays for Volumetric Absorptive Microsampling (VAMS) of Drugs in Different Blood Matrices: A Literature Review.
Molecules (Basel, Switzerland), Aug-14, Volume: 28, Issue: 16
2023
Significant Effects of Renal Function on Mycophenolic Acid Total Clearance in Pediatric Kidney Transplant Recipients with Population Pharmacokinetic Modeling.
Clinical pharmacokinetics, Volume: 62, Issue: 9
2023
Development of a novel UPLC-MS/MS method for the simultaneous quantification of mycophenolic mofetil, mycophenolic acid, and its major metabolites: Application to pharmacokinetic and tissue distribution study in rats.
Journal of pharmaceutical and biomedical analysis, Sep-20, Volume: 234
2023
Therapeutic drug monitoring of mycophenolic acid (MPA) using volumetric absorptive microsampling (VAMS) in pediatric renal transplant recipients: ultra-high-performance liquid chromatography-tandem mass spectrometry analytical method development, cross-va
Pharmacological reports : PR, Volume: 75, Issue: 4
2023
Clinical Evidence on the Purported Pharmacokinetic Interactions between Corticosteroids and Mycophenolic Acid.
Clinical pharmacokinetics, Volume: 62, Issue: 2
2023
Influence of SLCO1B1 Polymorphisms on the Pharmacokinetics of Mycophenolic Acid in Renal Transplant Recipients.
Current drug metabolism, Volume: 24, Issue: 2
2023
Volumetric Absorptive Microsampling to Enhance the Therapeutic Drug Monitoring of Tacrolimus and Mycophenolic Acid: A Systematic Review and Critical Assessment.
Therapeutic drug monitoring, 08-01, Volume: 45, Issue: 4
2023
Population Pharmacokinetics of Enteric-Coated Mycophenolate Sodium in Children after Renal Transplantation and Initial Dosage Recommendation Based on Body Surface Area.
Computational and mathematical methods in medicine, Volume: 2022
2022
The Immunobiogram, a novel in vitro diagnostic test to measure the pharmacodynamic response to immunosuppressive therapy in kidney transplant patients.
Transplant immunology, Volume: 75
2022
Noncompartmental pharmacokinetics of three intravenous mycophenolate mofetil concentrations in healthy Standardbred mares.
Veterinary dermatology, Volume: 34, Issue: 3
2023
[Pharmacokinetic study of mycophenolic acid in pediatric kidney transplantation].
Andes pediatrica : revista Chilena de pediatria, Volume: 93, Issue: 2
2022
Important lack of difference in tacrolimus and mycophenolic acid pharmacokinetics between Aboriginal and Caucasian kidney transplant recipients.
Nephrology (Carlton, Vic.), Volume: 27, Issue: 9
2022
Volumetric microsampling for simultaneous remote immunosuppressant and kidney function monitoring in outpatient kidney transplant recipients.
British journal of clinical pharmacology, Volume: 88, Issue: 11
2022
Pharmacokinetics of free and total mycophenolic acid in paediatric and adult renal transplant recipients: Exploratory analysis of the effects of clinical factors and gene variants.
Basic & clinical pharmacology & toxicology, Volume: 131, Issue: 1
2022
Population pharmacokinetics and Bayesian estimation of mycophenolate mofetil in patients with autoimmune hepatitis.
British journal of clinical pharmacology, Volume: 88, Issue: 11
2022
Pharmacokinetics of mycophenolic acid and external evaluation of two limited sampling strategies of drug exposure in patients with juvenile systematic lupus erythematosus.
European journal of clinical pharmacology, Volume: 78, Issue: 6
2022
Are in clinical practice measurements of concentrations and the calculation of mycophenolate mofetil pharmacokinetic parameters needed for optimizing therapy in patients with renal diseases or kidney transplantation?
Advances in clinical and experimental medicine : official organ Wroclaw Medical University, Volume: 31, Issue: 5
2022
Recent lessons learned from population pharmacokinetic studies of mycophenolic acid: physiological, genomic, and drug interactions leading to the prediction of drug effects.
Expert opinion on drug metabolism & toxicology, Volume: 17, Issue: 12
2021
Comparison of conventional dried blood spots and volumetric absorptive microsampling for tacrolimus and mycophenolic acid determination.
Journal of pharmaceutical and biomedical analysis, Jan-20, Volume: 208
2022
Pharmacokinetics of Mycophenolate Mofetil Metabolites in Older Patients on the Seventh Day After Renal Transplantation.
Transplantation proceedings, Volume: 53, Issue: 7
2021
Genetic polymorphisms in metabolic enzymes and transporters have no impact on mycophenolic acid pharmacokinetics in adult kidney transplant patients co-treated with tacrolimus: A population analysis.
Journal of clinical pharmacy and therapeutics, Volume: 46, Issue: 6
2021
Variants in mycophenolate and CMV antiviral drug pharmacokinetic and pharmacodynamic genes and leukopenia in heart transplant recipients.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, Volume: 40, Issue: 9
2021
The Impact of Genetic Polymorphisms on the Pharmacokinetics and Pharmacodynamics of Mycophenolic Acid: Systematic Review and Meta-analysis.
Clinical pharmacokinetics, Volume: 60, Issue: 10
2021
Pharmacokinetics of mycophenolate mofetil following single-dose intravenous and single- and multiple-dose oral administration and clinicopathologic effects of mycophenolate mofetil following long-term oral administration in healthy horses.
American journal of veterinary research, Volume: 82, Issue: 6
2021
Single-dose pharmacokinetics of mycophenolic acid following administration of immediate-release mycophenolate mofetil in healthy Beagle dogs.
Journal of veterinary pharmacology and therapeutics, Volume: 44, Issue: 4
2021
Assessment of pharmacokinetic mycophenolic acid clearance models using Monte Carlo numerical analysis.
Xenobiotica; the fate of foreign compounds in biological systems, Volume: 51, Issue: 4
2021
Population pharmacokinetics of mycophenolic acid in paediatric patients.
British journal of clinical pharmacology, Volume: 87, Issue: 4
2021
Population Pharmacokinetic Model of Plasma and Cellular Mycophenolic Acid in Kidney Transplant Patients from the CIMTRE Study.
Drugs in R&D, Volume: 20, Issue: 4
2020
Population pharmacokinetics of mycophenolic acid in Mexican patients with lupus nephritis.
Lupus, Volume: 29, Issue: 9
2020
A short overview on mycophenolic acid pharmacology and pharmacokinetics.
Clinical transplantation, Volume: 34, Issue: 8
2020
Pharmacokinetic evaluation of MFF in combinations with tacrolimus and cyclosporine. Findings of C0 and AUC.
Medicine, Volume: 99, Issue: 12
2020
Population pharmacokinetics and Bayesian estimators for intravenous mycophenolate mofetil in haematopoietic stem cell transplant patients.
British journal of clinical pharmacology, Volume: 86, Issue: 8
2020
Pharmacodynamic assessment of mycophenolic acid in resting and activated target cell population during the first year after renal transplantation.
British journal of clinical pharmacology, Volume: 86, Issue: 6
2020
Nuclear factor of activated T cells as potential pharmacodynamic biomarker for the risk of acute and subclinical rejection in de novo liver recipients.
Liver international : official journal of the International Association for the Study of the Liver, Volume: 40, Issue: 4
2020
Mycophenolic Acid and Its Pharmacokinetic Drug-Drug Interactions in Humans: Review of the Evidence and Clinical Implications.
Journal of clinical pharmacology, Volume: 60, Issue: 3
2020
The development of a population physiologically based pharmacokinetic model for mycophenolic mofetil and mycophenolic acid in humans using data from plasma, saliva, and kidney tissue.
Biopharmaceutics & drug disposition, Volume: 40, Issue: 9
2019
Prospective study of the changes in pharmacokinetics of immunosuppressive medications after laparoscopic sleeve gastrectomy.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, Volume: 20, Issue: 2
2020
Pharmacodynamic Monitoring of Mycophenolic Acid Therapy: Improved Liquid Chromatography-Tandem Mass Spectrometry Method for Measuring Inosin-5'-Monophosphate Dehydrogenase Activity.
Therapeutic drug monitoring, Volume: 42, Issue: 2
2020
Interaction Between Cyclosporine and Palbociclib in a Renal Transplant Patient: Case Report and Pharmacokinetic Perspective.
Journal of pharmacy practice, Volume: 33, Issue: 6
2020
Pharmacokinetics of Enteric-Coated Mycophenolate Sodium in Lupus Nephritis (POEMSLUN).
Therapeutic drug monitoring, Volume: 41, Issue: 6
2019
Population Pharmacokinetics of Mycophenolic Acid Co-Administered with Tacrolimus in Corticosteroid-Free Adult Kidney Transplant Patients.
Clinical pharmacokinetics, Volume: 58, Issue: 11
2019
Pharmacokinetic Drug-Drug Interactions Between Letermovir and the Immunosuppressants Cyclosporine, Tacrolimus, Sirolimus, and Mycophenolate Mofetil.
Journal of clinical pharmacology, Volume: 59, Issue: 10
2019
Genetic Variants Associated With Immunosuppressant Pharmacokinetics and Adverse Effects in the DeKAF Genomics Genome-wide Association Studies.
Transplantation, Volume: 103, Issue: 6
2019
Systematic external evaluation of published population pharmacokinetic models of mycophenolate mofetil in adult kidney transplant recipients co-administered with tacrolimus.
British journal of clinical pharmacology, Volume: 85, Issue: 4
2019
Nephrotic state substantially enhances apparent mycophenolic acid clearance
.
Clinical nephrology, Volume: 91, Issue: 3
2019
Population Pharmacokinetics and Bayesian Estimation of Mycophenolic Acid Exposure in Chinese Renal Allograft Recipients After Administration of EC-MPS.
Journal of clinical pharmacology, Volume: 59, Issue: 4
2019
Pharmacokinetics of free and total mycophenolic acid in adult lupus nephritis patients-implications for therapeutic drug monitoring.
