Page last updated: 2024-11-04

fludarabine

Description Research Excerpts Clinical Trials Roles Classes Pathways Study Profile Bioassays Related Drugs Related Conditions Protein Interactions Research Growth

Cross-References

ID SourceID
PubMed CID657237
CHEMBL ID1568
CHEBI ID94701
SCHEMBL ID3510
MeSH IDM0082555

Synonyms (112)

Synonym
adenine, 9-beta-d-arabinofuranosyl-2-fluoro-
ccris 3382
fludarabinum [latin]
9-beta-d-arabinofuranosyl-2-fluoroadenine
fludarabina [spanish]
2-fluoro-9-beta-d-arabinofuranosyladenine
einecs 244-525-5
nsc 118218h
f-ara-a
nsc 118218
fludarabine [inn]
9h-purin-6-amine, 9-beta-d-arabinofuranosyl-2-fluoro-
hsdb 6964
fludarabine (fludara) ,
AB00383071-11
fludarabine
smr000058874
2-f-ara-a
faraa
2-fluoro ara-a
nsc118218
2-fluoroadenine arabinoside
nsc-118218
21679-14-1
2-fluoroadenine-9-beta-d-arabinofuranoside, dna synthesis and methylation inhibitor
2-f-araa
DB01073
MLS000028687 ,
9-beta-d-arabinofuranosyl-2-fluoro-9h-purin-6-amine
fludarabine (inn)
D07966
fluradosa (tn)
CHEMBL1568
2-fluoro-ara-a
fluradosa
2-fluorovidarabine
(2r,3s,4s,5r)-2-(6-amino-2-fluoropurin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol
HMS3259F09
EN300-52641
tox21_113419
tox21_112795
dtxsid4039657 ,
dtxcid2019657
cas-21679-14-1
(2r,3s,4s,5r)-2-(6-amino-2-fluoro-9h-purin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol
HMS2232K13
unii-p2k93u8740
p2k93u8740 ,
fludarabinum
fludarabina
BCP9000693
(2r,3s,4s,5r)-2-(6-amino-2-fluoro-9h-purin-9-yl)-5-(hydroxymethyl)tetrahydrofuran-3,4-diol
BCPP000227
NCGC00181336-03
2-fluoroadenine-9-beta-d-arabinofuranoside
AM20090199
2-fluoro-9-.beta.-d-arabinofuranosyladenine
fludarabine [mi]
fludarabine [hsdb]
9-.beta.-d-arabinofuranosyl-2-fluoroadenine
fludarabine [who-dd]
fludarabine phosphate impurity, 2-fluoro-ara-adenine- [usp impurity]
9-.beta.-d-arabinofuranosyl-2-fluoro-9h-purin-6-amine
fludarabine phosphate impurity e [ep impurity]
fludarabine [vandf]
S1491
AKOS015916481
AKOS015854911
HG1007
9-|a-d-arabinofuranosyl-2-fluoro-9h-purin-6-amine
gtpl4802
HBUBKKRHXORPQB-FJFJXFQQSA-N
CCG-221147
HY-B0069
MLS006011578
NC00633
SCHEMBL3510
tox21_113419_1
NCGC00182047-03
9-?-d-arabinofuranosyl-2-fluoro-9h-purin-6-amine
DS-1271
2-fluoro-9-arabinoadenine
(2r,3s,4s,5r)-2-(6-amino-2-fluoro-purin-9-yl)-5-methylol-tetrahydrofuran-3,4-diol
bdbm68391
(2r,3s,4s,5r)-2-(6-azanyl-2-fluoranyl-purin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol
(2r,3s,4s,5r)-2-(6-amino-2-fluoro-9-purinyl)-5-(hydroxymethyl)oxolane-3,4-diol
cid_657237
9-(?-d-arabinofuranosyl)-2-fluoro-9h-purin-6-amine
AB00383071_13
mfcd00132942
fludarabine free base
C21550
2f-ara-a
EX-A833
sr-01000000270
SR-01000000270-2
CHEBI:94701
NCGC00021920-04
HMS3715F15
HMS3677N14
9-b-d-arabinofuranosyl-2-fluoroadenine
BCP01383
HMS3413N14
9-ss-d-arabinofuranosyl-2-fluoroadenine
f-ara-a;nsc 118218
BP-25389
2-fluoro-9-beta-arabinofuranosyladenine
fludarabinum (latin)
l01bb05
F0658
Z756440078
BP-55389

Research Excerpts

Toxicity

Fludarabine, cyclophosphamide, and rituximab (FCR) regimen is considered the treatment of choice for young fit patients with CLL. This combination is toxic for older patients. In search of less toxic alternatives, we hypothesized that combination of busulfan (Bu), fludarabsine (Flu), and clofarabines (Clo) would provide superior efficacy.

ExcerptReferenceRelevance
" In addition, case records from National Cancer Institute (NCI) Group C protocols were reviewed for fludarabine in chronic lymphocytic leukemia (CLL), and cladribine and pentostatin in hairy cell leukemia (HCL), as well as adverse drug reactions reported to the NCI from January 1980 through September 1993."( Neurotoxicity of purine analogs: a review.
Cheson, BD; Foss, FM; Sorensen, JM; Vena, DA, 1994
)
0.29
" In the FAMP group, a direct correlation was found between the LD50 values of both FAMP and Mitox and the number of synergistic interactions, while the Pearson correlation coefficient was not significant in the Pento group."( The in vitro cytotoxic effect of mitoxantrone in combination with fludarabine or pentostatin in B-cell chronic lymphocytic leukemia.
Brugiatelli, M; Callea, I; Console, G; Filangeri, M; Iacopino, P; Morabito, F; Musolino, C; Oliva, B; Sculli, G; Stelitano, C,
)
0.13
" As intravenous Campath-1H is almost invariably associated with reactions, sometimes of WHO grade 3-4, we adopted the subcutaneous route of administration, which proved to induce rare and mild adverse reactions but had comparable efficacy."( Safety and efficacy of subcutaneous Campath-1H for treating residual disease in patients with chronic lymphocytic leukemia responding to fludarabine.
Brando, B; Cafro, AM; Cairoli, R; Montillo, M; Morra, E; Oreste, P; Pungolino, E; Rossi, V; Tedeschi, A; Veronese, S, 2002
)
0.31
"Subcutaneous Campath-1H administered to CLL patients with residual BM disease after FAMP proved to be safe and effective."( Safety and efficacy of subcutaneous Campath-1H for treating residual disease in patients with chronic lymphocytic leukemia responding to fludarabine.
Brando, B; Cafro, AM; Cairoli, R; Montillo, M; Morra, E; Oreste, P; Pungolino, E; Rossi, V; Tedeschi, A; Veronese, S, 2002
)
0.31
" In summary, the combination of treosulfan and fludarabine is a safe and efficient conditioning regimen."( Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic hematopoietic stem cell transplantation.
Casper, J; Dölken, G; Freund, M; Hammer, U; Hartung, G; Kiefer, T; Kleine, HD; Knauf, W; Knopp, A; Steiner, B; Wegener, R; Wilhelm, S; Wolff, D, 2004
)
0.32
" The synergistic toxic effect of the two drugs led to a total elimination of the neoplastic population."( The conjugate Rituximab/saporin-S6 completely inhibits clonogenic growth of CD20-expressing cells and produces a synergistic toxic effect with Fludarabine.
Bolognesi, A; Farini, V; Lubelli, C; Polito, L; Ricci, F; Stirpe, F; Tazzari, PL; Zinzani, PL, 2004
)
0.32
" These results suggest that CD34 selection of reduced-intensity PBSC allografts may cause adverse effects upon specific antimicrobial immunity which can lead to fatal infections, particularly in high-risk patients."( Reduced-intensity conditioning followed by allografting of CD34-selected stem cells and < or =10(6)/kg T cells may have an adverse effect on transplant-related mortality.
Hartwig, UF; Herr, W; Huber, C; Kolbe, K; Kreiter, S; Meyer, RG; Schneider, PM; Ullmann, AJ; Wehler, T; Winkelmann, N, 2005
)
0.33
" Reduced-intensity allogeneic SCT may offer a less toxic approach to patients with AML."( Preliminary results of the safety of immunotherapy with gemtuzumab ozogamicin following reduced intensity allogeneic stem cell transplant in children with CD33+ acute myeloid leukemia.
Bhatia, M; Bradley, B; Cairo, MS; Cooney, E; Del Toro, G; Foley, S; Garvin, J; George, D; Harrison, L; Hawks, R; Militano, O; Roman, E; Satwani, P; Unal, E; van de Ven, C; Wolownik, K, 2005
)
0.33
"5 million adverse drug reaction (ADR) reports for 8620 drugs/biologics that are listed for 1191 Coding Symbols for Thesaurus of Adverse Reaction (COSTAR) terms of adverse effects."( Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
Benz, RD; Contrera, JF; Kruhlak, NL; Matthews, EJ; Weaver, JL, 2004
)
0.32
" The safe dose of fludarabine in this context remains controversial."( Clinical and imaging features of fludarabine neurotoxicity.
Brace, JR; Lee, MS; McKinney, AM; Santacruz, K, 2010
)
0.36
" This is a relatively safe drug for clinical use but may cause side effects, some of which may be life-threatening."( Severe pulmonary toxicity associated with fludarabine and possible contribution of rituximab.
Disel, U; Karakoc, E; Paydas, S; Yavuz, S, 2010
)
0.36
"Cytoreduction with 4 courses of R-FM was safe and highly efficient in patients with recurrent/refractory follicular lymphoma who had high tumor burden; however, better consolidation than FM is needed to further improve outcome."( Efficacy and safety of the combination of rituximab, fludarabine, and mitoxantrone for rituximab-naive, recurrent/refractory follicular non-Hodgkin lymphoma with high tumor burden: a multicenter phase 2 trial by the Groupe d'Etude des Lymphomes de l'Adult
Brice, P; Copin, MC; Fermé, C; Feugier, P; Lamy, T; Morschhauser, F; Mounier, N; Sebban, C; Solal-Celigny, P; Tilly, H, 2010
)
0.36
" In search of less toxic alternatives, we hypothesized that combination of busulfan (Bu), fludarabine (Flu) and clofarabine (Clo) would provide superior efficacy."( The synergistic cytotoxicity of clofarabine, fludarabine and busulfan in AML cells involves ATM pathway activation and chromatin remodeling.
Andersson, BS; Champlin, RE; Li, Y; Murray, D; Valdez, BC, 2011
)
0.37
" Only a few studies have evaluated the long-term adverse effects of purine analogs on the immune system or stem cell toxicity, and most of these are not very recent."( Purine analog toxicity in patients with hairy cell leukemia.
Tadmor, T, 2011
)
0.37
" Patients were evaluated for engraftment, adverse events, graft-versus-host disease, non-relapse mortality, relapse incidence, overall survival and disease-free survival."( Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial.
Baumgart, J; Beelen, DW; Blau, IW; Casper, J; Einsele, H; Finke, J; Freund, M; Giebel, S; Gramatzki, M; Holowiecki, J; Kienast, J; Larsson, K; Markiewicz, M; Mylius, HA; Pichlmeier, U; Repp, R; Ruutu, T; Schnitzler, M; Stelljes, M; Stuhler, G; Trenschel, R; Uharek, L; Volin, L; Zander, AR, 2011
)
0.37
" Non-hematologic adverse events of grade III-IV in severity included mainly infections and gastrointestinal symptoms (80% and 22% of the patients, respectively)."( Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial.
Baumgart, J; Beelen, DW; Blau, IW; Casper, J; Einsele, H; Finke, J; Freund, M; Giebel, S; Gramatzki, M; Holowiecki, J; Kienast, J; Larsson, K; Markiewicz, M; Mylius, HA; Pichlmeier, U; Repp, R; Ruutu, T; Schnitzler, M; Stelljes, M; Stuhler, G; Trenschel, R; Uharek, L; Volin, L; Zander, AR, 2011
)
0.37
" The most common adverse effects were hematologic, with 2 patients experiencing treatment-related myelodysplastic syndrome (MDS), evolving to acute myelogenous leukemia (AML) in 1 patient."( Safety and efficacy of combination therapy with fludarabine, mitoxantrone, and rituximab followed by yttrium-90 ibritumomab tiuxetan and maintenance rituximab as front-line therapy for patients with follicular or marginal zone lymphoma.
Bayer, R; Fung, HC; Gregory, SA; Karmali, R; Kassar, M; O'Brien, T; Shammo, JM; Venugopal, P, 2011
)
0.37
" Multivariate analysis (MVA) identified comorbidity score (HCT-CI) >2 and advanced disease as adverse factors with no independent impact of regimen."( Allo-SCT for AML and MDS with treosulfan compared with BU-based regimens: reduced toxicity vs reduced intensity.
Danylesko, I; Nagler, A; Shem-Tov, N; Shimoni, A; Volchek, Y; Yerushalmi, R, 2012
)
0.38
"Excessive adverse events were encountered in a Phase I/II study of cyclophosphamide (CY) dose deescalation in a fludarabine-based conditioning regimen for bone marrow transplantation from unrelated donors in patients with severe aplastic anemia."( Fludarabine-based conditioning for marrow transplantation from unrelated donors in severe aplastic anemia: early results of a cyclophosphamide dose deescalation study show life-threatening adverse events at predefined cyclophosphamide dose levels.
Adams, RH; Anderlini, P; Antin, JH; Arai, S; Carter, SL; Confer, DL; Deeg, HJ; Difronzo, NL; Eapen, M; Ewell, M; Gersten, ID; Horowitz, MM; Horwitz, M; Leifer, ES; McCarty, JM; Nakamura, R; Pulsipher, MA; Tolar, J, 2012
)
0.38
" This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT."( Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan.
Bernardo, ME; Bertaina, A; Caocci, G; Contoli, B; Giorgiani, G; La Nasa, G; Locatelli, F; Mastronuzzi, A; Pagliara, D; Pinto, RM; Piras, E; Vacca, A; Zecca, M, 2012
)
0.38
"Patients with class 3 thalassemia with high-risk features for adverse events after high-dose chemotherapy with hematopoietic stem cell transplantation (HSCT) are difficult to treat, tending to either suffer serious toxicity or fail to establish stable graft function."( Pretransplant immunosuppression followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control.
Andersson, BS; Anurathapan, U; Charoenkwan, P; Chuansumrit, A; Hongeng, S; Issaragrisil, S; Jetsrisuparb, A; Pakakasama, S; Rujkijyanont, P; Sirachainan, N; Sirireung, S; Songdej, D; Srisala, S; Sruamsiri, R; Ungkanont, A, 2013
)
0.39
" We conclude that FBM-A is an effective and safe conditioning regimen for adults up to age 69 with hematologic malignancies who have low-, intermediate-, or high-risk scores according to the DRI."( Reduced toxicity conditioning and allogeneic stem cell transplantation in adults using fludarabine, carmustine, melphalan, and antithymocyte globulin: outcomes depend on disease risk index but not age, comorbidity score, donor type, or human leukocyte ant
Adams, RH; Betcher, JA; Dueck, AC; Fauble, VD; Khera, N; Klein, JL; Leis, JF; Noel, P; Reeder, CB; Slack, JL; Sproat, LO, 2013
)
0.39
" Our data indicate that Flu (160 mg/m(2)) with targeted myeloablative Bu (90 mg·h/L) is less toxic than and equally effective as BuCy (Mel) in patients with similar indications for allo-HCT."( Fludarabine and exposure-targeted busulfan compares favorably with busulfan/cyclophosphamide-based regimens in pediatric hematopoietic cell transplantation: maintaining efficacy with less toxicity.
Bartelink, IH; Bierings, MB; Boelens, JJ; de Wildt, A; Gerhardt, CE; Lindemans, CA; van Maarseveen, EM; van Reij, EM; Versluys, B, 2014
)
0.4
" In an effort to minimize these adverse effects, we conducted bone marrow transplantation (BMT) from unrelated volunteer donors using a conditioning regimen without BU or TBI."( Fludarabine, cytarabine, granulocyte colony-stimulating factor and melphalan (FALG with L-PAM) as a reduced toxicity conditioning regimen in children with acute leukemia.
Kato, K; Matsumoto, K; Matsuyama, T; Yoshida, N, 2014
)
0.4
"This conditioning regimen of FLAG combined with L-PAM (which did not contain BU and TBI) was associated with good outcomes and minimal late adverse effects in children with acute leukemia who have undergone allogeneic BMT from unrelated volunteer donors."( Fludarabine, cytarabine, granulocyte colony-stimulating factor and melphalan (FALG with L-PAM) as a reduced toxicity conditioning regimen in children with acute leukemia.
Kato, K; Matsumoto, K; Matsuyama, T; Yoshida, N, 2014
)
0.4
"Modified Bu/Flu as a new RIC regimen is well tolerated and safe for patients who need allogeneic hematopoietic stem cell transplantation, especially in older patients and/or patients with severe comorbidities."( [The efficacy and safety of modified busulfan/fludarabine conditioning regimen in elderly or drug-intolerable patients with hematologic malignancies].
Chen, H; Fu, HX; Huang, XJ; Liu, DH; Liu, KY; Sun, YQ; Tang, FF; Wang, FR; Wang, Y; Xu, LP, 2013
)
0.39
" The FB3-ATG2 regimen is safe and efficient in both lymphoid and myeloid disorders."( Feasibility of the fludarabine busulfan 3 days and ATG 2 days reduced toxicity conditioning in 51 allogeneic hematopoietic stem cell transplantation: a single-center experience.
Chantepie, SP; Gac, AC; Reman, O, 2014
)
0.4
" There was a slightly increased incidence of adverse events with lumiliximab but these increases did not appear to lead to differences in eventual outcomes."( A randomized, open-label, multicentre, phase 2/3 study to evaluate the safety and efficacy of lumiliximab in combination with fludarabine, cyclophosphamide and rituximab versus fludarabine, cyclophosphamide and rituximab alone in subjects with relapsed ch
Awan, FT; Byrd, JC; Flinn, IW; Hellmann, A; Hillmen, P; Hughes, SG; Robak, T; Shannon, M; Trone, D, 2014
)
0.4
" Our RTMAC regimen would be less toxic and offers a high probability of survival for children with hematological malignancies."( Reduced-toxicity myeloablative conditioning consisting of 8-Gy total body irradiation, cyclophosphamide and fludarabine for pediatric hematological malignancies.
Hirabayashi, K; Koike, K; Kurata, T; Nakazawa, Y; Saito, S; Sakashita, K; Tanaka, M; Yanagisawa, R; Yoshikawa, K, 2014
)
0.4
" Moreover, it appears to be safe with a low NRM rate among high-risk patients."( The efficacy and safety of a new reduced-toxicity conditioning with 4 days of once-daily 100 mg/m(2) intravenous busulfan associated with fludarabine and antithymocyte globulins prior to allogeneic stem cell transplantation in patients with high-risk myel
Blaise, D; Calmels, B; Castagna, L; Chabannon, C; Crocchiolo, R; Devillier, R; El-Cheikh, J; Faucher, C; Furst, S; Granata, A; Harbi, S; Lemarie, C; Vey, N; Wanquet, A; Weiller, PJ, 2016
)
0.43
" The therapeutic efficacy of patients in 2 groups was analyzed according to the peripheral hemogram indexes, symptom and sign disappeared time as well as adverse reaction incidence."( [Clinical Efficacy and Safety of Rituximab Combined with Fludarabine and Cyclophosphamide for Treatment of Chronic Lymphocytic Leukemia].
Han, HJ; Lu, YW; Xia, RX, 2016
)
0.43
"application of rituximab combined with fludarabine and cyclophosphamide in the treatment of CLL shows the higher curative effect, can effectively improve the symptoms and reduce the incidence of adverse reactions."( [Clinical Efficacy and Safety of Rituximab Combined with Fludarabine and Cyclophosphamide for Treatment of Chronic Lymphocytic Leukemia].
Han, HJ; Lu, YW; Xia, RX, 2016
)
0.43
" In the safety analysis, the proportion of patients reporting adverse events was similar between the subcutaneous and intravenous groups (all grades: 82 [96%] of 85 patients and 81 [91%] of 89 patients; serious adverse events: 25 [29%] and 29 [33%] patients; grade ≥3: 59 [69%] and 63 [71%] patients, respectively)."( Pharmacokinetics, safety, and efficacy of subcutaneous versus intravenous rituximab plus chemotherapy as treatment for chronic lymphocytic leukaemia (SAWYER): a phase 1b, open-label, randomised controlled non-inferiority trial.
Assouline, S; Badoux, X; Berthillon, N; Brewster, M; Buccheri, V; Catalani, O; Delmer, A; Gaidano, G; Li, S; McIntyre, C; Sayyed, P; Trneny, M, 2016
)
0.43
" Odds ratio (OR) with 95% confidence interval (CI) were utilized to express the complete response, overall response and adverse events outcomes."( Efficacy and safety evaluation of fludarabine-based chemotherapy regimen for patients with non-Hodgkin lymphoma: A meta-analysis.
Chen, B; Gao, C; Ge, Z; Liu, R; Zhang, X, 2017
)
0.46
" Grade ≥3 adverse events occurred in 80."( Safety of obinutuzumab alone or combined with chemotherapy for previously untreated or relapsed/refractory chronic lymphocytic leukemia in the phase IIIb GREEN study.
Aktan, M; Bosch, F; Dartigeas, C; Ferra Coll, CM; Foà, R; Gresko, E; Kisro, J; Leblond, V; Merot, JL; Montillo, M; Raposo, J; Robson, S; Stilgenbauer, S, 2018
)
0.48
" The fludarabine, cyclophosphamide, and rituximab (FCR) regimen is considered the treatment of choice for young fit patients with CLL; however, this combination is toxic for older patients."( Low-dose fludarabine and cyclophosphamide combined with standard dose rituximab (LD-FCR) is an effective and safe regimen for elderly untreated patients with chronic lymphocytic leukemia: The Israeli CLL study group experience.
Aviv, A; Bairey, O; Braester, A; Fineman, R; Herishanu, Y; Levi, I; Polliack, A; Rahimi-Levene, N; Ruchlemer, R; Shvidel, L; Tadmor, T; Yuklea, M, 2019
)
0.51
" TBC combined with thiotepa is highly toxic to the skin with various cutaneous manifestations."( Skin toxicity following treosulfan-thiotepa-fludarabine-based conditioning regimen in non-malignant pediatric patients undergoing hematopoietic stem cell transplantation.
Altman Kohl, S; Even-Or, E; Molho-Pessach, V; Stepensky, P; Zaidman, I, 2020
)
0.56
" At the final analysis, no new safety signals were observed [Grade ≥ 3 adverse events (AEs): 1L 82·7%, R/R 84·5%; serious AEs: 1L 58·1%, R/R 62·5%]."( Safety and efficacy of obinutuzumab alone or with chemotherapy in previously untreated or relapsed/refractory chronic lymphocytic leukaemia patients: Final analysis of the Phase IIIb GREEN study.
Bosch, F; Böttcher, S; Foà, R; Ilhan, O; Kisro, J; Leblond, V; Mahé, B; Mikuskova, E; Osmanov, D; Perretti, T; Reda, G; Robinson, S; Stilgenbauer, S; Tausch, E; Trask, P; Turgut, M; Van Hoef, M; Wójtowicz, M, 2021
)
0.62
" Trends toward improved survival were seen in patients transplanted for myeloid disease using bone marrow as stem cell source who achieved a busulfan AUC > 4000 μmol*min/day with two-year relapse-free survival approaching 80% CONCLUSIONS: This conditioning regimen is safe and effective in patients with high-risk leukemias, particularly myeloid disease."( Reduced-toxicity conditioning regimen with busulfan, fludarabine, rATG, and 400 cGy TBI in pediatric patients undergoing hematopoietic stem cell transplant for high-risk hematologic malignancies.
Chaudhury, S; Duerst, RE; Jacobsohn, D; Kletzel, M; Kwon, S; Rossoff, J; Schneiderman, J; Tse, WT, 2021
)
0.62
" In this study, we aimed to investigate the side effect of Bu/Cy and Bu/Flu regimens on our patients who underwent allogeneic bone marrow transplantation."( Adverse Effects of Busulfan Plus Cyclophosphamide versus Busulfan Plus Fludarabine as Conditioning Regimens for Allogeneic Bone Marrow Transplantation.
Hajifathali, A; Mabani, M; Mehdizadeh, M; Parkhideh, S; Rezvani, H; Salari, S, 2021
)
0.62
"The therapeutic-related adverse effects of the two conditioning regimens in patients who underwent allogeneic bone marrow transplant were almost similar."( Adverse Effects of Busulfan Plus Cyclophosphamide versus Busulfan Plus Fludarabine as Conditioning Regimens for Allogeneic Bone Marrow Transplantation.
Hajifathali, A; Mabani, M; Mehdizadeh, M; Parkhideh, S; Rezvani, H; Salari, S, 2021
)
0.62
" Grade 3 adverse events included mucositis, diarrhea, and liver transaminitis (n = 3 each)."( Adding venetoclax to fludarabine/busulfan RIC transplant for high-risk MDS and AML is feasible, safe, and active.
Antin, JH; Brock, J; Cutler, CS; DeAngelo, DJ; Fell, G; Garcia, JS; Gooptu, M; Ho, VT; Karp, HQ; Kim, AS; Kim, HT; Koreth, J; Letai, A; Lindsley, RC; Loschi, F; Lucas, F; Mashaka, T; Murdock, HM; Nikiforow, S; Potter, D; Romee, R; Ryan, J; Shapiro, R; Soiffer, RJ; Stone, RM, 2021
)
0.62
"We prospectively studied clofarabine-fludarabine-busulfan (CloFluBu)-conditioning in allogeneic hematopoietic cell therapy (HCT) for lymphoid and myeloid malignancies and hypothesized that CloFluBu provides a less toxic alternative to conventional conditioning regimens, with adequate antileukemic activity."( Clofarabine-fludarabine-busulfan in HCT for pediatric leukemia: an effective, low toxicity, TBI-free conditioning regimen.
Bierings, M; Boelens, JJ; Bresters, D; de Koning, CCH; Kollen, WJ; Lankester, AC; Lindemans, CA; Nierkens, S; Versluijs, AB, 2022
)
0.72
" Her clinical course was complicated by high-grade pulmonary toxic effects 55 days after treatment that resulted in death."( Pulmonary Toxic Effects After Myeloablative Conditioning With Total Body Irradiation Delivered via Volumetric Modulated Arc Therapy With Fludarabine.
Abdul-Hay, M; Al-Homsi, AS; Byun, D; Galavis, P; Gerber, NK; Karp, JM; Modrek, AS; Teruel, J; Yuan, Y,
)
0.13
" The prediction of transplantation-related mortality (TRM) using the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score and an arbitrary upper age limit of 55 years for administering myeloablative conditioning (MAC) are common strategies to ensure a safe procedure."( Myeloablative Dose of Busulfan and Fludarabine Combined with In Vivo T Cell Depletion Is Safe and Effective Conditioning for Acute Myeloid Leukemia and Myelodysplastic Syndrome Patients.
Alshehri, H; Anteh, S; Avenoso, D; Bourlon, C; Bouziana, S; Dazzi, F; de Farias, M; Dragoi, OD; Gameil, A; Hannah, G; Kenyon, M; Krishnamurthy, P; Kulasekararaj, A; Leung, YT; Mehra, V; Pagliuca, A; Potter, V; Serpenti, F; Shah, MN; Slonim, LB; Wood, H, 2023
)
0.91

Pharmacokinetics

Fludarabine, carboplatin, topotecan regimen is a promising treatment based on potential pharmacodynamic interactions, which merits additional study in poor prognosis, acute leukemia patients.

ExcerptReferenceRelevance
" Concurrent with the phase I evaluation of 2-F-ara-AMP administered as a single intravenous (IV) bolus every 21 days to patients with advanced malignancy, plasma pharmacokinetic profiles of 2-F-ara-A were determined in 30 patients following the rapid infusion (2 to 5 minutes) of doses of 2-F-ara-AMP ranging from 80 to 260 mg/m2."( Pharmacokinetics of 2-F-ara-A (9-beta-D-arabinofuranosyl-2-fluoroadenine) in cancer patients during the phase I clinical investigation of fludarabine phosphate.
Grever, MR; Malspeis, L; Staubus, AE; Young, D, 1990
)
0.28
" Pharmacokinetic and pharmacodynamic studies were performed on specimens from 20 patients."( Pharmacokinetic and pharmacodynamic studies of fludarabine and cytosine arabinoside administered as loading boluses followed by continuous infusions after a phase I/II study in pediatric patients with relapsed leukemias. The Children's Cancer Group.
Avramis, VI; Kowck, R; Krailo, MD; Liu-Mares, W; Ramilo-Torno, LV; Reaman, GH; Sato, JK; Sharpe, A; Wiersma, S, 1998
)
0.3
"Serial plasma samples were collected for pharmacokinetic analysis on day 2 of the first cycle of therapy."( The pharmacokinetics and pharmacodynamics of fludarabine in rheumatoid arthritis.
Boumpas, DT; Davis, JC; Fessler, B; Knebel, W; Pucino, F; Sanders, WD; Yarboro, C,
)
0.13
"Fludarabine pharmacokinetics in patients with RA are characterized by an intermediate-length distribution phase (approximately 40 min), terminal half-life of 10."( The pharmacokinetics and pharmacodynamics of fludarabine in rheumatoid arthritis.
Boumpas, DT; Davis, JC; Fessler, B; Knebel, W; Pucino, F; Sanders, WD; Yarboro, C,
)
0.13
" Plasma and cellular pharmacokinetic measurements were conducted during the first 5 days."( Evaluation of the combination of nelarabine and fludarabine in leukemias: clinical response, pharmacokinetics, and pharmacodynamics in leukemia cells.
Ayres, M; Du, M; Gandhi, V; Hodge, JP; Keating, MJ; O'Brien, S; Plunkett, W; Ramakrishna, P; Rodriguez, CO; Rosner, GL; Weller, S, 2001
)
0.31
" Pharmacokinetic studies were done using 11 samples with the first and fourth doses of Bu."( Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes.
Andersson, BS; Brown, C; Chaudhry, A; Duggan, P; Glick, S; Gyonyor, E; Morris, D; Quinlan, D; Ruether, JD; Russell, JA; Stewart, D; Tran, HT, 2002
)
0.31
" Median busulfan clearance was 109 mL/min/m2 and median daily area-under-the-plasma-concentration-versus-time-curve was 4871 micromol-min, with negligible interdose variability in pharmacokinetic parameters."( Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS.
Andersson, BS; Champlin, RE; Couriel, D; de Lima, M; Giralt, S; Jones, R; Korbling, M; Madden, T; Pierre, B; Russell, JA; Shahjahan, M; Shpall, EJ; Thall, PF; Wang, X, 2004
)
0.32
"Fludarabine, carboplatin, topotecan regimen is a promising treatment based on potential pharmacodynamic interactions, which merits additional study in poor prognosis, acute leukemia patients."( A phase I and pharmacodynamic study of fludarabine, carboplatin, and topotecan in patients with relapsed, refractory, or high-risk acute leukemia.
Cooper, BW; Creger, RJ; Gerson, SL; Gosky, D; Hoppel, CL; Ingalls, ST; Koc, ON; Lazarus, HM; Meyerson, HJ; Nowell, GM; Pearson, G; Radivoyevitch, T; Tilby, MJ; Veal, GJ, 2004
)
0.32
" The pharmacokinetic (PK) properties of F-ara-A (9-beta-D-arabinosyl-2-fluoradenine) before and after application of busulfan were prospectively investigated in 16 patients with hematological malignancies."( F-ara-A pharmacokinetics during reduced-intensity conditioning therapy with fludarabine and busulfan.
Bergeman, T; Bonin, M; Bornhauser, M; Ehninger, G; Illmer, T; Leopold, T; Pursche, S; Schleyer, E, 2007
)
0.34
" pharmacokinetic data on 670 drugs representing, to our knowledge, the largest publicly available set of human clinical pharmacokinetic data."( Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
Lombardo, F; Obach, RS; Waters, NJ, 2008
)
0.35
" We now report full pharmacokinetic (PK) data, correlations of PK with clinical outcomes, and final response and progression-free survival (PFS)."( Clinical response and pharmacokinetics from a phase 1 study of an active dosing schedule of flavopiridol in relapsed chronic lymphocytic leukemia.
Blum, KA; Brooker-McEldowney, M; Byrd, JC; Dalton, JT; Farley, KL; Fischer, B; Grever, MR; Heerema, NA; Hurh, E; Jarjoura, D; Johnson, AJ; Lin, TS; Mitchell, SM; Moran, ME; Phelps, MA; Rozewski, DM; Schaaf, LJ; Wu, D, 2009
)
0.35
" Mean (+/-SD) elimination half-life did not differ significantly between IV and oral dosage groups (11."( Pharmacokinetics, bioavailability and effects on electrocardiographic parameters of oral fludarabine phosphate.
Greenblatt, DJ; Karyagina, EV; Lister-James, J; Lundberg, AS; Yin, W, 2010
)
0.36
" Forodesine has biologic activity in CLL; pharmacodynamic parameters suggest that an alternate dosing schedule and/or higher doses to achieve greater intracellular dGTP may be beneficial in this patient population."( Phase 2 and pharmacodynamic study of oral forodesine in patients with advanced, fludarabine-treated chronic lymphocytic leukemia.
Balakrishnan, K; Bantia, S; Bickel, S; Chen, Y; Gandhi, V; Kantarjian, H; Keating, MJ; Kilpatrick, JM; O'Brien, S; Ravandi, F; Tyler, BF; Verma, D, 2010
)
0.36
" The elimination half-life at steady state of rituximab in all patients was estimated to be 19."( Pharmacokinetics of rituximab in patients with CD20 positive B-cell malignancies.
Aarden, L; Baars, JW; Beijnen, JH; Huitema, AD; Tran, L, 2010
)
0.36
" Pharmacokinetic studies were fitted to a linear, 2-compartment model in which dose reduction led to incomplete saturation of CD52 binding sites and greater antibody clearance."( Impact of in vivo alemtuzumab dose before reduced intensity conditioning and HLA-identical sibling stem cell transplantation: pharmacokinetics, GVHD, and immune reconstitution.
Bloor, A; Chakraverty, R; Clark, A; Clark, R; Collin, M; Crawley, C; Fielding, A; Hale, G; Johnson, P; Kottaridis, P; Mackinnon, S; Milligan, D; Morris, E; Orti, G; Parker, A; Peggs, K; Pettengell, R; Pettitt, A; Roughton, M; Shen, J; Snowden, J; Thomson, K, 2010
)
0.36
" We retrospectively compared outcomes of a consecutive series of 51 AML patients treated with oral busulfan (1 mg/kg every 6 hours for 4 days) and cyclophosphamide (60 mg/kg IV × 2 days) - (Bu/Cy) with 100 consecutive AML patients treated with pharmacokinetic targeted IV busulfan (AUC < 6000 μM/L*min per day × 4 days) and fludarabine (40 mg/m2 × 4 days) - (t-IV Bu/Flu)."( Pharmacokinetic targeting of intravenous busulfan reduces conditioning regimen related toxicity following allogeneic hematopoietic cell transplantation for acute myelogenous leukemia.
Anasetti, C; Fernandez, HF; Field, T; Kharfan-Dabaja, MA; Kim, J; Nishihori, T; Perez, L; Perkins, J; Pidala, J, 2010
)
0.36
") busulfan can produce a more consistent pharmacokinetic profile than oral formulations can, but nonetheless, significant interpatient variability is evident."( Influence of GST gene polymorphisms on the clearance of intravenous busulfan in adult patients undergoing hematopoietic cell transplantation.
Bae, KS; Choi, Y; Han, SB; Hur, EH; Kim, DY; Kim, SD; Lee, JH; Lee, KH; Lim, HS; Lim, SN; Noh, GJ; Yun, SC, 2011
)
0.37
"Busulfan has a narrow therapeutic range, and in children, pharmacokinetic variability has been found to be high even after the use of intravenous (i."( Highly variable pharmacokinetics of once-daily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling.
Ahn, HS; Han, EJ; Jang, IJ; Jang, MK; Kang, HJ; Kim, H; Kim, NH; Lee, JW; Lee, SH; Park, KD; Shin, HY; Song, SH; Yu, KS; Yuk, YJ, 2012
)
0.38
" Marked differences were observed compared to pharmacokinetic parameters for non-Hodgkin lymphoma (NHL) obtained previously: in CLL, time-varying clearance at time zero (CL(2)) was faster, volumes of distribution (V(1) and V(2)) were larger, and rate of change (K(des)) from the targetmediated clearance pathway to catabolic elimination was lower than NHL."( Population pharmacokinetics of rituximab in patients with chronic lymphocytic leukemia.
Dmoszynska, A; Joshi, A; Li, J; Mangat, R; Robak, T; Valente, N; Visich, J; Wenger, M; Zhi, J, 2012
)
0.38
" The median half-life of rituximab during cycle 1 was 27 hours, compared to 199 hours during cycle 4 (P<0."( Rapid clearance of rituximab may contribute to the continued high incidence of autoimmune hematologic complications of chemoimmunotherapy for chronic lymphocytic leukemia.
Beum, PV; Lee, E; Lindorfer, MA; Marti, G; Mo, CC; Njuguna, N; Taylor, RP; Vire, B; Wiestner, A; Wilson, WH, 2013
)
0.39
" Plasma busulfan concentrations were determined by HPLC-UV and the pharmacokinetic parameters were calculated using the WinNonlin program."( Influence of fludarabine on the pharmacokinetics of oral busulfan during pretransplant conditioning for hematopoietic stem cell transplantation.
Colturato, VA; de Castro, FA; Lanchote, VL; Simões, BP; Voltarelli, JC, 2013
)
0.39
" A detailed in vitro structure-activity relationship of 1a in CLL compared to that of 22 synthetic analogues is described along with preliminary in vivo pharmacokinetic and metabolism studies on the most potent compounds."( Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
Castro, JE; Choi, MY; Molinski, TF; Stout, EP, 2014
)
0.4
" Pharmacokinetic treatment with targeted intravenous busulfan combined with fludarabine (BuFlu) was developed as a preparative regimen for acute leukemia and myelodysplasia."( Myeloablative intravenous pharmacokinetically targeted busulfan plus fludarabine as conditioning for allogeneic hematopoietic cell transplantation in patients with non-Hodgkin lymphoma.
Anasetti, C; Ayala, E; Figueroa, J; Kharfan-Dabaja, MA; Kim, J; Locke, F; Nishihori, T; Perkins, J; Riches, M; Yue, B, 2015
)
0.42
"Considerable interpatient variability exists in pharmacokinetic and fludarabine-based biomarkers, but these biomarkers are not associated with clinical outcomes in fludarabine/TBI-conditioned patients."( Association of fludarabine pharmacokinetic/dynamic biomarkers with donor chimerism in nonmyeloablative HCT recipients.
Bemer, MJ; Heimfeld, S; Mager, DE; McCune, JS; Sandmaier, BM; Storer, BE, 2015
)
0.42
" This regimen was associated with durable donor engraftment and relatively low rates of regimen related toxicity (RRT); future alemtuzumab pharmacokinetic studies may improve outcomes, by allowing targeted alemtuzumab clearance to reduce graft rejection and promote more rapid immune reconstitution."( Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection.
Abdel-Azim, H; Crooks, GM; Kapoor, N; Khazal, S; Kohn, DB; Mahadeo, KM; Shah, AJ; Zhao, Q, 2015
)
0.42
" Pharmacokinetic parameters were determined in six children who received intravenous treosulfan (dose range 12-24 g/m(2)) in combination with fludarabine prior to blood or marrow transplantation."( Development and Validation of a High Pressure Liquid Chromatography-UV Method for the Determination of Treosulfan and Its Epoxy Metabolites in Human Plasma and Its Application in Pharmacokinetic Studies.
Byrne, JA; Earl, JW; Fraser, CJ; Koyyalamudi, SR; Kuzhiumparambil, U; Nath, CE; O'Brien, TA; Shaw, PJ, 2016
)
0.43
" In part two of the study, we aimed to confirm the pharmacokinetic non-inferiority of subcutaneous rituximab, and investigate its safety and efficacy."( Pharmacokinetics, safety, and efficacy of subcutaneous versus intravenous rituximab plus chemotherapy as treatment for chronic lymphocytic leukaemia (SAWYER): a phase 1b, open-label, randomised controlled non-inferiority trial.
Assouline, S; Badoux, X; Berthillon, N; Brewster, M; Buccheri, V; Catalani, O; Delmer, A; Gaidano, G; Li, S; McIntyre, C; Sayyed, P; Trneny, M, 2016
)
0.43
" We did the primary analysis in patients in the intention-to-treat population with complete pharmacokinetic data (pharmacokinetic population)."( Pharmacokinetics, safety, and efficacy of subcutaneous versus intravenous rituximab plus chemotherapy as treatment for chronic lymphocytic leukaemia (SAWYER): a phase 1b, open-label, randomised controlled non-inferiority trial.
Assouline, S; Badoux, X; Berthillon, N; Brewster, M; Buccheri, V; Catalani, O; Delmer, A; Gaidano, G; Li, S; McIntyre, C; Sayyed, P; Trneny, M, 2016
)
0.43
"Between Aug 20, 2012, and June 17, 2013, we randomly assigned 176 patients to receive subcutaneous rituximab (n=88) or intravenous rituximab (n=88); 134 (76%) patients comprised the pharmacokinetic population."( Pharmacokinetics, safety, and efficacy of subcutaneous versus intravenous rituximab plus chemotherapy as treatment for chronic lymphocytic leukaemia (SAWYER): a phase 1b, open-label, randomised controlled non-inferiority trial.
Assouline, S; Badoux, X; Berthillon, N; Brewster, M; Buccheri, V; Catalani, O; Delmer, A; Gaidano, G; Li, S; McIntyre, C; Sayyed, P; Trneny, M, 2016
)
0.43
" Pharmacokinetic dosing of busulfan (Bu) is frequently done for myeloablative conditioning, but evidence for its use is limited in RIC transplants."( Fludarabine-Busulfan Reduced-Intensity Conditioning in Comparison with Fludarabine-Melphalan Is Associated with Increased Relapse Risk In Spite of Pharmacokinetic Dosing.
Al-Kali, A; Alkhateeb, HB; Damlaj, M; Gangat, N; Gastineau, DA; Hashmi, S; Hefazi, M; Hogan, WJ; Litzow, MR; Partain, DK; Patnaik, MM; Wolf, RC, 2016
)
0.43
" As no definitive pharmacokinetic (PK) basis for HCT dosing for the wide age and weight range in HCT is available, linear body surface area (BSA)-based dosing is still used."( Population Pharmacokinetics of Fludarabine in Children and Adults during Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation.
Boelens, JJ; Dorlo, TPC; Huitema, ADR; Kuball, J; Langenhorst, JB; Nierkens, S; van Kesteren, C; van Maarseveen, EM, 2019
)
0.51
" This multi-institutional prospective study compared pharmacokinetic (PK) parameters of Bu between BuFlu and BuCy."( Pharmacokinetics of intravenous busulfan as condition for hematopoietic stem cell transplantation: comparison between combinations with cyclophosphamide and fludarabine.
Hino, Y; Kato, J; Kikuchi, T; Koda, Y; Matsumoto, K; Mishina, T; Mori, T; Morita, K; Nagao, Y; Ohwada, C; Okamoto, S; Onizuka, M; Onoda, M; Sakaida, E; Shimizu, H; Shono, K; Takeda, Y; Yokota, A; Yokoyama, H, 2021
)
0.62
"Busulfan (Bu) is a common component of conditioning regimens before hematopoietic stem cell transplantation (HSCT) and is known for high interpatient pharmacokinetic (PK) variability."( Precision dosing of intravenous busulfan in pediatric hematopoietic stem cell transplantation: Results from a multicenter population pharmacokinetic study.
Ansari, M; Ben Hassine, K; Bittencourt, H; Bredius, RGM; Daali, Y; Kassir, N; Krajinovic, M; Lewis, V; Nath, CE; Nava, T; Shaw, PJ; Théoret, Y; Uppugunduri, CRS, 2021
)
0.62

Compound-Compound Interactions

The results of two multinational, randomized trials in CLL patients were reviewed. The individualized protocol of fludarabine combined with rituximab and amifostine showed the safety and effectiveness.

ExcerptReferenceRelevance
"The Children's Cancer Group (CCG) undertook a phase I study (CCG-0922) to determine a tolerable dose of idarubicin given with fludarabine and cytarabine in children with relapsed or refractory leukemia."( Phase I/II study of idarubicin given with continuous infusion fludarabine followed by continuous infusion cytarabine in children with acute leukemia: a report from the Children's Cancer Group.
Avramis, VI; Dinndorf, PA; Kelleher, JF; Krailo, MD; Liu-Mares, W; Mosher, RB; Reaman, GH; Sato, JK; Seibel, NL; Wiersma, S, 1997
)
0.3
"A dose of idarubicin 12 mg/m2/d for 3 days given in combination with fludarabine and cytarabine was tolerated."( Phase I/II study of idarubicin given with continuous infusion fludarabine followed by continuous infusion cytarabine in children with acute leukemia: a report from the Children's Cancer Group.
Avramis, VI; Dinndorf, PA; Kelleher, JF; Krailo, MD; Liu-Mares, W; Mosher, RB; Reaman, GH; Sato, JK; Seibel, NL; Wiersma, S, 1997
)
0.3
"Combinations of nucleoside analog drugs, such as F-araA and ara-C, combined with Topoisomerase II inhibitors, such as anthracyclines, are synergistic against human leukemic T-cells and induce apoptotic cell death."( Increased p21/WAF-1 and p53 protein levels following sequential three drug combination regimen of fludarabine, cytarabine and docetaxel induces apoptosis in human leukemia cells.
Avramis, VI; Cohen, LJ; Danenberg, P; Kwock, R; Mukherjee, SK; Nandy, P; Solorzano, MM,
)
0.13
" These results support that fludarabine in combination with cyclophosphamide and/or mitoxantrone can be highly effective in the treatment of B-CLL."( In vitro evaluation of fludarabine in combination with cyclophosphamide and/or mitoxantrone in B-cell chronic lymphocytic leukemia.
Bellosillo, B; Colomer, D; Gil, J; Montserrat, E; Pons, G; Villamor, N, 1999
)
0.3
" In conclusion, fludarabine combined with cyclophosphamide and dexamethasone is an effective therapy for recurrent indolent lymphoma."( The immunosuppression and potential for EBV reactivation of fludarabine combined with cyclophosphamide and dexamethasone in patients with lymphoproliferative disorders.
Arcaini, L; Astori, C; Baldanti, F; Bernasconi, C; Furione, M; Gerna, G; Lazzarino, M; Orlandi, E; Pagnucco, G; Paulli, M; Viglio, A, 1999
)
0.3
" Among these agents, cytarabine may be the best agent for the combination with fludarabine phosphate."( In vitro cytotoxic effects of fludarabine (2-F-ara-A) in combination with commonly used antileukemic agents by isobologram analysis.
Akutsu, M; Bai, L; Furukawa, Y; Ichikawa, A; Kano, Y; Kon, K; Suzuki, K; Tsunoda, S, 2000
)
0.31
" Because its toxicities are minimal and do not overlap with the toxicities of standard chemotherapy, it is an appealing agent to use in combination with chemotherapy."( Safety of fludarabine, mitoxantrone, and dexamethasone combined with rituximab in the treatment of stage IV indolent lymphoma.
Cabanillas, F; Dang, NH; Hagemeister, FB; Lee, MS; McAda, N; McLaughlin, P; Pate, O; Preti, AH; Rodriguez, MA; Romaguera, JE; Sarris, AH; Younes, A, 2000
)
0.31
" Two groups of infected mice were treated as follows: one group was treated by alternating the administration of Fludarabine and AZT (treatment A), while the other group received the same treatment plus GSH-loaded erythrocytes given with AZT (treatment A + L-RBC)."( New drug combinations for the treatment of murine AIDS and macrophage protection.
Brandi, G; Casabianca, A; Chiarantini, L; Fraternale, A; Magnani, M; Tonelli, A, 2001
)
0.31
"Fludarabine, 9-beta-D-arabinofuranosyl-2-fluoroadenine, is an adenine nucleoside analogue that has significant activity in hematological malignancies and has shown promising activity in combination with radiation in preclinical solid tumor models."( A phase I study of fludarabine combined with radiotherapy in patients with intermediate to locally advanced head and neck squamous cell carcinoma.
Ang, KK; Beauduin, M; Garden, AS; Grégoire, V; Hamoir, M; Hittelman, WN; Humblet, Y; Khuri, FR; Milas, L; Mitine, C; Rosier, JF; Scalliet, P, 2002
)
0.31
" The aim of study was to evaluate the activity and toxicity of FLU in combination with CY, the FLU-CY schedule, in patients with previously untreated B-CLL."( Fludarabine combined with cyclophosphamid is highly effective in the treatment of chronic lymphocytic leukemia.
Fricová, M; Guman, T; Kafková, A; Stecová, N; Tóthová, E, 2003
)
0.32
" Although fludarabine was administered, the platelet count did not decrease when combined with rituximab."( Successful treatment of lymphoma with fludarabine combined with rituximab after immune thrombocytopenia induced by fludarabine.
Asaka, M; Ehira, N; Fukushima, A; Imamura, M; Kanamori, H; Masauzi, N; Minami, H; Musashi, M; Obara, S; Ogura, N; Tanaka, J; Tsutsumi, Y; Yamato, H, 2005
)
0.33
" The aim of this study was to evaluate the cytotoxicity of SDX-101 combined with agents proven to be effective as first-line treatment of B-CLL: the purine nucleoside analogs, fludarabine (FA) and cladribine (2-CdA), and the monoclonal antibodies, anti-CD52 (alemtuzumab; ALT) and anti-CD20 (rituximab; RIT)."( Cytotoxic effect of R-etodolac (SDX-101) in combination with purine analogs or monoclonal antibodies on ex vivo B-cell chronic lymphocytic leukemia cells.
Cebula, B; Linke, A; Robak, P; Robak, T; Smolewski, P, 2006
)
0.33
" In this study, Cloretazine was evaluated both as a monotherapy and in combination with fludarabine in murine tumor and human tumor xenograft models."( Anti-tumor efficacy of Cloretazine (VNP40101M) alone and in combination with fludarabine in murine tumor and human xenograft tumor models.
King, I; Li, Z; Liu, L; Song, BL; Zheng, LM, 2007
)
0.34
"The current study was conducted to asses the safety profile and clinical activity of rituximab in combination with fludarabine and cyclophosphamide in patients with recurrent follicular lymphoma (FL)."( Rituximab in combination with fludarabine and cyclophosphamide in the treatment of patients with recurrent follicular lymphoma.
Brugiatelli, M; Federico, M; Liberati, M; Marcheselli, R; Merli, F; Orsucci, L; Pozzi, S; Sacchi, S; Tucci, A; Vallisa, D, 2007
)
0.34
" In conclusion, the individualized protocol of fludarabine combined with rituximab and amifostine showed the safety and effectiveness for treatment of aged patient with CLL."( [Curative efficacy of fludarabine combined with rituximab and amifostine on aged patient with B-chronic lymphocytic leukemia].
Li, SX; Liu, Y; Lu, XC; Zhu, HL, 2007
)
0.34
"This study was aimed to investigate the feasibility of low dose of fludarabine, cyclophosphamide combined with donor derived alloreactive NK cells as a new nonmyeloablative conditioning regimen in the haploidentical hematopoietic stem cell transplantation (haploidentical HSCT)."( [Application of low dose of fludarabine and cyclophosphamide combined with donor NK Cells as a non-myeloablative conditioning regimen for the haploidentical hematopoietic stem cell transplantation in mice].
An, XM; Cao, S; Qi, J; Ren, XB; Xin, N; Yu, JP, 2007
)
0.34
" Fourteen patients were treated with three cycles of rituximab (375 mg/m(2) weekly for 4 weeks) in combination with HDMP (1 gm/m(2) daily for 5 days)."( Rituximab in combination with high-dose methylprednisolone for the treatment of fludarabine refractory high-risk chronic lymphocytic leukemia.
Bole, J; Castro, JE; Kipps, TJ; Rassenti, L; Sandoval-Sus, JD, 2008
)
0.35
" Furthermore, local tumor irradiation combined with intratumoral dendritic cell administration significantly enhanced the therapeutic efficacy of tumor-reactive T cell adoptive transfer in this lymphodepleted liver tumor model."( Radiotherapy combined with intratumoral dendritic cell vaccination enhances the therapeutic efficacy of adoptive T-cell transfer.
Chang, AE; Davis, MA; Hoff, J; Li, M; Li, Q; Teitz-Tennenbaum, S; Wilder-Romans, K,
)
0.13
"The aim of study was to evaluate the clinical efficacy and toxicity of fludarabine combined with cytarabine (FA) regimen in the treatment of patients with refractory and/or relapsed acute myeloid leukemia (AML)."( [Clinical study on fludarabine combined with cytarabine regimen in the treatment of patients with refractory and relapsed acute myeloid leukemia].
Chen, YM; Dou, HJ; Hu, JP; Tang, Y; Yao, YY; Zhu, Q; Zou, LF, 2009
)
0.35
" To assess the efficacy and toxicity of oral fludarabine in combination with rituximab in patients with relapsed indolent B-cell non-Hodgkin lymphoma (B-NHL), we conducted a multicenter phase II study."( Phase II study of oral fludarabine in combination with rituximab for relapsed indolent B-cell non-Hodgkin lymphoma.
Ando, K; Hayashi, M; Hotta, T; Ishizawa, K; Itoh, K; Matsusako, M; Morishima, Y; Nakata, M; Nawano, S; Ogura, M; Taniwaki, M; Terauchi, T; Tobinai, K; Uchida, T; Watanabe, T; Yamamoto, J, 2009
)
0.35
"Preclinical data demonstrate enhanced antitumor effect when lumiliximab, an anti-CD23 monoclonal antibody, is combined with fludarabine or rituximab."( Phase 1/2 study of lumiliximab combined with fludarabine, cyclophosphamide, and rituximab in patients with relapsed or refractory chronic lymphocytic leukemia.
Byrd, JC; Castro, J; Flinn, IW; Harris, S; Heerema, N; Hughes, S; Kipps, TJ; Leigh, B; Lin, TS; Molina, A; O'Brien, S; Tangri, S; Wierda, W; Woodworth, J; Wynne, D, 2010
)
0.36
" To evaluate this effect in vivo, we performed a phase I study of chlorambucil combined with imatinib in relapsed CLL patients."( A phase I study of imatinib mesylate in combination with chlorambucil in previously treated chronic lymphocytic leukemia patients.
Aloyz, R; Amrein, L; Assouline, S; Caplan, S; Desjardins, P; Egorin, MJ; Hebb, J; Panasci, L; Rousseau, C, 2011
)
0.37
"To describe the clinical studies that led to the FDA approval of rituximab in combination with fludarabine and cyclophosphamide (FC) for the treatment of patients with chronic lymphocytic leukemia (CLL)."( U.S. Food and drug administration approval: rituximab in combination with fludarabine and cyclophosphamide for the treatment of patients with chronic lymphocytic leukemia.
Casak, SJ; Davis, G; Jarral, V; Keegan, P; Khandelwal, A; Lemery, SJ; Pazdur, R; Rothmann, MD; Shen, YL; Zhao, H, 2011
)
0.37
"The results of two multinational, randomized trials in CLL patients comparing rituximab combined with fludarabine and cyclophosphamide versus FC were reviewed."( U.S. Food and drug administration approval: rituximab in combination with fludarabine and cyclophosphamide for the treatment of patients with chronic lymphocytic leukemia.
Casak, SJ; Davis, G; Jarral, V; Keegan, P; Khandelwal, A; Lemery, SJ; Pazdur, R; Rothmann, MD; Shen, YL; Zhao, H, 2011
)
0.37
"On the basis of the demonstration of clinically meaningful prolongation of PFS, the FDA granted regular approval to rituximab in combination with FC for the treatment of patients with CLL."( U.S. Food and drug administration approval: rituximab in combination with fludarabine and cyclophosphamide for the treatment of patients with chronic lymphocytic leukemia.
Casak, SJ; Davis, G; Jarral, V; Keegan, P; Khandelwal, A; Lemery, SJ; Pazdur, R; Rothmann, MD; Shen, YL; Zhao, H, 2011
)
0.37
" The purpose of this phase II study was to determine the overall response rate, response duration and toxicity of cenersen administered in combination with FCR."( Phase II study of cenersen, an antisense inhibitor of p53, in combination with fludarabine, cyclophosphamide and rituximab for high-risk chronic lymphocytic leukemia.
Cook, H; Datto, M; Daugherty, FJ; Davis, PH; DeCastro, CM; Diehl, LF; Friedman, DR; Gockerman, JP; Lanasa, MC; Li, Z; Matta, KM; Moore, JO; Rehder, C; Rizzieri, D; Weinberg, JB, 2012
)
0.38
"The objective of this trial was to evaluate safety and efficacy of bendamustine combined with rituximab (BR) in patients with relapsed and/or refractory chronic lymphocytic leukemia (CLL)."( Bendamustine combined with rituximab in patients with relapsed and/or refractory chronic lymphocytic leukemia: a multicenter phase II trial of the German Chronic Lymphocytic Leukemia Study Group.
Bahlo, J; Böttcher, S; Bühler, A; Busch, R; Cramer, P; Döhner, H; Eckart, MJ; Eichhorst, BF; Elter, T; Fischer, K; Goede, V; Hallek, M; Kiehl, M; Kneba, M; Kranz, G; Schweighofer, CD; Staib, P; Stilgenbauer, S; Wendtner, CM; Winkler, D, 2011
)
0.37
" Bendamustine was administered at a dose of 70 mg/m(2) on days 1 and 2 combined with rituximab 375 mg/m(2) on day 0 of the first course and 500 mg/m(2) on day 1 during subsequent courses for up to six courses."( Bendamustine combined with rituximab in patients with relapsed and/or refractory chronic lymphocytic leukemia: a multicenter phase II trial of the German Chronic Lymphocytic Leukemia Study Group.
Bahlo, J; Böttcher, S; Bühler, A; Busch, R; Cramer, P; Döhner, H; Eckart, MJ; Eichhorst, BF; Elter, T; Fischer, K; Goede, V; Hallek, M; Kiehl, M; Kneba, M; Kranz, G; Schweighofer, CD; Staib, P; Stilgenbauer, S; Wendtner, CM; Winkler, D, 2011
)
0.37
" busulfan combined with fludarabine and analyzed the outcomes."( Highly variable pharmacokinetics of once-daily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling.
Ahn, HS; Han, EJ; Jang, IJ; Jang, MK; Kang, HJ; Kim, H; Kim, NH; Lee, JW; Lee, SH; Park, KD; Shin, HY; Song, SH; Yu, KS; Yuk, YJ, 2012
)
0.38
" Of 178 patients enrolled in the study, 165 were randomly assigned to receive 6 courses of oral fludarabine and cyclophosphamide (FC) in combination with rituximab (FCR; 375 mg/m(2) in cycle one, 500 mg/m(2) in all subsequent cycles) or alemtuzumab (FCCam; 30 mg subcutaneously injected on cycle days 1-3); each cycle was 28 days."( Excess mortality after treatment with fludarabine and cyclophosphamide in combination with alemtuzumab in previously untreated patients with chronic lymphocytic leukemia in a randomized phase 3 trial.
Aurran, T; Béné, MC; Casasnovas, O; Cazin, B; Chevret, S; Corront, B; Cymbalista, F; Delépine, R; Delmer, A; Feugier, P; Leblond, V; Lepretre, S; Letestu, R; Mahé, B; Maisonneuve, H; Royer, B; Tournilhac, O; Van Den Neste, E; Vaudaux, S, 2012
)
0.38
"Fludarabine is an adenine nucleoside analogue that has significant activity in hematological malignancies and has shown promising activity in combination with radiation in preclinical solid tumor models."( Fludarabine combined with radiotherapy in patients with locally advanced NSCLC lung carcinoma: a phase I study.
Christiansen, H; Griesinger, F; Lederer, K; Nitsche, M; Pradier, O; Schmidberger, H, 2012
)
0.38
" However, AUY922 in combination with fludarabine was significantly more effective at inducing apoptosis in cells in co-culture than either drug alone, an effect that was irrespective of ATM/TP53 dysfunction."( Heat shock protein-90 inhibitor, NVP-AUY922, is effective in combination with fludarabine against chronic lymphocytic leukemia cells cultured on CD40L-stromal layer and inhibits their activated/proliferative phenotype.
Best, OG; Mulligan, SP, 2012
)
0.38
"Cladribine (C) and fludarabine (F) combined with cyclophosphamide/mafosfamide in vivo, as well as ex vivo trigger apoptosis in CLL cells."( In vivo and ex vivo responses of CLL cells to purine analogs combined with alkylating agent.
Borowiak, A; Błoński, JZ; Cebula-Obrzut, B; Kiliańska, ZM; Kotkowska, A; Robak, T; Rogalińska, M; Smolewski, P; Wawrzyniak, E; Żołnierczyk, JD, 2013
)
0.39
" Thus, we examined in vivo the antitumor activity of galiximab used alone and in combination with chemotherapeutic agents in SCID mice bearing human lymphoma xenografts."( Galiximab (anti-CD80)-induced growth inhibition and prolongation of survival in vivo of B-NHL tumor xenografts and potentiation by the combination with fludarabine.
Berquist, L; Bonavida, B; Chu, P; Clanton, D; Hariharan, K; Ho, SN; Molina, A; Murphy, T; Vega, MI, 2013
)
0.39
" Together, our data suggest the potential for proteasome inhibitor based therapy in CLL and the rationale design of drug combination strategies based on CLL biology."( The investigational agent MLN2238 induces apoptosis and is cytotoxic to CLL cells in vitro, as a single agent and in combination with other drugs.
Advani, P; Akhtar, D; Chanan-Khan, A; Chitta, K; Colon-Otero, G; Foran, J; Khan, AN; Masood, A; Miller, KC; Paulus, A; Rivera, C; Roy, V, 2014
)
0.4
" In conclusion, we provide evidence that Notch is a therapeutic target in CLL cases with NOTCH1-activating mutations, supporting the use of Notch pathway inhibitors in combination with chemotherapy as a promising approach for the treatment of these high-risk CLL patients."( The γ-secretase inhibitor PF-03084014 combined with fludarabine antagonizes migration, invasion and angiogenesis in NOTCH1-mutated CLL cells.
Aymerich, M; Campo, E; Colomer, D; López-Guerra, M; López-Otín, C; Matas-Céspedes, A; Montraveta, A; Pérez-Galán, P; Roldán, J; Rosich, L; Roué, G; Villamor, N; Xargay-Torrent, S, 2015
)
0.42
" Early phase clinical studies with lumiliximab alone and in combination with fludarabine, cyclophosphamide and rituximab (FCR) established its potential efficacy and tolerability."( A randomized, open-label, multicentre, phase 2/3 study to evaluate the safety and efficacy of lumiliximab in combination with fludarabine, cyclophosphamide and rituximab versus fludarabine, cyclophosphamide and rituximab alone in subjects with relapsed ch
Awan, FT; Byrd, JC; Flinn, IW; Hellmann, A; Hillmen, P; Hughes, SG; Robak, T; Shannon, M; Trone, D, 2014
)
0.4
"This retrospective study compared efficacy and safety of fludarabine combined with intermediate-dose cytarabine (FA regimen) versus high-dose cytarabine (HiDAC regimen) as consolidation therapy in acute myeloid leukemia (AML) patients who achieved complete remission."( Retrospective comparison of fludarabine in combination with intermediate-dose cytarabine versus high-dose cytarabine as consolidation therapies for acute myeloid leukemia.
Chen, C; Chen, Y; Ding, Y; Fu, J; Liang, A; Lu, H; Wang, W; Wu, H; Zhang, W; Zou, S, 2014
)
0.4
"This study was aimed to investigate the morphological, immunophenotype, cytogenetic characteristics, clinical and therapy features in one elderly patient with chronic lymphocytic leukemia (CLL) combined with invasive aspergillose infection(IAI)."( [Elderly chronic lymphocytic leukemia combined with invasive aspergillosis infection in one case].
Li, BL; Li, J; Li, SX; Liu, Y; Wang, HT; Wang, TM; Zhai, B; Zhu, HL, 2015
)
0.42
" It is concluded that obatoclax can be safely administered to relapsed CLL patients in combination with FR and shows promising clinical activity."( Obatoclax in combination with fludarabine and rituximab is well-tolerated and shows promising clinical activity in relapsed chronic lymphocytic leukemia.
Brown, JR; Fisher, DC; Freedman, AS; Mikler, E; Neuberg, D; Reynolds, HM; Takebe, N; Tesar, B; Thompson, C; Werner, L; Yu, L, 2015
)
0.42
" Fifty-eight patients (median age, 67 years; range, 54 to 76) received radioimmunotherapy followed by fludarabine 150 mg/m(2) and busulfan 8 mg/kg combined with either 75 mg (n = 26) or 50 mg (n = 32) alemtuzumab."( Reduced-Intensity Conditioning Combined with (188)Rhenium Radioimmunotherapy before Allogeneic Hematopoietic Stem Cell Transplantation in Elderly Patients with Acute Myeloid Leukemia: The Role of In Vivo T Cell Depletion.
Bornhäuser, M; Ehninger, G; Kotzerke, J; Schetelig, J; Schneider, S; Strumpf, A; Wunderlich, G, 2015
)
0.42
" We performed a phase 2 study of alemtuzumab in combination with fludarabine in patients with relapsed disease."( A Phase 2 Trial of Fludarabine Combined With Subcutaneous Alemtuzumab for the Treatment of Relapsed/Refractory B-Cell Chronic Lymphocytic Leukemia.
Brown, JR; Cohen, JB; Flowers, CR; Jaye, DL; Katzen, HI; Lakhanpal, S; Leis, JF; Rosenthal, H; Sinha, R; Stock, W; Waller, EK, 2015
)
0.42
" These patients may benefit from rituximab combined with intensive chemotherapy."( [Curative effect analysis of rituximab combined with intensive chemotherapy for follicular lymphoma patients with bone marrow involvement].
Li, H; Li, Z; Liu, H; Liu, W; Lü, R; Qiu, L; Xiong, W; Yi, S; Zou, D, 2015
)
0.42
"To explore the clinical efficacy and safety of rituximab combined with fludarabine and cyclophosphamide for the treatment of the chronic lymphocytic leukemia (CLL)."( [Clinical Efficacy and Safety of Rituximab Combined with Fludarabine and Cyclophosphamide for Treatment of Chronic Lymphocytic Leukemia].
Han, HJ; Lu, YW; Xia, RX, 2016
)
0.43
" The patients in control group were treated with CHOP chemotherapy, the patients in observation group were treated with rituximab combined with fludarabine, cyclophosphamide treatment."( [Clinical Efficacy and Safety of Rituximab Combined with Fludarabine and Cyclophosphamide for Treatment of Chronic Lymphocytic Leukemia].
Han, HJ; Lu, YW; Xia, RX, 2016
)
0.43
"application of rituximab combined with fludarabine and cyclophosphamide in the treatment of CLL shows the higher curative effect, can effectively improve the symptoms and reduce the incidence of adverse reactions."( [Clinical Efficacy and Safety of Rituximab Combined with Fludarabine and Cyclophosphamide for Treatment of Chronic Lymphocytic Leukemia].
Han, HJ; Lu, YW; Xia, RX, 2016
)
0.43
" We performed an open-label, multi-center, phase II study to investigate the effect and quality of life (QoL) of treatment with vorinostat in combination with fludarabine, mitoxantrone and dexamethasone (V-FND) for relapsed or refractory MCL."( Results of a phase II study of vorinostat in combination with intravenous fludarabine, mitoxantrone, and dexamethasone in patients with relapsed or refractory mantle cell lymphoma: an interim analysis.
Kim, BS; Kim, HJ; Kim, JA; Kim, SJ; Kim, WS; Kong, JH; Park, SK; Park, Y; Shin, DY; Won, JH; Yoon, DH, 2016
)
0.43
"To investigate the efficacy and safety of haploidentical allo-HSCT in combination of reduced intensity preconditioning combined with cyclophosphamid (CTX)-induced immune tolerance after transplanitation for treatment of severe aplastic anemia (SAA)."( [Clinical Efficacy of Haploidentical Allo-HSCT of Reduced Intensity Preconditioning Combined with Induced Immune Tolerance after Transplantation for Severe Aplastic Anemia].
Chen, HR; Chen, P; Guo, Z; He, XP; Liu, XD; Lou, JX; Yang, K; Zhang, Y, 2016
)
0.43
"A total of 15 patients with SAA received the haploidentical allo-HSCT of reduced intensity preconditioning combined with CTX-induced immune tolerance after transplartation in the General hospital of Beijing military command of chinese PLA from June 2012 to December 2014."( [Clinical Efficacy of Haploidentical Allo-HSCT of Reduced Intensity Preconditioning Combined with Induced Immune Tolerance after Transplantation for Severe Aplastic Anemia].
Chen, HR; Chen, P; Guo, Z; He, XP; Liu, XD; Lou, JX; Yang, K; Zhang, Y, 2016
)
0.43
"The haploidentical allo-HSCT of reduced intensity preconditioning combined with CTX-induced immune tolerance after transplantation is safet and effective for SAA patients, that may be applied to clinical therapy."( [Clinical Efficacy of Haploidentical Allo-HSCT of Reduced Intensity Preconditioning Combined with Induced Immune Tolerance after Transplantation for Severe Aplastic Anemia].
Chen, HR; Chen, P; Guo, Z; He, XP; Liu, XD; Lou, JX; Yang, K; Zhang, Y, 2016
)
0.43
" Here, we conducted a single-arm, phase II trial to examine the efficacy and safety of lower-dose fludarabine and cyclophosphamide combined with a standard dose of rituximab (LD-FCR) in elderly patients with previously untreated CLL."( Low-dose fludarabine and cyclophosphamide combined with standard dose rituximab (LD-FCR) is an effective and safe regimen for elderly untreated patients with chronic lymphocytic leukemia: The Israeli CLL study group experience.
Aviv, A; Bairey, O; Braester, A; Fineman, R; Herishanu, Y; Levi, I; Polliack, A; Rahimi-Levene, N; Ruchlemer, R; Shvidel, L; Tadmor, T; Yuklea, M, 2019
)
0.51
" Subsequently, we demonstrated Entinostat (HDAC1 inhibitor) combination with Fludarabine significantly induced apoptosis in TP53 mutations CLL cells."( Entinostat combined with Fludarabine synergistically enhances the induction of apoptosis in TP53 mutated CLL cells via the HDAC1/HO-1 pathway.
Chen, B; Fang, Q; He, Z; Li, P; Ma, D; Ren, M; Wang, J; Zhe, N; Zhong, Q; Zhou, Z, 2019
)
0.51
" For this particular group we evaluated the efficacy and safety of fludarabine, cytarabine, granulocyte colony-stimulating factor (FLAG) in combination with etoposide (FLAG-Eto) in 36 patients."( Etoposide Combined with FLAG Salvage Therapy Is Effective in Multiple Relapsed/Refractory Acute Myeloid leukemia.
Dührsen, U; Flasshove, M; Hanoun, M; Noppeney, R; Schmitz, C; Westhus, J, 2020
)
0.56
" Additional drugs reported in combination with the Treo-Flu backbone are thiotepa and melphalan."( Treosulfan in combination with fludarabine as part of conditioning treatment prior to allogeneic hematopoietic stem cell transplantation.
Ussowicz, M, 2020
)
0.56
" This phase Ib/II study evaluated the safety and efficacy of fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin combined with the B-cell lymphoma-2 inhibitor venetoclax in ND-AML and R/R-AML."( Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia.
Adeoti, M; Alvarado, Y; Andreeff, M; Borthakur, G; Daver, N; DiNardo, CD; Garcia-Manero, G; Issa, G; Jabbour, E; Jain, N; Kadia, T; Kantarjian, HM; Konopleva, MY; Kornblau, S; Lachowiez, CA; Loghavi, S; Maiti, A; Masarova, L; Montalban Bravo, G; Pemmaraju, N; Ravandi, F; Sasaki, K; Short, NJ; Takahashi, K; Wang, S; Xiao, L; Yilmaz, M, 2021
)
0.62
"This study aimed to identify the maximum-tolerated dose (MTD) of cyclophosphamide when combined with bortezomib and fludarabine (B-FC) in a phase 1b trial, and to assess the efficacy and safety of this combination in a phase 2 trial in patients with relapsed or refractory MCL (rrMCL)."( Bortezomib in combination with fludarabine plus cyclophosphamide for patients with relapsed or refractory mantle-cell lymphoma: results of the LYM-4003 study.
Cai, QQ; Cao, JN; Feng, JF; Gao, Y; Huang, HQ; Jiang, WQ; Jin, J; Li, ZM; Wang, HQ; Wang, XX; Zhang, HL, 2021
)
0.62
" We treated 174 consecutive patients with newly diagnosed CBF-AML in a prospective clinical trial of FLAG-based induction/consolidation in combination with gemtuzumab ozogamicin (FLAG-GO; N = 65) or in combination with idarubicin (FLAG-IDA; N = 109)."( Retrospective comparison of survival and responses to Fludarabine, Cytarabine, GCSF (FLAG) in combination with gemtuzumab ozogamicin (GO) or Idarubicin (IDA) in patients with newly diagnosed core binding factor (CBF) acute myelogenous leukemia: MD Anderso
Alvarado, Y; Borthakur, G; Brandt, M; Daver, N; DiNardo, C; Garcia-Manero, G; Jabbour, EJ; Kadia, T; Kantarjian, H; Ohanian, M; Patel, K; Pemmaraju, N; Pierce, S; Ravandi, F; Takahashi, K; Wang, X, 2022
)
0.72
"Total body irradiation (TBI) at a dose of 12 Gy combined with cyclophosphamide (CyTBI12Gy) is one of the standard myeloablative regimens for patients with acute myeloid leukemia (AML) treated with allogeneic hematopoietic cell transplantation (allo-HCT)."( Fludarabine versus cyclophospamide in combination with myeloablative total body irradiation as conditioning for patients with acute myeloid leukemia treated with allogeneic hematopoietic cell transplantation. A study from the Acute Leukemia Working Party
Arat, M; Bourhis, JH; Cornelissen, JJ; Giebel, S; Grillo, G; Hilgendorf, I; Kröger, N; Labopin, M; Maertens, J; Mohty, M; Nagler, A; Poiré, X; Salmenniemi, U; Savani, B; Schroeder, T; Spyridonidis, A; Swoboda, R, 2023
)
0.91
"In this registry-based study we retrospectively compared outcomes of allogeneic hematopoietic cell transplantation (allo-HCT) for adult patients with acute lymphoblastic leukemia (ALL) following conditioning with total body irradiation (TBI) combined with either cyclophosphamide (Cy) or fludarabine (Flu)."( Fludarabine or cyclophosphamide in combination with total body irradiation as myeloablative conditioning prior to allogeneic hematopoietic cell transplantation for acute lymphoblastic leukemia: an analysis by the Acute Leukemia Working Party of the EBMT.
Aljurf, M; Arat, M; Bourhis, JH; Brissot, E; Forcade, E; Giebel, S; Helbig, G; Huynh, A; Kröger, N; Labopin, M; Labussière-Wallet, H; Lioure, B; Mohty, M; Nagler, A; Peric, Z; Reményi, P; Salmenniemi, U; Savani, BN; Socié, G; Spirydonidis, A; Srour, M; Tbakhi, A; Zahrani, MA, 2023
)
0.91
"In conclusion, ATG and PTCy combined with Flu-based increased intensity conditioning regimen is effective for acute leukemia in children."( Intensified conditioning regimen with fludarabine combined with post-transplantation cyclophosphamide for haploidentical allogeneic hematopoietic stem cell transplantation in children with high-risk acute leukemia.
Cao, J; Chen, D; Li, S; Liu, X; Lu, Y; Pei, R; Wang, T; Xu, X; Ye, P; Zheng, ZZ, 2023
)
0.91

Bioavailability

The pharmacokinetics, bioavailability and effects on electrocardiographic (ECG) parameters of fludarabine phosphate (2F-ara-AMP) were evaluated in adult patients with B-cell chronic lymphocytic leukemia. An oral dose of 40 mg/m2/day would provide similar systemic drug exposure to the standard intravenous dose of 25 mg/ m2/ day.

ExcerptReferenceRelevance
" Bioavailability of F-ara-A after subcutaneous dosing was approximately 105% of the bioavailability after intravenous administration."( Fludarabine pharmacokinetics after subcutaneous and intravenous administration in patients with lupus nephritis.
Boumpas, DT; Burstein, AH; Illei, GG; Kuo, GM; Pucino, F; Yarboro, C, 2001
)
0.31
" Although intracellular events involved in the pharmacologic action of these compounds have been extensively studied, the role of plasma membrane transporters in nucleoside-derived drug bioavailability and action in leukemia cells has not been comprehensively addressed."( Fludarabine uptake mechanisms in B-cell chronic lymphocytic leukemia.
Bellosillo, B; Casado, FJ; Colomer, D; Gil, J; Molina-Arcas, M; Montserrat, E; Pastor-Anglada, M, 2003
)
0.32
" Pharmacokinetic studies evaluating the bioavailability of oral fludarabine indicate that an oral dose of 40 mg/m2/day would provide similar systemic drug exposure to the standard intravenous dose of 25 mg/m2/day."( Oral fludarabine.
Figgitt, DP; Plosker, GL, 2003
)
0.32
" Pharmacokinetic studies suggest decreased bioavailability with successive cycles, probably due to accelerated metabolism."( High dose chlorambucil in the treatment of lymphoid malignancies.
Nicolle, A; Proctor, SJ; Summerfield, GP, 2004
)
0.32
" We have analyzed the role of plasma membrane transporters in nucleoside-derived drug bioavailability and action in CLL cells."( Equilibrative nucleoside transporter-2 (hENT2) protein expression correlates with ex vivo sensitivity to fludarabine in chronic lymphocytic leukemia (CLL) cells.
Bellosillo, B; Casado, FJ; Colomer, D; Gil, J; Huber-Ruano, I; Marcé, S; Molina-Arcas, M; Montserrat, E; Pastor-Anglada, M; Villamor, N, 2005
)
0.33
"This study determined the oral bioavailability of mycophenolic acid, the active metabolite of mycophenolate mofetil, in patients undergoing nonmyeloablative hematopoietic cell transplantation."( Highly variable mycophenolate mofetil bioavailability following nonmyeloablative hematopoietic cell transplantation.
Brunstein, C; DeFor, T; Ebeling, B; Green, K; Jacobson, P; McGlave, P; Rogosheske, J; Weisdorf, D, 2007
)
0.34
"The pharmacokinetics, bioavailability and effects on electrocardiographic (ECG) parameters of fludarabine phosphate (2F-ara-AMP) were evaluated in adult patients with B-cell chronic lymphocytic leukemia."( Pharmacokinetics, bioavailability and effects on electrocardiographic parameters of oral fludarabine phosphate.
Greenblatt, DJ; Karyagina, EV; Lister-James, J; Lundberg, AS; Yin, W, 2010
)
0.36
" P505-15 (also known as PRT062607) is a novel, highly selective, and orally bioavailable small molecule SYK inhibitor (SYK IC(50) = 1 nM) with anti-SYK activity that is at least 80-fold greater than its affinity for other kinases."( The selective SYK inhibitor P505-15 (PRT062607) inhibits B cell signaling and function in vitro and in vivo and augments the activity of fludarabine in chronic lymphocytic leukemia.
Baker, D; Betz, A; Burke, R; Coffey, G; DeGuzman, F; Druker, BJ; Fletcher, LB; Hollenbach, SJ; Loriaux, MM; Pak, Y; Pandey, A; Sinha, U; Spurgeon, SE; Tyner, JW, 2013
)
0.39
" In this context, we reviewed the sometimes paradoxical antioxidant properties of quercetin and the functional role of its glucuronide and/or sulfate conjugates to discuss the low bioavailability of the molecule measured in vivo."( The pleiotropic flavonoid quercetin: from its metabolism to the inhibition of protein kinases in chronic lymphocytic leukemia.
Russo, GL; Russo, M; Spagnuolo, C, 2014
)
0.4
" The oral bioavailability of F-ara-A from 1,2-dimyristoylglycerophosphate derivative 29 was similar to its oral bioavailability from fludarabine phosphate."( Phospholipid derivatives of cladribine and fludarabine: synthesis and biological properties.
Baranovsky, A; Bogushevich, S; Golubeva, M; Kalinichenko, E; Kulak, T; Kuzmitsky, B; Tsybulskaya, I, 2015
)
0.42
"The ATP-binding cassette transporter P-glycoprotein (P-gp) is known to limit both brain penetration and oral bioavailability of many chemotherapy drugs."( A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
Ambudkar, SV; Brimacombe, KR; Chen, L; Gottesman, MM; Guha, R; Hall, MD; Klumpp-Thomas, C; Lee, OW; Lee, TD; Lusvarghi, S; Robey, RW; Shen, M; Tebase, BG, 2019
)
0.51

Dosage Studied

Fludarabine exposure, previously shown to be highly variable when dosing is based on body surface area (BSA), is a predictor for survival in allogeneic hematopoietic cell transplantation (allo-HCT) These results suggest that individualized model-based dosing of fludrabine in infants and young children may reduce morbidity and mortality.