European journal of clinical pharmacology, Volume: 75, Issue: 3
2019
Population Pharmacokinetic Analysis of Immediate-Release Oral Tacrolimus Co-administered with Mycophenolate Mofetil in Corticosteroid-Free Adult Kidney Transplant Recipients.
European journal of drug metabolism and pharmacokinetics, Volume: 44, Issue: 3
2019
Longitudinal Pharmacokinetics of Mycophenolic Acid in Elderly Renal Transplant Recipients Compared to a Younger Control Group: Data from the nEverOld Trial.
European journal of drug metabolism and pharmacokinetics, Volume: 44, Issue: 2
2019
Pharmacokinetics and pharmacogenomics of mycophenolic acid and its clinical correlations in maintenance immunosuppression for lupus nephritis.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 05-01, Volume: 35, Issue: 5
2020
Therapeutic drug monitoring of tacrolimus and mycophenolic acid in outpatient renal transplant recipients using a volumetric dried blood spot sampling device.
British journal of clinical pharmacology, Volume: 84, Issue: 12
2018
Influence of uridine diphosphate-glucuronosyltransferases (1A9) polymorphisms on mycophenolic acid pharmacokinetics in patients with renal transplant.
Renal failure, Volume: 40, Issue: 1
2018
Optimization and application of an HPLC method for quantification of inosine-5'-monophosphate dehydrogenase activity as a pharmacodynamic biomarker of mycophenolic acid in Chinese renal transplant patients.
Clinica chimica acta; international journal of clinical chemistry, Volume: 485
2018
Pharmacokinetic Comparison of Two Mycophenolate Mofetil Formulations in Kidney Transplant Recipients.
Therapeutic drug monitoring, Volume: 40, Issue: 5
2018
Pharmacokinetics Evaluation of Mycophenolic Acid and Its Glucuronide Metabolite in Chinese Renal Transplant Recipients Receiving Enteric-Coated Mycophenolate Sodium and Tacrolimus.
Therapeutic drug monitoring, Volume: 40, Issue: 5
2018
Pharmacokinetic and Pharmacodynamic Markers of Mycophenolic Acid Associated with Effective Prophylaxis for Acute Graft-Versus-Host Disease and Neutrophil Engraftment in Cord Blood Transplant Patients.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 24, Issue: 7
2018
Pharmacokinetic Interactions Between Elbasvir/Grazoprevir and Immunosuppressant Drugs in Healthy Volunteers.
Journal of clinical pharmacology, Volume: 58, Issue: 5
2018
Influence of Genetic Polymorphisms on Mycophenolic Acid Pharmacokinetics and Patient Outcomes in Renal Transplantation.
Current drug metabolism, Volume: 19, Issue: 14
2018
Pharmacokinetics of Mycophenolic Acid after Intravenous Administration of Mycophenolate Mofetil to Healthy Cats.
Journal of veterinary internal medicine, Volume: 31, Issue: 6
2017
Population Pharmacokinetics of Mycophenolic Acid: An Update.
Clinical pharmacokinetics, Volume: 57, Issue: 5
2018
Pharmacodynamic Monitoring of Tacrolimus-Based Immunosuppression in CD14+ Monocytes After Kidney Transplantation.
Therapeutic drug monitoring, Volume: 39, Issue: 5
2017
Steady-state pharmacokinetics of mycophenolic acid in renal transplant patients: exploratory analysis of the effects of cyclosporine, recipients' and donors' ABCC2 gene variants, and their interactions.
European journal of clinical pharmacology, Volume: 73, Issue: 9
2017
Influence of cyclosporine and everolimus on the main mycophenolate mofetil pharmacokinetic parameters: Cross-sectional study.
Medicine, Volume: 96, Issue: 13
2017
Influence of Proton Pump Inhibitors on Mycophenolic Acid Pharmacokinetics in Patients With Renal Transplantation and the Relationship With Cytochrome 2C19 Gene Polymorphism.
Transplantation proceedings, Volume: 49, Issue: 3
2017
Pharmacokinetics of Mycophenolic Acid and Dose Optimization in Children After Intestinal Transplantation.
Therapeutic drug monitoring, Volume: 39, Issue: 1
2017
The pharmacokinetics and pharmacodynamics of mycophenolate mofetil in younger and elderly renal transplant recipients.
British journal of clinical pharmacology, Volume: 83, Issue: 4
2017
Population pharmacokinetics of mycophenolic acid and its glucuronide metabolite in lung transplant recipients with and without cystic fibrosis.
Xenobiotica; the fate of foreign compounds in biological systems, Volume: 47, Issue: 8
2017
Clinical Pharmacokinetics of Mycophenolic Acid in Hematopoietic Stem Cell Transplantation Recipients.
European journal of drug metabolism and pharmacokinetics, Volume: 42, Issue: 2
2017
1-Alpha, 25-dihydroxyvitamin D3 alters the pharmacokinetics of mycophenolic acid in renal transplant recipients by regulating two extrahepatic UDP-glucuronosyltransferases 1A8 and 1A10.
Translational research : the journal of laboratory and clinical medicine, Volume: 178
2016
Mycophenolic Acid Pharmacokinetics and Relapse in Children with Steroid-Dependent Idiopathic Nephrotic Syndrome.
Clinical journal of the American Society of Nephrology : CJASN, 10-07, Volume: 11, Issue: 10
2016
Pharmacokinetic Assessment of Drug-Drug Interactions of Isavuconazole With the Immunosuppressants Cyclosporine, Mycophenolic Acid, Prednisolone, Sirolimus, and Tacrolimus in Healthy Adults.
Clinical pharmacology in drug development, Volume: 6, Issue: 1
2017
Effects of poloxamer 407-induced hyperlipidemia on hepatic multidrug resistance protein 2 (Mrp2/Abcc2) and the pharmacokinetics of mycophenolic acid in rats.
Biopharmaceutics & drug disposition, Volume: 37, Issue: 6
2016
Pharmacokinetic Variability of Mycophenolic Acid in Pediatric and Adult Patients With Hematopoietic Stem Cell Transplantation.
Journal of clinical pharmacology, Volume: 56, Issue: 11
2016
Pharmacokinetic Analysis of Mycophenolate Mofetil and Enteric-Coated Mycophenolate Sodium in Calcineurin Inhibitor-Free Renal Transplant Recipients.
Therapeutic drug monitoring, Volume: 38, Issue: 3
2016
New challenges and promises in solid organ transplantation pharmacogenetics: the genetic variability of proteins involved in the pharmacodynamics of immunosuppressive drugs.
Pharmacogenomics, Volume: 17, Issue: 3
2016
Population Pharmacokinetic Approach of Immunosuppressive Therapy in Kidney Transplant Patients.
Current medicinal chemistry, Volume: 23, Issue: 19
2016
Pharmacokinetics and pharmacodynamics of mycophenolic acid in Nagase analbuminemic rats: Evaluation of protein binding effects using the modeling and simulation approach.
Drug metabolism and pharmacokinetics, Volume: 30, Issue: 6
2015
Pharmacokinetics, Pharmacodynamics, and Pharmacogenomics of Immunosuppressants in Allogeneic Hematopoietic Cell Transplantation: Part II.
Clinical pharmacokinetics, Volume: 55, Issue: 5
2016
Pharmacokinetics and Long-Term Safety and Tolerability of Everolimus in Renal Transplant Recipients Converted From Cyclosporine.
Therapeutic drug monitoring, Volume: 38, Issue: 1
2016
Mycophenolic mofetil optimized pharmacokinetic modelling, and exposure-effect associations in adult heart transplant recipients.
Pharmacological research, Volume: 99
2015
Synthesis and Pharmacokinetic Evaluation of Siderophore Biosynthesis Inhibitors for Mycobacterium tuberculosis.
Journal of medicinal chemistry, Jul-23, Volume: 58, Issue: 14
2015
Monitoring of mycophenolate mofetil metabolites in children with nephrotic syndrome and the proposed novel target values of pharmacokinetic parameters.
European journal of pharmaceutical sciences : official journal of the European Federation for Pharmaceutical Sciences, Sep-18, Volume: 77
2015
Population pharmacokinetics of mycophenolic acid in lung transplant recipients with and without cystic fibrosis.
European journal of clinical pharmacology, Volume: 71, Issue: 6
2015
No relevant pharmacokinetic interaction between pantoprazole and mycophenolate in renal transplant patients: a randomized crossover study.
British journal of clinical pharmacology, Volume: 80, Issue: 5
2015
Associations of UDP-glucuronosyltransferases polymorphisms with mycophenolate mofetil pharmacokinetics in Chinese renal transplant patients.
Acta pharmacologica Sinica, Volume: 36, Issue: 5
2015
Variability of mycophenolic acid elimination in the renal transplant recipients – population pharmacokinetic approach.
Renal failure, Volume: 37, Issue: 4
2015
Pharmacokinetics of mycophenolate sodium co-administered with tacrolimus in the first year after renal transplantation.
European journal of drug metabolism and pharmacokinetics, Volume: 41, Issue: 4
2016
Sensitive and validated LC-MS/MS methods to evaluate mycophenolic acid pharmacokinetics and pharmacodynamics in hematopoietic stem cell transplant patients.
Biomedical chromatography : BMC, Volume: 29, Issue: 9
2015
Relationship between pharmacokinetics and pharmacodynamics of calcineurin inhibitors in renal transplant patients.
Clinical transplantation, Volume: 29, Issue: 4
2015
Plasma and intracellular pharmacokinetic-pharmacodynamic analysis of mycophenolic acid in de novo kidney transplant patients.
Clinical biochemistry, Volume: 48, Issue: 6
2015
Influence of sex and race on mycophenolic acid pharmacokinetics in stable African American and Caucasian renal transplant recipients.
Clinical pharmacokinetics, Volume: 54, Issue: 4
2015
Short-term pharmacokinetic study of mycophenolate mofetil in neonatal swine.
Transplantation proceedings, Volume: 46, Issue: 10
2014
Population pharmacokinetics of mycophenolic acid and its main glucuronide metabolite: a comparison between healthy Chinese and Caucasian subjects receiving mycophenolate mofetil.
European journal of clinical pharmacology, Volume: 71, Issue: 1
2015
Population pharmacogenetic pharmacokinetic modeling for flip-flop phenomenon of enteric-coated mycophenolate sodium in kidney transplant recipients.
European journal of clinical pharmacology, Volume: 70, Issue: 10
2014
Pharmacokinetics of concentration-controlled mycophenolate mofetil in proliferative lupus nephritis: an observational cohort study.
Therapeutic drug monitoring, Volume: 36, Issue: 4
2014
Population pharmacokinetic-pharmacodynamic modelling of mycophenolic acid in paediatric renal transplant recipients in the early post-transplant period.
British journal of clinical pharmacology, Volume: 78, Issue: 5
2014
Pharmacokinetic and pharmacodynamic analysis of inosine monophosphate dehydrogenase activity in hematopoietic cell transplantation recipients treated with mycophenolate mofetil.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 20, Issue: 8
2014
Pharmacokinetics of mycophenolate mofetil in children with lupus and clinical findings in favour of therapeutic drug monitoring.
British journal of clinical pharmacology, Volume: 78, Issue: 4
2014
Circadian variation of mycophenolate mofetil pharmacokinetics in rats.
European journal of pharmaceutical sciences : official journal of the European Federation for Pharmaceutical Sciences, Jul-16, Volume: 58
2014
Effect of cyclosporine on steady-state pharmacokinetics of MPA in renal transplant recipients is not affected by the MPA formulation: analysis based on therapeutic drug monitoring data.
Therapeutic drug monitoring, Volume: 36, Issue: 4
2014
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Bioavailability (95)

ArticleYear
Temporal shifts in safety and efficacy profile of mycophenolate mofetil 2 g versus 3 g daily early after heart transplantation.
Pharmacotherapy, Volume: 42, Issue: 9
2022
Population pharmacokinetics and Bayesian estimation of mycophenolate mofetil in patients with autoimmune hepatitis.
British journal of clinical pharmacology, Volume: 88, Issue: 11
2022
Population pharmacokinetics of mycophenolic acid in paediatric patients.
British journal of clinical pharmacology, Volume: 87, Issue: 4
2021
Effect of the proton-pump Inhibitor pantoprazole on MycoPhenolic ACid exposure in kidney and liver transplant recipienTs (IMPACT study): a randomized trial.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 06-01, Volume: 35, Issue: 6
2020
Enhanced Antipsoriatic Activity of Mycophenolic Acid Against the TNF-α-Induced HaCaT Cell Proliferation by Conjugated Poloxamer Micelles.
Journal of pharmaceutical sciences, Volume: 109, Issue: 2
2020
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
Molecular pharmacology, Volume: 96, Issue: 5
2019
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
The Journal of biological chemistry, 11-15, Volume: 294, Issue: 46
2019
Population Pharmacokinetics and Bayesian Estimation of Mycophenolic Acid Exposure in Chinese Renal Allograft Recipients After Administration of EC-MPS.
Journal of clinical pharmacology, Volume: 59, Issue: 4
2019
Nanomedicine for immunosuppressive therapy: achievements in pre-clinical and clinical research.
Expert opinion on drug delivery, Volume: 15, Issue: 4
2018
Candida Esophagitis Associated With Mycophenolate Mofetil Treatment of Atopic Dermatitis.
Journal of drugs in dermatology : JDD, Oct-01, Volume: 15, Issue: 10
2016
Clinical Pharmacokinetics of Mycophenolic Acid in Hematopoietic Stem Cell Transplantation Recipients.
European journal of drug metabolism and pharmacokinetics, Volume: 42, Issue: 2
2017
Lymphatic Transport and Lymphocyte Targeting of a Triglyceride Mimetic Prodrug Is Enhanced in a Large Animal Model: Studies in Greyhound Dogs.
Molecular pharmaceutics, 10-03, Volume: 13, Issue: 10
2016
Bioavailability, Efficacy and Safety of Generic Immunosuppressive Drugs for Kidney Transplantation: A Systematic Review and Meta-Analysis.
American journal of nephrology, Volume: 44, Issue: 3
2016
Synthesis and Pharmacokinetic Evaluation of Siderophore Biosynthesis Inhibitors for Mycobacterium tuberculosis.
Journal of medicinal chemistry, Jul-23, Volume: 58, Issue: 14
2015
Population pharmacokinetics of mycophenolic acid in lung transplant recipients with and without cystic fibrosis.
European journal of clinical pharmacology, Volume: 71, Issue: 6
2015
Short-term pharmacokinetic study of mycophenolate mofetil in neonatal swine.
Transplantation proceedings, Volume: 46, Issue: 10
2014
Bioavailability of a generic of the immunosuppressive agent mycophenolate mofetil in pediatric patients.
Pediatric transplantation, Volume: 18, Issue: 6
2014
Pharmacology and toxicology of mycophenolate in organ transplant recipients: an update.
Archives of toxicology, Volume: 88, Issue: 7
2014
Effects of mycophenolic acid-glucosamine conjugates on the base of kidney targeted drug delivery.
International journal of pharmaceutics, Nov-01, Volume: 456, Issue: 1
2013
Sex differences in cyclosporine pharmacokinetics and ABCB1 gene expression in mononuclear blood cells in African American and Caucasian renal transplant recipients.
Journal of clinical pharmacology, Volume: 53, Issue: 10
2013
Determination of Mycophenolic acid in the vitreous humor using the HPLC-ESI-MS/MS method: application of intraocular pharmacokinetics study in rabbit eyes with ophthalmic implantable device.
Journal of pharmaceutical and biomedical analysis, Volume: 84
2013
Population pharmacokinetics of unbound mycophenolic acid in adult allogeneic haematopoietic cell transplantation: effect of pharmacogenetic factors.
British journal of clinical pharmacology, Volume: 75, Issue: 2
2013
Pre-transplant mycophenolate mofetil pharmacokinetics in Mexican children.
Proceedings of the Western Pharmacology Society, Volume: 54
2011
Population pharmacokinetics of mycophenolic acid and dose optimization with limited sampling strategy in liver transplant children.
British journal of clinical pharmacology, Volume: 74, Issue: 3
2012
The association of the UGT1A8, SLCO1B3 and ABCC2/ABCG2 genetic polymorphisms with the pharmacokinetics of mycophenolic acid and its phenolic glucuronide metabolite in Chinese individuals.
Clinica chimica acta; international journal of clinical chemistry, Apr-11, Volume: 413, Issue: 7-8
2012
Bayesian estimation of mycophenolate mofetil in lung transplantation, using a population pharmacokinetic model developed in kidney and lung transplant recipients.
Clinical pharmacokinetics, Jan-01, Volume: 51, Issue: 1
2012
[Relative bioavailability study of two oral formulations of mycophenolate mofetil in healthy volunteers].
Revista medica de Chile, Volume: 139, Issue: 7
2011
Omeprazole impairs the absorption of mycophenolate mofetil but not of enteric-coated mycophenolate sodium in healthy volunteers.
Journal of clinical pharmacology, Volume: 52, Issue: 8
2012
Single-dose, two-way crossover, bioequivalence study of Mycophenolate mofetil 500 mg tablet under fasting conditions in healthy male subjects.
Clinical therapeutics, Volume: 33, Issue: 3
2011
The proton pump inhibitor pantoprazole and its interaction with enteric-coated mycophenolate sodium in transplant recipients.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, Volume: 30, Issue: 5
2011
Nonlinear relationship between mycophenolate mofetil dose and mycophenolic acid exposure: implications for therapeutic drug monitoring.
Clinical journal of the American Society of Nephrology : CJASN, Volume: 6, Issue: 3
2011
Population pharmacokinetic analysis of mycophenolic acid coadministered with either tasocitinib (CP-690,550) or tacrolimus in adult renal allograft recipients.
Therapeutic drug monitoring, Volume: 32, Issue: 6
2010
Comparative bioavailability of two oral formulations of mycophenolate mofetil in healthy adult Uruguayan subjects: a case of highly variable rate of drug absorption.
International journal of clinical pharmacology and therapeutics, Volume: 48, Issue: 9
2010
Population pharmacokinetics of mycophenolic acid in children and young people undergoing blood or marrow and solid organ transplantation.
British journal of clinical pharmacology, Volume: 70, Issue: 4
2010
Population pharmacokinetics and Bayesian estimator of mycophenolic acid in children with idiopathic nephrotic syndrome.
British journal of clinical pharmacology, Volume: 69, Issue: 4
2010
Mycophenolic acid estimation by pooled sampling: a novel strategy.
Therapeutic drug monitoring, Volume: 32, Issue: 2
2010
Bioequivalence and pharmacokinetic comparison of two mycophenolate mofetil formulations in healthy Chinese male volunteers: an open-label, randomized-sequence, single-dose, two-way crossover study.
Clinical therapeutics, Volume: 32, Issue: 1
2010
Bioequivalence testing of immunosuppressants: concepts and misconceptions.
Kidney international. Supplement, Issue: 115
2010
Mycophenolate mofetil: An update.
Drugs of today (Barcelona, Spain : 1998), Volume: 45, Issue: 7
2009
Bioavailability of mycophenolate mofetil and enteric-coated mycophenolate sodium is differentially affected by pantoprazole in healthy volunteers.
Journal of clinical pharmacology, Volume: 49, Issue: 10
2009
Co-administration of grapefruit juice increases bioavailability of tacrolimus in liver transplant patients: a prospective study.
European journal of clinical pharmacology, Volume: 65, Issue: 9
2009
Comparison of 3 estimation methods of mycophenolic acid AUC based on a limited sampling strategy in renal transplant patients.
Therapeutic drug monitoring, Volume: 31, Issue: 2
2009
Population pharmacokinetics of mycophenolic acid : a comparison between enteric-coated mycophenolate sodium and mycophenolate mofetil in renal transplant recipients.
Clinical pharmacokinetics, Volume: 47, Issue: 12
2008
High-performance liquid chromatography method for the determination of mycophenolic acid in human plasma and application to a pharmacokinetic study of mycophenolic acid dispersible tablet.