ExcerptRelevanceReference
" The parent drug was almost quantitatively converted to 2-F-ara-A by apparent first-pass metabolism, with maximum levels of 2-F-ara-A and very low levels (less than 1 fmol/L) of 2-F-ara-AMP observed only in the plasma samples obtained shortly after dosing (2 to 4 minutes)."( Pharmacokinetics of 2-F-ara-A (9-beta-D-arabinofuranosyl-2-fluoroadenine) in cancer patients during the phase I clinical investigation of fludarabine phosphate.
Grever, MR; Malspeis, L; Staubus, AE; Young, D, 1990
)
0.28
" All patients were treated at a dosage of 25 mg/m2 per day for 5 consecutive days for a total of six courses."( Fludarabine treatment in resistant Waldenstrom's macroglobulinemia.
Aitini, E; Bendandi, M; Gherlinzoni, F; Salvucci, M; Tura, S; Zaccaria, A; Zinzani, PL, 1995
)
0.29
" Fludarabine was administered at a dosage of 25 mg/m2 daily for 5 days as a 30-minute intravenous infusion."( Immunosuppressive effects and clinical response of fludarabine in refractory chronic lymphocytic leukemia.
Bergmann, L; Fenchel, K; Hoelzer, D; Jahn, B; Mitrou, PS, 1993
)
0.29
" Twenty-one patients were treated at a dosage of 25 mg/m2 per day for 5 consecutive days."( Fludarabine: an active agent in the treatment of previously-treated and untreated low-grade non-Hodgkin's lymphoma.
Bendandi, M; Benfenati, D; Bocchia, M; Fanin, R; Gozzetti, A; Lauria, F; Raspadori, D; Rondelli, D; Zaja, F; Zinzani, PL, 1993
)
0.29
" FAMP was administered at a dosage of 25 mg/m2 daily for 5 days as 30 minute infusion every fifth week."( Clinical experience with fludarabine and its immunosuppressive effects in pretreated chronic lymphocytic leukemias and low-grade lymphomas.
Bergmann, L; Diehl, V; Engert, A; Fenchel, K; Hoelzer, D; Mitrou, PS; Pralle, H; Wijermans, P, 1995
)
0.29
" With this agent, a dosage regimen of 25-30 mg/m2 daily over 5 days seems to be the most effective to date."( Purine analogs in chronic lymphocytic leukemia and Waldenström's macroglobulinemia.
Kantarjian, H; Keating, MJ; O'Brien, S, 1996
)
0.29
" Other adverse effects such as nausea and vomiting or neurotoxicity are of mild to moderate severity when the recommended dosage is used."( [A new purine analog in the treatment of hematologic malignancy. I. Fludarabine].
Hájek, R; Krahulcová, E; Mayer, J; Penka, M; Vásová, I, 1997
)
0.3
"Previously untreated patients with low-grade lymphoma were entered onto dosing cohorts of four patients each."( Phase I study of fludarabine plus cyclophosphamide in patients with previously untreated low-grade lymphoma: results and and long-term follow-up--a report from the Eastern Cooperative Oncology Group.
Bennett, JM; Cassileth, PA; Gordon, LI; Hochster, HS; Oken, MM; Raphael, BG; Winter, JN, 2000
)
0.31
" However, further studies are required to optimize the CdA schedule and dosage in order to ameliorate its toxic profile while maintaining antitumor activity."( Fludarabine and cladribine in relapsed/refractory low-grade non-Hodgkin's lymphoma: a phase II randomized study.
Balzarotti, M; Bonadonna, G; De Paoli, A; Luoni, M; Rampinelli, I; Santoro, A; Tondini, C; Valagussa, P, 2000
)
0.31
" All four patients obtained no clinical benefit from the treatment; moreover, inflammation indices worsened and the prednisone dosage was increased during the trial, in spite of a significant fall in CD4+ T cells."( Unsuccessful treatment with fludarabine in four cases of refractory rheumatoid arthritis.
Bambara, LM; Biasi, D; Caramaschi, P; Carletto, A, 2000
)
0.31
" Bioavailability of F-ara-A after subcutaneous dosing was approximately 105% of the bioavailability after intravenous administration."( Fludarabine pharmacokinetics after subcutaneous and intravenous administration in patients with lupus nephritis.
Boumpas, DT; Burstein, AH; Illei, GG; Kuo, GM; Pucino, F; Yarboro, C, 2001
)
0.31
" They have shown a dose-response relationship and a higher response rate than previously seen in the lower-dose studies."( Emerging information on the use of rituximab in chronic lymphocytic leukemia.
Albitar, M; Keating, MJ; O'Brien, S, 2002
)
0.31
" The sensitivity of CLL cells to nine drugs (2'-chlorodeoxyadenosine, cisplatin, chlorambucil, cyclosporin A, doxorubicin, fludarabine, prednisolone, verapamil and vincristine) and two types of irradiation (gamma and UV-irradiation) was determined from dose-response curves of 4-day cultures ex vivo."( CD80 antigen expression as a predictor of ex vivo chemosensitivity in chronic lymphocytic leukemia.
Hulkkonen, J; Hurme, M; Kivekäs, I; Vilpo, J; Vilpo, L, 2002
)
0.31
" Dosage was increased gradually (target 30 mg, 3 times weekly, for a maximum of 12 weeks)."( Therapeutic role of alemtuzumab (Campath-1H) in patients who have failed fludarabine: results of a large international study.
Albitar, M; Binet, JL; Brettman, L; Byrd, J; Flinn, I; Hillmen, P; Jain, V; Keating, MJ; Rai, KR; Santabarbara, P; Wacker, B, 2002
)
0.31
" Granulocyte colony-stimulating factor (G-CSF), at the dosage of 5-10 microg/kg/die, or intermediate-dose Ara-C (800 mg/m(2)/q 12h x 6 doses), was administered to obtain peripheral blood stem cell (PBSC) mobilization."( Safety and efficacy of subcutaneous Campath-1H for treating residual disease in patients with chronic lymphocytic leukemia responding to fludarabine.
Brando, B; Cafro, AM; Cairoli, R; Montillo, M; Morra, E; Oreste, P; Pungolino, E; Rossi, V; Tedeschi, A; Veronese, S, 2002
)
0.31
" form of busulfan (Bu) has prompted investigation of administration schedules other than the 4-times-daily dosage commonly used with oral Bu."( Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes.
Andersson, BS; Brown, C; Chaudhry, A; Duggan, P; Glick, S; Gyonyor, E; Morris, D; Quinlan, D; Ruether, JD; Russell, JA; Stewart, D; Tran, HT, 2002
)
0.31
" Although increased dosing intensity and frequency leads to higher response rates than seen in patients treated with standard dose rituximab, responses are almost always partial remissions and the doses used are not feasible in routine clinical practice."( Advancing therapy for chronic lymphocytic leukemia--the role of rituximab.
Hillmen, P, 2004
)
0.32
") dosage regimen is 25 mg/m2 daily for 5 consecutive days, with treatment cycles repeated every 28 days."( Role of fludarabine as monotherapy in the treatment of chronic lymphocytic leukemia.
Leporrier, M, 2004
)
0.32
" Administration of cyclophosphamide at a lower dosage (< or =300 mg) appears to reduce the risk of myelosuppression without compromising efficacy."( Chemotherapy combination treatment regimens with fludarabine in chronic lymphocytic leukemia.
Eichhorst, BF; Hallek, M, 2004
)
0.32
" The optimum dose of chlorambucil has not been defined and there are numerous different dosing schedules available."( High dose chlorambucil in the treatment of lymphoid malignancies.
Nicolle, A; Proctor, SJ; Summerfield, GP, 2004
)
0.32
" In order to decrease the toxicity of the procedure, the dosage of total body irradiation was reduced from 12 to 8 Gy and subsequently the dose of cyclophosphamide from 120 to 80 mg/kg."( Reduced-intensity conditioning using TBI (8 Gy), fludarabine, cyclophosphamide and ATG in elderly CML patients provides excellent results especially when performed in the early course of the disease.
Kolb, HJ; Ledderose, G; Rolf, B; Schleuning, M; Schnittger, S; Schoch, C; Schwerdtfeger, R; Weisser, M, 2004
)
0.32
" According to MMF pharmacokinetic studies, which showed a short half-life of its active metabolite, mycophenolic acid, we increased MMF dosing from 15 mg/kg twice daily to 15 mg/kg 3 times daily to increase immunosuppression and reduce the incidence of both graft rejection and acute GVHD."( Unrelated donor granulocyte colony-stimulating factor-mobilized peripheral blood mononuclear cell transplantation after nonmyeloablative conditioning: the effect of postgrafting mycophenolate mofetil dosing.
Agura, E; Blume, KG; Bruno, B; Chauncey, TR; Kliem, C; Langston, AA; Maloney, DG; Maris, MB; Maziarz, RT; McSweeney, PA; Pulsipher, M; Sandmaier, BM; Shizuru, JA; Storb, R; Storer, BE; Wade, J, 2006
)
0.33
" Intravenous dosing resulted in a median area under the curve (AUC) of 28."( Highly variable mycophenolate mofetil bioavailability following nonmyeloablative hematopoietic cell transplantation.
Brunstein, C; DeFor, T; Ebeling, B; Green, K; Jacobson, P; McGlave, P; Rogosheske, J; Weisdorf, D, 2007
)
0.34
" The comparative effect of different dosage schemes, the role of possible response-prediction factors such as the endogenous erythropoietin level and the results achieved using darbopoietin alpha are reviewed."( [Anemia in chronic lymphatic leukemia: is erythropoietin the solution?].
Bendandi, M; de Gaona, ER; Iglesias, R; Panizo, C; Pérez-Calvo, J; Rifón, J,
)
0.13
" Nine patients were enrolled at increasing dosage levels of ara-C (8, 12, and 16 g/m2 per dose level)."( Phase I trial of FLAGM with high doses of cytosine arabinoside for relapsed, refractory acute myeloid leukemia: study of the Japan Adult Leukemia Study Group (JALSG).
Arai, Y; Doki, N; Ishida, F; Kano, Y; Kawai, Y; Komatsu, N; Miyawaki, S; Morii, T; Ogura, M; Ohno, R; Takeshita, A; Usui, N, 2007
)
0.34
" Pharmacokinetic assays of mycophenolic acid (MPA) showed a therapeutic area under the curve (AUC) at the dosage of 3 g daily, although a large inter- and intraindividual variations of MPA plasma levels were found."( Reduced-intensity conditioning allogeneic transplantation from unrelated donors: evaluation of mycophenolate mofetil plus cyclosporin A as graft-versus-host disease prophylaxis.
Caballero, D; Calvo, MV; de la Cámara, R; de Oteiza, JP; Heras, I; Martino, R; Pérez-Simón, JA; Rebollo, N; San Miguel, JF; Sierra, J; Valcarcel, D, 2008
)
0.35
" However, with MTD dosing this drug ratio is not optimal to produce synergy and future studies using ratiometric dosing are required to confirm these findings."( Phase I evaluation of gemcitabine, mitoxantrone, and their effect on plasma disposition of fludarabine in patients with relapsed or refractory acute myeloid leukemia.
Adams, DJ; Decastro, CM; Gockerman, JP; Moore, JO; Peterson, BL; Petros, WP; Rao, AV; Rizzieri, DA; Sand, GJ; Spasojevic, I; Younis, IR, 2008
)
0.35
" Body surface area-based dosing in the FLU/MEL regimen led to a wide range of MEL doses administered per kilogram body weight (2."( Characteristics and risk factors of oral mucositis after allogeneic stem cell transplantation with FLU/MEL conditioning regimen in context with BU/CY2.
Karas, M; Koza, V; Steinerova, K; Vokurka, S, 2009
)
0.35
" Mean (+/-SD) elimination half-life did not differ significantly between IV and oral dosage groups (11."( Pharmacokinetics, bioavailability and effects on electrocardiographic parameters of oral fludarabine phosphate.
Greenblatt, DJ; Karyagina, EV; Lister-James, J; Lundberg, AS; Yin, W, 2010
)
0.36
" These data suggest that clinical strategies are needed to optimize dosing of fludarabine to prevent overexposure and toxicity in HCT."( High fludarabine exposure and relationship with treatment-related mortality after nonmyeloablative hematopoietic cell transplantation.
Brunstein, CG; Cao, Q; Green, KG; Jacobson, PA; Long-Boyle, JR; McGlave, PB; Miller, JS; Rogosheske, J; Wagner, JE; Weisdorf, DJ, 2011
)
0.37
" Forodesine has biologic activity in CLL; pharmacodynamic parameters suggest that an alternate dosing schedule and/or higher doses to achieve greater intracellular dGTP may be beneficial in this patient population."( Phase 2 and pharmacodynamic study of oral forodesine in patients with advanced, fludarabine-treated chronic lymphocytic leukemia.
Balakrishnan, K; Bantia, S; Bickel, S; Chen, Y; Gandhi, V; Kantarjian, H; Keating, MJ; Kilpatrick, JM; O'Brien, S; Ravandi, F; Tyler, BF; Verma, D, 2010
)
0.36
" Bu in different dosing protocols, (2) compare intrasubject variability of Bu PK over repeated administrations; (3) examine the effect of concomitant administration of fludarabine on Bu PK, and (4) examine the effect of plasma concentrations of glutathione (GSH), the cosubstrate in Bu metabolism, on Bu clearance."( Linearity and stability of intravenous busulfan pharmacokinetics and the role of glutathione in busulfan elimination.
Almog, S; Gopher, A; Halkin, H; Hassoun, E; Kurnik, D; Loebstein, R; Nagler, A; Shimoni, A, 2011
)
0.37
" A dose-response relationship between busulfan exposure and outcome is known."( Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease.
Artz, AS; Del Cerro, P; Godley, LA; Hart, J; Horowitz, S; Innocenti, F; Larson, RA; O'Donnell, PH; Odenike, OM; Pai, RK; Stock, W; Undevia, SD; Van Besien, K, 2010
)
0.36
" In the remaining 53 patients, the first-dose AUC was within the target range and no dosing adjustments were required."( Pharmacokinetic targeting of i.v. BU with fludarabine as conditioning before hematopoietic cell transplant: the effect of first-dose area under the concentration time curve on transplant-related outcomes.
Anasetti, C; Ayala, E; Fancher, K; Fernandez, H; Field, T; Gardiner, JA; Kharfan-Dabaja, MA; Kim, J; Miller, S; Milone, MC; Perez, L; Perkins, J; Shaw, LM; Tate, C, 2011
)
0.37
" busulfan dosage scheme."( Influence of GST gene polymorphisms on the clearance of intravenous busulfan in adult patients undergoing hematopoietic cell transplantation.
Bae, KS; Choi, Y; Han, SB; Hur, EH; Kim, DY; Kim, SD; Lee, JH; Lee, KH; Lim, HS; Lim, SN; Noh, GJ; Yun, SC, 2011
)
0.37
"Because of the less graft-facilitating effect by bone marrow (BM), we need to assess a dosage of conditioning more accurately particularly in combination with reduced-intensity conditioning."( A prospective dose-finding trial using a modified continual reassessment method for optimization of fludarabine plus melphalan conditioning for marrow transplantation from unrelated donors in patients with hematopoietic malignancies.
Atsuta, Y; Imahashi, N; Ito, T; Kato, T; Miyamura, K; Morishita, Y; Murata, M; Naoe, T; Nishiwaki, S; Ohashi, H; Sawa, M; Suzuki, R; Terakura, S; Yasuda, T, 2011
)
0.37
" Although alemtuzumab in relapsed/refractory CLL may be beneficial, the optimal dosage and risk of infection related to its use remain thus far deeply controversial issues."( Multicenter study of subcutaneous alemtuzumab administered at reduced dose in patients with fludarabine-relapsed/refractory chronic lymphocytic leukemia: final analysis.
Argentieri, D; Bartomioli, M; Bezares, RF; Diaz, A; Garay, G; Milone, G; Ryser, R; Stemelin, G; Zubiaur, EL, 2011
)
0.37
" Busulfan clearance was not associated with sex or age, but was associated with the day of dosing and conditioning regimen (P = ."( Accurate targeting of daily intravenous busulfan with 8-hour blood sampling in hospitalized adult hematopoietic cell transplant recipients.
Blough, DK; Deeg, HJ; McCune, JS; McDonald, GB; O'Donnell, PV; Pawlikowski, MA; Rezvani, A; Yeh, RF, 2012
)
0.38
" Therefore, mAb therapies that rely substantially on effector mechanisms subject to exhaustion, including complement, may benefit from lower, more frequent dosing schemes optimized to sustain and maximize killing by cytotoxic immune effector systems."( Exhaustion of cytotoxic effector systems may limit monoclonal antibody-based immunotherapy in cancer patients.
Beum, PV; Beurskens, FJ; Engelberts, P; Farooqui, M; Lindorfer, MA; Mackus, WJ; Parren, PW; Taylor, RP; Wiestner, A, 2012
)
0.38
" We aimed to provide a rationale for optimal dosing and scheduling of this anti-CD20 antibody based on pharmacokinetics."( Rituximab serum concentrations during immuno-chemotherapy of follicular lymphoma correlate with patient gender, bone marrow infiltration and clinical response.
Burgstaller, S; Drach, J; Einberger, C; Fridrik, M; Gaiger, A; Greil, R; Heintel, D; Hopfinger, G; Jäger, U; Mannhalter, C; Oberaigner, W; Porpaczy, E; Putman, M; Raderer, M; Skrabs, C; Zeitlinger, M, 2012
)
0.38
"The results of this pilot trial suggest that more differentiated dosing schedules based on gender and bone marrow infiltration should be explored for rituximab therapy for lymphoma."( Rituximab serum concentrations during immuno-chemotherapy of follicular lymphoma correlate with patient gender, bone marrow infiltration and clinical response.
Burgstaller, S; Drach, J; Einberger, C; Fridrik, M; Gaiger, A; Greil, R; Heintel, D; Hopfinger, G; Jäger, U; Mannhalter, C; Oberaigner, W; Porpaczy, E; Putman, M; Raderer, M; Skrabs, C; Zeitlinger, M, 2012
)
0.38
" This study shows that, in routine clinical practice, there is low adherence to the original MD Anderson Cancer Center fludarabine-cyclophosphamide-rituximab schedule, and that the decision to modify dosage was mostly taken by the individual physician and was based on anticipated toxicity."( Impact of dose intensity on outcome of fludarabine, cyclophosphamide, and rituximab regimen given in the first-line therapy for chronic lymphocytic leukemia.
Borel, C; Bouvet, E; Cazin, B; Compaci, G; Laurent, G; Michallet, AS; Obéric, L; Ysebaert, L, 2013
)
0.39
" Oral dosing in mice prevented BCR-mediated splenomegaly and significantly inhibited NHL tumor growth in a xenograft model."( The selective SYK inhibitor P505-15 (PRT062607) inhibits B cell signaling and function in vitro and in vivo and augments the activity of fludarabine in chronic lymphocytic leukemia.
Baker, D; Betz, A; Burke, R; Coffey, G; DeGuzman, F; Druker, BJ; Fletcher, LB; Hollenbach, SJ; Loriaux, MM; Pak, Y; Pandey, A; Sinha, U; Spurgeon, SE; Tyner, JW, 2013
)
0.39
" More frequent dosing of rituximab may be required to prevent autoimmune complications in at-risk patients (clinicaltrials."( Rapid clearance of rituximab may contribute to the continued high incidence of autoimmune hematologic complications of chemoimmunotherapy for chronic lymphocytic leukemia.
Beum, PV; Lee, E; Lindorfer, MA; Marti, G; Mo, CC; Njuguna, N; Taylor, RP; Vire, B; Wiestner, A; Wilson, WH, 2013
)
0.39
" As a RIC regimen for CBT, 140 mg/m(2) melphalan with fludarabine and anti-lymphocyte globulin or anti-thymocyte globulin may be feasible, but further dosage optimization should be performed in controlled clinical trials."( Feasibility of reduced-intensity conditioning followed by unrelated cord blood transplantation for primary hemophagocytic lymphohistiocytosis: a nationwide retrospective analysis in Japan.
Atsuta, Y; Goto, H; Inagaki, J; Inoue, M; Ishii, E; Kato, K; Kawa, K; Koike, K; Ohga, S; Okada, K; Sawada, A; Suzuki, N; Suzuki, R; Yabe, H; Yasutomo, K, 2013
)
0.39
"We utilized a new reduced intensity conditioning (RIC) containing of new dosage of intravenous Bu (9."( [The efficacy and safety of modified busulfan/fludarabine conditioning regimen in elderly or drug-intolerable patients with hematologic malignancies].
Chen, H; Fu, HX; Huang, XJ; Liu, DH; Liu, KY; Sun, YQ; Tang, FF; Wang, FR; Wang, Y; Xu, LP, 2013
)
0.39
" Overall, patients dosed by DBW were more likely to undershoot goal AUC (-12."( Busulfan dosing (Q6 or Q24) with adjusted or actual body weight, does it matter?
Awan, FT; Clemmons, AB; DeRemer, DL; Evans, S, 2015
)
0.42
" B-cell depletion was observed along with complement consumption; median C2 and CH50 levels appeared lower during monthly dosing in patients who responded."( An open-label, single-arm, phase 1 study to assess biomarker effects, efficacy and safety of ofatumumab in patients with refractory chronic lymphocytic leukemia.
Butler, AC; Chan, G; Fang, L; Jewell, RC; Kipps, TJ; Laubscher, KH; Lewis, E; Lindeman, R; Patton, WN; Sedoti, D; Witman, P; Zhou, YY, 2015
)
0.42
" Precision dosing trials are warranted."( Alemtuzumab levels impact acute GVHD, mixed chimerism, and lymphocyte recovery following alemtuzumab, fludarabine, and melphalan RIC HCT.
Davies, SM; Filipovich, AH; Jodele, S; Lane, A; Marsh, RA; Mehta, PA; Neumeier, L, 2016
)
0.43
" We sought to develop a model for fludarabine dosing in adult HCT recipients that would allow for precise dose targeting and predict adverse clinical outcomes."( Personalized fludarabine dosing to reduce nonrelapse mortality in hematopoietic stem-cell transplant recipients receiving reduced intensity conditioning.
Brundage, R; Brunstein, CG; Cao, Q; Jacobson, PA; Jensen, K; Kirstein, MN; Kurtzweil, A; Long-Boyle, J; Rogosheske, J; Sanghavi, K; Wagner, J; Warlick, ED; Weisdorf, DJ; Wiseman, A, 2016
)
0.43
" Pharmacokinetic dosing of busulfan (Bu) is frequently done for myeloablative conditioning, but evidence for its use is limited in RIC transplants."( Fludarabine-Busulfan Reduced-Intensity Conditioning in Comparison with Fludarabine-Melphalan Is Associated with Increased Relapse Risk In Spite of Pharmacokinetic Dosing.
Al-Kali, A; Alkhateeb, HB; Damlaj, M; Gangat, N; Gastineau, DA; Hashmi, S; Hefazi, M; Hogan, WJ; Litzow, MR; Partain, DK; Patnaik, MM; Wolf, RC, 2016
)
0.43
" We previously suggested an optimal day 0 targeted range of alemtuzumab, but there are no pediatric data regarding the pharmacokinetics (PK) of subcutaneous alemtuzumab to guide precision dosing trials."( Pretransplant Absolute Lymphocyte Counts Impact the Pharmacokinetics of Alemtuzumab.
Chandra, S; Emoto, C; Fukuda, T; Khandelwal, P; Marsh, RA; Mehta, PA; Neumeier, L; Teusink-Cross, A; Vinks, AA, 2017
)
0.46
" These results suggest that individualized model-based dosing of fludarabine in infants and young children may reduce morbidity and mortality through improved rates of disease-free survival and limiting drug-related toxicity."( Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients.
Aweeka, F; Chan, D; Cowan, MJ; Dvorak, CC; Huang, L; Ivaturi, V; Jennissen, C; Liu, T; Long-Boyle, J; Orchard, PJ; Pai, SY; Stricherz, M; Tolar, J; Wahlstrom, J, 2017
)
0.46
"Unintentional passive diffusion of conventional small molecular weight pharmaceuticals across intact membranes of normal healthy cells in tissues and organ systems induces sequelae that limit therapeutic dosage and duration of administration."( Selectively Targeted Anti-Neoplastic Cytotoxicity of Three Immunopharmaceuticals with Covalently Bound Fludarabine, Gemcitabine and Dexamethasone Moieties Synthesized Utilizing Organic Chemistry Reactions in a Multi-Stage Regimen.
Coyne, CP; Narayanan, L, 2018
)
0.48
" As no definitive pharmacokinetic (PK) basis for HCT dosing for the wide age and weight range in HCT is available, linear body surface area (BSA)-based dosing is still used."( Population Pharmacokinetics of Fludarabine in Children and Adults during Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation.
Boelens, JJ; Dorlo, TPC; Huitema, ADR; Kuball, J; Langenhorst, JB; Nierkens, S; van Kesteren, C; van Maarseveen, EM, 2019
)
0.51
" Therefore, current linear BSA-based dosing leads to highly variable exposure, which may lead to variable treatment outcome."( Population Pharmacokinetics of Fludarabine in Children and Adults during Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation.
Boelens, JJ; Dorlo, TPC; Huitema, ADR; Kuball, J; Langenhorst, JB; Nierkens, S; van Kesteren, C; van Maarseveen, EM, 2019
)
0.51
" The RIC and MAC regimens were dosed at the average daily area under the concentration-vs-time curve (AUC) of 4000 µMol min and 5000-6000 µMol min, or total course AUC of 16,000 µMol min and 20,000-24,000 µMol min, respectively; PK-guided dosing removes overlap in systemic Bu exposure."( Reduced intensity vs. myeloablative conditioning with fludarabine and PK-guided busulfan in allogeneic stem cell transplantation for patients with AML/MDS.
Alatrash, G; Andersson, BS; Champlin, RE; Chen, J; Crain, AK; Di Stasi, A; Jones, RB; Kidwell, KM; Popat, U; Shpall, EJ; Thall, PF; Zope, M, 2019
)
0.51
" A randomized controlled trial (RCT) comparing alternative fludarabine dosing strategies to current practice may be warranted, but should be sufficiently powered for a relevant end point, while still feasible to enroll."( Clinical Trial Simulation To Optimize Trial Design for Fludarabine Dosing Strategies in Allogeneic Hematopoietic Cell Transplantation.
Boelens, JJ; de Witte, MA; Dorlo, TPC; Huitema, ADR; Langenhorst, JB; Nierkens, S; van Kesteren, C; van Maarseveen, EM, 2020
)
0.56
" Personalized PK-directed dosing to achieve optimal fludarabine exposure should be tested in prospective trials and, based on this analysis, may reduce disease relapse after CAR T-cell therapy."( Optimal fludarabine lymphodepletion is associated with improved outcomes after CAR T-cell therapy.
Baggott, C; Boelens, JJ; Brown, PA; Chinnabhandar, V; Curran, KJ; Egeler, E; Fabrizio, VA; Goksenin, AY; Hermiston, M; Karras, NA; Keating, AK; Krupski, C; Kunicki, M; Laetsch, TW; Mackall, CL; Margossian, SP; Mauguen, A; Mavroukakis, S; Moskop, A; Myers, GD; Pacenta, H; Phillips, CL; Prabhu, S; Qayed, M; Rabik, CA; Rossoff, J; Satwani, P; Schultz, LM; Stefanski, HE; Talano, JA; Verneris, MR; Wilcox, R, 2022
)
0.72
" Fludarabine exposure, previously shown to be highly variable when dosing is based on body surface area (BSA), is a predictor for survival in allogeneic hematopoietic cell transplantation (allo-HCT)."( Fludarabine exposure predicts outcome after CD19 CAR T-cell therapy in children and young adults with acute leukemia.
Admiraal, R; Bierings, M; Blok, H; Calkoen, FG; De Koning, C; Dekker, L; Hoogerbrugge, P; Huitema, ADR; Jiang, Y; Lindemans, CA; Nierkens, S; Nijstad, AL; Pieters, R; Spoon, M; Van Der Elst, KCM; Van Der Vlugt, M; Van Tinteren, H; Veldkamp, SR; Visscher, H; Vormoor, B; Vormoor, HJ, 2022
)
0.72
" Measured GFR (mGFR) may better predict drug dosing to mitigate toxicity and increase the chances of successful engraftment."( Relationship of iothalamate clearance and NRM in patients receiving fludarabine and melphalan reduced-intensity conditioning.
Alkhateeb, HB; Barreto, EF; Bartoo, GT; Hogan, WJ; Kutzke, JL; Leung, N; Litzow, MR; Mangaonkar, AA; Mara, KC; Merten, JA; Pawlenty, AG; Shah, MV, 2022
)
0.72
" These findings suggest that PK-directed fludarabine dosing to achieve an optimal AUC may result in improved outcomes for patients receiving axi-cel."( Identifying an optimal fludarabine exposure for improved outcomes after axi-cel therapy for aggressive B-cell non-Hodgkin lymphoma.
Bachanova, V; Bishop, MR; Brower, J; Chen, AI; Devlin, SM; Flynn, JR; Gonen, M; Ip, A; Leslie, LA; Maakaron, J; Maziarz, RT; McGuirk, JP; Nastoupil, LJ; Oluwole, OO; Parascondola, A; Perales, MA; Porter, DL; Riedell, PA; Schuster, SJ; Scordo, M; Shouval, R; Tomas, AA, 2023
)
0.91
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Drug Classes (1)

ClassDescription
purine nucleoside
[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]

Protein Targets (28)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Chain A, Beta-lactamaseEscherichia coli K-12Potency112.20200.044717.8581100.0000AID485294
Fumarate hydrataseHomo sapiens (human)Potency2.09310.00308.794948.0869AID1347053
TDP1 proteinHomo sapiens (human)Potency0.38010.000811.382244.6684AID686978; AID686979
AR proteinHomo sapiens (human)Potency16.34300.000221.22318,912.5098AID1259243; AID1259247; AID743035
Smad3Homo sapiens (human)Potency0.38200.00527.809829.0929AID588855; AID720534; AID720537
hypoxia-inducible factor 1, alpha subunit (basic helix-loop-helix transcription factor)Homo sapiens (human)Potency6.30960.00137.762544.6684AID914; AID915
nuclear receptor subfamily 1, group I, member 3Homo sapiens (human)Potency5.20810.001022.650876.6163AID1224838; AID1224893
EWS/FLI fusion proteinHomo sapiens (human)Potency0.68790.001310.157742.8575AID1259252; AID1259253; AID1259255; AID1259256
polyproteinZika virusPotency2.09310.00308.794948.0869AID1347053
67.9K proteinVaccinia virusPotency5.12090.00018.4406100.0000AID720579; AID720580
IDH1Homo sapiens (human)Potency2.59290.005210.865235.4813AID686970
cytochrome P450, family 19, subfamily A, polypeptide 1, isoform CRA_aHomo sapiens (human)Potency11.88320.001723.839378.1014AID743083
chromobox protein homolog 1Homo sapiens (human)Potency89.12510.006026.168889.1251AID540317
nuclear factor erythroid 2-related factor 2 isoform 2Homo sapiens (human)Potency11.08240.00419.984825.9290AID504444; AID720524
thyroid hormone receptor beta isoform 2Rattus norvegicus (Norway rat)Potency19.40810.000323.4451159.6830AID743065; AID743067
nuclear factor erythroid 2-related factor 2 isoform 1Homo sapiens (human)Potency13.59290.000627.21521,122.0200AID743202; AID743219
urokinase-type plasminogen activator precursorMus musculus (house mouse)Potency8.91250.15855.287912.5893AID540303
plasminogen precursorMus musculus (house mouse)Potency8.91250.15855.287912.5893AID540303
urokinase plasminogen activator surface receptor precursorMus musculus (house mouse)Potency8.91250.15855.287912.5893AID540303
gemininHomo sapiens (human)Potency7.30780.004611.374133.4983AID624296
lamin isoform A-delta10Homo sapiens (human)Potency35.48130.891312.067628.1838AID1487
Cellular tumor antigen p53Homo sapiens (human)Potency12.40030.002319.595674.0614AID651631; AID720552
[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)
corticotropin-releasing hormone receptor 2Homo sapiens (human)IC50 (µMol)47.10000.36807.095518.0000AID602180
corticotropin releasing factor-binding proteinHomo sapiens (human)IC50 (µMol)47.10000.36807.095518.0000AID602180
5'-nucleotidaseHomo sapiens (human)Ki700.00000.08840.16560.5100AID1589305; AID1589306
Adenosine receptor A3Rattus norvegicus (Norway rat)Ki10.40000.00030.91969.0000AID33337
[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)
corticotropin-releasing hormone receptor 2Homo sapiens (human)EC50 (µMol)53.00001.120011.561736.8000AID602473
corticotropin releasing factor-binding proteinHomo sapiens (human)EC50 (µMol)53.00001.120011.561736.8000AID602473
glycogen synthase kinase-3 beta isoform 1Homo sapiens (human)EC50 (µMol)300.00000.212522.156283.9400AID434954
[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)
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (139)

Processvia Protein(s)Taxonomy
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)
DNA metabolic process5'-nucleotidaseHomo sapiens (human)
leukocyte cell-cell adhesion5'-nucleotidaseHomo sapiens (human)
response to inorganic substance5'-nucleotidaseHomo sapiens (human)
response to ATP5'-nucleotidaseHomo sapiens (human)
ADP catabolic process5'-nucleotidaseHomo sapiens (human)
ATP metabolic process5'-nucleotidaseHomo sapiens (human)
adenosine biosynthetic process5'-nucleotidaseHomo sapiens (human)
negative regulation of inflammatory response5'-nucleotidaseHomo sapiens (human)
calcium ion homeostasis5'-nucleotidaseHomo sapiens (human)
inhibition of non-skeletal tissue mineralization5'-nucleotidaseHomo sapiens (human)
AMP catabolic process5'-nucleotidaseHomo sapiens (human)
adenosine catabolic processAdenosine deaminase Bos taurus (cattle)
cell adhesionAdenosine deaminase Bos taurus (cattle)
nucleotide metabolic processAdenosine deaminase Bos taurus (cattle)
purine ribonucleoside monophosphate biosynthetic processAdenosine deaminase Bos taurus (cattle)
inosine biosynthetic processAdenosine deaminase Bos taurus (cattle)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (43)

Processvia Protein(s)Taxonomy
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 binding5'-nucleotidaseHomo sapiens (human)
5'-deoxynucleotidase activity5'-nucleotidaseHomo sapiens (human)
protein binding5'-nucleotidaseHomo sapiens (human)
5'-nucleotidase activity5'-nucleotidaseHomo sapiens (human)
zinc ion binding5'-nucleotidaseHomo sapiens (human)
identical protein binding5'-nucleotidaseHomo sapiens (human)
thymidylate 5'-phosphatase activity5'-nucleotidaseHomo sapiens (human)
IMP 5'-nucleotidase activity5'-nucleotidaseHomo sapiens (human)
GMP 5'-nucleotidase activity5'-nucleotidaseHomo sapiens (human)
XMP 5'-nucleosidase activity5'-nucleotidaseHomo sapiens (human)
adenosine deaminase activityAdenosine deaminase Bos taurus (cattle)
protein bindingAdenosine deaminase Bos taurus (cattle)
zinc ion bindingAdenosine deaminase Bos taurus (cattle)
2'-deoxyadenosine deaminase activityAdenosine deaminase Bos taurus (cattle)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (27)

Processvia Protein(s)Taxonomy
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)
plasma membrane5'-nucleotidaseHomo sapiens (human)
nucleoplasm5'-nucleotidaseHomo sapiens (human)
cytosol5'-nucleotidaseHomo sapiens (human)
plasma membrane5'-nucleotidaseHomo sapiens (human)
external side of plasma membrane5'-nucleotidaseHomo sapiens (human)
cell surface5'-nucleotidaseHomo sapiens (human)
membrane5'-nucleotidaseHomo sapiens (human)
extracellular exosome5'-nucleotidaseHomo sapiens (human)
lysosomeAdenosine deaminase Bos taurus (cattle)
cytoplasmic vesicle lumenAdenosine deaminase Bos taurus (cattle)
anchoring junctionAdenosine deaminase Bos taurus (cattle)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (210)

Assay IDTitleYearJournalArticle
AID1346136Human ribonucleotide reductase regulatory subunit M2 (Nucleoside synthesis and metabolism)1982Molecular pharmacology, Mar, Volume: 21, Issue:2
In vitro biological activity of 9-beta-D-arabinofuranosyl-2-fluoroadenine and the biochemical actions of its triphosphate on DNA polymerases and ribonucleotide reductase from HeLa cells.
AID1346042Human ribonucleotide reductase catalytic subunit M1 (Nucleoside synthesis and metabolism)1982Molecular pharmacology, Mar, Volume: 21, Issue:2
In vitro biological activity of 9-beta-D-arabinofuranosyl-2-fluoroadenine and the biochemical actions of its triphosphate on DNA polymerases and ribonucleotide reductase from HeLa cells.
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.
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.
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.
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.
AID1223762Antitumor activity against CLL1 cells isolated from CLL patient assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID341996Antimicrobial activity against Streptococcus sp. 07941-1 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
AID33337Binding affinity determined by displacement of specific binding of [125I]N-(4-amino-3-iodophenethyl)-adenosine in membranes of CHO cells stably transfected with the rat adenosine A3 receptor1995Journal of medicinal chemistry, Mar-31, Volume: 38, Issue:7
Search for new purine- and ribose-modified adenosine analogues as selective agonists and antagonists at adenosine receptors.
AID540209Volume of distribution at steady state in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID1196587Cytotoxicity against patient PBMC after 48 hrs by CellTitre-Blue assay in presenc of mouse M210B4 cells2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID1196581Cytotoxicity against human HL60 cells after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID1196584Cytotoxicity against human SUDHL9 cells after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
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.
AID1079940Granulomatous liver disease, proven histopathologically. Value is number of references indexed. [column 'GRAN' in source]
AID409956Inhibition of mouse brain MAOB2008Journal of medicinal chemistry, Nov-13, Volume: 51, Issue:21
Quantitative structure-activity relationship and complex network approach to monoamine oxidase A and B inhibitors.
AID1196585Cytotoxicity against patient PBMC after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID1196583Cytotoxicity against human SU-DHL6 cells after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID1222933Apparent volume of distribution at steady state in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
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.
AID1079944Benign tumor, proven histopathologically. Value is number of references indexed. [column 'T.BEN' in source]
AID1079947Comments (NB not yet translated). [column 'COMMENTAIRES' in source]
AID1370965Antiproliferative activity against human HepG2 cells after 72 hrs by SRB assay2018Bioorganic & medicinal chemistry letters, 02-01, Volume: 28, Issue:3
Synthesis of novel 6-substituted amino-9-(β-d-ribofuranosyl)purine analogs and their bioactivities on human epithelial cancer cells.
AID510575Cell cycle arrest in human CEM cells assessed as accumulation at G2/M phase using propidium iodide staining at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1223763Antitumor activity against CLL2 cells isolated from CLL patient assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID1222920Tmax in CD-1 mouse brain at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID477295Octanol-water partition coefficient, log P of the compound2010European journal of medicinal chemistry, Apr, Volume: 45, Issue:4
QSPR modeling of octanol/water partition coefficient of antineoplastic agents by balance of correlations.
AID96165Inhibitory concentration required against human chronic myelogenous leukemic K-562 cell line after 48h, using [3H]thymidine incorporation assay2003Bioorganic & medicinal chemistry letters, Mar-10, Volume: 13, Issue:5
Cytotoxic activity of 6-alkynyl- and 6-alkenylpurines.
AID1223766Antitumor activity against CLL3 cells isolated from CLL patient assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID1197746Stimulation of human OATP1B3-mediated [3H]CCK-8 at 100 uM after 5 mins relative to control2015European journal of medicinal chemistry, Mar-06, Volume: 92Interaction of human organic anion transporter polypeptides 1B1 and 1B3 with antineoplastic compounds.
AID1079945Animal toxicity known. [column 'TOXIC' in source]
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.
AID342000Antimicrobial activity against Staphylococcus aureus CCM 885 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
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).
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).
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.
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.
AID634635Activity of Escherichia coli PNP assessed per mg of protein at 100 uM by UV spectrometric analysis2012European journal of medicinal chemistry, Jan, Volume: 47, Issue:1
Synthesis and evaluation of the substrate activity of C-6 substituted purine ribosides with E. coli purine nucleoside phosphorylase: palladium mediated cross-coupling of organozinc halides with 6-chloropurine nucleosides.
AID1079948Times to onset, minimal and maximal, observed in the indexed observations. [column 'DELAI' in source]
AID510579Inhibition of DNA synthesis in human CEM cells assessed as BrDU incorporation using propidium iodide staining at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID229569Ratio of kinetic parameters Vmax to the of Km.1985Journal of medicinal chemistry, Nov, Volume: 28, Issue:11
2-Fluoroformycin and 2-aminoformycin. Synthesis and biological activity.
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).
AID1174818Cytotoxicity against human HCT116 cells after 72 hrs by SRB assay2015European journal of medicinal chemistry, Jan-07, Volume: 89Synthesis of novel substituted purine derivatives and identification of the cell death mechanism.
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).
AID766997Inhibition of spliceosome-mediated splicing of DNAJB1 gene in human B-CLL cells at 10 uM after 4 hrs by RT-PCR analysis2013Journal of medicinal chemistry, Sep-12, Volume: 56, Issue:17
Stabilized cyclopropane analogs of the splicing inhibitor FD-895.
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.
AID1370962Antiproliferative activity against human HuH7 cells after 72 hrs by SRB assay2018Bioorganic & medicinal chemistry letters, 02-01, Volume: 28, Issue:3
Synthesis of novel 6-substituted amino-9-(β-d-ribofuranosyl)purine analogs and their bioactivities on human epithelial cancer cells.
AID510576Inhibition of RNA synthesis in human CEM cells assessed as BrU incorporation using propidium iodide staining at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1370967Antiproliferative activity against human FOCUS cells after 72 hrs by SRB assay2018Bioorganic & medicinal chemistry letters, 02-01, Volume: 28, Issue:3
Synthesis of novel 6-substituted amino-9-(β-d-ribofuranosyl)purine analogs and their bioactivities on human epithelial cancer cells.
AID1370964Antiproliferative activity against human MCF7 cells after 72 hrs by SRB assay2018Bioorganic & medicinal chemistry letters, 02-01, Volume: 28, Issue:3
Synthesis of novel 6-substituted amino-9-(β-d-ribofuranosyl)purine analogs and their bioactivities on human epithelial cancer cells.
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).
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).
AID510566Cytotoxicity against human K562 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1079941Liver damage due to vascular disease: peliosis hepatitis, hepatic veno-occlusive disease, Budd-Chiari syndrome. Value is number of references indexed. [column 'VASC' in source]
AID1222939Elimination half life in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1222924Cmax in CD-1 mouse brain at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1160916Cytotoxicity against human primary CLL cells2014Journal of medicinal chemistry, Sep-25, Volume: 57, Issue:18
Small-molecule inhibitors of 25-hydroxyvitamin D-24-hydroxylase (CYP24A1): synthesis and biological evaluation.
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).
AID1079939Cirrhosis, proven histopathologically. Value is number of references indexed. [column 'CIRRH' in source]
AID1222936Apparent total clearance in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1222913Drug transport in human CCRF-CEM cells assessed as ENT1-mediated uptake at 10 uM after 1 to 60 mins by liquid scintillation counting analysis2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID31885Binding affinity to adenosine A1 receptor in rat brain membranes by measuring displacement of specific [3H]PIA as radioligand.1995Journal of medicinal chemistry, Mar-31, Volume: 38, Issue:7
Search for new purine- and ribose-modified adenosine analogues as selective agonists and antagonists at adenosine receptors.
AID510577Inhibition of RNA synthesis in human CEM cells assessed as BrU incorporation using propidium iodide staining at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
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).
AID510574Cell cycle arrest in human CEM cells assessed as accumulation at G2/M phase using propidium iodide staining at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1222926AUC (0 to 4 hrs) in CD-1 mouse brain at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID33838Kinetic parameter was determined against adenosine deaminase expressed as Vmax1985Journal of medicinal chemistry, Nov, Volume: 28, Issue:11
2-Fluoroformycin and 2-aminoformycin. Synthesis and biological activity.
AID96168Inhibitory concentration required against human chronic myelogenous leukemic K-562 cell line after 5h, using [3H]thymidine incorporation assay2003Bioorganic & medicinal chemistry letters, Mar-10, Volume: 13, Issue:5
Cytotoxic activity of 6-alkynyl- and 6-alkenylpurines.
AID341999Antimicrobial activity against Staphylococcus aureus ATCC 29213 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
AID1079934Highest frequency of acute liver toxicity observed during clinical trials, expressed as a percentage. [column '% AIGUE' in source]
AID1589305Competitive type of inhibition of human 5'-nucleotidase using IMP as substrate assessed as inhibitor constant for free enzyme by Lineweaver-Burk plot analysis2019European journal of medicinal chemistry, Apr-15, Volume: 168Lead optimization and biological evaluation of fragment-based cN-II inhibitors.
AID1079946Presence of at least one case with successful reintroduction. [column 'REINT' in source]
AID510573Cell cycle arrest in human CEM cells assessed as accumulation at S phase using propidium iodide staining at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1222923Cmax in CD-1 mouse brain at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1079942Steatosis, proven histopathologically. Value is number of references indexed. [column 'STEAT' in source]
AID1222935Apparent total clearance in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID510581Induction of mitotic arrest in human CEM cells assessed as pH3Ser10 positive cells at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
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).
AID94831Compound was tested for cytotoxicity against K562 cell lines1992Journal of medicinal chemistry, Jan-24, Volume: 35, Issue:2
Synthesis and biologic activity of 2'-fluoro-2-halo derivatives of 9-beta-D-arabinofuranosyladenine.
AID1589306Non-competitive type of inhibition of human 5'-nucleotidase using IMP as substrate assessed as inhibitor constant for enzyme substrate complex by Lineweaver-Burk plot analysis2019European journal of medicinal chemistry, Apr-15, Volume: 168Lead optimization and biological evaluation of fragment-based cN-II inhibitors.
AID510565Cytotoxicity against human CEM-DNR-B cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID341995Antimicrobial activity against Escherichia coli ATCC 25922 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
AID510563Cytotoxicity against human A549 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1196579Cytotoxicity against human CCRF-CEM cells after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID1079932Highest frequency of moderate liver toxicity observed during clinical trials, expressed as a percentage. [column '% BIOL' in source]
AID1223765Antitumor activity against human HeLa cells assessed as cell viability by MTT assay2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID1222932Apparent volume of distribution at steady state in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1222919Tmax in CD-1 mouse brain at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID33816Kinetic parameter was determined against adenosine deaminase expressed as Km1985Journal of medicinal chemistry, Nov, Volume: 28, Issue:11
2-Fluoroformycin and 2-aminoformycin. Synthesis and biological activity.
AID1222943AUC (0 to infinity) in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in absence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID540213Half life in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID1370963Antiproliferative activity against human HCT116 cells after 72 hrs by SRB assay2018Bioorganic & medicinal chemistry letters, 02-01, Volume: 28, Issue:3
Synthesis of novel 6-substituted amino-9-(β-d-ribofuranosyl)purine analogs and their bioactivities on human epithelial cancer cells.
AID1223764Antitumor activity against human JVM2 cells assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID81745Compound was tested for cytotoxicity against HEp-2 cell lines1992Journal of medicinal chemistry, Jan-24, Volume: 35, Issue:2
Synthesis and biologic activity of 2'-fluoro-2-halo derivatives of 9-beta-D-arabinofuranosyladenine.
AID1174817Cytotoxicity against human HuH7 cells after 72 hrs by SRB assay2015European journal of medicinal chemistry, Jan-07, Volume: 89Synthesis of novel substituted purine derivatives and identification of the cell death mechanism.
AID96710Compound was tested for cytotoxicity against L1210 cell lines1992Journal of medicinal chemistry, Jan-24, Volume: 35, Issue:2
Synthesis and biologic activity of 2'-fluoro-2-halo derivatives of 9-beta-D-arabinofuranosyladenine.
AID1079935Cytolytic liver toxicity, either proven histopathologically or where the ratio of maximal ALT or AST activity above normal to that of Alkaline Phosphatase is > 5 (see ACUTE). Value is number of references indexed. [column 'CYTOL' in source]
AID1079943Malignant tumor, proven histopathologically. Value is number of references indexed. [column 'T.MAL' in source]
AID1370966Antiproliferative activity against human Mahlavu cells after 72 hrs by SRB assay2018Bioorganic & medicinal chemistry letters, 02-01, Volume: 28, Issue:3
Synthesis of novel 6-substituted amino-9-(β-d-ribofuranosyl)purine analogs and their bioactivities on human epithelial cancer cells.
AID96327Percent inhibition of [3H]-thymidine incorporation was determined in human chronic myelogenous leukemic K-562 cell line at 10 ug/mL after 48 hr2003Bioorganic & medicinal chemistry letters, Mar-10, Volume: 13, Issue:5
Cytotoxic activity of 6-alkynyl- and 6-alkenylpurines.
AID1222928AUC (0 to 4 hrs) in CD-1 mouse brain at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1222940Elimination half life in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID44000Compound was tested for cytotoxicity against CCRF-CEM cell lines1992Journal of medicinal chemistry, Jan-24, Volume: 35, Issue:2
Synthesis and biologic activity of 2'-fluoro-2-halo derivatives of 9-beta-D-arabinofuranosyladenine.
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).
AID510578Inhibition of DNA synthesis in human CEM cells assessed as BrDU incorporation using propidium iodide staining at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID510568Induction of apoptosis in human CEM cells using propidium iodide staining at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
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).
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).
AID510572Cell cycle arrest in human CEM cells assessed as accumulation at S phase using propidium iodide staining at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID540212Mean residence time in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID1079933Acute liver toxicity defined via clinical observations and clear clinical-chemistry results: serum ALT or AST activity > 6 N or serum alkaline phosphatases activity > 1.7 N. This category includes cytolytic, choleostatic and mixed liver toxicity. Value is
AID1079937Severe hepatitis, defined as possibly life-threatening liver failure or through clinical observations. Value is number of references indexed. [column 'MASS' in source]
AID1079938Chronic liver disease either proven histopathologically, or through a chonic elevation of serum amino-transferase activity after 6 months. Value is number of references indexed. [column 'CHRON' in source]
AID510567Cytotoxicity against human paclitaxel-resistant K562 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID341997Antimicrobial activity against Streptococcus sp. 07706-1 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
AID540211Fraction unbound in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID510570Cell cycle arrest in human CEM cells assessed as accumulation at G0/G1 phase using propidium iodide staining at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1196582Cytotoxicity against human K562 cells after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID1223768Antitumor activity against CLL5 cells isolated from CLL patient assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID341998Antimicrobial activity against Streptococcus sp. 07686-2 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
AID510580Induction of mitotic arrest in human CEM cells assessed as pH3Ser10 positive cells at IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
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).
AID510571Cell cycle arrest in human CEM cells assessed as accumulation at G0/G1 phase using propidium iodide staining at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1079936Choleostatic liver toxicity, either proven histopathologically or where the ratio of maximal ALT or AST activity above normal to that of Alkaline Phosphatase is < 2 (see ACUTE). Value is number of references indexed. [column 'CHOLE' in source]
AID1223769Antitumor activity against CLL6 cells isolated from CLL patient assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID409954Inhibition of mouse brain 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.
AID510569Induction of apoptosis in human CEM cells using propidium iodide staining at 5 times IC50 concentration after 24 hrs by flow cytometry2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID510564Cytotoxicity against human CEM cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Sep, Volume: 45, Issue:9
Synthesis of 5-[alkoxy-(4-nitro-phenyl)-methyl]-uridines and study of their cytotoxic activity.
AID1196586Cytotoxicity against healthy human PBMC after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
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).
AID342001Antimicrobial activity against Streptococcus pyogenes AP1 after 20 hrs2007Antimicrobial agents and chemotherapy, Aug, Volume: 51, Issue:8
Deoxyribonucleoside kinases activate nucleoside antibiotics in severely pathogenic bacteria.
AID540210Clearance in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID1079931Moderate liver toxicity, defined via clinical-chemistry results: ALT or AST serum activity 6 times the normal upper limit (N) or alkaline phosphatase serum activity of 1.7 N. Value is number of references indexed. [column 'BIOL' in source]
AID1223767Antitumor activity against CLL4 cells isolated from CLL patient assessed as cell viability after 48 hrs by FACS analysis2014Journal of medicinal chemistry, Jun-26, Volume: 57, Issue:12
Potent fluorinated agelastatin analogues for chronic lymphocytic leukemia: design, synthesis, and pharmacokinetic studies.
AID1196580Cytotoxicity against human Jurkat cells after 48 hrs by CellTitre-Blue assay2015Journal of medicinal chemistry, Feb-12, Volume: 58, Issue:3
SAR-guided development and characterization of a potent antitumor compound toward B-cell neoplasms with no detectable cytotoxicity toward healthy cells.
AID33792Binding affinity to adenosine A2A receptor in rat striatal membranes by measuring displacement of specific [3H]-CGS- 21680 as radioligand1995Journal of medicinal chemistry, Mar-31, Volume: 38, Issue:7
Search for new purine- and ribose-modified adenosine analogues as selective agonists and antagonists at adenosine receptors.
AID1222944AUC (0 to infinity) in CD-1 mouse at 60 mg/kg, iv administered as bolus dose in presence of ENT1 inhibitor NBMPR-P2013Drug metabolism and disposition: the biological fate of chemicals, Apr, Volume: 41, Issue:4
Transport of A1 adenosine receptor agonist tecadenoson by human and mouse nucleoside transporters: evidence for blood-brain barrier transport by murine equilibrative nucleoside transporter 1 mENT1.
AID1174819Cytotoxicity against human T47D cells after 72 hrs by SRB assay2015European journal of medicinal chemistry, Jan-07, Volume: 89Synthesis of novel substituted purine derivatives and identification of the cell death mechanism.
AID1079949Proposed mechanism(s) of liver damage. [column 'MEC' in source]
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
AID1745845Primary qHTS for Inhibitors of ATXN expression
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
AID504810Antagonists of the Thyroid Stimulating Hormone Receptor: HTS campaign2010Endocrinology, Jul, Volume: 151, Issue:7
A small molecule inverse agonist for the human thyroid-stimulating hormone receptor.
AID504812Inverse Agonists of the Thyroid Stimulating Hormone Receptor: HTS campaign2010Endocrinology, Jul, Volume: 151, Issue:7
A small molecule inverse agonist for the human thyroid-stimulating hormone receptor.
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.
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.
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.
AID1347159Primary screen GU Rhodamine 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.
AID1347160Primary screen NINDS Rhodamine 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.
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 (3,359)

TimeframeStudies, This Drug (%)All Drugs %
pre-199025 (0.74)18.7374
1990's434 (12.92)18.2507
2000's1337 (39.80)29.6817
2010's1302 (38.76)24.3611
2020's261 (7.77)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 (%)
Trials812 (23.52%)5.53%
Reviews330 (9.56%)6.00%
Case Studies578 (16.74%)4.05%
Observational16 (0.46%)0.25%
Other1,716 (49.71%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (1439)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Phase II Trial of Fludarabine & Cyclophosphamide Followed by Thalidomide for Angioimmunoblastic Lymphoma [NCT00958854]Phase 237 participants (Anticipated)Interventional2006-01-31Recruiting
A Phase 1-2 Study to Evaluate the Safety and Efficacy of IM19 CAR-T Cells in Patients With Relapsed and Refractory (R/R) Mantle Cell Lymphoma [NCT05155215]Phase 1/Phase 268 participants (Anticipated)Interventional2021-12-31Not yet recruiting
A Phase I Study of FT576 as Monotherapy and in Combination With Daratumumab in Subjects With Relapsed/Refractory Multiple Myeloma [NCT05182073]Phase 1168 participants (Anticipated)Interventional2021-11-24Recruiting
Fludarabine, Cyclophosphamide, Doxorubicin and Rituximab for the Treatment of Post-transplant Lymphoproliferative Disease (PTLD) [NCT01088724]Phase 44 participants (Actual)Interventional2002-02-28Completed
A Pilot Study of Intensified Lymphodepletion Followed by Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Systemic Lupus Erythematosus [NCT00076752]Phase 29 participants (Actual)Interventional2004-01-30Completed
A Phase I Clinical Trial of T-Cells Targeting B-Cell Maturation Antigen for Subjects With Relapsed/Refractory Multiple Myeloma [NCT03943472]Early Phase 110 participants (Anticipated)Interventional2019-07-08Recruiting
A Phase II/III Prospective, Open Label Study to Evaluate Safety and Efficacy of Intravenous Autologous CD19 CAR-T Cells for Relapsed/ Refractory B-Acute Lymphoblastic Leukaemia [NCT03937544]Phase 2/Phase 310 participants (Anticipated)Interventional2019-03-19Recruiting
A Phase III Randomized Trial to Evaluate the Efficacy and Safety of Second-Line Therapy With Fludarabine Plus Alemtuzumab vs. Fludarabine Alone in Patients With B-Cell Chronic Lymphocytic Leukemia [NCT00086580]Phase 3335 participants (Actual)Interventional2004-07-31Completed
Allogeneic HSCT Without Preparative Chemotherapy or With Low-Intensity Preparative Chemotherapy Using Sirolimus and Sirolimus-Generated Donor Th2 Cells for Therapy of Refractory Leukemia, Lymphoma, Myeloma, or Myelodysplastic Syndrome [NCT00074490]Phase 2442 participants (Actual)Interventional2004-01-01Terminated(stopped due to Premature closure due to inability to accrue to ARM IVD, cohorts 1 and 2)
Combined Phase I-Phase II Study of Autologous CD8+ T-cells Transiently Expressing a Chimeric Antigen Receptor Directed to B-Cell Maturation Antigen in Patients With Multiple Myeloma [NCT03448978]Phase 1/Phase 238 participants (Actual)Interventional2018-02-26Completed
Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy [NCT00303719]Phase 2342 participants (Actual)Interventional2002-03-26Terminated(stopped due to IRB Study Closure)
Fludarabine-based Conditioning for Allogeneic Marrow Transplantation From HLA-compatible Unrelated Donors in Severe Aplastic Anemia (BMT CTN #0301) [NCT00326417]Phase 1/Phase 297 participants (Actual)Interventional2006-01-31Completed
A Phase I/II Pilot Study of Memory-like NK Cells to Consolidate TCRαβ T Cell Depleted Haploidentical Transplant in High-risk AML [NCT06158828]Phase 1/Phase 248 participants (Anticipated)Interventional2024-03-31Not yet recruiting
A Phase 1/1b Dose Escalation/Dose Expansion Study of PRGN-3007 UltraCAR-T Cells in Patients With Advanced Hematologic and Solid Tumor Malignancies [NCT05694364]Phase 188 participants (Anticipated)Interventional2023-01-25Recruiting
A Phase I/Ib Study of JNJ-75276617 in Combination With Conventional Chemotherapy for Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias Harboring KMT2A, NPM1, or Nucleoporin Gene Alterations [NCT05521087]Phase 180 participants (Anticipated)Interventional2024-05-29Not yet recruiting
A Safety and Efficacy Study of JNJ-68284528 (Ciltacabtagene Autoleucel) Out-of-Specification (OOS) for Commercial Release in Patients With Multiple Myeloma [NCT05347485]Phase 286 participants (Actual)Interventional2022-05-13Active, not recruiting
A Phase 3 Randomized Study Comparing Bortezomib, Lenalidomide and Dexamethasone (VRd) Followed by Ciltacabtagene Autoleucel, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against BCMA Versus Bortezomib, Lenalidomide, and Dexamethasone (VRd) [NCT04923893]Phase 3650 participants (Anticipated)Interventional2021-08-19Recruiting
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
A Randomized Phase III Study of Ibrutinib (PCI-32765)-Based Therapy vs Standard Fludarabine, Cyclophosphamide, and Rituximab (FCR) Chemoimmunotherapy in Untreated Younger Patients With Chronic Lymphocytic Leukemia (CLL) [NCT02048813]Phase 3529 participants (Actual)Interventional2014-02-20Active, not recruiting
Prospective Non-randomized Multicenter Study to Assess the Efficacy Response Duration and Toxicity of RFC as First-line Treatment and R as Maintenance Treatment, in Patients Diagnosed of Follicular Non Hodgkin Lymphoma [NCT01124526]Phase 475 participants (Actual)Interventional2004-09-30Completed
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 Multicenter, Open-label, Phase 1/2 Clinical Trial to Evaluate the Safety and Anti-Tumor Activity of AB-201 in Subjects With Advanced HER2+ Solid Tumors [NCT05678205]Phase 1/Phase 2133 participants (Anticipated)Interventional2023-08-01Not yet recruiting
Allogeneic Hematopoietic Cell Transplantation for Patients With Non-Malignant Disorders Using Treosulfan, Fludarabine, and Thiotepa [NCT03980769]Phase 240 participants (Anticipated)Interventional2021-05-05Recruiting
Phase II Study of Reduced-Intensity Allogeneic Stem Cell Transplant for High-Risk Chronic Lymphocytic Leukemia (CLL) [NCT01027000]Phase 268 participants (Actual)Interventional2010-02-28Completed
Phase 1b Study Evaluating the Safety and Efficacy of Autologous CD30.CAR-T in Combination With PD-1 Checkpoint Inhibitor (Nivolumab) in Relapsed or Refractory Classical Hodgkin Lymphoma Patients After Failure of Frontline Therapy (ACTION) [NCT05352828]Phase 115 participants (Actual)Interventional2022-07-25Active, not recruiting
Randomized Comparison of Cyclophosphamide Versus Fludarabine in Addition to Anti-thymocyte Globulin for the Conditioning Therapy in Allogeneic Hematopoietic Cell Transplantation for Adult Acquired Aplastic Anemia [NCT01145976]Phase 398 participants (Anticipated)Interventional2010-03-31Recruiting
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
Haplo-identical Hematopoietic Stem Cell Transplantation Following Reduced-intensity Conditioning in Children With Neuroblastoma [NCT01156350]Phase 210 participants (Anticipated)Interventional2011-09-30Not yet recruiting
COBALT: Evaluation of CAR19 T-cells as an Optimal Bridge to Allogeneic Transplantation [NCT02431988]Phase 110 participants (Actual)Interventional2016-06-30Completed
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 Multicenter, Prospective, Non-randomized, Phase I-II Trial to Assess the Efficacy and Safety of the Combination of Oral Quizartinib and the FLAG-IDA Chemotherapy Regimen in First Relapsed/Refractory Acute Myeloid Leukemia (R/R AML) Patients [NCT04112589]Phase 1/Phase 263 participants (Actual)Interventional2019-12-26Active, not recruiting
A Phase II Trial of Alpha Beta T-cell and CD19 B-cell Depleted Peripheral Blood Stem Cell Transplantation Using the CliniMACS System for Patients With Non-Malignant Hematologic Disorders From Matched or Mismatched, Related or Unrelated Donors [NCT03615144]Phase 21 participants (Actual)Interventional2018-07-23Terminated(stopped due to Participant accrual low and the study was closed)
Phase I/II Study of CD5 CAR Engineered IL15-Transduced Cord Blood-Derived NK Cells in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapsed/Refractory Hematological Malignances [NCT05110742]Phase 1/Phase 248 participants (Anticipated)Interventional2023-11-30Not yet recruiting
ATTAMAGE-A1.: Phase I/II Study of Autologous CD8+ and CD4+ Transgenic T Cells Expressing High Affinity MAGE-A1-Specific T-Cell Receptor (TCR) Combined With Atezolizumab in Patients With Metastatic MAGE-A1 Expressing Cancer [NCT04639245]Phase 1/Phase 21 participants (Actual)Interventional2021-07-19Terminated(stopped due to Terminated due to slow accrual.)
Anti-Viral Central Memory CD8 Veto Cells in Haploidentical Hematopoietic Stem Cell Transplantation [NCT03622788]Phase 1/Phase 224 participants (Anticipated)Interventional2019-08-08Recruiting
A Phase 1/2 Study of CPX-351 (NSC# 775341) Alone Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) for Children With Relapsed Acute Myeloid Leukemia (AML) [NCT02642965]Phase 1/Phase 238 participants (Actual)Interventional2016-05-02Completed
Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia [NCT01129323]0 participants (Actual)Interventional2009-11-30Withdrawn(stopped due to This study did not accrue any subjects and due to this will be closed.)
A Phase I Trial of Ruxolitinib Combined With Tacrolimus and Sirolimus as Acute Graft-versus-Host Disease (aGVHD) Prophylaxis During Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis [NCT02528877]Phase 10 participants (Actual)Interventional2015-11-30Withdrawn(stopped due to The study design was revised so a new protocol will be opened.)
Population Pharmacokinetics of the Nucleoside Analogues Clofarabine and Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation (alloHCT) [NCT03609814]30 participants (Actual)Observational2016-01-26Completed
A Phase I Dose Escalation Trial of WT1-specific Donor-derived T Cells Following T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Relapsed/Refractory Multiple Myeloma [NCT01758328]Phase 129 participants (Anticipated)Interventional2012-12-31Active, not recruiting
A Phase-1 Trial of Adoptive Transfer of Vaccine-Primed CD3/CD28-Costimulated Autologous T-Cells Combined With Vaccine Boost and Bevacizumab for Recurrent Ovarian Fallopian Tube or Primary Peritoneal Cancer Previously Vaccinated With Autologous Tumor Vacci [NCT01312376]Phase 118 participants (Actual)Interventional2011-03-31Terminated
A Phase I Study of Adoptive Immunotherapy for Advanced B-Cell Maturation Antigen (BCMA)+ Multiple Myeloma With Autologous CD4+ and CD8+ T Cells Engineered to Express a BCMA-Specific Chimeric Antigen Receptor [NCT03338972]Phase 128 participants (Actual)Interventional2017-11-29Completed
Fludarabine, Cyclophosphamide, Rituximab and Bevacizumab in the Treatment of Relapsed Chronic Lymphocytic Leukemia [NCT00448019]Phase 264 participants (Actual)Interventional2007-02-28Completed
Multicenter, Open-label, Adaptive Design Phase I Trial With Genetically Modified T-cells Carrying Universal Chimeric Antigen Receptors (UniCAR02-T) in Combination With PSMA Peptide Target Module (TMpPSMA) for the Treatment of Patients With Progressive Dis [NCT04633148]Phase 151 participants (Anticipated)Interventional2020-11-23Recruiting
A Phase 1, Open-label, Multicenter Study to Evaluate the Safety of bb2121 in Subjects With High Risk, Newly Diagnosed Multiple Myeloma (KarMMa-4) [NCT04196491]Phase 113 participants (Actual)Interventional2020-05-27Completed
Related and Unrelated Donor Hematopoietic Stem Cell Transplant for DOCK8 Deficiency [NCT01176006]Phase 290 participants (Anticipated)Interventional2010-10-05Recruiting
Risk-ADAPTed Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation (ADAPT) [NCT06028828]Phase 260 participants (Anticipated)Interventional2023-09-11Recruiting
Pilot Open-label Trial of Nivolumab Combined With Chemotherapy in Refractory Myelodysplastic Syndromes. [NCT03259516]Phase 1/Phase 22 participants (Actual)Interventional2017-05-25Terminated(stopped due to Slow recruitment rate)
Genetically Engineered T Cells Expressing an Anti-CD19 Chimeric Antigen Receptor for Treatment of Patients With B Cell Malignancies [NCT02456350]Phase 136 participants (Anticipated)Interventional2015-04-30Recruiting
Phase II Multicentric, Randomized Trial, Exploring Intensified Rituximab Prephase Monotherapy Before Standard Fludarabine-Cyclophosphamide-Rituximab Regimen in Previously Untreated Symptomatic B-cell Chronic Lymphocytic Leukemia [NCT01370772]Phase 2140 participants (Actual)Interventional2011-05-31Completed
Phase I Study to Evaluate the Safety and Effectiveness of Anti-CD33 CAR NK Cell Therapy in Relapsed/Refractory Acute Myeloid Leukemia [NCT05008575]Phase 127 participants (Anticipated)Interventional2021-12-23Recruiting
Single Center Single Arm Clinical Prospective Study of Neoantigen Reactive T Cells (NRTs) Combined With Programmed Cell Death-1(PD-1) Inhibitor in the Treatment of Chinese Patients With Advanced Refractory Solid Tumors [NCT03171220]Phase 1/Phase 240 participants (Anticipated)Interventional2017-06-01Recruiting
Phase I/II Clinical Study to Evaluate the Safety and Efficacy of IM19 Chimeric Antigen Receptor T Cells(CAR-T) in the Treatment of Recurrent or Refractory (R/R) CD19 Positive Aggressive Non-Hodgkin's Lymphoma [NCT04440436]Phase 1/Phase 252 participants (Anticipated)Interventional2020-06-04Recruiting
Phase I Dose-Escalation Study of BCMA/CS1 Bispecific Chimeric Antigen Receptor (CAR)-T Cells for Relapsed/Refractory Multiple Myeloma [NCT05950113]Phase 130 participants (Anticipated)Interventional2024-03-28Not yet recruiting
A Randomized, Open-Label, Phase 2 Study Evaluating Lymphodepletion With ALLO-647, Fludarabine, and Cyclophosphamide, vs. Fludarabine and Cyclophosphamide Alone, in Subjects With Relapsed/Refractory Large B-Cell Lymphoma Receiving ALLO-501A Allogeneic CAR [NCT05714345]Phase 270 participants (Anticipated)Interventional2023-03-31Recruiting
A Phase 3, Randomized Study to Compare the Efficacy and Safety of Nemtabrutinib Versus Chemoimmunotherapy for Previously Untreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Without TP53 Aberrations [NCT05624554]Phase 3300 participants (Anticipated)Interventional2023-03-16Recruiting
A Phase Ib Study to Evaluate Safety and Persistence of ex Vivo Expanded Universal Donor NK Cells in Combination With IL-2 and TGFbeta Receptor I Inhibitor Vactosertib in Patients With Locally Advanced/Metastatic Colorectal Cancer and Relapsed/Refractory H [NCT05400122]Phase 112 participants (Anticipated)Interventional2022-09-09Suspended(stopped due to Insufficient staff)
A Randomized Phase 3 Trial of Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Children With Relapsed AML [NCT05183035]Phase 398 participants (Anticipated)Interventional2022-10-01Recruiting
A Phase 2 Multicenter Study Evaluating the Efficacy of KTE-X19 in Subjects With Relapsed/Refractory Mantle Cell Lymphoma [NCT04880434]Phase 290 participants (Anticipated)Interventional2021-04-27Active, not recruiting
TCRαβ+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 (Briquilimab) to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia [NCT04784052]Phase 1/Phase 212 participants (Anticipated)Interventional2021-12-07Recruiting
A Phase 1 Clinical Trial of Anti-CD19 Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Non-Hodgkin Lymphoma [NCT04545762]Phase 136 participants (Anticipated)Interventional2020-09-11Recruiting
Master Protocol to Assess the Safety and Recommended Phase 2 Dose of Next Generations of Autologous Enhanced NY-ESO-1/ LAGE-1a TCR Engineered T-cells, Alone or in Combination With Other Agents, in Participants With Advanced Tumors [NCT04526509]Phase 111 participants (Actual)Interventional2020-12-21Active, not recruiting
Allogeneic Hematopoietic Cell Transplantation With HLA-matched Donors : a Phase II Randomized Study Comparing 2 Nonmyeloablative Conditionings [NCT00603954]Phase 2107 participants (Actual)Interventional2008-01-31Completed
Clinical Study of Anti-CD19/BCMA Bispecific Chimeric Antigen Receptors (CARs) T Cell Therapy for Relapsed and Refractory Multiple Myeloma [NCT03706547]Phase 120 participants (Anticipated)Interventional2018-10-30Not yet recruiting
A Phase I, Open Label, Dose Escalation Study of ACE1702 Cell Immunotherapy in Subjects With Advanced or Metastatic HER2-expressing Solid Tumors [NCT04319757]Phase 136 participants (Anticipated)Interventional2020-05-19Recruiting
Open-Label Phase 1b/2a Clinical Trial to Assess the Safety, Tolerability and Antitumor Activity of Genetically Engineered NY-ESO-1 Specific (c259) T Cells (GSK3377794) in Combination With Anti-Cancer Agents Including Pembrolizumab in HLA-A2+ Participants [NCT03697824]Phase 20 participants (Actual)Interventional2019-02-25Withdrawn(stopped due to Internal decision, study will be replaced with a larger monotherapy trial)
Autologous Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease [NCT04224558]Phase 1/Phase 215 participants (Anticipated)Interventional2020-12-15Recruiting
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
A Phase 1 Multicenter Study of KITE-585, an Autologous Anti-BCMA CAR T-Cell Therapy, in Subjects With Relapsed/Refractory Multiple Myeloma [NCT03318861]Phase 117 participants (Actual)Interventional2017-10-20Terminated(stopped due to The study was terminated due to lack of efficacy)
A Randomized, Multi-Center, Phase III Study of Allogeneic Stem Cell Transplantation Comparing Regimen Intensity in Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia (BMT CTN #0901) [NCT01339910]Phase 3272 participants (Actual)Interventional2011-06-30Terminated(stopped due to Accrual terminated as recommended by the data and safety monitoring board.)
Purine-Alkylator Combination In Follicular Lymphoma Immuno-Chemotherapy for Older Patients: a Phase III Comparison of First-line R-CVP Versus R-FC [NCT01303887]Phase 3680 participants (Anticipated)Interventional2009-10-31Recruiting
Multi-center, Phase II Study to Assess the Safety and Efficacy of Haploidentical Bone Marrow Transplantation Using Reduced Intensity Conditioning(RIC)Regimen and Post-transplant Cyclophosphamide,in Patients With Poor Prognosis Lymphomas [NCT02049580]Phase 247 participants (Anticipated)Interventional2013-07-31Recruiting
A Phase 1, Multicenter, Open-Label, Dose Escalating Safety Study of Human Cord Blood Derived, Culture Expanded Natural Killer Cell (PNK-007) Infusion With Subcutaneous Recombinant Human IL-2 (RHIL-2) in Adults With Relapsed and/or Refractory Acute Myeloid [NCT02781467]Phase 110 participants (Actual)Interventional2016-07-11Terminated(stopped due to Business Decision)
A Pilot Study of Myeloablative Allogeneic or Haploidentical Stem Cell Transplantation With High Dose PT-Cy in Relapsed/Refractory AML [NCT02057770]Phase 125 participants (Actual)Interventional2014-02-28Terminated(stopped due to Low accrual rate)
NY-ESO-1 TCR Engineered Adoptive Cell Transfer Therapy With CTLA4 Blockade [NCT02070406]Phase 14 participants (Actual)Interventional2014-07-17Terminated(stopped due to low accrual)
A Phase 1 Study to Evaluate the Safety, Proliferation and Persistence of GEN-011, an Autologous Adoptive Cell Therapy Targeting Neoantigens in Solid Tumors [NCT04596033]Phase 149 participants (Actual)Interventional2020-11-11Terminated(stopped due to Business reasons)
Multi-center, Investigator Initiated Phase 1 Study of MAGE-A4 Specific TCR Gene Transferred T Lymphocytes With Solid Tumors [NCT02096614]Phase 118 participants (Actual)Interventional2014-04-30Completed
Phase 1 Study of FLAG-Ida With Pivekimab Sunirine (PVEK [IMGN632]) for Adults With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia and Other High-Grade Myeloid Neoplasms [NCT06034470]Phase 130 participants (Anticipated)Interventional2024-01-13Recruiting
An Open-label, Phase II, Two-stage, Study of Bisantrene(Xantrene) in Combination With Fludarabine and Clofarabine as Salvage Therapy for Adult Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) [NCT04989335]Phase 229 participants (Anticipated)Interventional2021-08-02Recruiting
A Phase I Study Evaluating Allogeneic Memory T Cells Engineered to Express Chimeric Antigen Receptors Specific for CD19 for the Treatment of Pediatric and Young Adult Patients ≤ 21 Years of Age With Relapsed or Refractory CD19-Positive Leukemia [NCT04881240]Phase 160 participants (Anticipated)Interventional2023-12-31Recruiting
Phase 1 Study of the Administration of T Lymphocytes Expressing the Kappa Chimeric Antigen Receptor (CAR) and CD28 Endodomain for Relapsed/Refractory Kappa+ Non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma. [NCT04223765]Phase 120 participants (Anticipated)Interventional2020-11-12Recruiting
A Feasibility Trial of MLN4924 (Pevonedistat, TAK 924) Given in Combination With Azacitidine, Fludarabine, and Cytarabine, in Children, Adolescents, and Young Adults With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome [NCT03813147]Phase 112 participants (Actual)Interventional2019-05-17Active, not recruiting
A Phase II Study Of Ibrutinib, Fludarabine, and Pembrolizumab in High-Risk or Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) [NCT03204188]Phase 215 participants (Actual)Interventional2017-09-22Active, not recruiting
A Phase II Study of Ofatumumab-Based Induction Chemoimmunotheraphy Followed by Consolidation Ofatumumab Immunotherapy in Previously Untreated Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma [NCT01145209]Phase 232 participants (Actual)Interventional2010-07-01Completed
Cladribine Combined With G-CSF and Cytarabine as a Salvage Treatment in Refractory/Relapsed Acute Lymphoblastic Leukemia [NCT05578378]Phase 2/Phase 332 participants (Anticipated)Interventional2022-01-01Recruiting
Population Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation [NCT01316549]67 participants (Actual)Observational2011-01-01Completed
Shortened-duration Tacrolimus Following Nonmyeloablative, Related Donor BMT With High-dose Posttransplantation Cyclophosphamide [NCT01342289]Phase 1127 participants (Actual)Interventional2011-08-31Completed
CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplant for Malignant Disease [NCT02061800]Phase 1/Phase 215 participants (Anticipated)Interventional2013-06-03Recruiting
Multicenter Phase II, Double-blind Placebo Controlled Trial of Maintenance Ixazomib After Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Multiple Myeloma (BMT CTN 1302) [NCT02440464]Phase 257 participants (Actual)Interventional2015-08-31Completed
Allogeneic Stem Cell Transplantation for Patients With Hematological Malignancies Using Multiple Unrelated Cord Blood Units [NCT00547196]10 participants (Actual)Interventional2005-08-16Active, not recruiting
A Randomized Phase II Study to Compare ATG or Post-Transplant Cyclophosphamide to Calcineurin Inhibitor-Methotrexate as GVHD Prophylaxis After Myeloablative Unrelated Donor Peripheral Blood Stem Cell Transplantation [NCT03602898]Phase 20 participants (Actual)Interventional2021-06-01Withdrawn(stopped due to Insufficient funding)
A Multiple Center Study on NOXA Expression-guided Randomized Chidamide Bridging Intervention in CAR-T Treated NHL Patients [NCT05370547]Phase 1/Phase 2120 participants (Anticipated)Interventional2022-05-25Recruiting
Phase I Trial of Escalated Doses of Targeted Marrow Irradiation (TMI) Combined With Fludarabine and Busulfan as Conditioning Regimen for Allogeneic Hematopoietic Progenitor Cell Transplantation [NCT02129582]Phase 114 participants (Actual)Interventional2014-11-05Completed
Donor-Derived Very-Rapid Manufactured CD19-Specific T Cells for Lymphoid Malignancies After Allogeneic Hematopoietic Stem-Cell Transplantation [NCT03579888]Phase 14 participants (Actual)Interventional2020-06-26Terminated(stopped due to Study halted prematurely)
A Multicenter, Open-label, Expanded Access Study of KTE-X19 for the Treatment of Subjects With Relapsed/Refractory B-Cell Malignancies [NCT04162756]0 participants Expanded AccessApproved for marketing
A Pilot Study of Genetically Engineered NY-ESO-1 Specific NY-ESO-1ᶜ²⁵⁹T in HLA-A2+ Patients With Synovial Sarcoma [NCT01343043]Phase 150 participants (Actual)Interventional2012-09-27Completed
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
Multicenter, Open-label, Phase 1 Study of Allo-RevCAR01-T-CD123 Consisting of Genetically Modified T Cells Carrying Reverse Chimeric Antigen Receptors (Allo RevCAR01 T) in Combination With CD123 Target Module (R-TM123) for the Treatment of Patients With S [NCT05949125]Phase 137 participants (Anticipated)Interventional2023-08-31Not yet recruiting
Efficacy and Safety of Autologous Cells Derived Anti-CD19 CAR-Engineered T Cells With Concurrent BTK Inhibitor for B Cell Lymphoma:a Single-center, Open-label, Pragmatic Clinical Trial [NCT05020392]Phase 324 participants (Anticipated)Interventional2021-09-14Recruiting
Modified TBF Regimen as Conditioning Regimen Prior to Allogeneic Hematopoietic Cell Transplantation for T Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma:Phase II Study [NCT05598593]Phase 270 participants (Anticipated)Interventional2022-10-23Recruiting
Observational Study of Low Dose FCR in the Treatment of Elderly/Comorbid Patients With Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma: The Q-lite Project [NCT02156726]200 participants (Anticipated)Observational2011-03-31Active, not recruiting
Nonmyeloablative Hematopoietic Cell Transplantation (HCT) for Patients With Hematologic Malignancies Using Related, HLA-Haploidentical Donors: A Pilot Trial of Peripheral Blood Stem Cells (PBSC) as the Donor Source [NCT02167958]Phase 128 participants (Actual)Interventional2015-02-11Completed
A Safety and Efficacy Study of Autologous Chimeric Antigen Receptor Engineered T Cells Redirected to EGFRvIII in Patients With Recurrent Glioblastoma Multiforme [NCT02844062]Phase 120 participants (Anticipated)Interventional2016-07-31Recruiting
A Phase I Clinical Trial of Human CD19/BCMA Bispecific CAR-T Cell Therapy for Subjects With Relapsed and Refractory POMES Syndrome. [NCT03879382]Phase 110 participants (Anticipated)Interventional2019-02-27Active, not recruiting
Clinical Study on the Safety and Efficacy of QN-030a in Acute Myeloid Leukemia [NCT05665114]Phase 118 participants (Anticipated)Interventional2022-12-24Recruiting
HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies [NCT00145626]Phase 240 participants (Actual)Interventional2004-05-31Completed
A Pilot Phase I Trial of IL-21 Expanded, Off the Shelf, Third-Party Natural Killer (NK) Cells in Combination With Mogamulizumab in Patients With Cutaneous T-Cell Lymphomas or Adult T-Cell Leukemia/Lymphomas [NCT04848064]Phase 112 participants (Anticipated)Interventional2022-05-06Recruiting
Safety and Efficacy of Ex-vivo Expanded Allogeneic γδ T-lymphocytes (OmnImmune®) in Patients With Active Relapsed or Refractory Acute Myeloid Leukaemia (AML) Who Are Not Eligible for or do Not Consent to High Dose Salvage Chemotherapy and/or Allogeneic Ha [NCT03790072]Phase 110 participants (Actual)Interventional2018-11-27Completed
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.)
A Phase II Study of Fludarabine and Rituximab for the Treatment of Marginal Zone Non-Hodgkin's Lymphoma [NCT00117156]Phase 226 participants (Actual)Interventional2003-12-31Completed
A Phase I/II Study of Fludarabine Plus Thalidomide as Frontline Therapy for Newly Diagnosed Patients With Chronic Lymphocytic Leukemia [NCT00096018]Phase 1/Phase 243 participants (Actual)Interventional2002-05-31Completed
Phase 1 and 2 Study of Combination Treatment of Bortezomib, Fludarabine and Cyclophosphamide in Patients With Recurrent Mantle Cell Lymphoma [NCT01322776]Phase 1/Phase 240 participants (Anticipated)Interventional2011-03-31Recruiting
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis [NCT01087398]Phase 2/Phase 310 participants (Anticipated)Interventional2009-09-30Recruiting
Phase II Study of Metastatic Cancer That Overexpresses p53 Using Lymphodepleting Conditioning Followed by Infusion of Anti-p53 T Cell Receptor (TCR)-Gene Engineered Lymphocytes [NCT00393029]Phase 212 participants (Actual)Interventional2006-10-31Completed
Randomized Trial of Unmanipulated Versus Expanded Cord Blood [NCT00067002]Phase 2110 participants (Actual)Interventional2003-04-30Completed
HEC73543 Versus Salvage Chemotherapy in Relapsed or Refractory FLT3-ITD Acute Myeloid Leukemia: a Multicenter, Open-label, Randomized Phase 3 Trial [NCT05586074]Phase 3324 participants (Anticipated)Interventional2023-03-03Recruiting
Total Body Irradiation/ Fludarabine/ Busulfan/ Cyclophosphamide (TFBC) Combined With Umbilical Cord Blood Transplantation (UCBT) in the Treatment of High-risk Malignant Hematological Diseases [NCT05929092]40 participants (Anticipated)Interventional2023-06-01Recruiting
Autologous TRAC Locus-inserted CD19-targeting Synthetic T-cell Receptor Antigen Receptor T (STAR-T) Cells for Relapsed/Refractory B-cell Non-Hodgkin's Lymphoma [NCT05631912]Phase 1/Phase 238 participants (Anticipated)Interventional2023-06-30Recruiting
A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy of Axicabtagene Ciloleucel Versus Standard of Care Therapy in Subjects With Relapsed/Refractory Diffuse Large B Cell Lymphoma [NCT03391466]Phase 3359 participants (Actual)Interventional2018-01-25Active, not recruiting
A Phase II Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes [NCT03121014]Phase 238 participants (Anticipated)Interventional2017-04-24Recruiting
A Phase 3 Multicenter, Randomized, Prospective, Open-label Trial of Standard Chemoimmunotherapy (FCR/BR) Versus Rituximab Plus Venetoclax (RVe) Versus Obinutuzumab (GA101) Plus Venetoclax (GVe) Versus Obinutuzumab Plus Ibrutinib Plus Venetoclax (GIVe) in [NCT02950051]Phase 3926 participants (Actual)Interventional2016-12-13Active, 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
Phase II Trial Of Non-Myeloablative Regimen Combining Melphalan, Fludarabine, And Anti-CD52 Monoclonal Antibody (CAMPATH-1H) Followed By An Unmodified Hematopoietic Cell Transplant From An HLA Compatible Related Or Unrelated Donor For Treatment Of Lymphoh [NCT00027560]Phase 251 participants (Actual)Interventional2001-07-31Completed
Phase IIa Study of Addition of Itacitinib With Tacrolimus/Sirolimus Regimen for GVHD Prophylaxis in Fludarabine and Melphalan Non-Myeloablative Conditioning Hematopoietic Cell Transplantation for Acute Leukemias, MDS or MF [NCT04339101]Phase 259 participants (Actual)Interventional2020-11-11Active, not recruiting
Clinical Study to Assess Efficacy and Safety of MDA-TIL (Autologous Expanded Tumor Infiltrating Lymphocytes) Across Multiple Tumor Types [NCT03610490]Phase 227 participants (Actual)Interventional2018-08-17Active, not recruiting
Allogeneic Transplantation Using Venetoclax, Timed Sequential Busulfan,Cladribine, and Fludarabine Conditioning in Patients With AML and MDS [NCT02250937]Phase 2116 participants (Anticipated)Interventional2014-10-27Active, not recruiting
A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Hematologic Malignancies [NCT04282174]Phase 20 participants (Actual)Interventional2022-09-30Withdrawn(stopped due to Study was split into two new studies before the first participant was enrolled.)
A Phase I Study to Examine the Toxicity of Allogeneneic Stem Cell Transplantation for Relapsed or Therapy Refractory Ewings [NCT02472392]Phase 110 participants (Anticipated)Interventional2013-04-30Completed
Novel BCMA-targeted CAR-T Cell Therapy for Multiple Myeloma [NCT04706936]Phase 125 participants (Anticipated)Interventional2021-04-01Recruiting
A Phase I Safety and Feasibility Study of Cellular Immunotherapy for Extensive Stage Small Cell Neuroendocrine Prostate Cancer Using Autologous T Cells Lentivirally Transduced to Express L1CAM-Specific Chimeric Antigen Receptor [NCT06094842]Phase 120 participants (Anticipated)Interventional2024-03-01Not yet recruiting
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
Phase 1 Trial for Patients With Advanced Hematologic Malignancies Undergoing Reduced Intensity Allogeneic HCT With a T-cell Depleted Graft With Infusion of Conventional T-cells and Regulatory T-cells [NCT05088356]Phase 140 participants (Anticipated)Interventional2021-09-07Recruiting
Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases [NCT04965597]Phase 240 participants (Anticipated)Interventional2022-04-19Recruiting
Phase Ib Clinical Trial of Autologous CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies [NCT04088864]Phase 152 participants (Anticipated)Interventional2020-01-10Suspended(stopped due to Business decision)
A Pilot Phase II Study Using Ibrutinib and Short-Course Fludarabine in Previously Untreated Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) [NCT02514083]Phase 229 participants (Actual)Interventional2015-12-09Active, not recruiting
Phase II Study of MK-3475 in Conjunction With Lymphodepletion, TIL, and High or Low Dose IL-2 in Patients With Metastatic Melanoma [NCT02500576]Phase 218 participants (Actual)Interventional2015-08-07Completed
Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia [NCT00737685]Phase 230 participants (Anticipated)Interventional2006-01-31Active, not recruiting
Phase II Trial of LMB-2, Fludarabine and Cyclophosphamide for Adult T-Cell Leukemia [NCT00924170]Phase 1/Phase 218 participants (Actual)Interventional2008-10-31Completed
CBA Versus FBA Conditioning Followed by Allogeneic HSCT in Treatment of High Risk and Refractory AML [NCT03384212]Phase 3120 participants (Anticipated)Interventional2016-08-01Recruiting
Phase I Study of Malignancies That Express NY-ESO-1 With T Cell Receptor-transduced T Cells Targeting NY-ESO-1 [NCT02457650]Phase 136 participants (Anticipated)Interventional2015-04-30Recruiting
Pilot Study of Autologous Anti-CD19 4-1BB CAR T Cells With Cell-intrinsic PD1 Inhibition in Relapsed or Refractory B-cell Lymphoma [NCT03208556]Phase 120 participants (Anticipated)Interventional2017-06-21Recruiting
Decitabine+ Fludarabine+Busulfan Conditioning Regimen for Elderly Acute Myeloid Leukemia in Complete Remission Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT03530085]Phase 2/Phase 360 participants (Anticipated)Interventional2018-06-15Recruiting
3RD GENERATION GD2 SPECIFIC CHIMERIC ANTIGEN RECEPTOR AND INDUCIBLE CASPASE 9 SAFETY SWITCH TRANSDUCED AUTOLOGOUS NATURAL KILLER T-CELLS TO TREAT CHILDREN WITH NEUROBLASTOMA (GINAKIT) [NCT02439788]Phase 10 participants (Actual)Interventional2017-08-31Withdrawn(stopped due to Based on newly available preclinical data we changed the CAR construct to a more effective version and will now study that product on a different protocol.)
An Open-Label, Multinational, Multicenter, Phase IIIB Study to Assess Safety of Rituximab Following Subcutaneous Administration in Patients With CD20+ DLBCL or CD20+ Follicular NHL Grade 1 to 3A [NCT02406092]Phase 3122 participants (Actual)Interventional2015-10-13Completed
A PHASE III, MULTICENTRE, RANDOMIZED, OPEN LABEL CLINICAL TRIAL OF AZACYTIDINE (VIDAZA®) VERSUS FLUDARABINE AND CYTARABINE (FLUGA SCHEME) IN ELDERLY PATIENTS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA. [NCT02319135]Phase 3289 participants (Actual)Interventional2014-10-31Completed
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies [NCT01652092]30 participants (Anticipated)Interventional2012-09-04Recruiting
A Phase 1/2 Study of Cytokine-Induced Memory-Like NK Cells in Patients With AML or MDS [NCT01898793]Phase 1/Phase 289 participants (Actual)Interventional2014-08-11Terminated(stopped due to Insufficient funding/staff)
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
The Safety,Tolerance and Efficacy of Neoantigen Targeting T Cells Suspension for Intravenous Infusion(Neo-T) to Advanced Solid Tumor [NCT05798546]Phase 121 participants (Anticipated)Interventional2022-09-28Recruiting
Prospective, Single-center, Single-arm, Open-label Study of Obinutuzumab, Zanubrutinib and Lenalidomide Sequential CD19/CD22 CAR-T in Patients With Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma [NCT05797948]20 participants (Anticipated)Interventional2022-07-01Enrolling by invitation
Phase I/II Study of CAR.70-engineered IL15-transduced Cord Blood-derived NK Cells in Conjunction With Lymphodepleting Chemotherapy for the Management of Advanced Renal Cell Carcinoma, Mesothelioma and Osteosarcoma [NCT05703854]Phase 1/Phase 250 participants (Anticipated)Interventional2023-03-29Recruiting
A Phase 2 Study of Cytokine-induced Memory-like NK Cells in Combination With Chemotherapy in Pediatric Patients With Refractory or Relapsed AML [NCT04354025]Phase 20 participants (Actual)Interventional2023-06-30Withdrawn(stopped due to Principal investigator decided not to move forward with the study.)
A Phase II Study of Dasatinib (Sprycel®) (NSC #732517) as Primary Therapy Followed by Transplantation for Adults >/= 18 Years With Newly Diagnosed Ph+ Acute Lymphoblastic Leukemia by CALGB, ECOG and SWOG [NCT01256398]Phase 266 participants (Actual)Interventional2010-12-14Completed
Multi-center, Investigator Initiated Phase 1 Study of NY-ESO-1 Specific TCR Gene Transferred T Lymphocytes With Solid Tumors [NCT02366546]Phase 19 participants (Actual)Interventional2015-03-31Active, not recruiting
A Pilot Study of EBV-TCR-T(YT-E001) in NPC Patients [NCT03648697]Phase 220 participants (Anticipated)Interventional2018-10-10Recruiting
CD19+ Chimeric Antigen Receptor T Cells for Patients With Advanced Lymphoid Malignancies [NCT02529813]Phase 126 participants (Actual)Interventional2015-12-16Completed
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
A 5 Day Course of Fludarabine and Cytarabine Followed by Full Intensity Allogeneic Stem Cell Transplantation (FA5-Bucy) in Treating Patients With High-risk, Recurrent or Refractory Acute Leukemia and Advanced Myelodysplastic Syndrome [NCT02328950]50 participants (Anticipated)Observational2014-12-31Recruiting
Safety and Efficacy Evaluation of IM19 CAR-T Cells on Refractory or Relapsed B-ALL Patients [NCT03142646]Phase 1/Phase 260 participants (Anticipated)Interventional2016-08-30Recruiting
Risk Stratified Treatment for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia: A Phase I/II Non-randomized Study of Trametinib and Azacitidine With or Without Chemotherapy (IND #164058) [NCT05849662]Phase 1/Phase 258 participants (Anticipated)Interventional2023-11-30Not yet recruiting
Phase 1b/2a Trial of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) From an HLA-partially Matched Related or Unrelated Donor After TCRαβ+ T-cell/CD19+ B-cell Depletion for Patients Who Will Receive a Kidney Transplant (KT) From the Same HSCT/KT [NCT05508009]Phase 1/Phase 212 participants (Anticipated)Interventional2023-01-10Recruiting
Randomized Study Comparing i.v. Busulfan (Busilvex®) Plus Fludarabine (BuFlu) Versus Busilvex® Plus Cyclophosphamide (BuCy2) as Conditioning Regimens Prior AlloHSCT in Patients (Age >= 40 and =<65 Years) With AML in Complete Remission. [NCT01191957]Phase 3252 participants (Actual)Interventional2008-01-31Completed
Phase I/II Study of CAR.70- Engineered IL15-transduced Cord Blood-derived NK Cells in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse/Refractory Hematological Malignances [NCT05092451]Phase 1/Phase 294 participants (Anticipated)Interventional2022-11-01Recruiting
A Phase II, Open-label, Single Arm Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Twice Daily Midostaurin (PKC412) Combined With Standard Chemotherapy and as a Single Agent Post-consolidation Therapy in Children With Untreated FLT3-mutate [NCT03591510]Phase 223 participants (Anticipated)Interventional2019-03-13Recruiting
Co-stimulation With Ipilimumab to Enhance Lymphodepletion Plus Adoptive Cell Transfer and High Dose IL-2 in Patients With Metastatic Melanoma [NCT01701674]13 participants (Actual)Interventional2012-10-09Active, not recruiting
Safety and Preliminary Efficacy of JK500 Cell Injection in Relapsed/Refractory Pediatric Acute Myeloid Leukemia [NCT05519384]Early Phase 112 participants (Anticipated)Interventional2022-09-14Recruiting
A Randomized Placebo-controlled Phase II Trial of Irradiated, Adenovirus Vector Transferred GM-CSF Secreting Autologous Leukemia Cell Vaccination (GVAX) Versus Placebo Vaccination in Patients With Advanced MDS/AML After Allogeneic Hematopoietic Stem Cell [NCT01773395]Phase 2123 participants (Actual)Interventional2013-01-08Terminated(stopped due to Recommendation by the Data and Safety Monitoring Board due to efficacy concerns)
Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome [NCT00357565]Phase 220 participants (Anticipated)Interventional2005-11-30Recruiting
An Investigator Sponsored Phase I Trial of Selinexor (KPT-330) Plus FLAG-Ida for the Treatment of Relapsing/Refractory AML [NCT03661515]Phase 116 participants (Actual)Interventional2018-07-17Completed
Immunotherapy Using Precision T Cells Specific to Personalized Neo-antigen for the Treatment of Advanced Solid Tumor [NCT03658785]Phase 1/Phase 240 participants (Anticipated)Interventional2018-12-10Not yet recruiting
A Phase 1 Study to Evaluate TAG72-Targeting Chimeric Antigen Receptor (CAR) T Cells in Patients With Advanced Epithelial Ovarian Cancer [NCT05225363]Phase 133 participants (Anticipated)Interventional2022-05-05Recruiting
A Phase 2 Clinical Trial to Evaluate the Efficacy of Zanubrutinib Followed Zanubrutinib Plus FCR (Fludarabine Cyclophosphamide and Rituximab) / BR(Bendamustine and Rituximab) in Newly Diagnosed Symptomatic CLL/SLL (STOP Trial) [NCT05287984]Phase 263 participants (Anticipated)Interventional2022-03-22Not yet recruiting
A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate the Safety and Clinical Activity of PBCAR20A in Subjects With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) or r/r Chronic Lymphocytic Leukemia (CLL) o [NCT04030195]Phase 1/Phase 218 participants (Actual)Interventional2020-03-24Completed
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
A Single Center, Open Label, Single Arm Exploratory Clinical Study of CD19-Directed Allogeneic Chimeric Antigen Receptor CART-cell Immunotherapy Cell Therapy in Pediatric Patients With Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia [NCT04173988]Early Phase 16 participants (Anticipated)Interventional2020-01-09Recruiting
Glypican 3-specific Chimeric Antigen Receptor Expressed in Autologous T Cells as Immunotherapy for Patients With Pediatric Solid Tumors [NCT02932956]Phase 110 participants (Actual)Interventional2018-12-17Active, not recruiting
The Safety and Efficacy of Double-target CART-19 and 20 Cells in Relapse and Refractory Patients With CD19+/CD20+ Non-Hodgkin's Lymphoma (NHL) [NCT06160362]20 participants (Anticipated)Interventional2023-10-31Recruiting
A Study to Infuse ROR1-Specific Autologous T Cells for Patients With Chronic Lymphocytic Leukemia (CLL) / Small Lymphocytic Lymphoma (SLL) [NCT02194374]Phase 10 participants (Actual)Interventional2015-01-31Withdrawn(stopped due to Study closed with no enrollment due to unavailability of reagent.)
Fludarabine-Based Conditioning for Matched Related Donor Bone Marrow Transplantation in Patients With Bone Marrow Failure Syndromes [NCT02928991]Early Phase 175 participants (Anticipated)Interventional2015-04-30Recruiting
Glypican 3-specific Chimeric Antigen Receptor Expressing T Cells as Immunotherapy for Patients With Hepatocellular Carcinoma [NCT02905188]Phase 19 participants (Actual)Interventional2019-03-28Completed
Anti-CD22 Immunoconjugate Inotuzumab Ozogamicin (CMC-544) Added to Fludarabine, Bendamustine and Rituximab and Allogeneic Transplantation for CD22 Positive-Lymphoid Malignancies [NCT01664910]Phase 1/Phase 227 participants (Actual)Interventional2012-10-29Completed
Multi-Institutional Prospective Pilot Study of Lupron to Enhance Lymphocyte Immune Reconstitution Following Allogeneic Bone Marrow Transplantation in Post-Pubertal Children and Adults With Molecular Imaging Evaluation [NCT01338987]Phase 276 participants (Actual)Interventional2011-04-19Completed
Phase I Clinical Trial of Human AntiCD19 Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Lymphoid Malignancies (Non-Hodgkin Lymphoma, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia) [NCT04732845]Phase 136 participants (Anticipated)Interventional2021-04-26Recruiting
Phase I Clinical Trial of AntiCD19 Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Non Hodgkin Lymphoma [NCT03434769]Phase 131 participants (Actual)Interventional2018-07-09Active, not recruiting
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
A Phase 1/2 Trial of Uproleselan Combined With High Dose Busulfan Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for Patients With Chemotherapy Resistant Acute Myeloid Leukemia [NCT05569512]Phase 1/Phase 228 participants (Anticipated)Interventional2022-10-06Recruiting
PNOC018 A Phase 1 Clinical Trial of Autologous T Cells Expressing a TCR Specific for H3.3K27M With Inhibition of Endogenous TCR (KIND T Cells) in HLA-A*0201-positive Participants With Newly Diagnosed H3.3K27M-positive Diffuse Midline Gliomas [NCT05478837]Phase 112 participants (Anticipated)Interventional2023-07-20Recruiting
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)
Phase I Study of Adoptive Immunotherapy With CD8+ and CD4+ Memory T Cells Transduced to Express an HA-1-Specific T Cell Receptor (TCR) for Children and Adults With Recurrent Acute Leukemia After Allogeneic Hematopoietic Stem Cell Transplantation (HCT) [NCT03326921]Phase 124 participants (Anticipated)Interventional2018-02-23Suspended(stopped due to Pause in funding)
Phase 1 Dose Escalation and Expansion Study of TROP2 CAR Engineered IL15-transduced Cord Blood-derived NK Cells in Patients With Advanced Solid Tumors (TROPIKANA) [NCT06066424]Phase 154 participants (Anticipated)Interventional2023-10-24Recruiting
International Collaborative Treatment Protocol for Children and Adolescents With Acute Lymphoblastic Leukemia - AIEOP-BFM ALL 2017 [NCT03643276]Phase 35,000 participants (Anticipated)Interventional2018-07-15Recruiting
A Randomized Trial for Adults With Newly Diagnosed Acute Lymphoblastic Leukemia [NCT01085617]Phase 31,033 participants (Actual)Interventional2010-12-31Active, not recruiting
Decitabine-primed Tandem Targeting CD19 and CD20 Chimeric Antigen Receptor T Cells Treatment in Relapsed and/or Refractory Non-Hodgkin's Lymphoma Patients [NCT04697940]Phase 1/Phase 233 participants (Anticipated)Interventional2020-12-15Recruiting
A Phase 1 Trial of CIML NK Cell Infusion for Myeloid Disease Relapse After Hematopoietic Cell Transplantation [NCT04024761]Phase 150 participants (Anticipated)Interventional2019-08-31Active, not recruiting
A Phase 2, Single-Center, Open Label Study of Autologous, Adoptive Cell Therapy Following a Reduced Intensity, Non-myeloablative, Lymphodepleting Induction Regimen in Metastatic Ovarian [NCT03412526]Phase 215 participants (Anticipated)Interventional2018-01-21Recruiting
Indoleamine-2,3-dioxygenase (IDO) Inhibition With INCB024360 and Intraperitoneal Delivery of Allogeneic Natural Killer Cells for Women With Recurrent Ovarian, Fallopian Tube, and Primary Peritoneal Cancer [NCT02118285]Phase 12 participants (Actual)Interventional2014-07-28Completed
Intra-osseous Co-transplant of Cord Blood and Mesenchymal Stromal Cells: A Feasibility Study [NCT02181478]Early Phase 16 participants (Actual)Interventional2015-07-22Completed
Randomized Phase III Trial Evaluating the Role of Autologous Stem Cell Transplantation in Previously Untreated Patients With Stage B and C Chronic Lymphocytic Leukemia [NCT00931645]Phase 3241 participants (Actual)Interventional2001-04-30Completed
A Pilot Study of EPOCH-F/R Induction Chemotherapy and Reduced-Intensity, HLA-Matched, Related Allogeneic Hematopoietic Stem Cell Transplantation, With Cyclosporine & Methotrexate GVHD Prophylaxis for Refractory or Relapsed Hematologic Malignancies [NCT00051311]Phase 262 participants (Actual)Interventional2003-01-03Completed
Pilot Study of Allogeneic/Syngeneic Blood Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas [NCT00043979]Phase 260 participants (Actual)Interventional2002-09-19Completed
Pilot Study Of Allogeneic Peripheral Blood Progenitor Cell Transplantation In Patients With Chemotherapy-Refractory Or Poor-Prognosis Metastatic Breast Cancer [NCT00074269]Phase 25 participants (Actual)Interventional2003-07-31Terminated(stopped due to Terminated early due to poor enrollment)
HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin [NCT01105273]Phase 1/Phase 212 participants (Actual)Interventional2009-07-31Completed
An Open-Label Phase I/II Study of Autologous Tumor-Draining Lymph Node-Derived Lymphocytes as Neoadjuvant Therapy for HER2-Negative Breast Cancer [NCT05981014]Phase 1/Phase 2196 participants (Anticipated)Interventional2023-08-01Recruiting
An Open-Label Phase I/II Study of Autologous Tumor-Draining Lymph Node-Derived Lymphocytes for Advanced HER2-Negative Breast Cancer [NCT05981001]Phase 1/Phase 2170 participants (Anticipated)Interventional2023-08-01Recruiting
Allogeneic Hematopoietic Cell Transplantation With Cyclophosphamide, Fludarabine, and Antithymocyte Globulin in Lower Risk Myelodysplastic Syndrome Phase 2 Extension Study [NCT06098326]Phase 230 participants (Anticipated)Interventional2018-03-06Active, not recruiting
Clinical Study of Universal Off-the-shelf Cell Products in Patients With CD19-positive Relapsed/Refractory B-cell Hematolymphatic Malignancies. [NCT06092047]Early Phase 110 participants (Anticipated)Interventional2023-10-31Not yet recruiting
A Dose-escalation Clinical Study of QH103 Cell Injection (CD19 CAR-γδT Cell Injection) in Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia (B-ALL). [NCT06056752]Phase 110 participants (Anticipated)Interventional2023-09-27Recruiting
Multiple-center Randomized Study to Compare Fludarabine and Busulfan Versus Fludarabine, Busulfan and Melphalan in Adult Patients With Acute Myeloid Leukemia (AML) and Myelodysplasia Syndrome (MDS) [NCT05991908]Phase 3222 participants (Anticipated)Interventional2023-10-19Recruiting
A Prospective Study of Tocilizumab for the Prevention of Graft Failure and Graft-versus-Host Disease in Haplo-Cord Transplantation [NCT04395222]Phase 221 participants (Actual)Interventional2020-10-07Active, not recruiting
B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR) [NCT04897321]Phase 132 participants (Anticipated)Interventional2022-07-06Recruiting
Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T Cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG) [NCT04196413]Phase 154 participants (Anticipated)Interventional2020-06-04Recruiting
Fludarabine/Clofarabine/Busulfan Combined With SAHA in Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation for Acute Leukemia [NCT02083250]Phase 170 participants (Actual)Interventional2014-03-06Completed
Phase 2 Study of Autologous Tumor Infiltrating Lymphocytes (LN-145) With Pembrolizumab, in Subjects Who Have Failed Cisplatin-Based Chemotherapy With Locally Advanced (Unresectable) or Metastatic Transitional Cell Cancer (TCC) of the Urothelium [NCT03935347]Phase 20 participants (Actual)Interventional2019-06-20Withdrawn(stopped due to no accrual)
A Clinical Study to Evaluate the Safety and Efficacy of BCMA-GPRC5D CAR-T in Patients With R/R MM Who Received Three or More Lines of Therapy [NCT05998928]Phase 210 participants (Anticipated)Interventional2023-07-27Recruiting
A Phase 1, Multicenter, Open-Label Study of CB-010, a CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy in Patients With Relapsed/Refractory B Cell Non-Hodgkin Lymphoma (ANTLER) [NCT04637763]Phase 172 participants (Anticipated)Interventional2021-05-26Recruiting
A Phase 1 Study of Adding Venetoclax to a Reduced Intensity Conditioning Regimen and to Maintenance in Combination With a Hypomethylating Agent After Allogeneic Hematopoietic Cell Transplantation for Patients With High Risk AML, MDS, and MDS/MPN Overlap S [NCT03613532]Phase 178 participants (Anticipated)Interventional2018-10-24Recruiting
A Phase 1 Study to Evaluate the Safety and Tolerability of a Combination Autologous CD19 CAR T Cell Therapy (SYNCAR-001 + STK-009) in Subjects With Relapsed or Refractory CD19+ Hematologic Malignancies [NCT05665062]Phase 136 participants (Anticipated)Interventional2022-06-24Recruiting
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
Itacitinib to Prevent Graft Versus Host Disease [NCT04859946]Phase 130 participants (Anticipated)Interventional2022-01-11Recruiting
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
An Assessment of the Safety and Feasibility of Administering T-Cells Expressing an Anti-CD19 Chimeric Antigen Receptor to Patients With B-Cell Lymphoma [NCT00924326]Phase 1/Phase 243 participants (Actual)Interventional2009-02-17Completed
Phase I Adoptive Cellular Therapy Trial With Endogenous CD8+ T Cells (ACTolog® IMA101) Alone or in Combination With Atezolizumab in Patients With Relapsed and/or Refractory Solid Cancers [NCT02876510]Phase 138 participants (Actual)Interventional2017-06-30Completed
Clinical Study of Redirected Autologous T Cells With a BCMA-targeted Chimeric Antigen Receptor in Patients With Refractory or Relapsed Multiple Myeloma [NCT03716856]Phase 111 participants (Actual)Interventional2018-03-23Active, not recruiting
A Randomized, Open-Label, Multicenter Phase 2 Study of VELCADE With Fludarabine in Comparison to Rituximab With Fludarabine in Follicular Lymphoma Patients Previously Treated With Rituximab [NCT00850499]Phase 212 participants (Actual)Interventional2009-09-30Terminated
A Phase II Study of Fludarabine Induction With Sequential High Dose Cyclophosphamide and Rituximab as Consolidation Therapy for Previously Untreated Patients With Intermediate and High-Risk Chronic Lymphocytic Leukemia [NCT00003659]Phase 239 participants (Actual)Interventional1998-09-30Completed
Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation in Patients With Multiple Sclerosis [NCT02529839]20 participants (Anticipated)Interventional2015-10-31Not yet recruiting
Phase IB Followed by Phase II Study of Trastuzumab Combined With Autologous Chimeric Receptor T Cells in HER2+ Advanced Breast Cancer and Other Solid Tumors [NCT06027983]Phase 1/Phase 236 participants (Anticipated)Interventional2023-11-01Not yet recruiting
Umbilical Cord Blood Transplant for Congenital Pediatric Disorders [NCT00950846]40 participants (Actual)Interventional2009-09-30Completed
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for B-Cell Non-Hodgkin Lymphoma Using Zevalin, Fludarabine and Melphalan [NCT00577278]Phase 241 participants (Actual)Interventional2007-10-03Active, not recruiting
A Pilot Trial of the Combination of Vemurafenib With Adoptive Cell Therapy in Patients With Metastatic Melanoma [NCT01585415]Phase 112 participants (Actual)Interventional2012-04-09Terminated
A Cancer Research UK Phase I Trial of Adoptive Transfer of Autologous Tumor Antigen-Specific T Cells With Preconditioning Chemotherapy and Intravenous IL2 in Patients With Advanced CEA Positive Tumors [NCT01212887]Phase 114 participants (Actual)Interventional2007-08-31Terminated(stopped due to due to safety concerns and lack of efficacy)
A Randomized Study of Once Daily Fludarabine-Clofarabine Versus Fludarabine Alone Combined With Intervenous Busulfan Followed by Allogeneic Hemopoietic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) [NCT01471444]Phase 3256 participants (Actual)Interventional2011-11-02Completed
Safety and Efficacy of Anti-CD7 CAR-Engineered T Cells for Relapsed/Refractory T Lymphoid Malignancies: a Single-center, Open-label, Non-randomized, Single-arm Clinical Study [NCT04823091]Phase 124 participants (Anticipated)Interventional2021-04-15Recruiting
Clinical Study on the Safety and Efficacy of QN-023a Targeting CD33 in Acute Myeloid Leukemia [NCT05665075]Phase 119 participants (Anticipated)Interventional2022-12-24Recruiting
The Clinical Research of Fourth Generation CART-cell Therapy in Refractory-Relapsed Ovarian Cancer [NCT03814447]Early Phase 110 participants (Anticipated)Interventional2019-08-16Recruiting
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 Phase II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation From Family Haploidentical Donors in Patients With Myelodysplastic Syndrome and Acute Leukemia Under Primary Antifungal Prophylaxis With Posaconazole. [NCT03434704]Phase 210 participants (Actual)Interventional2018-06-18Completed
Safety and Effectiveness of MESO-CAR T Cells Therapy for Relapsed and Refractory Epithelial Ovarian Cancer [NCT03916679]Phase 1/Phase 220 participants (Anticipated)Interventional2019-04-20Recruiting
A Phase 2 Study of Cytokine-induced Memory-like NK Cells in Relapsed/Refractory AML and MDS [NCT04893915]Phase 20 participants (Actual)Interventional2022-06-30Withdrawn(stopped due to No funding support)
Phase 1 Trial of Autologous T Cells Engineered to Express NY-ESO-1 TCR and CRISPR Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T Cells) [NCT03399448]Phase 13 participants (Actual)Interventional2018-09-05Terminated(stopped due to Sponsor has terminated trial to pursue other targets.)
A Phase I/II Study of the Selective Inhibitor of Nuclear Export Selinexor (KPT-330) in Combination With Fludarabine and Cytarabine in Patients With Refractory or Relapsed Leukemia or Myelodysplastic Syndrome [NCT03071276]Phase 237 participants (Actual)Interventional2016-01-14Terminated(stopped due to Due to slow enrollment)
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia [NCT02162420]50 participants (Anticipated)Interventional2015-01-10Recruiting
Phase I Study of Valproic Acid Expanded Cord Blood Stem Cells as an Allogeneic Donor Source for Adults With Hematological Malignancies [NCT03885947]Phase 17 participants (Actual)Interventional2019-02-21Completed
Optimization of the T Regulatory Cell and T Effector Cell Doses in Recipients of Double UCB Transplantation for Treatment of Hematological Malignancies [NCT01163201]Phase 1/Phase 20 participants (Actual)Interventional2014-01-31Withdrawn(stopped due to Replaced by a new study)
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
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
A Phase I Clinical Trial Using Genetically Engineered Autologous T Cells to Express Chimeric Antigen Receptor (CAR) for Treatment of Patients With Refractory or Relapsed CD19-positive B Lymphoid Malignancies [NCT05705570]Phase 130 participants (Anticipated)Interventional2023-03-01Not yet recruiting
A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies [NCT00423826]0 participants Expanded Access2007-01-31No longer available
A Clinical Trial of MESO-CAR T Cells Therapy for Relapsed and Refractory Ovarian Cancer [NCT03799913]Early Phase 120 participants (Anticipated)Interventional2019-04-10Recruiting
A Phase I Trial for the Evaluation of the In Vivo Persistence of Adoptively-transferred Tumor-Infiltrating Lymphocytes Cultured With a Pharmacologic Inhibitor of AKT in Patients With Metastatic Melanoma [NCT02489266]Phase 10 participants (Actual)Interventional2015-06-24Withdrawn
Multi-center, Open-label, Phase 1/2a Clinical Trial to Evaluate the Efficacy and Safety of Combination Therapy With MG4101 Plus Rituximab in Patient With Relapsed/Refractory Non-Hodgkin's Lymphoma of B-cell Origin [NCT03778619]Phase 1/Phase 29 participants (Actual)Interventional2018-11-28Active, not recruiting
T Cell Therapy for Patients With Metastatic Ovarian Cancer [NCT02482090]Phase 16 participants (Actual)Interventional2015-07-31Completed
A Phase 1 Study of Obatoclax Mesylate (GX15-070MS) in Combination With Fludarabine-Based Chemoimmunotherapy in Previously Treated Patients With B-Cell Chronic Lymphocytic Leukemia (B-CLL) [NCT00612612]Phase 128 participants (Actual)Interventional2008-01-31Terminated
Intentional Rejection of the Donor Graft Using Recipient Leukocyte Infusion(s) Following Nonmyeloablative Allogeneic Stem Cell Transplant [NCT00909948]Phase 17 participants (Actual)Interventional2008-11-30Terminated(stopped due to Slow Accrual)
Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults With Hematological Malignancies [NCT05735717]Phase 2150 participants (Anticipated)Interventional2023-05-11Recruiting
GD2-CAR PERSIST: Production and Engineering of GD2-Targeted, Receptor Modified T Cells (GD2CART) for Osteosarcoma or Neuroblastoma to Increase Systemic Tumor Exposure [NCT04539366]Phase 167 participants (Anticipated)Interventional2022-01-25Suspended(stopped due to Interim Monitoring)
Open-Label Pilot Study to Assess the Safety, Tolerability and Antitumor Activity of Genetically Engineered NY-ESO-1 Specific (c259) T Cells Alone or in Combination With Pembrolizumab in HLA-A2+ Subjects With NY-ESO-1 and/or LAGE-1a Positive Relapsed and R [NCT03168438]Phase 16 participants (Actual)Interventional2017-08-18Terminated(stopped due to The study was terminated following an internal review of the company's research and development portfolio)
Feasibility Study on Allogeneic Hematopoietic Stem Cell Transplantation Following Fludarabine-Busulfan-ALS-based Reduced-intensity Conditioning in Children With Hematological Malignancy or Solid Tumor Not Responding to Standard Therapy or for Which the In [NCT00750126]Phase 230 participants (Anticipated)Interventional2007-04-30Recruiting
A Phase I-II Study of Allogeneic CMV Specific Cytotoxic T Lymphocytes (CTL) for Patients With Refractory Glioblastoma Multiforme (GBM) [NCT00990496]Phase 125 participants (Actual)Interventional2009-09-30Terminated(stopped due to Accrual goals not met)
A Phase II Study of Twice Daily Cytarabine and Fludarabine in Acute Myelogenous Leukemia and High-Risk Myelodysplastic Syndrome [NCT01019317]Phase 2151 participants (Actual)Interventional2009-11-30Completed
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
Phase I/II Study of Metastatic Melanoma Using Lymphodepleting Conditioning Followed by Infusion of Tumor Infiltrating Lymphocytes Genetically Engineered to Express IL-12 [NCT01236573]Phase 1/Phase 234 participants (Actual)Interventional2010-10-31Terminated(stopped due to Unexpected toxicities, likely due to TIL/IL-12 & low % of durable responses.)
Phase I/II Study of Metastatic Cancer Using Lymphodepleting Conditioning Followed by Infusion of Anti-VEGFR2 Gene Engineered CD8+ Lymphocytes [NCT01218867]Phase 1/Phase 224 participants (Actual)Interventional2010-11-10Terminated(stopped due to No objective responses were observed.)
Nonmyeloablative Allogeneic Stem Cell Transplant for the Treatment of Hematologic Disorders [NCT00636909]Phase 225 participants (Actual)Interventional1999-07-31Completed
Fludarabine, Mitoxantrone, and Dexamethasone (FND) Plus Chimeric Anti-CD20 Monoclonal Antibody (Rituximab) for Stage IV Indolent Lymphoma [NCT00577993]Phase 3210 participants (Actual)Interventional1998-03-16Completed
T Cells co- Expressing a Second Generation Glypican 3-specific Chimeric Antigen Receptor With Cytokines Interleukin-21 and 15 as Immunotherapy for Patients With Liver Cancer [NCT04093648]Phase 10 participants (Actual)Interventional2020-01-31Withdrawn(stopped due to The key elements of this study were incorporated into another study.)
Fludarabine/Rituximab Combined With Escalating Doses of Lenalidomide Followed by Rituximab/Lenalidomide in Untreated Chronic Lymphocytic Leukemia (CLL) - a Dose-finding Study With Concomitant Evaluation of Safety and Efficacy. [NCT00738829]Phase 1/Phase 245 participants (Actual)Interventional2008-10-31Completed
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
Axicabtagene Ciloleucel in Relapsed or Refractory HIV-Associated Aggressive B-Cell Non-Hodgkin Lymphoma [NCT05077527]Phase 120 participants (Anticipated)Interventional2024-01-15Not yet recruiting
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
A Phase II Study of Sirolimus, Tacrolimus and Thymoglobulin®, as Graft-versus-Host- Disease Prophylaxis in Patients Undergoing Unrelated Donor Hematopoietic Cell Transplantation [NCT00691015]Phase 248 participants (Actual)Interventional2008-05-31Completed
Allogeneic Hematopoietic Cell Transplant for Hematological Cancers and Myelodysplastic Syndromes in HIV-Infected Individuals (BMT CTN #0903) [NCT01410344]Phase 220 participants (Actual)Interventional2011-09-30Completed
Trial of the Efficacy and Safety of High-dose Immunosuppressive Therapy Based on Fludarabine and Cyclophosphamide-containing Conditioning Regimen Followed by Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple Sclerosis. [NCT05832515]Phase 1200 participants (Anticipated)Interventional2020-10-01Recruiting
Phase II Study of Allogeneic Transplant of Hematopoietic Stem Cells From a Compatible Family Donor in the Treatment of Patients Over 55 Years With Hematological Malignancies [NCT00806767]Phase 282 participants (Anticipated)Interventional2007-03-31Completed
Hematopoietic Stem Cell Transplantation for the Treatment of Older Patients With Acute Myelogenous Leukemia [NCT00623935]Phase 256 participants (Actual)Interventional2007-03-31Completed
Phase II Study of Orally Fludarabine, Adriamycin and Dexamethasone (FAD) in Newly Diagnosed PTCL [NCT00840385]Phase 230 participants (Anticipated)Interventional2007-11-30Recruiting
Reduced Intensity Stem Cell Transplantation (RIST) for Patients With Hematological Malignancies Conditioned With Fludarabine and Busulfan [NCT00619645]Phase 28 participants (Actual)Interventional2007-06-30Completed
Reduced Intensity Conditioning Regimen for Low- and Intermediate-risk Myelodysplastic Syndrome Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation [NCT03412266]Phase 250 participants (Anticipated)Interventional2018-02-01Recruiting
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)
Phase I/II Clinical Trial of HER2 Targeted HypoSti.CAR-T Cells in Treating Patients With HER2 Positive Local Advanced or Metastatic Solid Tumors [NCT05681650]Phase 1/Phase 230 participants (Anticipated)Interventional2023-10-11Recruiting
A Phase 1/2a, Safety And Efficacy Study Of HLA-G- Targeted CAR-T Cells IVS-3001 In Subjects With Previously Treated Advanced HLA-G-Positive Solid Tumors [NCT05672459]Phase 1/Phase 2117 participants (Anticipated)Interventional2023-06-21Recruiting
A Single-Arm, Open-Label, Phase 1/2 Study Evaluating the Safety, Efficacy, and Cellular Kinetics/Pharmacodynamics of ALLO-501A, an Anti-CD19 Allogeneic CAR T Cell Therapy, and ALLO-647, an Anti-CD52 Monoclonal Antibody, in Subjects With Relapsed/Refractor [NCT04416984]Phase 1/Phase 2160 participants (Anticipated)Interventional2020-05-21Recruiting
Post-Transplant Bendamustine (PT-BEN) for GVHD Prophylaxis [NCT04022239]Phase 1/Phase 240 participants (Anticipated)Interventional2020-03-13Recruiting
Pilot Open-Label Study of Safety and Efficacy of Ruxolitinib Given Peri-Transplant During Reduced Intensity Allogeneic Hematopoietic Cell Transplantation (HCT) in Patients With Myelofibrosis [NCT02917096]Phase 118 participants (Actual)Interventional2016-11-13Completed
Phase I Study to Evaluate Cellular Immunotherapy Using Memory-Enriched T Cells Lentivirally Transduced to Express a CD19-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Receptor and a Truncated EGFR Following Lymphodepleting Chemotherapy in Adult P [NCT02153580]Phase 137 participants (Actual)Interventional2014-09-24Active, not recruiting
A Trial of Busulfan, Melphalan, Fludarabine and T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Followed by Post Transplantation Donor Lymphocyte Infusions for Patients With Relapsed or High-Risk Multiple Myeloma [NCT01131169]Phase 266 participants (Actual)Interventional2010-05-31Completed
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02 [NCT00258427]Phase 214 participants (Actual)Interventional2002-03-26Completed
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
Phase I/II Study Administering Peripheral Blood Lymphocytes Transduced With a Murine T-Cell Receptor Recognizing Human Thyroglobulin to Patients With Thyroglobulin Expressing Thyroid Cancer [NCT02390739]Phase 1/Phase 20 participants (Actual)Interventional2015-03-02Withdrawn
A Phase 1/2 Single Arm Open-Label Clinical Trial of Gavocabtagene Autoleucel (Gavo-cel) in Patients With Advanced Mesothelin-Expressing Cancer [NCT03907852]Phase 1/Phase 2175 participants (Anticipated)Interventional2019-04-15Recruiting
A Phase II Study of Crenolanib With Fludarabine and Cytarabine in Pediatric Patients With Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia [NCT03324243]Phase 20 participants (Actual)Interventional2018-01-31Withdrawn(stopped due to Withdrawn: Study halted prior to enrollment of first participant)
An Open-label, Randomised Phase II Study of Fludarabine With Pegylated Liposomal Doxorubicin Versus Pegylated Liposomal Doxorubicin Alone In Patients With Platinum Resistant/Refractory Ovarian Cancer [NCT03335241]Phase 2140 participants (Anticipated)Interventional2017-03-01Recruiting
Phase II Study Evaluating the Efficacy of Allogeneic Transplant Conditioning With Adaptive Dose Busulfan Intravenous (Busilvex®) in Patients at High Risk of Carrying Blood Diseases [NCT02483325]Phase 233 participants (Actual)Interventional2014-09-30Completed
Pilot Study of Adoptive Cell Transfer for the Treatment of Metastatic Cancer That Expresses NY-ESO-1 Using Lymphodepleting Conditioning Followed by Infusion of Anti-NY ESO-1 Murine TCR-Gene Engineered Lymphocytes [NCT02774291]Early Phase 13 participants (Actual)Interventional2017-04-20Terminated(stopped due to Due to lack of accrual the study was formally terminated on 01-JUL-2020. Primary Completion and Study Completion Dates have been revised based accordingly based on respective definitions)
Fludarabine and Cytarabine Versus High-dose Cytarabine in Consolidation Treatment of Core-bing Factor Acute Myeloid Leukemia: A Prospective, Multicenter, Randomized Study [NCT02926586]Phase 4200 participants (Anticipated)Interventional2017-01-01Recruiting
Clinical Study of Redirected Autologous T Cells With a Chimeric Antigen Receptor in Patients With Malignant Tumors [NCT03302403]18 participants (Actual)Interventional2017-12-29Active, not recruiting
Epigenetic Reprogramming in Relapse AML: A Phase 1 Study of Decitabine and Vorinostat Followed by Fludarabine, Cytarabine and G-CSF (FLAG) in Children and Young Adults With Relapsed/Refractory AML [NCT03263936]Phase 137 participants (Actual)Interventional2017-07-11Completed
A Phase 1, Multicenter, Open-Label Study of CB-012, a CRISPR-Edited Allogeneic Anti-CLL-1 CAR-T Cell Therapy in Patients With Relapsed/Refractory Acute Myeloid Leukemia [NCT06128044]Phase 170 participants (Anticipated)Interventional2023-12-05Not yet recruiting
A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies [NCT05579769]Phase 232 participants (Anticipated)Interventional2022-11-04Recruiting
A Phase I/II Study of HLA-matched Mobilized Peripheral Blood Hematopoietic Stem Cell Transplantation for Advanced Mycosis Fungoides/Sezary Syndrome Using Nonmyeloablative Conditioning With Campath-1H [NCT00047060]Phase 1/Phase 25 participants (Actual)Interventional2002-07-30Completed
CBA Versus FBA Conditioning Followed by Haploidentical Allogeneic HSCT in Treatment of High Risk and Refractory AML [NCT03384225]Phase 3120 participants (Anticipated)Interventional2016-08-01Recruiting
Dosimetry and Biodistribution of [18F]-Fludarabine in Lymphoid Malignancies [NCT02128945]Phase 110 participants (Actual)Interventional2014-04-30Completed
A PALG Prospective Multicenter Clinical Trial to Compare the Efficacy of Two Standard Induction Therapies (DA-90 vs DAC) and Two Standard Salvage Regimens (FLAG-IDA vs CLAG-M) in AML Patients ≤ 60 Years Old [NCT03257241]Phase 3582 participants (Anticipated)Interventional2017-07-03Recruiting
T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias [NCT03653338]Phase 1/Phase 25 participants (Anticipated)Interventional2018-08-02Recruiting
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
Phase II Study Testing Prophylaxis Feasibility of Graft Versus Host Disease With Only High Dose Cyclophosphamide Post-transplantation for Patients Eligible to a Reduced-intensity Conditioning Regiment Prior to Allogenic Transplantation With a Compatible F [NCT03263767]Phase 247 participants (Actual)Interventional2018-01-15Terminated(stopped due to Security criteria (MTD))
Relmacabtagene Autoleucel as Second-Line Therapy in Adult Patients With Aggressive B-cell NHL: a Single-arm, Multicenter, Open, Phase II Study [NCT06093841]Phase 246 participants (Anticipated)Interventional2023-11-30Not yet recruiting
T Regulatory Cell for Suppression of Acute Graft-vs-Host-Disease in Recipients of a Non-Myeloablative Umbilical Cord Blood Transplantation for Treatment of Hematological Malignancies [NCT02118311]Phase 20 participants (Actual)Interventional2016-06-30Withdrawn(stopped due to Changing study design. Will replace with a different protocol.)
Tumor Infiltrating Lymphocytes in Pediatric Malignant Solid Tumors: A Prospective Biobanking Study and Phase I Clinical Trial [NCT06047977]Phase 130 participants (Anticipated)Interventional2024-02-01Not yet recruiting
A Phase I Study of Bivalent CD79b and CD19 Directed CAR T Cells in Patients With Relapsed/Refractory Non-Hodgkin Lymphoma [NCT06026319]Phase 124 participants (Anticipated)Interventional2023-10-26Recruiting
A Phase 1 Multicenter Study Evaluating the Safety and Efficacy of ACE1831, an Allogeneic CD20-conjugated Gamma Delta T-cell Therapy, in Adult Subjects With Relapsed/Refractory CD20-expressing B-cell Malignancies [NCT05653271]Phase 142 participants (Anticipated)Interventional2023-01-21Recruiting
A Phase 2 Open-Label, Multicenter Study Evaluating The Safety And Efficacy of Axicabtagene Ciloleucel Concomitant With Prophylactic Steroids In Subjects With Relapsed Or Refractory Large B-Cell Lymphoma In The Outpatient Setting [NCT05459571]Phase 240 participants (Anticipated)Interventional2022-08-09Recruiting
Combined Transplantation of Unmanipulated Haploidentical and a SingleCord Blood Unit for Patients With Hematologic Malignancies [NCT01359254]Phase 21 participants (Actual)Interventional2010-04-30Terminated(stopped due to did not accrue enough patients.)
A Phase 1b Study of JCAR014, Autologous T Cells Engineered to Express a CD19-Specific Chimeric Antigen Receptor, in Combination With Durvalumab (MEDI4736) for Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma [NCT02706405]Phase 130 participants (Actual)Interventional2016-11-15Terminated(stopped due to Terminated due to slow accrual.)
A Cancer Research UK Phase I Trial of Anti-GD2 Chimeric Antigen Receptor (CAR) Transduced T-cells (1RG-CART) in Patients With Relapsed or Refractory Neuroblastoma [NCT02761915]Phase 117 participants (Actual)Interventional2016-02-29Completed
A Pilot Open-Label Clinical Trial Evaluating the Safety and Efficacy of Autologous T Cells Expressing Enhanced TCRs Specific for NY-ESO-1 in Subjects With Stage IIIb or Stage IV Non-Small Cell Lung Cancer (NSCLC) [NCT02588612]Phase 110 participants (Actual)Interventional2016-02-01Completed
A Phase I Trial of Anti-CD19 and Anti-CD20 Bicistronic Chimeric Antigen Receptor T- Cells for Treating B-cell Malignancies [NCT05797233]Phase 158 participants (Anticipated)Interventional2023-08-28Recruiting
A Phase 1, Multicenter, Open-Label Study of CB-011, a CRISPR-Edited Allogeneic Anti-BCMA CAR-T Cell Therapy in Patients With Relapsed/Refractory Multiple Myeloma (CaMMouflage Trial) [NCT05722418]Phase 150 participants (Anticipated)Interventional2023-02-06Recruiting
A Phase 1 Single Arm, Open Label Study to Evaluate the Safety of UF-KURE19 Cells in Patients With Relapsed or Refractory B Cell Non-Hodgkin Lymphomas [NCT05400109]Phase 110 participants (Anticipated)Interventional2023-04-26Recruiting
A Phase 1 Multicenter Study Evaluating the Safety and Efficacy of ALLO-316 Following ALLO-647 Containing Conditioning Regimen in Subjects With Advanced or Metastatic Clear Cell Renal Cell Carcinoma [NCT04696731]Phase 1120 participants (Anticipated)Interventional2021-02-24Recruiting
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major [NCT03171831]Phase 430 participants (Anticipated)Interventional2017-04-01Recruiting
A Collaboration Phase 2 Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia [NCT05955261]Phase 270 participants (Anticipated)Interventional2023-07-25Recruiting
Clinical Trial of CD40L-augmented Tumor Infiltrating Lymphocytes (CD40L TIL) for Patients With Oncogene-Driven Advanced Non-Small Cell Lung Cancer (NSCLC) [NCT05681780]Phase 1/Phase 220 participants (Anticipated)Interventional2022-12-23Recruiting
A Phase I Study to Evaluate PSCA-Targeting Chimeric Antigen Receptor (CAR)-T Cells for Patients With PSCA+ Metastatic Castration Resistant Prostate Cancer [NCT03873805]Phase 114 participants (Actual)Interventional2019-08-20Active, not recruiting
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
A Pilot Trial Examining Myeloid-Derived Suppressor Cells and Checkpoint Immune Regulators' Expression in Allogeneic Stem Cell Transplant Recipients Using Myeloablative Busulfan and Fludarabine [NCT02916979]Phase 120 participants (Actual)Interventional2016-09-06Completed
A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in Pediatric and Young Adult (<21year) Patients With High-Risk and Very High-Risk Myeloid Malignancies [NCT04990323]Phase 1/Phase 212 participants (Anticipated)Interventional2021-12-01Recruiting
A Randomized Study of Once Daily IV Busulfan With Fludarabine With Hemopoietic Stem Cell Transplantation for Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndrome (MDS) [NCT00469144]Phase 3233 participants (Actual)Interventional2005-06-30Completed
A Phase I Clinical Trial of T-cells Expressing an Anti-SLAMF7 CAR for Treating Multiple Myeloma [NCT03958656]Phase 113 participants (Actual)Interventional2019-06-13Completed
Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa [NCT00478244]7 participants (Actual)Interventional2007-04-30Terminated(stopped due to Competing studies)
Phase 1/2 Study of IDP-023 as a Single Agent and in Combination With Antibody Therapies in Patients With Advanced Hematologic Cancers [NCT06119685]Phase 1/Phase 2128 participants (Anticipated)Interventional2023-10-25Recruiting
A Phase II Study of Axicabtagene Ciloleucel, an Anti-CD19 Chimeric Antigen Receptor (CAR) Tcell Therapy, in Combination With Radiotherapy (RT) in Relapsed/Refractory Follicular Lymphoma [NCT06043323]Phase 220 participants (Anticipated)Interventional2024-03-31Not yet recruiting
"A Pilot Window-3 Study of Acalabrutinib Plus Rituximab Followed by Brexucabtagene Autoleucel Therapy in Patients With Previously Untreated High-risk Mantle Cell Lymphoma" [NCT05495464]Early Phase 120 participants (Anticipated)Interventional2022-11-18Recruiting
A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies [NCT05476770]Phase 154 participants (Anticipated)Interventional2022-11-11Recruiting
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
Phase II Study of the Administration of T Lymphocytes Expressing the CD30 Chimeric Antigen Receptor (CAR) for Relapsed/Refractory CD30+ Peripheral T Cell Lymphoma [NCT04083495]Phase 220 participants (Anticipated)Interventional2019-09-17Recruiting
A Phase II Randomized Controlled Trial Comparing GVHD-Reduction Strategies for Allogeneic Peripheral Blood Transplantation (PBSCT) for Patients With Acute Leukemia or Myelodysplastic Syndrome: Selective Depletion of CD45RA+ Naïve T Cells (TND) vs. Post-Tr [NCT03970096]Phase 2120 participants (Anticipated)Interventional2019-11-19Recruiting
Phase 1/2a, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate Safety and Clinical Activity of PBCAR0191 in Subjects With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) and r/r B-cell Acute Lymphoblastic Leukemia (B-A [NCT03666000]Phase 1/Phase 2120 participants (Anticipated)Interventional2019-03-11Recruiting
A Phase 1 Multicenter Study Evaluating the Safety and Tolerability of KTE-X19 in Adult Subjects With Relapsed/Refractory Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma [NCT03624036]Phase 116 participants (Actual)Interventional2018-11-15Terminated(stopped due to Development program terminated)
A Phase I Clinical Trial of T Cells Expressing a Novel Fully-human Anti-BCMA CAR for Treating Multiple Myeloma [NCT03602612]Phase 135 participants (Actual)Interventional2018-09-14Active, not recruiting
A Phase I/II Study Administering Peripheral Blood Lymphocytes Transduced With a CD70-Binding Chimeric Antigen Receptor to Patients With CD70-Expressing Cancers [NCT02830724]Phase 1/Phase 2124 participants (Anticipated)Interventional2017-04-06Recruiting
A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Adult Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (r/r ALL) (ZUMA-3) [NCT02614066]Phase 1/Phase 2125 participants (Actual)Interventional2016-03-07Completed
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
Study to Infuse Haploidentical Natural Killer Cells in Patients With Relapsed or Refractory Neuroblastoma [NCT00698009]Phase 21 participants (Actual)Interventional2008-06-30Terminated(stopped due to Slow accrual.)
Fludarabine, Campath, TBI T-Cell Deplete NMSCT With Post-Transplant T-Cell Infusions for CML Failing Imatinib Therapy With Imatinib (STI571) [NCT00416884]Phase 21 participants (Actual)Interventional2003-05-31Terminated(stopped due to Low enrollment)
Toward a Less Toxic Yet Highly Effective Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Children and Adolescents With Severe Sickle Cell Disease: A Pilot Study [NCT00968162]Phase 18 participants (Actual)Interventional2009-02-28Completed
Phase II Open Lable Clinical Study Efficacy and Safety of the Holistic Treatment for Young Patients With High-Risk Multiple Myeloma [NCT04008888]50 participants (Anticipated)Interventional2018-01-05Recruiting
Multicentre, Non-Randomised, Open-Label Phase II Study to Evaluate the Efficacy and Safety of Induction Treatment With Rituximab, Fludarabine, Cyclophosphamide, Followed by Rituximab Maintenance Therapy (R-Fc-Rm) in the First Line Treatment of Chronic Lym [NCT00545714]Phase 286 participants (Actual)Interventional2007-11-21Completed
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
T-Cell or Natural Killer (NK) Cell Adback in Patients With Lymphoid Malignancies Receiving Allogeneic Stem Cell Transplantation With Campath-IH Containing Conditioning Regimens [NCT00536978]Phase 222 participants (Actual)Interventional2007-09-30Completed
Randomized Comparison of Cyclophosphamide Versus Cyclophosphamide Plus Fludarabine In Addition To Anti-Thymocyte Globulin for the Conditioning Therapy in Allogeneic Hematopoietic Cell Transplantation for Bone Marrow Failure Syndrome [NCT00774527]Phase 382 participants (Actual)Interventional2003-03-31Completed
Phase Ib Study To Assess The Feasibility And Safety Of Adoptive Transfer Of Autologous Tumor-Infiltrating Lymphocytes In Advanced Solid Tumors [NCT03992326]Phase 16 participants (Actual)Interventional2019-09-01Terminated(stopped due to Internal competitive study that is open)
Phase I/II Study of Allogeneic Stem Cell Transplantation for Patients With Hematologic Diseases Using Haploidentical Family Donors and Sub-Myeloablative Conditioning With Campath 1H [NCT00058825]Phase 1/Phase 227 participants (Actual)Interventional2000-08-31Terminated(stopped due to Slow accrual due to practice changes meant study would take too long to finish)
CD34+Stem Cell Selection for Patients Receiving Partially Matched Family or Matched Unrelated Adult Donor Allogeneic Stem Cell Transplantations for Malignant and Non-Malignant Disease [NCT01049854]Phase 220 participants (Actual)Interventional2011-09-30Completed
Phase II Study Using Alemtuzumab Combined With Fludarabine for the Treatment of Relapsed/Refractory B-cell Chronic Lymphocytic Leukemia (B-CLL) [NCT00206726]Phase 260 participants (Actual)Interventional2005-05-31Completed
Active Immunization of Sibling Stem Cell Transplant Donors Against Purified Myeloma Protein of the Stem Cell Recipient With Multiple Myeloma in the Setting of Non-Myeloablative, HLA-Matched Allogeneic Peripheral Blood Stem Cell Transplantation [NCT00006184]Phase 220 participants (Actual)Interventional2001-02-08Completed
A Multi-Center Phase 2 Efficacy and Pharmacokinetic Study Evaluating Fludarabine, Cyclophosphamide, and Subcutaneous Campath (FCCam, Alemtuzumab) for Previously Untreated B-Cell Chronic Lymphocytic Leukemia [NCT00230282]Phase 225 participants (Actual)Interventional2004-07-31Completed
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
Vaccination to Enhance the Anti-Tumor Activity of GD2 Chimeric Antigen Receptor-Expressing, VZV-Specific T Cells in Subjects With Advanced Sarcomas and Neuroblastoma (VEGAS) [NCT01953900]Phase 126 participants (Anticipated)Interventional2014-04-30Active, not recruiting
Clinical Study to Evaluate the Safety and Efficacy of IM19 CAR-T Cell Therapy in Patients With Relapsed or Refractory CD19-positive Non-Hodgkin's Lymphoma [NCT03528421]Phase 130 participants (Anticipated)Interventional2018-05-22Recruiting
Lung Transplant in Tandem With Bone Marrow Transplant for Combined Lung and Bone Marrow Failure [NCT03500731]Phase 1/Phase 28 participants (Anticipated)Interventional2018-04-19Recruiting
A Phase I-II Study of Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies [NCT00943319]Phase 1/Phase 250 participants (Actual)Interventional2012-03-31Completed
Phase I Study of Infusion of Umbilical Cord Blood (UCB) Derived CD25+CD4+ T-Regulatory (Treg) Cells After Nonmyeloablative Cord Blood Transplantation [NCT00602693]Phase 141 participants (Actual)Interventional2007-07-23Completed
Total Body Irradiation With Fludarabine Conditioning Followed by Transplantation With Combined Umbilical Cord Blood Grafts [NCT00606437]Phase 140 participants (Actual)Interventional2005-09-30Completed
QN-019a as a Monotherapy and in Combination With Anti-CD20 Monoclonal Antibodies in Subjects With B-Cell Malignancies [NCT05379647]Phase 124 participants (Anticipated)Interventional2021-11-04Recruiting
Shanghai General Hospital, Shanghai, Jiao Tong University School of Medicine [NCT05379569]Phase 4142 participants (Anticipated)Interventional2022-05-15Recruiting
Phase 2 Study BRIDGING PRE-TRANSPLANT INFLAMMATORY DAMPENING for PRIMARY IMMUNE REGULATORY DISORDERS (BRIDGE Trial) [NCT05787574]Phase 239 participants (Anticipated)Interventional2023-03-15Recruiting
A Phase I Study to Evaluate the Safety, Tolerance and Efficacy of HRYZ-T101 TCR-T Cell for HPV18 Positive Advanced Solid Tumor [NCT05787535]Phase 117 participants (Anticipated)Interventional2023-03-21Recruiting
A Randomized Study of Fludarabine in Part of Induction and Postremission Treatment for de Novo Acute Myeloid Leukaemia in Elderly Patients [NCT00925873]Phase 3303 participants (Actual)Interventional1996-06-30Completed
Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies (UCALL) [NCT01247688]0 participants (Actual)Interventional2010-11-30Withdrawn(stopped due to This study was withdrawn due to low accrual.)
Allogeneic Hematopoietic Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors [NCT03615105]Phase 29 participants (Actual)Interventional2018-07-25Active, not recruiting
Phase II Study of Metastatic Melanoma Using a Chemoradiation Lymphodepleting Conditioning Regimen Followed by Infusion of Anti-Mart-1 and Anti-gp100 TCR-Gene Engineered Lymphocytes and Peptide Vaccines [NCT00923195]Phase 24 participants (Actual)Interventional2008-12-31Completed
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
Phase I/II Study of Metastatic Cancer That Expresses Her-2 Using Lymphodepleting Conditioning Followed by Infusion of Anti-Her-2 Gene Engineered Lymphocytes [NCT00924287]Phase 1/Phase 21 participants (Actual)Interventional2008-11-30Terminated(stopped due to This study was terminated after the first patient treated on study died as a result of the treatment.)
A Myeloablative Conditioning Regimen Consisting of Cyclophosphamide, Fludarabine and Total Body Irradiation Followed by the Transplantation of Unrelated Donor Double Unit Umbilical Cord Blood Grafts for Patients With Hematological Malignancy. [NCT00597519]Phase 228 participants (Actual)Interventional2006-03-31Completed
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.)
A Phase 1/2 Multi-center Study Evaluating the Safety and Efficacy of Axicabtagene Ciloleucel in Combination With Utomilumab in Subjects With Relapsed/Refractory Large B-Cell Lymphoma [NCT03704298]Phase 115 participants (Actual)Interventional2018-11-20Terminated(stopped due to Development program terminated)
"Phase II, Multicenter, Trial, Exploring Chemo-free Treatment (GA101+Ibrutinib) and MRD-driven Strategy in Previously Untreated Symptomatic B-chronic Lymphocytic Leukemia Medically Fit A Study From the Goelams/GCFLLC/MW Intergroup" [NCT02666898]Phase 2135 participants (Actual)Interventional2015-10-31Completed
Administration of T Cells Expressing Chondroitin-Sulfate-Proteoglycan-4 Specific Chimeric Antigen Receptors (CAR) in Subjects With Head and Neck Squamous Cell Carcinoma (HNSCC) [NCT06096038]Phase 1/Phase 233 participants (Anticipated)Interventional2023-12-31Not yet recruiting
Open-label, Phase I, Multi-center Study to Determine in Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Patients the Recommended Dose of CYAD-02 After a Non-myeloablative Preconditioning Chemotherapy Followed by a Potential Consolid [NCT04167696]Phase 127 participants (Anticipated)Interventional2019-11-25Recruiting
Genomics-Based Target Therapy for Children With Relapsed or Refractory Malignancy [NCT02638428]Phase 290 participants (Anticipated)Interventional2015-12-31Recruiting
Pilot Study of Expanded, Activated Haploidentical Natural Killer Cell Infusions for Non-B Lineage Hematologic Malignancies and Solid Tumors [NCT00640796]Phase 122 participants (Actual)Interventional2008-09-30Completed
A Phase 2, Multicenter, Randomized Open-Label Study To Determine the Efficacy of Lenalidomide (Revlimid®) Versus Investigator's Choice in Patients With Relapsed or Refractory Mantle Cell Lymphoma [NCT00875667]Phase 2254 participants (Actual)Interventional2009-04-30Completed
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study [NCT04009525]Phase 4800 participants (Anticipated)Interventional2019-06-01Recruiting
A Phase 1 Trial of the Wee1 Kinase Inhibitor AZD1775 in Combination With Flag Chemotherapy in Children, Adolescents, and Young Adults With Relapsed or Refractory Acute Myeloid Leukemia [NCT02791919]Phase 10 participants (Actual)Interventional2017-05-25Withdrawn(stopped due to Other - Protocol moved to Disapproved)
Nonmyeloablative Stem Cell Transplantation With CD8-depleted or Unmanipulated Peripheral Blood Stem Cells: A Prospective Randomized Phase II Trial [NCT00693927]Phase 254 participants (Actual)Interventional2002-03-31Completed
A Pilot Study of Sequential Autologous Stem Cell Transplantation and Immunotherapy With Reduced Intensity Allogeneic Stem Cell Transplant for High Risk Neuroblastoma [NCT00670410]Phase 115 participants (Actual)Interventional2003-11-30Terminated(stopped due to Poor accrual)
[NCT02577094]Phase 1/Phase 20 participants (Actual)InterventionalWithdrawn
Clinical Study of Natural Killer Cell Infusion in Patients With Acute Myeloid Leukemia [NCT04221971]Phase 110 participants (Anticipated)Interventional2020-10-31Not yet recruiting
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.)
Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors Using Fludarabine, Busulfan, 400 cGy Total Body Irradiation, and Thymoglobulin [NCT00679536]Phase 1/Phase 230 participants (Anticipated)Interventional2008-05-31Recruiting
An Open-Label, Multi-Centre, Randomised, Phase Ib Study to Investigate the Safety and Efficacy of RO5072759 Given in Combination With CHOP, FC or Bendamustine Chemotherapy in Patients With CD20+ B-Cell Follicular Non-Hodgkin's Lymphoma [NCT00825149]Phase 1137 participants (Actual)Interventional2009-02-28Completed
Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Treosulfan Based Preparative Regimen [NCT00796068]Phase 2130 participants (Actual)Interventional2009-02-24Completed
Safety and Efficacy of CD19/CD22 Dual Targeted CAR-T Cell Therapy in Patients With R/R B-Cell Acute Lymphoblastic Leukemia [NCT05225831]Early Phase 1100 participants (Anticipated)Interventional2021-08-15Recruiting
A Phase I/II Clinical Study of TC-N201 Injection for the Treatment of Advanced Solid Tumors With HLA-A2 Expression and Positive NY-ESO-1. [NCT05881525]Phase 118 participants (Anticipated)Interventional2023-06-01Recruiting
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
An Exploratory Clinical Study Evaluating the Safety and Efficacy of Anti-CEA-CAR-T Cells Injection in Patients With CEA+ Locally Advanced and/or Metastatic Solid Tumors [NCT06013111]Phase 19 participants (Anticipated)Interventional2023-09-01Not yet recruiting
Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome [NCT00885833]Phase 1/Phase 25 participants (Anticipated)Interventional2007-02-28Completed
Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia [NCT00882323]Phase 233 participants (Anticipated)Interventional2008-11-30Recruiting
Phase II Trial of Targeted Immune-Depleting Chemotherapy and Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplantation Using 8/8 and 7/8 HLA-matched Unrelated Donors and Utilizing Two Graft-versus-Host Disease Prophylaxis Regimens for the Treat [NCT00520130]Phase 1/Phase 292 participants (Actual)Interventional2007-10-30Completed
A Phase 1 Open-label, Multicenter Study Evaluating the Safety and Efficacy of KITE-363 or KITE-753, Autologous Anti-CD19/CD20 CAR T-cell Therapies, in Subjects With Relapsed and/or Refractory B-cell Lymphoma [NCT04989803]Phase 1114 participants (Anticipated)Interventional2021-10-27Recruiting
A Phase 3 Open-Label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation [NCT02421939]Phase 3371 participants (Actual)Interventional2015-10-20Active, not recruiting
A Proof of Concept Study of TBio-4101 (an Autologous Selected and Expanded Tumor Infiltrating Lymphocyte [TIL] Therapy) Using Short-Term Cultured, Selected Autologous TIL Following a Lymphodepleting Chemotherapy Regimen and Followed by IL-2 for Patients W [NCT05628883]Phase 125 participants (Anticipated)Interventional2022-11-22Recruiting
Pilot Study to Assess the Feasibility, Safety and Efficacy of Adoptive Transfer of Autologous Tumor-Infiltrating Lymphocytes Enriched for Tumor Antigen Specificity (NeoTIL) in Advanced Solid Tumors [NCT04643574]Phase 142 participants (Anticipated)Interventional2021-03-09Active, not recruiting
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.)
A Phase I Study of B-Cell Maturation Antigen (BCMA)-Specific Chimeric Antigen Receptor T Cells in Combination With JSMD194, a Small Molecule Inhibitor of Gamma Secretase, in Patients With Relapsed or Persistent Multiple Myeloma [NCT03502577]Phase 118 participants (Anticipated)Interventional2018-05-23Suspended(stopped due to Waiting on confirmation of additional funding)
Rasburicase to Prevent Graft -Versus-Host Disease [NCT00513474]Phase 146 participants (Actual)Interventional2008-01-31Completed
T-cell Based Immunotherapy for Treatment of Patients With Disseminated Melanoma. [NCT00937625]Phase 1/Phase 231 participants (Actual)Interventional2009-06-30Completed
Busulfan-Fludarabine-Clofarabine With Allogeneic Stem Cell Transplantation for Advanced Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Advanced, Gleevec Refractory Chronic Myeloid Leukemia. A Randomized Phase II Study. [NCT00469014]Phase 272 participants (Actual)Interventional2006-09-30Completed
Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia [NCT00301834]Phase 235 participants (Actual)Interventional2005-01-31Completed
An Open-labeled, Randomized, Two-dose, Parallel Group Trial of Ofatumumab, a Fully Human Monoclonal Anti-CD20 Antibody, in Combination With Fludarabine and Cyclophosphamide, in Patients With Previously Untreated B-cell CLL [NCT00410163]Phase 261 participants (Actual)Interventional2007-01-31Completed
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
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 Controlled Trial of Intermittent Fludarabine for Psoriatic Arthritis [NCT00001422]Phase 220 participants Interventional1995-06-30Completed
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases [NCT01019876]Phase 2/Phase 350 participants (Anticipated)Interventional2002-06-30Recruiting
Allogeneic Hematopoietic Cell Transplantation for Patients With Acute Myelogenous Leukemia in Remission Using HLA-Matched Sibling Donors, HLA-Matched Unrelated Donors, or HLA-Mismatched Familial Donors - A Phase 2 Study [NCT01020734]Phase 2263 participants (Actual)Interventional2011-05-31Completed
Phase II Trial of Combined Immunochemotherapy With Fludarabine, Mitoxantrone, Cyclophosphamide and Alemtuzumab (FMC-Alemtuzumab) in Patients With Previously Treated or Untreated T-Prolymphocytic Leukemia [NCT01186640]Phase 216 participants (Actual)Interventional2010-06-30Completed
A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT03600909]Phase 23 participants (Actual)Interventional2018-05-15Terminated(stopped due to Accrual has been slow)
IIT2017-03-Merin-HaploBFR: Bendamustine, Fludarabine, And Rituximab Conditioning For Haploidentical Stem Cell Transplantation With CD56-Enriched Donor Cell Infusion For Relapsed/Refractory Lymphoma, Multiple Myeloma, and CLL [NCT03524235]Phase 130 participants (Anticipated)Interventional2018-07-18Active, not recruiting
A Phase II Study Using the Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Neoantigens in Patients With Metastatic Cancer [NCT03412877]Phase 2270 participants (Anticipated)Interventional2018-09-06Recruiting
A Phase II Clinical Trial of Vemurafenib With Lymphodepletion Plus Adoptive Cell Transfer and High Dose IL-2 in Patients With Metastatic Melanoma [NCT01659151]Phase 217 participants (Actual)Interventional2012-08-03Active, not recruiting
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)
A Multi-Center Study of Conditioning With Treosulfan, Fludarabine and Escalating Doses of TBI for Allogeneic Hematopoietic Cell Transplantation in Patients With Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome (MDS), and Acute Lymphoblastic Leukemia [NCT00860574]Phase 296 participants (Actual)Interventional2009-02-28Completed
A Phase I and Dose Expansion Cohort Study of Panobinostat in Combination With Fludarabine and Cytarabine in Pediatric Patients With Refractory or Relapsed Acute Myeloid Leukemia or Myelodysplastic Syndrome [NCT02676323]Phase 119 participants (Actual)Interventional2016-05-03Terminated(stopped due to Slow accrual)
A Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body Irradiation [NCT00856388]62 participants (Actual)Interventional2009-01-14Completed
T Cells Expressing Fully-human Anti-CD19 and Anti-CD20 Chimeric Antigen Receptors for Treating B-cell Malignancies and Hodgkin Lymphoma [NCT04160195]Phase 12 participants (Actual)Interventional2019-12-20Terminated(stopped due to The original principal investigator left the National Institutes of Health (NCI) and the decision was made to close the study to enrollment.)
Autologous Hematopoietic Stem Cell Transplant for Crohn's Disease [NCT04154735]Phase 20 participants (Actual)Interventional2019-11-30Withdrawn(stopped due to Discontinued by Investigator)
A Pilot Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine [NCT00595127]21 participants (Actual)Interventional2001-06-30Completed
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)
A Phase I Clinical Trial Combining Nivolumab and Tumor Infiltrating Lymphocytes (TIL) for Patients With Advanced Non-Small Cell Lung Cancer [NCT03215810]Phase 120 participants (Actual)Interventional2017-10-11Completed
Randomized Comparison of Once-daily Intravenous Busulfan Plus Cyclophosphamide Versus Fludarabine as a Conditioning Regimen for Allogeneic Hematopoietic Cell Transplantation in Leukemia and Myelodysplastic Syndrome [NCT00774280]Phase 3130 participants (Actual)Interventional2002-05-31Completed
A Multicenter, Open Study to Assess the Antitumor Effect and Safety of Oral Fludarabine Phosphate (Fludara (SH T 586): 40 mg/m2/Day) Administered in 3 - 6 Treatment Cycles (1 Treatment Cycle: 5-consecutive Day Dosing, Followed by a Observation Period of 2 [NCT00688883]Phase 252 participants (Actual)Interventional2003-02-28Completed
Phase III Trial of Combined Immunochemotherapy With Fludarabine, Cyclophosphamide and Rituximab (FCR) Versus Bendamustine and Rituximab (BR) in Patients With Previously Untreated Chronic Lymphocytic Leukaemia [NCT00769522]Phase 3564 participants (Actual)Interventional2008-10-02Completed
Allogeneic γ9δ2 T Cells for the Treatment of Recurrent Hematologic Tumors After Allogeneic Hematopoietic Stem Cell Transplantation [NCT05755854]Phase 110 participants (Anticipated)Interventional2023-05-03Recruiting
Reduced Intensity Conditioning With Fludarabine and Busulfan Versus Fludarabine and Melphalan Before Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Randomised, Single-Center, Phase III Study [NCT05674539]Phase 3200 participants (Anticipated)Interventional2022-12-28Recruiting
Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related o [NCT00582933]Phase 296 participants (Actual)Interventional2001-05-31Completed
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
G-CSF and Plerixafor With Busulfan and Fludarabine for Allogeneic Stem Cell Transplantation for Myeloid Leukemias [NCT00822770]Phase 1/Phase 247 participants (Actual)Interventional2009-01-31Completed
HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease [NCT00578643]Phase 215 participants (Actual)Interventional2004-03-31Completed
Phase I-II Non-Randomized Study of Yttrium 90 Ibritumomab Tiuxetan (Zevalin) With Non Myeloablative Allogeneic Stem Cell Transplantation in Patients With Relapsed, Refractory, or Transformed Indolent Non Hodgkin Lymphoma [NCT00807196]Phase 1/Phase 220 participants (Anticipated)Interventional2008-09-30Recruiting
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
Fludarabine, Cyclophosphamide, and Multiple Dose Rituximab as Frontline Therapy in Chronic Lymphocytic Leukemia (CLL) [NCT00794820]Phase 266 participants (Actual)Interventional2003-12-31Completed
NK Cells Infusion as Consolidation Treatment of Acute Myeloid Leukemia in Children and Adolescents [NCT02763475]Phase 27 participants (Actual)Interventional2016-05-31Completed
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
A Phase I Study to Evaluate the Safety, Tolerance and Efficacy of CRTE7A2-01 TCR-T Cell for HPV16 Positive Advanced Cervical, Anal, or Head and Neck Cancers [NCT05122221]Phase 112 participants (Anticipated)Interventional2022-07-17Recruiting
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases [NCT00613561]Phase 225 participants (Anticipated)Interventional2007-12-31Recruiting
Post Transplant Cyclophosphamide for Unrelated and Related Allogeneic Hematopoietic Stem Cell Transplantation for Hematological Malignancies [NCT01349101]Phase 280 participants (Actual)Interventional2011-02-10Completed
Dasatinib in Combination With Chemotherapy for Relapsed or Refractory Core Binding Factor Acute Myeloid Leukemia: A Phase I Study (AflacLL1401) [NCT02680951]Phase 10 participants (Actual)Interventional2015-12-31Withdrawn(stopped due to Withdrawn due to lack of participants.)
Phase III, Multicentre, Randomised Study of Fludarabine/Cyclophosphamide Combination With or Without Rituximab in Patients With Untreated Mantle Cell Lymphoma [NCT00641095]Phase 2/Phase 3370 participants (Actual)Interventional2006-12-07Completed
A Randomised Trial of Chlorambucil Versus Fludarabine as Initial Therapy of Waldenström's Macroglobulinaemia and Splenic Lymphoma With Villous Lymphocytes [NCT00608374]Phase 3400 participants (Anticipated)Interventional2006-06-30Completed
High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma [NCT00790413]Early Phase 115 participants (Anticipated)Interventional2005-08-31Active, not recruiting
Phase III Randomized Trial of Fludarabine and Cyclophosphamide Versus Fludarabine for Previously Untreated Chronic Lymphocytic Leukemia [NCT00003764]Phase 3280 participants (Anticipated)Interventional2000-03-09Completed
A Phase II Study of Fludarabine (F), Rituxan (R) and Avastin (A) Followed by RA Maintenance in Patients With Relapsed/Refractory B-cell Chronic Lymphocytic Leukemia (CLL) [NCT00845104]Phase 20 participants (Actual)Interventional2009-01-31Withdrawn(stopped due to No patients enrolled.)
A Phase II Trial of Transplants From HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies and Other Lethal H [NCT01119066]Phase 2422 participants (Actual)Interventional2010-05-03Completed
A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults With Severe Sickle Cell Disease (BMT CTN 1503) [NCT02766465]Phase 2138 participants (Actual)Interventional2016-11-30Completed
Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia [NCT00578292]10 participants (Actual)Interventional2004-02-29Terminated(stopped due to Stem cell transplant was determined SOC for this disease (study is not relevant))
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 I/II Clinical Trail of TC-E202 Targeting HPV16 E6 for Relapsed/Refractory to Standard Treatment or Metastatic Cervical Carcinoma [NCT05357027]Phase 1/Phase 218 participants (Anticipated)Interventional2022-08-10Recruiting
Study of FT538 in Combination With Daratumumab in Acute Myeloid Leukemia [NCT04714372]Phase 111 participants (Actual)Interventional2021-11-03Active, not recruiting
A Phase II Study Using Autologous Young Tumor-Infiltrating Lymphocytes Derived From Patients With Non-Small Cell Lung Cancer Following Non-Myeloablative Lymphocyte Depleting Preparative Regimen [NCT02133196]Phase 285 participants (Anticipated)Interventional2014-10-23Recruiting
A Safety Study of Autologous T Cells Engineered to Target CD19 and CRISPR Gene Edited to Eliminate Endogenous HPK1 (XYF19 CAR-T Cells) for Relapsed or Refractory Haematopoietic Malignancies. [NCT04037566]Phase 140 participants (Anticipated)Interventional2019-08-31Recruiting
A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematological Malignancies. [NCT00890500]Phase 112 participants (Actual)Interventional2011-01-31Completed
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