Arzneimittel-Forschung, Volume: 58, Issue: 7
2008
Population pharmacokinetic modelling for enterohepatic circulation of mycophenolic acid in healthy Chinese and the influence of polymorphisms in UGT1A9.
British journal of clinical pharmacology, Volume: 65, Issue: 6
2008
Hologram QSAR model for the prediction of human oral bioavailability.
Bioorganic & medicinal chemistry, Dec-15, Volume: 15, Issue: 24
2007
Tacrolimus exposure and evolution of renal allograft histology in the first year after transplantation.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, Volume: 7, Issue: 9
2007
Pharmacokinetics of mycophenolic acid in liver transplant patients after intravenous and oral administration of mycophenolate mofetil.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 13, Issue: 6
2007
Bioavailability of a new generic formulation of mycophenolate mofetil MMF 500 versus CellCept in healthy adult volunteers.
Transplantation proceedings, Volume: 39, Issue: 4
2007
Mycophenolate mofetil inhibits tumor growth and angiogenesis in vitro but has variable antitumor effects in vivo, possibly related to bioavailability.
Transplantation, Mar-15, Volume: 83, Issue: 5
2007
Clinical pharmacokinetics and pharmacodynamics of mycophenolate in solid organ transplant recipients.
Clinical pharmacokinetics, Volume: 46, Issue: 1
2007
Highly variable mycophenolate mofetil bioavailability following nonmyeloablative hematopoietic cell transplantation.
Journal of clinical pharmacology, Volume: 47, Issue: 1
2007
Bioequivalence of enteric-coated mycophenolate sodium and mycophenolate mofetil: a meta-analysis of three studies in stable renal transplant recipients.
Transplantation, Dec-15, Volume: 82, Issue: 11
2006
Absence of an interaction between iron and mycophenolate mofetil absorption.
British journal of clinical pharmacology, Volume: 62, Issue: 4
2006
Absorption characteristics of EC-MPS--an enteric-coated formulation of mycophenolic sodium.
International journal of clinical pharmacology and therapeutics, Volume: 44, Issue: 8
2006
CYP3A5*3 influences sirolimus oral clearance in de novo and stable renal transplant recipients.
Clinical pharmacology and therapeutics, Volume: 80, Issue: 1
2006
Intravenous mycophenolate mofetil with low-dose oral tacrolimus and steroid induction for live donor liver transplantation.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 3, Issue: 2
2005
Enteric-coated mycophenolate sodium for transplant immunosuppression.
American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists, Nov-01, Volume: 62, Issue: 21
2005
Kidney immune cell infiltration and oxidative stress contribute to prenatally programmed hypertension.
Kidney international, Volume: 68, Issue: 5
2005
Population pharmacokinetics of mycophenolic acid in renal transplant recipients.
Clinical pharmacokinetics, Volume: 44, Issue: 10
2005
Mycophenolate mofetil: a dermatologic perspective.
Skin therapy letter, Volume: 10, Issue: 3
2005
Maximum a posteriori bayesian estimation of mycophenolic acid pharmacokinetics in renal transplant recipients at different postgrafting periods.
Therapeutic drug monitoring, Volume: 27, Issue: 3
2005
Pharmacokinetics and bioavailability of mycophenolic acid after intravenous administration and oral administration of mycophenolate mofetil to heart transplant recipients.
Therapeutic drug monitoring, Volume: 27, Issue: 3
2005
Mechanism of anti-human immunodeficiency virus activity of beta-D-6-cyclopropylamino-2',3'-didehydro-2',3'-dideoxyguanosine.
Antimicrobial agents and chemotherapy, Volume: 49, Issue: 5
2005
Enteric-coated mycophenolate sodium delivers bioequivalent MPA exposure compared with mycophenolate mofetil.
Clinical transplantation, Volume: 19, Issue: 2
2005
Pharmacokinetics of immunosuppressants: a perspective on ethnic differences.
International journal of clinical pharmacology and therapeutics, Volume: 42, Issue: 12
2004
Time-related clinical determinants of long-term tacrolimus pharmacokinetics in combination therapy with mycophenolic acid and corticosteroids: a prospective study in one hundred de novo renal transplant recipients.
Clinical pharmacokinetics, Volume: 43, Issue: 11
2004
Therapeutic drug monitoring of sirolimus: effect of concomitant immunosuppressive therapy and optimization of drug dosing.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, Volume: 4, Issue: 8
2004
Determination of mycophenolic acid in human plasma by high-performance liquid chromatography.
Journal of chromatography. A, Mar-26, Volume: 1031, Issue: 1-2
2004
Regulation of inosine monophosphate dehydrogenase type I and type II isoforms in human lymphocytes.
Biochemical pharmacology, Feb-15, Volume: 67, Issue: 4
2004
A randomized long-term trial of tacrolimus and sirolimus versus tacrolimus and mycophenolate mofetil versus cyclosporine (NEORAL) and sirolimus in renal transplantation. I. Drug interactions and rejection at one year.
Transplantation, Jan-27, Volume: 77, Issue: 2
2004
Gastric emptying time in renal transplant recipients treated with cyclosporine.
Transplantation proceedings, Volume: 35, Issue: 8
2003
Effect of cyclosporin pharmacokinetics on renal allograft outcome in African-Americans.
Clinical transplantation, Volume: 17, Issue: 3
2003
Pharmacokinetics of tacrolimus-based combination therapies.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Volume: 18 Suppl 1
2003
Caspofungin: new preparation. A last resort in invasive aspergillosis.
Prescrire international, Volume: 11, Issue: 61
2002
Glucocorticoids interfere with mycophenolate mofetil bioavailability in kidney transplantation.
Kidney international, Volume: 62, Issue: 3
2002
Effect of cyclosporine on mycophenolic acid area under the concentration-time curve in pediatric kidney transplant recipients.
Therapeutic drug monitoring, Volume: 23, Issue: 5
2001
The effect of selective bowel decontamination on the pharmacokinetics of mycophenolate mofetil in liver transplant recipients.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 7, Issue: 8
2001
Immunosuppressive drugs in paediatric liver transplantation.
Paediatric drugs, Volume: 3, Issue: 1
2001
Levels of mycophenolic acid and its glucuronide derivative in the plasma of liver, small bowel and kidney transplant patients receiving tacrolimus and cellcept combination therapy.
Transplant immunology, Volume: 8, Issue: 2
2000
[Mycophenolate mofetil: a new immunosuppressive drug in dermatology and its possible uses].
Der Hautarzt; Zeitschrift fur Dermatologie, Venerologie, und verwandte Gebiete, Volume: 51, Issue: 2
2000
Mizoribine and mycophenolate mofetil.
Current medicinal chemistry, Volume: 6, Issue: 7
1999
Mycophenolate mofetil and cyclosporine as graft-versus-host disease prophylaxis after allogeneic blood stem cell transplantation.
Transplantation, Feb-27, Volume: 67, Issue: 4
1999
Evidence that tacrolimus augments the bioavailability of mycophenolate mofetil through the inhibition of mycophenolic acid glucuronidation.
Therapeutic drug monitoring, Volume: 21, Issue: 1
1999
Unexpected augmentation of mycophenolic acid pharmacokinetics in renal transplant patients receiving tacrolimus and mycophenolate mofetil in combination therapy, and analogous in vitro findings.
Transplant immunology, Volume: 5, Issue: 3
1997
Clinically significant drug interactions with new immunosuppressive agents.
Drug safety, Volume: 16, Issue: 4
1997
Mycophenolate mofetil: a unique immunosuppressive agent.
American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists, Feb-01, Volume: 54, Issue: 3
1997
Pharmacokinetics and bioavailability of mycophenolate mofetil in healthy subjects after single-dose oral and intravenous administration.
Journal of clinical pharmacology, Volume: 36, Issue: 4
1996
From mice to man: the preclinical history of mycophenolate mofetil.
Clinical transplantation, Volume: 10, Issue: 1 Pt 2
1996
Single-dose pharmacokinetics of the new immunosuppressant RS-61443 in the rabbit.
Therapeutic drug monitoring, Volume: 15, Issue: 5
1993
Bioavailability improvement of mycophenolic acid through amino ester derivatization.
Pharmaceutical research, Volume: 7, Issue: 2
1990
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Dosage (761)

ArticleYear
[Clinical effect of different immunosuppressive treatment regimens in children with ocular myasthenia gravis: a retrospective analysis].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, Oct-15, Volume: 25, Issue: 10
2023
Long-term effects of average calcineurin inhibitor trough levels (over time) on renal function in a prospectively followed cohort of 150 kidney transplant recipients.
Clinical and translational science, Volume: 16, Issue: 11
2023
Mycophenolate mofetil as a treatment for presumed idiopathic chronic hepatitis in dogs: Six cases (2010-2022).
Veterinary medicine and science, Volume: 9, Issue: 6
2023
Long-term efficacy and safety of different corticosteroid courses plus mycophenolate mofetil for autoimmune encephalitis with neuronal surface antibodies without tumor.
Frontiers in immunology, Volume: 14
2023
[Off-label use of mycophenolate mofetil in immune-mediated diseases].
Revista medica de Chile, Volume: 150, Issue: 10
2022
A review of heart transplant immunosuppressants and nonmelanoma skin cancer.
Archives of dermatological research, Volume: 315, Issue: 9
2023
Utilizing therapeutic drug monitoring to optimize therapy with eculizumab and mycophenolate mofetil in a child with C3 glomerulonephritis.
Pediatric nephrology (Berlin, Germany), Volume: 38, Issue: 10
2023
SARS-CoV-2 vaccine alleviates disease burden and severity in liver transplant recipients even with low antibody titers.
Hepatology communications, 02-01, Volume: 7, Issue: 2
2023
Burden of neutropenia and leukopenia among adult kidney transplant recipients: A systematic literature review of observational studies.