A Phase II Multicenter Open-label Study of MabThera(Rituximab) Addition to Regularly Prescribed Chemotherapy in Patients With Untreated Mantle Cell Lymphoma [NCT01144403]Phase 28 participants (Actual)Interventional2010-06-30Terminated
A Phase II Study of Reduced Intensity Sibling Allogeneic Transplantation for Relapsed, Chemosensitive, PET-positive Hodgkin Lymphoma [NCT00907036]Phase 249 participants (Anticipated)Interventional2009-07-31Not yet recruiting
A Phase I/II Study of Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation [NCT02750254]Phase 15 participants (Actual)Interventional2016-06-27Terminated(stopped due to Toxicity. Only enrolled patients in phase I portion of trial.)
T-cell Based Immunotherapy for Treatment of Patients Squamous Cell Carcinoma in the Oral Cavity. A Pilot Study. [NCT00937300]Phase 10 participants (Actual)Interventional2009-06-30Withdrawn(stopped due to The patients eligible for this trial do not exist anymore due to change in procedures.)
A Pilot Study of Reduced Intensity Conditioning Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Relapsed CNS Involvement by Lymphoma [NCT02724904]Phase 1/Phase 20 participants (Actual)Interventional2017-05-31Withdrawn(stopped due to Drug logistics)
A Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors [NCT01804634]Phase 260 participants (Anticipated)Interventional2013-03-27Recruiting
An Open, Uncontrolled, Multicenter Clinical Trial to Explore the Safety, Efficacy, and Remission Phase of Chimeric Antigen Receptor T Cell (CAR-T) in the Treatment of Children With Relapsed and Refractory Acute Lymphoblastic Leukemia [NCT04626765]Early Phase 150 participants (Anticipated)Interventional2020-04-01Recruiting
A Clinical Trial to Explore the Safety, Efficacy, and Remission Phase of CAR-T Cell in the Treatment of Adult Relapsed Refractory (R/R) Acute Lymphoblastic Leukemia Bridging Allogeneic Hematopoietic Stem Cell Transplantation [NCT04626726]Early Phase 150 participants (Anticipated)Interventional2020-04-01Recruiting
T-cell Therapy in Combination With Nivolumab, Relatlimab and Ipilimumab for Patients With Advanced Ovarian-, Fallopian Tube- and Primary Peritoneal Cancer [NCT04611126]Phase 1/Phase 218 participants (Anticipated)Interventional2021-04-22Recruiting
A Phase 1 Study Evaluating the Safety of ABT-263 in Combination With Either Fludarabine/Cyclophosphamide/Rituximab (FCR) or Bendamustine/Rituximab (BR) in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia [NCT00868413]Phase 132 participants (Actual)Interventional2009-11-30Completed
Fludarabine, Bendamustine, and Rituximab (FBR) Non-Myeloablative Allogeneic Conditioning for Patients With Lymphoid Malignancies [NCT00880815]Phase 160 participants (Actual)Interventional2009-02-17Completed
Addition of Inotuzumab Ozogamicin Pre- and Post-Allogeneic Transplantation [NCT03856216]Phase 244 participants (Anticipated)Interventional2019-10-28Recruiting
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 Phase I Study to Evaluate the Safety of Escalating Doses of Lymphodepleting Conditioning Chemotherapy Prior to CD19 Chimeric Antigen Receptor T Cells in Subjects With Relapsed/Refractory Diffuse Large B-cell Lymphoma [NCT05052528]Phase 136 participants (Anticipated)Interventional2021-09-17Recruiting
A Dose Escalation Study of Intensity Modulated Total Marrow Irradiation (IMRT-TMI) With Fludarabine as a Myeloablative Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Relapsed and Refractory Hematologic Malign [NCT03696537]Phase 16 participants (Actual)Interventional2018-08-29Terminated(stopped due to Extreme toxicity)
Bendamustine Versus Fludarabine as 2nd-line Treatment in Chronic Lymphocytic Leukemia, Stage BINET B+C / RAI II-IV [NCT01423032]Phase 2/Phase 396 participants (Actual)Interventional2001-09-30Completed
The Treatment of Hematologic Malignancies With Single or Double Umbilical Cord Blood Unit Transplantation Followed by Graft-versus-Host Prophylaxis With Tacrolimus and Mycophenolate Mofetil [NCT00608517]6 participants (Actual)Interventional2005-09-30Terminated(stopped due to slow accrual)
A Phase II Study Using Short-Term Cultured Anti-Tumor Autologous Lymphocytes Following a Lymphocyte Depleting Regimen in Metastatic Melanoma [NCT00513604]Phase 2158 participants (Actual)Interventional2007-06-30Completed
Phase 1/2 Study of Metastatic Renal Cancer Using T-Cells Transduced With a T-Cell Receptor Which Recognizes TRAIL Bound to the DR4 Receptor [NCT00923390]Phase 15 participants (Actual)Interventional2009-03-02Terminated
Adoptive Transfer of Haploidentical NK Cells in Combination With Epratuzumab for the Treatment of Relapsed Acute Lymphoblastic Leukemia [NCT00941928]Phase 22 participants (Actual)Interventional2009-07-31Terminated(stopped due to Slow accrual)
A Phase II Trial of Haplotype Mismatched Hematopoietic Stem Cell Transplantation Using Highly Purified CD34 Cells in Patients With Hematological Malignancies [NCT00593554]Phase 29 participants (Actual)Interventional2007-08-07Terminated(stopped due to Slow accrual)
Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for Patients With GATA2 Mutations [NCT00923364]Phase 219 participants (Actual)Interventional2009-05-07Completed
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
Pilot Study of Reduced Intensity Haematopoietic Stem Cell Transplantation in Patients With Poor Risk Myelodysplastic Syndrome and Acute Myeloid Leukaemia Utilising Conditioning With Fludarabine, Busulphan and Thymoglobulin [NCT00915811]Phase 220 participants (Anticipated)Interventional2007-06-30Terminated(stopped due to FB ATG is now a standard for sib allo MDS patients)
A Multi-center Phase II Study of Selective Depletion of CD45RA+ T Cells From Allogeneic Peripheral Blood Stem Cell Grafts for the Prevention of GVHD [NCT00914940]Phase 241 participants (Actual)Interventional2009-12-17Terminated(stopped due to Did not reach one of the primary endpoints of decreased total acute GVHD)
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)
Preliminary Clinical Study of Autologous T Cells Modified Chimeric Antigen Receptor (CAR) Targeting GPC3 for the Treatment of Recurrent or Refractory Lung Squamous Cell Carcinoma [NCT02876978]Phase 120 participants (Anticipated)Interventional2016-03-31Recruiting
A Phase I Clinical Trial of T-Cells Targeting CD19 and CD22 for Subjects With CD19-positive Acute Lymphoblastic Leukemia [NCT04303520]Phase 120 participants (Anticipated)Interventional2018-05-03Recruiting
A Randomized, Multi-Center Phase III Trial Comparing Two Conditioning Regimens (CloFluBu and BuCyMel) in Children With Acute Myeloid Leukemia Undergoing Allogeneic Stem Cell Transplantation. [NCT05477589]Phase 3170 participants (Anticipated)Interventional2022-06-07Recruiting
Romidepsin Therapy in Conditioning and Maintenance in Patients With T-Cell Malignancies Receiving Allogeneic Stem Cell Transplant [NCT02512497]Phase 110 participants (Anticipated)Interventional2017-12-08Recruiting
Safety and Efficacy Study of Busulfan/FLAG Conditioning Regimen in Patients With Relapsed/Refractory Acute Leukemia Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation [NCT02784561]Phase 480 participants (Anticipated)Interventional2016-07-31Not yet recruiting
An Open-label, Randomized, Phase 3 Study Of Inotuzumab Ozogamicin (Cmc-544) Administered In Combination With Rituximab Compared To A Defined Investigator's Choice Therapy In Subjects With Relapsed Or Refractory, Cd22- Positive, Follicular B-cell Non Hodgk [NCT00562965]Phase 329 participants (Actual)Interventional2007-11-30Terminated(stopped due to See termination reason in detailed description.)
Reduced Dose Fludarabine/Cyclophosphamide Lymphodepletion Before Tumor-Infiltrating Lymphocyte Therapy With Lifileucel in Metastatic Melanoma [NCT06151847]Phase 212 participants (Anticipated)Interventional2024-01-27Recruiting
A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) [NCT05600426]Phase 3234 participants (Anticipated)Interventional2023-01-25Recruiting
A PHASE IB STUDY OF IMMUNOTHERAPY WITH EX VIVO PRE-ACTIVATED AND EXPANDED CB-NK CELLS IN COMBINATION WITH CETUXIMAB, IN COLORECTAL CANCER PATIENTS WITH MINIMAL RESIDUAL DISEASE (MRD) [NCT05040568]Phase 115 participants (Anticipated)Interventional2022-02-28Recruiting
A Phase I/II Study of Vaccination Against Minor Histocompatibility Antigens HA1 or HA2 After Allogeneic Stem Cell Transplantation for Advanced Hematologic Malignancies [NCT00943293]Phase 11 participants (Actual)Interventional2003-05-31Terminated(stopped due to Poor accrual; did not enter phase 2)
Clinical Trial Evaluating the Safety and Activity of Mutant KRAS G12V-specific TCR Transduced T Cell Therapy for Advanced Pancreatic Cancer [NCT04146298]Phase 1/Phase 230 participants (Anticipated)Interventional2021-10-21Recruiting
Prospective Phase II Clinical Trial of Myeloablative Conditioning Regimen With Fludarabine and Busulfan Plus 400 cGy Total Body Irradiation for Hematologic Malignancies [NCT00815568]Phase 2114 participants (Anticipated)Interventional2008-08-31Recruiting
Azacitidine Maintenance Therapy After Allogeneic Stem Cell Transplantation for Chronic Myelogenous Leukemia (CML) [NCT00813124]Phase 224 participants (Actual)Interventional2008-12-31Completed
Reduced Intensity Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) Supplemented With Donor Natural Killer (NK) Cell Infusions in Patients With High Risk Myeloid Malignancies Who Are Unsuitable for Fully Myeloablative Transplantation [NCT00303667]Phase 1/Phase 250 participants (Actual)Interventional2005-01-31Completed
SJCAR19: A Phase I/II Study Evaluating SJCAR19 (CD19-Specific CAR Engineered Autologous T-Cells) in Pediatric and Young Adult Patients ≤ 21 Years of Age With Relapsed or Refractory CD19+ Acute Lymphoblastic Leukemia [NCT03573700]Phase 1/Phase 226 participants (Actual)Interventional2018-07-24Active, not recruiting
A Genetic Risk-Stratified, Randomized Phase II Study of Four Fludarabine/Antibody Combinations for Patients With Symptomatic, Previously Untreated Chronic Lymphocytic Leukemia [NCT00602459]Phase 2418 participants (Actual)Interventional2008-01-15Completed
A Phase II Study Using Short-Term Cultured, Autologous Tumor-Infiltrating Lymphocytes Following a Lymphodepleting Regimen in Metastatic Cancers Plus the Administration of Pembrolizumab [NCT01174121]Phase 2332 participants (Anticipated)Interventional2010-08-26Recruiting
Allogeneic Stem Cell Transplantation With Rituximab Containing Nonablative Conditioning Regimen for Advanced/Recurrent Mantle Cell Lymphoma [NCT00525876]49 participants (Actual)Interventional2005-01-31Completed
Autologous Chimeric Antigen Receptor Engineered T Cell Immunotherapy for Desensitization in Patients Awaiting Kidney Transplantation [NCT06056102]Phase 120 participants (Anticipated)Interventional2024-01-05Not yet recruiting
Delayed Tolerance Through Mixed Chimerism [NCT05900401]Phase 1/Phase 220 participants (Anticipated)Interventional2023-10-01Recruiting
A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Myelodysplastic Syndrome [NCT05617625]Phase 250 participants (Anticipated)Interventional2024-03-31Suspended(stopped due to The study has been put on hold while pending CMS approval)
An Open-label, Non-randomized, Prospective Trial to Evaluate the Effect of Renal Function Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics in Patients With AML or MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT05534620]Phase 136 participants (Anticipated)Interventional2023-11-30Not yet recruiting
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
Allogeneic Peripheral Blood Progenitor Cell Transplantation in Patients With Incurable Solid Tumors: A Phase I Study [NCT00006126]Phase 10 participants (Actual)Interventional1999-09-30Withdrawn(stopped due to Unable to accrue subjects.)
Minimal Ablation and Cellular Immune Therapy of Chronic Lymphocytic Leukemia, Prolymphocytic Leukemia, Low-Grade Non-Hodgkin's Lymphoma, and Mantle Cell Lymphoma With Allogeneic Donor Stem Cells [NCT00006252]Phase 247 participants (Actual)Interventional2001-02-28Completed
Purine-Analog-Containing Non-Myeloablative Allogeneic Stem Cell Transplantation for Treatment of Hematologic Malignancies and Severe Aplastic Anemia [NCT00006379]Phase 258 participants (Actual)Interventional2000-06-30Completed
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
A Pilot Study of Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin for Treatment of CD25 Positive Hodgkin's Disease After Fludarabine and Cyclophosphamide [NCT00389506]0 participants (Actual)Interventional2006-09-30Withdrawn
A Phase I Clinical Trial of T-Cells Targeting B-Cell Maturation Antigen for Subjects With BCMA-positive Multiple Myeloma [NCT03093168]Phase 110 participants (Anticipated)Interventional2017-02-15Recruiting
Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation Following Reduced Intensity Conditioning in Treating Patients With Multiple Myeloma [NCT00802568]Phase 248 participants (Anticipated)Interventional2007-04-30Completed
A Pilot Study of Reduced Intensity Allogeneic Stem Cell Transplantation in Patients With Chronic Myeloid Leukemia (CML) and Adoptive Cellular Immunotherapy Only in Patients With Persistent Disease and Matched Family Donors [NCT00531310]Phase 25 participants (Actual)Interventional2003-01-31Terminated(stopped due to Poor accrual)
A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB) [NCT00881556]Early Phase 13 participants (Actual)Interventional2009-08-20Terminated
Phase I Study of Escalating Doses of Radiation Therapy Using Helical Tomotherapy in Combination With Fludarabine (FLU) and Melphalan (MEL) as a Preparative Regimen for Allogeneic Hematopoietic Stem Cell (HSC) Transplantation in Patients With Advanced and [NCT00800150]Phase 16 participants (Actual)Interventional2008-11-30Terminated(stopped due to Protocol objective could not be met. A new study with amended eligibility criteria will be developed.)
A Safety and Efficacy Study of Autologous Chimeric Switch Receptor Engineered T Cells Redirected to PD-L1 in Patients With Recurrent Glioblastoma Multiforme [NCT02937844]Phase 120 participants (Anticipated)Interventional2016-07-31Recruiting
Phase II Study of Early Allogeneic Blood Stem Cell Transplantation During Induction-chemotherapy Induced Aplasia in High-risk Acute Myeloid Leukemia [NCT00188136]Phase 232 participants (Actual)Interventional2002-08-31Completed
Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders [NCT00533923]Phase 225 participants (Anticipated)Interventional2002-12-31Completed
A Phase II Trial of Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic [NCT00629798]Phase 264 participants (Actual)Interventional2008-02-12Completed
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.)
Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study [NCT00160355]Phase 14 participants (Actual)Interventional2005-05-31Completed
Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia [NCT00455312]Phase 2/Phase 336 participants (Actual)Interventional2007-08-31Completed
High Dose Immune Suppression With Hematopoietic Stem Cell Support in Refractory Vasculitis, Necrotizing Vasculitis, Neurovascular Behcet's Disease, and Sjogren's Syndrome [NCT00278512]Phase 17 participants (Actual)Interventional2003-08-31Terminated(stopped due to No participant enrolled over five years. No plan to continue the study.)
Allogeneic Natural Killer Cells in Patients With Relapsed Acute Myelogenous Leukemia [NCT00274846]Phase 221 participants (Actual)Interventional2005-03-31Completed
A Phase II Study of R-FND, Followed by Zevalin Radioimmunotherapy, and Subsequent Maintenance Rituximab for Advanced Stage Follicular Lymphoma With High-Risk Features [NCT00290511]Phase 249 participants (Actual)Interventional2004-06-29Completed
Mini-Allogeneic Peripheral Blood Progenitor Cell Transplantation For Recurrent or Metastatic Breast Cancer [NCT00429572]Phase 219 participants (Actual)Interventional1998-01-31Completed
A Phase I/II Study of Llme Treated Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematological Malignancies [NCT00429416]Phase 1/Phase 214 participants (Actual)Interventional2004-03-31Completed
Adaptive Randomization of Fludarabine-Melphalan Versus Fludarabine-Cyclophosphamide Conditioning Regimen in Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation Using HLA-Matched Related Donor for Metastatic Renal Cell Carcinoma [NCT00429026]Phase 240 participants (Actual)Interventional2004-01-31Terminated(stopped due to Slow accrual, study terminated.)
Phase II Clinical Trial of Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis [NCT01047072]Phase 20 participants (Actual)InterventionalWithdrawn
Peripheral Blood Stem Cell Allotransplantation for Hematological Malignancies Using a Positive Stem Cell Selection Technique for T Cell Depletion, Followed by Delayed T Cell Add-Back [NCT00378534]Phase 2116 participants (Actual)Interventional2006-09-30Completed
Pilot Study Of Haplo-Identical Natural Killer Cell Transplantation For Acute Myeloid Leukemia [NCT00187096]49 participants (Actual)Interventional2005-09-30Completed
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Active Idiopathic Inflammatory Myopathy [NCT06154252]Phase 1/Phase 218 participants (Anticipated)Interventional2023-11-17Recruiting
A Phase 1 Open-label, Single Arm, Multicenter Study Evaluating the Safety and Efficacy of KITE-197 in Subjects With Relapsed or Refractory Large B-cell Lymphoma [NCT06079164]Phase 139 participants (Anticipated)Interventional2023-11-09Recruiting
A Phase 1 Open-label, Multicenter Study Evaluating the Safety of KITE-222, an Autologous Anti-CLL-1 CAR T-cell Therapy, in Subjects With Relapsed/Refractory Acute Myeloid Leukemia [NCT04789408]Phase 140 participants (Anticipated)Interventional2021-07-19Recruiting
Phase Ib Study of Fludarabine, Cytarabine (Ara-C) and Pegylated Erwinase (Pegcrisantaspase) in Patients With Relapsed or Refractory Leukemia [NCT04526795]Phase 162 participants (Anticipated)Interventional2021-04-09Recruiting
A Phase 1/2, Multicenter, Open-Label, Single Arm, Dose Escalation and Expansion Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) P [NCT04240002]Phase 1/Phase 297 participants (Anticipated)Interventional2020-09-04Recruiting
Timed Sequential Busulfan and Post Transplant Cyclophosphamide for Allogeneic Transplantation [NCT02861417]Phase 2204 participants (Actual)Interventional2016-08-05Active, not recruiting
A Prospective, Randomized, Open Label, Phase III Trial of Fludarabine, Cyclophosphamide, and Rituximab vs. Pentostatin, Cyclophosphamide, and Rituximab in Previously Untreated or Treated B-cell Chronic Lymphocytic Leukemia [NCT00254163]Phase 3184 participants (Actual)Interventional2003-12-31Completed
Treatment of Patients With Metastatic Melanoma Using a Transplant of Autologous Lymphocytes Reactive With Tumor Following a Myeloablative Lymphocyte Depleting Regimen of Chemotherapy, Total Body Irradiation and Reconstitution With CD34+ Cells [NCT00096382]Phase 234 participants (Actual)Interventional2004-09-30Completed
A Phase II Study of Bortezomib in Combination With Rituximab, Fludarabine, Mitoxantrone, and Dexamethasone (VR-FND) for Relapsed or Refractory Follicular Lymphoma [NCT00510887]Phase 214 participants (Actual)Interventional2007-01-31Terminated(stopped due to Low accrual.)
Study of Allogeneic Transplantation in Patients With Cutaneous T-Cell Lymphoma (CTCL) [NCT00506129]Phase 233 participants (Actual)Interventional2003-09-30Completed
Conditioning for Hematopoietic Cell Transplantation With Fludarabine Plus Targeted IV Busulfan and GVHD Prophylaxis With Thymoglobulin, Tacrolimus and Methotrexate in Patients With Myeloid Malignancies [NCT01056614]Phase 223 participants (Actual)Interventional2004-09-30Completed
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
Imatinib Mesylate, Busulfan, Fludarabine, Antithymocyte Globulin and Allogeneic Stem Cell Transplantation for Chronic Myelogenous Leukemia [NCT00499889]Phase 242 participants (Actual)Interventional2003-02-28Terminated(stopped due to Support issue.)
Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (AML/MDS/JMML) [NCT01020539]Phase 118 participants (Actual)Interventional2002-09-11Completed
Phase II Trial of Rituximab/Fludarabine Followed by CAMPATH-1H in the First-Line Treatment of Patients With Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) [NCT00193466]Phase 240 participants Interventional2002-01-31Completed
Phase II Study Evaluating The Infusion Of Autologous Tumor-Infiltrating Lymphocytes (TILs) And Low-Dose Interleukin-2 (IL-2) Therapy Following A Preparative Regimen Of Non-Myeloablative Lymphodepletion Using Cyclophosphamide And Fludarabine In Patients Wi [NCT01883323]Phase 212 participants (Actual)Interventional2013-06-30Completed
TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies [NCT03849651]Phase 2140 participants (Anticipated)Interventional2019-01-31Recruiting
A Phase II Study of Ibrutinib in Combination With Fludarabine, Cyclophosphamide, and Rituximab (iFCR) in Previously Untreated, Younger Patients With Chronic Lymphocytic Leukemia [NCT02251548]Phase 285 participants (Actual)Interventional2014-10-31Active, not recruiting
A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation [NCT01962415]Phase 2100 participants (Anticipated)Interventional2014-02-04Recruiting
Allogeneic Bone Marrow Transplantation With Matched Unrelated Donors for Patients With Hematologic Malignancies Using a Preparative Regimen of Busulfan, Cyclophosphamide, and Fludarabine [NCT00208923]Phase 255 participants (Anticipated)Interventional1998-07-31Completed
Phase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency [NCT00228852]Phase 1/Phase 20 participants InterventionalCompleted
Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia [NCT01949129]Phase 2/Phase 31,000 participants (Anticipated)Interventional2013-04-30Recruiting
A Multicenter Clinical Study on the Safety and Efficacy of CAR-T in the Treatment of Relapsed / Refractory Non Hodgkin's Lymphoma [NCT04666168]200 participants (Anticipated)Interventional2020-10-22Recruiting
Multicenter Clinical Study on the Safety and Effectiveness of CAR-T in the Treatment of Relapsed/Refractory Plasma Cell Tumors [NCT04665076]60 participants (Anticipated)Interventional2020-10-22Recruiting
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 Phase 1, Multicenter, Open-Label Study Of CC-97540 (BMS-986353), CD19-Targeted Nex-T Chimeric Antigen Receptor (CAR) T Cells, in Participants With Severe, Refractory Systemic Lupus Erythematosus (SLE) [NCT05869955]Phase 143 participants (Anticipated)Interventional2023-09-13Recruiting
Phase II Study of Metastatic Cancer That Expresses NY-ESO-1 Using Lymphodepleting Conditioning Followed by Infusion of Anti-NY ESO-1 Murine TCR-Gene Engineered Lymphocytes [NCT01967823]Phase 211 participants (Actual)Interventional2013-10-24Completed
Clinical Study to Assess Efficacy and Safety of LN-145/LN-145-S1 (Autologous Centrally Manufactured Tumor Infiltrating Lymphocytes) Across Multiple Tumor Types [NCT03449108]Phase 230 participants (Actual)Interventional2018-04-27Active, not recruiting
Personalized NK Cell Therapy in CBT [NCT02727803]Phase 2100 participants (Anticipated)Interventional2016-05-19Recruiting
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
Pilot Study of Crenolanib Combined With Standard Salvage Chmetherapy in Subjects With Relapsed/Refractory Acute Myeloid Leukemia [NCT02626338]Phase 1/Phase 216 participants (Actual)Interventional2016-02-29Completed
HPV-16/18 E6/E7-Specific T Lymphocytes in Patients With Relapsed HPV-Associated Cancers [NCT02379520]Phase 132 participants (Anticipated)Interventional2015-09-30Active, not recruiting
Subcutaneous MabCampath® (Alemtuzumab) and Oral Fludara® (Fludarabinephosphate) for the Treatment of Refractory or Relapsed Chronic Lymphocytic Leukemia in 2nd or 3rd Line of Treatment: A Pilot Trial (FLUSALEM) for the Determination of Safety, Efficacy an [NCT00565981]Phase 210 participants (Actual)Interventional2004-03-31Completed
Randomized Trial Comparing the Efficacy of Chlorambucil to Fludarabine in Patients With Advanced Waldenström Macroglobulinemia,Lymphoplasmacytic Lymphoma or Splenic Marginal Zone Lymphoma [NCT00566332]Phase 3414 participants (Actual)Interventional2001-06-30Completed
The VICTORY Study: A Phase I Study of Venetoclax in Combination With Non-myeloablative Conditioning Allogeneic Haematopoietic Stem Cell Transplantation [NCT05005299]Phase 118 participants (Anticipated)Interventional2022-06-08Recruiting
Sequential Myeloablative Autologous Stem Cell Transplantation Followed by Allogeneic Non-Myeloablative Stem Cell Transplantation for Patients With Poor Risk Lymphomas [NCT01181271]Phase 242 participants (Actual)Interventional2010-08-31Completed
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
An Adaptive, Comparative, Randomized, Parallel-group, Multi Center, Phase Ib Study of Subcutaneous (SC) Rituximab Versus Intravenous (IV) Rituximab Both in Combination With Chemotherapy (Fludarabine and Cyclophosphamide), in Patients With Previously Untre [NCT01292603]Phase 1240 participants (Actual)Interventional2011-04-18Completed
Phase I Study of Bortezomib With or Without Total Marrow Irradiation (TMI) Using Intensity Modulated Radiation Therapy (IMRT) in Combination With Fludarabine (FLU) and Melphalan (MEL) as a Preparative Regimen for Allogeneic Hematopoietic Stem Cell (HSC) T [NCT01163357]Phase 118 participants (Actual)Interventional2011-01-28Active, not recruiting
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents [NCT02014506]Phase 1/Phase 230 participants (Anticipated)Interventional2017-01-31Recruiting
A Reduced Intensity Conditioning Regimen and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematologic Malignancies. [NCT00739141]Phase 286 participants (Actual)Interventional2008-08-12Completed
A Randomized Comparison of Fludarabine in Combination With Cytarabine Versus High -Dose Cytarabine in Post-remission Therapy for AML1-ETO Acute Myeloid Leukemia [NCT02024308]Phase 462 participants (Anticipated)Interventional2010-11-30Recruiting
Anti-CD19 Universal CAR-T Cells for CD19+ B Cell Hematologic Malignancies: a Multi-center, Uncontrolled Trial [NCT04264039]Early Phase 130 participants (Anticipated)Interventional2020-04-01Not yet 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
[NCT00005898]Phase 1/Phase 230 participants Interventional2000-02-29Completed
Allogeneic Transplantation Using Mini-Conditioning for Treatment of Stage IV Breast Cancer [NCT00006261]Phase 20 participants (Actual)Interventional2000-05-31Withdrawn
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
Protocol for Patients With High Risk (Resistant, Refractory, Relapsed or Adverse Cytogenetic) AML [NCT00005863]Phase 30 participants Interventional1998-08-31Completed
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
A Phase I Pilot Trial to Evaluate the Toxicity of Epstein-Barr Virus Specific T-Lymphocytes or Peripheral Blood Mononuclear Cells for the Treatment of Relapsed/Refractory Hodgkin's Disease [NCT00006100]Phase 10 participants Interventional2000-04-30Active, not recruiting
Randomized Trial of MCD Versus FMD in Untreated Advanced Follicular Lymphoma [NCT00006250]Phase 3500 participants (Anticipated)Interventional2000-05-31Active, not recruiting
A Phase 1, Open-Label, Dose-Finding Study of BMS-986453, Dual Targeting BCMAxGPRC5D Chimeric Antigen Receptor T Cells, in Participants With Relapsed and/or Refractory Multiple Myeloma [NCT06153251]Phase 1130 participants (Anticipated)Interventional2024-01-08Not yet recruiting
An Open-label, Multicenter Phase 2 Study Evaluating the Efficacy and Safety of CRG-022, a CD22-directed Autologous Chimeric Antigen Receptor (CAR) T-cell Therapy in Patients With Relapsed/Refractory Large B-Cell Lymphoma After CD19-directed CAR T-cell The [NCT05972720]Phase 2123 participants (Anticipated)Interventional2023-08-01Recruiting
A Phase 2, Open-Label, Multi-Center Study of Innate Cell Engager AFM13 in Combination With Allogeneic Natural Killer Cells (AB-101) in Subjects With Recurrent or Refractory Hodgkin Lymphoma and CD-30 Positive Peripheral T-Cell Lymphoma [NCT05883449]Phase 2154 participants (Anticipated)Interventional2023-10-10Recruiting
A Phase II Study Using the Administration of Autologous T-Cells Engineered Using the Sleeping Beauty Transposon/Transposase System to Express T-Cell Receptors Reactive Against Mutated Neoantigens in Patients With Metastatic Cancer [NCT04102436]Phase 2210 participants (Anticipated)Interventional2024-01-03Not yet recruiting
An Open, Uncontrolled, Multicenter Clinical Trial to Explore the Safety, Efficacy, and Remission Phase of Chimeric Antigen Receptor T Cell (CAR-T) in the Treatment of Relapsed Refractory (R/R) Multiple Myeloma (MM) [NCT04626752]Early Phase 150 participants (Anticipated)Interventional2020-04-01Recruiting
Fludarabine, BBR 2778 (Pixantrone) and Rituximab (FP-R) vs Fludarabine and Rituximab (F-R) for Relapsed or Refractory Indolent Non-Hodgkin's Lymphoma [NCT00577161]Phase 30 participants (Actual)Interventional2007-09-30Withdrawn(stopped due to closed to enrollment)
MINIALO-VELCADE2005: A Phase II National, Open-label, Multicenter, no Controlled Study of Treated With Bortezomib (Velcade) Multiple Myeloma Patients Pre and Post Allogeneic Haematopoietic Progenitor Cell Transplant With no Myeloablative Conditioning [NCT00564200]Phase 230 participants (Anticipated)Interventional2007-11-30Completed
Randomized Phase-III Trial Comparing Fludarabine and Cyclophosphamide Plus Rituximab (FCR) to FC and MabCampath (FCCam) for Previously Untreated Fit Patients With Chronic Lymphocytic Leukemia (CLL) [NCT00564512]Phase 3178 participants (Actual)Interventional2007-11-30Completed
Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome Using Reduced Intensity Busulfan and Fludarabine Conditioning [NCT00475020]Phase 263 participants (Actual)Interventional2006-01-04Completed
A Phase I Study Evaluating The Use Of Rft5-Dga To Deplete Alloreactive Cells For Patients With Fanconi Anemia After Haploidentical Stem Cell Transplantation [NCT00586274]Phase 11 participants (Actual)Interventional2002-03-31Terminated(stopped due to Poor accrual)
A Phase I Study to Investigate the Safety and Clinical Activity of Idelalisib in Combination With Chemotherapeutic Agents, Immunomodulatory Agents and Anti-CD20 mAb in Subjects With Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma, Mantle Cell [NCT01088048]Phase 1241 participants (Actual)Interventional2010-03-25Completed
Assessment of Safety and Recommended Phase 2 Dose of Autologous T Cells Engineered With an Affinity-enhanced TCR Targeting NYESO1 and LAGE1a, and Co-expressing CD8α (GSK3901961) in Participants With NYESO1 and/or LAGE1a Positive Previously Treated Advance [NCT06048705]Phase 17 participants (Actual)Interventional2021-03-09Terminated(stopped due to The study was terminated due to a change in GSK's R&D priorities.)
A Phase I/II, Single Arm, Multi-center Study Evaluating the Safety and Efficacy of HY004 in Adult Patients With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (r/r B-ALL) [NCT06009107]Phase 1/Phase 250 participants (Anticipated)Interventional2023-10-18Not yet recruiting
A Phase I, Open-Label, Multicenter Study of FT536 as Monotherapy and in Combination With Monoclonal Antibodies in Subjects With Advanced Solid Tumors [NCT05395052]Phase 15 participants (Actual)Interventional2022-05-31Terminated(stopped due to This study was terminated by the Sponsor.)
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)
A Phase I, Open-Label, Multicenter Study of FT538 in Combination With Monoclonal Antibodies in Subjects With Advanced Solid Tumors [NCT05069935]Phase 116 participants (Actual)Interventional2021-10-15Terminated(stopped due to This study was terminated by the Sponsor.)
Clinical Study of ssCART-19 Cells in Patients With CD19 Positive Relapsed or Refractory Acute Lymphoblastic Leukemia [NCT04825496]Phase 118 participants (Anticipated)Interventional2021-04-09Recruiting
A Phase I, Open-Label, Multicenter Study of FT538 as Monotherapy in Relapsed/Refractory Acute Myelogenous Leukemia and in Combination With Monoclonal Antibodies in Relapsed/Refractory Multiple Myeloma [NCT04614636]Phase 142 participants (Actual)Interventional2020-10-17Terminated(stopped due to This study was terminated by the Sponsor.)
A Phase I, Open-Label, Multicenter Study of FT516 in Combination With Monoclonal Antibodies in Subjects With Advanced Solid Tumors [NCT04551885]Phase 112 participants (Actual)Interventional2020-09-07Terminated(stopped due to This study was terminated by the Sponsor.)
Pilot Study of Anakinra to Mitigate CAR-T Toxicity in Subjects With Relapsed or Refractory Large B-Cell Lymphoma [NCT04432506]Phase 220 participants (Anticipated)Interventional2020-07-27Active, not recruiting
A Phase I Clinical Trial Testing the Safety of IL-21-Expanded, Off-the-shelf, Third-party Natural Killer Cells (KDS-1001) for the Induction of Relapsed/Refractory Acute Myeloid Leukemia [NCT04220684]Phase 121 participants (Anticipated)Interventional2020-06-01Recruiting
A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion Study to Evaluate the Safety and Clinical Activity of PBCAR269A, With or Without Nirogacestat, in Study Participants With Relapsed/Refractory Multiple Myeloma [NCT04171843]Phase 148 participants (Actual)Interventional2020-04-30Terminated(stopped due to Study was terminated due to lack of sufficient therapeutic effect)
A Phase II Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia [NCT03164057]Phase 2206 participants (Actual)Interventional2017-06-15Active, not recruiting
A Phase II Trial of Pembrolizumab in Combination With Chimeric Antigen Receptor Therapy in Patients With Relapsed/Refractory Primary Mediastinal B-cell Lymphoma [NCT05934448]Phase 235 participants (Anticipated)Interventional2023-10-31Not yet recruiting
Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressing Autologous T Cells as an Immunotherapy for Children With Solid Tumors (CARE) [NCT04715191]Phase 124 participants (Anticipated)Interventional2024-01-03Not yet recruiting
Transplantation Using Reduced Intensity Approach for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow [NCT02757885]Phase 210 participants (Actual)Interventional2019-07-10Completed
A Phase 1b/2 Study of IPI-145 in Combination With Fludarabine, Cyclophosphamide, and Rituximab (iFCR) in Previously Untreated, Younger Patients With Chronic Lymphocytic Leukemia. [NCT02158091]Phase 1/Phase 232 participants (Actual)Interventional2014-06-27Active, not recruiting
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
A Prospective Randomized Study of Cell Transfer Therapy for Metastatic Melanoma Using Short-Term Cultured Tumor-Infiltrating Lymphocytes Plus IL-2 Following Either a Non-Myeloablative Lymphocyte Depleting Chemotherapy Regimen Alone or in Conjunction With [NCT01319565]Phase 2102 participants (Actual)Interventional2011-03-24Active, not recruiting
A Phase 1/2 Single-center Study Evaluating the Safety and Efficacy of TRAC and Power3 Genes Knock-out Allogeneic CD19-targeting CAR-T Cell (ATHENA) Therapy in Adults With Refractory/Relapsed B-cell Non-Hodgkin Lymphoma [NCT06014073]Phase 1/Phase 230 participants (Anticipated)Interventional2023-09-06Recruiting
BMT-06: Phase II Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Cyclophosphamide and Post-Transplant Cyclophosphamide Conditioning for Partially HLA Mismatched (Haploidentical) Allogeneic Transplantation in Patie [NCT04187105]Phase 227 participants (Anticipated)Interventional2020-01-27Recruiting
Multi-Center Phase II Randomized Controlled Trial of Naïve T Cell Depletion for Prevention of Chronic Graft-Versus-Host Disease in Children and Young Adults [NCT03779854]Phase 268 participants (Anticipated)Interventional2019-08-29Recruiting
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
Phase I Study of Cellular Immunotherapy Using T Cells Lentivirally Transduced to Express a CD123-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR for Patients With CD123+ Relapsed/Refractory Acute Myeloid Leukem [NCT02159495]Phase 131 participants (Actual)Interventional2015-12-15Active, not recruiting
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
Single Patient Protocol: A Phase II Study Using the Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Mutated Neoantigens in a Patient With Metastatic Cancer Plus the Administration of Pembrolizumab [NCT04135092]0 participants Expanded AccessNo longer available
Phase II Trial of Dasatinib With Fludarabine and Rituximab in Relapsed and Refractory Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma [NCT01173679]Phase 210 participants (Actual)Interventional2010-07-31Terminated(stopped due to Slow accrual)
An Open Label Clinical Trial to Evaluate the Safety and Efficacy of CT103A Cells for the Treatment of Relapsed/Refractory Antibody-associated Idiopathic Inflammatory Diseases of the Nervous System [NCT04561557]Early Phase 118 participants (Anticipated)Interventional2020-09-22Recruiting
MT2007-19R: WCC #53 Allogeneic Natural Killer Cells in Patients With Recurrent Ovarian Cancer, Fallopian Tube, and Primary Peritoneal Cancer [NCT00652899]Phase 214 participants (Actual)Interventional2008-03-31Terminated(stopped due to Withdrawn due to toxicity)
Fludarabine and 400 CGY Total Body Irradiation for Recipients of HLA-Matched or Mis-Matched Family or Unrelated Donor Hematopoietic Stem Cell Transplants Who Have Rejected Their First Allogeneic Stem Cell Transplant [NCT00472329]Phase 220 participants (Anticipated)Interventional2007-03-31Recruiting
Phase I/II Trial to Determine the Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Ste [NCT05436418]Phase 1/Phase 2240 participants (Anticipated)Interventional2022-11-18Recruiting
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
Phase II Study of Metastatic Cancer That Expresses NY-ESO-1 Using Lymphodepleting Conditioning Followed by Infusion of Anti-NY ESO-1 TCR-Gene Engineered Lymphocytes [NCT00670748]Phase 245 participants (Actual)Interventional2008-05-29Terminated(stopped due to A more highly selected protocol with ESO TCR opened for pts with melanoma)
Chimeric Antigen Receptor (CAR)-T Cell Therapy for Patients With Hematologic Malignancies [NCT03642626]240 participants (Anticipated)Observational2018-12-18Recruiting
A Prospective Multicenter Pilot Trial to Evaluate the Efficacy of a Treatment With Fludarabine, Cyclophosphamide, Lenalidomide (FCL) for Advanced Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL) Patients. [NCT00727415]Phase 1/Phase 242 participants (Actual)Interventional2008-02-29Completed
"Phase II Study of a Reduced-toxicity Submyeloablative Conditioning Regimen Prior to Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies" [NCT00841724]Phase 282 participants (Actual)Interventional2009-01-31Completed
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
Phase II Study of Fludarabine, Cytarabine (ARA-C) and Erwinase IV in Patients With Relapsed or Refractory Hematologic Malignancies [NCT02718755]Phase 20 participants (Actual)Interventional2018-05-31Withdrawn(stopped due to Problem with drug supply)
A Pilot Study of a Novel Sequential Treatment Utilizing CPX-351 as Salvage Chemotherapy Followed by Allogeneic Stem-Cell Transplantation (SCT) Utilizing a Haplo-cord Graft for Patients With Relapsed or Refractory Leukemia or Myelodysplastic Syndrome [NCT03393611]Phase 114 participants (Actual)Interventional2012-11-30Completed
Autologous and Allogeneic Transplantation for T-Cell Lymphoma: Impact of Campath -1H and Soluble CD52 [NCT00505921]Phase 227 participants (Actual)Interventional2003-03-31Terminated(stopped due to Slow Accrual.)
A Phase 1, Open-Label, Multicenter Study of KYV-101, an Autologous Fully-Human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Refractory Lupus Nephritis [NCT05938725]Phase 112 participants (Anticipated)Interventional2023-04-28Recruiting
A Phase 1 Study Evaluating SC291, a Hypoimmune, Allogeneic CD19-directed CAR T Cell Therapy, in Relapsed and/or Refractory B-cell Malignancies (ARDENT) [NCT05878184]Phase 154 participants (Anticipated)Interventional2023-05-02Recruiting
A Phase 2 Study of SNDX-5613 in Combination With Chemotherapy for Patients With Relapsed or Refractory KMT2A-Rearranged Infant Leukemia [NCT05761171]Phase 278 participants (Anticipated)Interventional2023-11-20Recruiting
A Phase 1b Dose Escalation Study of Metabolically Fit CD19 Chimeric Antigen Receptor (CAR) T Cells With CD34 Selection Markers in Adult Patients With Relapsed or Refractory CD19 B-Cell Non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia/Small Lymphocyti [NCT05702853]Phase 1/Phase 227 participants (Anticipated)Interventional2023-11-06Recruiting
Phase I Study of In Vivo Expansion of Melan-A/MART-1 Antigen-Specific CD8 T Lymphocytes Following Transient Immunosuppression in Patients With Advanced Melanoma [NCT00324623]Phase 18 participants (Actual)Interventional2005-09-30Completed
A Phase II Study of Fludarabine, Velcade and Rituximab for Relapsed or Refractory Follicular Non-Hodgkin Lymphoma: Hoosier Oncology Group LYM08-134 [NCT01186458]Phase 24 participants (Actual)Interventional2010-10-31Terminated(stopped due to Slow accrual)
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
International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia - Incorporating an Embedded Dose Finding Study for Gemtuzumab Ozogamicin in Combination With Induction Chemotherapy [NCT02724163]Phase 3700 participants (Anticipated)Interventional2016-04-30Recruiting
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 Single-Arm, Open-Label Study to Evaluate Safety and Efficacy of B7H3 or HBsAg Targeting CAR-T in Treating Advanced Hepatocellular Carcinoma [NCT05323201]Phase 1/Phase 215 participants (Anticipated)Interventional2022-02-10Recruiting
Anti-CD7 Universal CAR-T Cells for CD7+ T/NK Cell Hematologic Malignancies: a Multi-center, Uncontrolled Trial [NCT04264078]Early Phase 130 participants (Anticipated)Interventional2021-03-01Recruiting
IL-1 Receptor Antagonist to Prevent Severe Chimeric Antigen Receptor T-Cell Related Encephalopathy Syndrome [NCT04205838]Phase 236 participants (Anticipated)Interventional2020-03-04Recruiting
Administration of Her2 Chimeric Antigen Receptor Expressing T Cells for Subjects With Advanced Sarcoma (HEROS) [NCT00902044]Phase 136 participants (Anticipated)Interventional2010-02-11Active, not recruiting
NY-ESO-1 TCR Engineered Adoptive Cell Transfer Therapy With Nivolumab PD-1 Blockade [NCT02775292]Phase 11 participants (Actual)Interventional2017-01-03Completed
Unrelated Donor Hematopoietic Stem Cell Transplantation After Nonmyeloablative Conditioning For Patients With Hematological Malignancies [NCT00627666]Phase 252 participants (Anticipated)Interventional2003-01-31Completed
A Phase I/II Study of Competitive Transfer of αCD19-TCRz-CD28 and αCD19-TCRz-CD137 Chimeric Antigen Receptor T-Cells in Patients With Refractory CD19+ B-lineage Leukemia/Lymphoma [NCT02685670]Phase 1/Phase 220 participants (Anticipated)Interventional2016-02-29Recruiting
Allogeneic Stem Cell Transplant to Induce Mixed Donor Chimerism in Patients With Sickle Cell Disease and Thalassemia [NCT00408447]Phase 253 participants (Actual)Interventional2004-09-30Active, not recruiting
A Phase I, Open-label, Dose Escalation Study to Assess the Safety and Tolerability of FP253 in Combination With Fludarabine Phosphate [NCT00625430]Phase 118 participants (Anticipated)Interventional2008-03-31Recruiting
Allogenic Immunotherapy Based on Natural Killer Cell Adoptive Transfer in Metastatic Gastrointestinal Carcinoma Treated With Cetuximab : a Phase I Trial [NCT02845999]Phase 19 participants (Actual)Interventional2009-11-30Completed
Nonmyeloablative Stem Cell Transplantation With or Without Lenalidomide for Chronic Lymphocytic Leukemia (RV-CLL-PI-0294) [NCT00899431]Phase 239 participants (Actual)Interventional2009-05-06Terminated(stopped due to Terminated per PI's request at the time of continuing review)
Efficacy and Safety of Orelabrutinib, Fludarabine, Cyclophosphamide, and Obinutuzumab (GA-101) (oFCG) in the Treatment of Newly Diagnosed Chronic Lymphocytic Leukemia (CLL) / Small Lymphocytic Lymphoma (SLL) [NCT05322733]Phase 225 participants (Anticipated)Interventional2022-04-15Recruiting
Evaluation of Melphalan+Bortezomib as a Conditioning Regimen for Autologous and Allogeneic Stem Cell Transplants in Multiple Myeloma After Cytoreductive Therapy [NCT00948922]Phase 2124 participants (Actual)Interventional2009-06-18Completed
To Investigate the Safety and Preliminary Efficacy of EBV CAR-T Cells in the Treatment of Relapsed/Refractory EBV-positive Nasopharyngeal Carcinoma [NCT05654077]Early Phase 124 participants (Anticipated)Interventional2022-01-18Recruiting
A Multicenter Clinical Study on the Safety and Effectiveness of CAR-T in the Treatment of Relapsed/Refractory Hodgkin's Lymphoma [NCT04665063]20 participants (Anticipated)Interventional2020-10-22Recruiting
Graft-versus-host Disease Prophylaxis With Post-transplantation Cyclophosphamide and Ruxolitinib in Patients With Myelofibrosis [NCT02806375]Phase 1/Phase 220 participants (Actual)Interventional2016-01-31Completed
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
A Randomized Phase III Trial Comparing the Combination of Fludarabine, BBR 2778 (Pixantrone) and Rituximab (FP-R) With the Combination of Fludarabine and Rituximab (F-R) in the Treatment of Patients With Relapsed or Refractory Indolent Non-Hodgkin's Lymph [NCT00551239]Phase 30 participants (Actual)Interventional2007-08-31Withdrawn
Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia [NCT01423500]Phase 3405 participants (Anticipated)Interventional2007-01-31Active, not recruiting
A Phase II Study to Evaluate the Efficacy and Safety of Oral Fludarabine Phosphate in Combination With Mitoxantrone as First Line Treatment in Follicular NHL [NCT00185445]Phase 262 participants (Actual)Interventional2004-06-30Completed
Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia [NCT01423747]Phase 3400 participants (Anticipated)Interventional2003-07-31Active, not recruiting
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
A Pilot Study of NY-ESO-1c259T Cells in Subjects With Advanced Myxoid/ Round Cell Liposarcoma [NCT02992743]Phase 223 participants (Actual)Interventional2016-12-06Completed
Treatment of Patients With Metastatic Melanoma by Lymphodepleting Conditioning Followed by Infusion of TCR-Gene Engineered Lymphocytes and Subsequent Fowlpox gp100 Vaccination [NCT00085462]Phase 161 participants (Anticipated)Interventional2004-05-31Completed
Propylene Glycol-Free Melphalan HCl (EVOMELA®) in Combination With Fludarabine and Total Body Irradiation Based Reduced Intensity Conditioning for Haploidentical Transplantation [NCT03159702]Phase 243 participants (Anticipated)Interventional2017-12-08Recruiting
An Open Label, Multicenter, Non Randomized Phase II Study to Evaluate Anti-tumor Activity and Safety of a Combination of Fludarabine, Mitoxantrone and Rituximab in Relapsed or Primary Failing Advanced Follicular Non-Hodgkin's Lymphoma. [NCT00169208]Phase 250 participants (Actual)Interventional2001-04-30Completed
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
A Prospective Phase I/IIa, Open-label, Multicenter Trial to Evaluate the Safety and Efficacy of oNKord®, an Off-the-shelf, ex Vivo-cultured Allogeneic NK Cell Preparation, in Subjects With Acute Myeloid Leukemia Who Are in Morphologic Complete Remission W [NCT04632316]Phase 1/Phase 233 participants (Anticipated)Interventional2020-12-08Recruiting
Multicenter Phase I/II Study of Haploidentical Hematopoietic Cell Transplantation With CD3/CD19 Depleted Grafts in Patients With Treatment Refractory Hematologic Malignancies [NCT00202917]Phase 1/Phase 261 participants (Actual)Interventional2004-02-29Completed
Phase II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Cord Blood Transplantation in Pediatric Acute Myeloid Leukemia [NCT00887042]Phase 1/Phase 235 participants (Anticipated)Interventional2006-06-30Active, not recruiting
Dose-escalation Study of Graft-versus-host Disease Prophylaxis With High-dose Post-transplantation Bendamustine in Patients With Refractory Acute Leukemia [NCT02799147]Phase 1/Phase 227 participants (Actual)Interventional2016-06-30Completed
A Phase 1/2 Single Arm Open-Label Clinical Trial of TC-510 In Patients With Advanced Mesothelin-Expressing Cancer [NCT05451849]Phase 1/Phase 26 participants (Actual)Interventional2022-06-21Active, not recruiting
UAB 0775 Phase II Trial of Non-Myeloablative Allogeneic Hematopoietic Cell Transplantation Protocol From HLA Matched Related Donors for The Treatment of Patients With Low Grade B Cell Malignancies [NCT00714259]Phase 2/Phase 35 participants (Actual)Interventional2008-07-31Terminated(stopped due to Accrual goals could not be met within a timely manner)
A Phase I/II Trial in Patients With Metastatic Gastrointestinal Epithelial Cancer Administering Tumor-Infiltrating Lymphocytes in Which the Gene Encoding CISH Was Inactivated Using the CRISPR/Cas9 System [NCT04426669]Phase 1/Phase 220 participants (Anticipated)Interventional2020-05-15Recruiting
Phase I Clinical Trial Evaluating Safety of CD19 CAR-T Cells in Patients With Relapsed or Refractory Acute B-cell Lymphoblastic Leukemia (R/R B-ALL) [NCT04653493]Phase 122 participants (Anticipated)Interventional2021-08-31Not yet recruiting
A Phase I, Single-arm, Open Label, Dose Escalation, Multicenter Study of Off-the-shelf Natural Killer (NK) Cells (SAR445419) in Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML) [NCT05712278]Phase 112 participants (Anticipated)Interventional2023-06-16Recruiting
Phase 1/2 Dose Escalation Study of CD19/CD22 Bicistronic Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory CD19/CD22-expressing B Cell Malignancies [NCT05442515]Phase 1/Phase 2116 participants (Anticipated)Interventional2022-12-28Recruiting
A Randomized Phase III Study of the Treatment of Children and Adolescents With Refractory or Relapsed Acute Myeloid Leukemia [NCT00186966]Phase 3394 participants (Actual)Interventional2002-03-31Completed
A Study of Children With Refractory or Relapsed Acute Lymphoblastic Leukemia (ALLR16) [NCT00187083]Phase 340 participants (Actual)Interventional1997-02-28Completed
Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients With Symptomatic Sickle Cell Disease. (BMTCTN1507) [NCT03263559]Phase 280 participants (Anticipated)Interventional2017-10-03Active, not recruiting
A Randomized Phase II Trial of Fludarabine, Cyclophosphamide and Mitoxantrone (FCM) With or Without Rituximab in Previously Treated Chronic Lymphocytic Leukemia [NCT00337246]Phase 256 participants (Anticipated)Interventional2005-07-31Completed
A Pilot Phase II Trial of Combination Therapy With Fludarabine, Mitoxantrone and Rituximab in Mantle Cell Lymphoma [NCT00183989]Phase 230 participants (Anticipated)Interventional2000-08-31Terminated(stopped due to Insufficient accrual)
[NCT00186797]28 participants Interventional2002-12-31Completed
A Multicenter Study to Confirm the Efficacy and Safety of Fludara i.v. (Fludarabine Phosphate, SH L 573), Administered in 6 Treatment Cycles (1 Treatment Cycle: 5-consecutive Day Dosing, Followed by an Observation Period of 23 Days) in Untreated Chronic L [NCT00220311]Phase 410 participants (Actual)Interventional2000-11-30Completed
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
T Cell Receptor Alpha/Beta T Cell Depleted (α/β TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA) [NCT03579875]Phase 248 participants (Anticipated)Interventional2018-11-13Recruiting
Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases [NCT01962636]200 participants (Anticipated)Interventional2016-12-31Recruiting
The Safety and Efficacy of CART-19 Cells in Relapse and Refractory Patients With CD19+ B-cell Acute Lymphoblastic Leukemia. [NCT02924753]Phase 120 participants (Anticipated)Interventional2016-07-18Recruiting
A Phase 1 Open-Label Dose-Escalation Study of the Safety of Adoptively Transferred Autologous CD8+ T Lymphocytes Targeting HPV-16 E6/E7, HPV-18 E6/E7 and Survivin in Patients With Relapsed or Refractory HPV-related Oropharyngeal Cancers [NCT05582590]Phase 136 participants (Anticipated)Interventional2023-03-31Not yet 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
"High Dose Interleukin-2 (IL-2) Therapy In Lymphodepleted Primed Patients With Metastatic Melanoma" [NCT00085423]Phase 220 participants (Actual)Interventional2004-02-29Completed
Haploidentical Donor NK Cell Adoptive Therapy and Double T Cell Depleted Umbilical Cord Blood Transplantation With Post-Transplant IL-2 Immune Therapy For Refractory Acute Myeloid Leukemia [NCT00871689]Phase 22 participants (Actual)Interventional2009-01-31Terminated(stopped due to Due to graft failure.)
A Phase I/II Study of Fludarabine, Cyclophosphamide, Rituximab, and Vorinostat Followed by Rituximab and Vorinostat Maintenance Therapy in Patients With Previously Untreated B-Cell Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) [NCT00918723]Phase 1/Phase 240 participants (Actual)Interventional2009-06-30Completed
Clinical Study on the Safety and Efficacy of Anti-PSMA CAR NK Cells in Metastatic Castration-resistant Prostate Cancer (mCRPC) [NCT03692663]Early Phase 19 participants (Anticipated)Interventional2018-12-01Recruiting
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies Using Melphalan for T-Cell Tolerization [NCT01350258]Phase 1/Phase 28 participants (Actual)Interventional2011-04-30Terminated(stopped due to Poor accrual)
Phase I/II Pilot Study of Allogeneic Peripheral Blood Stem Cell Infusion For Patients With High Risk Chronic Lymphocytic Leukemia [NCT00002838]Phase 1/Phase 213 participants (Actual)Interventional1995-12-31Completed
Phase I/II Study of HLA-Matched Peripheral Blood Mobilized Hematopoietic Progenitor Cell Transplantation Followed by Allogeneic T-cell Infusion as Adoptive Immunotherapy in Patients With Metastatic Melanoma [NCT00003552]Phase 1/Phase 20 participants Interventional1999-01-31Terminated
Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies [NCT01471067]Phase 133 participants (Actual)Interventional2012-07-13Completed
A Single-center Open-label Phase I Study of ALT-801 for ex Vivo Maturation and in Vivo Retargeting of Haploidentical Natural Killer Cells Delivered Following Fludarabine, Cytarabine, and G-CSF in Patients With Relapsed/Refractory Acute Myeloid Leukemia [NCT01478074]Phase 10 participants (Actual)Interventional2011-11-30Withdrawn(stopped due to The treatment planned was determined to be of low feasibility as no subject was found eligible and able to enroll after screening over 30 subjects)
Observational Study for Evaluation of Quality of Life in Patients Under Treatment for B-Chronic Lymphocytic Leukemia. [NCT00344825]300 participants (Actual)Observational2004-01-31Completed
FLAT: Fludarabine, Cytarabine and Topotecan in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia [NCT00488709]Phase 447 participants (Actual)Interventional2003-05-31Completed
Dose Finding Study of Gelonin Purging of Autologous Stem Cells for Transplantation of Patients With AML/MDS in First or Subsequent Remission [NCT00043810]Phase 1/Phase 23 participants (Actual)Interventional2002-07-31Terminated
Multicenter, Phase 2 Clinical Trial Evaluating the Efficacy and Tolerability of Induction and Consolidation Chemotherapy Comprising Fludarabine, Cytarabine, and Attenuated-dose Idarubicin in Elderly Patients With AML(Acute Myeloid Leukemia) [NCT01247493]Phase 2108 participants (Actual)Interventional2007-06-30Completed
Allogenic CD19-targeting Chimeric Antigen Receptor γδT Cells Therapy in Patients With Relapsed or Refractory B-cell Non-Hodgkin's Lymphoma [NCT05554939]Phase 1/Phase 230 participants (Anticipated)Interventional2022-12-11Recruiting
FLAG-IDA Chemotherapy Induction Follow by Intensive Chemotherapy Postremission +/- Autologous Hemopoietic Stem Cell Transplantation or Bone Marrow Transplantation in Patients With High Risk Myelodysplastic Syndromes or Secondary Acute Myeloblastic Leukemi [NCT00487448]Phase 4200 participants (Anticipated)Interventional1998-07-31Completed
Infusion of Allogeneic, 3rd Party CD19-specific T Cells for Patients With Refractory CD19+ B-Lineage Lymphoid Malignancies [NCT02274506]Phase 10 participants (Actual)Interventional2014-10-20Withdrawn(stopped due to PI's response to ePAAC, has withdrawn the protocol acknowledging the PI's action.)
Phase I/II Randomized Trial of Cord Blood-derived NK Cells Genetically Engineered With NY-ESO-1 TCR/IL-15 Cell Receptor for Relapsed/Refractory Multiple Myeloma [NCT06066359]Phase 1/Phase 244 participants (Anticipated)Interventional2023-11-30Recruiting
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
A Phase 1 and Pharmacokinetic Study of Uproleselan (GMI-1271, NSC #801708) in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia Myelodysplastic Syndrome or Mixed Phenotype Acute Leukemia That Expresses E-Selectin Ligand [NCT05146739]Phase 118 participants (Anticipated)Interventional2023-12-23Recruiting
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
A Phase I Trial of Lymphodepletion Plus Adoptive Cell Therapy With High-Dose IL-2 in Adolescent and Young Adult Patients With Soft Tissue Sarcoma [NCT04052334]Phase 110 participants (Actual)Interventional2019-09-27Active, not recruiting
NON-T-CELL DEPLETED HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING [NCT00521430]30 participants (Anticipated)Interventional2004-04-30Completed
Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation [NCT00516152]Phase 236 participants (Anticipated)Interventional2002-11-30Completed
A Phase II Study of Fludarabine + Rituximab Induction Followed by Alemtuzumab (Campath-1H, NSC #715969, IND #10864) Administered Subcutaneously as Consolidation in Untreated Patients With B-Cell Chronic Lymphocytic Leukemia [NCT00098670]Phase 2102 participants (Actual)Interventional2004-10-31Completed
T Cell Therapy for Patients With Metastatic Renal Cell Carcinoma [NCT02926053]Phase 16 participants (Anticipated)Interventional2016-12-31Recruiting
[NCT00529880]Phase 219 participants (Anticipated)Interventional2004-12-31Recruiting
A Phase 1 Study of NKX101, an Activating Chimeric Receptor Natural Killer Cell Therapy, in Subjects With Hematological Malignancies or Dysplasias [NCT04623944]Phase 190 participants (Anticipated)Interventional2020-09-21Recruiting
Phase I Study of the Administration of T Lymphocytes Co-Expressing the CD30 Chimeric Antigen Receptor (CAR) and CCR4 for Relapsed/Refractory CD30+ Hodgkin Lymphoma and Cutaneous T-Cell Lymphoma [NCT03602157]Phase 159 participants (Anticipated)Interventional2018-12-12Recruiting
A Phase I Clinical Trial of Cyclophosphamide Followed by Intravenous and Intraperitoneal Infusion of Autologous T Cells Genetically Engineered to Secrete IL-12 and to Target the MUC16ecto Antigen in Patients With Recurrent MUC16ecto+ Solid Tumors [NCT02498912]Phase 118 participants (Actual)Interventional2015-08-31Active, not recruiting
A Phase 1/2 Multicenter Study Evaluating the Safety and Efficacy of KTE-C19 in Adults With Refractory Aggressive Non-Hodgkin Lymphoma [NCT02348216]Phase 1/Phase 2307 participants (Actual)Interventional2015-04-21Completed
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.)
Autologous T Cells With a Chimeric Antigen Receptor in Patients With CD19-positive Malignant B Cell Leukemia and Lymphoma [NCT02933775]Phase 145 participants (Anticipated)Interventional2016-10-31Not yet recruiting
Prospective Randomized Study to Compare Efficacy and Safety of RFC-Lite (Rituximab, Fludarabine, Cyclophosphamide) Regimen With LR (Rituximab, Chlorambucil) as a First-Line Therapy in Patients With B-Cell Chronic Lymphocytic Leukemia and Unfavorable Somat [NCT01283386]Phase 426 participants (Actual)Interventional2011-04-27Terminated
Benadamustine, Fludarabine and Busulfan Conditioning in Recipients of Haploidentical Stem Cell Transplantation (FluBuBe) [NCT04942730]Phase 240 participants (Anticipated)Interventional2021-01-21Recruiting
A Phase 1 Open-Label, Multi-Center Study of PSMA Targeted Genetically Modified Chimeric Antigen Receptor T Cells in Patients With Metastatic Castration Resistant Prostate Cancer [NCT04227275]Phase 116 participants (Actual)Interventional2019-11-22Terminated(stopped due to Based on the safety events and evidence of biologic activity without sustained clinical responses the Sponsor finds the risk/benefit analysis unfavorable for patients and has terminated the study.)
A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT02143830]Phase 270 participants (Anticipated)Interventional2014-04-30Recruiting
Reduced Intensity Conditioning and Transplantation of Partially HLA-Mismatched Peripheral Blood Stem Cells for Patients With Hematologic Malignancies [NCT02581007]Phase 225 participants (Actual)Interventional2015-10-26Completed
Ma-Spore ALL 2010 Study [NCT02894645]Phase 4500 participants (Anticipated)Interventional2008-10-31Recruiting
Study to Evaluate the Efficacy and Safety of Relmacabtagene Autoleucel (Relma-cel) as First-Line Therapy in Adult Participants With High-Risk Large B-Cell Lymphoma [NCT05590221]Phase 220 participants (Anticipated)Interventional2023-01-03Recruiting
A Multi-Center, Open-Label, Phase 1/2 Clinical Trial to Evaluate the Safety and Anti-Tumor Activity of AB-101 Monotherapy and AB-101 With Immunotherapy in Patients With Relapsed/Refractory Non-Hodgkin Lymphoma of B-Cell Origin. [NCT04673617]Phase 1/Phase 2108 participants (Anticipated)Interventional2021-03-29Recruiting
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
A Phase I Dose Escalation Study of Lenalidomide (Revlimid) in Combination With Fludarabine-Rituximab (Rituxan) for Previously Untreated CLL/SLL [NCT00543114]Phase 19 participants (Actual)Interventional2007-10-31Terminated(stopped due to lack of efficacy and tolerability)
A PedAL/EuPAL Phase 1/2 Trial of IMGN632 in Pediatric Patients With Relapsed or Refractory Leukemia [NCT05320380]Phase 1/Phase 20 participants (Actual)Interventional2023-08-01Withdrawn(stopped due to Withdrawn per CS0150757)
Phase 2 Study of Personalized r-ATG Dosing to Improve Survival Through Enhanced Immune Reconstitution in Pediatric and Adult Patients Undergoing Ex-vivo CD34-Selected Allogeneic-HCT (PRAISE-IR) [NCT04872595]Phase 256 participants (Anticipated)Interventional2021-04-30Recruiting
A Phase I/II Study Administering Peripheral Blood Lymphocytes Transduced With a Murine T-Cell Receptor Recognizing the G12D Variant of Mutated RAS in HLA-A*11:01 Patients [NCT03745326]Phase 1/Phase 270 participants (Anticipated)Interventional2019-05-16Recruiting
A Phase I/II Study Administering Peripheral Blood Lymphocytes Transduced With a Murine T-Cell Receptor Recognizing the G12V Variant of Mutated RAS in HLA-A*11:01 Patients [NCT03190941]Phase 1/Phase 2110 participants (Anticipated)Interventional2017-09-21Recruiting
Phase I Study of Cellular Immunotherapy Using Memory Enriched T Cells Lentivirally Transduced to Express an IL13Rα2-Targeting, Hinge-Optimized, 41BB-Costimulatory Chimeric Receptor and a Truncated CD19 for Children With Recurrent/Refractory Malignant Brai [NCT04510051]Phase 118 participants (Anticipated)Interventional2020-12-04Recruiting
Cord Blood Ex-Vivo MSC Expansion Plus Fucosylation to Enhance Homing and Engraftment [NCT03096782]Phase 26 participants (Actual)Interventional2017-10-13Completed
A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT00987480]Phase 245 participants (Actual)Interventional2009-09-25Completed
Phase I/Ib Study of Adoptive NK Expressing an Affinity-enhanced T-cell Receptor (TCR) Reactive Against the NY-ESO-1-specific Cord Blood-derived NK Cells (NY-ESO-1 TCR/IL-15 NK) in Conjunction With Lymphodepleting Chemotherapy for the Management of Advance [NCT06083883]Phase 144 participants (Anticipated)Interventional2024-02-01Not yet recruiting
Reduced Intensity Conditioning (RIC) and Transplantation of HLA(Human Leukocyte Antigen)-Haploidentical Related Bone Marrow (Haplo-BM) For Patients With Hematologic Diseases [NCT02145039]2 participants (Actual)Interventional2014-10-31Terminated(stopped due to Replaced by another study.)
Pre-administration of Rabbit Antithymocyte Globulin to Optimize Donor T-Cell Engraftment Following Reduced Intensity Allogeneic Peripheral Blood Progenitor Cell Transplantation From Matched-Related Donors [NCT00787761]Phase 224 participants (Actual)Interventional2007-04-30Completed
Sequential Myeloablative Stem Cell Transplantation and Reduced Intensity Allogeneic Stem Cell Transplantation in Patients With Refractory or Recurrent Non-Hodgkin's Lymphoma and Hodgkin's Disease [NCT00802113]Phase 1/Phase 230 participants (Actual)Interventional2003-06-30Completed
Reduced Intensity Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Patients With Hematologic Malignancies [NCT00818961]Phase 236 participants (Actual)Interventional2005-05-31Terminated(stopped due to terminated early due to meeting end point with fewer patients than anticipated)
Lenalidomide Following Rituximab and Fludarabine in Untreated Chronic Lymphocytic Leukemia [NCT00860457]Phase 222 participants (Actual)Interventional2008-02-29Completed
Allogeneic Hematopoietic Cell Transplantation for Patients With Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen [NCT00919503]Phase 298 participants (Actual)Interventional2009-07-31Completed
A Phase 2 Study of Acalabrutinib in Combination With Lisocabtagene Maraleucel in Relapsed/Refractory Aggressive B-cell Lymphomas [NCT05583149]Phase 227 participants (Anticipated)Interventional2022-10-31Not yet recruiting
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.)
Randomized Phase III Study in Low Grade Lymphoma Comparing Maintenance Anti-CD20 Antibody Versus Observation Following Induction Therapy [NCT00003204]Phase 3515 participants (Actual)Interventional1998-03-31Completed
A Phase I-II Study for the Treatment of Steroid Resistant GVHD With Fludarabine [NCT00004194]Phase 1/Phase 20 participants InterventionalActive, not recruiting
Chronic Lymphocytic Leukemia Trial 4: A Randomized Comparison of Chlorambucil, Fludarabine and Fludarabine Plus Cyclophosphamide [NCT00004218]Phase 30 participants Interventional1999-10-31Completed
A Study to Determine the Safety and Efficacy of Using CD8-High Density Microparticles (CD8-HDM) to Deplete CD8+ Cells From Donor Lymphocyte Infusion in Order to Reduce Graft-versus-Host Disease (GvHD) Without Compromising an Anti-Leukemia Effect in Patien [NCT00004878]Phase 20 participants (Actual)InterventionalWithdrawn(stopped due to study never opened)
Phase I Study of Bryostatin 1 (NSC 339555) and Fludarabine in Patients With Chronic Lymphocytic Leukemia and Indolent Non-Hodgkin's Lymphoma [NCT00005580]Phase 154 participants (Actual)Interventional1998-09-30Completed
Chemotherapy (CT) Followed by Donor Lymphocyte Infusion (DLI) Plus Interleukin 2 (IL-2) for Patients With Relapse Acute Myeloid or Lymphoid Leukemia After Allogeneic Hematopoietic Transplant [NCT00005802]Phase 1/Phase 20 participants Interventional1999-06-30Completed
Prospective Study of Efficacy and Safety of RFC (Rituximab, Fludarabine, Cyclophosphamide) Regimen as a First-Line Therapy in Patients With B-Cell Chronic Lymphocytic Leukemia and Favorable Somatic Status [NCT01271010]Phase 489 participants (Actual)Interventional2011-06-17Terminated
A Single-Arm Phase I/II Study Evaluating the Safety and Clinical Efficacy Of the 2-nd Generation CD19 Autologous CAR T Cells on the CliniMACS Prodigy Automated Manufacturing Platform in Treatment of Paediatric And Young Adult Patients With Relapsed/Refrac [NCT03467256]Phase 1/Phase 218 participants (Anticipated)Interventional2018-05-14Active, not recruiting
Haploidentical Stem Cell Transplant Using Post Transplant Cyclophosphamide for GvHD Prophylaxis: A Pilot Study [NCT02248597]Phase 227 participants (Actual)Interventional2015-02-25Completed
Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation [NCT01472055]Phase 246 participants (Anticipated)Interventional2011-10-31Recruiting
A Phase II Study of Thiotepa Added to Fludarabine and Melphalan as the Preparative Regime for Alternative Donor Transplantation [NCT03342196]Phase 240 participants (Actual)Interventional2018-03-21Active, not recruiting
A Randomized Phase II Study of Treosulfan, Fludarabine and Low-Dose TBI as Conditioning for Allogeneic Hematopoietic Cell Transplantation in Patients With Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML) [NCT01894477]Phase 2102 participants (Actual)Interventional2013-11-30Active, not recruiting
Natural Killer Cells in Allogeneic Cord Blood Transplantation [NCT01619761]Phase 113 participants (Actual)Interventional2013-05-03Active, not recruiting
A Phase I Trial of PS-341 and Fludarabine for Relapsed and Refractory Indolent Non-Hodgkin's Lymphoma and Chronic Lymphocytic Leukemia [NCT00068315]Phase 118 participants (Actual)Interventional2003-07-31Completed
Safety And Efficacy of Sub-Myeloablative Allogeneic Stem Cell Transplantation For Patients With Myeloproliferative Disorder (MPD), Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous Leukemia [NCT00069992]Phase 27 participants (Actual)Interventional2001-12-31Terminated(stopped due to Closed due to competing protocols)
Fludarabine Versus Chlorambucil in First Line Therapy of Elderly Patients (More Than 65 Years) With Advanced Chronic Lymphocytic Leukemia [NCT00262795]Phase 3206 participants (Actual)Interventional2003-09-30Completed
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 Phase I Trial Of Sequential Administration Of Triapine (3-Aminopyridine-2-Carboxaldehyde Thiosemicarbazone) Followed By Fludarabine In Adults With Relapsed And Refractory Leukemias And Myelodysplasias [NCT00077558]Phase 10 participants Interventional2004-01-31Completed
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
Abbreviated Fludarabine / Mitoxantrone / Rituximab Chemotherapy Followed by Zevalin for Relapsed Mantle Cell Lymphoma [NCT00119730]Phase 230 participants (Actual)Interventional2005-02-28Completed
Phase II Trial Comparing Combination Treatment With Fludarabine and Alemtuzumab to Fludarabine and Rituximab in Patients With B-Cell Chronic Lymphocytic Leukemia Requiring Treatment After First Line Therapy [NCT00086775]Phase 20 participants Interventional2003-07-31Completed
Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation [NCT00301912]Phase 20 participants (Actual)Interventional2002-01-31Withdrawn(stopped due to Withdrawn because study never opened to accrual)
Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies [NCT00301951]Phase 17 participants (Actual)Interventional2004-09-30Completed
Pilot Study of Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies [NCT00304018]Phase 15 participants (Actual)Interventional2002-10-31Completed
Low-Dose Allogeneic Peripheral Blood Stem Cell Transplantation for High-Risk Low Grade Hematologic Malignancies [NCT00296023]25 participants (Actual)Interventional1999-01-31Completed
Optimizing Lymphodepletion to Improve Outcomes in Patients Receiving Anti Cluster of Differentiation Antigen 19 (Anti-CD19) Chimeric Antigen Receptor T (CAR T) Cell Therapy With Kymriah/tIsagenlecleucel (LOKI) [NCT06003179]Phase 140 participants (Anticipated)Interventional2023-12-31Not yet recruiting
A Phase I/IIa Multicenter Study Evaluating the Safety and Efficacy of CAR20(NAP)-T in Patients With Relapsed/Refractory B Cell Lymphoma (CARMA-01 Study) [NCT06002659]Phase 1/Phase 218 participants (Anticipated)Interventional2023-12-01Not yet recruiting
Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed/Refractory Acute Myeloid Leukemia [NCT06001788]Phase 1171 participants (Anticipated)Interventional2023-12-31Recruiting
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 Phase I Trial of T Cell Receptor Gene Therapy Targeting KK-LC-1 for Gastric, Breast, Cervical, Lung and Other KK-LC-1 Positive Epithelial Cancers [NCT05035407]Phase 1100 participants (Anticipated)Interventional2022-03-08Recruiting
Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia [NCT04558736]Phase 221 participants (Anticipated)Interventional2021-01-21Active, not recruiting
Multicenter, Open-label, Adaptive Design Phase I Trial With Genetically Modified T-cells Carrying Universal Chimeric Antigen Receptors (UniCAR02-T) in Combination With CD123 Target Module (TM123) for the Treatment of Patients With Hematologic and Lymphati [NCT04230265]Phase 190 participants (Anticipated)Interventional2020-01-28Recruiting
Phase I/II Study of Dual CD19-CD22 Chimeric Antigen Receptor (CAR) T Cells in Patients With Advanced CD19+ CD22+ Lymphoid Malignancies [NCT04029038]Phase 1/Phase 20 participants (Actual)Interventional2019-05-15Withdrawn(stopped due to 0 participant accrual)
Phase 1 Study of Lentivirally Transduced T Cells Engineered to Contain Anti-CD123 Linked to TCRζ and 4-1BB Signaling Domains in Subjects With Refractory or Relapsed Acute Myeloid Leukemia [NCT03766126]Phase 112 participants (Anticipated)Interventional2018-12-06Active, not 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 Study in Metastatic Melanoma Using a Lymphodepleting Conditioning Followed by Infusion of Anti-MART-1 TCR-Gene Engineered Lymphocytes and Subsequent Peptide Immunization [NCT00091104]Phase 1136 participants (Anticipated)Interventional2004-07-31Completed
A Pilot Trial of Therapeutic Vaccination With a Modified gp100 Melanoma Peptide (gp100:209-217(210M)), Montanide ISA 51, and KLH With Reconstitution After Chemotherapy to Induce Lymphopenia in Patients With Metastatic Melanoma [NCT00091143]Phase 120 participants (Anticipated)Interventional2004-07-31Completed
Phase II Study in Metastatic Renal Cell Cancer Using Cultured, Tumor-Reactive Lymphocytes and Interleukin-2 [NCT00091611]Phase 13 participants (Actual)Interventional2004-09-30Terminated(stopped due to This study was terminated due to low accrual.)
The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells [NCT00084695]Phase 225 participants (Anticipated)Interventional2003-09-30Recruiting
A Phase II Pilot Study of Tumor-Loaded Dendritic Cells Alone or Following a Non-Myeloablative Conditioning Regimen in Patients With Metastatic Renal Cell Carcinoma [NCT00093522]Phase 228 participants (Anticipated)Interventional2004-08-31Active, not recruiting
A Phase I Study to Assess the Safety and Tolerability of ex Vivo Next-generation Neoantigen-selected Tumor-infiltrating Lymphocyte (TIL) Therapy in Advanced Epithelial Tumors and Immune Checkpoint Blockade (ICB) Resistant Solid Tumors [NCT05141474]Early Phase 110 participants (Anticipated)Interventional2021-10-28Recruiting
A Multicenter, Single Arm, Open Label, Phase I Clinical Study to Evaluate the Safety, Tolerability and Efficacy of HRYZ-T101 Injection for HPV18 Positive Solid Tumor [NCT05952947]Phase 132 participants (Anticipated)Interventional2023-10-31Not yet recruiting
Pilot Study Evaluating the Use of Ex Vivo Expanded Cord Blood Progenitors as Supportive Care Following Chemotherapy (FLAG) in Patients With AML or Acute Leukemia of Ambiguous Lineage [NCT01701323]Phase 17 participants (Actual)Interventional2012-12-10Terminated
A Prospective, Open-Label, Multicenter Randomized Phase III Study to Compare The Efficacy and Safety of A Combined Regimen of Venetoclax and Obinutuzumab Versus Fludarabine, Cyclophosphamide, and Rituximab (FCR)/Bendamustine and Rituximab (BR) in FIT Pati [NCT04285567]Phase 3166 participants (Actual)Interventional2020-05-28Active, not recruiting
UARK 2003-18, A Phase II Study of KIR-Ligand Mismatched Haplo-Identical Natural Killer Cells Transfused Before Autologous Stem Cell Transplant in Relapsed Multiple Myeloma [NCT00089453]Phase 110 participants (Actual)Interventional2003-09-30Completed
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
Alloreactive NK Cells With Busulfan, Fludarabine and Thymoglobulin for Allogeneic Stem Cell Transplantation for AML and MDS [NCT00402558]Phase 115 participants (Actual)Interventional2006-05-31Completed
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
Reduced Intensity Conditioning Regimen for Haplo-identical Family Donor Stem Cell Transplants for Hematologic Malignancies With Delayed Add-back of Non-alloreactive T Cells [NCT00104975]Phase 120 participants (Anticipated)Interventional2005-02-28Completed
A Programme of Treatment Development for Older Patients With Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome [NCT00454480]Phase 2/Phase 32,000 participants (Anticipated)Interventional2006-08-31Completed
Anti-Third Party T Lymphocytes With Nonmyeloablative Stem Cell Transplantation for Treatment of Indolent Lymphoid Malignancies [NCT00473551]Phase 14 participants (Actual)Interventional2007-05-31Terminated(stopped due to Terminated due to slow accrual.)
A Phase IB Study to Assess the Safety and Efficacy of Neoadjuvant Administration of Autologous Tumor Infiltrating Lymphocytes (LN144/Lifileucel) and Pembrolizumab for Treatment of Patients With Locally Advanced (Stage IIIB-D)/Metastatic (Stage IV) Melanom [NCT05176470]Phase 115 participants (Anticipated)Interventional2022-07-01Recruiting
Cord Blood Expansion on Mesenchymal Stem Cells [NCT00498316]Phase 198 participants (Actual)Interventional2007-07-03Completed
Phase II Study of Sequential Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis [NCT00133367]Phase 232 participants (Anticipated)Interventional2005-08-31Completed
A Phase I/II Trial of T Cell Receptor Gene Therapy Targeting HPV-16 E7 for HPV-Associated Cancers [NCT02858310]Phase 1/Phase 2180 participants (Anticipated)Interventional2017-01-27Recruiting
Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies (UCAML) [NCT01247701]16 participants (Actual)Interventional2010-11-30Completed
Phase 2 Study of 5 Days Azacytidine Priming Prior to Fludarabine, Cytarabine and Granulocyte-Colony Stimulating Factor (G-CSF) Combination for Patients With Relapsed or Refractory AML [NCT02275663]Phase 237 participants (Anticipated)Interventional2014-12-31Recruiting
A Phase I Clinical Study to Assess the Safety and Efficacy of CD70-targeted CAR-NKT in the Treatment of Relapsed or Metastatic Advanced Renal Cell Carcinoma. [NCT06182735]Phase 19 participants (Anticipated)Interventional2023-07-17Recruiting
A Phase 3 Randomized, Open-Label, Multicenter Study Evaluating the Efficacy of Axicabtagene Ciloleucel Versus Standard of Care Therapy in Subjects With Relapsed/Refractory Follicular Lymphoma [NCT05371093]Phase 3230 participants (Anticipated)Interventional2022-09-12Recruiting
Phase I/II First-in-Human Trial With CAR19 Regulatory T Cells (CAR19-tTreg) in Adults With Relapsed/Refractory CD19+ B Acute Lymphocytic Leukemia [NCT05114837]Phase 1/Phase 231 participants (Anticipated)Interventional2024-05-31Not yet recruiting
Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS [NCT04708054]Phase 2100 participants (Anticipated)Interventional2021-10-21Recruiting
A Phase 1 Study Evaluating the Safety and Efficacy of HPV16 E7 T Cell Receptor Engineered T Cells (KITE-439) in HLA-A*02:01+ Subjects With Relapsed/Refractory HPV16+ Cancers [NCT03912831]Phase 18 participants (Actual)Interventional2019-04-30Terminated(stopped due to Development program terminated)
First-Line Therapy With Ibrutinib, Fludarabine, Cyclophosphamide, and Obinutuzumab (GA-101) (iFCG) for Patients With Chronic Lymphocytic Leukemia (CLL) With Mutated IGHV Gene and Non-Del(17p) [NCT02629809]Phase 281 participants (Actual)Interventional2016-03-18Active, not recruiting
Administration Of TGF-beta Resistant Cytotoxic T-Lymphocytes to Patients With EBV-positive Nasopharyngeal Carcinoma (RESIST-NPC) [NCT02065362]Phase 114 participants (Anticipated)Interventional2015-02-28Active, not recruiting
A Pilot Study of Lymphodepletion Plus Adoptive Cell Transfer With TGF-Beta Resistant (DNRII) and NGFR Transduced T-Cells Followed by High Dose Interleukin-2 in Patients With Metastatic Melanoma [NCT01955460]Phase 115 participants (Anticipated)Interventional2014-10-15Recruiting
A Phase 2 Study of Fludarabine, Cytarabine, Filgrastim-sndz,Gemtuzumab Ozogamicin and Idarubicin in Newly Diagnosed Core Binding Factor Associated Acute Myelogenous Leukemia [NCT00801489]Phase 2270 participants (Anticipated)Interventional2007-04-04Recruiting
Allogeneic Stem Cell Transplantation Followed By Adoptive Immunotherapy for Patients With Relapsed and Refractory Hodgkin's Disease [NCT00385788]Phase 252 participants (Actual)Interventional2005-07-31Completed
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
Phase II Study of Metastatic Melanoma Using Lymphodepleting Conditioning Followed by Infusion of Anti-gp100:154-162 TCR-Gene Engineered Lymphocytes [NCT00509496]Phase 221 participants (Actual)Interventional2007-06-30Terminated(stopped due to Low accrual)
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
Phase II Study of Metastatic Melanoma Using Lymphodepleting Conditioning Followed by Infusion of Anti-MART-1 F5 TCR-Gene Engineered Lymphocytes and ALVAC Virus Immunization [NCT00612222]Phase 24 participants (Actual)Interventional2008-01-31Terminated(stopped due to The study was terminated due to low accrual.)
Reduced Intensity Hematopoietic Cell Transplantation for Patients With Resistant Langerhans Cell Histiocytosis [NCT00618540]Phase 21 participants (Actual)Interventional2007-01-31Terminated(stopped due to Slow accrual)
Phase II Study of the Addition of Azacitidine (NSC#102816) to Reduced-Intensity Conditioning Allogeneic Transplantation for Myelodysplasia (MDS) and Older Patients With AML [NCT01168219]Phase 268 participants (Actual)Interventional2010-07-15Completed
A Phase 1 Safety and Efficacy Study of ADI-001 Anti-CD20 CAR-engineered Allogeneic Gamma Delta (γδ) T Cells in Adults With B Cell Malignancies [NCT04735471]Phase 178 participants (Anticipated)Interventional2021-03-04Recruiting
CD34 Selected Allogeneic Hematopoietic Cell Transplantation With Myeloablative Conditioning and CD8+ Memory T Cell Infusion For Patients With Myelodysplastic Syndrome, Acute Leukemia, and Chronic Myelogenous Leukemia [NCT04151706]Phase 220 participants (Anticipated)Interventional2020-02-27Suspended(stopped due to Interim analysis)
Umbilical Cord Blood Transplantation From Unrelated Donors [NCT03016806]Phase 130 participants (Anticipated)Interventional2015-06-30Recruiting
Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Malignant Hematological Diseases [NCT00582894]17 participants (Actual)Interventional2005-02-28Completed
Cd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi Anemia [NCT00590460]Phase 1/Phase 25 participants (Actual)Interventional2001-07-31Terminated(stopped due to slow accrual)
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)
T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies [NCT00683046]Phase 2204 participants (Actual)Interventional2001-11-30Completed
Prospective Identification of Significant Prognostic Factors in Patients Treated With Fludarabine, Cyclophosphamide, and Rituximab (FCR) as Initial Therapy for Chronic Lymphocytic Leukemia. [NCT00759798]Phase 2289 participants (Actual)Interventional2008-08-13Completed
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
Combined Haploidentical-Cord Blood Transplantation for Adults and Children [NCT00943800]87 participants (Actual)Interventional2006-10-09Completed
A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial) [NCT01005576]Phase 221 participants (Actual)Interventional2010-01-31Completed
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
A Randomised Phase II Study in Metastatic Melanoma to Evaluate the Efficacy of Adoptive Cellular Therapy With Tumour Infiltrating Lymphocytes (TIL) and Assessment of High Versus Low Dose Interleukin-2 [NCT01995344]Phase 22 participants (Actual)Interventional2014-03-01Terminated(stopped due to Withdrawal of funding)
A Phase I/II Clinical Trial of Fludarabine, Bendamustine, and Rituximab (FBR) in Previously Treated Patients With Chronic Lymphocytic Leukemia (CLL) [NCT01096992]Phase 1/Phase 251 participants (Actual)Interventional2010-04-19Completed
Lymphodepleting Chemotherapy and T-Cell Suppression Followed By Allogeneic Natural Killer Cells and IL-2 in Patients With Recurrent Ovarian, Fallopian Tube, Primary Peritoneal Cancer and Advanced Metastatic Breast Cancer (MT2009-30) [NCT01105650]Phase 213 participants (Actual)Interventional2010-07-31Completed
Phase II Study in Patients With Metastatic Melanoma Using a Non-Myeloablative Lymphocyte Depleting Regimen of Chemotherapy Followed by Infusion of MART-1 Reactive Peripheral Blood Lymphocytes (PBL) With or Without High Dose Aldesleukin [NCT01495572]Phase 25 participants (Actual)Interventional2011-12-31Terminated(stopped due to Due to slow accrual, the investigator decided to close the study.)
A Phase I Study of FT819 in Subjects With B-cell Malignancies [NCT04629729]Phase 1396 participants (Anticipated)Interventional2021-07-26Recruiting
A Phase 2 Multicenter Study Evaluating the Efficacy and Safety of Axicabtagene Ciloleucel as First-Line Therapy in Subjects With High-Risk Large B-Cell Lymphoma (ZUMA-12) [NCT03761056]Phase 242 participants (Actual)Interventional2019-01-29Completed
A Phase II Study of Hematopoietic Stem Cell Therapy for Young Adults With Severe Sickle Cell Disease [NCT01565616]Phase 222 participants (Actual)Interventional2012-03-31Completed
Phase I/II Study of CD40 Ligand Expressing MSLN-CAR T Cell Treatment in MSLN Positive Advanced/Metastatic Solid Tumors [NCT05693844]Phase 1/Phase 230 participants (Anticipated)Interventional2023-01-20Recruiting
An Open, Uncontrolled, Multicenter Clinical Trial to Explore the Safety, Efficacy, and Remission Phase of Chimeric Antigen Receptor T Cell (CAR-T) in the Treatment of Relapsed Refractory (R/R) Non-Hodgkin Lymphoma (NHL) [NCT04626739]Early Phase 1100 participants (Anticipated)Interventional2020-04-01Recruiting
A Randomized, Open-Label, Phase 3 Trial to Compare the Efficacy and Safety of Idecabtagene Vicleucel With Lenalidomide Maintenance Versus Lenalidomide Maintenance Therapy Alone in Adult Participants With Newly Diagnosed Multiple Myeloma Who Have Suboptima [NCT06045806]Phase 3618 participants (Anticipated)Interventional2023-10-16Recruiting
Clinical Trial of CAR-T in the Treatment of Relapsed and Refractory Hematopoietic and Lymphoid Tissue Tumors in Children [NCT04610125]30 participants (Anticipated)Interventional2020-06-23Recruiting
An Open-Label, Multicenter, Phase Ib Trial of GA101 (RO5072759) in Combination With Chemotherapy in Patients With Previously Untreated Chronic Lymphocytic Leukemia [NCT01300247]Phase 141 participants (Actual)Interventional2011-05-31Completed
A Phase II, Multicenter, Single Arm Study to Determine the Efficacy and Safety of Low Dose Fludarabine and Cyclophosphamide Combined With Standard Dose Rituximab as Primary Therapy in Elderly Untreated Patients (>/=65 Years Old) With Chronic Lymphocytic L [NCT01263704]Phase 242 participants (Actual)Interventional2011-07-17Completed
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) [NCT01666080]30 participants (Anticipated)Interventional2012-08-31Recruiting
A Phase 1 Study to Evaluate Intracerebroventricular (ICV) Administration of CD19-Targeting Chimeric Antigen Receptor (CAR) T Cells in Patients With Primary CNS Lymphoma [NCT05625594]Phase 120 participants (Anticipated)Interventional2023-06-29Recruiting
A Phase 1/2 Trial (CheckCell-2) in Patients With Metastatic Non-small Cell Lung Cancer (NSCLC) Administering Tumor-Infiltrating Lymphocytes (TILs) in Which the Gene Encoding CISH Was Inactivated Using the CRISPR/Cas9 System [NCT05566223]Phase 1/Phase 270 participants (Anticipated)Interventional2023-02-28Not yet recruiting
A Feasibility Trial of Post-Transplant Infusion of Allogeneic Regulatory T Cells and Allogeneic Conventional T Cells in Patients With Hematologic Malignancies Undergoing Allogeneic Myeloablative Hematopoietic Cell Transplantation From Haploidentical-Relat [NCT01050764]Phase 1/Phase 210 participants (Actual)Interventional2009-06-30Terminated(stopped due to Safety)
Dose Escalation Trial of Cloretazine (VNP40101M) and Hematopoietic Cell Transplantation for Patients With Selected, Poor-Prognosis Hematologic Malignancies [NCT00521859]Phase 15 participants (Actual)Interventional2007-08-31Completed
HDAC Inhibitor Valproic Acid as an Effective Therapy for Chronic Lymphocytic Leukemia [NCT00524667]Phase 26 participants (Actual)Interventional2008-01-31Terminated(stopped due to Terminated due to poor accrual.)
Pacritinib Prior to Transplant for Patients With Myeloproliferative Neoplasms (MPN) [NCT02410551]Phase 24 participants (Actual)Interventional2015-06-15Terminated
A Phase I/II Study to Evaluate the Safety of Cellular Immunotherapy Using Autologous T Cells Engineered to Express a CD20-Specific Chimeric Antigen Receptor for Patients With Relapsed or Refractory B Cell Non-Hodgkin Lymphomas [NCT03277729]Phase 1/Phase 250 participants (Anticipated)Interventional2017-12-05Recruiting
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
Bispecific NK Engager AFM13 Combined With NK Cells for Patients With Recurrent of Refractory CD30 Positive Hodgkin or Non-Hodgkin Lymphomas [NCT04074746]Phase 1/Phase 230 participants (Anticipated)Interventional2020-07-18Active, not recruiting
A Phase 1 Safety Study of Adoptive Cellular Therapy Using Autologous T Cells Transduced With Lentivirus to Express a CD33 Specific Chimeric Antigen Receptor in Patients With Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia [NCT03126864]Phase 111 participants (Actual)Interventional2017-08-04Terminated(stopped due to Terminated per the PI's request.)
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 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity Conditioning [NCT05031897]Phase 267 participants (Anticipated)Interventional2021-10-25Recruiting
Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders [NCT00053989]Phase 241 participants (Actual)Interventional2002-01-29Completed
[NCT01540812]418 participants (Actual)Observational2012-02-29Completed
Phase II Study of Haploidentical Allogeneic Peripheral Blood Stem Cell Transplantation in Patients With High-Risk Acute Myeloid Leukemia in First Remission [NCT00101140]Phase 20 participants (Actual)InterventionalWithdrawn
A Phase I/II Combination Study of Topotecan, Fludarabine, Cytosine Arabinoside and G-CSF (T-FLAG) Induction Therapy in Patients With Poor Prognosis AML, MDS and Relapsed/Refractory ALL Followed by Maintenance of Either PBSC Transplant or 13 Cis-Retinoic A [NCT00003619]Phase 1/Phase 20 participants Interventional1998-02-28Completed
A Clinical Trial Evaluating I131-Tositumomab (Anti-CD20) With Escalating Doses of Fludarabine Followed by Autologous or Syngeneic Stem Cell Transplantation for Relapsed or Refractory B-cell Non-Hodgkin's Lymphoma in Patients 60 Years of Age and Older [NCT00110071]Phase 138 participants (Actual)Interventional2005-01-31Completed
A Phase I/II Study of Total Body Irradiation, Thiotepa, and Fludarabine as Conditioning for Haploidentical CD34+ Purified Peripheral Blood Stem Cell Transplants [NCT00112567]Phase 1/Phase 220 participants (Anticipated)Interventional2003-04-30Completed
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
Allogeneic Breast Protocol 1: T-Cell Depleted Allogeneic Blood Stem Cell Transplantation Using an Immunoablative Conditioning Regimen in Metastatic Breast Cancer [NCT00020176]Phase 20 participants Interventional2000-06-30Completed
Randomized Phase II Study of Thalidomide Versus Thalidomide Plus Fludarabine for Patients With Chronic Lymphocytic Leukemia Previously Treated With Fludarabine [NCT00009984]Phase 270 participants (Actual)Interventional2002-03-31Terminated
Phase II Study of Combination Rituxan (Rituximab, Mabthera) and Fludarabine Therapy in Lymphoplasmacytic Lymphoma (Waldenstrom's Macroglobulinemia) [NCT00020800]Phase 27 participants (Actual)Interventional2001-09-30Completed
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
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
Autologous Followed By Non-Myeloablative Allogeneic Transplant For Multiple Myeloma [NCT00028600]Phase 260 participants (Actual)Interventional2001-11-30Completed
Allogeneic Stem Cell Transplantation Following Nonmyeloablative Chemotherapy in Patients With Hemoglobinopathies [NCT00034528]Phase 22 participants (Actual)Interventional2001-09-30Terminated(stopped due to Due to slow recruitment)
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
Allo SCT From HLA Haploidentical Related Donors Using Sub-Myeloablative Conditioning For Patients With High Risk Hemoglobinopathies: Hemo SS, Hemo SC, Hemo SB0/+ Thalassemia, Homozygous B0/+ Thalassemia or Severe B0/+ Thalassemia Variants [NCT00040417]Phase 215 participants Interventional2000-08-31Terminated(stopped due to unable to accrue patients)
Transplantation With T-Cell Depleted Autologous Peripheral Stem Cells for Severe Systemic Sclerosis: A Phase I Dose Escalation Study [NCT00040651]Phase 115 participants Interventional2002-07-31Terminated
Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL): DNA Microarray Gene Expression Analysis [NCT00001586]Phase 2105 participants (Actual)Interventional1997-09-30Completed
Phase I/II Study of Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancy, Using MHC Identical or Near Identical Donors and Sub-Myeloablative Conditioning With CAMPATH 1H (DIMSUM) [NCT00048412]Phase 1/Phase 240 participants Interventional2000-06-30Completed
A Phase II Study of Oral Fludarabine Phosphate in Patients With Previously Untreated B-Cell Chronic Lymphocytic Leukemia [NCT00049075]Phase 2128 participants (Actual)Interventional2002-08-08Completed
A Single Dose-escalation Study to Evaluate the Safety and Efficacy of Allogeneic CAR-T Targeting CD19 Bridging Hematopoietic Stem Cell Transplantation in Patients With Refractory or Relapsed B Cell Acute Lymphoblastic Leukemia [NCT05576181]Phase 119 participants (Anticipated)Interventional2022-10-15Not yet recruiting
A Single Dose-escalation Study to Evaluate the Safety and Efficacy of Allogeneic CAR-T Targeting CD19 (ThisCART19A) in Adult Patients With B Cell Malignancies After Failure of Autologous Chimeric Antigen Receptor T- Cell(CAR-T) Therapy [NCT05640713]Phase 112 participants (Anticipated)Interventional2022-12-01Not yet recruiting
National Mantle Cell Lymphoma Trial - Phase II Randomized Study of Fludarabine/Cyclophosphamide Combination With or Without Rituximab in Patients With Untreated Mantle Cell Lymphoma [NCT00053092]Phase 282 participants (Anticipated)Interventional2002-10-31Completed
Phase II Study of Fludarabine, Carboplatin, and Topotecan With Thalidomide for Patients With Relapsed/Refractory or High Risk Acute Myelogenous Leukemia, Chronic Myeloid Leukemia and Advanced Myelodysplastic Syndromes [NCT00053287]Phase 242 participants (Actual)Interventional2002-09-30Completed
A Single-Arm, Open-Label, Phase 1 Study of the Safety, Efficacy, and Cellular Kinetics/Pharmacodynamics of ALLO-715 to Evaluate an Anti-BCMA Allogeneic CAR T Cell Therapy With or Without Nirogacestat in Subjects With Relapsed/Refractory Multiple Myeloma [NCT04093596]Phase 1132 participants (Anticipated)Interventional2019-09-23Active, not recruiting
Phase II Trial in Intrafamilial Allogeneic Cell Transplant in Patients With Metastatic Kidney Cancer [NCT00056095]Phase 257 participants (Actual)Interventional2002-11-04Completed
A Study of Allogeneic Blood Stem Cell Transplantation With Purine Analog-Based Conditioning For Patients With Advanced Hodgkin's Disease [NCT00423709]46 participants (Actual)Interventional1998-01-31Completed
Allogeneic Adoptive Immunochemotherapy For Treatment Of Renal Cell Carcinoma [NCT00073879]0 participants Interventional2003-04-30Completed
Clinical Study of the Safety and Initial Efficacy of BGT007 Cells in the Treatment of Patients With Relapsed /Metastatic Nasopharyngeal Carcinoma [NCT05616468]Early Phase 123 participants (Anticipated)Interventional2022-12-30Recruiting
Treatment Of Patients With Metastatic Melanoma Using Nonmyeloablative But Lymphocyte Depleting Regimen Followed By The Administration Of In Vitro Sensitized Lymphocytes Reactive With ESO-1 Antigen [NCT00079144]Phase 20 participants Interventional2004-01-31Completed
Phase II Trial Using Aldesleukin (IL-2) Following a Lymphodepleting Chemotherapy and Reinfusion of Autologous Lymphocytes Depleted of T Regulatory Lymphocytes in Metastatic Melanoma [NCT00138229]Phase 26 participants (Actual)Interventional2005-07-31Terminated
Randomized Study for Patients With Follicular Lymphoma Needing Treatment [NCT00140569]Phase 3400 participants Interventional1994-01-31Completed
Feasibility/Phase II Trial of Fludarabine, Rituximab, and Alemtuzumab for Previously Treated B-Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) [NCT00143065]Phase 28 participants (Actual)Interventional2005-08-31Completed
Phase II Clinical Protocol for the Treatment of Patients With Previously Untreated CLL With Four or Six Cycles of Fludarabine and Cyclophosphamide With Rituximab (FCR) Plus Lenalidomide Followed by Lenalidomide Consolidation/ Maintenance [NCT01723839]Phase 221 participants (Actual)Interventional2012-02-22Completed
Treatment of Patients With Metastatic Melanoma Using Cloned Lymphocytes Following the Administration of a Non-Myeloablative But Lymphocyte Depleting Regimen [NCT00001832]Phase 2170 participants (Actual)Interventional1999-08-31Completed
[NCT00010361]20 participants Interventional2000-11-30Completed
Low Dose Chlorambucil Maintenance Vs. No Treatment Following High-Dose Chlorambucil Induction In Patients With Advanced B-Chronic Lymphocytic Leukemia. A Randomized Phase III Study Of The EORTC LG (CLL-3) [NCT00017108]Phase 30 participants Interventional2001-03-31Active, not recruiting
Phase II Window Evaluation of the Farnesyl Transferase Inhibitor (R115777) Followed by 13-CIS Retinoic Acid, Cytosine Arabinoside and Fludarabine Plus Hematopoietic Stem Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia [NCT00025038]Phase 2100 participants (Actual)Interventional2001-06-30Completed
Adoptive Immunotherapy by Allogeneic Stem Cell Transplantation for Metastatic Renal Cell Carcinoma: A Phase II Study [NCT00027573]Phase 236 participants (Actual)Interventional2001-10-31Completed
Fludarabine And Busulfan As Conditioning For Patients With Chronic Myeloid Leukemia Or Myelodysplastic Syndrome Transplanted With Hematopoietic Stem Cells From HLA-Compatible Related Or Unrelated Donors [NCT00027924]Phase 20 participants Interventional2001-10-31Completed
Allogeneic Hematopoietic Cell Transplantation From HLA-matched Donor After Flu-Mel-PTCy Versus Flu-Mel-ATG Reduced-intensity Conditioning: a Phase II Randomized Study From the Belgian Hematology Society (BHS) [NCT03852407]Phase 2114 participants (Anticipated)Interventional2019-02-04Recruiting
Phase II Study of Rituximab in Combination With Fludarabine and Cyclophosphamide for the Treatment of Relapsed Follicular Lymphoma [NCT00393107]Phase 254 participants Interventional2000-03-31Completed
Allogeneic Hematopoietic Stem Cell Transplantation For The Treatment Of High Risk Multiple Myeloma With Reduced Toxicity Myeloablative Conditioning Regimen [NCT00615589]Phase 222 participants (Actual)Interventional2008-02-29Terminated(stopped due to Low accrual)
Fludarabine Versus Fludarabine Plus Cyclophosphamide in First Line Therapy of Younger Patients (Up to 65 Years) With Advanced Chronic Lymphocytic Leukemia (CLL) [NCT00276848]Phase 3375 participants (Actual)Interventional1999-07-31Completed
Campath 1H (Alemtuzumab) Combined With High-Dose Therapy and Autologous Stem Cell Transplantation in Chronic Lymphocytic Leukemia [NCT00276809]Phase 230 participants (Anticipated)Interventional2001-06-30Completed
Randomized Phase III Trial Comparing Early Treatment With Fludarabine/Cyclophosphamide + Rituximab Versus Deferred Treatment in Untreated Binet Stage A Patients With CLL and High Risk of Progression [NCT00275054]Phase 3825 participants (Actual)Interventional2005-10-31Completed
Pivotal Study for High Dose Therapy and Autologous Stem Cell Transplantation in Early Stages of CLL [NCT00275015]Phase 2169 participants (Actual)Interventional1998-01-31Completed
A Phase I Clinical Trial With TriPRIL CAR T Cells for the Treatment of Patients With Relapsed or Refractory Multiple Myeloma [NCT05020444]Phase 118 participants (Anticipated)Interventional2021-10-05Recruiting
Phase 1/2 Trial of TC-110 T Cells in Adults With Relapsed or Refractory Non-Hodgkin Lymphoma (NHL) or Acute Lymphoblastic Leukemia (ALL) [NCT04323657]Phase 1/Phase 26 participants (Actual)Interventional2020-03-27Completed
Safety and Efficacy of Campath in Nonmyeloablative Transplantation [NCT00038844]65 participants (Actual)Interventional2001-06-30Completed
Phase I and Pharmacokinetic Study of UCN-01 and Fludarabine in Relapsed or Refractory Low-Grade Lymphoid Malignancies [NCT00019838]Phase 10 participants Interventional1999-07-31Completed
Safety and Efficacy of Sequential Treatment With a Combination of Rituximab, Fludarabine and Cyclophosphamide Followed by Zevalin (Rituximab and Y-Ibritumomab Tiuxetan) - A Phase I/II Study for Treatment of Patients With Relapsed Indolent and Transformed [NCT00397800]Phase 1/Phase 212 participants (Anticipated)Interventional2005-06-30Active, not recruiting
Randomized Study Of Fludarabine And Cyclophosphamide With Or Without Genasense (Bcl-2 Antisense Oligonucleotide) In Subjects With Relapsed Or Refractory Chronic Lymphocytic Leukemia [NCT00024440]Phase 30 participants Interventional2001-07-31Completed
Unrelated Umbilical Cord Blood As An Alternate Source Of Stem Cells Transplantation [NCT00055653]Phase 20 participants Interventional2003-01-31Completed
Phase II Trial of T-Cell Depleted Hematopoietic Stem Cell Transplants (SBA-E-BM) From HLA Compatible Related or Unrelated Donors After a Myeloablative Preparative Regimen of Hyperfractionated TBI, Thiotepa and Cyclophosphamide (TBI/Thio/cy) for Treatment [NCT00028730]Phase 225 participants (Actual)Interventional2001-08-31Completed
Anti-CD45 (YTH-24 & YTH 54) and ANTI-CD52 (CAMPATH-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases [NCT00056979]Phase 1/Phase 22 participants (Actual)Interventional2002-06-30Terminated
A Phase I Study of Flavopiridol, Fludarabine and Rituximab in Indolent B-cell Lymphoproliferative Disorders and Mantle Cell Lymphoma [NCT00058227]Phase 137 participants (Actual)Interventional2003-04-30Completed
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
A Phase I Trial of BBR 2778 in Combination With Fludarabine, Dexamethasone and Rituximab in the Treatment of Patients With Relapsed or Refractory Indolent Non-Hodgkin's Lymphoma [NCT00060684]Phase 130 participants Interventional2001-12-31Completed
Pilot Study Of T-Cell-Depleted Peripheral Blood Stem Cell Transplantation From Partially Matched Related Donors For Patients With High-Risk Leukemia [NCT00066417]Phase 251 participants (Anticipated)InterventionalTerminated(stopped due to Trial was withdrawn for drug availability issues.)
Phase I/II Evaluation of Safety and Activity of Mylotarg Plus Melphalan and Fludarabine as Preparative Therapy for Older or Medically Infirm Patients Undergoing Allogeneic Bone Marrow and Peripheral Blood Stem Cell Transplantation [NCT00038831]Phase 1/Phase 247 participants (Actual)Interventional2001-05-31Completed
Immunomodulation by Ultraviolet B-Irradiation (UVB) to Facilitate Allogeneic Stem Cell Transplantation for Treatment of Hematologic Malignancies [NCT00068523]10 participants (Actual)Interventional2003-06-30Completed
Efficacy of Maintenance Therapy With Rituximab After Induction Chemotherapy (R-CHOP vs. R-FC) for Elderly Patients With Mantle Cell Lymphoma Not Suitable for Autologous Stem Cell Transplantation [NCT00209209]Phase 3570 participants (Anticipated)Interventional2004-01-14Active, not recruiting
Protocol for the Treatment of Adults Aged NCT00209833]Phase 2/Phase 3200 participants Interventional1999-01-31Active, not recruiting
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)
A Pilot Study of Genasense® (G3139, Oblimersen Sodium, Bcl-2 Antisense Oligonucleotide), Fludarabine and Rituximab in Previously Treated Subjects With Chronic Lymphocytic Leukemia [NCT00078234]Phase 1/Phase 225 participants (Actual)Interventional2003-11-30Completed
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 Phase II Study of Single Agent Depsipeptide (NSC 630176) Followed by a Phase I Study of Rituximab/Fludarabine Combination With an Escalating Dose of Depsipeptide in Relapsed or Refractory Low Grade B Cell Lymphomas [NCT00079443]Phase 260 participants (Actual)Interventional2004-01-31Terminated
T-Cell Depleted, Reduced-Intensity Allogeneic Stem Cell Transplantation From Haploidentical Related Donors For Hematologic Malignancies [NCT00080925]Phase 120 participants (Anticipated)Interventional2004-02-29Completed
Multicenter Phase II Trial of Fludarabine and Cyclophosphamide in Combination With Alemtuzumab (FC-Cam) for Patients With Relapsed Chronic Lymphocytic Leukemia - CLL-2L Protocol of the German CLL-Study Group (GCLLSG) [NCT00147901]Phase 261 participants (Actual)Interventional2005-01-31Completed
Pilot Study Of Multiple Umbilical Cord Blood Unit Transplantation Following Non-Myeloablative Conditioning In Patients With Hematologic Disorders Or Severe Aplastic Anemia [NCT00054236]Phase 155 participants (Actual)Interventional2002-05-31Completed
Phase I/II Study Of UCN-01 In Combination With Fludarabine In Patients With Relapsed Or Refractory Chronic Lymphocytic Leukemia Or Small Lymphocytic Lymphoma [NCT00045513]Phase 1/Phase 20 participants Interventional2002-06-30Completed
Phase I/II Study of CD45 Antibodies and Alemtuzumab Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation of Patients With Hematological Diseases [NCT00056966]Phase 1/Phase 224 participants (Actual)Interventional2002-11-30Completed
Pilot/Feasibility Study To Evaluate The Safety Of Cellular Immunotherapy For CD19+ Follicular Lymphoma Using Autologous Cytolytic T Cells Genetically-Modified To Be CD19-Specific And Co-Express HyTK [NCT00182650]Phase 15 participants (Anticipated)Interventional2004-06-30Completed
Phase I/II Study of Allogeneic Stem Cell Transplantation For Patients With Graft Failure Following Allogeneic Transplantation Using MHC Identical or Near Identical Donors and Submyeloablative Conditioning With CAMPATH 1H (CAMGRAFT) [NCT00048399]Phase 1/Phase 240 participants Interventional2000-12-31Terminated(stopped due to accrual was slow)
Treatment of Elderly Patients (>60 Years) With Acute Myeloblastic Leukemia or Advanced MDS (RAEB-T): An Open Randomized Study to Test the Efficacy of G-CSF-Priming and a Feasibility Trial of Dose-Reduced Allogeneic Transplantation and of Autologous Stem C [NCT00199147]Phase 4250 participants Interventional2000-01-31Recruiting
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)
Tumor Infiltrating Lymphocytes (TIL Cells) Transduced With An Interleukin-2 (SBIL-2) Gene Following The Administration Of A Nonmyeloablative But Lymphocyte Depleting Regimen in Metastatic Melanoma [NCT00062036]Phase 1/Phase 233 participants (Anticipated)Interventional2003-06-30Completed
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
A Phase II Study of Cell Transfer Therapy for Metastatic Melanoma Using 41BB Selected Tumor Infiltrating Lymphocytes Plus IL-2 Following a Non-Myeloablative Lymphocyte Depleting Chemotherapy Regimen [NCT02111863]Phase 26 participants (Actual)Interventional2014-02-21Terminated(stopped due to Study terminated due to low accrual and change in research focus.)
Allogeneic Hematopoietic Cell Transplantation for Patients With Hematologic Disorders Who Are Ineligible or Inappropriate for Treatment With a More Intensive Therapeutic Regimen [NCT00448201]Phase 271 participants (Actual)Interventional2011-01-07Completed
Allogeneic Natural Killer Cells in Patients With Advanced Metastatic Breast Cancer [NCT00376805]Phase 26 participants (Actual)Interventional2006-04-30Terminated(stopped due to Withdrawn due to toxicity)
Allogeneic Hematopoietic Cell Transplantation for Patients With Hematologic Disorders Who Are Undergoing Dose-Adjusted Treatment With A Maximally Intensive Busulfex-Based Therapeutic Regimen [NCT00448357]Phase 1/Phase 254 participants (Actual)Interventional2005-10-31Completed
Phase I Dose Escalation Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies [NCT03241940]Phase 150 participants (Anticipated)Interventional2017-10-20Recruiting
Phase 1 Dose Escalation Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells With or Without NKTR-255 in Adults With Recurrent or Refractory B Cell Malignancies [NCT03233854]Phase 160 participants (Anticipated)Interventional2017-09-01Recruiting
A Phase I-II Study of Oxaliplatin, Fludarabine, Cytarabine, and Rituximab in Patients With Richter's Transformation, Prolymphocytic Leukemia, Aggressive, Relapsed or Refractory B-Cell Chronic Lymphocytic Leukemia [NCT00472849]Phase 1/Phase 292 participants (Actual)Interventional2007-05-31Completed
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 Phase I/II Study of Fludarabine, Rituximab, and Lenalidomide in Minimally Treated and Untreated Patients With Chronic Lymphocytic Leukemia [NCT00536341]Phase 1/Phase 264 participants (Actual)Interventional2008-01-31Completed
A Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Patients With High Risk Hemoglobinopathy Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism [NCT00427661]8 participants (Actual)Interventional2002-06-30Completed
Phase I/II Trial of Fludarabine in Combination With Intravenous Busulfan and Allogeneic Progenitor Cell Support for Patients With Hematologic Malignancies [NCT00506857]Phase 1/Phase 282 participants (Actual)Interventional2003-11-30Completed
Transplantation of Haploidentical CD34+ Purified Peripheral Blood Stem Cells With NK-Cell Add-Back Following Conditioning With Total Body Irradiation, Thiotepa, Fludarabine and OKT3 [NCT00450983]Phase 21 participants (Actual)Interventional2006-12-31Terminated
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.)
A Randomized Phase II Trial of Fludarabine/Melphalan 140 VS. Fludarabine/Melphalan 100 Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation for Patients With Multiple Myeloma [NCT00505895]Phase 252 participants (Actual)Interventional2002-01-31Completed
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 I Trial of GFRα4 CAR T Cells in Adult Patients With Recurrent or Metastatic Medullary Thyroid Cancer [NCT04877613]Phase 118 participants (Anticipated)Interventional2021-08-19Recruiting
A Phase II Study of High-Dose Intravenous Busulfan and Fludarabine With Allogeneic Marrow and Peripheral Blood Progenitor Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes [NCT00502905]Phase 2200 participants (Actual)Interventional2003-10-31Completed
CD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency Disorders [NCT00579137]Phase 1/Phase 23 participants (Actual)Interventional2007-10-31Terminated(stopped due to slow accrual)
Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis [NCT00622895]Phase 1/Phase 23 participants (Actual)Interventional2006-09-01Completed
Reduced-Intensity Preparative Regiment With Fludarabine, Busulfan, And Alemtuzumab (Campath 1H) Followed By Allogeneic Hematopoietic Stem Cell Transplant For Malignant And Non-Malignant Hematological Diseases [NCT00579111]Phase 1/Phase 24 participants (Actual)Interventional2007-06-30Terminated(stopped due to slow accrual)
Non-Myeloablative Chemotherapy Followed by HLA-Matched Related Allogeneic Stem Cell Transplantation for Hematologic Malignancies [NCT00741455]18 participants (Actual)Interventional2004-06-30Completed
Phase I/II Study of Metastatic Cancer That Expresses MAGE-A3/12 Using Lymphodepleting Conditioning Followed by Infusion of Anti-MAGE-A3/12 TCR-Gene Engineered Lymphocytes [NCT01273181]Phase 1/Phase 29 participants (Actual)Interventional2010-12-31Terminated
Busulfan (IV) and Fludarabine Followed by Post-allogeneic Transplantation Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies. [NCT00800839]Phase 256 participants (Actual)Interventional2008-09-30Completed
Phase II Study of CD62L+-Derived T Lymphocytes Transduced With a T Cell Receptor Recognizing the NY-ESO-1 Antigen and Aldesleukin Following Lymphodepletion in Patients With NY-ESO-1 Expressing Melanoma [NCT02062359]Phase 22 participants (Actual)Interventional2014-02-28Terminated(stopped due to Study was closed due to poor accrual.)
T Cell Therapy in Combination With Peginterferon for Patients With Metastatic Melanoma [NCT02379195]Phase 1/Phase 212 participants (Actual)Interventional2014-11-30Completed
Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis [NCT00775931]Phase 2/Phase 37 participants (Actual)Interventional2008-08-31Completed
Exploratory Study Evaluating the Interest of PET to 18F-Fludarabine for the Initial Assessment and End-treatment Evaluation of Patients With Symptomatic Multiple Myeloma in the First Line of Treatment, Not Candidates for Marrow Autograft [NCT03832127]Phase 135 participants (Anticipated)Interventional2022-04-01Not yet recruiting
A Phase I/IIa Open Label, Non-Randomized, Multicenter Study of CYNK-101 in Combination With Trastuzumab and Pembrolizumab in Patients With Locally Advanced Unresectable or Metastatic HER2-Positive Gastric or Gastroesophageal Junction (G/GEJ) Aenocarcinoma [NCT05207722]Phase 1/Phase 252 participants (Anticipated)Interventional2022-04-14Active, not recruiting
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 Feasibility Study of Early Allogeneic Hematopoietic Cell Transplantation for Relapsed or Refractory High-Grade Myeloid Neoplasms" [NCT02756572]Phase 230 participants (Actual)Interventional2016-09-22Completed
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
Open-label Phase I, Multi-center Study to Determine the Recommended Dose of CYAD-211 After a Non-myeloablative Preconditioning Chemotherapy in Multiple Myeloma Patients With Relapsed or Refractory Disease [NCT04613557]Phase 118 participants (Actual)Interventional2020-11-16Active, not recruiting
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis [NCT04384692]Phase 245 participants (Anticipated)Interventional2020-12-18Recruiting
Phase 1/2, Open-label Study Evaluating Safety of Repeat Administration of Ad/PNP-F-araAMP (Ad/PNP Administered Intratumorally Followed by Intravenous Fludarabine Phosphate) in Subjects With Recurrent, Local Head and Neck Cancer [NCT03754933]Phase 1/Phase 210 participants (Anticipated)Interventional2019-02-11Recruiting
A Pilot Study of Lymphodepletion Plus Adoptive Cell Transfer With T -Cells Transduced With CXCR2 and NGFR Followed by High Dose Interleukin-2 in Patients With Metastatic Melanoma [NCT01740557]Phase 1/Phase 210 participants (Actual)Interventional2015-01-28Completed
A Phase I Study of 5-Azacytidine in Combination With Chemotherapy for Children With Relapsed or Refractory ALL or AML [NCT01861002]Phase 115 participants (Actual)Interventional2013-05-22Completed
An Multicenter, Randomized, Controlled, Prospective Clinical Study of Mitoxantrone Liposome Combined With PTCy as Conditioning Regimen in Allo-HSCT in Acute Leukemia [NCT05739630]Phase 2/Phase 360 participants (Anticipated)Interventional2023-01-01Recruiting
Renal Allograft Tolerance Through Mixed Chimerism - SMC/MGH [NCT04540380]Phase 16 participants (Anticipated)Interventional2021-09-01Recruiting
Hematopoietic Stem Cell Transplant for Sickle Cell Disease [NCT02065596]Phase 1/Phase 225 participants (Anticipated)Interventional2015-10-19Completed
MT2007-12 Allogeneic Natural Killer Cells With Rituximab in Patients With CD20 Positive Relapsed Non-Hodgkin Lymphoma or Chronic Lymphocytic Leukemia. Strategies to Increase Sensitivity of CLL Tumor Cells to Natural Killer Cell-Immune-Mediated Cytolysis [NCT00625729]Phase 1/Phase 26 participants (Actual)Interventional2008-01-31Terminated(stopped due to No patients exhibited natural killer cell expansion (primary endpoint).)
Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide [NCT02282904]Phase 1/Phase 27 participants (Actual)Interventional2014-10-23Terminated
Clinical Study of the Efficacy and Safety of XPO-1 Inhibitors in Combination With CAR-T Cells in Relapsed Refractory B-cell Non-Hodgkin's Lymphoma [NCT05322330]Phase 220 participants (Anticipated)Interventional2022-02-10Recruiting
Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis [NCT00572897]Phase 266 participants (Actual)Interventional2007-08-31Completed
Phase I Clinical Trial Using an Engineered Peripheral Blood Graft for Haploidentical Transplantation [NCT02960646]Phase 111 participants (Actual)Interventional2017-01-18Completed
Phase I Trial of Anti-GPC3 Chimeric T Cells for Subjects With GPC3-Positive Advanced Hepatocellular Carcinoma [NCT03084380]Phase 1/Phase 220 participants (Anticipated)Interventional2017-06-01Not yet recruiting
Open-label, Single-arm Trial to Evaluate Antitumor Activity, Safety, and Pharmacokinetics of Isatuximab Used in Combination With Chemotherapy in Pediatric Patients From 28 Days to Less Than 18 Years of Age With Relapsed/Refractory B or T Acute Lymphoblast [NCT03860844]Phase 267 participants (Actual)Interventional2019-08-06Terminated(stopped due to Study was prematurely stopped due to sponsor decision (stage 2 efficacy criteria not met); not due to safety concerns.)
A Phase I/II Study of the Selective Inhibitor of Nuclear Export Selinexor (KPT-330) in Combination With Fludarabine and Cytarabine in Patients With Refractory or Relapsed Leukemia or Myelodysplastic Syndrome [NCT02212561]Phase 119 participants (Actual)Interventional2014-08-31Completed
Single Patient Protocol: A Study Using the Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Mutated or Viral Neoantigens in a Patient With Metastatic Cancer Plus the Administration of Pembrolizumab [NCT04536922]Phase 20 participants (Actual)Interventional2021-01-27Withdrawn(stopped due to Subject no longer able to participate in this single pt study.)
Prognostic Factors and Risk-Adapted Therapy in Patients With Early Stage Chronic Lymphocytic Leukemia [NCT00262782]Phase 3877 participants (Actual)Interventional1997-04-30Completed
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime [NCT01821781]Phase 220 participants (Anticipated)Interventional2013-03-31Recruiting
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
First Line Therapy With Methotrexate (MTX) and Second Line Therapy With Fludarabine of Patients With T-Cell Large Granular Lymphocyte Leukemia (T-LGL) [NCT00278265]Phase 212 participants (Actual)Interventional2005-06-30Terminated(stopped due to slow recruitment)
Treatment of Advanced Chronic Lymphocytic Leukemia (CLL) Fludarabine, Mitoxantrone and Cyclophosphamide With or Without G-CSF [NCT00416910]Phase 383 participants (Actual)Interventional1999-07-31Terminated(stopped due to low recruitment)
Purine Analog-Based Conditioning for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia [NCT00427336]9 participants (Actual)Interventional2000-12-31Completed
Darbepoetin Alfa in Patients With Chronic Lymphocytic Leukemia and Comorbidity [NCT00281892]Phase 397 participants (Actual)Interventional2004-09-30Completed
Phase II Trial of Combined Immunochemotherapy With Fludarabine, Cyclophosphamide and Rituximab in Patients With B-PLL [NCT00281931]Phase 221 participants (Anticipated)Interventional1999-09-30Terminated
Nonmyeloablative Bone Marrow Transplants in Hematologic Malignancies: Dose Finding Study for Post-Transplant Immunosuppression [NCT00255710]Phase 160 participants (Anticipated)Interventional2002-07-31Completed
Non-Myeloablative HLA-Matched Sibling Allogeneic Peripheral Blood Stem Cell Transplantation for Metastatic Renal Cell Carcinoma [NCT00262886]Phase 235 participants (Anticipated)Interventional2001-08-31Completed
Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases [NCT00295971]Phase 121 participants (Actual)Interventional2005-04-30Completed
Feasibility of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation Followed by Donor Lymphocyte Infusions for Children at High Risk for Complications With Conventional Transplantation [NCT00301860]8 participants (Actual)Interventional2003-01-31Terminated(stopped due to lack of efficacy)
Study of CD19/CD20 Bispecific Chimeric Antigen Receptor (CAR)-T Cells for the Treatment of Relapsed or Refractory B-Cell Lymphomas and Chronic Lymphocytic Leukemia (CD20 - Cluster of Differentiation Antigen 20) [NCT04007029]Phase 124 participants (Anticipated)Interventional2019-10-04Recruiting
Consolidation With Campath-1H After FMC Induction in Patients With T-cell Chronic Lymphocytic Leukemia [NCT00278213]Phase 217 participants (Anticipated)Interventional2002-09-30Completed
A Phase Ib Open-Label Study Evaluating the Safety and Efficacy of NKTR-255 in Combination With CD19-Directed CAR-T Cell Therapy in Patients With Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL) [NCT05359211]Phase 124 participants (Anticipated)Interventional2022-12-08Recruiting
Evaluation of Safety and Efficacy of Treosulfan-cytarabine-fludarabine (FLAT) Combination Prior to Autologous Stem Cell Transplant (HSCT) in Elderly Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) [NCT03961919]Phase 215 participants (Actual)Interventional2009-02-10Completed
BRAVO: Newly-Diagnosed Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Recovery From Focal Radiotherapy Alone or Focal Radiotherapy and Dose-intensified Temozolomide (Phase I) [NCT03396575]Phase 121 participants (Anticipated)Interventional2018-07-17Active, not recruiting
A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Pediatric and Adolescent Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4) [NCT02625480]Phase 1/Phase 2116 participants (Anticipated)Interventional2016-02-01Recruiting
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
A Phase II Study Using a Peptide Vaccine With or Without Aldesleukin Following a Lymphodepleting Chemotherapy and Reinfusion of Autologous Lymphocytes Depleted of T Regulatory Lymphocytes in Metastatic Melanoma [NCT00303836]Phase 258 participants (Actual)Interventional2005-11-30Terminated
Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission [NCT00305708]Phase 1/Phase 240 participants (Anticipated)Interventional2000-08-31Completed
A Multicenter Study to Assess the Antitumor Effect and Safety of Fludarabine Phosphate Tablet (SH T 586) in Combination With Rituximab Administered in 6 Treatment Cycles (1 Treatment Cycle: Rituximab 375 mg/m2 iv on Day 1 Along With 5-Consecutive Day Oral [NCT00311129]Phase 241 participants (Actual)Interventional2005-12-31Completed
A Dose Ranging Trial of MART-1/gp100/Tyrosinase/NY-ESO-1 Peptide-Pulsed Dendritic Cells Matured Using Cytokines With Autologous Lymphocyte Infusion With or Without Escalating Doses of Fludarabine for Patients With Chemotherapy-naive Metastatic Melanoma [NCT00313508]Phase 118 participants (Actual)Interventional2006-02-28Completed
A Phase 2, Open-Label, Multicenter, Basket Study Evaluating the Efficacy of Brexucabtagene Autoleucel in Adults With Rare B-cell Malignancies (ZUMA-25) [NCT05537766]Phase 290 participants (Anticipated)Interventional2022-11-01Recruiting
A Dose Escalation Study of Intensity Modulated Total Marrow Irradiation (IMRT-TMI) Followed by Fludarabine as a Myeloablative Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Relapsed and Refractory Hematologic [NCT05201183]Phase 1/Phase 20 participants (Actual)Interventional2023-10-31Withdrawn(stopped due to insufficient staff and updates in standard practice have made it difficult to recruit participants)
A Phase II Study Of Pooled Unrelated Donor Umbilical Cord Blood (UCB) Transplant For Patients With Hematologic Malignancies Needing Allogeneic Stem Cell Transplant But Do Not Have A Related HLA-Matched Donor [NCT01500161]Phase 21 participants (Actual)Interventional2011-11-30Terminated(stopped due to Insufficient accruals)
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
Phase Ib Study of TBI-1301 (NY-ESO-1 Specific TCR Gene Transduced Autologous T Lymphocytes) in Patients With Solid Tumors [NCT02869217]Phase 122 participants (Anticipated)Interventional2016-09-30Active, not recruiting
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
Phase II Trial of Reduced Intensity Conditioning (RIC) and Allogeneic Transplantation of Umbilical Cord Blood (UCB) From Unrelated Donors in Patients With Hematologic Malignancies [NCT01622556]Phase 26 participants (Actual)Interventional2012-01-31Terminated
A Phase I Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas [NCT01625351]Phase 123 participants (Actual)Interventional2012-08-20Completed
A Randomized Trial Comparing CD3/CD19 Depleted or CD3 Depleted/CD56 Selected Haploidentical Donor Natural Killer (NK) Cell Based Therapy in Older Adults With Acute Myelogenous Leukemia in First Complete Remission [NCT01639456]Phase 20 participants (Actual)Interventional2013-10-31Withdrawn(stopped due to study was abandoned and a new study was written to replace this one)
Hematopoietic Stem Cell Transplantation Using Alternate Donor Umbilical Cord Blood Options [NCT01652014]Phase 20 participants (Actual)Interventional2014-01-31Withdrawn(stopped due to Funding unavailable)
Autologous CD19 Specific T-cell Infusion in Patients With B-cell Chronic Lymphocytic Leukemia (B-CLL) [NCT01653717]Phase 130 participants (Anticipated)Interventional2013-06-11Completed
Phase I Trial of Fludarabine and Methoxyamine (TRC102) for Relapsed or Refractory Hematologic Malignancies [NCT01658319]Phase 120 participants (Actual)Interventional2011-05-31Completed
Use of Umbilical Cord Blood Cell in the Preparative Regimen of Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT00427557]Phase 231 participants (Actual)Interventional2006-10-31Completed
A Phase II Study of High Dose Chemotherapy With Autologous Hematopoietic Progenitor Cell Transplant for Multiple Sclerosis That Failed at Least Two Lines of Therapy [NCT01679041]Phase 21 participants (Actual)Interventional2012-11-30Terminated(stopped due to Insufficient accruals; PI leaving site)
A Phase I Study of Venetoclax to Augment Epigenetic Modification and Chemotherapy in Pediatric and Young Adult Patients With Relapsed and Refractory Acute Myeloid Leukemia [NCT05317403]Phase 140 participants (Anticipated)Interventional2023-03-31Recruiting
Dose Escalation Study of CD19 Chimeric Antigen Receptor (CAR) T Cells With a CD34 Selection Marker in Adults With Recurrent or Refractory B Cell Malignancies [NCT04214886]Phase 124 participants (Anticipated)Interventional2019-12-31Active, not recruiting
Phase II Trial of HLA Haploidentical Natural Killer Cell Infusion for Treatment of Relapsed or Persistent Leukemia Following Allogeneic Hematopoietic Stem Cell Transplant [NCT00526292]Phase 212 participants (Actual)Interventional2007-08-31Completed
Dose Escalation Study Phase I/II of Umbilical Cord Blood-Derived CAR-Engineered NK Cells in Conjunction With Lymphodepleting Chemotherapy in Patients With Relapsed/Refractory B-Lymphoid Malignancies [NCT03056339]Phase 1/Phase 244 participants (Actual)Interventional2017-06-21Completed
An Open-label Phase II Study of the Efficacy and Safety of the Combination of Fludarabine, Cyclophosphamide and Rituximab in Patients With Chronic Lymphocytic Leukaemia Who Are Newly Diagnosed, Have Relapsed or Are Resistant to First-Line Treatment [NCT00812669]Phase 252 participants (Actual)Interventional2008-08-18Completed
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
Epigenetic Priming With 5-Azacytidine Prior to in Vivo T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With High Risk Myeloid Malignancies in Morphologic Remission [NCT02497404]Phase 240 participants (Actual)Interventional2015-02-13Completed
Treatment Protocol for Newky Diagnosed Adult Ph-Chromosome Positive (BCR::ABL1) Acute Lymphoblastic Leukemia (LALPh2022) [NCT06175702]150 participants (Anticipated)Observational2023-12-25Not yet recruiting
CARTIMMUNE: A Single-Center Study of Patients With Autoimmune Diseases Receiving an Autologous Fully-Human Anti-CD19 Chimeric Antigen Receptor T-Cell (KYV 101) [NCT06152172]Phase 124 participants (Anticipated)Interventional2024-02-29Not yet recruiting
Phase I/II Study of TROP2 CAR Engineered IL15-transduced Cord Blood-derived NK Cells Delivered Intraperitoneally for the Management of Platinum Resistant Ovarian Cancer, Mesonephric-like Adenocarcinoma, and Pancreatic Cancer [NCT05922930]Phase 1/Phase 251 participants (Anticipated)Interventional2023-10-11Recruiting
An Adaptive Phase 3, Randomized, Open-Label, Multicenter Study to Compare the Efficacy and Safety of Axicabtagene Ciloleucel Versus Standard of Care Therapy as First-Line Therapy in Subjects With High-Risk Large B-Cell Lymphoma (ZUMA-23) [NCT05605899]Phase 3300 participants (Anticipated)Interventional2023-02-10Recruiting
A Phase 3 Randomized Study Comparing Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (AS [NCT05257083]Phase 3750 participants (Anticipated)Interventional2023-10-10Recruiting
Phase I Study of GPC3 Targeted CAR-T Cell Therapy in Advanced GPC3 Expressing Hepatocellular Carcinoma (HCC) [NCT05003895]Phase 138 participants (Anticipated)Interventional2021-12-08Recruiting
A Phase II Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children and Young Adults With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT02626715]Phase 222 participants (Actual)Interventional2015-09-04Completed
Haploidentical Stem Cell Transplantation With Fixed Dose of T Cells After in Vitro T Cell Depletion Using CD3 Monoclonal Antibody for Children With Acquired Severe Aplastic Anemia [NCT01759732]Phase 210 participants (Anticipated)Interventional2012-09-30Recruiting
Phase 2 Multicenter, Study to Assess the Efficacy and the Safety of Front-line Fludarabine, Cyclophoshamide and Ofatumumab (FCO2) Chemoimmunotherapy in Young (≤65 Yrs) Patients With Chronic Lymphocytic Leukemia (CLL). [NCT01762202]Phase 280 participants (Actual)Interventional2013-11-05Completed
A Phase I/II Clinical Trial Testing the Safety and Feasibility of IL-21-Expanded Natural Killer Cells for the Induction of Relapsed/Refractory Acute Myeloid Leukemia [NCT01787474]Phase 130 participants (Actual)Interventional2014-05-19Completed
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
A Phase II Multicentre, Randomized, Controlled Open-label Study on the Use of Anti-thymocyte Globulin and Rituximab for Immunomodulation of Graft-versus-host Disease in Allogeneic Matched Transplants for Non Malignancies [NCT01810926]Phase 2130 participants (Anticipated)Interventional2011-09-30Recruiting
Prospective, Phase II Randomized Study to Compare Busulfan-fludarabine Reduced-intensity Conditioning (RIC) With Thiotepa-fludarabine RIC Regimen Prior to Allogeneic Transplantation of Hematopoietic Cells for the Treatment of Myelofibrosis [NCT01814475]Phase 262 participants (Actual)Interventional2011-07-31Completed
A Phase 2 Open-Label, Multicenter Study Evaluating the Safety and Efficacy of Axicabtagene Ciloleucel in Combination With Rituximab in Participants With Refractory Large B-Cell Lymphoma (ZUMA-14) [NCT04002401]Phase 227 participants (Actual)Interventional2019-11-05Completed
Lymphodepleting Chemotherapy With Rituximab and Allogeneic Natural Killer Cells for Patients With Refractory Lymphoid Malignancies (MT2009-15) [NCT01181258]Phase 216 participants (Actual)Interventional2010-08-31Completed
Phase 1 Trial of Ivosidenib and FLAG Chemotherapy in Relapsed/Refractory IDH1+ Acute Myeloid Leukemia (AML) [NCT04250051]Phase 125 participants (Anticipated)Interventional2020-12-21Recruiting
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
Partially HLA Mismatched (Haploidentical) Allogeneic Bone Marrow Transplantation for Patients With Hematologic Malignancies [NCT01749293]Phase 23 participants (Actual)Interventional2012-08-30Terminated(stopped due to The protocol design is being reconfigured in order to open a new study.)
Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis After Allogeneic Non-myeloablative Peripheral Blood Stem Cell Transplantation [NCT00282282]Phase 231 participants (Actual)Interventional2006-01-31Completed
G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With Secondary Acute Myeloid Leukemia Evolving From MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT05449899]Phase 2/Phase 3232 participants (Anticipated)Interventional2022-07-31Recruiting
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
Autologous Activated T-Cells Transduced With A 3rd Generation GD-2 Chimeric Antigen Receptor And iCaspase9 Safety Switch Administered To Patients With Relapsed Or Refractory Neuroblastoma (GRAIN) [NCT01822652]Phase 111 participants (Actual)Interventional2013-08-31Active, not recruiting
Pilot Study of Affinity-enhanced Anti-NY-ESO-1 TCR Engineered Autologous T Cells in NSCLC Patients [NCT03029273]Phase 120 participants (Anticipated)Interventional2017-03-21Recruiting
Safety and Efficiency of Anti-CD19/CD22 Tandem Fully Human Chimeric Antigen Receptor (CAR)-Transduced T-cell Therapy for Pediatric and Young Adult Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia: a Single Centre, Non-randomised, Open [NCT04499573]Phase 1/Phase 250 participants (Anticipated)Interventional2020-07-27Active, not recruiting
Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation [NCT02007863]2 participants (Actual)Interventional2008-08-31Completed
A Phase II Study of Lymphodepletion Followed by Autologous Tumor-Infiltrating Lymphocytes and High-Dose Aldesleukin for Human Papillomavirus-Associated Cancers [NCT01585428]Phase 229 participants (Actual)Interventional2012-04-13Completed
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
Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc) [NCT00684255]Phase 11 participants (Actual)Interventional2007-08-31Terminated(stopped due to inactive)
A Randomized Study of Combined Haplo-identical Umbilical Cord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies [NCT01745913]Phase 22 participants (Actual)Interventional2012-10-26Terminated(stopped due to Slow accrual)
Biparental HLA Haplotype Disparate T-cell Depleted Transplants for Patients Lacking an HLACompatible Donor [NCT01598025]3 participants (Actual)Interventional2012-05-02Terminated(stopped due to Closed due to poor accrual)
A Pilot Study Using High Dose Busulfan and Bortezomib as Part of Allogeneic Transplant Conditioning Regimen for High Risk Multiple Myeloma Patients. [NCT01534143]Phase 21 participants (Actual)Interventional2012-02-29Terminated(stopped due to Data was not collected, because funding was unavailable to continue study.)
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation With Bevacizumab for Advanced Solid Tumor [NCT00523809]Phase 25 participants (Actual)Interventional2007-08-31Terminated(stopped due to Slow accrual.)
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
Methotrexate Alone Versus Combination of Methotrexate and Subcutaneous Fludarabine for Severe Rheumatoid Arthritis: Safety, Tolerance and Efficacy [NCT00001677]Phase 240 participants Interventional1998-06-30Completed
Phase 1 Dose Escalation Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory CD19/CD22-expressing B Cell Malignancies [NCT03448393]Phase 1140 participants (Anticipated)Interventional2018-03-26Recruiting
A Phase I and Expansion Cohort Study of Selinexor and Venetoclax in Combination With Chemotherapy in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia [NCT04898894]Phase 142 participants (Anticipated)Interventional2021-11-15Recruiting
NK Cells With HLA Compatible Hematopoietic Transplantation for High Risk Myeloid Malignancies [NCT01823198]Phase 1/Phase 263 participants (Actual)Interventional2013-06-11Completed
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
Compassionate Use Administration of Autologous CAR T Cells Targeting the CD19 Antigen and Containing the Inducible Caspase 9 Safety Switch [NCT03594162]0 participants Expanded AccessNo longer available
Multicenter, Prospective, Open-label, Single-arm, Phase I-II Clinical Trial to Analyze Induction Therapy With a Combination of Fludarabine, Idarubicin, Cytarabine, G-CSF and Plerixafor for the Treatment of Young Patients With Relapsed or Refractory AML [NCT01435343]Phase 1/Phase 255 participants (Actual)Interventional2012-07-31Completed
Phase I Clinical Trial of Anti-CD19/20/22 Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Lymphoid Malignancies (Non-Hodgkin Lymphoma, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia, B-Prolymphocytic Leukemia) [NCT05418088]Phase 136 participants (Anticipated)Interventional2022-06-30Recruiting
Reduced Intensity Matched Sibling Bone Marrow Transplantation for Sickle Cell Anemia in Patients 2-30 Years Old [NCT01877837]Phase 330 participants (Actual)Interventional2011-06-30Completed
Phase I/Ib Study of TBI-2001 for Patients With Relapsed or Refractory CD19+ B-cell Lymphoma, Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma (SLL) [NCT05963217]Phase 119 participants (Anticipated)Interventional2023-07-26Recruiting
Phase I Study of Autologous Transgenic T-Cells Expressing High Affinity Mesothelin-Specific T-Cell Receptor (TCR) (FH-TCR Tᴍsʟɴ) in Patients With Metastatic Pancreatic Ductal Adenocarcinoma [NCT04809766]Phase 115 participants (Anticipated)Interventional2021-12-14Recruiting
A Phase 2 Multi-Center Study Evaluating the Safety and Efficacy of CD30-Directed Genetically Modified Autologous T Cells (CD30.CAR-T) in Adult and Pediatric Patients With Relapsed or Refractory Classical Hodgkin Lymphoma [NCT04268706]Phase 297 participants (Anticipated)Interventional2021-02-01Active, not recruiting
T-cell Therapy in Combination With Checkpoint Inhibitors for Patients With Advanced Ovarian-, Fallopian Tube- and Primary Peritoneal Cancer [NCT03287674]Phase 1/Phase 27 participants (Actual)Interventional2017-10-09Completed
Delayed Infusion of Ex Vivo Anergized Peripheral Blood Mononuclear Cells Following CD34 Selected Peripheral Blood Stem Cell Transplantation From a Haploidentical Donor for Patients With Acute Leukemia and Myelodysplasia [NCT00376480]Phase 119 participants (Actual)Interventional2005-06-30Completed
Autologous Hematopoietic Stem Cell Transplant for Patients With Systemic Sclerosis and Cardiac Dysfunction [NCT03593902]Phase 2/Phase 39 participants (Actual)Interventional2018-05-17Terminated(stopped due to PI Sabbatical)
Transplantation Of Umbilical Cord Blood From Unrelated Donors In Patients With Haematological Diseases Using A Myeloablative Conditioning Regimen [NCT02310997]Phase 211 participants (Actual)Interventional2011-07-31Terminated(stopped due to Trial closed early due to poor recruitment)
Clinical Study on the Safety and Efficacy of QN-030a in Acute Myeloid Leukemia With Minimal Residual Disease [NCT05601830]Phase 118 participants (Anticipated)Interventional2022-10-28Recruiting
PRO#0118: A Phase I Study of Decitabin in Combination With Fludarabin and Busulfan as a Reduced Intensity Conditioning Regimen for the Treatment of Myeloid Malignancies [NCT01455506]Phase 120 participants (Actual)Interventional2009-05-31Completed
A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia [NCT01187017]Phase 1/Phase 21 participants (Actual)Interventional2010-08-31Completed
Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Anemia From HLA Matched or Partially-Matched Related Donors [NCT01350232]2 participants (Actual)Interventional2009-09-30Terminated(stopped due to Poor accrual)
A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells [NCT03333486]Phase 231 participants (Actual)Interventional2017-12-07Active, not recruiting
Phase 1 Trial of Umbilical Cord Blood Natural Killer Cells (CB-NK) Expressing Soluble IL-15 (sIL-15) and PD-L1 +/- Atezolizumab in Non-Small Cell Lung Cancer Patients Refractory to PD-1/PD-L1 Immune Checkpoint Inhibitors [NCT05334329]Phase 121 participants (Anticipated)Interventional2022-07-20Recruiting
Phase II Study of Timed Sequential Busulfan in Combination With Fludarabine in Allogeneic Stem Cell Transplantation [NCT01572662]Phase 2201 participants (Actual)Interventional2012-04-11Completed
Non-Myeloablative Chemotherapy Followed by Unrelated Allogeneic Stem Cell Transplantation in Patients With Advanced Hematologic Malignancies: A Pilot Study [NCT00004114]Phase 10 participants (Actual)InterventionalWithdrawn(stopped due to Study never opened)
A Single Dose-escalation and Dose-expansion Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Allogeneic CAR-T Targeting CD19 in Patients With Refractory or Relapsed B Cell Lymphoma. [NCT05776407]Phase 1/Phase 212 participants (Anticipated)Interventional2023-05-31Not yet recruiting
Multicenter Trial for Treatment of Acute Lymphocytic Leukemia in Adults (Pilot Study 06/99) [NCT00199056]Phase 4225 participants Interventional1999-10-31Completed
A Phase I Study of Fludarabine With Radiotherapy in Patients With Locally Advanced Squamous Cell Carcinoma of the Head and Neck [NCT00004246]Phase 116 participants (Actual)Interventional1995-06-07Completed
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
Phase I, Open-label Study Evaluating the Safety of Escalating Doses of Ad/PNP-F-araAMP (Ad/PNP Administered Intratumorally With Co-administration of Fludarabine Phosphate Intravenously) in Subjects With Advanced Solid Tumors [NCT01310179]Phase 112 participants (Actual)Interventional2011-02-28Completed
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 Phase II Study to Evaluate the Efficacy of Posttransplant Cyclophosphamide for Prevention of Chronic Graft-versus-Host Disease After Allogeneic Peripheral Blood Stem Cell Transplantation [NCT01427881]Phase 243 participants (Actual)Interventional2011-09-30Completed
Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism [NCT00176852]Phase 2/Phase 322 participants (Actual)Interventional2002-06-30Completed
A Phase II Study of Cyclophosphamide, Fludarabine, Alemtuzumab, and Rituximab (CFAR) in Previously Treated Patients With Chronic Lymphocytic Leukemia (CLL) [NCT01082939]Phase 280 participants (Actual)Interventional2002-12-31Completed
An International Randomised Clinical Trial of Therapeutic Interventions to Assess the Effects on Outcome in Adults With Acute Myeloid Leukaemia and High Risk Myelodysplasia Undergoing Allogeneic Stem Cell Transplantation. [NCT04217278]Phase 2/Phase 3333 participants (Actual)Interventional2020-01-27Active, not recruiting
Allogeneic Stem Cell Transplant With a Novel Conditioning Therapy Using Helical Tomotherapy, Melphalan, and Fludarabine in Hematological Malignancies [NCT00544466]Phase 1/Phase 275 participants (Actual)Interventional2006-07-31Active, not recruiting
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)
Phase I/II Randomized Study of Clofarabine, Idarubicin, and Cytarabine (CIA) Versus Fludarabine, Idarubicin, and Cytarabine (FLAI) in Acute Myelogenous Leukemia and High-Risk Myelodysplastic Syndrome [NCT01289457]Phase 1/Phase 2282 participants (Actual)Interventional2011-02-02Completed
A Phase II Study of EL625 in Patients in Persistent Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma [NCT00636155]Phase 220 participants (Actual)Interventional2008-02-29Terminated(stopped due to funding)
Phase II Study of Metastatic Melanoma Using a Nonmyeloablative Lymphodepleting Regimen Followed by Melanoma-Reactive T-Cells Sensitized in Vitro With Peptide-Pulsed Drosophila Cells [NCT01271907]Phase 23 participants (Actual)Interventional2011-04-15Terminated(stopped due to Study was terminated due to withdrawal of CRADA partner.)
A Multicenter, Prospective Trial to Evaluate the Role of NK Cell KIR Epitope Mismatch on Mortality and Disease Relapse in T-Cell Depleted Hematopoietic Stem Cell Transplantation From HLA-C Mismatched, Unrelated Donors for Myeloid Malignancies [NCT00392782]Phase 224 participants (Actual)Interventional2005-07-31Terminated
Phase II Trial of Triapine (NSC #663249, 3-Aminopyridine-2-Carboxaldehyde Thiosemicarbone) Plus Fludarabine (NSC #312887, Fludarabine Monophosphate) in Adults With Aggressive Myeloproliferative Disorders (MPDs) Including Chronic Myelomonocytic Leukemia (C [NCT00381550]Phase 235 participants (Actual)Interventional2006-08-31Completed
Prospective Randomised Multicenter Study for Therapy Optimization of Recurrent, Progressive Low Grade Non-Hodgkin Lymphomas and Mantle Cell Lymphomas [NCT01456351]Phase 3230 participants (Actual)Interventional2003-09-30Completed
Once Daily Intravenous Busulfex as Part of Reduced-toxicity Conditioning for Patients With Relapsed/Refractory Hodgkin's and Non-Hodgkin's Lymphomas Undergoing Allogeneic Hematopoietic Progenitor Cell Transplantation - A Multicenter Phase II Study [NCT01203020]Phase 222 participants (Actual)Interventional2010-10-12Completed
Phase I/II Study of Metastatic Cancer That Expresses NY-ESO-1 Using Lymphodepleting Conditioning Followed by Infusion of Gene Engineered Lymphocytes Cotransduced With Genes Encoding IL-12 and Anti-NY ESO-1 TCR [NCT01457131]Phase 12 participants (Actual)Interventional2011-10-06Terminated
Phase II Study of Lymphocytes Generated With Engineered Cells for Costimulation Enhancement in Patients With Metastatic Melanoma Following Lymphodepletion [NCT01369875]Phase 22 participants (Actual)Interventional2011-06-17Terminated(stopped due to Premature closure. Protocol did not meet its primary objective.)
CAR- PRISM (PRecision Intervention Smoldering Myeloma): A Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against BCMA in High-Risk Smoldering Myeloma [NCT05767359]Phase 220 participants (Anticipated)Interventional2023-04-19Recruiting
Administration of T Lymphocytes Expressing the CD30 Chimeric Antigen Receptor (CAR) for Patients With CD30+ Nonseminomatous Germ Cell Tumors (NSGCT) [NCT05634785]Phase 218 participants (Anticipated)Interventional2022-12-09Recruiting
A Phase 1 Study of Autologous Activated T-cells Targeting the B7-H3 Antigen in Subjects With Recurrent Epithelial Ovarian Cancer [NCT04670068]Phase 121 participants (Anticipated)Interventional2021-01-27Recruiting
Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA [NCT03821987]55 participants (Anticipated)Interventional2018-12-17Recruiting
A Phase 1/2 Study of Vadastuximab Talirine Administered in Sequence With Allogeneic Hematopoietic Stem Cell Transplant in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) [NCT02614560]Phase 1/Phase 214 participants (Actual)Interventional2015-11-30Terminated
German Multicenter Study for Treatment Optimisation in Acute Lymphoblastic Leukemia in Adults and Adolescents Above 15 Years (Amend 3) (GMALL 07/2003) [NCT00198991]Phase 41,883 participants (Actual)Interventional2003-04-30Completed
Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies [NCT00153985]Phase 22 participants (Actual)Interventional2004-03-31Completed
A Phase II Study Of Allogeneic Transplant For Older Patients With AML In First Morphologic Complete Remission Using A Non-Myeloablative Preparative Regimen [NCT00070135]Phase 2121 participants (Actual)Interventional2004-01-31Completed
A Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation [NCT01596699]Phase 216 participants (Actual)Interventional2012-05-24Terminated(stopped due to Triggering of futility rule; Interim analysis suggested no statistical superiority over historic controls)
Donor Statin Treatment for Prevention of Severe Acute GVHD After Nonmyeloablative Hematopoietic Cell Transplantation [NCT01527045]Phase 247 participants (Actual)Interventional2012-09-25Completed
Safety and Efficacy of Universal Chimeric Antigen Receptor-modified AT19 Cells in Patients With CD19+ Relapsed/Refractory Hematological Malignancies: a Single-center, Open-label, Single-arm Clinical Study [NCT04796688]Phase 127 participants (Anticipated)Interventional2021-03-10Recruiting
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
A Phase I Study to Evaluate Safety, Feasibility and Immunologic Response of Adoptive T Cell Transfer With or Without Dendritic Cell Vaccination in Patients With Metastatic Melanoma [NCT01946373]Phase 120 participants (Anticipated)Interventional2013-10-31Recruiting
Safety and Efficacy of 90Y Zevalin in Nonmyeloablative Transplantation for Lymphoid Malignancies [NCT00048737]Phase 1/Phase 270 participants (Actual)Interventional2002-10-31Completed
A Multicenter Phase I-II Study of Tumor Vaccine Following Chemotherapy in Patients With Metastatic Breast Cancer Untreated With Chemo/Radiation in the Previous 18 Months: Vaccine-Induced Bias of T-Cell Repertoire Reconstitution After T-Cell Re-Infusion [NCT00048893]Phase 1/Phase 237 participants (Actual)Interventional2002-11-30Terminated(stopped due to The study was closed to accrual due to very poor enrollment.)
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
Phase II Study of Fludarabine and Mitoxantrone, Followed by GM-CSF(Granulocyte-macrophage Colony-stimulating Factor) and Rituximab in Patients With Low Grade Non-Hodgkins Lymphoma: An Analysis of Efficacy and Tolerability [NCT00208975]Phase 215 participants (Actual)Interventional2002-07-31Terminated(stopped due to slow accrual)
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
Phase I Clinical Trial of TSA-CTL (Tumor Specific Antigen-Induced Cytotoxic T Lymphocytes) In the Treatment of Advanced Solid Tumors [NCT02959905]Phase 111 participants (Actual)Interventional2016-12-22Completed
A Phase 1-2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-C19 in Combination With Atezolizumab in Subjects With Refractory Diffuse Large B-Cell Lymphoma (DLBCL) [NCT02926833]Phase 1/Phase 237 participants (Actual)Interventional2016-09-29Completed
A Study of a Reduced-Intensity Conditioning Regimen With Treosulfan and Fludarabine for Allogeneic Hematopoietic Cell Transplantation for Patients With Acute Leukemia [NCT00253513]Phase 1/Phase 260 participants (Actual)Interventional2005-06-30Completed
Protocol Title: An Open Label, Multi-centre Phase 1/2a Study of Modified and Unmodified Autologous Tumour Infiltrating Lymphocytes (TIL) in Patients With Platinum-resistant Ovarian Cancer [NCT04389229]Phase 1/Phase 20 participants (Actual)Interventional2020-07-01Withdrawn(stopped due to Sponsor decision)
Megadose CD34 Selected Progenitor Cells for Transplantation in Patients With Advanced Hematological Malignant Diseases [NCT00038857]Phase 229 participants (Actual)Interventional2001-09-30Completed
AML Therapy With Irradiated Allogeneic Cells [NCT02105116]6 participants (Actual)Interventional2014-02-28Terminated(stopped due to No patients were eligible to receive the experimental component of the protocol therapy.)
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)
A Single-center, Single-arm Exploratory Clinical Trial to Evaluate the Safety and Efficacy of Fully Human Anti-CD5 Chimeric Antigen Receptor T Cells (CT125A Cells) for the Treatment of Relapsed/Refractory CD5+ Hematopoietic Malignancies [NCT04767308]Early Phase 118 participants (Anticipated)Interventional2021-03-31Not yet recruiting
Phase II Clinical Protocol for the Treatment of Patients With Previously Untreated Chronic Lymphocytic Leukemia [NCT00280241]Phase 265 participants (Actual)Interventional2004-06-30Completed
Phase II Study in Metastatic Melanoma or Kidney Cancer Using Autologous Natural Killer Cells Plus Aldesleukin (IL-2) Following a Lymphodepleting Chemotherapy [NCT00328861]Phase 28 participants (Actual)Interventional2006-05-31Completed
A Phase II Trial of Fludarabine in Combination With Daunorubicin and Cytarabine Liposome for Adults With Newly-diagnosed Acute Myeloid Leukemia: University of California Hematologic Malignancies Consortium Protocol 1914 [NCT04425655]Phase 22 participants (Actual)Interventional2020-08-05Terminated(stopped due to Original investigator for the trial has left)
Adoptive Transfer of ImmPACT Expanded Multiple Antigen Specific Endogenously Derived T Cells (MASE-T) in Combination With Lymphodepletion and Anti-PD-1 to Patients With Metastatic Melanoma [NCT04904185]Phase 112 participants (Anticipated)Interventional2021-08-17Recruiting
A Phase I/II Trial of PD-1 Knockout EBV-CTLs for Advanced Stage EBV Associated Malignancies [NCT03044743]Phase 1/Phase 220 participants (Anticipated)Interventional2017-04-07Recruiting
Safety and Efficacy Study of Chimeric Antigen Receptor T (CAR-T) Cells in the Treatment of Relapsed/Refractory Hematological Malignancies [NCT05528887]Phase 110 participants (Anticipated)Interventional2021-09-16Recruiting
Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation [NCT02790515]Phase 252 participants (Anticipated)Interventional2016-07-14Recruiting
Phase I/II Study Using a Non-Myeloablative Lymphocyte Depleting Regimen of Chemotherapy Followed by Infusion of Allogeneic Tumor-Reactive Lymphocyte Cell Line DMF5 in Metastatic Melanoma [NCT00924001]Phase 1/Phase 21 participants (Actual)Interventional2007-08-31Terminated(stopped due to study was stopped due to low accrual)
Therapy for Chronic Cold Agglutinin Disease: A Prospective, Non-randomized International Multicentre Study on the Safety and Efficacy of Rituximab in Combination With Fludarabine. [NCT00373594]Phase 230 participants (Anticipated)Interventional2005-06-30Completed
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
A Pilot Study of Daunorubicin-cytarabine Liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) as Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia Followed by Consolidation With a CD34+-Selected Allograft [NCT04982354]Phase 1/Phase 220 participants (Anticipated)Interventional2022-07-05Recruiting
Phase 2 Study of a Reduced-toxicity Myeloablative Conditionning Regimen Using Fludarabine and Full Doses of iv Busulfan in Pediatric Patients Not Eligible for Standard Myeloablative Conditioning Regimens [NCT01572181]Phase 250 participants (Actual)Interventional2012-04-30Completed
A Phase II Investigation of Vorinostat in Combination With Intravenous Fludarabine, Mitoxantrone, and Dexamethasone in Patients With Relapsed or Refractory Mantle Cell Lymphoma [NCT01578343]Phase 220 participants (Actual)Interventional2012-06-30Terminated(stopped due to we collected data of a total of 19 patients for an interim analysis. but there are less than 7 responses out of the initial 19 patients.)
Subcutaneous Bortezomib, Cyclophosphamide and Rituximab (BCR) Versus Fludarabine, Cyclophosphamide and Rituximab (FCR) for Initial Therapy of Waldenstrőm's Macroglobulinaemia (WM): a Randomized Phase II Trial [NCT01592981]Phase 260 participants (Actual)Interventional2013-01-31Completed
FT500 as Monotherapy and in Combination With Immune Checkpoint Inhibitors in Subjects With Advanced Solid Tumors (Phase 1) [NCT03841110]Phase 137 participants (Actual)Interventional2019-02-15Completed
A Pilot Clinical Trial Combining PD-1 Blockade, CD137 Agonism and Adoptive Cell Therapy for Metastatic Melanoma [NCT02652455]Early Phase 111 participants (Actual)Interventional2016-03-08Completed
Allogeneic Stem Cell Transplantation With 3-days Busulfan Plus Fludarabine as Conditioning in Patients With Relapsed or Refractory T-, NK/T-cell Lymphomas [NCT02859402]Phase 234 participants (Anticipated)Interventional2016-12-31Recruiting
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
Busulfan Dose Escalation Study Based on AUC in the Setting of Busulfan/Fludarabine Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) [NCT00361140]Phase 472 participants (Actual)Interventional2005-08-31Completed
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
Phase II Study Using a Myeloablative Lymphocyte Depleting Regimen of Chemotherapy and Intensive Total Body Irradiation Followed by Infusion of Tumor Reactive Lymphocytes and Reconstitution With CD34+ Stem Cells in Metastatic Melanoma [NCT00314106]Phase 226 participants (Actual)Interventional2006-04-30Completed
An Open-label, Phase I/II Study to Assess the Safety and Clinical Activity of NKR-2 Treatment Administration After a Non-myeloablative Preconditioning Chemotherapy in Relapse/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Patients. [NCT03466320]Phase 1/Phase 221 participants (Actual)Interventional2018-09-18Completed
Adoptive Transfer of Haploidentical Natural Killer Cells to Treat Refractory or Relapsed AML MT2010-02 [NCT01106950]Phase 215 participants (Actual)Interventional2010-07-31Terminated(stopped due to study drug (Ontak) no longer available)
A Phase I/II, Multicenter, Open-Label, Multi-Arm Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in the Treatment of Pediatric and Young Adult Patie [NCT04029688]Phase 1/Phase 2183 participants (Anticipated)Interventional2020-01-27Recruiting
A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib Administered in Combination With Re-Induction Chemotherapy, and as a Single-Agent Continuation Therapy, in Pediatric Re [NCT03793478]Phase 1/Phase 265 participants (Anticipated)Interventional2018-08-15Recruiting
Phase 3 Open-label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation [NCT03182244]Phase 3276 participants (Actual)Interventional2018-01-15Active, not recruiting
Adoptive Transfer of NY-ESO-1 TCR Engineered Peripheral Blood Mononuclear Cells (PBMC) After a Nonmyeloablative Conditioning Regimen, With Administration of NY-ESO-1157-165 Pulsed Dendritic Cells and Interleukin-2, in Patients With Advanced Malignancies [NCT01697527]Phase 26 participants (Actual)Interventional2012-11-02Active, not recruiting
Phase I Study to Evaluate the Safety of Cellular Adoptive Immunotherapy Using Autologous CD8+ Antigen-Specific T Cell Clones Following Fludarabine Lymphodepletion for Patients With Metastatic Melanoma [NCT00317759]Phase 112 participants (Anticipated)Interventional2003-05-31Completed
Conditioning For Hematopoietic Cell Transplantation With Fludarabine Plus Targeted IV Busulfan and GVHD Prophylaxis With Thymoglobulin, Tacrolimus and Methotrexate in Patients With Myeloid Malignancies [NCT00346359]Phase 240 participants (Anticipated)Interventional2006-03-31Completed
Infusion of Off-the-Shelf Ex Vivo Expanded Cryopreserved Progenitor Cells to Facilitate the Engraftment of a Single CCR5Δ32 Homozygous or Heterozygous Cord Blood Unit in Patients With HIV and Hematological Malignancies [NCT04083170]Phase 210 participants (Anticipated)Interventional2022-10-06Recruiting
A Single-Arm, Open-Label, Phase 1 Study Evaluating the Safety, Efficacy, and Cellular Kinetics/Pharmacodynamics of ALLO-501, an Anti-CD19 Allogeneic CAR T Cell Therapy, And ALLO-647, An Anti-CD52 Monoclonal Antibody, in Patients With Relapsed/Refractory L [NCT03939026]Phase 174 participants (Anticipated)Interventional2019-05-01Active, not recruiting
A Phase I/II Study of IL-15 Administration Following a Non-Myeloablative Lymphocyte Depleting Chemotherapy Regimen and Autologous Lymphocyte Transfer in Metastatic Melanoma [NCT01369888]Phase 1/Phase 23 participants (Actual)Interventional2011-05-31Terminated(stopped due to Closed this protocol due to autoimmune toxicity.)
Combination of Cyclophosphamide and Fludarabine for Lupus Nephritis: Tolerance, Toxicity, Efficacy and Effects on B and T Lymphocyte Regeneration [NCT00001676]Phase 115 participants Interventional1998-01-31Completed
Safety and Efficacy Study of Novel Mesothelin CAR-T Cell Therapy in Patients With Mesothelin-positive Advanced Refractory Solid Tumors [NCT05531708]Phase 10 participants (Actual)Interventional2021-04-02Withdrawn(stopped due to The study is not initiated and we want it to be withdrawn.)
Safety and Efficacy Study of Novel CAR-T Cell Therapy in the Treatment of Hematopoietic and Lymphoid Malignancies [NCT05513612]Phase 10 participants (Actual)Interventional2020-08-01Withdrawn(stopped due to The study is not initiated and we want it to be withdrawn.)
Double Umbilical Cord Blood Transplantation for Patients With Malignant and Non-Malignant Disorders [NCT00801931]Phase 1/Phase 21 participants (Actual)Interventional2007-09-06Terminated(stopped due to Poor accrual)
Phase I/II Study of Adoptive Immunotherapy After Allogeneic HCT With Virus Specific CD8+ T Cells That Have Been Transduced to Express a WT1-Specific T Cell Receptor for Patients With Relapsed AML [NCT01640301]Phase 1/Phase 247 participants (Actual)Interventional2012-12-06Terminated(stopped due to Terminated due to slow accrual.)
A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS) [NCT00002798]Phase 3880 participants (Actual)Interventional1996-08-31Completed
A Randomized Phase II Study of Concurrent Fludarabine + Chimeric Anti-CD20 Monoclonal Antibody IDEC-C2B8 (Rituximab) [NSC# 687451] Induction Followed By Rituximab Consolidation In Untreated Patients With B-Cell Chronic Lymphocytic Leukemia [NCT00003248]Phase 2104 participants (Actual)Interventional1998-03-31Completed
PHASE II TRIAL OF FLUDARABINE AND SANDOSTATIN FOR RELAPSED LOW-GRADE NON-HODGKIN'S LYMPHOMA [NCT00002779]Phase 234 participants (Actual)Interventional1998-02-28Completed
A Phase I Study of G3139 (NSC 683428) in Combination With Salvage Chemotherapy for Treatment of Refractory and Relapsed Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL) [NCT00004862]Phase 124 participants (Actual)Interventional1999-10-31Completed
A Phase I-II Study of the Treatment of Metastatic Cancer That Expresses MAGE-A3 Using Lymphodepleting Conditioning Followed by Infusion of Anti-MAGE-A3 HLA-A*01 Restricted TCR-Gene Engineered Lymphocytes and Aldesleukin [NCT02153905]Phase 1/Phase 23 participants (Actual)Interventional2014-07-03Terminated(stopped due to Slow, insufficient accrual.)
Use of G-CSF Stimulated HLA-Identical Allogeneic Peripheral Blood Stem Cells for Patients With High Risk Acute Myelogenous Leukemia or CML in Blast Crisis [NCT00002833]Phase 253 participants (Actual)Interventional1994-10-31Completed
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
Allogeneic Stem Cell Transplantation of Renal Cell Cancer and Metastatic Melanoma After Non-Myeloablative Chemotherapy [NCT00004135]Phase 219 participants (Actual)Interventional1999-02-28Completed
A Phase II Study of Alternating Cycles of Fludarabine and Cyclophosphamide in Previously Untreated Patients With B-cell CLL [NCT00003829]Phase 234 participants (Actual)Interventional1999-08-31Completed
A Multi-Center, Open Label, Randomized, Active Controlled Phase II/III Clinical Trial to Evaluate the Safety and Efficacy of Processed Unrelated Bone Marrow in Patients With Acute or Chronic Leukemia [NCT00004255]Phase 2/Phase 30 participants Interventional2000-03-31Completed
Phase I Study of Fludarabine, Carboplatin, and Topotecan for Patients With Relapsed/Refractory Acute Leukemia and Advanced Myelodysplastic Syndromes [NCT00005593]Phase 131 participants (Actual)Interventional1998-09-30Completed
A Phase I/II Study of the Treatment of Metastatic Cancer That Expresses MAGE-A3 Using Lymphodepleting Conditioning Followed by Infusion of HLA-DP0401/0402 Restricted Anti-MAGE-A3 TCR-Gene Engineered Lymphocytes and Aldesleukin [NCT02111850]Phase 1/Phase 221 participants (Actual)Interventional2014-02-07Completed
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)
Transplantation of Unrelated Donor Hematopoietic Stem Cells for the Treatment of Hematological Malignancies [NCT00281879]Phase 2200 participants (Actual)Interventional2006-02-28Terminated
Master Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, Alone or in Combination With Other Agents, in HLA-A2+ Participants With NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO) [NCT03967223]Phase 2103 participants (Actual)Interventional2019-12-31Active, not recruiting
Phase I Study of Autologous CD8+ and CD4+ Transgenic T Cells Expressing High Affinity KRASG12V Mutation-Specific T Cell Receptors in Participants With Metastatic Pancreatic, Colorectal and Non-Small Cell Lung Cancers With KRAS G12V Mutations [NCT06043713]Phase 124 participants (Anticipated)Interventional2024-01-01Recruiting
A Single-center, Open-label Clinical Study of TIL Cells for the Treatment of the Recurrent/Metastatic Solid Tumors Patients Who Had Failed Standard Therapy [NCT05649618]Early Phase 142 participants (Anticipated)Interventional2022-12-05Not yet recruiting
A Phase I/II Study of T Cell Receptor Gene Therapy Targeting HPV-16 E6 for HPV-Associated Cancers [NCT02280811]Phase 1/Phase 212 participants (Actual)Interventional2014-10-14Completed
A Single-arm, Open-label, Dose-finding Clinical Study of TCR-T Cells in Patients With HLA-A2-expressing and NY-ESO-1-positive Recurrent or Metastatic Solid Tumors [NCT05648994]Early Phase 130 participants (Anticipated)Interventional2022-12-31Not yet recruiting
Safety and Efficacy of Anti-FLT3 CAR- T Cell (TAA05 Cell Injection) in the Treatment of Relapsed/ Refractory Acute Myeloid Leukemia [NCT05445011]Phase 112 participants (Anticipated)Interventional2022-06-14Recruiting
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
Evaluation of Safety and Antitumor Activity of Lete-Cel (GSK3377794) in HLA-A2+ Participants With NY-ESO-1 Positive Previously Untreated Advanced (Metastatic or Unresectable) Synovial Sarcoma and Myxoid/Round Cell Liposarcoma [NCT05993299]Phase 27 participants (Actual)Interventional2019-12-31Active, not recruiting
Phase I Study to Evaluate the Safety and Efficacy of NK Cell Therapy in Acute Myeloid Leukemia (AML). [NCT05987696]Phase 1102 participants (Anticipated)Interventional2023-08-10Not yet recruiting
Phase I, Open-Label, Study of Tumor Infiltrating Lymphocytes Engineered With Membrane Bound IL15 Plus Acetazolamide in Adult Patients With Metastatic Melanoma [NCT05470283]Phase 130 participants (Anticipated)Interventional2022-09-07Recruiting
A Single-Arm, Open-Label, Phase 1/2 Study Evaluating the Safety, Efficacy, and Cellular Kinetics/Pharmacodynamics of ALLO-647 and ALLO-605, an Anti- BCMA Allogeneic CAR T Cell Therapy in Patients With Relapsed/Refractory Multiple Myeloma [NCT05000450]Phase 1/Phase 2136 participants (Anticipated)Interventional2021-06-06Active, not recruiting
Lymphodepletion Plus Adoptive Cell Transfer With or Without Dendritic Cell Immunization in Patients With Metastatic Melanoma [NCT00338377]Phase 21,230 participants (Actual)Interventional2006-02-01Active, not recruiting
Phase I Study to Assess Feasibility and Safety of Adoptive Transfer of Autologous Tumor-Infiltrating Lymphocytes in Combination With Interleukin-2 Followed by Nivolumab Rescue for Advanced Metastatic Melanoma [NCT03475134]Phase 118 participants (Actual)Interventional2018-02-21Active, not recruiting
A Pilot/Phase 1 Study of Immunosuppression-free Regulatory T-cell Graft-engineered Haploidentical Hematopoietic Cell Transplantation in Relapsed/Refractory and Ultra-High-risk AML/MDS [NCT04678401]Phase 120 participants (Anticipated)Interventional2021-01-12Recruiting
A Phase 2 Study to Evaluate the Efficacy and Safety of Adoptive Transfer of Autologous Tumor Infiltrating Lymphocytes in Patients With Advanced Solid Cancers [NCT03935893]Phase 2240 participants (Anticipated)Interventional2019-12-03Recruiting
CD45A-Depleted Haploidentical Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation [NCT02259348]Phase 212 participants (Actual)Interventional2014-10-31Terminated(stopped due to Investigator's decision.)
Phase I Safety Study of Descartes-11 in Patients With Relapsed/Refractory Multiple Myeloma [NCT03994705]Phase 1/Phase 225 participants (Actual)Interventional2019-08-06Active, not recruiting
A Phase 1/2, Open-label, Single Arm, Multicohort, Multicenter Trial to Evaluate the Safety and Efficacy of JCAR017 in Pediatric Subjects With Relapsed/Refractory (r/r) B-cell Acute Lymphoblastic Leukemia (B-ALL) and B-cell Non-Hodgkin Lymphoma (B-NHL). [NCT03743246]Phase 1/Phase 2121 participants (Anticipated)Interventional2018-10-17Recruiting
Phase 3 Multicenter Randomized Trial to Evaluate Efficacy and Safety of CPI-613 in Combination With HD Cyt. and Mito. vs HD Cyt. and Mito. Therapy and Control Sub-groups in Older Patients With R/R AML [NCT03504410]Phase 3200 participants (Actual)Interventional2018-11-12Terminated(stopped due to Futile)
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)
A Phase II Study of Fludarabine Induction Followed by CAMPATH-1H Consolidation in Untreated Patients With B-Cell Chronic Lymphocytic Leukemia [NCT00004857]Phase 286 participants (Actual)Interventional2000-01-31Completed
Phase II Study of Fludarabine + Idarubicin + Aracytine in Refractory or Relapsed ALL in Children [NCT00003729]Phase 213 participants (Actual)Interventional1998-12-31Terminated(stopped due to low accrual)
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
A Phase II Study of Reduced Intensity Double Umbilical Cord Blood Transplantation Using Fludarabine, Melphalan, and Low Dose Total Body Radiation [NCT01408563]Phase 233 participants (Actual)Interventional2011-12-31Completed
Phase 3 Randomized Trial of DFP-10917 vs Non-Intensive Reinduction (LoDAC, Azacitidine, Decitabine, Venetoclax Combination Regimens) or Intensive Reinduction (High & Intermediate Dose Cytarabine Regimens) for Acute Myelogenous Leukemia Patients in Second, [NCT03926624]Phase 3450 participants (Anticipated)Interventional2019-11-22Recruiting
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
Multicenter Phase II Study of Haploidentical Hematopoietic Cell Transplantation With CD3/CD19 Depleted Grafts After a Reduced Intensity Conditioning Regimen for Adult Patients With Acute Leukemia [NCT00961142]Phase 223 participants (Actual)Interventional2009-06-30Terminated(stopped due to slow recruitment)
G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With High-risk MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT05453552]Phase 2/Phase 3242 participants (Anticipated)Interventional2022-07-01Recruiting
A Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haploidentical Peripheral Blood Stem Cell Transplantation [NCT03018223]Phase 132 participants (Actual)Interventional2017-01-31Completed
G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With RAEB-1, RAEB-2 and AML Secondary to MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT04713956]Phase 2/Phase 3242 participants (Anticipated)Interventional2021-01-15Recruiting
Umbilical Cord Blood Transplant With Co-Infusion of T Regulatory Cells [NCT00376519]Phase 13 participants (Actual)Interventional2007-05-31Terminated(stopped due to Slow accrual)
Treatment Optimization in Adult Patients With Newly Diagnosed Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma by Individualised, Targeted and Intensified Treatment - a Phase IV-trial With a Phase III-part to Evaluate Safety and Efficacy of Ne [NCT02881086]Phase 31,000 participants (Actual)Interventional2016-08-31Active, not recruiting
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
Reduced Toxicity Conditioning Prior to Unrelated Cord Cell Transplantation for High Risk Myeloid Malignancies [NCT02333838]Phase 231 participants (Actual)Interventional2015-05-31Active, not recruiting
A Phase I, Open-Label, Multicenter Study of FT596 as a Monotherapy and in Combination With Rituximab or Obinutuzumab in Subjects With Relapsed/Refractory B-cell Lymphoma and Chronic Lymphocytic Leukemia [NCT04245722]Phase 198 participants (Actual)Interventional2020-03-19Terminated(stopped due to The study was terminated by the Sponsor.)
A Phase I Study of FT516 as Monotherapy in Relapsed/Refractory Acute Myelogenous Leukemia and in Combination With Monoclonal Antibodies in Relapsed/Refractory B-Cell Lymphoma [NCT04023071]Phase 172 participants (Actual)Interventional2019-10-04Terminated(stopped due to The study was terminated by the Sponsor.)
A Phase 2 Multicenter Study of Axicabtagene Ciloleucel in Subjects With Relapsed/Refractory Indolent Non-Hodgkin Lymphoma (iNHL) [NCT03105336]Phase 2159 participants (Actual)Interventional2017-06-20Active, not recruiting
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia [NCT02845596]40 participants (Anticipated)Interventional2016-08-31Active, not recruiting
A Phase 2 Multicenter Study Evaluating the Efficacy of KTE-X19 in Subjects With Relapsed/Refractory Mantle Cell Lymphoma [NCT02601313]Phase 2105 participants (Actual)Interventional2015-11-09Completed
AN OPEN-LABEL, RANDOMIZED PHASE 3 STUDY OF INOTUZUMAB OZOGAMICIN COMPARED TO A DEFINED INVESTIGATOR'S CHOICE IN ADULT PATIENTS WITH RELAPSED OR REFRACTORY CD22-POSITIVE ACUTE LYMPHOBLASTIC LEUKEMIA (ALL) [NCT01564784]Phase 3326 participants (Actual)Interventional2012-08-02Completed
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
A Phase I-II Study of Oxaliplatin, Fludarabine, and Cytarabine in Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes at First Relapse With Complete Remission Duration < 1 Year [NCT00480987]Phase 1/Phase 227 participants (Actual)Interventional2007-07-31Terminated(stopped due to Lack of support.)
Phase I Study to Evaluate the Safety and Effectiveness of Dual CD33-CLL1 CAR-T Therapy in Relapsed/Refractory Acute Myeloid Leukemia [NCT05016063]Early Phase 132 participants (Anticipated)Interventional2021-09-01Not yet recruiting
A Phase I Open, Single-arm Study of JWCAR029 (CD19-targeted Chimeric Antigen Receptor T Cells) for Patients With Relapsed or Refractory in B-cell Acute Lymphoblastic Leukemia [NCT05727683]Phase 133 participants (Anticipated)Interventional2022-04-28Recruiting
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
Cladribine Dose Escalation in Conditioning Regimen Prior to Allo-HSCT for Refractory Acute Leukemia and Myelodysplastic Syndromes [NCT03235973]Phase 129 participants (Anticipated)Interventional2018-04-28Recruiting
An Open-label, Multi-center, Single-arm Phase 1/2 Study to Assess Tolerability, Safety and Efficacy of CRC01 in Adult Patients With Relapsed or Refractory Large B-cell Lymphoma [NCT04836507]Phase 1/Phase 291 participants (Anticipated)Interventional2021-03-02Recruiting
Fludarabine Added to Induction Treatment in Untreated Multiple Myeloma Patients: A Randomised, Placebo Controlled, Double Blind Phase II Trial: NMSG #13/03 [NCT00382694]Phase 280 participants Interventional2005-05-31Active, not recruiting
A Phase 1/2 Open-label, Multicenter Study of Lenzilumab and Axicabtagene Ciloleucel in Subjects With Relapsed or Refractory Large B-cell Lymphoma (ZUMA-19) [NCT04314843]Phase 16 participants (Actual)Interventional2020-05-26Terminated(stopped due to Development program terminated.)
A Randomized,Open,Multicenter and Prospective Study of the Optimized Dose of Anti-Thymoglobuline in Haploidentical Allogeneic Stem Cell Transplantation [NCT03190733]Phase 4192 participants (Anticipated)Interventional2017-08-30Not yet recruiting
Pilot Study of Infusion of Fucosylated Regulatory T Cells to Prevent Graft Versus Host Disease [NCT02423915]Phase 15 participants (Actual)Interventional2015-07-30Completed
Phase I/II Study Evaluating the Infusion of Tumor-Infiltrating Lymphocytes (TILs) & Low-Dose Interleukin-2 (IL-2) Therapy Following a Preparative Regimen of Non-myeloablative Lymphodepletion Using Cyclophosphamide & Fludarabine in Patients With Malignant [NCT02414945]Phase 1/Phase 29 participants (Actual)Interventional2015-05-15Completed
An Open, Single-arm Clinical Study of Autologous T Cells (CAR-T) Targeting B7-H3 Chimeric Antigen Receptor Gene in the Treatment of Patients With Advanced Gastrointestinal Tumors [NCT05515185]Early Phase 130 participants (Anticipated)Interventional2022-09-09Not yet recruiting
Safety and Efficacy Evaluation of IM19 CAR-T Cells On CD19+ Refractory or Relapsed B-ALL Patients [NCT03173417]Phase 1/Phase 2177 participants (Actual)Interventional2017-05-23Completed
Phase I-II Study of Crenolanib Combined With Standard Salvage Chemotherapy, and Crenolanib Combined With 5-Azacitidine in Acute Myeloid Leukemia Patients With FLT3 Activating Mutations [NCT02400281]Phase 1/Phase 228 participants (Actual)Interventional2015-09-30Completed
A Phase 2, Single-Center, Open Label Study of Autologous, Adoptive Cell Therapy Following a Reduced Intensity, Non-myeloablative, Lymphodepleting Induction Regimen in Metastatic Melanoma Patients [NCT03166397]Phase 230 participants (Anticipated)Interventional2017-06-05Recruiting
A Multicenter, Single-Arm, Phase II Study to Evaluate the Efficacy and Safety of Rituximab Plus Fludarabine and Cyclophosphamide (FCR) as First-Line Treatment in Patients With B-Cell Chronic Lymphocytic Leukemia (CLL) [NCT02533401]Phase 234 participants (Actual)Interventional2006-02-28Completed
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
Epigenetic Reprogramming in Relapse AML: A Phase 1 Study of Decitabine and Vorinostat Followed by Fludarabine, Cytarabine and G-CSF (FLAG) in Children and Young Adults With Relapsed/Refractory AML [NCT02412475]Phase 13 participants (Actual)Interventional2015-02-21Terminated(stopped due to We opened a competing study with the TACL consortium)
Venetoclax Combining With Fludarabine and Melphalan as Conditioning Regimen Prior to Allogeneic Hematopoietic Stem Cell Transplantation for Older Patients With Hematologic Malignancies [NCT05084027]Phase 250 participants (Anticipated)Interventional2021-10-07Recruiting
T Cells Expressing a Fully-Human Anti-CD19 Chimeric Antigen Receptor for Treating B-cell Malignancies [NCT02659943]Phase 127 participants (Actual)Interventional2016-01-21Completed
A Prospective Randomized and Phase II Trial for Metastatic Melanoma Using Adoptive Cell Therapy With Tumor-Infiltrating Lymphocytes Plus IL-2 Either Alone or Following the Administration of Pembrolizumab [NCT02621021]Phase 220 participants (Actual)Interventional2015-12-04Suspended(stopped due to The team is revising the protocol design and has chosen to put the study on hold until those changes are approved.)
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)
A Prospective Phase II Clinical Study to Assess the Efficacy and Toxicity of High-dose Chemotherapy Followed by Allogeneic Stem Cell Transplantation as Treatment of Primary Progressive and Relapsed Aggressive Non-Hodgkin Lymphoma [NCT04121507]Phase 260 participants (Actual)Interventional2019-06-24Completed
Phase I/II Study of CD19 CART Cells for Patients With Relapse and Refractory CD19+ B-cell Lymphoma. [NCT03146533]Phase 1/Phase 220 participants (Anticipated)Interventional2017-05-31Recruiting
FLUCLORIC: Randomized Multicentric Phase III Study Comparing the Efficacy of 2 Reduced Intensity Conditioning Regimens (Clofarabine/Busulfan vs Fludarabine/Busulfan) in Adults With AML and Eligible to Allogeneic Stem Cell Transplantation [NCT05917405]Phase 2302 participants (Anticipated)Interventional2023-09-14Recruiting
A Randomized Phase II Trial of Consolidation Therapy Following CD19 CAR T-Cell Treatment for Relapsed/Refractory Diffuse Large B-Cell Lymphoma or Grade IIIB Follicular Lymphoma [NCT05633615]Phase 2396 participants (Anticipated)Interventional2023-06-12Recruiting
Phase 1 Study of Lintivirally Transduced T Cells Engineered to Contain Anti-CD38 Linked to TCRζ and 4-1BB Signaling Domains in Subjects With Acute Myeloid Leukemia and Multiple Myeloma [NCT05442580]Phase 136 participants (Anticipated)Interventional2023-05-30Recruiting
A Phase 1/2, Open Label, Multicenter Trial to Assess the Safety and Efficacy of MB-102 in Patients With Relapsed or Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm [NCT04109482]Phase 1/Phase 23 participants (Actual)Interventional2020-02-17Terminated(stopped due to Business reasons.)
A Phase 1 Study Evaluating the Safety and Efficacy of MAGE-A3/A6 T Cell Receptor Engineered T Cells (KITE-718) in HLA-DPB1*04:01 Positive Subjects With Advanced Cancers [NCT03139370]Phase 116 participants (Actual)Interventional2017-12-27Terminated(stopped due to The study was terminated due to the sponsor's decision to discontinue the clinical trial.)
Phase I Study of CART-PSMA-TGFβRDN Cells in Patients With Advanced Castrate Resistant Prostate Cancer [NCT03089203]Phase 119 participants (Anticipated)Interventional2017-03-08Recruiting
Treatment of Patients With Relapsed or Refractory CD19+ Lymphoid Disease With T Lymphocytes Transduced by RV-SFG.CD19.CD28.4-1BBzeta Retroviral Vector - a Unicenter Phase I/II Clinical Trial [NCT03676504]Phase 1/Phase 268 participants (Anticipated)Interventional2018-09-07Recruiting
A Phase II Trial Of Autologous Stem Cell Transplant Followed By Mini-Allogeneic Stem Cell Transplant In Lieu Of Standard Allogeneic Bone Marrow Transplantation For Treatment Of Multiple Myeloma [NCT00014508]Phase 20 participants Interventional2001-11-19Completed
Phase I Study of Escalating Doses of Total Marrow and Lymphoid Irradiation (TMLI) Combined With Fludarabine and Melphalan as Conditioning for Allogeneic Hematopoietic Cell Transplantation in Patients With High-Risk Acute Leukemia or Myelodysplastic Syndro [NCT03494569]Phase 136 participants (Anticipated)Interventional2018-07-06Recruiting
Phase I Study of Autologous Tumor-Draining Lymph Node-Derived Lymphocytes as Neoadjuvant Therapy for HER2-Negative Breast Cancer [NCT06121570]Phase 124 participants (Anticipated)Interventional2023-11-01Recruiting
A Phase III Intergroup CLL Study of Asymptomatic Patients With Untreated Chronic Lymphocytic Leukemia Randomized to Early Intervention Versus Observation With Later Treatment in the High Risk Genetic Subset With IGVH Unmutated Disease [NCT00513747]Phase 384 participants (Actual)Interventional2008-01-31Terminated(stopped due to Insufficient accrual)
A Phase II Study of Cyclophosphamide, Fludarabine, Alemtuzumab, and Rituximab (CFAR) in High-Risk Previously Untreated Patients With CLL [NCT00525603]Phase 260 participants (Actual)Interventional2005-06-30Completed
A Phase II Trial to Assess the Activity of NY-ESO-1 Targeted T Cells in Advanced Oesophagogastric Cancer [NCT01795976]Phase 22 participants (Actual)Interventional2014-10-31Terminated(stopped due to Withdrawal of funding)
Phase II Study of Metastatic Melanoma Using Lymphodepleting Conditioning Followed by Infusion of Anti-gp100:154-162 TCR-Gene Engineered Lymphocytes and ALVAC Virus Immunization [NCT00610311]Phase 23 participants (Actual)Interventional2008-01-31Terminated(stopped due to The study was terminated due to low accrual.)
A Phase II Study of Sirolimus, Tacrolimus and Thymoglobulin, as Graft-versus-Host Prophylaxis in Patients Undergoing Unrelated Donor Hematopoietic Cell Transplantation [NCT00589563]Phase 232 participants (Actual)Interventional2007-05-31Completed
A Comparison of Reduced Dose Total Body Irradiation (TBI) and Cyclophosphamide With Fludarabine and Melphalan Reduced Intensity Conditioning in Adults With Acute Lymphoblastic Leukaemia (ALL) in Complete Remission. (ALL-RIC) [NCT03821610]Phase 2242 participants (Anticipated)Interventional2018-11-22Active, not recruiting
A Phase I Study of Administrating CD19 Chimeric Antigen Receptor Expressing T Cells Followed by Allogeneic Stem Cell Transplantation in Patients With Refractory CD19+ B-linage Leukemia/Lymphoma [NCT03110640]Phase 120 participants (Anticipated)Interventional2016-10-01Recruiting
Randomized Study of Different Non-myeloablative Conditioning Regimens With Hematopoietic Stem Cell Support in Patients With Scleroderma (Autologous Systemic Sclerosis Immune Suppression Trial - II ASSIST-IIb) [NCT01445821]Phase 344 participants (Actual)Interventional2011-09-15Terminated(stopped due to Developed a better regimen: DIAD. Cast. 2018 NCT03593902)
Phase III Trial of Combined Immunochemotherapy With Fludarabine, Cyclophosphamide and Rituximab (FCR) Versus Chemotherapy With Fludarabine and Cyclophosphamide (FC) Alone in Patients With Previously Untreated Chronic Lymphocytic Leukaemia [NCT00281918]Phase 3817 participants (Actual)Interventional2003-07-31Completed
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
T-Cell Depleted Double UCB With Post Transplant IL-2 for Refractory Myeloid Leukemia [NCT01464359]Phase 23 participants (Actual)Interventional2011-10-31Terminated(stopped due to Slow accrual)
Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies [NCT01499147]100 participants (Actual)Interventional2000-02-29Completed
Bortezomib Combined With Fludarabine and Cytarabine for Mantle Cell Lymphoma Patients:a Single Arm, Open-labelled, Phase 2 Study [NCT03016988]Phase 250 participants (Anticipated)Interventional2017-01-31Not yet recruiting
MT2008-15: Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Nonmyeloablative Preparative Regimen and Priming With Complement 3a Fragment (C3a) [NCT00963872]Phase 1/Phase 231 participants (Actual)Interventional2010-03-31Terminated(stopped due to Lack of efficacy after interim analysis)
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
A Study of Umbilical Cord Blood-Derived Natural Killer Cells in Conjunction With Lymphodepleting Chemotherapy and Lenalidomide as Immunotherapy in Patients With Hematologic Malignancies [NCT02280525]Phase 18 participants (Actual)Interventional2015-03-05Completed
A Phase 1/2 Trial Evaluating Treatment of Emergent Graft Versus Host Disease (GvHD) With AP1903 After Planned Donor Infusions (DLIs) of T-cells Genetically Modified With the iCasp9 Suicide Gene in Patients With Hematologic Malignancies [NCT01875237]Phase 1/Phase 23 participants (Actual)Interventional2013-12-27Terminated
Phase I Study of Escalating Doses of 90Y-DOTA-Anti-CD25 Monoclonal Antibody Added to the Conditioning Regimen of Fludarabine, Melphalan, and Organ Sparing Total Marrow and Lymphoid Irradiation (TMLI) as Conditioning for Allogeneic Hematopoietic Cell Trans [NCT05139004]Phase 112 participants (Anticipated)Interventional2022-07-19Recruiting
A Phase I Dose Escalation Trial of Autologous Third-generation Anti-CD19 Chimeric Antigen Receptor T-cells (WZTL-002) for Relapsed and Refractory B-cell Lymphomas [NCT04049513]Phase 130 participants (Anticipated)Interventional2019-10-11Recruiting
A Two-Stage Phase 1 Open-Label Study of huJCAR014, CD19-Targeted Chimeric Antigen Receptor (CAR)-Modified T Cells Bearing a Human Binding Domain, in Adult Patients With Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma and Acute Lymphocytic Leukemia [NCT03103971]Phase 155 participants (Actual)Interventional2017-11-03Active, not recruiting
Phase 2 Study of Planned Donor Lymphocyte Infusion After Reduced Intensity Allogeneic Stem Cell Transplantation [NCT01518153]Phase 216 participants (Actual)Interventional2012-02-29Terminated(stopped due to Objectives not met.)
A Multicenter, Open-Label, Single-Arm, Phase IIIb, International Study Evaluating the Safety of Obinutuzumab Alone or in Combination With Chemotherapy in Patients With Previously Untreated or Relapsed/Refractory Chronic Lymphocytic Leukemia [NCT01905943]Phase 3979 participants (Actual)Interventional2013-11-04Completed
Clinical Study on QN-023a Targeting CD33 in Relapsed/Refractory Acute Myeloid Leukemia [NCT05601466]Phase 118 participants (Anticipated)Interventional2022-10-28Recruiting
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 Study of Thymic Shielding in Recipients of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Stem Cell Transplantation in Patients With Fanconi Anemia [NCT00167206]Phase 1/Phase 216 participants (Actual)Interventional2004-03-31Terminated(stopped due to Treatment with thymic shielding found safe, another study started.)
Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders [NCT00176878]Phase 2/Phase 310 participants (Actual)Interventional2000-06-30Completed
Autologous Versus Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Chemosensitive Follicular Non-Hodgkin's Lymphoma Beyond First Complete Response or First Partial Response (BMT CTN #0202) [NCT00096460]Phase 2/Phase 330 participants (Actual)Interventional2004-08-31Terminated(stopped due to lower than anticipated accrual)
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
A Phase 1 Open-Label, Multi-Center First in Human Study of TnMUC1-Targeted Genetically-Modified Chimeric Antigen Receptor T Cells in Patients With Advanced TnMUC1-Positive Solid Tumors and Multiple Myeloma [NCT04025216]Phase 116 participants (Actual)Interventional2019-10-10Terminated(stopped due to The sponsor finds the risk/benefit analysis unfavorable and has terminated the study.)
A Phase II Study Evaluating the Safety and Efficacy of the Combination of Zanubrutinib Plus Lisocabtagene Maraleucel for Richter's Syndrome [NCT05873712]Phase 224 participants (Anticipated)Interventional2023-07-28Recruiting
A Phase 1b Clinical Trial of Anti-BCMA Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Multiple Myeloma [NCT05577000]Phase 124 participants (Anticipated)Interventional2021-10-18Recruiting
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
A Phase I Study of Autologous Activated T-Cells Expressing a 2nd Generation GD2 Chimeric Antigen Receptor, IL-15, and iCaspase9 Safety Switch Administered To Patients With Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma [NCT03721068]Phase 118 participants (Anticipated)Interventional2019-02-19Recruiting
Randomized Phase III Study Comparing a Non-myeloablative Lymphocyte Depleting Regimen of Chemotherapy Followed by Infusion of Tumor Infiltrating Lymphocytes and Interleukin-2 to Standard Ipilimumab Treatment in Metastatic Melanoma [NCT02278887]Phase 3168 participants (Actual)Interventional2014-09-23Active, not recruiting
CD123-Directed Autologous T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML) [NCT04318678]Phase 132 participants (Anticipated)Interventional2020-07-29Active, not recruiting
A Phase II Study of Selective Depletion of CD45RA+ T Cells From Allogeneic Peripheral Blood Stem Cell Grafts for the Prevention of GVHD in Children [NCT01858740]Phase 220 participants (Actual)Interventional2013-12-17Completed
A Phase I Study of Adoptive Transfer of Autologous Tumour Antigen-Specific T Cells With Pre-conditioning Chemotherapy and Intravenous IL2 in Patients With CD19 Positive Malignancy [NCT01493453]Phase 117 participants (Actual)Interventional2008-03-31Terminated(stopped due to Investigation into serious breach)
Nonmyeloablative Allogeneic Stem Cell Transplantation For Relapsed Hodgkin's or Non-Hodgkin's Lymphoma After Autologous Transplantation ( A BMT Study) [NCT00121186]Phase 21 participants (Actual)Interventional2005-07-31Terminated(stopped due to poor accrual)
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)
Phase 2 Study of the Activity and Safety of Fludarabine, Cyclophosphamide, and Mitoxantrone Plus Rituximab (FCM-R) With Pegfilgrastim (Neulasta) as Frontline Therapy for Patients < 70 Years With Chronic Lymphocytic Leukemia [NCT00254410]Phase 230 participants (Actual)Interventional2005-03-14Completed
Fludarabine, Cyclophosphamide, and Rituximab (FCR) Plus Sargramostim (GM-CSF) as Frontline Therapy for Symptomatic Chronic Lymphocytic Leukemia [NCT00381004]Phase 260 participants (Actual)Interventional2006-09-30Completed
A Phase I-II Study of Oxaliplatin, Fludarabine, Cytarabine and Rituximab in Patients With Richter's Transformation, Prolymphocytic Leukemia or Refractory/Relapsed B-Cell Chronic Lymphocytic Leukemia [NCT00452374]Phase 1/Phase 248 participants (Actual)Interventional2004-11-30Completed
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
Lymphodepleting Chemotherapy With Fludarabine and Cyclophosphamide Prior to Infusion of CAR T Cell Therapy in Patients With Moderate-Severe Renal Dysfunction [NCT05909059]Phase 220 participants (Anticipated)Interventional2023-12-31Not yet recruiting
Open-label, Phase 1 Study of CD19 t-haNK as a Single Agent and in Combination With an IL-15 Superagonist (N-803) and Rituximab in Subjects With Selected CD19+ and CD20+ Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma. [NCT05618925]Phase 120 participants (Anticipated)Interventional2023-11-01Not yet recruiting
A Phase II Randomized Study to Assess Outcomes With Treosulfan-Based Versus Clofarabine-Based Conditioning in Patients With Myelodysplastic Syndromes With Excess Blasts (MDS-EB), or Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Tra [NCT04994808]Phase 280 participants (Anticipated)Interventional2023-08-11Recruiting
Phase II Trial of Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in High Risk AML Patients Who Are Not Eligible for Allogeneic Stem Cell Transplantation [NCT02771197]Phase 220 participants (Actual)Interventional2016-09-28Completed
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
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
"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
Phase II Study of Metastatic Cancer That Overexpresses p53 Using Lymphodepleting Conditioning Followed by Infusion of Anti-P53 TCR-Gene Engineered Lymphocytes and Dendritic Cell Vaccination [NCT00704938]Phase 23 participants (Actual)Interventional2008-06-30Terminated(stopped due to Terminated due to withdrawal of support from our collaborator.)
A Phase I Study of Autologous Activated T-cells Targeting the CD19 Antigen and Containing the Inducible Caspase 9 Safety Switch in Subjects With Relapsed/Refractory B-cell Lymphoma [NCT03696784]Phase 130 participants (Anticipated)Interventional2019-03-12Recruiting
Phase I/Ib Clinical Trial of Autologous CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults With Recurrent or Refractory B Cell Malignancies [NCT04088890]Phase 192 participants (Anticipated)Interventional2019-09-12Recruiting
ADMINISTRATION OF MOST CLOSELY MATCHED THIRD PARTY RAPIDLY GENERATED LMP, BARF1 and EBNA1 SPECIFIC CYTOTOXIC T-LYMPHOCYTES TO PATIENTS WITH EBV-POSITIVE LYMPHOMA AND OTHER EBV-POSITIVE MALIGNANCIES [NCT02287311]Phase 142 participants (Anticipated)Interventional2015-02-28Recruiting
Anti-CD30 CAR T Cells With Fully-human Binding Domains for Treating CD30-expressing Lymphomas Including Anaplastic Large Cell Lymphomas [NCT03049449]Phase 126 participants (Actual)Interventional2017-03-17Completed
A Phase II Study for Metastatic Melanoma Using High-Dose Chemotherapy Preparative Regimen Followed by Cell Transfer Therapy Using Tumor-Infiltrating Lymphocytes Plus IL-2 With the Administration of Pembrolizumab in the Retreatment Arm [NCT01993719]Phase 233 participants (Actual)Interventional2013-12-12Completed
Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia [NCT00003816]Phase 2/Phase 3361 participants (Actual)Interventional1998-10-19Completed
A Clinical Study of Low-dose Total Body Irradiation and Fludarabine/Busulfan/Melphalan as a Conditioning Regimen for Secondary Umbilical Cord Blood Transplantation in Patients With Hematological Malignancies Who Relapsed After Allo-HSCT [NCT06125483]38 participants (Anticipated)Interventional2023-11-01Recruiting
A Phase I Trial Evaluating Escalating Doses of ²¹¹At-Labeled Anti-CD38 Monoclonal Antibody Followed by HLA-Matched or Haploidentical Donor Hematopoietic Cell Transplantation for High-Risk Multiple Myeloma [NCT04579523]Phase 130 participants (Anticipated)Interventional2024-02-01Not yet recruiting
International Collaborative Treatment Protocol For Children And Adolescents With Acute Lymphoblastic Leukemia [NCT01117441]Phase 36,136 participants (Actual)Interventional2010-06-30Completed
Haploidentical Donor Hematopoietic Stem Cell Transplantation [NCT02660281]Phase 174 participants (Actual)Interventional2015-10-31Completed
A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Leukemia or Lymphoma [NCT05565105]Phase 2100 participants (Anticipated)Interventional2024-03-31Not yet recruiting
The Safety and Clinical Efficacy of Human CD19/CD22 Bispecific Chimeric Antigen Receptor (CAR)-T Cell Therapy for Subjects With Measurable Residual Disease(MRD)-Positive B Cell Acute Lymphoblastic Leukemia [NCT03919526]Phase 119 participants (Actual)Interventional2019-08-11Completed
Safety and Efficacy of Cord Blood Derived Anti-CD19 CAR-Engineered NK Cells for Relapsed/Refractory B Lymphoid Malignancies: a Single-center, Open-label, Single-arm Clinical Study [NCT04796675]Phase 127 participants (Anticipated)Interventional2021-04-10Recruiting
Cellular Adoptive Immunotherapy Using Autologous Tumor-Infiltrating Lymphocytes Following Lymphodepletion With Cyclophosphamide and Fludarabine for Patients With Metastatic Melanoma [NCT01807182]Phase 211 participants (Actual)Interventional2013-08-20Completed
Allogeneic Peripheral Blood Stem Cell Transplantation Using a Non-Myeloablative Preparative Regimen for Patients With Hematologic Malignancies [NCT00004145]Phase 216 participants (Actual)Interventional1998-11-30Completed
A Phase I Study of Anti-CD19 CAR T-cell Therapy With Axicabtagene Ciloleucel (Axi-cel) in Patients With Relapsed/Refractory Primary and Secondary Central Nervous System (CNS) Lymphoma [NCT04608487]Phase 118 participants (Actual)Interventional2020-12-04Active, not recruiting
Pilot Study of JAK Inhibitor Therapy Followed by Reduced Intensity Haploidentical Transplantation for Patients With Myelofibrosis [NCT04370301]Phase 210 participants (Anticipated)Interventional2021-02-09Recruiting
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) [NCT03597594]Phase 1/Phase 24 participants (Actual)Interventional2021-09-02Active, not recruiting
A Pilot Study of CD19-Specific Car T Cells as Consolidation for Older Adults With Acute Lymphoblastic Leukemia in First Remission [NCT05707273]Phase 121 participants (Anticipated)Interventional2023-04-26Recruiting
Phase 2 Study of the Combination of Lisocabtagene Maraleucel, Nivolumab, and Ibrutinib in Richter's Transformation [NCT05672173]Phase 220 participants (Anticipated)Interventional2023-06-02Recruiting
PRO#1278: A Phase III Study of Fludarabine and Busulfan Versus Fludarabine, Busulfan and Low Dose Total Body Irradiation in Patients Receiving an Allogeneic Hematopoietic Stem Cell Transplant [NCT01366612]Phase 353 participants (Actual)Interventional2010-06-16Terminated(stopped due to Lack of Accrual)
A Pilot Study of the Administration of Young Tumor Infiltrating Lymphocytes Following a Non-Myeloablative Lymphocyte Depleting Chemotherapy Regimen in Metastatic Melanoma [NCT01468818]Phase 218 participants (Actual)Interventional2011-09-30Terminated(stopped due to Study was closed prematurely due to slow and insufficient accrual.)
A Prospective Multicenter Observational Study to Assess Long-term Outcome of Participants Who Have Received oNKord® [NCT05290662]50 participants (Anticipated)Observational [Patient Registry]2022-06-14Recruiting
Phase II Pilot Trial to Evaluate the Efficacy of a Combined Therapy Approach for Young CLL Patients With Advanced and Progressive Disease Stratified According to the Biological Prognostic Features [NCT00462332]Phase 286 participants (Actual)Interventional2007-05-31Completed
Phase II Study of Metastatic Melanoma Using Lymphodepleting Conditioning Followed by Infusion of Anti-MART-1 F5 TCR-Gene Engineered Lymphocytes [NCT00509288]Phase 224 participants (Actual)Interventional2007-06-30Completed
Busulfan, Fludarabine, Donor Stem Cell Transplant, and Cyclophosphamide in Treating Participants With Multiple Myeloma or Myelofibrosis [NCT03303950]Phase 26 participants (Actual)Interventional2018-03-30Terminated(stopped due to Slow accrual)
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 Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation [NCT00744692]Phase 122 participants (Actual)Interventional2008-10-31Completed
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.)
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.)
Trial of Allogeneic BMT for Hematologic Malignancies Using HLA-matched Related or Unrelated Donors With Fludarabine and IV Busulfan as Pre-transplant Conditioning Followed by Post-transplant Immunosuppression With High-dose Cyclophosphamide [NCT00809276]Phase 1/Phase 292 participants (Actual)Interventional2009-05-31Completed
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
Natural Killer (NK) Cells and Nonmyeloablative Stem Cell Transplantation for Chronic Myelogenous Leukemia (CML) [NCT01390402]Phase 26 participants (Actual)Interventional2012-01-31Completed
A Phase 1, Multicenter, Open-label Study of BMS-986403 in Subjects With Relapsed and/or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) [NCT05244070]Phase 15 participants (Actual)Interventional2022-09-14Terminated(stopped due to Business objectives have changed.)
Phase I/II Study of Metastatic Cancer Using Lymphodepleting Conditioning Followed by Infusion of Anti-mesothelin Gene Engineered Lymphocytes [NCT01583686]Phase 1/Phase 215 participants (Actual)Interventional2012-05-04Terminated(stopped due to Study terminated due to slow/insufficient accrual.)
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)
Itacitinib to Prevent Graft Versus Host Disease [NCT04127721]Phase 20 participants (Actual)Interventional2020-09-22Withdrawn(stopped due to 0 ACTUAL Enrollment must have Overall Recruitment Status)
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation for Multiple Myeloma Using a Conditioning Regimen of Fludarabine, Melphalan, and Bortezomib [NCT01453101]Phase 254 participants (Actual)Interventional2010-06-09Completed
Single Arm Phase II Study of Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Acute Lymphoblastic Leukemia (ALL) in Older Patients Using Fludarabine and Total Body Irradiation (FluTBI) Regimen [NCT01991457]Phase 219 participants (Actual)Interventional2013-08-27Completed
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.)
Phase 1 Study of Lentivirally Transduced T Cells Engineered to Contain Anti-CD123 Linked to TCRζ and 4-1BB Signaling Domains in Pediatric Subjects With Refractory or Relapsed Acute Myeloid Leukemia [NCT04678336]Phase 112 participants (Anticipated)Interventional2021-02-15Active, not recruiting
A Study of CD19 CAR-T Cells for Patients With Relapse and Refractory CD19+ B-cell Acute Lymphoblastic Leukemia [NCT03263208]Phase 1/Phase 220 participants (Anticipated)Interventional2017-08-16Recruiting
An Exploratory Clinical Study Evaluating the Safety and Efficacy of Anti-HER2-CAR-T Cells Injection in Patients With HER2+ Locally Advanced and/ or Metastatic Solid Tumors [NCT06101082]Phase 19 participants (Anticipated)Interventional2023-10-25Not yet recruiting
A Phase 1 Study of FT522 in Combination With Rituximab in Participants With Relapsed/Refractory B-Cell Lymphoma [NCT05950334]Phase 1322 participants (Anticipated)Interventional2023-11-01Recruiting
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)
A Phase 1, Multicenter, Open-Label Study of CC-95266 in Subjects With Relapsed and/or Refractory Multiple Myeloma [NCT04674813]Phase 1180 participants (Anticipated)Interventional2021-02-24Recruiting
Phase 1 Dose Escalation Study of Systemically Administered IL13Ra2 Chimeric Antigen Receptor (CAR) T Cells After a Nonmyeloablative Conditioning Regimen in Patients With Metastatic Melanoma [NCT04119024]Phase 124 participants (Anticipated)Interventional2019-11-27Recruiting
Adoptive Transfer of NY-ESO-1 TCR Engineered Peripheral Blood Mononuclear Cells (PBMC) and Peripheral Blood Stem Cells (PBSC) After a Myeloablative Conditioning Regimen, With Administration of Interleukin-2, in Patients With Advanced Malignancies [NCT03240861]Phase 15 participants (Actual)Interventional2017-07-26Terminated(stopped due to slow accrual)
Phase III Randomized, Double-blind, Placebo-controlled Study Investigating the Efficacy of the Addition of Crenolanib to Salvage Chemotherapy Versus Salvage Chemotherapy Alone in Subjects ≤ 75 Years of Age With Relapsed/Refractory FLT3 Mutated Acute Myelo [NCT03250338]Phase 3322 participants (Anticipated)Interventional2018-06-05Recruiting
Phase II Study in Patients With Metastatic Ocular Melanoma Using a Non-Myeloablative Lymphocyte Depleting Regimen of Chemotherapy Followed by Infusion of Autologous Tumor-Infiltrating Lymphocytes With or Without High Dose Aldesleukin [NCT01814046]Phase 224 participants (Actual)Interventional2013-03-01Terminated(stopped due to Investigator left the National Institutes of Health (NIH))
Pilot Study: Infusion of Off-the-Shelf Ex Vivo Expanded Cryopreserved Progenitor Cells (Dilanubicel) in the Setting of Single Cord Blood Transplantation for Patients With Hematologic Malignancies [NCT03399773]Phase 215 participants (Anticipated)Interventional2022-05-10Recruiting
Treatment of Graft Failure After Hematopoietic Stem Cell Transplantation [NCT02161783]50 participants (Anticipated)Observational2014-10-06Recruiting
A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT01071239]Phase 21 participants (Actual)Interventional2009-04-30Completed
Immunotherapy With CD19/22 CAR Redirected T-cells for High Risk, Relapsed Paediatric CD19+ and/or CD22+ Acute Lymphoblastic Leukaemia and Other Haematological Malignancies [NCT02443831]Phase 133 participants (Anticipated)Interventional2016-04-30Active, not recruiting
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
Assessment of Safety and Recommended Phase 2 Dose of Autologous T Cells Engineered With an Affinity-enhanced TCR Targeting NY ESO 1 and LAGE 1a, and Co-expressing the dnTGF-βRII (GSK3845097) in Participants With NY ESO 1 and/or LAGE 1a Positive Previously [NCT05943990]Phase 15 participants (Actual)Interventional2020-12-21Terminated(stopped due to The study was terminated due to a change in GSK's R&D priorities.)
Compassionate Use Re-Infusion of ATLCAR.CD30 [NCT03914885]0 participants Expanded AccessNo longer available
Reduced Intensity Conditioning Regimen for Elderly or High Comorbidity Burden Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation [NCT03412409]Phase 250 participants (Anticipated)Interventional2018-02-01Recruiting
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)
Clinical Study of Redirected Autologous T Cells With a BCMA-targeted Chimeric Antigen Receptor in Patients With Refractory or Relapsed Multiple Myeloma [NCT03380039]6 participants (Actual)Interventional2017-10-13Active, not recruiting
Phase I Study of Autologous Tumor-Draining Lymph Node-Derived Lymphocytes for Advanced HER2-Negative Breast Cancer [NCT06121557]Phase 124 participants (Anticipated)Interventional2023-11-01Recruiting
Phase Ib Clinical Trial of Autologous Anti-NY-ESO-1 TCR-Engineered T Cells in Patients With Relapsed/Refractory Locally Advanced or Metastatic NY-ESO-1-Expressing Triple Negative Breast Cancer [NCT05989828]Phase 120 participants (Anticipated)Interventional2023-11-27Not yet recruiting
A Phase II Trial of Reduced Intensity Fludarabine and Total Body Irradiation-Based Conditioning Prior to Haplo-Identical Transplantation for Patients With Hematologic Malignancies [NCT05417971]20 participants (Anticipated)Observational2022-08-29Recruiting
"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
Phase I Dose Escalation Trial of CD19 Directed Chimeric Antigen Receptor T Cell Therapy in the Treatment of Relapsed/Refractory B Cell Malignancies [NCT04892277]Phase 125 participants (Anticipated)Interventional2022-07-14Recruiting
Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning for Transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT) For Patients With Hematologic Malignancies [NCT04191187]Phase 237 participants (Anticipated)Interventional2019-12-06Active, not recruiting
Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: A Sickle Transplant Advocacy and Research Alliance (STAR) Trial [NCT04018937]Phase 258 participants (Anticipated)Interventional2019-03-22Recruiting
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 Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor [NCT02828592]Phase 220 participants (Anticipated)Interventional2016-09-09Recruiting
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)
A Phase 3, Open-Label, Multicenter, Randomized Study of SKLB1028 Versus Salvage Chemotherapy in Patients With FLT3-mutated Acute Myeloid Leukemia Refractory to or Relapsed After First-line Treatment(ALIVE) [NCT04716114]Phase 3315 participants (Anticipated)Interventional2021-03-24Recruiting
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
Phase Ib Trial of Pembrolizumab Administered in Combination With or Following Adoptive Cell Therapy- A Multiple Cohort Study; The ACTIVATE (Adoptive Cell Therapy InVigorated to Augment Tumor Eradication) Trial [NCT03158935]Phase 18 participants (Actual)Interventional2017-07-07Completed
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
Phase I Study to Evaluate the Safety and Effectiveness of Anti-BCMA CAR-NK Therapy in Relapsed or Refractory Multiple Myeloma [NCT05008536]Early Phase 127 participants (Anticipated)Interventional2021-10-01Recruiting
A Phase I/II Study of the Safety and Feasibility of Administering T Cells Expressing Anti-EGFRvIII Chimeric Antigen Receptor to Patients With Malignant Gliomas Expressing EGFRvIII [NCT01454596]Phase 1/Phase 218 participants (Actual)Interventional2012-05-16Completed
A Phase I Clinical Trial of T-Cells Targeting B-Cell Maturation Antigen for Previously Treated Multiple Myeloma [NCT02215967]Phase 130 participants (Actual)Interventional2014-08-12Completed
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
CHAIROS - Effect of Early Brief Intensification by Chemoimmunotherapy With FCR Followed by FR and Rituximab Maintenance on Clinical Response in Chemo-naïve Patients With B-CLL [NCT02013817]Phase 243 participants (Actual)Interventional2005-10-11Completed
A Phase 2, Open-label, Single Arm, Multicohort, Multicenter Trial to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects With Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma (NHL) [NCT04245839]Phase 2213 participants (Anticipated)Interventional2020-07-14Active, not recruiting
Phase I Study to Evaluate Cellular Immunotherapy Using Memory-Enriched T Cells Lentivirally Transduced to Express a CS1-Targeting, Hinge-Optimized, 41BB-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR Following Lymphodepleting Chemotherapy in [NCT03710421]Phase 130 participants (Anticipated)Interventional2019-04-23Recruiting
Allogeneic Hematopoietic Stem Cell Transplantation of α/β T-Lymphocyte Depleted Graft Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia [NCT03531736]Phase 117 participants (Actual)Interventional2018-05-09Active, not recruiting
A Phase 1b/2 Study of the BCL-2 Inhibitor Venetoclax in Combination With Standard Intensive AML Induction/Consolidation Therapy With FLAG-IDA in Patients With Newly Diagnosed or Relapsed/Refractory AML [NCT03214562]Phase 1/Phase 2116 participants (Anticipated)Interventional2017-09-26Recruiting
Administration of Autologous CAR-T Cells Targeting the CD19 Antigen and Containing the Inducible caspase9 Safety Switch in Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia [NCT03016377]Phase 1/Phase 254 participants (Anticipated)Interventional2012-03-22Recruiting
A Study of BCMA CAR-T Cells for Patients With Relapse and Refractory Multiple Myeloma [NCT03322735]Phase 1/Phase 210 participants (Anticipated)Interventional2017-12-08Recruiting
The Safety and Efficacy of CART-19 Cells in Relapse and Refractory Patients With CD19+ B-cell Lymphoma [NCT03101709]Phase 130 participants (Anticipated)Interventional2016-08-31Recruiting
T-cell Therapy in Combination With Vemurafenib for Patients With BRAF Mutated Metastatic Melanoma [NCT02354690]Phase 1/Phase 213 participants (Actual)Interventional2014-11-30Completed
Safety and Efficacy Evaluation of IM19 Chimeric Antigen Receptor-modified T Cells (IM19CAR-T) In CD19+ B Cell Malignancies [NCT03344705]Phase 120 participants (Anticipated)Interventional2017-08-21Recruiting
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.)
A Pilot Study of Condensed Busulfan, Melphalan, and Fludarabine Conditioning Prior to Ex-vivo CD34+ Selected Allogeneic Hematopoietic Cell Transplantation [NCT04098393]Phase 145 participants (Anticipated)Interventional2019-09-18Recruiting
Phase 1 A/B Study of LY2606368 in Combination With Cytarabine and Fludarabine in Patients With Relapsed/Refractory Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (HRMDS) [NCT02649764]Phase 115 participants (Actual)Interventional2016-05-04Completed
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
Open-label, Multicenter, Randomized, Comparative, Phase III Study to Evaluate the Efficacy and Safety of FCR vs. FC Alone in Previously Treated Patients With CD20 Positive B-cell CLL [NCT00090051]Phase 3552 participants (Actual)Interventional2003-07-31Completed
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
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