Transplant infectious disease : an official journal of the Transplantation Society, Volume: 25, Issue: 1
2023
Mycophenolate-Induced Hepatotoxicity Precipitates Tacrolimus Nephrotoxicity in a Kidney Transplant Recipient: A Case Report.
Transplantation proceedings, Volume: 54, Issue: 10
2022
Population Pharmacokinetics of Enteric-Coated Mycophenolate Sodium in Children after Renal Transplantation and Initial Dosage Recommendation Based on Body Surface Area.
Computational and mathematical methods in medicine, Volume: 2022
2022
Fingerprick Microsampling Methods Can Replace Venepuncture for Simultaneous Therapeutic Drug Monitoring of Tacrolimus, Mycophenolic Acid, and Prednisolone Concentrations in Adult Kidney Transplant Patients.
Therapeutic drug monitoring, 02-01, Volume: 45, Issue: 1
2023
The Immunobiogram, a novel in vitro diagnostic test to measure the pharmacodynamic response to immunosuppressive therapy in kidney transplant patients.
Transplant immunology, Volume: 75
2022
Impacts of dosing and drug withdrawal period on tacrolimus-based triple therapy in a non-human primate renal transplantation model.
Transplant immunology, Volume: 75
2022
Influence of mycophenolate mofetil dosage and plasma levels on the occurrence of chronic lung allograft dysfunction in lung transplants: a retrospective cohort analysis.
Swiss medical weekly, 07-18, Volume: 152
2022
Evaluation of nefrotoxicity by tacrolimus and micophenolate mofetil associated with kidney ischemia and reperfusion: experimental study in rats.
Revista do Colegio Brasileiro de Cirurgioes, Volume: 49
2022
Effectiveness of delayed second dose of AZD1222 vaccine in patients with autoimmune rheumatic disease.
Clinical rheumatology, Volume: 41, Issue: 11
2022
[Pharmacokinetic study of mycophenolic acid in pediatric kidney transplantation].
Andes pediatrica : revista Chilena de pediatria, Volume: 93, Issue: 2
2022
Important lack of difference in tacrolimus and mycophenolic acid pharmacokinetics between Aboriginal and Caucasian kidney transplant recipients.
Nephrology (Carlton, Vic.), Volume: 27, Issue: 9
2022
[Benefit of therapeutic drug monitoring of immunosuppressants and immunomodulators in the management of autoimmune diseases].
La Revue de medecine interne, Volume: 43, Issue: 7
2022
In Vitro Predictive Dissolution Test Should Be Developed and Recommended as a Bioequivalence Standard for the Immediate-Release Solid Oral Dosage Forms of the Highly Variable Mycophenolate Mofetil.
Molecular pharmaceutics, 07-04, Volume: 19, Issue: 7
2022
The efficacy and safety of mycophenolate mofetil in Thai neuromyelitis optica spectrum disorder patients.
Multiple sclerosis and related disorders, Volume: 63
2022
Two-year outcomes of low-exposure extended-release tacrolimus and mycophenolate mofetil regimen in de novo kidney transplantation: A multi-center randomized controlled trial.
Clinical transplantation, Volume: 36, Issue: 6
2022
A suspected case of a multiple autoimmune syndrome in a poodle dog.
Veterinary medicine and science, Volume: 8, Issue: 2
2022
Effect of MMF Immunosuppression Based on CNI Reduction on CNI-Related Renal Damage after Lung Transplantation.
Journal of healthcare engineering, Volume: 2022
2022
Is the failure of recent trials on withdrawal of calcineurin inhibitors due to inadequate mycophenolic acid dosing?
Journal of nephrology, Volume: 35, Issue: 7
2022
Serum albumin level is associated with mycophenolic acid concentration in children with idiopathic nephrotic syndrome.
European journal of pediatrics, Volume: 181, Issue: 3
2022
A retrospective evaluation of the steroid sparing effects of oral mycophenolate mofetil (MMF) as an adjunct immunosuppressant for the treatment of canine pemphigus foliaceus.
Veterinary dermatology, Volume: 33, Issue: 1
2022
[Steroidi tra necessità e tossicità].
Giornale italiano di nefrologia : organo ufficiale della Societa italiana di nefrologia, Sep-07, Volume: 38, Issue: Suppl 77
2021
Mycophenolate Mofetil as a Rescue Therapy in Frequently Relapsing/Steroid-Dependent Nephrotic Syndrome in Children; Ability to Maintain Remission.
Iranian journal of kidney diseases, Volume: 15, Issue: 5
2021
A Systematic Review of Multiple Linear Regression-Based Limited Sampling Strategies for Mycophenolic Acid Area Under the Concentration-Time Curve Estimation.
European journal of drug metabolism and pharmacokinetics, Volume: 46, Issue: 6
2021
Impact of risk-stratified mycophenolate dosing in heart transplantation.
Clinical transplantation, Volume: 35, Issue: 11
2021
Genetic polymorphisms in metabolic enzymes and transporters have no impact on mycophenolic acid pharmacokinetics in adult kidney transplant patients co-treated with tacrolimus: A population analysis.
Journal of clinical pharmacy and therapeutics, Volume: 46, Issue: 6
2021
A retrospective study of adverse effects of mycophenolate mofetil administration to dogs with immune-mediated disease.
Journal of veterinary internal medicine, Volume: 35, Issue: 5
2021
The Impact of Genetic Polymorphisms on the Pharmacokinetics and Pharmacodynamics of Mycophenolic Acid: Systematic Review and Meta-analysis.
Clinical pharmacokinetics, Volume: 60, Issue: 10
2021
Differential efficacy of mycophenolate mofetil in adults with relapsing myelin oligodendrocyte glycoprotein antibody-associated disorders.
Multiple sclerosis and related disorders, Volume: 53
2021
Clock gene Bmal1 controls diurnal rhythms in expression and activity of intestinal carboxylesterase 1.
The Journal of pharmacy and pharmacology, Mar-01, Volume: 73, Issue: 1
2021
A systematic overview of the spermatotoxic and genotoxic effects of methotrexate, ganciclovir and mycophenolate mofetil.
Acta obstetricia et gynecologica Scandinavica, Volume: 100, Issue: 9
2021
The use of mycophenolate mofetil area under the curve.
Current opinion in rheumatology, 05-01, Volume: 33, Issue: 3
2021
Personalized Therapy for Mycophenolate: Consensus Report by the International Association of Therapeutic Drug Monitoring and Clinical Toxicology.
Therapeutic drug monitoring, 04-01, Volume: 43, Issue: 2
2021
Pneumocystis Jirovecii Pneumonia in Systemic Lupus Erythematosus: A Nationwide Cohort Study in Taiwan.
Arthritis care & research, Volume: 74, Issue: 9
2022
Single-dose pharmacokinetics of mycophenolic acid following administration of immediate-release mycophenolate mofetil in healthy Beagle dogs.
Journal of veterinary pharmacology and therapeutics, Volume: 44, Issue: 4
2021
Immunosuppressant quantification in intravenous microdialysate - towards novel quasi-continuous therapeutic drug monitoring in transplanted patients.
Clinical chemistry and laboratory medicine, 04-27, Volume: 59, Issue: 5
2021
Voclosporin: a novel calcineurin inhibitor with no impact on mycophenolic acid levels in patients with SLE.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 04-25, Volume: 37, Issue: 5
2022
Kidney Transplantation and COVID-19: Two Case Reports.
Transplantation proceedings, Volume: 53, Issue: 4
2021
Comparison of a Point-of-Care Testing with Enzyme-Multiplied Immunoassay Technique and Liquid Chromatography Combined With Tandem Mass Spectrometry Methods for Therapeutic Drug Monitoring of Mycophenolic Acid: A Preliminary Study.
Therapeutic drug monitoring, 10-01, Volume: 43, Issue: 5
2021
Recent updates on the management of autoimmune hepatitis.
Clinical and molecular hepatology, Volume: 27, Issue: 1
2021
Clinical course of COVID-19 disease in immunosuppressed renal transplant patients
Turkish journal of medical sciences, 04-30, Volume: 51, Issue: 2
2021
Weight-based mycophenolate mofetil dosing predicts acute GVHD and relapse after allogeneic hematopoietic cell transplantation.
European journal of haematology, Volume: 106, Issue: 2
2021
Evaluation of tacrolimus and mycophenolic acid removal by simultaneous continuous hemodiafiltration and plasma exchange in a lung transplant patient.
Journal of clinical pharmacy and therapeutics, Volume: 46, Issue: 2
2021
Population Pharmacokinetic Model of Plasma and Cellular Mycophenolic Acid in Kidney Transplant Patients from the CIMTRE Study.
Drugs in R&D, Volume: 20, Issue: 4
2020
Rare Oral Presentation of a Mycophenolate Mofetil-Related Other Iatrogenic Immunodeficiency-Associated Lymphoproliferative Disorder (MMF-OIA-LPD) Lesion: A Case Report and Literature Review.
Journal of oral and maxillofacial surgery : official journal of the American Association of Oral and Maxillofacial Surgeons, Volume: 79, Issue: 2
2021
Target-oriented delivery of self-assembled immunosuppressant cocktails prolongs allogeneic orthotopic liver transplant survival.
Journal of controlled release : official journal of the Controlled Release Society, 12-10, Volume: 328
2020
Establishment of a Liquid Chromatography-Tandem Mass Spectrometry Method for the Determination of Immunosuppressant Levels in the Peripheral Blood Mononuclear Cells of Chinese Renal Transplant Recipients.
Therapeutic drug monitoring, Volume: 42, Issue: 5
2020
Mycophenolic acid area under the concentration-time curve is associated with therapeutic response in childhood-onset lupus nephritis.
Pediatric nephrology (Berlin, Germany), Volume: 36, Issue: 2
2021
Treatment of Pemphigus Vulgaris and Foliaceus with Adjuvant Rituximab Compared to Immunosuppression Alone: Real-Life Experience.
Dermatology (Basel, Switzerland), Volume: 237, Issue: 2
2021
Kidney allograft recipients, immunosuppression, and coronavirus disease-2019: a report of consecutive cases from a New York City transplant center.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 07-01, Volume: 35, Issue: 7
2020
Inosine 5'-Monophosphate Dehydrogenase Activity for the Longitudinal Monitoring of Mycophenolic Acid Treatment in Kidney Allograft Recipients.