TrialOutcome
NCT00001586 (2) [back to overview]Change in Gene Expression Post Chemo
NCT00001586 (2) [back to overview]Number of Participants With Adverse Events
NCT00001832 (2) [back to overview]Number of Participants With Adverse Events
NCT00001832 (2) [back to overview]Clinical Response
NCT00003659 (3) [back to overview]Utilize Flow Cytometry and Polymerase Chain Reaction as Sensitive Measures of Minimal Residual Disease
NCT00003659 (3) [back to overview]Overall Response Rate
NCT00003659 (3) [back to overview]Overall Survival Status
NCT00003816 (4) [back to overview]4 yr OS
NCT00003816 (4) [back to overview]4 Year PFS
NCT00003816 (4) [back to overview]Toxicity/TRM at Day 100
NCT00003816 (4) [back to overview]CR Rate
NCT00005803 (4) [back to overview]Progression Free-survival (PFS)
NCT00005803 (4) [back to overview]Overall Survival (OS)
NCT00005803 (4) [back to overview]Non-Relapse Mortality
NCT00005803 (4) [back to overview]Engraftment of HLA Identical PBSC Allografts
NCT00006184 (2) [back to overview]Number of Participants With Adverse Events
NCT00006184 (2) [back to overview]Immune Response
NCT00027560 (6) [back to overview]Acute Graft-versus-Host Disease Unrelated and Mismatched Related Patients
NCT00027560 (6) [back to overview]Extensive Chronic Graft-versus-Host Disease Matched Related Patients
NCT00027560 (6) [back to overview]Overall Survival
NCT00027560 (6) [back to overview]Overall Survival
NCT00027560 (6) [back to overview]Acute Graft-versus-Host Disease Matched Related Patients
NCT00027560 (6) [back to overview]Extensive Chronic Graft-versus-Host Disease Unrelated and Mismatched Related Patients
NCT00036738 (5) [back to overview]Relapse Free Survival
NCT00036738 (5) [back to overview]Transplant-related Mortality
NCT00036738 (5) [back to overview]Overall Survival
NCT00036738 (5) [back to overview]Leukemia-free Survival
NCT00036738 (5) [back to overview]Transplant-related Mortality
NCT00038857 (1) [back to overview]Number of Participants With Absolute Neutrophil Count Engraftment
NCT00040846 (5) [back to overview]Evaluate the Risk for Disease Progression and Relapse
NCT00040846 (5) [back to overview]Evaluate the Risk of Transplant Related Mortality.
NCT00040846 (5) [back to overview]Evaluate the Risk/Incidence of Infections
NCT00040846 (5) [back to overview]Determine Whether Engraftment Can be Maintained With a Single Dose Fludarabine, DLI and Continued MMF/CSP, Defined as Rejection Rate < 20%.
NCT00040846 (5) [back to overview]Evaluate the Risk of Occurrence of Acute and Chronic GVHD
NCT00043979 (14) [back to overview]Number of Participants With Engraftment
NCT00043979 (14) [back to overview]Toxicity
NCT00043979 (14) [back to overview]Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin)
NCT00043979 (14) [back to overview]Median Survival From Date of Progression
NCT00043979 (14) [back to overview]Number of Participants to Complete Conversion to >95% Donor Chimerism
NCT00043979 (14) [back to overview]Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy
NCT00043979 (14) [back to overview]Median Time to Reach Absolute Neutrophil Count of 500/mm(3)
NCT00043979 (14) [back to overview]Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant
NCT00043979 (14) [back to overview]Number of Participants With Acute and Chronic GVHD
NCT00043979 (14) [back to overview]Median Time to Reach a Platelet Count of 50,000/mm(3)
NCT00043979 (14) [back to overview]Median Progression Free Survival
NCT00043979 (14) [back to overview]Cluster of Differentiation 4 (CD4) Reconstitution
NCT00043979 (14) [back to overview]Number of Participants Who Experienced Graft Versus Tumor Effect (GVT)
NCT00043979 (14) [back to overview]Early Post Transplantation Relapse
NCT00045435 (6) [back to overview]Disease-free Survival-incidence of Survival Without Relapse
NCT00045435 (6) [back to overview]Incidence of Rejection
NCT00045435 (6) [back to overview]Incidence of Acute and Chronic GVHD
NCT00045435 (6) [back to overview]Overall Survival
NCT00045435 (6) [back to overview]Nonrelapse Mortality (NRM)-Incidence of Nonrelapse Death
NCT00045435 (6) [back to overview]Incidence of Relapse
NCT00047060 (11) [back to overview]Number of Participants Who Experienced Transplant Related Mortality
NCT00047060 (11) [back to overview]Number of Participants That Remained Disease-free
NCT00047060 (11) [back to overview]Number of Participants That Experienced Red Blood Cell Recovery
NCT00047060 (11) [back to overview]Efficacy of Nonmyeloablative Preparative Regimen
NCT00047060 (11) [back to overview]Number of Participants That Experienced Platelet Recovery
NCT00047060 (11) [back to overview]Number of Participants That Experienced Graft Failure
NCT00047060 (11) [back to overview]Number of Participants That Experienced Engraftment
NCT00047060 (11) [back to overview]Number of Participants Overall Survival
NCT00047060 (11) [back to overview]Number of Participants Who Experienced Acute GVHD Grades II-IV
NCT00047060 (11) [back to overview]Number of Participant Who Experienced Chronic Graft Versus Host Disease
NCT00047060 (11) [back to overview]Median Time in Months to Achieve Full Myeloid and Full Donor T-cell Chimerism
NCT00048737 (1) [back to overview]Number of Participants With Graft Failure
NCT00048893 (1) [back to overview]Number of Participants With Adverse Events
NCT00049504 (3) [back to overview]Donor Engraftment (Chimerism)
NCT00049504 (3) [back to overview]Non-relapse-related Mortality
NCT00049504 (3) [back to overview]Incidence of Grades III-IV Acute GVHD
NCT00051311 (9) [back to overview]Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis
NCT00051311 (9) [back to overview]Percentage of Recipients Who Achieved Donor Chimerism at Day +14
NCT00051311 (9) [back to overview]Number of Recipients With Non-serious Adverse Events
NCT00051311 (9) [back to overview]Median Time to Neutrophil Recovery
NCT00051311 (9) [back to overview]Median Time to Platelet Recovery
NCT00051311 (9) [back to overview]Number of Recipients Evaluable Who Achieved Full Donor Chimerism at Day+100
NCT00051311 (9) [back to overview]Median Cycles of Induction Chemotherapy With Fludarabine, Cyclophosphamide, Etoposide, Doxorubicin, Vincristine, and Prednisone Given to Recipients
NCT00051311 (9) [back to overview]Number of Recipients Who Developed Chronic Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis
NCT00051311 (9) [back to overview]Number of Recipients With a Response to Reduced-intensity Stem Cell Transplantation (RIST)
NCT00053014 (2) [back to overview]Serious Adverse Events
NCT00053014 (2) [back to overview]Overall Survival
NCT00053989 (4) [back to overview]Acute GvHD
NCT00053989 (4) [back to overview]OS
NCT00053989 (4) [back to overview]PFS
NCT00053989 (4) [back to overview]Day 100 TRM
NCT00054353 (8) [back to overview]PFS
NCT00054353 (8) [back to overview]Relapse Rate
NCT00054353 (8) [back to overview]Response Rate
NCT00054353 (8) [back to overview]Engraftment
NCT00054353 (8) [back to overview]Incidence of Acute GVHD (Grades III-IV)
NCT00054353 (8) [back to overview]OS
NCT00054353 (8) [back to overview]Non-relapse Mortality
NCT00054353 (8) [back to overview]Incidence of Chronic (Extensive) GVHD
NCT00058825 (9) [back to overview]Median Time to Engraftment With the Isolex/CLINIMACs System
NCT00058825 (9) [back to overview]2-year Overall Survival
NCT00058825 (9) [back to overview]Donor Chimerism Engraftment of Greater Than 50%
NCT00058825 (9) [back to overview]Transplant Related Mortality (TRM)
NCT00058825 (9) [back to overview]Number of Patients Who Engrafted With the Isolex/CLINIMACs System
NCT00058825 (9) [back to overview]Time in Days to ANC Engraftment
NCT00058825 (9) [back to overview]Acute Graft Versus Host Disease
NCT00058825 (9) [back to overview]Chronic Graft Versus Host Disease
NCT00058825 (9) [back to overview]2-year Relapse-free Survival
NCT00060424 (5) [back to overview]Transplant-related Mortality
NCT00060424 (5) [back to overview]Rate of Relapse
NCT00060424 (5) [back to overview]Acute Grade II-IV GVHD and Chronic (Extensive) GVHD
NCT00060424 (5) [back to overview]Rate and Types of Infections
NCT00060424 (5) [back to overview]Overall Survival
NCT00067002 (5) [back to overview]Number of Participants Severity of Acute GVHD by Treatment Arm
NCT00067002 (5) [back to overview]Rate of Chronic GVHD
NCT00067002 (5) [back to overview]Number of Participants With Engraftment
NCT00067002 (5) [back to overview]Rate of Acute Graft Versus Host Disease (GVHD)
NCT00067002 (5) [back to overview]Time To Neutrophil Engraftment
NCT00070135 (3) [back to overview]Non-relapse Mortality (NRM)
NCT00070135 (3) [back to overview]2 Year Disease Free Survival In Unrelated Donor Recipient Group
NCT00070135 (3) [back to overview]2 Year DFS for All Patients
NCT00074269 (7) [back to overview]Response as Measured at 12 Months Post Allografting
NCT00074269 (7) [back to overview]Progression-free Survival
NCT00074269 (7) [back to overview]Overall Survival
NCT00074269 (7) [back to overview]Number of Participants With Long-term Engraftment of Allogeneic Stem Cells and Lymphocytes
NCT00074269 (7) [back to overview]Number of Participants With Adverse Events
NCT00074269 (7) [back to overview]Number of Participants With Acute and Chronic Graft-versus-host Disease (GVHD)
NCT00074269 (7) [back to overview]Frequency of the Induction of Full Donor Chimerism of Lymphocytes as Measured at 1 Month Post Allografting
NCT00074490 (4) [back to overview]Percentage of Patients With Opportunistic Infection
NCT00074490 (4) [back to overview]Percentage of Patients With ≥ Grade 2 Acute Graft Versus Host Disease (GVHD)
NCT00074490 (4) [back to overview]Percentage of Patients to Receive T Cell Infusion
NCT00074490 (4) [back to overview]Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0)
NCT00075478 (8) [back to overview]Incidence of Graft Rejection
NCT00075478 (8) [back to overview]Incidence of Relapse-related Mortality
NCT00075478 (8) [back to overview]Incidence of Relapse/Progression
NCT00075478 (8) [back to overview]Incidence of Non-relapse Mortality
NCT00075478 (8) [back to overview]Incidence of Grades II-IV Acute GVHD
NCT00075478 (8) [back to overview]Incidence of Chronic Extensive GVHD
NCT00075478 (8) [back to overview]Progression-free Survival
NCT00075478 (8) [back to overview]Overall Survival
NCT00076752 (16) [back to overview]Anti-Nuclear Antibody
NCT00076752 (16) [back to overview]Anti-Smith-Ribonuclear Protein Antibody
NCT00076752 (16) [back to overview]Cluster of Differentiation 19 (CD19) + Cells
NCT00076752 (16) [back to overview]Cluster of Differentiation 3 (CD3) + Cells
NCT00076752 (16) [back to overview]Cluster of Differentiation 4 (CD4) + Cells
NCT00076752 (16) [back to overview]Cluster of Differentiation 8 (CD8) + Cells
NCT00076752 (16) [back to overview]Extractable Nuclear Antigen (ENA)
NCT00076752 (16) [back to overview]Natural Killer Cells
NCT00076752 (16) [back to overview]Platelet Count
NCT00076752 (16) [back to overview]Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)
NCT00076752 (16) [back to overview]Number of Participants With Adverse Events
NCT00076752 (16) [back to overview]White Blood Cells
NCT00076752 (16) [back to overview]Relapse-free Complete Clinical Response
NCT00076752 (16) [back to overview]Absolute Lymphocyte Count
NCT00076752 (16) [back to overview]Absolute Neutrophil Count
NCT00076752 (16) [back to overview]Anti-Double Stranded Deoxyribonucleic Acid (DNA) Antibody
NCT00085423 (3) [back to overview]Number of partiCIPANTS WITH OBJECTIVE RESPONSE AS MEASURED BY RECIST
NCT00085423 (3) [back to overview]Number of Participants With Lymphocyte Recovery as Measured by Blood Count
NCT00085423 (3) [back to overview]Time to Progression as Measured by RECIST
NCT00086580 (20) [back to overview]Mean EQ-5D™ Index Scores to Measure Quality of Life at End of Treatment
NCT00086580 (20) [back to overview]Participant Best Response to Treatment Assessed by the Independent Response Review Panel (IRRP)
NCT00086580 (20) [back to overview]Total Volume of Distribution (Vss) of Fludarabine
NCT00086580 (20) [back to overview]Participants With Minimal Residual Disease (MRD)
NCT00086580 (20) [back to overview]Mean Systemic Clearance (CL) of Fludarabine
NCT00086580 (20) [back to overview]Mean EuroQol Visual Analogue Scale (EQ-VAS) Scores to Measure Quality of Life at End of Treatment
NCT00086580 (20) [back to overview]Mean EuroQol Visual Analogue Scale (EQ-VAS) Scores to Measure Quality of Life at Baseline
NCT00086580 (20) [back to overview]Area Under the Curve (AUC) of Fludarabine From (AUC 0-tau)
NCT00086580 (20) [back to overview]Mean EQ-5D™ Index Scores to Measure Quality of Life at Baseline
NCT00086580 (20) [back to overview]Maximum Plasma Concentration (Cmax) of Fludarabine
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates of Overall Survival Time for Participants With Rai Stage I-II
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates of Overall Survival Time
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates for Time to Alternative Therapy
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates for Progression-free Survival (PFS) Based on Independent Response Review Panel (IRRP) for Participants With Rai Stage III-IV
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates for Progression-free Survival (PFS) Based on Independent Response Review Panel (IRRP) Assessment
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates for Duration of Response Assessed by the Independent Response Review Panel (IRRP)
NCT00086580 (20) [back to overview]Kaplan Meier Estimates for Time to Disease Progression Assessed by the Independent Response Review Panel (IRRP)
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates of Overall Survival Time for Participants With Rai Stage III-IV
NCT00086580 (20) [back to overview]Kaplan-Meier Estimates for Progression-free Survival (PFS) Based on Independent Response Review Panel (IRRP) for Participants With Rai Stage I-II
NCT00086580 (20) [back to overview]Summary of Participants With Adverse Experiences (AEs)
NCT00089011 (8) [back to overview]Incidences of Grades II-IV Acute GVHD
NCT00089011 (8) [back to overview]Incidence of Grade III/IV GVHD
NCT00089011 (8) [back to overview]Incidences of Graft Rejection
NCT00089011 (8) [back to overview]Overall Survival
NCT00089011 (8) [back to overview]Incidence of Chronic Extensive GVHD
NCT00089011 (8) [back to overview]Rates of Relapse-related Mortality
NCT00089011 (8) [back to overview]Rates of Disease Progression
NCT00089011 (8) [back to overview]Rate and Duration of Steroid Use for the Treatment of Chronic GVHD
NCT00090051 (15) [back to overview]Final Analysis: Time to New Chronic Lymphocytic Leukemia (CLL) Treatment
NCT00090051 (15) [back to overview]Final Analysis: Time to Overall Survival Event
NCT00090051 (15) [back to overview]Final Analysis: Percentage of Participants With Complete Response
NCT00090051 (15) [back to overview]Progression-free Survival (PFS) as Assessed by the Independent Review Committee (IRC)
NCT00090051 (15) [back to overview]Number of Participants With Disease-free Survival (DFS) Events
NCT00090051 (15) [back to overview]Number of Participants With Event-free Survival (EFS) Events
NCT00090051 (15) [back to overview]Number of Participants With Overall Survival (OS) Events
NCT00090051 (15) [back to overview]Number of Participants With Progression-free Survival (PFS) Events Assessed by the Independent Review Committee (IRC)
NCT00090051 (15) [back to overview]Final Analysis: Time to Disease-Free Survival Event
NCT00090051 (15) [back to overview]Overall Survival (OS)
NCT00090051 (15) [back to overview]Disease-free Survival (DFS)
NCT00090051 (15) [back to overview]Final Analysis: Time to Progression-Free Survival Event
NCT00090051 (15) [back to overview]Final Analysis: Duration of Response
NCT00090051 (15) [back to overview]Final Analysis: Time to Event-Free Survival Event
NCT00090051 (15) [back to overview]Event-free Survival (EFS)
NCT00096018 (2) [back to overview]Duration of Response
NCT00096018 (2) [back to overview]Overall Responders (Complete and Partial Response)
NCT00096382 (2) [back to overview]Clinical Tumor Regression
NCT00096382 (2) [back to overview]Safety
NCT00096460 (1) [back to overview]Lymphoma Progression-free Survival
NCT00098670 (5) [back to overview]Number of Participants With a Complete Response After Treatment With Fludarabine & Rituximab Followed by Alemtuzumab
NCT00098670 (5) [back to overview]Number of Participants With Severe Non-Hematologic Adverse Events During Treatment With Alemtuzumab
NCT00098670 (5) [back to overview]2 Year Survival
NCT00098670 (5) [back to overview]2 Year Progression Free Survival
NCT00098670 (5) [back to overview]Number of Participants With a Complete or Partial Response After Induction Therapy With Fludarabine & Rituximab
NCT00104858 (8) [back to overview]Number of Participants With Regimen-related Toxicity and Infections
NCT00104858 (8) [back to overview]Number of Participants With Grade II-III and III-IV Acute GVHD and Chronic GVHD
NCT00104858 (8) [back to overview]Rituxan Concentration
NCT00104858 (8) [back to overview]Donor and Host Polymorphisms of the FCgammaRIIIa Receptor and CD32 and Their Impact on Disease Response and Relapse
NCT00104858 (8) [back to overview]Overall Survival
NCT00104858 (8) [back to overview]Number of Patients Achieving Complete Response and Partial Response (Overall Response Rate)
NCT00104858 (8) [back to overview]Number of Participants With Treatment-related Mortality
NCT00104858 (8) [back to overview]Number of Participants With Relapse/Progression
NCT00105001 (5) [back to overview]Number of Participants Surviving Without Progression
NCT00105001 (5) [back to overview]Number of Participants Utilizing High-Dose Corticosteroids
NCT00105001 (5) [back to overview]Number of Non-Relapse Mortalities
NCT00105001 (5) [back to overview]Number of Participants Surviving Overall
NCT00105001 (5) [back to overview]Number of Participants With Grades II-IV Acute GVHD
NCT00112593 (6) [back to overview]Progression of HIV
NCT00112593 (6) [back to overview]Death From Regimen Toxicity or Opportunistic Infection
NCT00112593 (6) [back to overview]Death From GVHD
NCT00112593 (6) [back to overview]Successful Induction of Mixed Hematopoietic Chimerism as Assessed by the Percentage of Peripheral Blood T Cells That Are of Donor Origin
NCT00112593 (6) [back to overview]Reconstitution of HIV-specific Immunity
NCT00112593 (6) [back to overview]Overall Survival
NCT00117156 (5) [back to overview]Delayed Pneumonia Toxicity Rate
NCT00117156 (5) [back to overview]Objective Response Rate
NCT00117156 (5) [back to overview]Delayed Bone Marrow Toxicity Rate
NCT00117156 (5) [back to overview]3.1-Year Progression-Free Survival
NCT00117156 (5) [back to overview]3.1-Year Overall Survival
NCT00118352 (6) [back to overview]Incidence of Non-relapse Mortality
NCT00118352 (6) [back to overview]Disease Progression/Relapse
NCT00118352 (6) [back to overview]Incidence of Infection
NCT00118352 (6) [back to overview]Incidence of Grade III-IV Acute GVHD
NCT00118352 (6) [back to overview]Incidence of High-dose Corticosteroid Utilization.
NCT00118352 (6) [back to overview]Incidence of Graft Rejection
NCT00119366 (3) [back to overview]Participant Disease Response Within 4 Weeks After Transplant
NCT00119366 (3) [back to overview]Number of Participants With 100% Donor Chimerism at Day 28 and Day 84
NCT00119366 (3) [back to overview]Two-year Disease-free Survival of Study Participants Who Completed the Study Regimen
NCT00119392 (5) [back to overview]Overall and Progression-free Survival
NCT00119392 (5) [back to overview]Incidence and Severity of Acute Graft-versus-host Disease (GVHD) and Chronic GVHD.
NCT00119392 (5) [back to overview]Engraftment and Hematopoietic Toxicity
NCT00119392 (5) [back to overview]Treatment Related Mortality (TRM)
NCT00119392 (5) [back to overview]Response Rates
NCT00121186 (2) [back to overview]Overall Survival
NCT00121186 (2) [back to overview]Progression-free Survival
NCT00134004 (5) [back to overview]Progression-free Survival
NCT00134004 (5) [back to overview]Graft Failure Rate
NCT00134004 (5) [back to overview]Hematologic and Non-hematologic Toxicities as Measured by NCI Common Toxicity Criteria for Adverse Events, v 3.0 Weekly Until 1 Year After Transplantation
NCT00134004 (5) [back to overview]Relapse Rate
NCT00134004 (5) [back to overview]Transplant-related Mortality
NCT00145626 (13) [back to overview]Number of Transplant-Related Adverse Outcomes: Engraftment Failure
NCT00145626 (13) [back to overview]Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation
NCT00145626 (13) [back to overview]Kinetics of Lymphohematopoietic Reconstitution
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Minimal Residual Disease (MRD)
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Median Dose of NK Cells
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Donor Type
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Disease Status at HSCT
NCT00145626 (13) [back to overview]One-year Survival
NCT00145626 (13) [back to overview]Number of Transplant-Related Adverse Outcomes: Regimen-Related Mortality
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Median Dose of CD34
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Median Age of Donor at HSCT
NCT00145626 (13) [back to overview]Number of Transplant-Related Adverse Outcomes: Fatal Acute Graft-Versus Host Disease (GVHD)
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Match N/6 HLA Loci
NCT00153985 (3) [back to overview]The Incidence of Grade II-IV Acute Graft vs. Host Disease.
NCT00153985 (3) [back to overview]Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.
NCT00153985 (3) [back to overview]Solid Organ Toxicity Related to the Conditioning Regimen.
NCT00167206 (9) [back to overview]Immune Reconstitution - Mean Value (1 Year)
NCT00167206 (9) [back to overview]Number of Patients Who Exhibited Regimen-related Toxicity (RRT)
NCT00167206 (9) [back to overview]Number of Patients Who Exhibited Secondary Graft Failure
NCT00167206 (9) [back to overview]Number of Patients With Acute Graft Versus-Host Disease (aGVHD)
NCT00167206 (9) [back to overview]Number of Patients With Chronic Graft Versus-Host Disease (GVHD)
NCT00167206 (9) [back to overview]Immune Reconstitution - Mean Value (2 Years)
NCT00167206 (9) [back to overview]Number of Patients Alive at 1 Year
NCT00167206 (9) [back to overview]Number of Patients Alive at 2 Years
NCT00167206 (9) [back to overview]Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment
NCT00176852 (15) [back to overview]Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
NCT00176852 (15) [back to overview]The Incidence of Chimerism at 6 Months
NCT00176852 (15) [back to overview]The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
NCT00176852 (15) [back to overview]The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
NCT00176852 (15) [back to overview]Disease Free Survival
NCT00176852 (15) [back to overview]Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
NCT00176852 (15) [back to overview]Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
NCT00176852 (15) [back to overview]Overall Survival
NCT00176852 (15) [back to overview]Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
NCT00176852 (15) [back to overview]The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)
NCT00176852 (15) [back to overview]The Incidence of Chimerism at 1 Year
NCT00176852 (15) [back to overview]Overall Survival
NCT00176852 (15) [back to overview]The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)
NCT00176852 (15) [back to overview]Disease Free Survival
NCT00176852 (15) [back to overview]The Incidence of Chimerism at 100 Days
NCT00176865 (9) [back to overview]Percentage of Donor Chimerism at 100 Days
NCT00176865 (9) [back to overview]Number of Subjects With Mixed Chimerism
NCT00176865 (9) [back to overview]Incidence of Chronic Graft Versus Host Disease (cGVHD)
NCT00176865 (9) [back to overview]Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD)
NCT00176865 (9) [back to overview]Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD)
NCT00176865 (9) [back to overview]Number of Subjects Alive at One Year
NCT00176865 (9) [back to overview]Number of Subjects Alive at 100 Days
NCT00176865 (9) [back to overview]Percentage of Donor Chimerism at 365 Days
NCT00176865 (9) [back to overview]Percentage of Donor Chimerism at 180 Days
NCT00176878 (6) [back to overview]Number of Patients With Disease Recurrence
NCT00176878 (6) [back to overview]Number of Patients With Grade 2-4 Acute Graft Versus Host Disease
NCT00176878 (6) [back to overview]Number of Patients With Succcessful Engraftment After Transplantation
NCT00176878 (6) [back to overview]Number of Patients Alive at Three Years (Survival)
NCT00176878 (6) [back to overview]Number of Patients With Chronic Graft Versus Host Disease
NCT00176878 (6) [back to overview]Number of Patients Alive (Survival) at 2 Years
NCT00187096 (10) [back to overview]Duration of Engraftment of Natural Killer (NK) Cells
NCT00187096 (10) [back to overview]Overall Survival
NCT00187096 (10) [back to overview]Number of Patients Experiencing Grade 3 or 4 Toxicities During Conditioning and up to 100 Days Post-transplant
NCT00187096 (10) [back to overview]Percent of Peak NK Cell Chimerism
NCT00187096 (10) [back to overview]Proportion of Patients Experiencing Grade 3 or 4 Toxicities During Conditioning and up to 100 Days Post-transplant
NCT00187096 (10) [back to overview]Relapse-free Survival
NCT00187096 (10) [back to overview]Number of KIR-mismatched NK Cells
NCT00187096 (10) [back to overview]Number of Participants With Evidence of NK Cells Lysing a Target Cell Line (K562)
NCT00187096 (10) [back to overview]Percent of Detectable Donor NK Cells at Day 28
NCT00187096 (10) [back to overview]Day That Maximum NK Cell Engraftment Was Reached
NCT00206726 (6) [back to overview]Percentage of Participants With Overall Response at Different Observation Times
NCT00206726 (6) [back to overview]Complete Response (CR)
NCT00206726 (6) [back to overview]Overall Survival (OS)
NCT00206726 (6) [back to overview]Overall Response (OR)
NCT00206726 (6) [back to overview]Progression-free Survival (PFS)
NCT00206726 (6) [back to overview]Number of Participants With Minimal Residual Disease (MRD)
NCT00208975 (1) [back to overview]Number of Patients's Who Had Complete Response and Partial Response to the Treatment of Fludarabine and Cyclophosphamide Followed by GM-CSF and Rituximab.
NCT00230282 (2) [back to overview]Number of Subjects Maintaining Partial Response (PR) or Complete Response (CR)
NCT00230282 (2) [back to overview]Duration of Response
NCT00245037 (7) [back to overview]Progression-Free Survival
NCT00245037 (7) [back to overview]Acute Graft-Versus-Host Disease (aGVHD) Outcome
NCT00245037 (7) [back to overview]Chronic Graft-Versus-Host Disease (cGVHD) Outcome
NCT00245037 (7) [back to overview]Non-relapse Mortality
NCT00245037 (7) [back to overview]Overall Survival
NCT00245037 (7) [back to overview]Relapse Mortality
NCT00245037 (7) [back to overview]Regimen-Related Toxicities
NCT00253513 (4) [back to overview]Number of Patients Experiencing Regimen-related Toxicity Events in Study Population
NCT00253513 (4) [back to overview]Number of Patients Experiencing Graft Failure
NCT00253513 (4) [back to overview]Number of Subjects Who Are Without Disease at One Year as Indicator of Disease Free Survival.
NCT00253513 (4) [back to overview]Incidence (Percent of Participants) With Nonrelapse Mortality (NRM) by Day 200 (Secondary Phase Only)
NCT00254163 (9) [back to overview]Infective Event Rate
NCT00254163 (9) [back to overview]Hematologic Recovery
NCT00254163 (9) [back to overview]Infection Rate
NCT00254163 (9) [back to overview]Mean Absolute Neutrophil Count (ANC) at Post-treatment
NCT00254163 (9) [back to overview]Objective Remission Rate (ORR)
NCT00254163 (9) [back to overview]Percentage of Patients Hospitalized
NCT00254163 (9) [back to overview]Progression-free Survival (PFS) Rate at 1-year
NCT00254163 (9) [back to overview]Progression-free Survival (PFS) Rate at 2-year
NCT00254163 (9) [back to overview]Complete Remission (CR)
NCT00254410 (4) [back to overview]Clinical Response Rate at 6 Months
NCT00254410 (4) [back to overview]Clinical Response Rate at 3 Months
NCT00254410 (4) [back to overview]Molecular Response Rate at 3 Months
NCT00254410 (4) [back to overview]Molecular Response Rate at 6 Months
NCT00255684 (2) [back to overview]Number of Participants Who Survived 100 Days or Longer
NCT00255684 (2) [back to overview]Number of Participants Who Developed Acute Graft Versus Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Major Infections
NCT00258427 (8) [back to overview]Number of Participants Experiencing Acute Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Acute Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Chronic Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Chronic Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Graft Failure
NCT00258427 (8) [back to overview]Number of Participants Experiencing Overall Survival
NCT00258427 (8) [back to overview]Number of Participants Experiencing Relapse
NCT00274846 (4) [back to overview]Median Time to Disease Relapse (Months)
NCT00274846 (4) [back to overview]Overall Survival Time of Patients With Complete Remission
NCT00274846 (4) [back to overview]Number of Patients With Natural Killer (NK) Cell Expansion
NCT00274846 (4) [back to overview]Number of Patients With Complete Remission
NCT00278512 (1) [back to overview]Survival
NCT00280241 (4) [back to overview]Overall Survival Rate
NCT00280241 (4) [back to overview]Tolerability of Rituximab, Cyclophosphamide and Fludarabine in Patients With Previously Untreated CLL/SLL
NCT00280241 (4) [back to overview]Duration of Response
NCT00280241 (4) [back to overview]Efficacy of Rituximab, Cyclophosphamide and Fludarabine in Patients With Previously Untreated CLL/SLL
NCT00281918 (11) [back to overview]Final Analysis: Time to Overall Survival Event
NCT00281918 (11) [back to overview]Final Analysis: Time to Progression-free Survival Event
NCT00281918 (11) [back to overview]Event-free Survival (EFS)
NCT00281918 (11) [back to overview]Final Analysis: Duration of Response
NCT00281918 (11) [back to overview]Final Analysis: Time to Disease-free Survival (DFS) Event in Participants With Complete Response (CR)
NCT00281918 (11) [back to overview]Overall Survival (OS)
NCT00281918 (11) [back to overview]Final Analysis: Percentage of Participants With Complete Response (CR) and Partial Response
NCT00281918 (11) [back to overview]Final Analysis: Time to Event-free Survival Event
NCT00281918 (11) [back to overview]Final Analysis: Time to New Treatment for Chronic Lymphocytic Leukemia(CLL)
NCT00281918 (11) [back to overview]Disease-free Survival (DFS) of Patients With Confirmed Complete Response (CR).
NCT00281918 (11) [back to overview]Progression-free Survival (PFS)
NCT00282282 (3) [back to overview]Percentage of Participants With ≥90 Percent Donor-derived Hematopoeisis Around 100 Days Post Transplantation
NCT00282282 (3) [back to overview]Incidence of Grade II-IV Acute GVHD (aGVHD) Developing by Day 100 Following Non-myeloablative PBSC Transplantation Using Tacrolimus and Sirolimus.
NCT00282282 (3) [back to overview]Disease Response.
NCT00282412 (1) [back to overview]Survival
NCT00290511 (7) [back to overview]Median Progression Free Survival
NCT00290511 (7) [back to overview]Overall Survival
NCT00290511 (7) [back to overview]Percentage of Participants With Overall Survival Rate at 10 Years
NCT00290511 (7) [back to overview]Number of Participants With Time to Progression (TTP)
NCT00290511 (7) [back to overview]Tolerance and Efficacy of Maintenance Therapy With Rituximab
NCT00290511 (7) [back to overview]Tolerance and Efficacy of Maintenance Therapy With Yttrium-90 Ibritumomab Tiuxetan (YIT)
NCT00290511 (7) [back to overview]Progression Free Survival at 10 Years
NCT00301834 (5) [back to overview]Disease-free Survival With Correction of Disease at One Year Post Transplantation
NCT00301834 (5) [back to overview]Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation
NCT00301834 (5) [back to overview]Cytomegalovirus (CMV) Viral Infection and Disease Symptoms
NCT00301834 (5) [back to overview]Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation
NCT00301834 (5) [back to overview]Treatment-related Mortality at 100 Days and 1 Year Post Transplantation
NCT00303667 (9) [back to overview]Number of Patients With Treatment-Related Mortality
NCT00303667 (9) [back to overview]Number of Patients With Graft Failure
NCT00303667 (9) [back to overview]Disease-free Survival at 1 Year
NCT00303667 (9) [back to overview]Disease-free Survival at 6 Months
NCT00303667 (9) [back to overview]In Vivo Expansion of a Donor NK Cells NK Cell Product
NCT00303667 (9) [back to overview]Incidence of Chronic Graft Versus Host Disease
NCT00303667 (9) [back to overview]Incidence of Grade III-IV Acute Graft Versus Host Disease
NCT00303667 (9) [back to overview]Incidence of Post-transplant Lymphoproliferative Disorder (PTLD)
NCT00303667 (9) [back to overview]Number of Patients With Disease Relapse
NCT00303719 (5) [back to overview]Overall Survival
NCT00303719 (5) [back to overview]Neutrophil and Donor Cell Engraftment
NCT00303719 (5) [back to overview]Acute Graft-Versus-Host Disease
NCT00303719 (5) [back to overview]Transplant Related Mortality
NCT00303719 (5) [back to overview]Serious Adverse Events
NCT00305682 (15) [back to overview]Number of Participants With Chronic Graft-Versus-Host Disease
NCT00305682 (15) [back to overview]Number of Participants Experiencing Relapse (Incidence of Relapse)
NCT00305682 (15) [back to overview]Number of Participants Experiencing Relapse (Incidence of Relapse) at 2 Years
NCT00305682 (15) [back to overview]Number of Participants Who Were Alive at 1 Year Post Transplant
NCT00305682 (15) [back to overview]Number of Participants Who Were Dead at 6 Months After Study Completion
NCT00305682 (15) [back to overview]Number of Participants With Acute Graft-versus-host Disease (GVHD)
NCT00305682 (15) [back to overview]Number of Participants Who Were Alive at 2 Years Post Transplant
NCT00305682 (15) [back to overview]Percentage of Donor Chimerism at 180 Days
NCT00305682 (15) [back to overview]Percentage of Donor Chimerism at 21 Days
NCT00305682 (15) [back to overview]Percentage of Donor Chimerism at 365 Days
NCT00305682 (15) [back to overview]Number of Participants Experiencing Progression-free Survival
NCT00305682 (15) [back to overview]Number of Participants Experiencing Progression-free Survival at 2 Years
NCT00305682 (15) [back to overview]Number of Participants With Neutrophil Engraftment
NCT00305682 (15) [back to overview]Number of Participants With Platelet Engraftment
NCT00305682 (15) [back to overview]Percentage of Donor Chimerism at 100 Days
NCT00309842 (11) [back to overview]Number of Participants With Neutrophil Engraftment
NCT00309842 (11) [back to overview]Number of Participants With Chronic Graft-Versus-Host Disease
NCT00309842 (11) [back to overview]Number of Participants Who Were Alive at 1 Year Transplant Overall Survival
NCT00309842 (11) [back to overview]Number of Participants Who Died Due to Transplant
NCT00309842 (11) [back to overview]Number of Participants With Platelet Engraftment
NCT00309842 (11) [back to overview]Percentage Chimerism at 1 Year
NCT00309842 (11) [back to overview]Number of Participants With Acute Graft-Versus-Host Disease
NCT00309842 (11) [back to overview]Percentage Chimerism on Day 21
NCT00309842 (11) [back to overview]Percentage Chimerism on Day 100
NCT00309842 (11) [back to overview]Percentage Chimerism at 6 Months
NCT00309842 (11) [back to overview]Percentage Chimerism at 2 Years
NCT00314106 (2) [back to overview]Number of Participants With Adverse Events
NCT00314106 (2) [back to overview]Complete Response
NCT00322101 (6) [back to overview]Incidence and Severity of Acute and Chronic Graft-vs-host Disease
NCT00322101 (6) [back to overview]Progression-free Survival
NCT00322101 (6) [back to overview]Non-relapse Mortality
NCT00322101 (6) [back to overview]Donor Cell Engraftment
NCT00322101 (6) [back to overview]Overall Survival
NCT00322101 (6) [back to overview]Incidence of Disease Progression/Relapse
NCT00326417 (5) [back to overview]Chronic GVHD
NCT00326417 (5) [back to overview]Overall Survival (OS)
NCT00326417 (5) [back to overview]Disease-free Survival (DFS)
NCT00326417 (5) [back to overview]Cumulative Incidence of Graft Failure
NCT00326417 (5) [back to overview]Acute Graft vs Host Disease (GVHD)
NCT00328861 (2) [back to overview]Safety
NCT00328861 (2) [back to overview]Objective Response
NCT00352976 (18) [back to overview]Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
NCT00352976 (18) [back to overview]Average IgG Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
NCT00352976 (18) [back to overview]Number of Participants With Secondary Graft Failure at 100 Days
NCT00352976 (18) [back to overview]Number of Participant With Neutrophil Recovery
NCT00352976 (18) [back to overview]Number of Participants Experiencing Overall Survival
NCT00352976 (18) [back to overview]Number of Participants Experiencing Infections by Day 365
NCT00352976 (18) [back to overview]Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
NCT00352976 (18) [back to overview]Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 180 Days
NCT00352976 (18) [back to overview]Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 100 Days
NCT00352976 (18) [back to overview]Average IgG Levels as a Measure of Immune Reconstitution After Transplant, by 180 Days
NCT00352976 (18) [back to overview]Number of Participants Experiencing Infections by Day 180
NCT00352976 (18) [back to overview]Number of Participants Experiencing Infections by Day 100
NCT00352976 (18) [back to overview]Number of Participants Experiencing Grade ≥3 Regimen Related Toxicity
NCT00352976 (18) [back to overview]Number of Participants Experiencing Chronic GVHD
NCT00352976 (18) [back to overview]Average Immunoglobulin G (IgG) Levels as a Measure of Immune Reconstitution After Transplant, by 100 Days
NCT00352976 (18) [back to overview]Number of Participants Experiencing Acute Graft-versus-host Disease (GVHD)
NCT00352976 (18) [back to overview]Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 100 Days
NCT00352976 (18) [back to overview]Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 180 Days
NCT00354172 (15) [back to overview]Chimerism After Double Umbilical Cord Blood Transplant (UCBT)
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Died Due to Transplant.
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Attained Neutrophil Engraftment
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Attained Platelet Engraftment
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Died by 12 Months
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Died by 24 Months
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Experienced Relapse by 12 Months
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Experienced Relapse by 24 Months
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Were Disease-free and Alive at 12 Months
NCT00354172 (15) [back to overview]Number of Participants (Patients) Who Were Disease-free and Alive at 6 Months
NCT00354172 (15) [back to overview]Number of Participants (Patients) With Acute Graft-versus-host Disease (GVHD) Grade II-IV
NCT00354172 (15) [back to overview]Number of Participants (Patients) With Acute Graft-versus-Host Disease at Grade III-IV
NCT00354172 (15) [back to overview]Number of Participants (Patients) With Chronic Graft-Versus-Host Disease
NCT00354172 (15) [back to overview]Number of Participants (Patients) With Successful Natural Killer Cell Expansion
NCT00354172 (15) [back to overview]Number of Patients Who Were Disease-free and Alive at 24 Months
NCT00358657 (9) [back to overview]Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)
NCT00358657 (9) [back to overview]Incidence of Chronic GVHD
NCT00358657 (9) [back to overview]Proportion of Patients Who Achieve Greater Than 5% Donor T-cell Chimerism
NCT00358657 (9) [back to overview]Number of Patients With Transplant Related Mortality
NCT00358657 (9) [back to overview]Number of Patients With Infections
NCT00358657 (9) [back to overview]Graft Rejection
NCT00358657 (9) [back to overview]Immune Reconstitution
NCT00358657 (9) [back to overview]Graft Failure
NCT00358657 (9) [back to overview]Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)
NCT00361140 (2) [back to overview]Severe Venous Occlusive Disease (VOD)/ Sinusoidal Obstructive Syndrome (SOS)
NCT00361140 (2) [back to overview]Non-relapse Mortality
NCT00376805 (4) [back to overview]Overall Median Number of Days Patients Alive After Treatment
NCT00376805 (4) [back to overview]Number of Patients Who Had Expansion of Natural Killer Cells
NCT00376805 (4) [back to overview]Number of Patients by Disease Response
NCT00376805 (4) [back to overview]Number of Patients Who Died While on Study
NCT00378534 (5) [back to overview]Survival and Non-relapse Mortality at Day +200 Using the Miltenyi Reagent System
NCT00378534 (5) [back to overview]Number of Participants Who Developed Limited Chronic GVHD
NCT00378534 (5) [back to overview]Number of Participants With Relapse of Disease
NCT00378534 (5) [back to overview]Number of Participants Who Develop Extensive GVHD
NCT00378534 (5) [back to overview]Number of Participants Who Developed Acute GVHD Grades I, II, III, IV
NCT00381004 (3) [back to overview]Participant Overall Response Rate (ORR) at 6 Months Includes Complete Remissions, Partial Remission, or Nodule Partial Remissions.
NCT00381004 (3) [back to overview]Number of Participants With Overall Response Includes Complete Remissions, Partial Remission, or Nodule Partial Remissions.
NCT00381004 (3) [back to overview]Number of Participants Progression-free
NCT00381550 (2) [back to overview]Incidence of Grade 3 or 4 Drug-related Non-hematologic Toxicity as Assessed by NCI CTCAE v3.0
NCT00381550 (2) [back to overview]Response Rate Including Complete Response, Partial Response, and Hematological Improvement Assessed by Blood Cell Counts, Number of Blasts in Bone Marrow, and Clinical Evaluation
NCT00385788 (1) [back to overview]Transplant Related Mortality Rate
NCT00387959 (1) [back to overview]Survival at 1 Year After Transplantation
NCT00392782 (7) [back to overview]Incidence of Chronic Graft-versus-host Disease (GVHD)
NCT00392782 (7) [back to overview]Incidence of Disease Relapse
NCT00392782 (7) [back to overview]Incidence of Disease-free Survival
NCT00392782 (7) [back to overview]Incidence of Grade II-IV Acute Graft-vs-host Disease (GVHD)
NCT00392782 (7) [back to overview]Incidence of Graft Failure
NCT00392782 (7) [back to overview]Overall Survival
NCT00392782 (7) [back to overview]Transplant-related Mortality
NCT00393029 (3) [back to overview]Clinical Tumor Regression
NCT00393029 (3) [back to overview]In Vivo Survival of T-cell Receptor (TCR) Gene-engineered Cells in Participants
NCT00393029 (3) [back to overview]The Number of Participants With Adverse Events
NCT00397813 (5) [back to overview]Number of Patients Who Engrafted
NCT00397813 (5) [back to overview]Number of Patients Who Had Infections
NCT00397813 (5) [back to overview]Number of Patients With HCT Failure.
NCT00397813 (5) [back to overview]Number of Patients With Relapse/Progression
NCT00397813 (5) [back to overview]Number of Patients With Progression-free Survival
NCT00410163 (18) [back to overview]AUC(0-inf) and AUC(0-672) After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)
NCT00410163 (18) [back to overview]Percent Change From Screening (Visit 1) in Complement (CH50) Levels at Visit 9 (Week 4)
NCT00410163 (18) [back to overview]Progression-Free Survival
NCT00410163 (18) [back to overview]Time to Next Anti-chronic Lymphocytic Leukemia (CLL) Therapy or Death
NCT00410163 (18) [back to overview]Median Percent Change in CD5+CD19+ and CD5+CD20+ Cells in Peripheral Blood From Onset of Course 3 Throughout Follow-up (FU) Compared to Screening
NCT00410163 (18) [back to overview]Median Percent Change in Tumor Size From Baseline (Visit 2, Wk 0) at Visits 9, 21, 25, 29, 33, 34, 35, and 37
NCT00410163 (18) [back to overview]Number of Participants (Par.) Who Were Classified as Responders and Non-responders
NCT00410163 (18) [back to overview]Vss After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)
NCT00410163 (18) [back to overview]CL After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)
NCT00410163 (18) [back to overview]Duration of Response
NCT00410163 (18) [back to overview]t1/2 After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)
NCT00410163 (18) [back to overview]Number of Participants With Positive Human Anti-human Anti Bodies (HAHA) at Visits 1, 21, 35, and 39
NCT00410163 (18) [back to overview]Number of Participants (Par.) With Complete Remission (CR), Measured From Start of Treatment Until 3 Months After Last Infusion
NCT00410163 (18) [back to overview]Number of Participants Classified as Responders Having CR Who Tested Negative for Minimal Residual Disease (MRD)
NCT00410163 (18) [back to overview]Number of Participants Who Experienced Any Adverse Event From First Treatment (Visit 2) to Visit 43 (Month 60)
NCT00410163 (18) [back to overview]Ctrough and Cmax at the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)
NCT00410163 (18) [back to overview]Number of Participants Who Reported Myelosuppression (Anemia, Leukopenia, Neutropenia, and Thrombocytopenia)
NCT00410163 (18) [back to overview]Number of Participants With Progression or Death
NCT00412360 (9) [back to overview]Percentage of Participants With Treatment-related Mortality
NCT00412360 (9) [back to overview]Percentage of Participants With Acute Graft-versus-host Disease (GVHD)
NCT00412360 (9) [back to overview]Percentage of Participants With Chronic GVHD
NCT00412360 (9) [back to overview]Percentage of Participants With Neutrophil and Platelet Engraftment
NCT00412360 (9) [back to overview]Time to Neutrophil and Platelet Engraftment
NCT00412360 (9) [back to overview]Percentage of Participants With Disease-free Survival
NCT00412360 (9) [back to overview]Number of Participants With Engraftment Syndrome
NCT00412360 (9) [back to overview]Percentage of Participants With Overall Survival
NCT00412360 (9) [back to overview]Percentage of Participants With Relapse
NCT00416884 (1) [back to overview]Number of Participants With Treatment-related Mortality
NCT00425802 (8) [back to overview]Incidence of Chronic GVHD at 1 Year
NCT00425802 (8) [back to overview]Incidence of Moderate to Severe Grades II to IV Graft Versus Host Disease (GVHD) at 100 Days
NCT00425802 (8) [back to overview]Overall Survival at 1 Year
NCT00425802 (8) [back to overview]Time to Neutrophil Engraftment
NCT00425802 (8) [back to overview]Immune Reconstruction/CD4+ Count at 3 Months
NCT00425802 (8) [back to overview]Time to Platelet Engraftment
NCT00425802 (8) [back to overview]Immune Reconstruction/CD4+ Count at 6 Months
NCT00425802 (8) [back to overview]Immune Reconstruction/CD4+ Count at 1 Year
NCT00427336 (1) [back to overview]Number of Patients With Engraftment Response
NCT00427557 (1) [back to overview]Number of Participants With Engraftment
NCT00427661 (3) [back to overview]Engraftment at 1 Year Post BMT.
NCT00427661 (3) [back to overview]Development of GVHD Within 1 Year of BMT
NCT00427661 (3) [back to overview]Incidence of Grade 2-4 Acute GVHD.
NCT00429416 (5) [back to overview]Number of Patients Who Achieve a CD4 Count > 200/Micro-liters
NCT00429416 (5) [back to overview]Incidence of Grade II-IV Acute Graft-Versus-Host-Disease (GVHD)
NCT00429416 (5) [back to overview]Safety of CD34+ Stem Cell Infusions Followed by LLME as Measured by 100-Day Mortality
NCT00429416 (5) [back to overview]Rate of Serious Infectious Complications
NCT00429416 (5) [back to overview]Rate of Engraftment of Non-Myeloablative Transplants
NCT00429572 (5) [back to overview]Grade II-IV Toxicity
NCT00429572 (5) [back to overview]Time to Progressive Disease
NCT00429572 (5) [back to overview]Overall Survival
NCT00429572 (5) [back to overview]Number of Participants With Tumor Response
NCT00429572 (5) [back to overview]Number of Participants With Acute or Chronic GVHD And Response to Therapy
NCT00448019 (3) [back to overview]Overall Response Rate (ORR) to Fludarabine, Cyclophosphamide, Rituximab, and Bevacizumab Therapy in Previously Treated CLL
NCT00448019 (3) [back to overview]Progression Free Survival (PFS) Rate
NCT00448019 (3) [back to overview]Number of Participants With Complete or Partial Response to Fludarabine, Cyclophosphamide, Rituximab, and Bevacizumab Therapy in Previously Treated Chronic Lymphocytic Leukemia (CLL)
NCT00448201 (4) [back to overview]Complete or Mixed Donor Chimerism at 30, 60, and 90 Days Post-transplant
NCT00448201 (4) [back to overview]Graft-vs-host Disease at 6 Months Post-transplant
NCT00448201 (4) [back to overview]Treatment-related Mortality
NCT00448201 (4) [back to overview]5-year Disease-free Survival
NCT00448357 (6) [back to overview]Incidence of DNA Chimerism in Patients Between One Month Post Transplant
NCT00448357 (6) [back to overview]Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912)
NCT00448357 (6) [back to overview]Overall Survival
NCT00448357 (6) [back to overview]Number of Participants With Dose Limiting Toxicities (DLTs)
NCT00448357 (6) [back to overview]Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen
NCT00448357 (6) [back to overview]Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant
NCT00450983 (4) [back to overview]Risk for Graft Failure
NCT00450983 (4) [back to overview]Risk for Mortality From Infection Before Day 180
NCT00450983 (4) [back to overview]Risk of Developing Grades III-IV Acute Graft-vs-host Disease (GVHD)
NCT00450983 (4) [back to overview]Risk for Life-threatening Infections
NCT00452374 (2) [back to overview]Number of Participants With a Complete Response or Partial Response
NCT00452374 (2) [back to overview]Maximum Tolerated Dose (MTD) Oxaliplatin
NCT00453388 (4) [back to overview]Number of Patients Who Engraft at Each Dose of TBI Used
NCT00453388 (4) [back to overview]Incidence of Transplant-related Mortality
NCT00453388 (4) [back to overview]Incidence of Grades III-IV Acute GVHD
NCT00453388 (4) [back to overview]Incidence of Adverse Events
NCT00455312 (10) [back to overview]Overall Survival
NCT00455312 (10) [back to overview]Overall Survival
NCT00455312 (10) [back to overview]Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD)
NCT00455312 (10) [back to overview]Incidence of Chronic GVHD
NCT00455312 (10) [back to overview]Incidence of Chronic GVHD
NCT00455312 (10) [back to overview]Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD)
NCT00455312 (10) [back to overview]Incidence of Late Secondary Malignancies
NCT00455312 (10) [back to overview]Incidence of Regimen Related Mortality at 100 Days
NCT00455312 (10) [back to overview]Neutrophil Engraftment
NCT00455312 (10) [back to overview]Incidence of Pulmonary Complications
NCT00462332 (2) [back to overview]Length of Survival
NCT00462332 (2) [back to overview]Number of Patients With Complete Response
NCT00469014 (1) [back to overview]Treatment Related Mortality
NCT00469144 (2) [back to overview]3 Year Progression Free Survival
NCT00469144 (2) [back to overview]Treatment-related Mortality (TRM)
NCT00472849 (2) [back to overview]Maximum Total Tolerated Dose (MTD) of Daily Combination Fludarabine 30 mg/m^2 and Cytarabine 500 mg/m^2 Among 3 Dose Levels (Dose Level 1: 2 Days, Dose Level 2: 3 Days or Dose Level 3: 4 Days)
NCT00472849 (2) [back to overview]Overall Response: Number of Participants With Complete Remission, Nodular Partial Remission, and Partial Remission
NCT00475020 (2) [back to overview]Rate of Non-relapse Mortality at 100 Days Post-transplant
NCT00475020 (2) [back to overview]Efficacy of This Therapy 3 Years Post-transplant
NCT00478244 (10) [back to overview]Number of Patients With Acute Graft-Versus-Host Disease (GVHD)
NCT00478244 (10) [back to overview]Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)
NCT00478244 (10) [back to overview]Number of Patients With Detectable Collagen Type VII
NCT00478244 (10) [back to overview]Number of Patients With Donor Derived Cells in Skin
NCT00478244 (10) [back to overview]Number of Patients With Neutrophil Engraftment
NCT00478244 (10) [back to overview]Number of Patients With Platelet Engraftment
NCT00478244 (10) [back to overview]Number of Patients With Resistance to Blister Formation
NCT00478244 (10) [back to overview]Number of Patients With Transplant-Related Mortality
NCT00478244 (10) [back to overview]Number of Patients With >70% Donor Chimerism
NCT00478244 (10) [back to overview]Overall Survival
NCT00480987 (1) [back to overview]Number of Participants With Objective Response
NCT00489281 (3) [back to overview]Transplant-related Mortality
NCT00489281 (3) [back to overview]Donor Chimerism at 1 Year
NCT00489281 (3) [back to overview]Donor Chimerism at 30 Days
NCT00499889 (3) [back to overview]Number of Participants in Complete Molecular Remission at 1 Year
NCT00499889 (3) [back to overview]Participants' With mCR Response to Post Transplant DLI
NCT00499889 (3) [back to overview]Participants' With mCR Response to Post Transplant Imatinib Mesylate Therapy
NCT00502905 (1) [back to overview]Number of Participants With Successful Engraftment
NCT00505895 (2) [back to overview]Number of Participants With Successful Engraftment at Day 100
NCT00505895 (2) [back to overview]Acute Grade II-IV Graft Versus Host Disease (GVHD)
NCT00505921 (1) [back to overview]Participant Progression Free Survival at 2 Years
NCT00506129 (2) [back to overview]Participant's Response According to Physician's Global Assessment of Clinical Condition (PGA)
NCT00506129 (2) [back to overview]Average Overall Survival (OS) Length
NCT00506857 (2) [back to overview]Maximum Tolerated Dose (MTD)
NCT00506857 (2) [back to overview]Number of Participants With Graft Versus Host Disease (GVHD)
NCT00509288 (2) [back to overview]Toxicity
NCT00509288 (2) [back to overview]Clinical Tumor Regression.
NCT00509496 (2) [back to overview]Clinical Tumor Regression.
NCT00509496 (2) [back to overview]Toxicity
NCT00510887 (6) [back to overview]Number of Participants With Neuropathy, Any Grade
NCT00510887 (6) [back to overview]Percentage of Subjects Experiencing Progression Free Survival
NCT00510887 (6) [back to overview]Percentage of Subjects Experiencing Overall Survival
NCT00510887 (6) [back to overview]Number of Participants With a Grade 3-4 Hematologic Toxicity.
NCT00510887 (6) [back to overview]Duration of Response
NCT00510887 (6) [back to overview]Complete and Partial Response
NCT00513474 (3) [back to overview]Number of Participant With Adverse Events (AE)
NCT00513474 (3) [back to overview]Percentage of Participants With Grades II to IV Acute Graft-Versus-Host Disease (aGVHD)
NCT00513474 (3) [back to overview]Uric Acid Levels
NCT00513604 (2) [back to overview]Clinical Response
NCT00513604 (2) [back to overview]Toxicity
NCT00513747 (5) [back to overview]Overall Survival (OS) for High Risk Patients
NCT00513747 (5) [back to overview]Disease-Free Survival in High Risk Patients
NCT00513747 (5) [back to overview]Time to Second Treatment in High Risk Patients
NCT00513747 (5) [back to overview]Time to First Treatment Survival in Low Risk Patients
NCT00513747 (5) [back to overview]Overall Survival in Low Risk Patients
NCT00520130 (17) [back to overview]Percentage of Participants With Grade II-IV Acute Graft Versus Host Disease (GVHD)
NCT00520130 (17) [back to overview]Days to Engraftment of Platelets
NCT00520130 (17) [back to overview]Early Treatment Related Mortality
NCT00520130 (17) [back to overview]Overall Survival
NCT00520130 (17) [back to overview]Percentage of Participants With Grade III-IV Acute Graft Versus Host Disease (GVHD)
NCT00520130 (17) [back to overview]Days to Engraftment of Neutrophils
NCT00520130 (17) [back to overview]Percentage of Participants With Late Treatment Related Mortality
NCT00520130 (17) [back to overview]Toxicities
NCT00520130 (17) [back to overview]Changes in CD8 T Cell Receptor Vbeta Repertoire
NCT00520130 (17) [back to overview]Immune Reconstitution of Cluster of Differentiation 4 (CD4) T Cell Populations
NCT00520130 (17) [back to overview]Immune Reconstitution of Cluster of Differentiation 8 (CD8) T Cell Populations
NCT00520130 (17) [back to overview]Immune Reconstitution of Normal Killer (NK) Cells
NCT00520130 (17) [back to overview]Percentage of Participants With Chronic Graft Versus Host Disease (cGVHD)
NCT00520130 (17) [back to overview]Recovery of Naïve Cluster of Differentiation 4 (CD4) T Cells
NCT00520130 (17) [back to overview]Recovery of Naïve Cluster of Differentiation 8 (CD8) T Cells
NCT00520130 (17) [back to overview]Days to Engraftment of Lymphocytes
NCT00520130 (17) [back to overview]Changes in Cluster of Differentiation 4 (CD4) T Cell Receptor Vbeta Repertoire
NCT00525603 (1) [back to overview]Overall Participant Response
NCT00525876 (1) [back to overview]Overall Survival at 100 Days Post Transplant (Number of Surviving Participants)
NCT00526292 (1) [back to overview]Treatment Efficacy as Defined by Complete or Partial Remission
NCT00536341 (4) [back to overview]Complete Response Rate
NCT00536341 (4) [back to overview]Overall Survival
NCT00536341 (4) [back to overview]Number of Adverse Events as a Measure of Safety and Tolerability
NCT00536341 (4) [back to overview]Progression-Free Survival
NCT00544115 (2) [back to overview]Two-year Overall Survival
NCT00544115 (2) [back to overview]Neutrophil Engraftment - The Days Till ANC Recovery
NCT00544466 (2) [back to overview]Overall Survival on Day 180 Days Post-transplant
NCT00544466 (2) [back to overview]Number of Grade 3 and Above Toxicities of Helical Tomotherapy (HT) in Combination With Fludarabine and Melphalan Followed by Allogeneic Stem Cell Transplantation.
NCT00545714 (14) [back to overview]Percentage of Participants With Immunoglobulin Heavy Locus (IgH) Rearrangement
NCT00545714 (14) [back to overview]Overall Survival (OS)
NCT00545714 (14) [back to overview]Duration of Response (DOR)
NCT00545714 (14) [back to overview]Percentage of Participants With Positive and Negative Zeta-Chain-Associated Protein Kinase 70 (ZAP-70) Expression
NCT00545714 (14) [back to overview]Percentage of Participants With Genetic Abnormalities
NCT00545714 (14) [back to overview]Percentage of Participants With Cluster of Differentiation (CD) 38 Cells >/=30% in Peripheral Blood
NCT00545714 (14) [back to overview]Percentage of Participants With Clinical Response of CR or PR as Assessed by Multiparameter Flow Cytometry
NCT00545714 (14) [back to overview]Percentage of Participants With Clinical Response of CR or PR Among Participants With Negative Minimal Residual Disease (MRD) as Assessed by Multiparameter Flow Cytometry
NCT00545714 (14) [back to overview]Treatment-Free Survival (TFS)
NCT00545714 (14) [back to overview]Progression-Free Survival (PFS)
NCT00545714 (14) [back to overview]Percentage of Participants With CR With Incomplete Bone Marrow Recovery (CRi)
NCT00545714 (14) [back to overview]Percentage of Participants With CR Achieved After the Rituximab, Fludarabine, and Cyclophosphamide Regimen
NCT00545714 (14) [back to overview]Percentage of Participants Who Died
NCT00545714 (14) [back to overview]Percentage of Participants With PD or Death
NCT00547196 (2) [back to overview]Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB)
NCT00547196 (2) [back to overview]Survival Rate at Day 180 After Allogeneic Transplant From Umbilical Cord Blood (UCB)
NCT00553098 (11) [back to overview]Overall Survival
NCT00553098 (11) [back to overview]Number of Patients Who Achieve Greater Than 50% Donor T-cell Chimerism
NCT00553098 (11) [back to overview]Clinical Significant Infection, Requiring Treatment, Within 100 Days Post Transplant
NCT00553098 (11) [back to overview]Immune Reconstitution by 1 Year Post Transplant
NCT00553098 (11) [back to overview]Number of Patients Diagnosed With Overall Grade 1 or Grade 2 Acute GVHD
NCT00553098 (11) [back to overview]Number of Patients Diagnosed With Acute GVHD
NCT00553098 (11) [back to overview]Number of Patients Diagnosed With Overall Grade III or Grade IV Acute GVHD
NCT00553098 (11) [back to overview]Greater Than 50% CD33+ Donor Chimerisms at 1 Year Post Transplant
NCT00553098 (11) [back to overview]Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant
NCT00553098 (11) [back to overview]Disease Response by 1 Year Post Transplant
NCT00553098 (11) [back to overview]Number of Patients Diagnosed With Chronic GVHD
NCT00562965 (7) [back to overview]Percentage of Participants With Objective Response (OR)
NCT00562965 (7) [back to overview]Number of Participants With Grade 3 or 4 Treatment Emergent Adverse Events (TEAEs)
NCT00562965 (7) [back to overview]Overall Survival Probability at Months 6, 12 and 24
NCT00562965 (7) [back to overview]Number of Participants With Clinically Significant Change From Baseline in Laboratory Findings
NCT00562965 (7) [back to overview]Number of Participants With Clinically Significant Change From Baseline in Vital Signs
NCT00562965 (7) [back to overview]Number of Participants With Clinically Significant Change From Baseline in QT Interval Findings
NCT00562965 (7) [back to overview]Progression-Free Survival (PFS)
NCT00566696 (7) [back to overview]To Estimate the Cumulative Incidence of Relapse for Research Participants Who Receive This Study Treatment.
NCT00566696 (7) [back to overview]To Estimate the Rate of Overall Grade III-IV Acute GVHD, and the Rate and Severity of Chronic GVHD in Research Participants.
NCT00566696 (7) [back to overview]Disease-Free Survival (DFS)
NCT00566696 (7) [back to overview]Event-free Survival (EFS)
NCT00566696 (7) [back to overview]Incidence of Regimen-related Mortality
NCT00566696 (7) [back to overview]Overall Survival (OS)
NCT00566696 (7) [back to overview]Incidence of Non-hematologic Regimen-related Toxicities
NCT00572897 (6) [back to overview]Response Outcomes
NCT00572897 (6) [back to overview]Transplant-related Mortality
NCT00572897 (6) [back to overview]The Primary Endpoint is Progression-free Survival.
NCT00572897 (6) [back to overview]PLT
NCT00572897 (6) [back to overview]Absolute Neutrophil Count (ANC)
NCT00572897 (6) [back to overview]Overall Survival
NCT00574496 (3) [back to overview]Disease Relapse or Progression as Measured by CT Scan or PET
NCT00574496 (3) [back to overview]Overall Survival
NCT00574496 (3) [back to overview]Progression-free Survival at 1 Year
NCT00577278 (3) [back to overview]Overall Survival at Two Years
NCT00577278 (3) [back to overview]Relapse/Progression Rate at Two Years
NCT00577278 (3) [back to overview]Progression-free Survival at Two Years
NCT00577993 (2) [back to overview]Number of Participants With Overall Survival (10 Years) by Treatment
NCT00577993 (2) [back to overview]Number of Participants With Progression Free Survival (10 Years) by Treatment
NCT00578292 (8) [back to overview]Immune Reconstitution
NCT00578292 (8) [back to overview]Hematopoietic Reconstitution
NCT00578292 (8) [back to overview]Event-free Survival
NCT00578292 (8) [back to overview]Engraftment Rate After Transplant
NCT00578292 (8) [back to overview]Number of Participants With CHRONIC GVHD
NCT00578292 (8) [back to overview]Number of Participants With Infectious Complications
NCT00578292 (8) [back to overview]Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)
NCT00578292 (8) [back to overview]Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)
NCT00578643 (2) [back to overview]Percentage of Participants With Engraftment
NCT00578643 (2) [back to overview]Number of Patients That Have Complete Donor Chimerism After Transplant.
NCT00579111 (2) [back to overview]Number of Patients With Treatment Related Grade III or IV Non-hematological Toxicity
NCT00579111 (2) [back to overview]Number of Patients With Successful Donor Engraftment
NCT00579137 (4) [back to overview]Patients Alive at 1 Year
NCT00579137 (4) [back to overview]Number of Patients With Grade III to IV Acute GVHD
NCT00579137 (4) [back to overview]Number of Patients With Grade III or IV Toxicity
NCT00579137 (4) [back to overview]Number of Patients With Donor Engraftment
NCT00582894 (4) [back to overview]Number of Participants Experiencing Engraftment Donor Chimerism (EDC)
NCT00582894 (4) [back to overview]Number of Participants Relapse-Free
NCT00582894 (4) [back to overview]Number of Participants Overall Survival as a Function of Time.
NCT00582894 (4) [back to overview]Number of Participants Experiencing Transplant Related Mortality (TRM)
NCT00582933 (1) [back to overview]Death From GVHD
NCT00589316 (1) [back to overview]Two-Year Disease-free Survival of Study Participants Who Completed the Study Regimen
NCT00589563 (14) [back to overview]Overall Survival at Two Years Post HSCT
NCT00589563 (14) [back to overview]Occurrence of Thrombotic Microangiopathy
NCT00589563 (14) [back to overview]Occurence of Sinusoidal Obstructive Syndrome (SOS)
NCT00589563 (14) [back to overview]Non-relapse Mortality at Two Years Post HSCT
NCT00589563 (14) [back to overview]Incidence of Disease Relapse/Progression at 2 Years Post HSCT
NCT00589563 (14) [back to overview]Event Free Survival at Two Years Post HSCT
NCT00589563 (14) [back to overview]Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease (GVHD) at Day 100
NCT00589563 (14) [back to overview]Cumulative Incidence of Chronic GVHD
NCT00589563 (14) [back to overview]Non-relapse Mortality at 100 Days Post HSCT
NCT00589563 (14) [back to overview]Severity of Chronic GVHD
NCT00589563 (14) [back to overview]Severity of Acute GVHD
NCT00589563 (14) [back to overview]Occurence of Infections Including Cytomegalovirus and Epstein-Barr Virus Reactivation
NCT00589563 (14) [back to overview]Time to Platelet Count Recovery (Engraftment)
NCT00589563 (14) [back to overview]Time to Absolute Neutrophil Count Recovery (Engraftment)
NCT00590460 (10) [back to overview]Patients With Treated Related Death
NCT00590460 (10) [back to overview]Patients With Limited Chronic GVHD From Day 100 to 365
NCT00590460 (10) [back to overview]Patients With Grade III - IV Acute GVHD
NCT00590460 (10) [back to overview]Patients With Grade II - IV Acute Graft Versus Host Disease (GVHD)
NCT00590460 (10) [back to overview]Days to Absolute Neutrophil Count (ANC) of 500/mm3
NCT00590460 (10) [back to overview]Patients With Extensive Chronic GVHD From Day 100 to 365
NCT00590460 (10) [back to overview]Number of Patients With Donor Engraftment
NCT00590460 (10) [back to overview]Number of Patients Alive at 1 Year Post Transplant
NCT00590460 (10) [back to overview]Number of Patients With Graft Failure
NCT00590460 (10) [back to overview]Days to Platelet Count of 20,000/mm3 Without Transfusions
NCT00593554 (7) [back to overview]Treatment-related Mortality (TRM) Rate at 6 Months After Transplantation
NCT00593554 (7) [back to overview]Time to Platelet Engraftment
NCT00593554 (7) [back to overview]Acute Graft vs. Host Disease (GvHD)
NCT00593554 (7) [back to overview]Chronic Graft vs. Host Disease (GvHD)
NCT00593554 (7) [back to overview]Frequency of Infection
NCT00593554 (7) [back to overview]Regimen-related Toxicity
NCT00593554 (7) [back to overview]Time to Neutrophil Engraftment
NCT00594308 (5) [back to overview]Number of Patients With Acute Grade II-IV GVHD
NCT00594308 (5) [back to overview]Patients Who Experience Serious Transplant Related Toxicities as Evaluated by Bone Marrow Transplant-adjusted NCI Common Toxicity Criteria.
NCT00594308 (5) [back to overview]Number of Days for Absolute Neutrophil Count to Recover
NCT00594308 (5) [back to overview]Number of Patients Engrafting at Day +30 by Short Tandem Repeat (STR) on Peripheral Blood Mononuclear Cells (PBMC's).
NCT00594308 (5) [back to overview]Time to Resolution of Cytopenias: Platelet Transfusion Independence
NCT00595127 (1) [back to overview]Incidence & Quality of Engraftment & Hematopoietic Reconstitution
NCT00597519 (1) [back to overview]Overall Response
NCT00602459 (6) [back to overview]Time-to-progression in Patients With Del(11q22.3)
NCT00602459 (6) [back to overview]Time-to-progression in Patients Without Del(11q22.3)
NCT00602459 (6) [back to overview]Overall Response Rates in Patients With Del(11q22.3)
NCT00602459 (6) [back to overview]PFS Rate of Patients With Del(11q22.3)
NCT00602459 (6) [back to overview]Overall Response Rate in Patients Without Del(11q22.3)
NCT00602459 (6) [back to overview]2-Year Progression Free Survival (PFS) Rate
NCT00603954 (11) [back to overview]Quality and Timing of Immunologic Reconstitution: Concentration of ATG Levels
NCT00603954 (11) [back to overview]Percentage of 4-year Progression Free Survival in the 2 Groups
NCT00603954 (11) [back to overview]Percentage of 5-year Overall Survival in the 2 Groups
NCT00603954 (11) [back to overview]Percentage of Participants With Chronic GVHD in the 2 Groups
NCT00603954 (11) [back to overview]Percentage of 5-year Progression Free Survival in the 2 Groups
NCT00603954 (11) [back to overview]Number of Participants With Graft Rejection as Defined by Whole Blood and T Cell Chimerism
NCT00603954 (11) [back to overview]Percentage of 4-year Overall Survival in the 2 Groups
NCT00603954 (11) [back to overview]Incidences of Bacterial, Fungal and Viral Infections in the 2 Groups.
NCT00603954 (11) [back to overview]Percentage of Non Relapse Mortality in the 2 Groups
NCT00603954 (11) [back to overview]Percentage of Participants With Grade II-IV Acute GVHD Between the 2 Groups
NCT00603954 (11) [back to overview]Percentage of Relapse Rate in the 2 Groups
NCT00608517 (7) [back to overview]Number of Participants With 100-day Non-relapse Mortality
NCT00608517 (7) [back to overview]Number of Participants With Chronic Graft Versus Host Disease (GVHD)
NCT00608517 (7) [back to overview]Number of Subjects With All-cause Mortality
NCT00608517 (7) [back to overview]Number of Participants With Sustained Donor Engraftment of Umbilical Cord Blood Stem Cells
NCT00608517 (7) [back to overview]Overall Survival
NCT00608517 (7) [back to overview]Number of Participants Who Relapsed at 1 Year
NCT00608517 (7) [back to overview]Number of Participants With Acute Graft-versus-host Disease (GVHD)
NCT00610311 (2) [back to overview]Number of Participants With Metastatic Melanoma Who Develop Clinical Tumor Regression (CR or PR)
NCT00610311 (2) [back to overview]Number of Participants With Adverse Events
NCT00612222 (2) [back to overview]Number of Participants With Adverse Events
NCT00612222 (2) [back to overview]Number of Participants With Metastatic Melanoma Who Develop Clinical Tumor Regression (CR or PR)
NCT00615589 (5) [back to overview]The Percentage of Patients Free From Progression at 1 Year
NCT00615589 (5) [back to overview]Non Relapse Mortality (NRM) at 1 Year and 3 yearsThe Percentage of Deaths Not Attributable to Disease Relapse or Progression
NCT00615589 (5) [back to overview]The Percentage of Patients Alive 1 Year Post Transplant
NCT00615589 (5) [back to overview]Percentage of Patients With Treatment Related Mortality (TRM)
NCT00615589 (5) [back to overview]Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)
NCT00618540 (8) [back to overview]Overall Survival
NCT00618540 (8) [back to overview]Platelet Engraftment
NCT00618540 (8) [back to overview]Incidence of Chronic GVHD
NCT00618540 (8) [back to overview]Transplantation-related Death
NCT00618540 (8) [back to overview]Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)
NCT00618540 (8) [back to overview]Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD)
NCT00618540 (8) [back to overview]Neutrophil Engraftment
NCT00618540 (8) [back to overview]Disease-free Survival at 12 Months Post Transplantation
NCT00619645 (2) [back to overview]Number of Patients With Day 100 Transplant-related Mortality
NCT00619645 (2) [back to overview]Number of Patients Alive 24 Months Post Day 100 Transplant
NCT00622895 (10) [back to overview]Skin Score
NCT00622895 (10) [back to overview]Event-free Survival (EFS)
NCT00622895 (10) [back to overview]EFS
NCT00622895 (10) [back to overview]Incidence of Graft Rejection
NCT00622895 (10) [back to overview]Overall Survival
NCT00622895 (10) [back to overview]The Percent of Participants With Definite and Probable Viral, Fungal, and Bacterial Infections
NCT00622895 (10) [back to overview]Treatment-related Mortality
NCT00622895 (10) [back to overview]Incidence and Severity of Graft-versus-host Disease (GVHD)
NCT00622895 (10) [back to overview]Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36)
NCT00622895 (10) [back to overview]Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ)
NCT00623935 (2) [back to overview]Percentage of Participants Alive at 1 Year
NCT00623935 (2) [back to overview]Percentage of Participants With Relapse Free Survival at 1 Year
NCT00625729 (6) [back to overview]Number of Patients Whose Disease Progressed After Treatment
NCT00625729 (6) [back to overview]Number of Patients Exhibiting Natural Killer Cell Expansion
NCT00625729 (6) [back to overview]Number of Patients With Adequate Natural Killer Cells Infused
NCT00625729 (6) [back to overview]Number of Patients With Interleukin-15 Production and NK Cell Expansion
NCT00625729 (6) [back to overview]Number of Patients With Overall Survival
NCT00625729 (6) [back to overview]Number of Patients With Overall Response
NCT00630253 (5) [back to overview]Number of Participants With Chronic Graft-Versus-Host Disease (GVHD)
NCT00630253 (5) [back to overview]Number of Participants With Transplant Related Deaths
NCT00630253 (5) [back to overview]Number of Participants Experiencing Overall Survival
NCT00630253 (5) [back to overview]Number of Participants With Acute Graft-Versus-Host Disease (GVHD)
NCT00630253 (5) [back to overview]Number of Participants Experiencing Graft Failure
NCT00636155 (3) [back to overview]Progression Free Survival
NCT00636155 (3) [back to overview]Overall Survival
NCT00636155 (3) [back to overview]Number of Patients With an Overall Response (Complete Response + Partial Response)
NCT00652899 (4) [back to overview]Number of Patients With In Vivo Expansion of Infused Allogeneic Natural Killer (NK) Cell Product
NCT00652899 (4) [back to overview]Median Overall Survival Number of Days Patients Alive After Treatment
NCT00652899 (4) [back to overview]Median Number of Days to Progression
NCT00652899 (4) [back to overview]Number of Patients Per Disease Response
NCT00670748 (3) [back to overview]Number of Participants With Serious and Non-Serious Adverse Events
NCT00670748 (3) [back to overview]Clinical Response Per the Response Evaluation Criteria in Solid Tumors (RECIST)
NCT00670748 (3) [back to overview]Number of Participants With In Vivo Survival of T-Cell Receptor (TCR)-Engineered Cells
NCT00683046 (2) [back to overview]Median Disease-free Survival
NCT00683046 (2) [back to overview]Median Overall Survival
NCT00691015 (8) [back to overview]Severity of Acute Graft-versus-host Disease (GVHD)
NCT00691015 (8) [back to overview]Incidence of Acute Graft-versus-host Disease (GVHD)
NCT00691015 (8) [back to overview]Time to Engraftment (i.e., Absolute Neutrophil Recovery [ANC > 500/mm³] )
NCT00691015 (8) [back to overview]Safety, as Defined by Serious Adverse Events and Adverse Events Related to Study Treatment.
NCT00691015 (8) [back to overview]Incidence of Chronic GVHD.
NCT00691015 (8) [back to overview]Incidence of Infections, Including Bacterial, Fungal, and Viral Infections (i.e., CMV and EBV Reactivation, Including Post-transplant Lymphoproliferative Disorders)
NCT00691015 (8) [back to overview]Karnofsky Performance Status Performance Status
NCT00691015 (8) [back to overview]Overall Survival.
NCT00704938 (2) [back to overview]Clinical Response (Complete Response + Partial Response)
NCT00704938 (2) [back to overview]Number of Participants With Adverse Events
NCT00714259 (7) [back to overview]Composite Incidence of Acute and Chronic Graft Versus Host Disease
NCT00714259 (7) [back to overview]Number of Participants With Detectable Donor Chimerism at up to 100 Days Post Transplant
NCT00714259 (7) [back to overview]Progression Free Survival Post Transplant
NCT00714259 (7) [back to overview]Progressive Free Survival Post Transplant
NCT00714259 (7) [back to overview]Progressive Free Survival Post Transplant
NCT00714259 (7) [back to overview]Progressive Free Survival Post Transplant
NCT00714259 (7) [back to overview]Non-relapse Treatment Related Mortality
NCT00719849 (16) [back to overview]Probability of Progression-free Survival at 1 Year
NCT00719849 (16) [back to overview]Probability of Progression-free Survival at 2 Years
NCT00719849 (16) [back to overview]Probability of Survival at 1 Year
NCT00719849 (16) [back to overview]Probability of Survival at 2 Years
NCT00719849 (16) [back to overview]Incidence of Neutrophil Engraftment at Day 42
NCT00719849 (16) [back to overview]Chimerism
NCT00719849 (16) [back to overview]Incidence of Chronic Graft-versus-host-disease (GVHD) at 1 Year
NCT00719849 (16) [back to overview]Incidence of Clinically Significant Infections at 1 Year
NCT00719849 (16) [back to overview]Incidence of Clinically Significant Infections at 6 Months
NCT00719849 (16) [back to overview]Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD) at Day 100
NCT00719849 (16) [back to overview]Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD) at Day 100
NCT00719849 (16) [back to overview]Incidence of Clinically Significant Infections at 2 Years
NCT00719849 (16) [back to overview]Incidence of Non-relapse Mortality at 6 Months
NCT00719849 (16) [back to overview]Incidence of Platelet Engraftment at 6 Months
NCT00719849 (16) [back to overview]Incidence of Relapse at 1 Year
NCT00719849 (16) [back to overview]Incidence of Relapse at 2 Years
NCT00723099 (9) [back to overview]Number of Participants With Graft Failure/Rejection
NCT00723099 (9) [back to overview]Time to Platelet Engraftment of > 20,000 Cells Per mm3
NCT00723099 (9) [back to overview]Percent of Patients With Non-relapse Mortality
NCT00723099 (9) [back to overview]Percent of Patients With Non-relapse Mortality
NCT00723099 (9) [back to overview]Percent of Patients With Grade II-IV Acute Graft Versus Host Disease
NCT00723099 (9) [back to overview]Percent of Patients With Chronic GVHD
NCT00723099 (9) [back to overview]Percent of Patients With Acute GVHD Grades III-IV
NCT00723099 (9) [back to overview]Overall Survival
NCT00723099 (9) [back to overview]Median Time to ANC > 500
NCT00727415 (6) [back to overview]Number of Patients Reaching Disease-free Survival (DSF) Overall
NCT00727415 (6) [back to overview]Maximum Tolerated Dose of Lenalidomide (Phase I)
NCT00727415 (6) [back to overview]Number of Patients With Severe Infections
NCT00727415 (6) [back to overview]Correlation Between Complete Response (CR) and Baseline Biologic Parameters (i.e., IgHV, CD38, Etc.).
NCT00727415 (6) [back to overview]Toxicity as Assessed by NCI CTCAE v3.0
NCT00727415 (6) [back to overview]Overall Complete Response (CR) Rate (Phase II)
NCT00739141 (4) [back to overview]Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients
NCT00739141 (4) [back to overview]Progression Free Survival/PFS at 1 Year Post UCBT.
NCT00739141 (4) [back to overview]Percentage of Participants With Sustained CB-derived Platelet Engraftment
NCT00739141 (4) [back to overview]Percentage of Participants With Sustained CB-derived Neutrophil Engraftment
NCT00741455 (3) [back to overview]Number of Participants With Successful Bone Marrow Engraftment
NCT00741455 (3) [back to overview]Number of Participants Who Achieve Complete Donor Chimerism
NCT00741455 (3) [back to overview]Overall Survival Measured in Participants
NCT00744692 (9) [back to overview]To Describe the Incidence of Grade 3-4 Organ Toxicity
NCT00744692 (9) [back to overview]To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant
NCT00744692 (9) [back to overview]To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure
NCT00744692 (9) [back to overview]To Evaluate the Pace of Immune Reconstitution.
NCT00744692 (9) [back to overview]To Describe the Pace of Neutrophil Recovery
NCT00744692 (9) [back to overview]Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.
NCT00744692 (9) [back to overview]To Describe the Pace of Platelet Recovery
NCT00744692 (9) [back to overview]To Determine the Overall Survival at day180 Post-transplant
NCT00744692 (9) [back to overview]To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)
NCT00759798 (1) [back to overview]Number of Participants With Complete Remission (CR)
NCT00775931 (4) [back to overview]Transplant Related Mortality at 100 Days
NCT00775931 (4) [back to overview]Number of Patients Who Achieved Donor Cell Engraftment
NCT00775931 (4) [back to overview]Incidence of Grade II - IV Acute Graft-versus-host Disease
NCT00775931 (4) [back to overview]Transplant Related Toxicity
NCT00782379 (11) [back to overview]Number of Patients Who Experienced Severe Graft-versus-host Disease (GVHD)(Grade 3 or 4)
NCT00782379 (11) [back to overview]Non-relapse Mortality at Day 100 After Peripheral Blood Stem Cell Transplantation (PBSCT)
NCT00782379 (11) [back to overview]Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 60 Post-transplantation
NCT00782379 (11) [back to overview]Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 90 Post-transplantation
NCT00782379 (11) [back to overview]Disease Free Survival at Day 100
NCT00782379 (11) [back to overview]Overall Survival at 12 Months
NCT00782379 (11) [back to overview]Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 30 Post-transplantation
NCT00782379 (11) [back to overview]Disease Free Survival at 12 Months
NCT00782379 (11) [back to overview]Non-relapse Mortality at 1 Year After Peripheral Blood Stem Cell Transplantation (PBSCT)
NCT00782379 (11) [back to overview]Overall Survival at Day 100
NCT00782379 (11) [back to overview]Incidence of Graft Rejection for Patients at Day 100
NCT00787761 (8) [back to overview]Achievement of > 90% (Full) Donor Chimerism in the T-cell Lineage as Measured by PCR at Day 30 Post-transplantation
NCT00787761 (8) [back to overview]T-cell and Myeloid Chimerism at Days 90 Post-transplantation (>90% Chimerism)
NCT00787761 (8) [back to overview]Disease-free Survival (DFS) at 24 Months
NCT00787761 (8) [back to overview]Number of Patients Experiencing Extensive Chronic Graft Versus Host Disease (GVHD)
NCT00787761 (8) [back to overview]Non-relapse Mortality (NRM) at Day 180 Post-transplantation
NCT00787761 (8) [back to overview]Number of Patients Who Experience Severe (Grade 3 or 4) Acute Graft-versus-host Disease
NCT00787761 (8) [back to overview]Overall Survival (OS) at 24 Months
NCT00787761 (8) [back to overview]T-cell and Myeloid Chimerism at Days 180 Post-transplantation (>90%)
NCT00789776 (7) [back to overview]Number of Participants With Dose Limiting Toxicities
NCT00789776 (7) [back to overview]Number of Participants With Grades III-IV Acute GVHD
NCT00789776 (7) [back to overview]Number of Participants With Relapsed Disease
NCT00789776 (7) [back to overview]Number of Subjects Surviving Post-transplant.
NCT00789776 (7) [back to overview]Number of Participants Who Experienced Chronic Extensive GVHD
NCT00789776 (7) [back to overview]Number of Non-relapse Participant Mortalities
NCT00789776 (7) [back to overview]Number of Participants Who Experienced Graft Failure
NCT00793572 (6) [back to overview]Number of Patients Surviving Overall
NCT00793572 (6) [back to overview]Number of Patients Surviving Progression-free
NCT00793572 (6) [back to overview]Number of Patients With Chronic GVHD
NCT00793572 (6) [back to overview]Number of Patients With Grade II-IV Acute GVHD
NCT00793572 (6) [back to overview]Number of Patients With Toxicities Related to Bortezomib Maintenance Therapy
NCT00793572 (6) [back to overview]Number of Patients With Non-relapse Mortality
NCT00794820 (3) [back to overview]Complete Remission (CR) Rate of FCR3 in Treatment-naïve Participants With Chronic Lymphocytic Leukemia (CLL) at 6 Months
NCT00794820 (3) [back to overview]Overall Survival (OS) Rate
NCT00794820 (3) [back to overview]Remission Duration/Time to Progression (TTP)
NCT00795132 (3) [back to overview]Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments
NCT00795132 (3) [back to overview]Two Year Overall Survival
NCT00795132 (3) [back to overview]Number of Participants Who Experienced Transplantation-related Mortality (TRM)
NCT00796068 (12) [back to overview]Non-relapse Mortality
NCT00796068 (12) [back to overview]Incidence of Clinically Significant Infections
NCT00796068 (12) [back to overview]Incidence of Acute or Chronic Graft-versus-host Disease (GVHD)
NCT00796068 (12) [back to overview]Duration (Days) Until Participants Obtained Platelet Engraftment
NCT00796068 (12) [back to overview]The Number of Participants Alive at Two-years Follow up.
NCT00796068 (12) [back to overview]Number of Patients With Non-relapse Mortality (NRM)
NCT00796068 (12) [back to overview]Number of Participants With Secondary Graft Failure
NCT00796068 (12) [back to overview]Number of Participants With Graft Failure/Rejection
NCT00796068 (12) [back to overview]Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100
NCT00796068 (12) [back to overview]Number of Participants Surviving up to 2 Years Without Disease Progression
NCT00796068 (12) [back to overview]Incidence of Relapse or Disease Progression
NCT00796068 (12) [back to overview]Number of Participants Surviving by 1 Year
NCT00800839 (6) [back to overview]2-year Progression-Free Survival
NCT00800839 (6) [back to overview]Cumulative Incidence of Grade II to IV Acute GVHD
NCT00800839 (6) [back to overview]Day-100 Treatment-Related Mortality
NCT00800839 (6) [back to overview]Rate of Engraftment
NCT00800839 (6) [back to overview]Cumulative Incidence of Grade III to IV Acute GVHD
NCT00800839 (6) [back to overview]2-year Overall Survival
NCT00802113 (7) [back to overview]Time to Neutrophil Engraftment
NCT00802113 (7) [back to overview]Time to Platelet Engraftment
NCT00802113 (7) [back to overview]Total Number of Subjects With a Disease Relapse or Progression Following MAC AutoSCT
NCT00802113 (7) [back to overview]Total Number of Subjects That Experienced Transplant-related Mortality (TRM)
NCT00802113 (7) [back to overview]Total Number of Subjects With a Complete Response (CR) Following Myeloablative Conditioning (MAC) and Autologous Stem Cell Transplantation (AutoSCT)
NCT00802113 (7) [back to overview]Total Number of Subjects With Grade II-IV Acute Graft-versus-Host-Disease (GVHD)
NCT00802113 (7) [back to overview]Total Number of Subjects With Partial Response or Stable Disease Following MAC AutoSCT
NCT00809276 (1) [back to overview]To Determine the Optimal Regimen of Post-graft Immunosuppression With High-dose Cy Following Fludarabine, Busulfan, and Transplantation of Fully HLA-matched Bone Marrow That Leads to an Acceptable Incidence of Grades III/IV Acute GVHD
NCT00813124 (1) [back to overview]Number of Participants With Molecular Response
NCT00818961 (11) [back to overview]Overall Survival at 1 Year
NCT00818961 (11) [back to overview]Number of Patients Experiencing Grade 2-4 Acute Graft-versus-host Disease Post-transplant
NCT00818961 (11) [back to overview]Platelet Engraftment
NCT00818961 (11) [back to overview]Number of Patients Experiencing Chronic Graft Versus Host Disease
NCT00818961 (11) [back to overview]Non-relapse Mortality at Day 100
NCT00818961 (11) [back to overview]Complete Donor Chimerism
NCT00818961 (11) [back to overview]Non-relapse Mortality at 1 Year Post-transplant
NCT00818961 (11) [back to overview]Neutrophil Recovery
NCT00818961 (11) [back to overview]Number of Patients Requiring the Use of Donor Leukocyte Infusion (DLI) for Early Mixed T-cell Chimerism
NCT00818961 (11) [back to overview]Number of Patients Experiencing Veno-occlusive Disease (VOD) Post-transplant
NCT00818961 (11) [back to overview]Survival at Day 100
NCT00822770 (3) [back to overview]Engraftment Response Rate: Number of Transplanted Participants With Complete Chimerism at Day 30
NCT00822770 (3) [back to overview]Number of Participants Alive With no Disease Progression at Time of Allo Transplant
NCT00822770 (3) [back to overview]Number of Participants With Grade 4 Dose Limiting Toxicity to Determine Maximum Tolerated Dose (MTD) Plerixafor
NCT00827099 (2) [back to overview]Number of Participants With 100 Day Transplant-related Mortality (TRM)
NCT00827099 (2) [back to overview]Number of Patients That Engrafted Blood Counts by 30 Days After Transplant
NCT00850499 (2) [back to overview]Overall Response Rate
NCT00850499 (2) [back to overview]Complete Response Rate
NCT00856388 (8) [back to overview]Rate of Complete Donor Chimerism - Blood
NCT00856388 (8) [back to overview]Median Time to Platelet Engraftment
NCT00856388 (8) [back to overview]1 yr Extenstive Chronic GVHD
NCT00856388 (8) [back to overview]3 yr Overall Survival
NCT00856388 (8) [back to overview]Acute GVHD Grade III-IV
NCT00856388 (8) [back to overview]Day 100 TRM
NCT00856388 (8) [back to overview]Median Time to ANC Engraftment
NCT00856388 (8) [back to overview]Rate of Complete Donor Chimerism - Blood
NCT00860457 (1) [back to overview]Complete Response Rate
NCT00860574 (8) [back to overview]Incidence of Chronic GVHD
NCT00860574 (8) [back to overview]Incidence of Grades II-IV Acute GVHD
NCT00860574 (8) [back to overview]Median Donor CD3 + T Lymphocyte Chimerism in Peripheral Blood
NCT00860574 (8) [back to overview]Non Relapse Mortality (NRM) Incidence
NCT00860574 (8) [back to overview]Non Relapse Mortality Incidence
NCT00860574 (8) [back to overview]Relapse-free Survival
NCT00860574 (8) [back to overview]Overall Survival (OS)
NCT00860574 (8) [back to overview]Relapse Incidence
NCT00871689 (8) [back to overview]Number of Patients With Successful Natural Killer Expansion
NCT00871689 (8) [back to overview]Number of Patients With Grade III-IV Acute Graft-Versus-Host (GVHD) Disease
NCT00871689 (8) [back to overview]Number of Patients With Neutrophil Engraftment
NCT00871689 (8) [back to overview]Incidence of Primary Graft Failure
NCT00871689 (8) [back to overview]Median Overall Survival
NCT00871689 (8) [back to overview]Number of Patients With Acute Graft-Versus-Host (GVHD) Disease
NCT00871689 (8) [back to overview]Number of Patients With Complete Remission of Disease
NCT00871689 (8) [back to overview]Number of Patients With Transplant-Related Death (TRD)
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Financial Problems Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Fatigue Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Kaplan Meier Estimate of Time to Treatment Failure (TTF) as Assessed by the Investigator
NCT00875667 (47) [back to overview]Kaplan Meier Estimate of Time to Treatment Failure as Assessed by the Investigator at the Final Analysis
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Constipation
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Diarhoea
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Dyspnoea Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Fatigue Domain
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Financial Problems Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Global Health Status / QoL Domain
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Insomnia Domain
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Nausea / Vomiting Domain
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Pain Domain
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Role Functioning Domain
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Social Functioning Domain
NCT00875667 (47) [back to overview]Number of Participants With Treatment Emergent Adverse Events
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Appetite Loss Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Dyspnoea Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Diarhoea Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Cognitive Functioning Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Pain Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Constipation Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Kaplan Meier Estimate of Time to Progression According to the IRC Central Review
NCT00875667 (47) [back to overview]Kaplan Meier Estimate of Time to First Response as Assessed by the Investigator at the Final Analysis
NCT00875667 (47) [back to overview]Kaplan Meier Estimate of Time to First Response (TTFR) According to the IRC Central Review
NCT00875667 (47) [back to overview]Kaplan Meier Estimate for Progression Free Survival by Investigator's Assessment at the Final Analysis
NCT00875667 (47) [back to overview]Kaplan Meier Estimate for Progression Free Survival (PFS) by Independent Review Committee (IRC) Central Review
NCT00875667 (47) [back to overview]Kaplan Meier Estimate for Overall Survival as Assessed by the Investigator at the Final Analysis
NCT00875667 (47) [back to overview]Kaplan Meier Estimate for Overall Survival (OS) According to the IRC Central Review
NCT00875667 (47) [back to overview]Kaplan Meier Estimate for Duration of Response as Assessed by the Investigator at the Final Analysis
NCT00875667 (47) [back to overview]Kaplan Meier Estimate for Duration of Response (DOR) According to the IRC Central Review
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Cognitive Functioning Domain
NCT00875667 (47) [back to overview]Kaplan Meier Estimate of Time to Progression as Assessed by the Investigator at the Final Analysis
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Appetite Loss Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Percentage of Participants With a Complete Response, Unconfirmed Complete Response, Partial Response and Stable Disease at the Final Analysis
NCT00875667 (47) [back to overview]Percentage of Participants With a Complete Response, Unconfirmed Complete Response, Partial Response and Stable Disease According to the IRC Central Review
NCT00875667 (47) [back to overview]Percentage of Participants Who Achieved an Overall Response as Assessed by the Investigator at the Final Analysis
NCT00875667 (47) [back to overview]Percentage of Participants Who Achieved an Overall Response According to the IRC Central Review
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Social Functioning Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Role Functioning Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Nausea and Vomiting Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Insomnia Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Maximum Change From Baseline in the EORTC QLQ-C30 Global Health Status / QoL Domain to Treatment Discontinuation Visit
NCT00875667 (47) [back to overview]Mean Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
NCT00899431 (1) [back to overview]Percentage of Participants With GVHD (Graft Versus Host Disease)
NCT00914940 (4) [back to overview]Number of Participants Who Did Not Engraft After Receiving a CD45RA+ T Cell Depleted PBSC Transplant
NCT00914940 (4) [back to overview]Number of Participants Who Have Relapsed Within 5 Years of CD45RA+ T Cell Depleted PBSC Transplant
NCT00914940 (4) [back to overview]Number of Participants With Chronic GVHD
NCT00914940 (4) [back to overview]Transplant-related Mortality by Day 100
NCT00918723 (4) [back to overview]To Estimate the Rate of Conversion of Partial Response (PR) to Complete Response (CR) After Fludarabine, Cyclophosphamide and Rituximab (FCR) Plus Vorinostat
NCT00918723 (4) [back to overview]Maximum Tolerated Dose (MTD) of Vorinostat That Can be Combined With Fludarabine Phosphate, Cyclophosphamide and Rituximab (FCR) (Phase I)
NCT00918723 (4) [back to overview]Overall Survival
NCT00918723 (4) [back to overview]Percentage of Patients With Progression-free Survival at 2 Years
NCT00919503 (10) [back to overview]Number of Participants With Infections
NCT00919503 (10) [back to overview]Number of Patients With Grade II-IV Acute Graft-versus-host Disease
NCT00919503 (10) [back to overview]Number of Patients With of Chronic Graft-versus-host Disease
NCT00919503 (10) [back to overview]Overall Survival
NCT00919503 (10) [back to overview]Preliminary Efficacy
NCT00919503 (10) [back to overview]Donor Chimerism CD3 at 100 Days Post Transplant
NCT00919503 (10) [back to overview]Donor Chimerism CD33 at Day 100 Post Transplant
NCT00919503 (10) [back to overview]Immune Reconstitution Following Hematopoietic Cell Transplantation
NCT00919503 (10) [back to overview]Disease Response at One Year Following Hematopoietic Cell Transplantation
NCT00919503 (10) [back to overview]Non-relapse Mortality
NCT00923195 (2) [back to overview]Toxicity Profile
NCT00923195 (2) [back to overview]Complete Response Rates for Patients With Metastatic Melanoma
NCT00923364 (7) [back to overview]Overall Survival
NCT00923364 (7) [back to overview]Incidence of Acute and Chronic Graft-versus-host Disease (GVHD)
NCT00923364 (7) [back to overview]Number of Participants With Serious and Non-serious Adverse Events
NCT00923364 (7) [back to overview]Number of Participants With Disease Free Survival
NCT00923364 (7) [back to overview]Days to Platelet Engraftment
NCT00923364 (7) [back to overview]Days to Neutrophil Engraftment
NCT00923364 (7) [back to overview]Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2)
NCT00924001 (3) [back to overview]Number of Participiants With In-vivo Survival of Infused Cells
NCT00924001 (3) [back to overview]Number of Participants With an Objective Clinical Tumor Regression Response According to RECIST Criteria
NCT00924001 (3) [back to overview]Number of Participants With Adverse Events
NCT00924170 (17) [back to overview]Area Under the Plasma Concentration (AUC) - LMB2
NCT00924170 (17) [back to overview]Duration of Response (Complete Response + Partial Response)
NCT00924170 (17) [back to overview]Half Life (t1/2) of LMB-2
NCT00924170 (17) [back to overview]Number of Participants With Dose Limiting Toxicity (DLT)
NCT00924170 (17) [back to overview]Number of Participants With Serious and Non-serious Adverse Events
NCT00924170 (17) [back to overview]Overall Survival (OS)
NCT00924170 (17) [back to overview]Peak Level of LMB-2 in Adult T-Cell Lymphoma
NCT00924170 (17) [back to overview]Percentage of Patients Who Developed Neutralizing Antibodies After One or More Cycles of LMB-2
NCT00924170 (17) [back to overview]Plasma Clearance (CL) of LMB-2
NCT00924170 (17) [back to overview]Progression Free Survival (PFS)
NCT00924170 (17) [back to overview]Volume of Distribution of LMB-2
NCT00924170 (17) [back to overview]Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2
NCT00924170 (17) [back to overview]Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders
NCT00924170 (17) [back to overview]Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders
NCT00924170 (17) [back to overview]Percentage of Participants With a Minimally Durable Clinical Response Rate
NCT00924170 (17) [back to overview]Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide
NCT00924170 (17) [back to overview]Post Treatment Effects of LMB-2 + Fludarabine and Cyclophosphamide (FC) on Normal B and T Cell Subsets by Flow Cytometry
NCT00924287 (2) [back to overview]Number of Participants With Adverse Events
NCT00924287 (2) [back to overview]Number of Participants With In Vivo Survival of Transfused Cells
NCT00924326 (2) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0).
NCT00924326 (2) [back to overview]Number of Participants With a Response Assessed by the Response Criteria for Malignant Lymphoma
NCT00943319 (3) [back to overview]Maximum Tolerated Dose
NCT00943319 (3) [back to overview]Overall Survival
NCT00943319 (3) [back to overview]Disease Free Survival
NCT00943800 (3) [back to overview]Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
NCT00943800 (3) [back to overview]Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
NCT00943800 (3) [back to overview]Percentage of Participants With Neutrophil Engraftment
NCT00946023 (12) [back to overview]Relapse
NCT00946023 (12) [back to overview]Progression-free Survival
NCT00946023 (12) [back to overview]Graft Failure
NCT00946023 (12) [back to overview]Incidence of Chronic GVHD
NCT00946023 (12) [back to overview]Incidence of Grades II-IV Acute Graft-versus-Host-Disease (GVHD)
NCT00946023 (12) [back to overview]Incidence of Grades III-IV Acute GVHD
NCT00946023 (12) [back to overview]Non-relapse Mortality
NCT00946023 (12) [back to overview]Engraftment
NCT00946023 (12) [back to overview]Overall Survival
NCT00946023 (12) [back to overview]Overall Survival
NCT00946023 (12) [back to overview]Progression-free Survival
NCT00946023 (12) [back to overview]Relapse
NCT00948922 (3) [back to overview]Overall Survival (OS) Rate
NCT00948922 (3) [back to overview]Progression Free Survival (PFS)
NCT00948922 (3) [back to overview]Percentage of Participants With Acute or Chronic Graft-versus-host Disease (GVHD) Following Transplant
NCT00950846 (6) [back to overview]Number of Participants With Chronic GvHD
NCT00950846 (6) [back to overview]Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT00950846 (6) [back to overview]Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT00950846 (6) [back to overview]Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT00950846 (6) [back to overview]Number of Participants With Donor Engraftment After Transplant.
NCT00950846 (6) [back to overview]Incidence of Severe Grade III-IV Acute GvHD at Day 100.
NCT00963872 (20) [back to overview]Incidence of Grades III-IV Graft-vs-host Disease
NCT00963872 (20) [back to overview]Neutrophil Engraftment
NCT00963872 (20) [back to overview]Non-Relapse Mortality
NCT00963872 (20) [back to overview]Non-relapse Mortality
NCT00963872 (20) [back to overview]Number of Patients With the Complement 3a (C3a) Unit Predominating
NCT00963872 (20) [back to overview]Overall Survival
NCT00963872 (20) [back to overview]Overall Survival at Day 720
NCT00963872 (20) [back to overview]Relapse of Disease
NCT00963872 (20) [back to overview]Relapse of Disease
NCT00963872 (20) [back to overview]Incidence of Grades II-IV Graft-vs-host Disease
NCT00963872 (20) [back to overview]Donor Chimerism in Blood
NCT00963872 (20) [back to overview]Donor Chimerism in Blood
NCT00963872 (20) [back to overview]Donor Chimerism
NCT00963872 (20) [back to overview]Donor Chimerism
NCT00963872 (20) [back to overview]Donor Chimerism
NCT00963872 (20) [back to overview]Disease Progression
NCT00963872 (20) [back to overview]Disease Progression
NCT00963872 (20) [back to overview]Chronic Graft-Versus-Host Disease
NCT00963872 (20) [back to overview]Bone Marrow Chimerism
NCT00963872 (20) [back to overview]Platelet Recovery
NCT00987480 (4) [back to overview]The Incidence of Acute GvHD
NCT00987480 (4) [back to overview]The Incidence of Early Transplant Related Mortality
NCT00987480 (4) [back to overview]Overall Survival at 3 Years
NCT00987480 (4) [back to overview]Disease-free Survival at 3 Years
NCT01005576 (4) [back to overview]Incidence of Disease Recurrence
NCT01005576 (4) [back to overview]Median Time to ANC Engraftment
NCT01005576 (4) [back to overview]Median Time to Platelet Engraftment
NCT01005576 (4) [back to overview]Primary Objective: Event-free Survival at 1 Year.
NCT01008462 (7) [back to overview]Number of Patients With Grade II-IV Acute Graft-versus-Host-Disease and/or Chronic Extensive Graft-versus-Host-Disease
NCT01008462 (7) [back to overview]Event-Free Survival (EFS)
NCT01008462 (7) [back to overview]Number of Patients Who Engrafted
NCT01008462 (7) [back to overview]Number of Patients Who Had Infections
NCT01008462 (7) [back to overview]Number of Patients With Relapsed/Progressive Disease
NCT01008462 (7) [back to overview]Overall Survival
NCT01008462 (7) [back to overview]Non-relapse Mortality (NRM)
NCT01010217 (6) [back to overview]Number of Participants With Non-relapse Mortality (NRM)
NCT01010217 (6) [back to overview]Grade III-IV aGVHD
NCT01010217 (6) [back to overview]Disease Free Survival
NCT01010217 (6) [back to overview]cGVHD
NCT01010217 (6) [back to overview]Number of Participants With Non Related Mortality (NRM)
NCT01010217 (6) [back to overview]Engraftments
NCT01019317 (1) [back to overview]Participants With a Complete Response
NCT01027000 (1) [back to overview]2-year Progression-free Survival in Early Disease Participants
NCT01028716 (12) [back to overview]Disease-free Survival
NCT01028716 (12) [back to overview]Point Estimate of Overall Survival at 3 Years
NCT01028716 (12) [back to overview]Percentage of Participants With Chronic Graft Versus Host Disease
NCT01028716 (12) [back to overview]Relapse of Malignancy After Transplantation
NCT01028716 (12) [back to overview]Incidence of Grades III/IV Acute Graft Versus Host Disease
NCT01028716 (12) [back to overview]Time to Neutrophil Recovery
NCT01028716 (12) [back to overview]Time to Platelet Recovery
NCT01028716 (12) [back to overview]Toxicity of Treatment Regimen Determined by Number of Adverse Events Per Organ System
NCT01028716 (12) [back to overview]Incidence of Primary Graft Failure
NCT01028716 (12) [back to overview]Non-relapse Mortality at 1 Year
NCT01028716 (12) [back to overview]Number of Platelet Transfusions
NCT01028716 (12) [back to overview]Number of Red Blood Cell Transfusions
NCT01044745 (4) [back to overview]Transplant-related Mortality (TRM)
NCT01044745 (4) [back to overview]Overall Survival
NCT01044745 (4) [back to overview]Event-free Survival
NCT01044745 (4) [back to overview]Number of Participants With Grades II-IV Acute GVHD
NCT01050764 (6) [back to overview]Serious Infections
NCT01050764 (6) [back to overview]Overall Survival (OS), 1 Year
NCT01050764 (6) [back to overview]Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells
NCT01050764 (6) [back to overview]To Measure the Incidence and Severity of Acute and Chronic GvHD
NCT01050764 (6) [back to overview]Acute Graft-versus-Host-Disease (aGvHD)
NCT01050764 (6) [back to overview]Median Overall Survival (OS)
NCT01082939 (1) [back to overview]Number of Participants With an Overall Response
NCT01088048 (15) [back to overview]Plasma Concentration of IDELA (Cohort 4)
NCT01088048 (15) [back to overview]Plasma Concentration of IDELA (Cohort 6)
NCT01088048 (15) [back to overview]Plasma Concentration of IDELA (Cohort 7)
NCT01088048 (15) [back to overview]Plasma Concentration of IDELA (Cohort 1, Cohorts 2 and 3, Cohort 5)
NCT01088048 (15) [back to overview]Overall Survival
NCT01088048 (15) [back to overview]Progression-free Survival
NCT01088048 (15) [back to overview]Time to Response
NCT01088048 (15) [back to overview]Toxicity of Administration of IDELA
NCT01088048 (15) [back to overview]Duration of Exposure to IDELA
NCT01088048 (15) [back to overview]Plasma Concentration of Lenalidomide
NCT01088048 (15) [back to overview]Sub-study: Plasma Concentration of IDELA (Cohorts 1-4)
NCT01088048 (15) [back to overview]Plasma Concentration of Bendamustine
NCT01088048 (15) [back to overview]Overall Response Rate
NCT01088048 (15) [back to overview]Plasma Concentration of Everolimus
NCT01088048 (15) [back to overview]Duration of Response
NCT01096992 (2) [back to overview]Maximum Tolerated Dose (MTD) of Bendamustine Combined With Fixed-Dose Fludarabine and Rituximab (FBR)
NCT01096992 (2) [back to overview]Overall Response Rate of Bendamustine Combined With Fixed-Dose Fludarabine and Rituximab (FBR)
NCT01105650 (4) [back to overview]Response Rate
NCT01105650 (4) [back to overview]Overall Survival
NCT01105650 (4) [back to overview]Time to Disease Progression
NCT01105650 (4) [back to overview]Number of Participants With Progressive Disease at One Year
NCT01106950 (6) [back to overview]Percent of Patients With Successful Expansion of Natural Killer Cells After Infusion
NCT01106950 (6) [back to overview]Percent of Patients With Natural Killer Cell Expansion Versus KIR Genotype Versus Treg Depletion
NCT01106950 (6) [back to overview]Percent of Patients With Incidence of Relapse
NCT01106950 (6) [back to overview]Percent of Patients With Disease Free Survival
NCT01106950 (6) [back to overview]Percent of Patients With Complete Remission of Disease
NCT01106950 (6) [back to overview]Number of Patients With Treatment-Related Death
NCT01119066 (6) [back to overview]Survival and Disease-free Survival (DFS)
NCT01119066 (6) [back to overview]Survival and Disease-free Survival (DFS)
NCT01119066 (6) [back to overview]The Incidence of Durable Hematopoietic Engraftment for T-cell Depleted Transplants Fractionated by the CliniMACS System Administered After Each of the Four Disease Targeted Cytoreduction Regimens.
NCT01119066 (6) [back to overview]Survival and Disease-free Survival (DFS)
NCT01119066 (6) [back to overview]Number of Participants With Acute and Chronic GVHD Following T-cell Depleted, CD34+ Progenitor Cell Enriched Transplants Fractionated by the CliniMACS System.
NCT01119066 (6) [back to overview]Incidence of Non-relapse Mortality (Transplant-related Mortality) Following Each Cytoreduction Regimen and a Transplant Fractionated by the CliniMACS System.
NCT01131169 (2) [back to overview]Proportion of Participants With Relapsed Multiple Myeloma With Progression-free (PFS)
NCT01131169 (2) [back to overview]Proportion of Participants With Relapsed Multiple Myeloma Alive at 2 Years
NCT01135329 (1) [back to overview]Graft Failure
NCT01144403 (4) [back to overview]Number of Participant With Adverse Event (AE)
NCT01144403 (4) [back to overview]Progression-free Survival (PFS)
NCT01144403 (4) [back to overview]Overall Survival (OS)
NCT01144403 (4) [back to overview]Overall Response Rate (ORR)
NCT01145209 (6) [back to overview]Number of Grade 3 and 4 Treatment Related Adverse Events
NCT01145209 (6) [back to overview]Progression Free Survival Rate 2 Years After Initiation of Induction Therapy
NCT01145209 (6) [back to overview]Participants With MRD Negativity at the Completion of Consolidation Immunotherapy Who Failed to Achieve MRD Negativity
NCT01145209 (6) [back to overview]Participants With Minimal Residual Disease (MRD) Negativity
NCT01145209 (6) [back to overview]Participants With Complete Response Rates Following Induction Chemoimmunotherapy.
NCT01145209 (6) [back to overview]Median Relationship of CD20 Expression With MRD Negativity Rate
NCT01168219 (3) [back to overview]Progression-free Survival
NCT01168219 (3) [back to overview]Overall Survival (OS)
NCT01168219 (3) [back to overview]100-day Mortality
NCT01173679 (2) [back to overview]Toxicities
NCT01173679 (2) [back to overview]Progression-Free and Overall Survival
NCT01181258 (4) [back to overview]Number of Patients With an Objective Response
NCT01181258 (4) [back to overview]Time to Disease Progression
NCT01181258 (4) [back to overview]Serious Adverse Events
NCT01181258 (4) [back to overview]Patients With Expansion of NK Cells
NCT01181271 (12) [back to overview]Cumulative Incidence of Non-relapse Mortality
NCT01181271 (12) [back to overview]Estimated Two Year Overall Survival Rate for All Participants
NCT01181271 (12) [back to overview]Cumulative Incidence of Disease Relapse
NCT01181271 (12) [back to overview]Estimated Two Year Overall Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants
NCT01181271 (12) [back to overview]Estimated Two Year Progression Free Survival Rate for Participants Undergoing Only Autologous Transplant
NCT01181271 (12) [back to overview]Number of Days After Allogeneic Transplant Until Absolute Neutrophil Count Was Equal to or Greater Than 500/uL
NCT01181271 (12) [back to overview]Peripheral Blood All-cell Donor Chimerism
NCT01181271 (12) [back to overview]Estimated Two Year Overall Survival Rate for Participants Undergoing Only Autologous Transplant
NCT01181271 (12) [back to overview]Estimated Two Year Progression Free Survival Rate for All Participants
NCT01181271 (12) [back to overview]Estimated Two Year Progression Free Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants
NCT01181271 (12) [back to overview]Cumulative Incidence of Grades II to IV Acute Graft Versus Host Disease (GVHD)
NCT01181271 (12) [back to overview]Cumulative Incidence of Extensive Chronic Graft-versus-host-disease
NCT01186458 (1) [back to overview]Toxicity
NCT01187017 (4) [back to overview]Number of Patients Who Experienced Disease Relapse
NCT01187017 (4) [back to overview]Participant Survival Following Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia
NCT01187017 (4) [back to overview]Response Rate at 6 Months
NCT01187017 (4) [back to overview]Number of Participants With Clonal Evolution
NCT01203020 (9) [back to overview]Rates of Acute and Chronic Graft Versus Host Disease (GVHD).
NCT01203020 (9) [back to overview]Non-Relapse Mortality (NRM) Following RTC Transplantation at 1 Year.
NCT01203020 (9) [back to overview]Non-Relapse Mortality (NRM) Following RTC Transplantation at 2 Years.
NCT01203020 (9) [back to overview]Relapse Rate (RR) Following Transplantation at 2-year.
NCT01203020 (9) [back to overview]Relapse Rate (RR) Following Transplantation at 1-year.
NCT01203020 (9) [back to overview]Percentage of 1 Year Overall Survival (OS)
NCT01203020 (9) [back to overview]Percentage of 2-year Progression Free Survival (PFS)
NCT01203020 (9) [back to overview]Percentage of 2 Year Overall Survival (OS)
NCT01203020 (9) [back to overview]Percentage of 1-year Progression Free Survival (PFS)
NCT01218867 (2) [back to overview]Number of Participants With a Response to Therapy
NCT01218867 (2) [back to overview]Number of Participants With Serious and Non-Serious Adverse Events
NCT01231412 (6) [back to overview]Number of Non-Relapse Mortalities
NCT01231412 (6) [back to overview]Number of of Participants Surviving Overall
NCT01231412 (6) [back to overview]Number of Participants With Relapse/Progression
NCT01231412 (6) [back to overview]Number of Patients With Chronic Extensive GVHD
NCT01231412 (6) [back to overview]Number of Patients With Grades II-IV Acute GVHD
NCT01231412 (6) [back to overview]Number of Patients With Grades III-IV Acute GVHD
NCT01236573 (3) [back to overview]Maximum Tolerated Dose (MTD)
NCT01236573 (3) [back to overview]Number of Participants With Adverse Events
NCT01236573 (3) [back to overview]Response (Complete Response (CR) + Partial Response (PR)) to Therapy
NCT01247701 (7) [back to overview]Number of Participants With Chronic GvHD
NCT01247701 (7) [back to overview]Number of Participants With Platelet Engraftment
NCT01247701 (7) [back to overview]Number of Participants With Severe Acute GVHD Grade III-IV
NCT01247701 (7) [back to overview]Number of Participants With Donor Engraftment After Transplant.
NCT01247701 (7) [back to overview]Number of Participants With Relapse Rate After Transplant
NCT01247701 (7) [back to overview]Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT01247701 (7) [back to overview]Number of Participants With Neutrophil Engraftment
NCT01251575 (3) [back to overview]Number of Patients With Grade III-IV Acute GVHD
NCT01251575 (3) [back to overview]Number of Non-Relapse Mortalities
NCT01251575 (3) [back to overview]Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD)
NCT01256398 (6) [back to overview]Disease Free Survival (DFS)
NCT01256398 (6) [back to overview]Probability of Being BCR-ABL Negative in the Bone Marrow and Peripheral Blood at the Completion of the CNS Prophylaxis Course (Restricted to Those Patients Achieving a CR)
NCT01256398 (6) [back to overview]Disease Free Survival Defined From the Date of First Induction Complete Response (CR) to Relapse or Death Due to Any Cause
NCT01256398 (6) [back to overview]Overall Survival (OS)
NCT01256398 (6) [back to overview]Feasibility of Maintenance Therapy in This Patient Population (Restricted to Those Patients Achieving a CR). Feasibility Will be Defined as the Number of Deaths Ocuring.
NCT01256398 (6) [back to overview]Response
NCT01263704 (6) [back to overview]Quality of Life (QoL): Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Questionnaire
NCT01263704 (6) [back to overview]Percentage of Participants With Neutropenic Fever, Infection, >/= Grade 3 Drug-Related Neutropenia, >/= Grade 3 Drug-Related Thrombocytopenia, Hospitalizations
NCT01263704 (6) [back to overview]Hospitalization Days
NCT01263704 (6) [back to overview]Overall Response Rate
NCT01263704 (6) [back to overview]Progression-free Survival (PFS)
NCT01263704 (6) [back to overview]Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
NCT01271010 (10) [back to overview]Event-free Survival
NCT01271010 (10) [back to overview]Duration of Response
NCT01271010 (10) [back to overview]Percentage of Participants With Phenotypic Remission
NCT01271010 (10) [back to overview]Progression-free Survival
NCT01271010 (10) [back to overview]Percentage of Participants With Partial Remission
NCT01271010 (10) [back to overview]Percentage of Participants With Disease Progression
NCT01271010 (10) [back to overview]Percentage of Participants With Complete Remission
NCT01271010 (10) [back to overview]Percentage of Participants With Stable Disease
NCT01271010 (10) [back to overview]Overall Survival
NCT01271010 (10) [back to overview]Percentage of Participants With Adverse Events (AEs) and Serious AEs
NCT01271907 (2) [back to overview]Clinical Response
NCT01271907 (2) [back to overview]Safety of Drosophila Generated PBL Administered in Combination With a Lymphodepleting Preparative Regimen and Supportive Systemic Aldesleukin
NCT01273181 (2) [back to overview]Toxicity Profile
NCT01273181 (2) [back to overview]Clinical Tumor Regression (Complete Response (CR) + Partial Response (PR)) in Patients With Metastatic Cancer
NCT01283386 (10) [back to overview]Duration of Response
NCT01283386 (10) [back to overview]Event-free Survival
NCT01283386 (10) [back to overview]Overall Survival
NCT01283386 (10) [back to overview]Percentage of Participants With Complete Remission
NCT01283386 (10) [back to overview]Percentage of Participants With Disease Progression
NCT01283386 (10) [back to overview]Percentage of Participants With Partial Remission
NCT01283386 (10) [back to overview]Percentage of Participants With Phenotypic Remission
NCT01283386 (10) [back to overview]Percentage of Participants With Stable Disease
NCT01283386 (10) [back to overview]Progression-free Survival
NCT01283386 (10) [back to overview]Percentage of Participants With Adverse Events (AEs) and Serious AEs
NCT01289457 (4) [back to overview]Overall Survival
NCT01289457 (4) [back to overview]Response Rates of Clofarabine, Idarubicin, and Cytarabine (CIA) Versus Fludarabine, Idarubicin, and Cytarabine (FLAI)
NCT01289457 (4) [back to overview]Event-Free Survival (EFS) at 2 Years
NCT01289457 (4) [back to overview]Maximum Tolerated Dose (MTD) of Clofarabine, Idarubicin, and Cytarabine
NCT01292603 (15) [back to overview]Part 2: Percentage of Participants With Total B-Cell Depletion by Visit
NCT01292603 (15) [back to overview]Part 1: Total Cluster Differentiation19 Positive (CD19+) B-Cell Counts by Visit
NCT01292603 (15) [back to overview]Part 2: Time to Cmax (Tmax) of Rituximab at Cycle 6
NCT01292603 (15) [back to overview]Part 1: Percentage of Participants and Nurses Recording a Preference For Either SC or IV Administration
NCT01292603 (15) [back to overview]Part 1: Percentage of Participants With Anti-Rituximab Antibodies
NCT01292603 (15) [back to overview]Part 1: Percentage of Participants With Total B-Cell Depletion by Visit
NCT01292603 (15) [back to overview]Part 2: Terminal Half-Life of Rituximab at Cycle 6
NCT01292603 (15) [back to overview]Part 1: Subcutaneous Rituximab Dose Resulting in Trough Concentration (Ctrough) Levels Non-Inferior to Intravenous Rituximab
NCT01292603 (15) [back to overview]Part 2: Physician/Nurse Opinion on Time Savings With Rituximab SC Compared With Rituximab IV
NCT01292603 (15) [back to overview]Part 2: Total CD19+ B-Cell Counts by Visit
NCT01292603 (15) [back to overview]Part 2: Percentage of Participants With Anti-Rituximab Antibodies
NCT01292603 (15) [back to overview]Part 2: Physician/Nurse Opinion on Convenience of Rituximab SC Compared With Rituximab IV
NCT01292603 (15) [back to overview]Part 2: Maximum Observed Concentration (Cmax) of Rituximab at Cycle 6
NCT01292603 (15) [back to overview]Part 2: Observed Area Under the Serum Concentration-Curve (AUC) of Rituximab at Cycle 6
NCT01292603 (15) [back to overview]Part 2: Rituximab C Trough Levels at Cycle 5
NCT01300247 (7) [back to overview]Number of Participants With Human Anti-Human Antibodies (HAHAs)
NCT01300247 (7) [back to overview]Duration of Objective Response (DOR), Assessed by the Investigator According to IWCLL Guidelines
NCT01300247 (7) [back to overview]Percentage of Participants Who Had B-Cell Recovery
NCT01300247 (7) [back to overview]Percentage of Participants Who Had B-Cell Depletion
NCT01300247 (7) [back to overview]Percentage of Participants With Objective Response, Assessed According to International Workshop on Chronic Lymphocytic Leukemia (IWCLL) Guidelines
NCT01300247 (7) [back to overview]Percentage of Participants Who Were Alive and Progression Free
NCT01300247 (7) [back to overview]Percentage of Participants Who Were Alive
NCT01300572 (9) [back to overview]Rates of Non-relapse Mortality
NCT01300572 (9) [back to overview]Estimation of Absorbed Radiation Doses to Normal Organs, Marrow and Tumor
NCT01300572 (9) [back to overview]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.
NCT01300572 (9) [back to overview]Rates of Engraftment
NCT01300572 (9) [back to overview]Rates of Donor Chimerism
NCT01300572 (9) [back to overview]Achievement of Remission
NCT01300572 (9) [back to overview]Duration of Remission
NCT01300572 (9) [back to overview]Disease-free Survival
NCT01300572 (9) [back to overview]Overall Survival
NCT01310179 (2) [back to overview]Number of Participants With Side Effects After Ad/PNP-F-araAMP Treatment
NCT01310179 (2) [back to overview]Treatment Outcome and Percent Change in Tumor Volume
NCT01338987 (4) [back to overview]Percentage of B Cells at One Year Post-transplant in Participants Who Did/Did Not Receive Leuprolide Following Bone Marrow Transplant (BMT)
NCT01338987 (4) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
NCT01338987 (4) [back to overview]Number of Adverse Events Related to Study Drug Experienced by Participants After Second Bone Marrow Transplant (BMT)
NCT01338987 (4) [back to overview]Time to Engraftment in First Transplant Recipients Only With Median Thoracic Spine Standardized Uptake Values (SUV) of 1.4 or Greater Than Those Patients With SUV's Less Than 1.4
NCT01339910 (10) [back to overview]Number of Participants With Primary Graft Failure
NCT01339910 (10) [back to overview]Number of Participants With Donor Cell Engraftment
NCT01339910 (10) [back to overview]Percentage of Participants With Neutrophil and Platelet Engraftment
NCT01339910 (10) [back to overview]Percentage of Participants With Acute Graft Versus Host Disease (GVHD)
NCT01339910 (10) [back to overview]Number of Participants With Secondary Graft Failure
NCT01339910 (10) [back to overview]Percentage of Participants With Relapse-Free Survival (RFS)
NCT01339910 (10) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT01339910 (10) [back to overview]Percentage of Participants With Disease Relapse
NCT01339910 (10) [back to overview]Percentage of Participants With Chronic GVHD
NCT01339910 (10) [back to overview]Percentage of Participants With Treatment-related Mortality
NCT01343043 (8) [back to overview]Time to Maximum Persistence of NY-ESO-1 Genetically Engineered T Cells
NCT01343043 (8) [back to overview]Duration of Overall Response
NCT01343043 (8) [back to overview]Number of Participants With at Least One Confirmed Positive Post-Baseline Anti-infused (NY-ESO-1 Genetically Engineered T) Cell Antibody Result
NCT01343043 (8) [back to overview]Objective Response Rate (ORR)
NCT01343043 (8) [back to overview]Best Overall Response
NCT01343043 (8) [back to overview]Overall Survival
NCT01343043 (8) [back to overview]Number of Participants With Non-serious Adverse Events (Non-SAEs) and Serious Adverse Events (SAEs)
NCT01343043 (8) [back to overview]Progression Free Survival
NCT01366612 (1) [back to overview]To Compare the Relapse Rate at 1 Year of Patients With Myeloid Malignancies Receiving Each Treatment
NCT01369875 (2) [back to overview]Number of Participants With Clinical Tumor Regression.
NCT01369875 (2) [back to overview]Number of Participants With Serious and Non-Serious Adverse Events
NCT01369888 (2) [back to overview]Phase 1: Maximum Tolerated Dose (MTD) of Intravenous Recombinant IL-15 as a Daily Intravenous Bolus for 10 Consecutive Days in Patients With Metastatic Melanoma Who Have Received a Lymphodepleting Chemotherapy and ACT TIL.
NCT01369888 (2) [back to overview]Number of Participants With Adverse Events
NCT01390402 (1) [back to overview]Number of Participants With Molecular Complete Remission at 3 Month Post Transplant
NCT01408563 (12) [back to overview]Relapse-free Survival
NCT01408563 (12) [back to overview]Rates of Grade II-IV and Grade III-IV Acute Graft Versus Host Disease (GVHD) at 100 Days
NCT01408563 (12) [back to overview]Rate of Post-transplant Lymphoma
NCT01408563 (12) [back to overview]Overall Survival
NCT01408563 (12) [back to overview]Median Time to Neutrophil Engraftment
NCT01408563 (12) [back to overview]Number of Participants With Primary Graft Failure
NCT01408563 (12) [back to overview]Median Time to Platelet Engraftment
NCT01408563 (12) [back to overview]Median Thrombopoietin Levels After Transplant
NCT01408563 (12) [back to overview]Immune Reconstitution - Median CD4 Count at 12 Months
NCT01408563 (12) [back to overview]100-day Treatment Related Mortality
NCT01408563 (12) [back to overview]1 Year Relapse Rate
NCT01408563 (12) [back to overview]The Rate of Chronic GVHD
NCT01410344 (7) [back to overview]Percentage of Participants With Acute Graft-Versus-Host Disease (GVHD)
NCT01410344 (7) [back to overview]Percentage of Participants With Relapse/Progression
NCT01410344 (7) [back to overview]Chimerism
NCT01410344 (7) [back to overview]Percentage of Participants Recovering Hematologic Function
NCT01410344 (7) [back to overview]Percentage of Participants With Overall Survival
NCT01410344 (7) [back to overview]Percentage of Participants With Chronic Graft-Versus-Host Disease (GVHD)
NCT01410344 (7) [back to overview]Percentage of Participants With Non-Relapse Mortality
NCT01427881 (9) [back to overview]Chronic GVHD Requiring Systemic Immunosuppressive Treatment
NCT01427881 (9) [back to overview]Grades II-IV and III-IV Acute GVHD
NCT01427881 (9) [back to overview]Persistent or Recurrent Malignancy After HCT
NCT01427881 (9) [back to overview]Overall Survival
NCT01427881 (9) [back to overview]Hematologic Recovery
NCT01427881 (9) [back to overview]Non-relapse Mortality
NCT01427881 (9) [back to overview]Graft Failure
NCT01427881 (9) [back to overview]Donor Engraftment
NCT01427881 (9) [back to overview]Disease-free Survival
NCT01434472 (7) [back to overview]Progression-free Survival
NCT01434472 (7) [back to overview]Overall Survival
NCT01434472 (7) [back to overview]Hematopoietic Toxicity
NCT01434472 (7) [back to overview]Absolute Neutrophil Count (ANC) Engraftment
NCT01434472 (7) [back to overview]Treatment-related Mortality
NCT01434472 (7) [back to overview]Platelet Engraftment
NCT01434472 (7) [back to overview]Response Rates
NCT01445821 (2) [back to overview]Number of Participants With Treatment Failure
NCT01445821 (2) [back to overview]Survival of Treatment
NCT01453101 (3) [back to overview]Overall Response Rate
NCT01453101 (3) [back to overview]Overall Survival (OS)
NCT01453101 (3) [back to overview]Progression Free Survival
NCT01454596 (5) [back to overview]Circulating Chimeric Antigen Receptor (CAR+) Cells in Peripheral Blood at 1 Month Post Treatment
NCT01454596 (5) [back to overview]Number of Patients With an Objective Response
NCT01454596 (5) [back to overview]Number of Treatment Related Adverse Events
NCT01454596 (5) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
NCT01454596 (5) [back to overview]Progression Free Survival
NCT01464359 (9) [back to overview]Incidence of Graft Failure
NCT01464359 (9) [back to overview]Incidence of Acute Graft-Versus-Host Disease
NCT01464359 (9) [back to overview]Duration of Survival
NCT01464359 (9) [back to overview]Duration of Survival
NCT01464359 (9) [back to overview]Duration of Survival
NCT01464359 (9) [back to overview]Disease Free Survival
NCT01464359 (9) [back to overview]Clinical Disease Response
NCT01464359 (9) [back to overview]Clinical Disease Response
NCT01464359 (9) [back to overview]Transplant-Related Mortality
NCT01468818 (1) [back to overview]Objective Response in Patients With Metastatic Melanoma
NCT01471444 (4) [back to overview]Overall Survival (OS) Post Transplant at 1, 3 and 5 Years
NCT01471444 (4) [back to overview]Number of Participants in the Study Who Are With no Grade 3 or 4 Acute Graft-versus-host Disease at Any Time During the First 100 Days Post Transplant.
NCT01471444 (4) [back to overview]Number of Participants With Non Relapse Mortality at 100 Day Post Transplant
NCT01471444 (4) [back to overview]Progression-Free Survival (PFS)
NCT01490723 (2) [back to overview]Treatment-Related Mortality (TRM)
NCT01490723 (2) [back to overview]Overall Survival (OS)
NCT01495572 (2) [back to overview]Number of Participants With Adverse Events
NCT01495572 (2) [back to overview]Clinical Tumor Response
NCT01499147 (4) [back to overview]Participants With 100 Day Transplant-related Mortality.
NCT01499147 (4) [back to overview]Number of Participants With Engraftment.
NCT01499147 (4) [back to overview]Number of Participants With Moderate to Severe (Grade 2-4) Acute Graft Versus Host Disease (GVHD).
NCT01499147 (4) [back to overview]Time to ANC and Platelet Engraftment
NCT01518153 (2) [back to overview]Overall Survival (OS)
NCT01518153 (2) [back to overview]Success Rate
NCT01527045 (8) [back to overview]Number of Patients With Grades II-IV Acute Graft-versus-host-disease (GVHD)
NCT01527045 (8) [back to overview]Number of Patients Requiring Secondary Systemic Immunosuppressive Therapy
NCT01527045 (8) [back to overview]Number of Non-relapse Mortalities
NCT01527045 (8) [back to overview]Number of Donors Discontinuing Atorvastatin Due to Toxicity
NCT01527045 (8) [back to overview]Number of Patients With Recurrent or Progressive Malignancy
NCT01527045 (8) [back to overview]Number of Patients With Grade III-IV Acute Graft-versus-host Disease (GVHD) Post-transplant
NCT01527045 (8) [back to overview]Number of Patients With Chronic Extensive GVHD
NCT01527045 (8) [back to overview]Number of Patients Surviving Overall
NCT01529827 (4) [back to overview]Progression Free Survival (PFS) at One Year
NCT01529827 (4) [back to overview]Median Time to Neutrophil Engraftment
NCT01529827 (4) [back to overview]Clinical Response
NCT01529827 (4) [back to overview]Transplant Related Mortality (TRM)
NCT01564784 (12) [back to overview]Duration of Remission (DoR) for Participants Who Achieved CR/CRi (Per Investigator Assessment)
NCT01564784 (12) [back to overview]Maximum Observed Inotuzumab Ozogamicin Serum Concentration (Cmax) and Pre-Dose Inotuzumab Ozogamicin Serum Concentration (Ctrough) Following Single and Multiple Dosing
NCT01564784 (12) [back to overview]Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, Core 30 (EORTC QLQ-C30) Score
NCT01564784 (12) [back to overview]Change From Baseline in EQ-5D VAS
NCT01564784 (12) [back to overview]Progression-Free Survival (PFS)
NCT01564784 (12) [back to overview]Percentage of Participants With Veno-Occlusive Liver Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS) Following Post Study HSCT
NCT01564784 (12) [back to overview]Percentage of Participants With Hematologic Remission (Complete Remission [CR]/Complete Remission With Incomplete Hematologic Recovery [CRi]) as Assessed by the Endpoint Adjudication Committee (EAC)
NCT01564784 (12) [back to overview]Change From Baseline in EuroQol 5 Dimension Health Questionnaire (EQ-5D) Index Score
NCT01564784 (12) [back to overview]Overall Survival (OS)
NCT01564784 (12) [back to overview]Percentage of Participants Who Had a Hematopoietic Stem-Cell Transplant (HSCT)
NCT01564784 (12) [back to overview]Cytogenetic Status (Based on Local Laboratory Analysis) of Participants With CR/CRi (Per EAC Assessment)
NCT01564784 (12) [back to overview]Percentage of Participants Achieving MRD Negativity (Based on Central Laboratory Analysis) in Participants Achieving a CR/CRi (Per EAC Assessment)
NCT01565616 (8) [back to overview]Graft Failure
NCT01565616 (8) [back to overview]Chronic Graft Versus Host Disease (GVHD)
NCT01565616 (8) [back to overview]Time to Neutrophil and Platelet Engraftment
NCT01565616 (8) [back to overview]PROMIS-57 Scores Health Related Quality of Life
NCT01565616 (8) [back to overview]Event -Free Survival Rate
NCT01565616 (8) [back to overview]Transplant Related Outcomes
NCT01565616 (8) [back to overview]Acute Graft Versus Host Disease (GVHD)
NCT01565616 (8) [back to overview]Overall Survival
NCT01570348 (11) [back to overview]EFS
NCT01570348 (11) [back to overview]Event-free Survival (EFS)
NCT01570348 (11) [back to overview]Incidence and Severity of GVHD
NCT01570348 (11) [back to overview]Incidence of Disease-modifying Drugs for CD Initiated Post-transplant
NCT01570348 (11) [back to overview]Overall Survival
NCT01570348 (11) [back to overview]Quality of Life Measured Using the Previously Validated Short Inflammatory Bowel Disease Questionnaire
NCT01570348 (11) [back to overview]Regimen-related Toxicity Graded According to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4
NCT01570348 (11) [back to overview]Treatment-related Mortality (TRM)
NCT01570348 (11) [back to overview]Incidence of Graft Rejection
NCT01570348 (11) [back to overview]Development of Infectious Complications
NCT01570348 (11) [back to overview]Disease Activity
NCT01572662 (3) [back to overview]Overall Survival
NCT01572662 (3) [back to overview]Overall Survival
NCT01572662 (3) [back to overview]Non-Relapse Mortality Rate (NRM)
NCT01583686 (1) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
NCT01585428 (1) [back to overview]Number of Patients With Serious and Non-serious Adverse Events
NCT01596699 (3) [back to overview]Number of Participants With Treatment-Related Adverse Events as a Measure of Safety and Tolerability
NCT01596699 (3) [back to overview]Engraftment Rate of Patients With Non-malignant Diseases (Stratum A)
NCT01596699 (3) [back to overview]Mixed-donor Chimerism Rate of Patients With High-risk Myeloid Malignancies (Stratum B)
NCT01640301 (11) [back to overview]Treatment-related Toxicity Rate (Arm II)
NCT01640301 (11) [back to overview]Treatment-related Toxicity Rate (Arm I)
NCT01640301 (11) [back to overview]Maintenance of T Cell Receptor (TCR) Expression of Transduced T Cells (Arm I) i.e. Persistence
NCT01640301 (11) [back to overview]Maintenance of Function of Transduced T Cells (Arm I)
NCT01640301 (11) [back to overview]Incidence of Relapse After T Cell Therapy (Arm II)
NCT01640301 (11) [back to overview]Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm I)
NCT01640301 (11) [back to overview]Anti-leukemic Potential Efficacy, in Terms of Duration of Response (Arm II)
NCT01640301 (11) [back to overview]Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm II)
NCT01640301 (11) [back to overview]Count of Participants Who Experienced Grade III-IV Acute Graft Versus Host Disease (GVHD) (Arm II)
NCT01640301 (11) [back to overview]Count of Participants Who Experienced Grade III-IV Acute Graft-versus-host Disease (GVHD) (Arm I)
NCT01640301 (11) [back to overview]Disease-free Survival After T Cell Therapy
NCT01659151 (3) [back to overview]Number of Participants With Progression Free Survival (PFS)
NCT01659151 (3) [back to overview]Percentage of Participants With Overall Response (OR)
NCT01659151 (3) [back to overview]Percentage of Participant Drop Out Rate
NCT01690520 (6) [back to overview]Time to Platelet Engraftment (20k)
NCT01690520 (6) [back to overview]Time to Neutrophil Engraftment
NCT01690520 (6) [back to overview]Overall Survival
NCT01690520 (6) [back to overview]Proportion of Patients With Severe Acute Graft Versus Host Disease
NCT01690520 (6) [back to overview]Proportion of Participants With Chronic Graft Versus Host Disease
NCT01690520 (6) [back to overview]Non-relapse Mortality
NCT01701674 (4) [back to overview]Occurrence of Dose Limiting Toxicity (DLT) Events
NCT01701674 (4) [back to overview]Rate of Meeting Feasibility Requirements
NCT01701674 (4) [back to overview]Progression Free Survival (PFS)
NCT01701674 (4) [back to overview]Overall Response Rate (ORR)
NCT01707004 (8) [back to overview]Progression Free Survival
NCT01707004 (8) [back to overview]Percentage of Participants With Platelet Recovery by Day 30
NCT01707004 (8) [back to overview]Time to Neutrophil Recovery
NCT01707004 (8) [back to overview]Cumulative Incidence of Chronic GVHD According to BMTCTN
NCT01707004 (8) [back to overview]Cumulative Incidence of Grade III-IV Acute GVHD
NCT01707004 (8) [back to overview]Number of Participants With Complete Remission After Transplantation
NCT01707004 (8) [back to overview]Number of Participants With Primary Graft Failure
NCT01707004 (8) [back to overview]Overall Survival (OS)
NCT01723839 (2) [back to overview]Overall Response Rate
NCT01723839 (2) [back to overview]Complete Response
NCT01749293 (3) [back to overview]Number of Participants That Engrafted and the Number of Participants That Had Full Donor Chimerism at Day 60
NCT01749293 (3) [back to overview]Number of Participants That Had an Overall Survival Rate
NCT01749293 (3) [back to overview]Number of Participants That Had an Event Free Survival Rate
NCT01773395 (5) [back to overview]18-Month Overall Survival
NCT01773395 (5) [back to overview]Percentage of Participants Experiencing Grade 3 or Higher Non-Hematologic or Grade 4 or Higher Hematologic Adverse Events
NCT01773395 (5) [back to overview]Percentage of Participants Experiencing Acute and Chronic Graft-Versus-Host Disease
NCT01773395 (5) [back to overview]Percentage of Participants With Relapse and/or Non-Relapse Mortality
NCT01773395 (5) [back to overview]18-Month Progression Free Survival
NCT01807182 (3) [back to overview]Count of Participants Who Experienced Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0
NCT01807182 (3) [back to overview]Number of Participants Who Experienced in Vivo Persistence of Adoptively Transferred T Cells Following TIL Infusion
NCT01807182 (3) [back to overview]A Count of Participants With Biomarker Expression Above Threshold
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Successful Engraftment
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Transplant-related Mortality (TRM)
NCT01807611 (6) [back to overview]Overall Survival
NCT01807611 (6) [back to overview]Event-free Survival
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD)
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Malignant Relapse
NCT01814046 (3) [back to overview]Percentage of Participants With Ocular Melanoma Treated With Young Tumor Infiltrating Lymphocytes (TIL) With or Without High Dose Aldesleukin With an Objective Response Rate of (Complete Response (CR) + Partial Response (PR))
NCT01814046 (3) [back to overview]Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0)
NCT01814046 (3) [back to overview]Count of Participants With Changes in Visual Symptoms
NCT01823198 (3) [back to overview]Overall Survival
NCT01823198 (3) [back to overview]Number of Participants With Grade 3 Toxicities
NCT01823198 (3) [back to overview]Number of Participants Who Experienced Dose-limiting Toxicities (DLT)
NCT01824693 (4) [back to overview]Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
NCT01824693 (4) [back to overview]Percent Probability of Event-free Survival (EFS)
NCT01824693 (4) [back to overview]Percent Probability of 18 Months-relapse Event Between Arms
NCT01824693 (4) [back to overview]Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100
NCT01861002 (1) [back to overview]Number of Participants Who Experienced a Dose Limiting Toxicity (DLT)
NCT01875237 (8) [back to overview]To Assess the Proportions of GvHD Response Post-administration of AP1903.
NCT01875237 (8) [back to overview]To Assess the Proportion of Patients Developing Grade I-IV Acute GvHD
NCT01875237 (8) [back to overview]To Assess the Incidence of GvHD Treatment Failure Post-administration of AP1903.
NCT01875237 (8) [back to overview]To Evaluate the Safety of Donor Lymphocyte Infusion Followed by Dimerizer Drug, AP1903 by Number of Participants With Adverse Events.
NCT01875237 (8) [back to overview]To Assess Post Donor Lymphocyte Infusion (DLI) Chimerism
NCT01875237 (8) [back to overview]To Assess the Incidence of Acute GvHD Flare After CR/PR Requiring Additional Agent for Systemic Therapy Before Day 56 Post-administration of AP1903.
NCT01875237 (8) [back to overview]To Assess the Incidence of Epstein-Barr Virus -PTLD or EBV Reactivation Requiring Therapy Post DLI.
NCT01875237 (8) [back to overview]Number of Participants Assessed Post Donor Lymphocyte Infusion (DLI): Disease-free Survival & Non-relapse Mortality, Chimerism and GVHD.
NCT01877837 (2) [back to overview]Overall Survival
NCT01877837 (2) [back to overview]Number of Participants With Graft Failure
NCT01894477 (2) [back to overview]Number of Participants With Acute GVHD, Graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
NCT01894477 (2) [back to overview]Number of Participants That Did Not Progress Within 6 Months
NCT01898793 (8) [back to overview]Duration of Remission (DOR) (Phase I, Phase II, and Pediatric)
NCT01898793 (8) [back to overview]Maximal Tolerated or Tested Dose (MT/TD) of CIML-NK Cells (Phase I)
NCT01898793 (8) [back to overview]Time to Progression (Phase I, Phase II, and Pediatric)
NCT01898793 (8) [back to overview]Safety of CIML NK Cells as Measured by the Number of Participants With Treatment Related Adverse Events (Pediatric Cohort)
NCT01898793 (8) [back to overview]Toxicity as Measured by the Number of Participants With Treatment Related Adverse Events (Phase I and Phase II)
NCT01898793 (8) [back to overview]Complete Remission Rate (CR/CRi) in Participants With Relapsed or Refractory AML Following CIML NK Therapy (Phase II)
NCT01898793 (8) [back to overview]Overall Survival (OS) (Phase I, Phase II, and Pediatric)
NCT01898793 (8) [back to overview]Disease Free Survival (DFS) (Phase I, Phase II, and Pediatric)
NCT01905943 (12) [back to overview]Number of Participants With Adverse Events of Special Interest (AESIs)
NCT01905943 (12) [back to overview]Number of Participants With Adverse Events of Particular Interest (AEPIs)
NCT01905943 (12) [back to overview]Number of Participants With Adverse Events (AEs)
NCT01905943 (12) [back to overview]Percentage of Participants With Overall Response (OR) at Final Response Assessment (FRA)
NCT01905943 (12) [back to overview]Percentage of Participants With Best Overall Response (BOR)
NCT01905943 (12) [back to overview]Median Time to Response (TTR)
NCT01905943 (12) [back to overview]Median Time to Progression-Free Survival (PFS)
NCT01905943 (12) [back to overview]Median Time to Overall Survival (OS)
NCT01905943 (12) [back to overview]Median Time to Duration of Response (DoR)
NCT01905943 (12) [back to overview]Median Time to New Anti-Leukemia Therapy (TTNT)
NCT01905943 (12) [back to overview]Percentage of Participants With Minimal Residual Disease (MRD)-Negativity as Assessed by Flow Cytometry
NCT01905943 (12) [back to overview]Median Time to Event-Free Survival (EFS)
NCT01967823 (6) [back to overview]Percentage of Participants With a Response
NCT01967823 (6) [back to overview]Number of Participants With Serious and Non-serious Treatment Related Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)
NCT01967823 (6) [back to overview]Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells
NCT01967823 (6) [back to overview]Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells
NCT01967823 (6) [back to overview]Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells
NCT01967823 (6) [back to overview]Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells
NCT01991457 (1) [back to overview]Number of Subjects Disease-free Survival
NCT01993719 (13) [back to overview]Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)
NCT01993719 (13) [back to overview]Overall Response Rate (ORR)
NCT01993719 (13) [back to overview]Overall Response Rate (ORR)
NCT01993719 (13) [back to overview]Progression-free Survival (PFS)
NCT01993719 (13) [back to overview]Number of Participants Who Have a Clinical Response to Treatment (Objective Tumor Regression)
NCT01993719 (13) [back to overview]Overall Response Rate (ORR)
NCT01993719 (13) [back to overview]Number of Treatment-related Adverse Events for Participants Who Received Pembrolizumab
NCT01993719 (13) [back to overview]Progression-free Survival (PFS)
NCT01993719 (13) [back to overview]Progression-free Survival (PFS)
NCT01993719 (13) [back to overview]Overall Progression Free Survival (PFS)
NCT01993719 (13) [back to overview]Overall Survival
NCT01993719 (13) [back to overview]Overall Survival
NCT01993719 (13) [back to overview]Number of Participants With Treatment-related Grade 3-5 Adverse Events in Arm 1N and Arm 1P
NCT02007863 (1) [back to overview]Number of Successful Unrelated Cord Blood (UCB) Transplants
NCT02013817 (6) [back to overview]Percentage of Participants With the Best Clinical Response by Visit (Clinical Assessment)
NCT02013817 (6) [back to overview]Percentage of Participants With the Best Clinical Response by Visit (Clinical + Radiological Assessment)
NCT02013817 (6) [back to overview]Time to Next Treatment - Percentage of Participants With an Event
NCT02013817 (6) [back to overview]Time to Next Treatment - Time to Event
NCT02013817 (6) [back to overview]Percentage of Participants With a Best Clinical Response of Clinical Remission (CR)
NCT02013817 (6) [back to overview]Percentage of Participants With Adverse Events (AEs)
NCT02029638 (18) [back to overview]Number of Transplanted Participants With Engraftment Syndrome
NCT02029638 (18) [back to overview]Number of Transplanted Participants With Chronic T Cell-Mediated or Antibody-Mediated Rejection
NCT02029638 (18) [back to overview]Number of Adverse Events (AEs) by Severity- Including Infection, Wound Complications, Post-Transplant Diabetes, Hemorrhagic Cystitis and Malignancy
NCT02029638 (18) [back to overview]Number of Participants Free From Return to Immunosuppression for the Duration of the Study
NCT02029638 (18) [back to overview]Number of Adverse Events (AEs)- Including Infection, Wound Complications, Post-transplant Diabetes, Hemorrhagic Cystitis and Malignancy
NCT02029638 (18) [back to overview]Number of Participants Experiencing an Incidence of Graft-versus-Host Disease Post-Transplant
NCT02029638 (18) [back to overview]Number of Days Post-Transplant to the First Episode of Acute Rejection Requiring Treatment
NCT02029638 (18) [back to overview]Number of Days From Transplant to Platelet Count Recovery
NCT02029638 (18) [back to overview]Number of Days From Neutrophil Nadir to Absolute Neutrophil Recovery
NCT02029638 (18) [back to overview]Duration in Days of Graft-versus-Host Disease in Transplanted Participants
NCT02029638 (18) [back to overview]Histological Severity of Biopsies Demonstrating Acute Rejection as Defined by Banff 2007 Classification Renal Allograft Pathology
NCT02029638 (18) [back to overview]Duration in Days of Adverse Events (AEs)- Including Infection, Wound Complications, Post-transplant Diabetes, Hemorrhagic Cystitis and Malignancy
NCT02029638 (18) [back to overview]Number of Transplanted Participants With Acute Renal Allograft Rejection
NCT02029638 (18) [back to overview]Number of Transplanted Participants Who Died
NCT02029638 (18) [back to overview]Number of Transplanted Participants Who Developed Donor-Specific Antibody During Study Participation
NCT02029638 (18) [back to overview]Number of Transplanted Participants Who Developed Donor- Specific Antibody After Initiation of Immunosuppression Withdrawal
NCT02029638 (18) [back to overview]Percent of Participants Who Achieved Operational Tolerance
NCT02029638 (18) [back to overview]Number of Transplanted Participants Who Remained Off Immunosuppression for at Least 52 Weeks, Including Those in Whom the 52 Week Biopsy Was Not Performed
NCT02048813 (2) [back to overview]Overall Survival (OS) Rate at 3 Years
NCT02048813 (2) [back to overview]Progression-free Survival (PFS) Rate at 3 Years
NCT02062359 (1) [back to overview]Number of Participants With Adverse Events
NCT02080195 (5) [back to overview]Acute Graft Versus Host Disease (GVHD)
NCT02080195 (5) [back to overview]Survival
NCT02080195 (5) [back to overview]Graft Failure
NCT02080195 (5) [back to overview]The Feasibility of the Conditioning Regimen and Post Transplantation Cyclophosphamide in Refractory SLE Patients With Donors Having Various Degrees of Matching
NCT02080195 (5) [back to overview]Chronic Graft Versus Host Disease (GVHD)
NCT02111850 (6) [back to overview]Maximum Tolerated Cell Dose (MTD) of Cluster of Differentiation 4 (CD4) Cells Transduced With an Anti-MAGE-A3-DP0401/0402 Restricted (MAGE-A3-DP4) T Cell Receptor and Aldesleukin
NCT02111850 (6) [back to overview]Number of Engineered T Cell Receptor (TCR) Cells That Survived at 4 Weeks
NCT02111850 (6) [back to overview]Number of Participants With Dose-limiting Toxicity (DLT)
NCT02111850 (6) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0).
NCT02111850 (6) [back to overview]Number of Adverse Events With Grades ≥1 That Are Possibly, Probably, and/or Definitely Related to Treatment
NCT02111850 (6) [back to overview]Percentage of Participants Who Have a Clinical Response to Treatment (Objective Tumor Regression)
NCT02111863 (3) [back to overview]Count of Participants With Serious and Non-Serious Adverse Events
NCT02111863 (3) [back to overview]Overall Survival (OS) of Patients Receiving a Lymphocyte Depleting Preparative Regimen
NCT02111863 (3) [back to overview]Objective Response Rate of Patients With Metastatic Melanoma
NCT02145039 (4) [back to overview]Number of Patients With Hematopoietic Engraftment
NCT02145039 (4) [back to overview]2 Year Survival
NCT02145039 (4) [back to overview]Number of Patients Experiencing Acute Graft-versus-host Disease by 100 Days
NCT02145039 (4) [back to overview]Number of Patients Experiencing Transplant Related Mortality (TRM)
NCT02153905 (4) [back to overview]Number of Patients With Objective Tumor Regression
NCT02153905 (4) [back to overview]Number of Treatment Related Adverse Events Related to T-Cell Receptor (TCR) Gene-Engineered Cells
NCT02153905 (4) [back to overview]Number of Participants With Dose-Limiting Toxicity (DLT)
NCT02153905 (4) [back to overview]Number of Participants With Serious and Non-Serious Adverse Events
NCT02158091 (2) [back to overview]Number of Patients Who Had a Minimal Residual Disease (MRD) Negative Complete Response (CR) 2 Months After Chemotherapy
NCT02158091 (2) [back to overview]Number of Patients Who Experienced a Dose Limiting Toxicity (DLT) During Phase I
NCT02199041 (9) [back to overview]Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
NCT02199041 (9) [back to overview]Number of Participants With Neutrophil Engraftment
NCT02199041 (9) [back to overview]Number of Participants With Overall Survival (OS)
NCT02199041 (9) [back to overview]Number of Participants With Secondary Graft Failure
NCT02199041 (9) [back to overview]Number of Participants With Transplant-related Morbidity
NCT02199041 (9) [back to overview]Number of Participants With Malignant Relapse
NCT02199041 (9) [back to overview]Number of Participants With Transplant-related Mortality (TRM)
NCT02199041 (9) [back to overview]Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
NCT02199041 (9) [back to overview]Number of Participants With Event-free Survival (EFS)
NCT02215967 (3) [back to overview]Number of Participants With Best Response
NCT02215967 (3) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
NCT02215967 (3) [back to overview]Number of Participants With Dose Limiting Toxicities
NCT02224872 (11) [back to overview]Number of Patients With Primary or Secondary Graft Failure Following Transplant
NCT02224872 (11) [back to overview]Number of Patients That Expired Due to Non-relapsed-related Mortality Following Transplant
NCT02224872 (11) [back to overview]Participants With Chronic GVHD at One Year
NCT02224872 (11) [back to overview]Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant
NCT02224872 (11) [back to overview]Number of Participants With Grade II-IV or Grade III-IV Acute GVHD
NCT02224872 (11) [back to overview]Participants That Were GVHD Free, Relapse Free Survival (GRFS)
NCT02224872 (11) [back to overview]Number of Patients That Have Survived at One Year
NCT02224872 (11) [back to overview]Number of Patients That Expired Due to Transplant Related Mortality
NCT02224872 (11) [back to overview]Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant
NCT02224872 (11) [back to overview]Number of Participants With Major Toxicities Related to Transplant
NCT02224872 (11) [back to overview]Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe?
NCT02248597 (5) [back to overview]12 Month Disease Free Survival Probability
NCT02248597 (5) [back to overview]Progression Free Survival
NCT02248597 (5) [back to overview]Rate of Acute GvHD
NCT02248597 (5) [back to overview]Overall Survival
NCT02248597 (5) [back to overview]Relapse-free Mortality
NCT02251548 (12) [back to overview]Participants Who Convert From Bone Marrow MRD Negativity to MRD Positivity in Participants Who Discontinue Ibrutinib
NCT02251548 (12) [back to overview]Rate of MRD Negative CR After 3 Cycles of iFCR
NCT02251548 (12) [back to overview]Complete Response Rate (CRR)
NCT02251548 (12) [back to overview]Overall Response Rate
NCT02251548 (12) [back to overview]Partial Response Rate (PRR)
NCT02251548 (12) [back to overview]1-year Combined Response With MRD From Bone Marrow
NCT02251548 (12) [back to overview]Part I: Participants Who Achieve a Minimal Residual Disease (MRD) Negative Complete Response (CR) in the Bone Marrow at 2 Months Post FCR
NCT02251548 (12) [back to overview]Number of Participants Who Convert From Bone Marrow MRD Negative PR to Bone Marrow MRD Negative CR After 2 Years on Treatment
NCT02251548 (12) [back to overview]Number of Participants Who Convert From Bone Marrow MRD Negative PR to Bone Marrow MRD Negative CR After 1 Year of Ibrutinib Maintenance
NCT02251548 (12) [back to overview]Median Overall Survival (OS)
NCT02251548 (12) [back to overview]Median Progression-Free Survival (PFS)
NCT02251548 (12) [back to overview]Median Time to Bone Marrow MRD Negativity
NCT02259348 (9) [back to overview]Percentage of Participants Engrafted by Day 42 Post-transplant
NCT02259348 (9) [back to overview]Overall Survival (OS)
NCT02259348 (9) [back to overview]Mean of Days to Absolute Neutrophil Count (ANC) Engraftment
NCT02259348 (9) [back to overview]Incidence of Malignant Relapse
NCT02259348 (9) [back to overview]Median Days to Absolute Neutrophil Count (ANC) Engraftment
NCT02259348 (9) [back to overview]Event-free Survival (EFS)
NCT02259348 (9) [back to overview]Incidence and Severity of Chronic GvHD
NCT02259348 (9) [back to overview]Incidence and Severity of Acute GvHD
NCT02259348 (9) [back to overview]Rate of Transplant-related Mortality (TRM)
NCT02280811 (7) [back to overview]Expression of Programmed Cell Death 1 (PD-1) by Circulating E6 T-Cell Receptor (TCR) T-Cells
NCT02280811 (7) [back to overview]Maximum Tolerated Dose (MTD)
NCT02280811 (7) [back to overview]Number of Participants With a Dose Limiting Toxicity (DLT)
NCT02280811 (7) [back to overview]Number of Participants With Serious and Non-serious Adverse Events
NCT02280811 (7) [back to overview]Objective Tumor Response Rate (Complete or Partial Response)
NCT02280811 (7) [back to overview]Percentage of Cluster of Differentiation 3 (CD3+) Cells That Are E6 T-Cell Receptor Memory of Circulating T-Cells in Responders and Non-responders
NCT02280811 (7) [back to overview]Duration of Response
NCT02282904 (2) [back to overview]To Determine the Safety of This Allogeneic HSCT Approach in Patients With CGD Including Transplant Related Toxicity, the Incidence of Acute and Chronic Graft-versus-host Disease, Immune Reconstitution, Overalland Disease-free Survival.
NCT02282904 (2) [back to overview]To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD
NCT02348216 (29) [back to overview]Phase 2 Pivotal Study (Cohorts 1 and 2): Best Overall Response Using IRRC Per Cheson 2007
NCT02348216 (29) [back to overview]Pharmacokinetics: Peak Level of Anti-CD19 CAR T Cells in Blood
NCT02348216 (29) [back to overview]Phase 1 Study: Number of Participants Experiencing Adverse Events (AEs) Defined as Dose Limiting Toxicities (DLTs)
NCT02348216 (29) [back to overview]Phase 1 Study: ORR as Assessed by Investigator Per Revised IWG Response Criteria for Malignant Lymphoma
NCT02348216 (29) [back to overview]Phase 2 Pivotal Study (Cohorts 1 and 2): Duration of Response (DOR) Using IRRC Per Cheson 2007
NCT02348216 (29) [back to overview]Pharmacodynamics: Peak Level of Cytokine (Ferritin) in Serum (Phase 1 and Phase 2 Cohorts 1 and 2)
NCT02348216 (29) [back to overview]Pharmacodynamics: Peak Level of Cytokine (CRP) in Serum
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study (Cohort 4): Percentage of Participants With Treatment-Emergent CRS and Neurologic Toxicities by Severity Grades
NCT02348216 (29) [back to overview]Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 4 and Grade 3 or Higher Resulting From Decreased Parameter Value
NCT02348216 (29) [back to overview]Phase 2: Progression-Free Survival (PFS) as Assessed by Investigator Per Revised IWG Response Criteria for Malignant Lymphoma
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study: Number of Participants With the European Quality of Life Five Dimension Five Level Scale (EQ-5D) Score
NCT02348216 (29) [back to overview]Phase 2 Pivotal Study (Cohorts 1 and 2): ORR Per Independent Radiological Review Committee (IRRC)
NCT02348216 (29) [back to overview]Pharmacodynamics: Peak Level of Cytokine (Ferritin) in Serum (Phase 2 Cohort 3)
NCT02348216 (29) [back to overview]Phase 2 Pivotal Study (Cohorts 1 and 2): Overall Response Rate (ORR) as Assessed by Investigator Per Revised International Working Group (IWG) Response Criteria for Malignant Lymphoma
NCT02348216 (29) [back to overview]Phase 2 Pivotal Study (Cohorts 1 and 2): PFS Using IRRC Per Cheson 2007
NCT02348216 (29) [back to overview]Percentage of Participants With Positive Replication Competent Retrovirus (RCR)
NCT02348216 (29) [back to overview]Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAEs)
NCT02348216 (29) [back to overview]Percentage of Participants With Anti-Axicabtagene Ciloleucel Antibodies
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study (Cohorts 3, 4, 5, and 6): ORR as Assessed by Investigator Per the Revised IWG Response Criteria for Malignant Lymphoma
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study: EQ-5D Visual Analogue Scale (VAS) Score
NCT02348216 (29) [back to overview]Phase 2: Duration of Response (DOR) as Assessed by Investigator Per Revised IWG Response Criteria for Malignant Lymphoma
NCT02348216 (29) [back to overview]Phase 2: Overall Survival (OS)
NCT02348216 (29) [back to overview]Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 4 and Grade 3 or Higher Resulting From Increased Parameter Value
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study (Cohort 3): Percentage of Participants With Treatment-Emergent Cytokine Release Syndrome (CRS) and Neurologic Toxicities by Severity Grades
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study (Cohort 5): Percentage of Participants With Treatment-Emergent CRS and Neurologic Toxicities by Severity Grades
NCT02348216 (29) [back to overview]Pharmacodynamics: Peak Level of Cytokines (Ferritin, ICAM-1, IL-2 R, Perforin, and VCAM-1) in Serum (Phase 2 Cohorts 4, 5, and 6)
NCT02348216 (29) [back to overview]Phase 2 Safety Management Study (Cohort 6): Percentage of Participants With Treatment-Emergent CRS and Neurologic Toxicities by Severity Grades
NCT02348216 (29) [back to overview]Pharmacodynamics: Peak Level of Cytokines (IP-10, Granzyme B, IFN-gamma, IL-1 RA, IL-10, IL-15, IL-2, IL-6, IL-7, IL-8, TNF Alpha, and GM-CSF) in Serum (Phase 2 Cohorts 4, 5, and 6)
NCT02348216 (29) [back to overview]Pharmacodynamics: Peak Level of Cytokines in Serum (Phase 1 and Phase 2 Cohorts 1, 2, and 3)
NCT02354690 (5) [back to overview]Treatment Related Immune Responses
NCT02354690 (5) [back to overview]Number of Reported Adverse Events
NCT02354690 (5) [back to overview]Objective Response Rate
NCT02354690 (5) [back to overview]Overall Survival
NCT02354690 (5) [back to overview]Progression Free Survival
NCT02379195 (5) [back to overview]Objective Response Rate
NCT02379195 (5) [back to overview]Number of Participants With Adverse Events/Serious Adverse Events
NCT02379195 (5) [back to overview]Treatment Related Immune Responses
NCT02379195 (5) [back to overview]Progression Free Survival
NCT02379195 (5) [back to overview]Overall Survival
NCT02421939 (12) [back to overview]Percentage of Participants With Complete Remission and Complete Remission With Partial Hematological Recovery (CR/CRh) in the Gilteritinib Arm
NCT02421939 (12) [back to overview]Percentage of Participants Who Achieved Transfusion Conversion and Maintenance
NCT02421939 (12) [back to overview]Percentage of Participants Who Underwent Hematopoietic Stem Cell Transplant
NCT02421939 (12) [back to overview]Change From Baseline in Brief Fatigue Inventory (BFI)
NCT02421939 (12) [back to overview]Percentage of Participants With Composite Complete Remission (CRc Rate)
NCT02421939 (12) [back to overview]Percentage of Participants With Complete Remission (CR) With Partial Hematological Recovery (CRh)
NCT02421939 (12) [back to overview]Percentage of Participants With Complete Remission (CR) Rate
NCT02421939 (12) [back to overview]Duration of Remission
NCT02421939 (12) [back to overview]Duration of Overall Survival (OS)
NCT02421939 (12) [back to overview]Duration of Leukemia-Free Survival (LFS)
NCT02421939 (12) [back to overview]Duration of Event-Free Survival (EFS)
NCT02421939 (12) [back to overview]Number of Participants With Adverse Events
NCT02435901 (3) [back to overview]Event Free Survival; Number of Participants Who Survived at 2 Years
NCT02435901 (3) [back to overview]Assessment of Treatment Related Mortality and Morbidity
NCT02435901 (3) [back to overview]Number of Participants With Sustained Cell Engraftment of Donor Cells
NCT02440464 (14) [back to overview]Percentage of Participants With Infections Post-randomization by Infection Type
NCT02440464 (14) [back to overview]Percentage of Participants With Disease Progression
NCT02440464 (14) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT02440464 (14) [back to overview]Percentage of Participants With Progression-Free Survival
NCT02440464 (14) [back to overview]Percentage of Participants With Toxicities Post-randomization by Toxicity Type
NCT02440464 (14) [back to overview]Percentage of Participants With Treatment-Related Mortality (TRM)
NCT02440464 (14) [back to overview]Percentage of Participants With Best Response to Treatment After Randomization
NCT02440464 (14) [back to overview]Percentage of Participants With Response to Treatment
NCT02440464 (14) [back to overview]Percentage of Participants With Infections Post-randomization by Time Point
NCT02440464 (14) [back to overview]Percentage of Participants With Acute GVHD (Grades III-IV)
NCT02440464 (14) [back to overview]Medical Outcomes Study (MOS) - Short Form 36 (SF-36) Score
NCT02440464 (14) [back to overview]Percentage of Participants With Chronic GVHD
NCT02440464 (14) [back to overview]Functional Assessment of Cancer Therapy (FACT) - Bone Marrow Transplant (BMT) Total Score
NCT02440464 (14) [back to overview]Percentage of Participants With Toxicities Post-randomization by Time Point
NCT02497404 (9) [back to overview]Acute Graft-versus-Host Disease (GVHD)
NCT02497404 (9) [back to overview]Disease Free Survival at 1 Year Post-transplant
NCT02497404 (9) [back to overview]Overall Survival at 6 Months Post-transplant
NCT02497404 (9) [back to overview]Disease Free Survival at 6 Months Post-transplant
NCT02497404 (9) [back to overview]Graft Failure
NCT02497404 (9) [back to overview]High-Risk Extensive Chronic Graft-versus-Host-Disease
NCT02497404 (9) [back to overview]Overall Survival at 1 Year Post-Transplant
NCT02497404 (9) [back to overview]Overall Survival at 2 Years Post-Transplant
NCT02497404 (9) [back to overview]Disease Free Survival at 2 Years Post-transplant
NCT02533401 (5) [back to overview]Progression-Free Survival (PFS)
NCT02533401 (5) [back to overview]Percentage of Participants With Death or Disease Progression
NCT02533401 (5) [back to overview]Percentage of Participants Who Died
NCT02533401 (5) [back to overview]Overall Survival (OS)
NCT02533401 (5) [back to overview]Percentage of Participants With Complete Response (CR), Nodular Partial Response (nPR), or Partial Response (PR)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Disease Relapse, Days 90-180 (D90)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Non-relapse Mortality, Day 360 (D60)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Non-relapse Mortality, Day 360 (D90)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Non-relapse Mortality, Days 60-180 (D60)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D90)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Relapse, Day 360 (D60)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Relapse, Day 360 (D90)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Graft Failure, Days 60-180 (D60)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Graft Failure, Days 90-180 (D90)
NCT02556931 (22) [back to overview]Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D90 Cohort)
NCT02556931 (22) [back to overview]Number of Number of Participants Who Experience Graft Failure, Day 360 (D90)
NCT02556931 (22) [back to overview]Number of Number of Participants With Severe Chronic GVHD, Day 360 (D60)
NCT02556931 (22) [back to overview]Number of Number of Participants Who Experience Graft Failure, Day 360 (D60)
NCT02556931 (22) [back to overview]Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60)
NCT02556931 (22) [back to overview]Number of Participants With Chronic GVHD, Days 60-180 (D60)
NCT02556931 (22) [back to overview]Number of Participants With Chronic GVHD, Days 90-180 (D90)
NCT02556931 (22) [back to overview]Number of Participants With Grades III-IV Acute GVHD, Days 60-180 (D60)
NCT02556931 (22) [back to overview]Number of Participants With Grades III-IV Acute GVHD, Days 90-180 (D90)
NCT02556931 (22) [back to overview]Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D60 Cohort)
NCT02556931 (22) [back to overview]Number of Number of Participants With Severe Chronic GVHD, Day 360 (D90)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Disease Relapse, Days 60-180 (D60)
NCT02556931 (22) [back to overview]Number of Participants Who Experience Non-relapse Mortality, Days 90-180 (D90)
NCT02581007 (4) [back to overview]Graft Rejection
NCT02581007 (4) [back to overview]GVHD Incidence
NCT02581007 (4) [back to overview]Overall Survival
NCT02581007 (4) [back to overview]Relapse Incidence
NCT02588612 (10) [back to overview]Number of Participants With Hematology Results by Maximum Grade Increase Post-Baseline
NCT02588612 (10) [back to overview]Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
NCT02588612 (10) [back to overview]Disease Control Rate (DCR)
NCT02588612 (10) [back to overview]Duration of Response
NCT02588612 (10) [back to overview]Overall Response Rate (ORR)
NCT02588612 (10) [back to overview]Progression-Free Survival (PFS) by Investigator Assessment
NCT02588612 (10) [back to overview]Number of Participants With Worst Case Post-Baseline Abnormal Electrocardiogram (ECG) Findings
NCT02588612 (10) [back to overview]Time to Response
NCT02588612 (10) [back to overview]Change From Baseline in Oxygen Saturation
NCT02588612 (10) [back to overview]Number of Participants With Any Grade Increase in Clinical Chemistry Parameters
NCT02601313 (8) [back to overview]Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Self-Care Scale Score
NCT02601313 (8) [back to overview]Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Usual Activity Scale Score
NCT02601313 (8) [back to overview]Percentage of Participants With Objective Response (OR) Per the Lugano Classification According to Independent Radiology Review Committee (IRRC) in Cohort 2
NCT02601313 (8) [back to overview]Change Over Time in EQ-5D Visual Analogue Scale (VAS) Score
NCT02601313 (8) [back to overview]Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Pain / Discomfort Activity Scale Score
NCT02601313 (8) [back to overview]Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Mobility Scale Score
NCT02601313 (8) [back to overview]Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Anxiety / Depression Activity Scale Score
NCT02601313 (8) [back to overview]Percentage of Participants With Objective Response (OR) Per the Lugano Classification According to Independent Radiology Review Committee (IRRC) in Cohort 1
NCT02614066 (18) [back to overview]Phase 2: Percentage of Participants Experiencing Treatment Emergent Adverse Events (TEAEs)
NCT02614066 (18) [back to overview]Phase 2: Percentage of Participants With Anti-KTE-X19 Antibodies
NCT02614066 (18) [back to overview]Phase 2: Change From Baseline Over Time in EQ-5D: Visual Analogue Scale (VAS)
NCT02614066 (18) [back to overview]Phase 2: Number of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Value
NCT02614066 (18) [back to overview]Phase 2: Number of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Value
NCT02614066 (18) [back to overview]Phase 2: Number of Participants With 5-Level European Quality of Life-5 Dimensions (EQ-5D-5L): Health Utility Index Scale
NCT02614066 (18) [back to overview]Phase 2: Relapse-free Survival (RFS)
NCT02614066 (18) [back to overview]Phase 1: Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs)
NCT02614066 (18) [back to overview]Phase 2: Complete Remission With Incomplete Hematologic Recovery (CRi) Rate Per Independent Review
NCT02614066 (18) [back to overview]Phase 2: Duration of Remission (DOR) Per Independent Review
NCT02614066 (18) [back to overview]Phase 2: Minimum Residual Disease (MRD) Negative Remission Rate
NCT02614066 (18) [back to overview]Phase 2: Percentage of Participants With Allogeneic Stem Cell Transplant (Allo-SCT)
NCT02614066 (18) [back to overview]Phase 2: MRD Negative Rate Among Complete Remission (CR) Participants
NCT02614066 (18) [back to overview]Phase 2: Complete Remission (CR) Rate Per Independent Review
NCT02614066 (18) [back to overview]Phase 2: MRD Negative Rate Among Complete Remission With Incomplete Hematologic Recovery (CRi) Participants
NCT02614066 (18) [back to overview]Phase 2: OCR Rate (CR + CRi) Per Investigator Review
NCT02614066 (18) [back to overview]Phase 2: Overall Complete Remission (OCR) Rate (Complete Remission [CR]+ Complete Remission With Incomplete Hematologic Recovery [CRi]) as Assessed Per Independent Review
NCT02614066 (18) [back to overview]Phase 2: Overall Survival (OS)
NCT02614560 (7) [back to overview]Incidence of Laboratory Abnormalities
NCT02614560 (7) [back to overview]Rate of MRD Negativity
NCT02614560 (7) [back to overview]Best Response of CR or CRi
NCT02614560 (7) [back to overview]Duration of Response
NCT02614560 (7) [back to overview]Overall Survival
NCT02614560 (7) [back to overview]Incidence of Adverse Events
NCT02614560 (7) [back to overview]1-year Survival Rate
NCT02626338 (1) [back to overview]Clinical Response to Crenolanib With Standard Salvage Chemotherapy
NCT02642965 (11) [back to overview]Liposome-encapsulated Cytarabine Time of Maximum Concentration
NCT02642965 (11) [back to overview]Liposome-encapsulated Cytarabine Volume of Distribution
NCT02642965 (11) [back to overview]Liposome-encapsulated Daunorubicin Area Under the Curve
NCT02642965 (11) [back to overview]Liposome-encapsulated Daunorubicin Clearance
NCT02642965 (11) [back to overview]Liposome-encapsulated Daunorubicin Volume of Distribution
NCT02642965 (11) [back to overview]Number of Participants With a Dose-limiting Toxicity
NCT02642965 (11) [back to overview]Percentage of Responders (Complete Response or Complete Remission With Partial or Incomplete Platelet Recovery) After First Cycle of Therapy
NCT02642965 (11) [back to overview]Percentage of Responders (Complete Response or Complete Remission With Partial Platelet Recovery) After up to 2 Cycles
NCT02642965 (11) [back to overview]Liposome-encapsulated Daunorubicin Time of Maximum Concentration
NCT02642965 (11) [back to overview]Liposome-encapsulated Cytarabine Area Under the Curve
NCT02642965 (11) [back to overview]Liposome-encapsulated Cytarabine Clearance
NCT02652468 (7) [back to overview]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
NCT02652468 (7) [back to overview]Overall Survival (OS)
NCT02652468 (7) [back to overview]Number of Participants With Severe Chronic GVHD
NCT02652468 (7) [back to overview]Number of Participants With Graft Failure
NCT02652468 (7) [back to overview]Number of Participants With Grade III-IV Acute GVHD as Determined by International Bone Marrow Transplant Registry (IBMTR) Severity Index Criteria
NCT02652468 (7) [back to overview]Progression-free Survival
NCT02652468 (7) [back to overview]Number of Participants With Treatment-related Mortality
NCT02659943 (12) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
NCT02659943 (12) [back to overview]Number of Participants With Evidence of Immunogenicity of the Chimeric Antigen Receptor (CAR) T-cell Product
NCT02659943 (12) [back to overview]Number of Participants With a Dose-Limiting Toxicity (DLT)
NCT02659943 (12) [back to overview]Number of Participants Who Had Anti-Lymphoma Activity
NCT02659943 (12) [back to overview]MTD
NCT02659943 (12) [back to overview]Median Peak Chimeric Antigen Receptor (CAR) T Cells Level for Participants Treated
NCT02659943 (12) [back to overview]Maximum Feasible Dose
NCT02659943 (12) [back to overview]Number of Participants With a Duration of Best Response in Months
NCT02659943 (12) [back to overview]Number of Participants Who Had a Second or Third Infusion of Chimeric Antigen Receptor (CAR)+ T Cells
NCT02659943 (12) [back to overview]Number of Participants Who Had a Best Response of Complete Remission (CR), Partial Remission (PR), Stable Disease (SD), and Progressive Disease (PD)
NCT02659943 (12) [back to overview]Number of Participants That Had Any Grade 2, 3, 4 and 5 Adverse Events
NCT02659943 (12) [back to overview]Percentage of Enrolled Participants Who Actually Get Treated
NCT02706405 (14) [back to overview]Highest Treatment Dose Administered on Study
NCT02706405 (14) [back to overview]Rate of Complete Response (CR) by Investigator Assessment Using Lugano Criteria
NCT02706405 (14) [back to overview]Progression Free Survival
NCT02706405 (14) [back to overview]Overall Survival
NCT02706405 (14) [back to overview]Objective Response Rate by Investigator Assessment Using Lugano Criteria
NCT02706405 (14) [back to overview]Maximum JCAR014 Cmax in Blood by Quantitative Polymerase Chain Reaction (qPCR) Analysis
NCT02706405 (14) [back to overview]Maximum JCAR014 Cmax by Flow Cytometry
NCT02706405 (14) [back to overview]Duration of Response
NCT02706405 (14) [back to overview]Dose Limiting Toxicity (DLT) Rates
NCT02706405 (14) [back to overview]Count of Participants Who Experienced Adverse Events
NCT02706405 (14) [back to overview]AUC of JCAR014 Cells by qPCR Analysis
NCT02706405 (14) [back to overview]Area Under the Curve (AUC) of JCAR014 by Flow Cytometry
NCT02706405 (14) [back to overview]Time to Loss of JCAR014 Detection in Blood by qPCR Analysis
NCT02706405 (14) [back to overview]Rate of Partial Response (PR) by Investigator Assessment Using Lugano Criteria
NCT02756572 (19) [back to overview]Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - GENDER
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Received Early Transplant.
NCT02756572 (19) [back to overview]Treatment Related Mortality Among Patients Who Received Early Transplant vs Patients Who Did Not Receive Early Transplant
NCT02756572 (19) [back to overview]Relapse-free Survival (RFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Relapse-free Survival (RFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Received Early Transplant.
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant
NCT02756572 (19) [back to overview]Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - TREATMENT RELATED MORTALITY
NCT02756572 (19) [back to overview]Demonstrate the Feasibility of Collecting Patient-reported Outcomes for Trial Participants
NCT02756572 (19) [back to overview]Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - AGE
NCT02756572 (19) [back to overview]Event-free Survival (EFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Event-free Survival (EFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Relapsed-free Survival 6 Months After Transplant
NCT02756572 (19) [back to overview]Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Enrollment and Incidence of Early Transplant
NCT02756572 (19) [back to overview]Acute Graft Versus Host Disease Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 84
NCT02756572 (19) [back to overview]Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 28
NCT02756572 (19) [back to overview]Demonstrate the Feasibility of Collecting Resource Utilization Data for Trial Participants
NCT02757885 (12) [back to overview]Veno-occlusive Disease (VOD) Rate
NCT02757885 (12) [back to overview]Rate of Disease Recurrence
NCT02757885 (12) [back to overview]Primary Graft Rejection Rate
NCT02757885 (12) [back to overview]Overall Survival Rate
NCT02757885 (12) [back to overview]Late Graft Rejection Rate
NCT02757885 (12) [back to overview]Infection Rate
NCT02757885 (12) [back to overview]Frequency of Stroke
NCT02757885 (12) [back to overview]Frequency of Idiopathic Pneumonia Syndrome (IPS)
NCT02757885 (12) [back to overview]Rate of Central Nervous System (CNS) Toxicity
NCT02757885 (12) [back to overview]Event-free Survival (EFS) Rate
NCT02757885 (12) [back to overview]Cumulative Incidence of Neutrophil Engraftment and Platelet Engraftment.
NCT02757885 (12) [back to overview]Chimerism Rate Following Hematopoietic Cell Transplantation for Sickle Cell Disease
NCT02761915 (7) [back to overview]Assessment of Tumour Response From Baseline (RECIST)
NCT02761915 (7) [back to overview]Safety and Tolerability of 1RG-CART Therapy
NCT02761915 (7) [back to overview]To Determine Recommended Phase II Regimen by Assessing the Number of DLTs at Each Dose Level
NCT02761915 (7) [back to overview]1RG-CART Counts in the Peripheral Blood
NCT02761915 (7) [back to overview]To Evaluate Anti-tumour Activity (Overall Survival)
NCT02761915 (7) [back to overview]To Evaluate Anti-tumour Activity (Progression Free Survival)
NCT02761915 (7) [back to overview]Assessment of Tumour Response From Baseline (irRC)
NCT02771197 (2) [back to overview]Number of Patients With 2-year Relapse Risk
NCT02771197 (2) [back to overview]Assess Safety of Pembrolizumab by Recording the Number of Participants With Treatment-related Adverse Events
NCT02774291 (1) [back to overview]Number of Participants With Toxicity Graded According to NCI-CTCAE Version 4.0
NCT02833805 (12) [back to overview]Number of Participants Who Experience Primary Graft Failure
NCT02833805 (12) [back to overview]Number of Participants Who Experience Grades III-IV Acute GVHD
NCT02833805 (12) [back to overview]Number of Participants Who Experience Grades II-IV Acute GVHD
NCT02833805 (12) [back to overview]Overall Survival at One Year
NCT02833805 (12) [back to overview]GVHD-free Relapse-free Survival (GRFS)
NCT02833805 (12) [back to overview]Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts
NCT02833805 (12) [back to overview]Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts
NCT02833805 (12) [back to overview]Number of Participants Who Experience Chronic GVHD
NCT02833805 (12) [back to overview]Overall Survival and Engraftment at One Year
NCT02833805 (12) [back to overview]Number of Participants With Full Donor Chimerism
NCT02833805 (12) [back to overview]Number of Participants Who Experience Secondary Graft Failure
NCT02833805 (12) [back to overview]Transplant-related Mortality
NCT02918292 (15) [back to overview]Percentage of Participants With Cytomegalovirus (CMV), Epstein Barr Virus (EBV) or Post-Transplant Lymphoproliferative Disease (PTLD)
NCT02918292 (15) [back to overview]Percentage of Participants With Neutrophil Recovery
NCT02918292 (15) [back to overview]Percentage of Participants With Acute Graft-vs-host-disease (GVHD)
NCT02918292 (15) [back to overview]Percentage of Participants With Chronic GVHD
NCT02918292 (15) [back to overview]Percentage of Participants With Graft-Failure-Free Survival
NCT02918292 (15) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT02918292 (15) [back to overview]Percentage of Participants With Platelet Recovery
NCT02918292 (15) [back to overview]Percentage of Participants With Primary Graft Failure
NCT02918292 (15) [back to overview]Percentage of Participants With Secondary Graft Failure
NCT02918292 (15) [back to overview]Frequencies of Infections Categorized by Infection Type
NCT02918292 (15) [back to overview]Health Related Quality of Life (HR-QoL) - PedsQL Stem Cell Transplant Module
NCT02918292 (15) [back to overview]Immune Reconstitution of Flow Cytometry
NCT02918292 (15) [back to overview]Immune Reconstitution of Quantitative Immunoglobulins
NCT02918292 (15) [back to overview]Participants With Grade 3-5 Toxicities by SOC
NCT02918292 (15) [back to overview]Health Related Quality of Life (HR-QoL) - Medical Outcomes Study Short Form (MOS SF-36)
NCT02926833 (22) [back to overview]Phase 1: Number of Participants Experiencing Dose-Limiting Toxicities (DLTs)
NCT02926833 (22) [back to overview]Phase 1 and 2: Atezolizumab Levels in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Atezolizumab Levels in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Atezolizumab Levels in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Atezolizumab Levels in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Peak Serum Levels of C-X-C Motif Chemokine 10 (CXCL10), Interferon-Gamma (IFN-γ), Interleukin-1 Receptor Antagonist (IL-1RA), Interleukin (IL)-2, IL-6, IL-8, IL-15, and Tumor Necrosis Factor-Alpha (TNF-α) in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants With Hematology Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Values
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants With Serum Chemistry Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Values
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants With Serum Chemistry Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Values
NCT02926833 (22) [back to overview]Phase 1 and 2: Complete Response Rate (CRR)
NCT02926833 (22) [back to overview]Phase 1 and 2: Duration of Response (DOR)
NCT02926833 (22) [back to overview]Phase 1 and 2: Objective Response Rate (ORR)
NCT02926833 (22) [back to overview]Phase 1 and 2: Overall Survival (OS)
NCT02926833 (22) [back to overview]Phase 1 and 2: Peak Anti-CD19 CAR T-Cell (KTE-C19) Level (Maximum Observed Plasma Concentration) in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Peak Serum Levels of C-Reactive Protein (CRP) in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Peak Serum Levels of Ferritin in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Peak Serum Levels of Interleukin-2 Receptor Alpha (IL-2Rα) in Blood
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants Experiencing Adverse Events (AEs)
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants With Anti-Atezolizumab Antibodies
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants With Anti-KTE-C19 Antibodies
NCT02926833 (22) [back to overview]Phase 1 and 2: Percentage of Participants With Hematology Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Values
NCT02926833 (22) [back to overview]Phase 1 and 2: Progression-Free Survival (PFS)
NCT02992743 (22) [back to overview]Overall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Independent Reviewer
NCT02992743 (22) [back to overview]Area Under the Time Curve From Zero to Time 28 Days AUC(0-28) of GSK3377794
NCT02992743 (22) [back to overview]Change From Baseline in ECG Mean Heart Rate
NCT02992743 (22) [back to overview]Progression Free Survival (PFS) Assessed by Investigator
NCT02992743 (22) [back to overview]Time to Cmax (Tmax)
NCT02992743 (22) [back to overview]Time to Response (TTR) Assessed by Independent Reviewer
NCT02992743 (22) [back to overview]Duration of Response (DOR) Assessed by Independent Reviewer
NCT02992743 (22) [back to overview]Number of Participants With Insertional Oncogenesis
NCT02992743 (22) [back to overview]Duration of Response (DOR) Assessed by Investigator
NCT02992743 (22) [back to overview]Maximum Transgene Expansion (Cmax) of GSK3377794
NCT02992743 (22) [back to overview]Progression Free Survival (PFS) Assessed by Independent Reviewer
NCT02992743 (22) [back to overview]Time to Response (TTR) Assessed by Investigator
NCT02992743 (22) [back to overview]Best Overall Response (BOR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Independent Reviewer Assessment
NCT02992743 (22) [back to overview]Best Overall Response (BOR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Investigator Assessment
NCT02992743 (22) [back to overview]Change From Baseline in Electrocardiogram (ECG) Parameters- PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval
NCT02992743 (22) [back to overview]Number of Participants With Adverse Event of Special Interest (AESI)
NCT02992743 (22) [back to overview]Number of Participants With Any Grade Increase in Clinical Chemistry Results at Worst-case Post-baseline
NCT02992743 (22) [back to overview]Number of Participants With Any Grade Increase in Hematology Results at Worst-case Post-baseline
NCT02992743 (22) [back to overview]Number of Participants With Serious Adverse Events (SAEs) and Non-SAEs
NCT02992743 (22) [back to overview]Number of Participants With Positive Anti-drug Antibodies (ADAs)
NCT02992743 (22) [back to overview]Number of Participants With Replication Competent Lentivirus (RCL)
NCT02992743 (22) [back to overview]Overall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Investigator Assessment
NCT03018223 (4) [back to overview]Incidence of Chronic GVHD
NCT03018223 (4) [back to overview]Incidence of Grade II-IV Acute Graft vs. Host Disease (GVHD)
NCT03018223 (4) [back to overview]Progression Free Survival (PFS)
NCT03018223 (4) [back to overview]Overall Survival (OS)
NCT03049449 (10) [back to overview]Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells
NCT03049449 (10) [back to overview]Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells
NCT03049449 (10) [back to overview]Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells
NCT03049449 (10) [back to overview]Number of Participants With a Dose Limiting Toxicity (DLT)
NCT03049449 (10) [back to overview]Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
NCT03049449 (10) [back to overview]Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells
NCT03049449 (10) [back to overview]Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells
NCT03049449 (10) [back to overview]Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells
NCT03049449 (10) [back to overview]Number of Participants With Complete Response (CR), Partial Response (PR), Stable Disease (SD), or Progression as Their Best Response
NCT03049449 (10) [back to overview]Maximum Tolerated Dose of Anti-Tumor Necrosis Factor (TNF) Receptor Superfamily Member 8 (CD30) Chimeric Antigen Receptor (CAR) in Participants With Advanced CD30-expressing Lymphomas
NCT03071276 (3) [back to overview]The Overall Response (OR) Rate (Complete Response + Incomplete Count Recovery + Partial Response)
NCT03071276 (3) [back to overview]Complete Response
NCT03071276 (3) [back to overview]Complete Response or Complete Response With Incomplete Count Recovery
NCT03096782 (3) [back to overview]Time to Engraftment
NCT03096782 (3) [back to overview]Disease-free Survival
NCT03096782 (3) [back to overview]Overall Survival
NCT03128359 (3) [back to overview]Overall Survival (OS) at 1 Year
NCT03128359 (3) [back to overview]Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS) at 1 Year
NCT03128359 (3) [back to overview]Acute Graft Versus Host Disease (aGVHD) of Grades 2-4 According to the Consensus Grading
NCT03168438 (13) [back to overview]Maximum Persistence (Cmax) of GSK3377794
NCT03168438 (13) [back to overview]Duration of Response
NCT03168438 (13) [back to overview]Area Under the Plasma Concentration-time Curve From Zero to Day 28 (AUC[0-28])
NCT03168438 (13) [back to overview]Number of Participants With Treatment Limiting Toxicities-GSK3377794+Pembrolizumab Arm Only
NCT03168438 (13) [back to overview]Number of Participants With Worst-case Results for Coagulation Parameters Relative to Normal Range Post-Baseline Relative to Baseline
NCT03168438 (13) [back to overview]Number of Participants With Worst-case Hematology Results by Any Grade Increase Post-Baseline Relative to Baseline
NCT03168438 (13) [back to overview]Number of Participants With Worst-case Chemistry Results by Any Grade Increase Post-Baseline Relative to Baseline
NCT03168438 (13) [back to overview]Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
NCT03168438 (13) [back to overview]Time to Response
NCT03168438 (13) [back to overview]Time to Maximum Persistence
NCT03168438 (13) [back to overview]Progression-free Survival
NCT03168438 (13) [back to overview]Overall Response Rate
NCT03168438 (13) [back to overview]Number of Participants With Worst-case Post Baseline Abnormal Electrocardiogram (ECG) Findings
NCT03215810 (2) [back to overview]Number of Participants With Objective Response
NCT03215810 (2) [back to overview]Rate of Dose Limiting Toxicity (DLT)
NCT03287674 (4) [back to overview]Progression Free Survival
NCT03287674 (4) [back to overview]Overall Survival
NCT03287674 (4) [back to overview]Treatment Related Immune Responses
NCT03287674 (4) [back to overview]Number of Participants With Reported Adverse Events by Type
NCT03303950 (8) [back to overview]Number of Participants With Minimal Residual Disease (MRD) Response
NCT03303950 (8) [back to overview]Disease Free Survival at One Year
NCT03303950 (8) [back to overview]Incidence of Acute Graft Versus Host Disease (GVHD)
NCT03303950 (8) [back to overview]Incidence of Chronic GVHD
NCT03303950 (8) [back to overview]Non-relapse Mortality (NRM) at Day 100
NCT03303950 (8) [back to overview]Non-relapse Mortality (NRM) at Day 365
NCT03303950 (8) [back to overview]Overall Survival at One Year
NCT03303950 (8) [back to overview]Number of Participants With Different Clinical Responses
NCT03318861 (8) [back to overview]Overall Survival (OS)
NCT03318861 (8) [back to overview]Percentage of Participants Experiencing Clinically Significant Laboratory Abnormalities
NCT03318861 (8) [back to overview]Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs)
NCT03318861 (8) [back to overview]Percentage of Participants Experiencing Treatment-Emergent Adverse Events
NCT03318861 (8) [back to overview]Progression Free Survival (PFS) as Determined by Study Investigators According to the IMWG Consensus Panel 1
NCT03318861 (8) [back to overview]Time to Next Treatment (TTNT)
NCT03318861 (8) [back to overview]Duration of Response (DOR) as Determined by Study Investigators According to the IMWG Consensus Panel 1
NCT03318861 (8) [back to overview]Objective Response Rate (ORR) as Determined by Study Investigators According to the International Myeloma Working Group (IMWG) Consensus Panel 1 Criteria
NCT03338972 (7) [back to overview]Count of Patients That Experienced Adverse Events
NCT03338972 (7) [back to overview]Dose-limiting Toxicities (DLT) Rate
NCT03338972 (7) [back to overview]Duration of Persistence of Adoptively Transferred BCMA CAR-T Cells
NCT03338972 (7) [back to overview]Number of Participants With Detectable BCMA CART Cell Migration to Primary Disease Site (Bone Marrow) at Day 28
NCT03338972 (7) [back to overview]Objective Response Rate (ORR)
NCT03338972 (7) [back to overview]Overall Survival (OS)
NCT03338972 (7) [back to overview]Progression-free Survival (PFS)
NCT03504410 (1) [back to overview]Complete Remission (CR)
NCT03593902 (2) [back to overview]Survival of Treatment
NCT03593902 (2) [back to overview]Change in Skin Score by mRSS
NCT03602612 (3) [back to overview]Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0).
NCT03602612 (3) [back to overview]Number of Participants at Each Dose Level Who Experience a Dose-Limiting Toxicity (DLT)
NCT03602612 (3) [back to overview]Maximum Tolerated Dose (MTD) of Anti-B Cell Maturation Antigen (BCMA) - Chimeric Antigen Receptor (CAR)-T Cells
NCT03624036 (4) [back to overview]Objective Response Rate (ORR) Per Investigator Review Assessed by International Workshop on CLL (IWCLL) 2018 Criteria
NCT03624036 (4) [back to overview]Peak Level of Anti-CD19 CAR T-Cells in Blood
NCT03624036 (4) [back to overview]Percentage of Participants Experiencing Treatment Emergent Adverse Events (TEAEs)
NCT03624036 (4) [back to overview]Number of Participants Experiencing Dose Limiting Toxicities (DLTs)
NCT03761056 (15) [back to overview]Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Value
NCT03761056 (15) [back to overview]Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Value
NCT03761056 (15) [back to overview]Duration of Response (DOR) Per the Lugano Classification
NCT03761056 (15) [back to overview]Event-Free Survival (EFS)
NCT03761056 (15) [back to overview]Complete Response (CR) Rate Per the Lugano Classification as Determined by Study Investigators
NCT03761056 (15) [back to overview]Objective Response Rate (ORR) Per the Lugano Classification as Determined by Study Investigators
NCT03761056 (15) [back to overview]Time to Peak Serum Level of Granzyme B, Interferon-gamma (IFNg), Interleukin (IL)-2, IL-5, IL-6, IL-8, CRP, and Ferritin
NCT03761056 (15) [back to overview]Overall Survival (OS)
NCT03761056 (15) [back to overview]Peak Serum Level of Granzyme B, Interferon-gamma (IFNg), Interleukin (IL)-2, IL-5, IL-6, IL-8
NCT03761056 (15) [back to overview]Percentage of Participants With Treatment-Emergent Adverse Events (TEAE) and Treatment-Emergent Serious Adverse Events (SAE)
NCT03761056 (15) [back to overview]Peak Serum Level of C-Reactive Protein (CRP)
NCT03761056 (15) [back to overview]Peak Serum Level of Ferritin
NCT03761056 (15) [back to overview]Pharmacokinetics: Peak Level of Anti-CD19 CAR T Cells in Blood
NCT03761056 (15) [back to overview]Progression-Free Survival (PFS)
NCT03761056 (15) [back to overview]Relapse With Central Nervous Disease (CNS) Disease
NCT03813147 (8) [back to overview]Elimination Half-life of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS
NCT03813147 (8) [back to overview]Maximum Time to Concentration of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS
NCT03813147 (8) [back to overview]Total Plasma Clearance of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS
NCT03813147 (8) [back to overview]Maximum Concentration of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS
NCT03813147 (8) [back to overview]Number of Participants With Adverse Events Attributable to MLN4924 (Pevonedistat)
NCT03813147 (8) [back to overview]Number of Participants With Antitumor Activity of MLN4924 (Pevonedistat)
NCT03813147 (8) [back to overview]Number of Participants With Dose Limiting Toxicities of MLN4924 (Pevonedistat)
NCT03813147 (8) [back to overview]Area Under the Plasma Concentration Versus Time Curve of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS
NCT03860844 (13) [back to overview]AML: AUC of Isatuximab
NCT03860844 (13) [back to overview]Percentage of Participants With Complete Response (CR) Rate
NCT03860844 (13) [back to overview]Overall Response Rate (ORR)
NCT03860844 (13) [back to overview]Number of Participants With Infusion Reactions (IRs)
NCT03860844 (13) [back to overview]CD38 Receptor Occupancy
NCT03860844 (13) [back to overview]AML: Plasma Concentration Reached by Isatuximab Before Next Dose Administration (Ctrough)
NCT03860844 (13) [back to overview]Cluster of Differentiation (CD)38 Receptor Density
NCT03860844 (13) [back to overview]Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
NCT03860844 (13) [back to overview]CD38 Receptor Occupancy
NCT03860844 (13) [back to overview]B-ALL and T-ALL: Plasma Concentration Reached by Isatuximab Before Next Dose Administration (Ctrough)
NCT03860844 (13) [back to overview]B-ALL and T-ALL: Concentrations at the End of Infusion (Ceoi) of Isatuximab
NCT03860844 (13) [back to overview]B-ALL and T-ALL: Area Under the Concentration Time Curve (AUC) of Isatuximab
NCT03860844 (13) [back to overview]AML: Ceoi of Isatuximab
NCT03873805 (2) [back to overview]Number of Participants Experiencing a Dose-limiting Toxicity (DLT)
NCT03873805 (2) [back to overview]Grade 3 Toxicity Profile
NCT03912831 (1) [back to overview]Phase 1A: Percentage of Participants Experiencing Adverse Events Defined as Dose-Limiting Toxicities (DLTs)
NCT03958656 (3) [back to overview]Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
NCT03958656 (3) [back to overview]Number of Participants That Had Any Grade ≤2, and 3, 4 and 5 Adverse Events Following Administration of T Cells Expressing Anti- Signaling Lymphocytic Activation Molecule F7 (SLAMF7) Chimeric Antigen Receptor (CAR)
NCT03958656 (3) [back to overview]Number of Participants With a Response
NCT04002115 (7) [back to overview]Severity of Chronic GVHD
NCT04002115 (7) [back to overview]Rate of Chronic GVHD
NCT04002115 (7) [back to overview]Severity of Acute Graft-versus-host Disease (GVHD)
NCT04002115 (7) [back to overview]Complete Remission (CR) Rate at Day 30 Post HSCT
NCT04002115 (7) [back to overview]Non-relapse Related Mortality
NCT04002115 (7) [back to overview]Rate of Acute Graft-versus-host Disease (GVHD)
NCT04002115 (7) [back to overview]Neutrophil Engraftment
NCT04030195 (4) [back to overview]Objective Response Rate
NCT04030195 (4) [back to overview]Maximum Tolerated Dose (MTD)
NCT04030195 (4) [back to overview]Number of Participants With Dose-Limiting Toxicities
NCT04030195 (4) [back to overview]Progression-free Survival (PFS)
NCT04160195 (7) [back to overview]Last Time-Point at Which Chimeric Antigen Receptors (CAR) T Cells Were Detected in the Blood
NCT04160195 (7) [back to overview]Maximum Tolerated Dose (MTD) of Chimeric Antigen Receptors (CAR) T Cells
NCT04160195 (7) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
NCT04160195 (7) [back to overview]Percentage of Peak Blood Chimeric Antigen Receptors (CAR) T Cells
NCT04160195 (7) [back to overview]Percentage of Peripheral Blood Mononuclear Cells (PBMC) of Chimeric Antigen Receptors (CAR) T Cells
NCT04160195 (7) [back to overview]Number of Participants Administered T Cells Expressing a Novel Fully- Human Anti-cluster of Differentiation 19 (CD19) and Anti-cluster of Differentiation 20 (CD20) Chimeric Antigen Receptors (CAR) Who Experienced a Dose-limiting Toxicity (DLT)
NCT04160195 (7) [back to overview]Number of Participants With Clinical Response
NCT04205240 (2) [back to overview]Number of Patients With Grade II-IV Acute Graft-versus Host Disease (GvHD (aGVHD)
NCT04205240 (2) [back to overview]Number of Patients With a Partial Response
NCT04339101 (3) [back to overview]Progression Free Survival (PFS)
NCT04339101 (3) [back to overview]Cumulative Incidence of Grade II-IV Acute GVHD
NCT04339101 (3) [back to overview]Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year
NCT04395222 (2) [back to overview]Proportion of Failure of the Haplo-Graft
NCT04395222 (2) [back to overview]Percentage of Subjects With Successful Haplo-derived Neutrophil Engraftment
NCT04639245 (6) [back to overview]Participants That Displayed Transgenic T Cells in Tumor Tissue
NCT04639245 (6) [back to overview]Overall Survival
NCT04639245 (6) [back to overview]Count of Participants That Experienced Treatment-related Unexpected Grade 3 or Higher Adverse Events
NCT04639245 (6) [back to overview]Peripheral Blood Concentration of Infused Transgenic T Cells Over Time
NCT04639245 (6) [back to overview]Progression-free Survival
NCT04639245 (6) [back to overview]Objective Response Rates
NCT05993299 (5) [back to overview]Time to Cmax (Tmax)
NCT05993299 (5) [back to overview]Overall Response Rate (ORR)
NCT05993299 (5) [back to overview]Maximum Transgene Expansion (Cmax)
NCT05993299 (5) [back to overview]Disease Control Rate (DCR)
NCT05993299 (5) [back to overview]Area Under the Time Curve From Zero to Time 28 Days (AUC[0-28])