Transplantation, 04-01, Volume: 105, Issue: 4
2021
Lack of concordance between EMIT assay and LC-MS/MS for Therapeutic Drug Monitoring of Mycophenolic Acid: Potential increased risk for graft rejection?
Journal of pharmaceutical and biomedical analysis, Aug-05, Volume: 187
2020
A new model to determine Optimal Exposure to Tacrolimus and Mycophenolate Mofetil after renal transplantation.
Clinical transplantation, Volume: 34, Issue: 7
2020
The impact of age on patient tolerance of mycophenolate following kidney transplantation.
Nephrology (Carlton, Vic.), Volume: 25, Issue: 7
2020
Pharmacokinetic evaluation of MFF in combinations with tacrolimus and cyclosporine. Findings of C0 and AUC.
Medicine, Volume: 99, Issue: 12
2020
Relapse predictors and serologically unstable condition of IgG4-related disease: a large Chinese cohort.
Rheumatology (Oxford, England), 08-01, Volume: 59, Issue: 8
2020
Population pharmacokinetics and Bayesian estimators for intravenous mycophenolate mofetil in haematopoietic stem cell transplant patients.
British journal of clinical pharmacology, Volume: 86, Issue: 8
2020
Mycophenolate-induced oral ulcers: Case report and literature review.
American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists, Mar-24, Volume: 77, Issue: 7
2020
Immunosuppressants in Organ Transplantation.
Handbook of experimental pharmacology, Volume: 261
2020
Clickable, acid labile immunosuppressive prodrugs for in vivo targeting.
Biomaterials science, Jan-01, Volume: 8, Issue: 1
2020
Optimizing Mycophenolic Acid Exposure in Kidney Transplant Recipients: Time for Target Concentration Intervention.
Transplantation, Volume: 103, Issue: 10
2019
Limited Sampling Strategy for the Estimation of Mycophenolic Acid and its Acyl Glucuronide Metabolite Area under the Concentration-Time Curve in Japanese Lung Transplant Recipients.
Journal of pharmacy & pharmaceutical sciences : a publication of the Canadian Society for Pharmaceutical Sciences, Societe canadienne des sciences pharmaceutiques, Volume: 22, Issue: 1
2019
Prednisolone Concentrations in Plasma (Total and Unbound) and Saliva of Adult Kidney Transplant Recipients.
Therapeutic drug monitoring, Volume: 41, Issue: 6
2019
Generation and Validation of a Limited Sampling Strategy to Monitor Mycophenolic Acid Exposure in Children With Nephrotic Syndrome.
Therapeutic drug monitoring, Volume: 41, Issue: 6
2019

Proceedings. Mathematical, physical, and engineering sciences, Volume: 475, Issue: 2227
2019
Model based development of tacrolimus dosing algorithm considering CYP3A5 genotypes and mycophenolate mofetil drug interaction in stable kidney transplant recipients.
Scientific reports, 08-13, Volume: 9, Issue: 1
2019
Renal Transplantation in Patients With Atypical Hemolytic Uremic Syndrome: A Single Center Experience.
Transplantation proceedings, Volume: 51, Issue: 7
2019
Mammalian Target of Rapamycin Inhibitors Combined With Calcineurin Inhibitors as Initial Immunosuppression in Renal Transplantation: A Meta-analysis.
Transplantation, Volume: 103, Issue: 10
2019
Pharmacokinetics of Enteric-Coated Mycophenolate Sodium in Lupus Nephritis (POEMSLUN).
Therapeutic drug monitoring, Volume: 41, Issue: 6
2019
Early pharmacokinetics of low dosage mycophenolate exposure in Thai kidney transplant recipients.
International journal of clinical pharmacy, Volume: 41, Issue: 4
2019
Successful Treatment for BK Virus Nephropathy by Leflunomide in a Kidney Transplant Patient: A Case Report.
Transplantation proceedings, Volume: 51, Issue: 5
2019
Population Pharmacokinetics of Mycophenolic Acid Co-Administered with Tacrolimus in Corticosteroid-Free Adult Kidney Transplant Patients.
Clinical pharmacokinetics, Volume: 58, Issue: 11
2019
Adverse effects of mycophenolic acid in renal transplant recipients: gender differences.
International journal of clinical pharmacy, Volume: 41, Issue: 3
2019
Pharmacokinetic Drug-Drug Interactions Between Letermovir and the Immunosuppressants Cyclosporine, Tacrolimus, Sirolimus, and Mycophenolate Mofetil.
Journal of clinical pharmacology, Volume: 59, Issue: 10
2019
Canine sterile neutrophilic dermatosis (resembling Sweet's syndrome) with severe extracutaneous manifestations.
Schweizer Archiv fur Tierheilkunde, Volume: 161, Issue: 4
2019
Low-dose cyclosporine for active lupus nephritis: a dose titration approach.
Clinical rheumatology, Volume: 38, Issue: 8
2019
Efficacy of mycophenolate mofetil in Japanese patients with systemic lupus erythematosus.
Clinical rheumatology, Volume: 38, Issue: 6
2019
Prophylaxis and treatment with mycophenolate mofetil in children with graft-versus-host disease undergoing allogeneic hematopoietic stem cell transplantation: a nationwide survey in Japan.
International journal of hematology, Volume: 109, Issue: 4
2019
Systematic external evaluation of published population pharmacokinetic models of mycophenolate mofetil in adult kidney transplant recipients co-administered with tacrolimus.
British journal of clinical pharmacology, Volume: 85, Issue: 4
2019
Change in Mycophenolate and Tacrolimus Exposure by Transplant Vintage and Race.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 17, Issue: 6
2019
The impact of tacrolimus exposure on extrarenal adverse effects in adult renal transplant recipients.
British journal of clinical pharmacology, Volume: 85, Issue: 3
2019
Management of De Novo Mycobacterial Infection After Lung Transplantation Without Rifampicin: Case Series of a Single Institution.
Transplantation proceedings, Volume: 50, Issue: 9
2018
Short-Term Intensified Dosage Regimen of Mycophenolic Acid is Associated with Less Acute Rejection in Kidney Transplantation from Donation after Circulatory Death.
Urologia internationalis, Volume: 101, Issue: 4
2018
Pilot study comparing the childhood arthritis and rheumatology research alliance consensus treatment plans for induction therapy of juvenile proliferative lupus nephritis.
Pediatric rheumatology online journal, Oct-22, Volume: 16, Issue: 1
2018
Effect of Early Immunosuppression Therapy on De Novo Anti-Human-Leukocyte-Antigen Antibody After Kidney Transplantation.
Transplantation proceedings, Volume: 50, Issue: 8
2018
Liver Transplantation for Progressive Familial Intrahepatic Cholestasis.
Annals of transplantation, Sep-25, Volume: 23
2018
Mycophenolate mofetil in paediatric autoimmune or immune-mediated diseases of the central nervous system: clinical experience and recommendations.
Developmental medicine and child neurology, Volume: 61, Issue: 4
2019
Therapeutic drug monitoring of tacrolimus and mycophenolic acid in outpatient renal transplant recipients using a volumetric dried blood spot sampling device.
British journal of clinical pharmacology, Volume: 84, Issue: 12
2018
Development and in vitro characterization of chitosan-coated polymeric nanoparticles for oral delivery and sustained release of the immunosuppressant drug mycophenolate mofetil.
Drug development and industrial pharmacy, Volume: 45, Issue: 1
2019
Mycophenolic acid concentrations in peripheral blood mononuclear cells are associated with the incidence of rejection in renal transplant recipients.
British journal of clinical pharmacology, Volume: 84, Issue: 10
2018
Optimization and application of an HPLC method for quantification of inosine-5'-monophosphate dehydrogenase activity as a pharmacodynamic biomarker of mycophenolic acid in Chinese renal transplant patients.
Clinica chimica acta; international journal of clinical chemistry, Volume: 485
2018
Immunosuppressive treatment for proliferative lupus nephritis.
The Cochrane database of systematic reviews, 06-29, Volume: 6
2018
Reduced Tacrolimus Trough Level Is Reflected by Estimated Glomerular Filtration Rate (eGFR) Changes in Stable Renal Transplantation Recipients: Results of the OPTIMUM Phase 3 Randomized Controlled Study.
Annals of transplantation, Jun-12, Volume: 23
2018
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Interactions (110)

ArticleYear
A Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Tacrolimus and Corticosteroids in Combination With or Without Mycophenolate Mofetil in Liver Transplantation Recipients Infected With Hepatitis B Virus.
Transplantation proceedings, Volume: 55, Issue: 2
2023
Drug-Drug Interactions Between Mycophenolic Acid and Proton Pump Inhibitors: A Systematic Review and Meta-Analysis.
Therapeutic drug monitoring, 06-01, Volume: 44, Issue: 3
2022
Low-dose antithymocyte globulin plus low-dose posttransplant cyclophosphamide combined with cyclosporine and mycophenolate mofetil for prevention of graft-versus-host disease after HLA-matched unrelated donor peripheral blood stem cell transplantation.
Bone marrow transplantation, Volume: 56, Issue: 10
2021
Low-Dose Corticosteroid Combined With Mycophenolate Mofetil for IgA Nephropathy With Stage 3 or 4 CKD: A Retrospective Cohort Study.
Clinical therapeutics, Volume: 43, Issue: 5
2021
Comparison of a Point-of-Care Testing with Enzyme-Multiplied Immunoassay Technique and Liquid Chromatography Combined With Tandem Mass Spectrometry Methods for Therapeutic Drug Monitoring of Mycophenolic Acid: A Preliminary Study.
Therapeutic drug monitoring, 10-01, Volume: 43, Issue: 5
2021
Evaluation of the inhibitory effect of tacrolimus combined with mycophenolate mofetil on mesangial cell proliferation based on the cell cycle.
International journal of molecular medicine, Volume: 46, Issue: 4
2020
Mycophenolic Acid and Its Pharmacokinetic Drug-Drug Interactions in Humans: Review of the Evidence and Clinical Implications.
Journal of clinical pharmacology, Volume: 60, Issue: 3
2020
Model based development of tacrolimus dosing algorithm considering CYP3A5 genotypes and mycophenolate mofetil drug interaction in stable kidney transplant recipients.
Scientific reports, 08-13, Volume: 9, Issue: 1
2019
Population Pharmacokinetics of Mycophenolic Acid Co-Administered with Tacrolimus in Corticosteroid-Free Adult Kidney Transplant Patients.
Clinical pharmacokinetics, Volume: 58, Issue: 11
2019
[Clinical effect of tacrolimus combined with glucocorticoid in the treatment of IgA nephropathy in children].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, Volume: 21, Issue: 3
2019
Systematic external evaluation of published population pharmacokinetic models of mycophenolate mofetil in adult kidney transplant recipients co-administered with tacrolimus.
British journal of clinical pharmacology, Volume: 85, Issue: 4
2019
Comparison of Sirolimus Combined With Tacrolimus and Mycophenolate Mofetil Combined With Tacrolimus in Kidney Transplantation Recipients: A Meta-Analysis.
Transplantation proceedings, Volume: 50, Issue: 10
2018
Population Pharmacokinetic Analysis of Immediate-Release Oral Tacrolimus Co-administered with Mycophenolate Mofetil in Corticosteroid-Free Adult Kidney Transplant Recipients.
European journal of drug metabolism and pharmacokinetics, Volume: 44, Issue: 3
2019
Mycophenolate Mofetil Alone and in Combination with Tacrolimus Inhibits the Proliferation of HT-29 Human Colonic Adenocarcinoma Cell Line and Might Interfere with Colonic Tumorigenesis.
Anticancer research, Volume: 38, Issue: 6
2018
Failure of remission induction by glucocorticoids alone or in combination with immunosuppressive agents in IgG4-related disease: a prospective study of 215 patients.
Arthritis research & therapy, 04-10, Volume: 20, Issue: 1
2018
Kidney Transplant in a Human Immunodeficiency Virus-Positive Patient: Case Report of Drug Interactions.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 21, Issue: 4
2023
De novo low-dose sirolimus versus mycophenolate mofetil in combination with extended-release tacrolimus in kidney transplant recipients: a multicentre, open-label, randomized, controlled, non-inferiority trial.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Aug-01, Volume: 32, Issue: 8
2017
Managing Drug-Drug Interaction Between Ombitasvir, Paritaprevir/Ritonavir, Dasabuvir, and Mycophenolate Mofetil.
Therapeutic drug monitoring, Volume: 39, Issue: 4
2017
A Retrospective Study on Mycophenolic Acid Drug Interactions: Effect of Prednisone, Sirolimus, and Tacrolimus With MPA.
Therapeutic drug monitoring, Volume: 39, Issue: 3
2017
Low-dose mycophenolate mofetil in tablet form or capsule form combined with tacrolimus in the early period after kidney transplantation: a prospective randomized trial
.
Clinical nephrology, Volume: 86 (2016), Issue: 12
2016
A Prospective Randomized, Comparative Trial of High-Dose Mizoribine Versus Mycophenolate Mofetil in Combination With Tacrolimus and Basiliximab for Living Donor Renal Transplant: A Multicenter Trial.
Experimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation, Volume: 14, Issue: 5
2016
Mycophenolate mofetil combined with systemic corticosteroids prevents progression to chronic recurrent inflammation and development of 'sunset glow fundus' in initial-onset acute uveitis associated with Vogt-Koyanagi-Harada disease.
Acta ophthalmologica, Volume: 95, Issue: 1
2017
Pharmacokinetic Assessment of Drug-Drug Interactions of Isavuconazole With the Immunosuppressants Cyclosporine, Mycophenolic Acid, Prednisolone, Sirolimus, and Tacrolimus in Healthy Adults.
Clinical pharmacology in drug development, Volume: 6, Issue: 1
2017
[Comparison of therapeutic effects of prednisone combined with mycophenolate mofetil versus cyclosporin A in children with steroid-resistant nephrotic syndrome].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, Volume: 18, Issue: 2
2016
The Use of Methotrexate, Alone or in Combination With Other Therapies, for the Treatment of Palmoplantar Psoriasis.
Journal of drugs in dermatology : JDD, Volume: 14, Issue: 8
2015
Enteric-coated mycophenolate sodium given in combination with tacrolimus has a lower incidence of serious infections in Asian renal-transplant recipients compared with mycophenolate mofetil.
International journal of clinical practice. Supplement, Issue: 183
2015
mTOR inhibitor versus mycophenolic acid as the primary immunosuppression regime combined with calcineurin inhibitor for kidney transplant recipients: a meta-analysis.
BMC nephrology, Jul-01, Volume: 16
2015
Therapeutic effect of double-filtration plasmapheresis combined with methylprednisolone to treat diffuse proliferative lupus nephritis.
Journal of clinical apheresis, Volume: 31, Issue: 4
2016
Modeling approach for multiple transporters-mediated drug-drug interactions in sandwich-cultured human hepatocytes: effect of cyclosporin A on hepatic disposition of mycophenolic acid phenyl-glucuronide.
Drug metabolism and pharmacokinetics, Volume: 30, Issue: 2
2015
Safety of reduced dose of mycophenolate mofetil combined with tacrolimus in living-donor liver transplantation.
Clinical and molecular hepatology, Volume: 20, Issue: 3
2014
Anti-SLA/LP alone or in combination with anti-Ro52 and fine specificity of anti-Ro52 antibodies in patients with autoimmune hepatitis.
Liver international : official journal of the International Association for the Study of the Liver, Volume: 35, Issue: 2
2015
Effects of ASKP1240 combined with tacrolimus or mycophenolate mofetil on renal allograft survival in Cynomolgus monkeys.
Transplantation, Aug-15, Volume: 98, Issue: 3
2014
Everolimus in combination with mycophenolate mofetil as pre- and post-transplantation immunosuppression after nonmyeloablative hematopoietic stem cell transplantation in canine littermates.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 20, Issue: 9
2014
Cyclosporine A and tacrolimus combined with enteric-coated mycophenolate sodium influence the plasma mycophenolic acid concentration - a randomised controlled trial in Chinese live related donor kidney transplant recipients.
International journal of clinical practice. Supplement, Issue: 181
2014
High prevalence of potential drug interactions affecting mycophenolic acid pharmacokinetics in nonmyeloablative hematopoietic stem cell transplant recipients.
International journal of clinical pharmacology and therapeutics, Volume: 51, Issue: 9
2013
Alefacept combined with tacrolimus, mycophenolate mofetil and steroids in de novo kidney transplantation: a randomized controlled trial.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, Volume: 13, Issue: 7
2013
Enteric-coated mycophenolate sodium in combination with full dose or reduced dose cyclosporine, basiliximab and corticosteroids in Australian de novo kidney transplant patients.
Nephrology (Carlton, Vic.), Volume: 18, Issue: 1
2013
The outcomes of mycophenolate mofetil therapy combined with systemic corticosteroids in acute uveitis associated with Vogt-Koyanagi-Harada disease.
Acta ophthalmologica, Volume: 90, Issue: 8
2012
Effects of mycophenolic acid alone and in combination with its metabolite mycophenolic acid glucuronide on rat embryos in vitro.
Archives of toxicology, Volume: 87, Issue: 2
2013
Everolimus in combination with cyclosporin a as pre- and posttransplantation immunosuppressive therapy in nonmyeloablative allogeneic hematopoietic stem cell transplantation.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 18, Issue: 7
2012
A Phase I study of gemtuzumab ozogamicin (GO) in combination with busulfan and cyclophosphamide (Bu/Cy) and allogeneic stem cell transplantation in children with poor-risk CD33+ AML: a new targeted immunochemotherapy myeloablative conditioning (MAC) regim
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 18, Issue: 2
2012
[Drug interactions and immunosuppression in organ transplant recipients].
Tidsskrift for den Norske laegeforening : tidsskrift for praktisk medicin, ny raekke, Oct-18, Volume: 131, Issue: 20
2011
Tolerance of enteric-coated mycophenolate sodium in combination with calcineurin inhibitor in kidney transplant recipients: Polish experience.
Transplantation proceedings, Volume: 43, Issue: 8
2011
Efficacy and safety of enteric-coated mycophenolate sodium in combination with two glucocorticoid regimens for the treatment of active lupus nephritis.
Lupus, Volume: 20, Issue: 14
2011
FTY720 combined with tacrolimus in de novo renal transplantation: 1-year, multicenter, open-label randomized study.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Volume: 26, Issue: 11
2011
Imatinib combined with myeloablative allogeneic hematopoietic stem cell transplantation for advanced phases of chronic myeloid leukemia.
Leukemia research, Volume: 35, Issue: 10
2011
Effects of mycophenolate mofetil combined with corticosteroids for induction therapy of microscopic polyangiitis.
American journal of nephrology, Volume: 33, Issue: 2
2011
Pharmacokinetics of sotrastaurin combined with tacrolimus or mycophenolic acid in de novo kidney transplant recipients.
Transplantation, Feb-15, Volume: 91, Issue: 3
2011
Population pharmacokinetic analysis of mycophenolic acid coadministered with either tasocitinib (CP-690,550) or tacrolimus in adult renal allograft recipients.
Therapeutic drug monitoring, Volume: 32, Issue: 6
2010
In vitro and in vivo immunomodulatory effects of cobalt protoporphyrin administered in combination with immunosuppressive drugs.
Transplant immunology, Volume: 24, Issue: 1
2010
[Prophylactic effect of CsA, MTX, MMF combined with ATG on GVHD in patients underwent unrelated peripheral blood hematopoietic stem cell transplantation].
Zhongguo shi yan xue ye xue za zhi, Volume: 18, Issue: 2
2010
Basiliximab versus daclizumab combined with triple immunosuppression in deceased donor renal transplantation: a prospective, randomized study.
Transplantation, Apr-27, Volume: 89, Issue: 8
2010
Human mesenchymal stem cells and immunosuppressive drug interactions in allogeneic responses: an in vitro study using human cells.
Transplantation proceedings, Volume: 41, Issue: 8
2009
Long-term renal function in liver transplant recipients and impact of immunosuppressive regimens (calcineurin inhibitors alone or in combination with mycophenolate mofetil): the TRY study.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 15, Issue: 9
2009
Drug interactions in transplant patients: what everyone should know.
Current opinion in nephrology and hypertension, Volume: 18, Issue: 5
2009
A comparison of a standard-dose prednisone regimen and mycophenolate mofetil combined with a lower prednisone dose in Chinese adults with idiopathic nephrotic syndrome who were carriers of hepatitis B surface antigen: a prospective cohort study.
Clinical therapeutics, Volume: 31, Issue: 4
2009
Mycophenolate mofetil combined with tacrolimus and minidose methotrexate after unrelated donor bone marrow transplantation with reduced-intensity conditioning.
International journal of hematology, Volume: 89, Issue: 4
2009
Tacrolimus pharmacokinetic drug interactions: effect of prednisone, mycophenolic acid or sirolimus.
Fundamental & clinical pharmacology, Volume: 23, Issue: 1
2009
Could mycophenolate mofetil combined with benazapril delay tubulointerstitial fibrosis in 5/6 nephrectomized rats?
Chinese medical journal, Jan-20, Volume: 122, Issue: 2
2009
Pharmacokinetics, efficacy, and safety of mycophenolate mofetil in combination with standard-dose or reduced-dose tacrolimus in liver transplant recipients.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 15, Issue: 2
2009
Unified QSAR approach to antimicrobials. 4. Multi-target QSAR modeling and comparative multi-distance study of the giant components of antiviral drug-drug complex networks.
Bioorganic & medicinal chemistry, Jan-15, Volume: 17, Issue: 2
2009
Heart transplantation under cyclosporine or tacrolimus combined with mycophenolate mofetil or everolimus.
Transplantation proceedings, Volume: 40, Issue: 8
2008
A prospective multicentre study of mycophenolate mofetil combined with prednisolone as induction therapy in 213 patients with active lupus nephritis.
Lupus, Volume: 17, Issue: 7
2008
[Daclizumab in combination with mycophenolate mofetil and a late introduction of Tacrolimus at low doses, as a therapeutic approach in the elderly renal transplant donor-recipients pairs in kidney transplant].
Nefrologia : publicacion oficial de la Sociedad Espanola Nefrologia, Volume: 28, Issue: 3
2008
Efficacy and safety of universal valganciclovir prophylaxis combined with a tacrolimus/mycophenolate-based regimen in kidney transplantation.
Swiss medical weekly, Dec-01, Volume: 137, Issue: 47-48
2007
Pharmacokinetics, safety, and efficacy of mycophenolate mofetil in combination with sirolimus or ciclosporin in renal transplant patients.
British journal of clinical pharmacology, Volume: 64, Issue: 6
2007
Rescue therapy by immunoadsorption in combination with tacrolimus and mycophenolate mofetil for C4d-positive acute humoral renal allograft rejection.
Transplantation proceedings, Volume: 38, Issue: 10
2006
Mycophenolate mofetil in combination with reduction of calcineurin inhibitors for chronic renal dysfunction after liver transplantation.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 12, Issue: 12
2006
Tolerability of enteric-coated mycophenolate sodium to 1 year in combination with cyclosporine and corticosteroids in renal transplant recipients.
Transplantation proceedings, Volume: 38, Issue: 9
2006
Sirolimus monotherapy versus sirolimus in combination with steroids and/or MMF for immunosuppression after liver transplantation.
Digestive diseases and sciences, Volume: 51, Issue: 10
2006
Efficacy of mycophenolic acid combined with KRP-203, a novel immunomodulator, in a rat heart transplantation model.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, Volume: 25, Issue: 3
2006
Tacrolimus combined with mycophenolate mofetil can effectively reverse C4d-positive steroid-resistant acute rejection in Chinese renal allograft recipients.
Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, Volume: 21, Issue: 2
2006
Clinical pharmacokinetics of tacrolimus after the first oral administration in combination with mycophenolate mofetil and prednisone in Chinese renal transplant recipients.
Transplantation proceedings, Volume: 37, Issue: 10
2005
Best single time points to predict the area-under-the-curve in long-term heart transplant patients taking mycophenolate mofetil in combination with cyclosporine or tacrolimus.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, Volume: 24, Issue: 10
2005
A prospective, randomized trial of tacrolimus in combination with sirolimus or mycophenolate mofetil in kidney transplantation: results at 1 year.
Transplantation, Aug-15, Volume: 80, Issue: 3
2005
Drug interaction between mycophenolate mofetil and rifampin: possible induction of uridine diphosphate-glucuronosyltransferase.
Clinical pharmacology and therapeutics, Volume: 78, Issue: 1
2005
A phase I/II study of mycophenolate mofetil in combination with cyclosporine for prophylaxis of acute graft-versus-host disease after myeloablative conditioning and allogeneic hematopoietic cell transplantation.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume: 11, Issue: 7
2005
UDP-glucuronosyltransferases and clinical drug-drug interactions.
Pharmacology & therapeutics, Volume: 106, Issue: 1
2005
A prospective, randomized-controlled pilot study of ursodeoxycholic acid combined with mycophenolate mofetil in the treatment of primary sclerosing cholangitis.
Alimentary pharmacology & therapeutics, Nov-01, Volume: 20, Issue: 9
2004
C4d-positive acute humoral renal allograft rejection: rescue therapy by immunoadsorption in combination with tacrolimus and mycophenolate mofetil.
Transplantation proceedings, Volume: 36, Issue: 7
2004
Low-dose sirolimus in combination with mycophenolate mofetil improves kidney graft function late after renal transplantation and suggests pharmacokinetic interaction of both immunosuppressive drugs.
Kidney & blood pressure research, Volume: 27, Issue: 3
2004
A randomized long-term trial of tacrolimus and sirolimus versus tacrolimus and mycophenolate mofetil versus cyclosporine (NEORAL) and sirolimus in renal transplantation. I. Drug interactions and rejection at one year.
Transplantation, Jan-27, Volume: 77, Issue: 2
2004
The use of daclizumab as induction therapy in combination with tacrolimus and mycophenolate mofetil in recipients with previous transplants.
Clinical transplantation, Volume: 17, Issue: 5
2003
Assessment of immunosuppressive drug interactions: inhibition of lymphocyte function in peripheral human blood.
Journal of immunological methods, Volume: 283, Issue: 1-2
2003
Twelve-month evaluation of the clinical pharmacokinetics of total and free mycophenolic acid and its glucuronide metabolites in renal allograft recipients on low dose tacrolimus in combination with mycophenolate mofetil.
Therapeutic drug monitoring, Volume: 25, Issue: 5
2003
Long-term changes in mycophenolic acid exposure in combination with tacrolimus and corticosteroids are dose dependent and not reflected by trough plasma concentration: a prospective study in 100 de novo renal allograft recipients.
Journal of clinical pharmacology, Volume: 43, Issue: 8
2003
Tacrolimus dose requirement in renal transplant recipients is significantly higher when used in combination with corticosteroids.
British journal of clinical pharmacology, Volume: 56, Issue: 3
2003
Randomized trial of tacrolimus in combination with sirolimus or mycophenolate mofetil in kidney transplantation: results at 6 months.
Transplantation, Apr-27, Volume: 75, Issue: 8
2003
Cyclosporine A versus tacrolimus in combination with mycophenolate mofetil and steroids as primary immunosuppression after lung transplantation: one-year results of a 2-center prospective randomized trial.
The Journal of thoracic and cardiovascular surgery, Volume: 125, Issue: 4
2003
Long-term mycophenolate mofetil monotherapy in combination with calcineurin inhibitors for chronic renal dysfunction after liver transplantation.
Transplantation, Jan-27, Volume: 75, Issue: 2
2003
Efficacy of mycophenolate sodium as monotherapy and in combination with FTY720 in a DA-to-Lewis-rat heart-transplantation model.
Transplantation, Nov-27, Volume: 74, Issue: 10
2002
Mycophenolate mofetil in combination with recombinant interferon alfa-2a in interferon-nonresponder patients with chronic hepatitis C.
Journal of hepatology, Volume: 37, Issue: 6
2002
Dose proportional inhibition of HIV-1 replication by mycophenolic acid and synergistic inhibition in combination with abacavir, didanosine, and tenofovir.
Antiviral research, Volume: 55, Issue: 1
2002
Anti-interleukin-2 receptor therapy in combination with mycophenolate mofetil is associated with more severe hepatitis C recurrence after liver transplantation.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 7, Issue: 12
2001
Mycophenolate mofetil combined with a cyclooxygenase-2 inhibitor ameliorates murine lupus nephritis.
Kidney international, Volume: 60, Issue: 2
2001
Dose optimization of mycophenolate mofetil when administered with a low dose of tacrolimus in cadaveric renal transplant recipients.
Transplantation, Jul-15, Volume: 72, Issue: 1
2001
Pharmacokinetic basis for the efficient and safe use of low-dose mycophenolate mofetil in combination with tacrolimus in kidney transplantation.
Clinical chemistry, Volume: 47, Issue: 7
2001
A randomized double-blind comparative study of mycophenolate mofetil and azathioprine in combination with cyclosporine and corticosteroids in primary liver transplant recipients.
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society, Volume: 7, Issue: 5
2001
Safety and efficacy of tacrolimus in combination with mycophenolate mofetil (MMF) in cadaveric renal transplant recipients. FK506/MMF Dose-Ranging Kidney Transplant Study Group.
Transplantation, Mar-15, Volume: 69, Issue: 5
2000
Drug interaction between mycophenolate mofetil and tacrolimus detectable within therapeutic mycophenolic acid monitoring in renal transplant patients.
Therapeutic drug monitoring, Volume: 21, Issue: 5
1999
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]