Change in Gene Expression Post Chemo

Changes in lymphocyte gene expression was measured by deoxyribonucleic acid (DNA) microarray analysis of circulating leukemic cells after completion of study treatment. A change in expression is defined as a >50% increase in circulating leukemic cells or a 30% decrease in circulating leukemic cells. (NCT00001586)
Timeframe: 6 hours post treatment, and 24 hours post treatment

InterventionPercent change in cells (Number)
6 hours post treatment (e.g. ># cells)24 hours post treatment (e.g. > # cells)
Intermediate-high Risk B-Cell Pts3050

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. (NCT00001586)
Timeframe: 13 years, 10.5 months

InterventionParticipants (Number)
Intermediate-high Risk B-Cell Pts18

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00001832)
Timeframe: 10.5 months

InterventionParticipants (Number)
Abl Cells IV + Cyclophosphamide 30 mg/kg3
Abl Cells IV + Cyclophosphamide 60 mg/kg3
Abl Cells IV+Low Dose IV IL-2 (Initial)3
Abl Cells IV+High Dose IV IL-2 (Initial)6
Abl Cells IV + MTD IL-250
Abl Cells IA + MTD (Prior Cells IV on 6)4
Abl Cells IA + MTD IL-27
Abl Cells IA+MTD IL-2 (MART-1 Reactive)8
Abl Cells IV + MTD IL-2 no GCSF6
Abl Cells IV+MTD IL-2 no GCSF(gp100 Reactive)1
Abl Cells IV+MTD IL-2 no GCSF (MART-1reactive)7
Abl Cells IV + SQ IL-2 With GCSF6
Abl Cells IV + SQ IL-2 With GCSF (MART-1 Reactive)3
Abl Cells IV + SQ IL-2 With GCSF (no Reactivity)2

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Clinical Response

Complete response (CR) is defined as the disappearance of all clinical evidence of disease. Partial response (PR) is a 50% or greater decrease in the sum of the products of perpendicular diameters of all measurable lesions for at least one month. No new lesions may appear, and none may increase. Minor response (MR) is a 25-49% decrease in the sum of the products of the perpendicular diameters of all measurable lesions. Appearance of new lesions following a PR or CR are considered relapses. Patients with progressive disease (PD) and no evidence of stable disease will be taken off study after receiving IL-2. (NCT00001832)
Timeframe: Every three to four weeks after the treatment, for up to 5 years.

,,,,,,,,,,,,,
InterventionParticipants (Number)
Complete ResponsePartial ResponseMinor ResponseProgressive DiseaseMixed ResponseNo ResponseStable Disease
Abl Cells IA + MTD (Prior Cells IV on 6)0000040
Abl Cells IA + MTD IL-21000050
Abl Cells IA+MTD IL-2 (MART-1 Reactive)1100150
Abl Cells IV + Cyclophosphamide 30 mg/kg0000030
Abl Cells IV + Cyclophosphamide 60 mg/kg0000030
Abl Cells IV + MTD IL-2314000321
Abl Cells IV + MTD IL-2 no GCSF1100040
Abl Cells IV + SQ IL-2 With GCSF0300030
Abl Cells IV + SQ IL-2 With GCSF (MART-1 Reactive)0100020
Abl Cells IV + SQ IL-2 With GCSF (no Reactivity)0000020
Abl Cells IV+High Dose IV IL-2 (Initial)0000060
Abl Cells IV+Low Dose IV IL-2 (Initial)0000030
Abl Cells IV+MTD IL-2 no GCSF (MART-1reactive)0200050
Abl Cells IV+MTD IL-2 no GCSF(gp100 Reactive)0010000

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Utilize Flow Cytometry and Polymerase Chain Reaction as Sensitive Measures of Minimal Residual Disease

The flow cytometric response and the molecular polymerase chain reaction (PCR) response was captured as indicated in the protocol. Immunophenotypic analysis of bone marrow and/ or peripheral blood demonstrate a normal k:λ ratio and a normal number of CD5/CD19 (or CD5/CD20) dual staining cells (<5% of the lymphocyte gate). (NCT00003659)
Timeframe: 3 years

Interventionparticipants (Number)
Flow cytometric complete responseMolencular (PCR) complete response
Intermediate or High Risk Chronic Lymphocytic Leukemia2012

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Overall Response Rate

Response was determined as indicated in the protocol. The categories are: complete response, nodular partial response, partial response and failure. The major criteria for determination of response to therapy in patients with CLL include physical examination and examination of the peripheral blood and bone marrow. The laboratory and radiographic studies which were abnormal pre-study, will be repeated to document the degree of maximal response. (NCT00003659)
Timeframe: 3 years

Interventionparticipants (Number)
Complete ResponseNodular Partial ResponsePartial ResponseFailure
Intermediate or High Risk Chronic Lymphocytic Leukemia22284

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Overall Survival Status

The 5 year survival rate. The survival of patients with this disease is dependent on the stage of disease. Two useful staging systems are: Three-stage Rai System Clinical Feature and the Binet System. (NCT00003659)
Timeframe: up to 5 years

Interventionparticipants (Number)
Intermediate or High Risk Chronic Lymphocytic Leukemia26

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4 yr OS

Overall survival estimate at 4 years post BMT (NCT00003816)
Timeframe: 4-year

Interventionpercentage of participants (Number)
BuCy56.4
CyTBI56.5
FluMel38.2
VpCyTBI39.1
Other53.3

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4 Year PFS

progression free survival estimate at 4 years post BMT (events are disease progression/relapse and death due to any cause) (NCT00003816)
Timeframe: 4 years

Interventionpercentage of participants (Number)
BuCy49.1
CyTBI52.2
FluMel33.2
VpCyTBI26.1
Other40

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Toxicity/TRM at Day 100

Death due to treatment related causes before day +100 after BMT (NCT00003816)
Timeframe: Day +100

InterventionParticipants (Count of Participants)
BuCy3
CyTBI4
FluMel31
VpCyTBI4
Other4

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CR Rate

Rate of Complete Remission by Day +100 (NCT00003816)
Timeframe: day 100

InterventionParticipants (Count of Participants)
BuCy42
CyTBI55
FluMel134
VpCyTBI18
Other7

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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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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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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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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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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00006184)
Timeframe: 9 years

InterventionParticipants (Count of Participants)
Recipient - Chemotherapy Group10
Donor - Vaccination Generation Group10

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Immune Response

Immune cell depletion is defined as immunosuppression of participants T cells prior to transplant measured by cluster of differentiation 4 (CD4) counts (i.e. cells) > 50 cells per ul.Immune T-cell depletion helps to reduce the ability to reject allogeneic cells in participants and is required for engraftment. Engraftment is the body's ability to accept donor cells. (NCT00006184)
Timeframe: 105 days

InterventionParticpants (Number)
Recipient - Chemotherapy Group7

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Overall Survival

(NCT00027560)
Timeframe: 12 months post transplant

Interventionparticipants (Number)
TREATMENT OF LYMPHOHEMATOPOIETIC MALIGNANCIES37

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Overall Survival

(NCT00027560)
Timeframe: 24 months post transplant

Interventionparticipants (Number)
TREATMENT OF LYMPHOHEMATOPOIETIC MALIGNANCIES30

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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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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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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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Number of Participants With Absolute Neutrophil Count Engraftment

Absolute neutrophil engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L. Baseline to Day 30 post transplant. (NCT00038857)
Timeframe: Day 0 up to Day 30

Interventionparticipant (Number)
Melphalan + Thiotepa + Fludarabine + Rabbit ATG + CD34 PBPC21

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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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Number of Participants With Engraftment

Engraftment is defined as rapid conversion to complete donor chimerism and is assessed by blood counts and chimerism, >95% donor engraftment at day 100 in >75% of patients. (NCT00043979)
Timeframe: 100 days

InterventionParticipants (Number)
Arm 2-Recipients23

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Toxicity

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00043979)
Timeframe: 16.5 months

InterventionParticipants (Number)
Arm 2-Recipients30

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Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin)

Response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST). RECIST criteria offer a simplified, conservative, extraction of imaging data for wide application in clinical trials. They presume that linear measures are an adequate substitute for 2-D (dimensional) methods and registers four response categories: Complete response (CR) is disappearance of all target lesions. Partial response (PR) is 30% increase in the sum of the longest diameter of target lesions. Progressive disease (PD) is 20% increase in the sum of the longest diameter of target lesions. Stable disease (SD) is small changes that do not meet above criteria. For the purposes of this study very good partial response ((VGPR) is >75% reduction in disease) was also employed. (NCT00043979)
Timeframe: up to 10 cycles of therapy or 280 days

InterventionParticipants (Count of Participants)
Complete Response (CR)Progressive Disease (PD)Partial Response (PR)Very Good Partial Response (VGPR)
Arm 2-Recipients2442

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Median Survival From Date of Progression

Median survival from date of progression is based on the time from on-study date until progression or last follow-up. (NCT00043979)
Timeframe: up to 77 months

InterventionMonths (Median)
Participants who did not receive a transplant(n=7)Participants who received a transplant (n=23)
Arm 2-Recipients3.319.1

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Number of Participants to Complete Conversion to >95% Donor Chimerism

Participants who tolerated the transplantation regimen and accepted >95% of the donors blood, marrow, and/or tissue. (NCT00043979)
Timeframe: up to 30 days

InterventionParticipants (Count of Participants)
Day +14Day +28
Arm 2-Recipients2323

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Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy

Site of radiotherapy (high energy radiation) and/or toxicity experienced by the participants post HSCT radiotherapy. Grading was preformed using the Modified Glucksberg Criteria. (NCT00043979)
Timeframe: up to 6 cycles or 168 days

InterventionParticipants (Count of Participants)
Chest wall; G2 skinAbdomen; G4 GIPancreas; G4 LFTs, G4 pancreatitisPleura, mediastinum; G4 LFTs, G2 mucositisChest wall; G4 skin, G3 mucositisSpine, skull; G2 nausea+vomiting, G2 fatiguePelvis; G4 enteritisPulmonary (cyberknife)Brain; B3 mucositisWhole lung; G3 mucositis, G3 skin, G5 lungL arm, R shoulder, B/L femur
Arm 2-Recipients11111111111

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Median Time to Reach Absolute Neutrophil Count of 500/mm(3)

Days for participants to achieve a neutrophil count of 500/mm(3). (NCT00043979)
Timeframe: up to 12 days

InterventionDays (Median)
Arm 2-Recipients9

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Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant

Participants who are alive at two years following Allo-Hematopoietic Stem Cell Transplant. (NCT00043979)
Timeframe: 2 years

Interventionpercentage of participants (Number)
From date of enrollmentFrom date of transplantation
Arm 2-Recipients39.134.8

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Number of Participants With Acute and Chronic GVHD

Acute GVHD as by Modified Glucksberg Criteria occurring before day 100. Chronic GVHD as per Seattle criteria occurring after day 100. (NCT00043979)
Timeframe: up to 5 years or death

,
Interventionparticipants (Number)
acute GVHDchronic GVHD
Recipients -Cyclosporine GVHD Prophylaxis1212
Recipients -Tacrolimus/Sirolimus GVHD Prophylaxis55

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Median Time to Reach a Platelet Count of 50,000/mm(3)

Days for participants to achieve a platelet count of 50,000/mm(3). (NCT00043979)
Timeframe: up to 43 days

InterventionDays (Median)
Arm 2-Recipients15

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Median Progression Free Survival

Progression free survival was based on the time from on-study date until progression or last follow-up. (NCT00043979)
Timeframe: up to 77 months

InterventionMonths (Median)
Arm 2-Recipients15.9

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Cluster of Differentiation 4 (CD4) Reconstitution

The median CD4 count with a range of 85-1565 (absolute count) was used to determine recovery and were considered recovered if in this range. The CD4 count was established by flow cytometry testing. (NCT00043979)
Timeframe: Day +28-42

Interventionmm(3) (Median)
Arm 2-Recipients284

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Number of Participants Who Experienced Graft Versus Tumor Effect (GVT)

GVT is defined as tumor response after day 42 post-transplantation without cytotoxic therapy. (NCT00043979)
Timeframe: up to day 100

InterventionParticipants (Count of Participants)
Arm 2-Recipients0

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Early Post Transplantation Relapse

Participants who experienced recurrence or progression of disease following transplant. (NCT00043979)
Timeframe: up to 300 days

InterventionDays (Median)
Arm 2-Recipients100

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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 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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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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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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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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Number of Participants That Remained Disease-free

Number of participants that remained Disease-free survival following stem cell transplant. Disease-free survival is defined as survival free of disease relapse or disease progression following stem cell transplant. (NCT00047060)
Timeframe: up to 100 days

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H4

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Number of Participants That Experienced Red Blood Cell Recovery

Number of participants that experienced red blood cell recovery following stem cell transplant. Red blood cell recovery is defined as achieving transfusion independence. (NCT00047060)
Timeframe: up to 100 days

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H5

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Efficacy of Nonmyeloablative Preparative Regimen

Proportion of subjects achieving a complete response. Complete response (CR) is defined as: disappearance of all signs and symptoms of cutaneous T-cell lymphomas (CTCL) for a period of at least one month. (NCT00047060)
Timeframe: 36 months

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H4

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Number of Participants That Experienced Platelet Recovery

Number of participants that experienced platelet recovery up to day 100 following stem cell transplant. Platelet recovery is defined as platelet count is greater than 50 x 10^9/l without platelet transfusion. (NCT00047060)
Timeframe: up to 100 days

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H5

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Number of Participants That Experienced Graft Failure

Number of participants that experienced graft failure. Graft failure is defined as: the failure to achieve sustained engraftment following stem cell transplantation. (NCT00047060)
Timeframe: up to 100 days

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H0

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Number of Participants That Experienced Engraftment

Number of participants that experienced engraftment following stem cell transplant. Engraftment is defined as neutrophil count is greater than 0.5 x10^9. (NCT00047060)
Timeframe: up to 100 days

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H5

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Number of Participants Overall Survival

Number of participants overall survival following stem cell transplant. Overall survival is defined as number participants alive following stem cell transplant (NCT00047060)
Timeframe: up to 5 years

InterventionParticipants (Count of Participants)
Stem Cell Transplant Therapy With Campath-1H3

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Number of Participants Who Experienced Acute GVHD Grades II-IV

"Number of participants who experienced acute GVHD grades II-IV~Acute-GVHD was graded and staged prospectively using criteria from the 1994 Consensus Conference on Acute- GVHD Grading.~Grades are defined as:~Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day.~Grade III: Skin = Rash on >50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea > 1500 mL/day.~Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin >15 mg/dL; Lower GI = Severe abdominal pain with or without ileus.~Grade II GVHD as moderate, grade III as severe, and grade IV life-threatening." (NCT00047060)
Timeframe: up to 100 days

InterventionParticipants (Count of Participants)
Grade IIGrade III-IV
Stem Cell Transplant Therapy With Campath-1H01

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Number of Participant Who Experienced Chronic Graft Versus Host Disease

"Number of participant who experienced chronic graft versus host disease (GVHD) following stem cell transplant The diagnosis of clinical features of chronic-GVHD was determined prospectively and classified retrospectively into limited or extensive based on the Revised Seattle Classification. Chronic GvHD severity categorized as limited is defined as: localized skin lesions with or without limited hepatic involvement and extensive is defined as: generalized skin involvement, major hepatic complications, or involvement of any other organ." (NCT00047060)
Timeframe: Day 100 up to 3 years

InterventionParticipants (Count of Participants)
LimitedExtensive
Stem Cell Transplant Therapy With Campath-1H30

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Median Time in Months to Achieve Full Myeloid and Full Donor T-cell Chimerism

Median time in months to achieve full myeloid and full donor T-cell Chimerism. Myeloid (CD34+) and T-cell (CD3+) chimerisms were determined by PCR analysis of short tandem repeats (STR). Full donor chimerism is defined as >95% donor- derived cells in the peripheral blood in a specific lineage. (NCT00047060)
Timeframe: Up to 22 months

Interventionmonths (Median)
Myeloid ChimerismDonor T-cell Chimerism
Stem Cell Transplant Therapy With Campath-1H97

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Number of Participants With Graft Failure

Graft failure is defined as either lack of hematologic recovery or lack of or loss of detectable donor cells. (NCT00048737)
Timeframe: 100 days

Interventionparticipants (Number)
90Y Zevalin in ASCT1

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Number of Participants With Adverse Events

Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. (NCT00048893)
Timeframe: 91 months

InterventionParticipants (Number)
Carcinoembryonic Antigen (CEA)-Tricom Vaccines11

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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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Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis

Here is the number of recipients who developed grades I-IV acute graft versus host disease (GVHD) following a combination of cyclosporine and a mini-dose methotrexate regimen for graft-versus-host disease (GVHD) prophylaxis. Grades I-IV acute GVHD clinical staging was assessed by the Glucksberg Criteria. Grade I-IV acute GVHD can occur in the skin, liver, and/or gut. Grade 1: rash < 25% body surface area (BSA), total bilirubin 2-3 mg/dl, and diarrhea 500-1000 ml/d. Grade 2: 25-50% BSA, total bilirubin 3-6 mg/dl, and diarrhea 1000-1500 ml/d. Grade 3: generalized erythroderma, total bilirubin 6-15 mg/dl, and diarrhea >1500 ml/d. Grade 4: desquamation and bullae, total bilirubin >15 mg/dl, and pain +/- ileus. (NCT00051311)
Timeframe: At least 100 days of follow-up post reduced-intensity stem cell transplantation (RIST)

InterventionParticipants (Count of Participants)
Grade I GVHDGrade II GVHDGrade III GVHDGrade IV GVHD
Recipient1760

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Percentage of Recipients Who Achieved Donor Chimerism at Day +14

Recipients who achieved engraftment with donor chimerism. Engraftment is defined as a target peripheral blood cluster of differentiation 4 (CD4) count of 100 cells/ul after induction therapy. (NCT00051311)
Timeframe: Day+14

Interventionpercentage of recipients (Number)
Recipient90

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Number of Recipients With Non-serious Adverse Events

Here is the number of recipients with non-serious adverse events assessed by the Common Toxicity Criteria version 2.0. A non-serious adverse event is any untoward medical occurrence. (NCT00051311)
Timeframe: Date treatment consent signed to date off study, approximately 58 months and 13 days.

InterventionParticipants (Count of Participants)
Recipient31

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Median Time to Neutrophil Recovery

Neutrophil recovery is defined as a neutrophil count ≥5000 µl for three consecutive days. (NCT00051311)
Timeframe: Up to 2 months after stem cell transplant

InterventionDays (Median)
Recipient14

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Median Time to Platelet Recovery

Platelet recovery is defined as >50,000 mm3 platelet cell count after transfusion. (NCT00051311)
Timeframe: Up to 2 months after stem cell transplant

InterventionDays (Median)
Recipient13

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Number of Recipients Evaluable Who Achieved Full Donor Chimerism at Day+100

Recipients who achieved engraftment with donor chimerism. Engraftment is defined as a target peripheral blood cluster of differentiation 4 (CD4) count of 100 cells/ul after induction therapy. (NCT00051311)
Timeframe: Day+100

InterventionParticipants (Count of Participants)
Recipient29

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Median Cycles of Induction Chemotherapy With Fludarabine, Cyclophosphamide, Etoposide, Doxorubicin, Vincristine, and Prednisone Given to Recipients

Median cycles of induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone given to recipients. (NCT00051311)
Timeframe: Up to cycle 3

InterventionCycles (Median)
Recipient2

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Number of Recipients Who Developed Chronic Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis

Here is the number of recipients who developed chronic graft versus host disease (GVHD) following a combination of cyclosporine and a mini-dose methotrexate regimen for graft-versus-host disease (GVHD) prophylaxis. Grades I-IV chronic GVHD clinical staging was assessed by the Glucksberg Criteria. Grade I-IV chronic GVHD can occur in the skin, liver, and/or gut. Grade 1: rash < 25% body surface area (BSA), total bilirubin 2-3 mg/dl, and diarrhea 500-1000 ml/d. Grade 2: 25-50% BSA, total bilirubin 3-6 mg/dl, and diarrhea 1000-1500 ml/d. Grade 3: generalized erythroderma, total bilirubin 6-15 mg/dl, and diarrhea >1500 ml/d. Grade 4: desquamation and bullae, total bilirubin >15 mg/dl, and pain +/- ileus. (NCT00051311)
Timeframe: At least 100 days of follow-up post reduced-intensity stem cell transplantation (RIST)

InterventionParticipants (Count of Participants)
Recipient23

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Number of Recipients With a Response to Reduced-intensity Stem Cell Transplantation (RIST)

Complete remission (CR) is complete disappearance of all detectable signs and symptoms of lymphoma for a period of at least one month. Complete remission unconfirmed (CRu) is a residual lymph node mass > 1.5 cm in greatest transverse diameter will be considered CRu if it has regressed by more than 75% in the SPD, does not change over at least one month, is negative by PET or gallium, and is negative on any biopsies obtained (biopsy not required). Progressive disease (PD) is a 25% or greater increase in SPD of all measured lesions compared to the smallest previous measurements, or appearance of any new lesion(s). (NCT00051311)
Timeframe: At least 100 days after post reduced-intensity stem cell transplantation (RIST).

InterventionParticipants (Count of Participants)
Complete Remission or Complete Remission UnconfirmedProgressive Disease
Recipient1516

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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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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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Acute GvHD

overall grade II-IV acute GvHD (NCT00053989)
Timeframe: Day +100

InterventionParticipants (Count of Participants)
All Patients16

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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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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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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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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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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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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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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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Median Time to Engraftment With the Isolex/CLINIMACs System

Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days. (NCT00058825)
Timeframe: 30 days

Interventiondays (Median)
Isolex12
CLINIMACs12

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2-year Overall Survival

Overall survival (OS) was calculated from the time of transplant to death from any cause or censored at last follow-up. Survival data were analyzed by Kaplan-Meier method. (NCT00058825)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Stem Cell Transplant33.3

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Donor Chimerism Engraftment of Greater Than 50%

Number of patients that engrafted who showed a chimerism (donor cells) of greater than 50% in the first 30 days (NCT00058825)
Timeframe: 30 days

Interventionparticipants (Number)
YesNo
Stem Cell Transplant202

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Number of Patients Who Engrafted With the Isolex/CLINIMACs System

Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days. (NCT00058825)
Timeframe: 30 days

Interventionparticipants (Number)
Isolex15
CLINIMACs7

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Time in Days to ANC Engraftment

Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days. (NCT00058825)
Timeframe: 30 days

Interventiondays (Median)
Stem Cell Transplant12

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Acute Graft Versus Host Disease

Number of patients with Acute Graft Versus Host Disease within 100 days post-transplant (NCT00058825)
Timeframe: 100 days

Interventionparticipants (Number)
Grade 0Grade IGrade IIGrade IIIGrade IV
Stem Cell Transplant260010

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Chronic Graft Versus Host Disease

Number of patients with Chronic Graft Versus Host Disease within 1 year post-transplant (NCT00058825)
Timeframe: 1 year

Interventionparticipants (Number)
YesNo
Stem Cell Transplant126

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2-year Relapse-free Survival

Relapse-free survival (RFS) was calculated from the time of transplant to the date of relapse, death, or last follow-up, whichever occurred first. Survival data were analyzed by Kaplan-Meier method. (NCT00058825)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Stem Cell Transplant25.9

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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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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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Number of Participants Severity of Acute GVHD by Treatment Arm

The severity of acute GVHD is determined by an assessment of the degree of involvement of the skin, liver, and gastrointestinal tract. The stages of individual organ involvement is assessed using Glucksberg grade (I-IV) where Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT00067002)
Timeframe: Following first 100 days, up to one year

,
InterventionParticipants (Count of Participants)
Grade ≤ 2Grade ≥ 3No GVHD
Expanded CB Arm15227
Un-Manipulated CB Arm20619

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Rate of Chronic GVHD

Number of participants who present GVHD post-transplant and display features of chronic GVHD. Diagnostic and distinctive features of chronic GVHD are present. There are no features of acute GVHD. (NCT00067002)
Timeframe: Up to one year

,
InterventionParticipants (Count of Participants)
Overall Chronic GVHDLimitedExtensive
Expanded CB Arm201413
Un-Manipulated CB Arm16109

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Number of Participants With Engraftment

Engraftment defined as a sustained absolute neutrophil count (ANC) > 0.5 x 10^9/L for at least 3 consecutive days. Engraftment Failure defined as ANC <500/ul by day +42 and participant has no evidence of donor chimerism on bone marrow examination. (NCT00067002)
Timeframe: First 100 days, evaluation and blood tests twice weekly

InterventionParticipants (Count of Participants)
Un-Manipulated CB Arm45
Expanded CB Arm44

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Rate of Acute Graft Versus Host Disease (GVHD)

Number of participants who display features of acute GVHD within 100 days of transplant. (NCT00067002)
Timeframe: Review over first 100 days

InterventionParticipants (Count of Participants)
Un-Manipulated CB Arm29
Expanded CB Arm24

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Time To Neutrophil Engraftment

Engraftment is defined as a sustained ANC > 0.5 x 10^9/L for at least 3 consecutive days. Engraftment date is the first of the 3 days with sustained absolute neutrophil count (ANC) >/= 0.5 x 10^9/L. (NCT00067002)
Timeframe: First 100 days, evaluation and blood tests twice weekly

InterventionDays (Median)
Un-Manipulated CB Arm17
Expanded CB Arm15

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Non-relapse Mortality (NRM)

Percentage of patients who died due to causes other than relapse (NCT00070135)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
Treatment (Fludarabine, Busulfan, Allogeneic PBSC)16

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2 Year Disease Free Survival In Unrelated Donor Recipient Group

"Percentage of participants who were alive and relapse free at 2 years for patients who were matched with an unrelated donor for transplant. The 2 year disease free survival, with 95% confidence interval, was estimated using the Kaplan Meier method.~A relapse is defined as any of the following:~Reappearance of leukemia blasts cells in peripheral blood~>5% blasts in the marrow, not attributable to another cause (e.g., bone marrow regeneration)~If there are no circulating blasts, but the marrow contains 5-20% blasts, a repeat bone marrow ≥ 1 week later with >5% blasts is necessary to meet the criteria for relapse~The development of extramedullary leukemia or leukemic cells in the cerebral spinal fluid" (NCT00070135)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Treatment (Fludarabine, Busulfan, Allogeneic PBSC)40

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2 Year DFS for All Patients

Percentage of participants who were alive and relapse free at 2 years for all patients. The 2 year disease free survival, with 95% confidence interval, was estimated using the Kaplan Meier method. (NCT00070135)
Timeframe: Up to 2 years

Interventionpercentage of participants (Number)
Treatment (Fludarabine, Busulfan, Allogeneic PBSC)42

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Response as Measured at 12 Months Post Allografting

response (partial and complete) assessed by CT scan at 12 months post allografting (NCT00074269)
Timeframe: From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 12 months

InterventionParticipants (Count of Participants)
Treatment3

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Progression-free Survival

Progression assessed by CT scan (NCT00074269)
Timeframe: From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 6 months

Interventiondays (Median)
Treatment110

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Overall Survival

(NCT00074269)
Timeframe: 1 year from the time of transplant

Interventiondays (Median)
Treatment279.4

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Number of Participants With Long-term Engraftment of Allogeneic Stem Cells and Lymphocytes

Long-term Engraftment of Allogeneic Stem Cells and Lymphocytes based on cell counts of ANC >1000 for 3 consecutive days and platelet count of >50,000 (NCT00074269)
Timeframe: 30 days post transplant

InterventionParticipants (Count of Participants)
Treatment5

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Number of Participants With Adverse Events

(NCT00074269)
Timeframe: 5 years post transplant

InterventionParticipants (Count of Participants)
Treatment5

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Number of Participants With Acute and Chronic Graft-versus-host Disease (GVHD)

(NCT00074269)
Timeframe: 100 days post transplant

InterventionParticipants (Count of Participants)
Treatment3

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Frequency of the Induction of Full Donor Chimerism of Lymphocytes as Measured at 1 Month Post Allografting

(NCT00074269)
Timeframe: 1 month post allografting

InterventionParticipants (Count of Participants)
Treatment5

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Percentage of Patients With Opportunistic Infection

Participants are susceptible to opportunistic infections such as bacterial, fungal, viral, protozoan infections and more due to immune suppression from chemotherapy drugs used to treat their disease. (NCT00074490)
Timeframe: First 100 days post-transplant

Interventionpercentage of participants (Number)
Arm IVD Cohort 1 (Th2 DLI)0
Arm IVD Cohort 3 (Multiple Th2 DLI)11.11
Arm IVA (12-day Expanded Th2 DLI)7.50
Arm IVB (6-day Expanded Th2 DLI)9.09
Arm IVC (6-day Expanded Th2 DLI and High Dose Sirolimus)11.90

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Percentage of Patients With ≥ Grade 2 Acute Graft Versus Host Disease (GVHD)

GVHD of the skin, liver and gut were graded on a scale of 1, 2, 3, and 4 (e.g. the grades are not added together) using the National Institutes of Health Consensus Criteria. Grade 1 is minimal GVHD, Grade 2 is moderate GVHD, Grade 3 is severe GVHD and Grade 4 is very severe GVHD. Grade 4 is a worse outcome than Grade 1. (NCT00074490)
Timeframe: first 100 days post-transplant

Interventionpercentage of patients (Number)
Arm IVD Cohort 1 (Th2 DLI)0
Arm IVD Cohort 3 (Multiple Th2 DLI)11.11
Arm IVA (12-day Expanded Th2 DLI)10
Arm IVB (6-day Expanded Th2 DLI)40.91
Arm IVC (6-day Expanded Th2 DLI and High Dose Sirolimus)40.48

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Percentage of Patients to Receive T Cell Infusion

T cells administered by intravenous infusion after patient received transplant. (NCT00074490)
Timeframe: first 100 days post-transplant

Interventionpercentage of patients (Number)
Arm IVD Cohort 1 (Th2 DLI)100
Arm IVD Cohort 3 (Multiple Th2 DLI)100
Arm IVA (12-day Expanded Th2 DLI)100
Arm IVB (6-day Expanded Th2 DLI)100
Arm IVC (6-day Expanded Th2 DLI and High Dose Sirolimus)100

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Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT00074490)
Timeframe: Date treatment consent signed to date off study, approximately 5 years

InterventionParticipants (Count of Participants)
Arm IVD Cohort 1 (Th2 DLI)1
Arm IVD Cohort 3 (Multiple Th2 DLI)27
Arm IVA (12-day Expanded Th2 DLI)40
Arm IVB (6-day Expanded Th2 DLI)44
Arm IVC (6-day Expanded Th2 DLI and High Dose Sirolimus)42

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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 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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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 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 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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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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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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Anti-Nuclear Antibody

Anti-Nuclear antibody is a well accepted biological clinical laboratory marker of systemic lupus. Range of normal values is 0-0.9 EU. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionEU (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years 9
Autologous HSCT in SLE5.44.73.73.22.72.62.82.52.5

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Anti-Smith-Ribonuclear Protein Antibody

Anti-Smith-Ribonuclear protein antibody is a well accepted biological clinical laboratory marker of systemic lupus.Range of normal values is 0-19 EU. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months and 2 years.

InterventionEU (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years
Autologous HSCT in SLE4951.631.529.828.52016.6725.67

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Cluster of Differentiation 19 (CD19) + Cells

The CD19 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 47-409 u/L. (NCT00076752)
Timeframe: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.

Interventioncells/mL^3 (Mean)
Day 01 month3 months6 months1 year3 years
Autologous HSCT in SLE0.010.236.745.32142.69246.83

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Cluster of Differentiation 3 (CD3) + Cells

The CD3+Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 650-2108 uL. (NCT00076752)
Timeframe: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.

Interventioncells/mL^3 (Mean)
Day 01 month3 months6 months1 year2 years
Autologous HSCT in SLE2.99239.47435.97699.471493.291678.76

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Cluster of Differentiation 4 (CD4) + Cells

The CD4 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 358-1259 uL. (NCT00076752)
Timeframe: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.

Interventioncells/mL^3 (Mean)
Day 01 month3 months6 months1 year2 years
Autologous HSCT in SLE2.58103.37112.8316.25702.87958.03

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Cluster of Differentiation 8 (CD8) + Cells

The CD8 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 194-836 u/L. (NCT00076752)
Timeframe: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.

Interventioncells/mL^3 (Mean)
Day 01 month3 months6 months1 year2 years
Autologous HSCT in SLE0.34138.68318.47334.91736.67674.69

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Extractable Nuclear Antigen (ENA)

Extractable nuclear antigen is a well accepted biological clinical laboratory marker of systemic lupus. Range of normal values is 0-19. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionEU (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE66.964.861.558.551.357.260.650.626.3

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Natural Killer Cells

The natural killer cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 87-505 uL. (NCT00076752)
Timeframe: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.

Interventioncells/mL^3 (Mean)
Day 01 month3 months6 months1 year3 years
Autologous HSCT in SLE0.03117.06116.57123.18158.9115.18

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Platelet Count

The platelet count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 162-380 K/uL. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionK/uL (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE251113166187170226210308272

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Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)

The SLEDAI activity index test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Complete clinical response is defined as complete clinical response in the target organ and no clinical signs of active lupus as determined by a SLEDAI score of ≤3; partial response is at least 50% improvement in general disease activity as measured by SLEDAI. Remission is a SLEDAI score <3 and prednisone <10mg/day. 6+ indicates active disease requiring therapy. A score of 0 indicates a better outcome and a score greater then 6+ indicates a worse outcome. (NCT00076752)
Timeframe: Day -7, day 0, 1 month, 3 months, 6 months, 1 year, 18 months, 2 years and 3 years.

Interventionscores on a scale. (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE4.254.133.631.600000

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT00076752)
Timeframe: 18 months

Interventionparticipants (Number)
Autologous HSCT in SLE8

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White Blood Cells

The white blood cell test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 3.4-9.6 K/uL. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionK/uL (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE6.890.4710.323.844.215.815.247.176.34

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Relapse-free Complete Clinical Response

Complete clinical response is defined as complete clinical response in the target organ and no clinical signs of active lupus as determined by a Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score of ≤3; prednisone ≤10mg/day at 6 months and ≤5mg/day at 12 months or later. (NCT00076752)
Timeframe: 60 months

InterventionMonths (Median)
Autologous HSCT in SLE54

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Absolute Lymphocyte Count

The absolute lymphocyte count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 0.45-4.9 K/uL. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionK/uL (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE0.540.00650.420.530.821.751.81.81.75

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Absolute Neutrophil Count

The absolute neutrophil count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 1.29-7.5 K/uL. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionK/uL (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE5.660.459.112.722.783.442.724.043.77

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Anti-Double Stranded Deoxyribonucleic Acid (DNA) Antibody

Anti-Double stranded deoxyribonucleic acid antibody is a well accepted biological clinical laboratory marker especially specific for systemic lupus. Range of normal values is 0-24 IU. (NCT00076752)
Timeframe: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.

InterventionIU (Mean)
Day -7Day 01 month3 months6 months1 year18 months2 years3 years
Autologous HSCT in SLE17.38.80000000

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Number of partiCIPANTS WITH OBJECTIVE RESPONSE AS MEASURED BY RECIST

Objective response as measured by radiological and physical examination using RECIST criteria. (NCT00085423)
Timeframe: Response at 12 weeks

Interventionparticipants (Number)
Group 13

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Number of Participants With Lymphocyte Recovery as Measured by Blood Count

Lymphocyte recovery to a greater than 1000 cells/mcL was determined by differential peripheral blood cell counts on sequential days as noted in time frame. (NCT00085423)
Timeframe: on days 1-15, weekly for 2 weeks, and then every 2-3 months

Interventionparticipants (Number)
Group 118

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Time to Progression as Measured by RECIST

Clinical outcome used the National Cancer Institute's Response Evaluation Criteria in Solid Tumors (RECIST)1.0. (NCT00085423)
Timeframe: From date of randomization until the first date of documented progression or date of death from any cause, which ever came first, assessed up till 100 months

Interventionyears (Mean)
Group 1.3

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Mean EQ-5D™ Index Scores to Measure Quality of Life at End of Treatment

EQ-5D™ is a trademark of the EuroQol Group. EQ-5D™ is a standardized instrument for use as a measure of health outcome. The questionnaire asks about health status along 5 dimensions: mobility, self care, usual activities, pain/discomfort, and anxiety/depression, which are rated at three possible levels (no problems, some problems, extreme problems). The score ranges from best (+1) to worst (-0.59). (NCT00086580)
Timeframe: up to month 6 (end of treatment)

Interventionunits on a scale (Mean)
Combination Arm (FluCAM)0.8049
Fludarabine Alone0.7749

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Participant Best Response to Treatment Assessed by the Independent Response Review Panel (IRRP)

Participants were evaluated by the IRRP according to National Cancer Institute (NCI) 1996 response criteria. The best response observed during the study is summarized. Response categories include Complete Response (CR) with normal physical exam, marrow cells and blood values, Partial Response (PR) with a >= 50% decrease from baseline in lymphocytes, lymphadenopathy and liver or spleen exam, Stable Disease (SD) without significant progression from baseline, or Progressive Disease (PD) with increased size/number of nodes, size of liver or spleen, increase in lymphocytes, aggressive histology. (NCT00086580)
Timeframe: Up to 9 months

,
Interventionparticipants (Number)
Overall Response (CR+PR)Complete Response (CR)Partial Response (PR)Progressive Disease (PD)Stable Disease (SD)Not Evaluable (NE)
Combination Arm (FluCAM)1372111661213
Fludarabine Alone126711992111

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Total Volume of Distribution (Vss) of Fludarabine

The total volume of distribution (Vss) is the apparent volume in which fludarabine is distributed immediately after it has been injected intravenously and equilibrated between plasma and the surrounding tissues. Total volume of distribution (Vss) of fludarabine is derived from plasma concentration versus time data. (NCT00086580)
Timeframe: month 4 (cycle 4): first day of dosing (pre-dose, 0.5 hr end of infusion), second day of dosing (pre-dose, 0.5 hr end of infusion), third day of dosing (pre-dose, 0.25 hr, 0.5 hr end of infusion, 1,2,3,4,6,24,48,72 hr after start of fludarabine infusion)

Interventionliters (Mean)
Combination Arm (FluCAM)117
Fludarabine Alone172

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Participants With Minimal Residual Disease (MRD)

MRD negativity in this report was defined by the absence of tumor cells in bone marrow, using 4-color flow cytometry. MRD was assessed in participants with a clinical complete response (CR) or partial response (PR) without recovery of blood counts. MRD represents a very positive outcome. (NCT00086580)
Timeframe: up to 9 months

Interventionparticipants (Number)
Combination Arm (FluCAM)6
Fludarabine Alone0

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Mean Systemic Clearance (CL) of Fludarabine

Clearance of drug from plasma is affected by the absorption, distribution, metabolism and elimination of the drug. Mean systemic clearance of fludarabine is derived from plasma concentration versus time data. (NCT00086580)
Timeframe: month 4 (cycle 4): first day of dosing (pre-dose, 0.5 hr end of infusion), second day of dosing (pre-dose, 0.5 hr end of infusion), third day of dosing (pre-dose, 0.25 hr, 0.5 hr end of infusion, 1,2,3,4,6,24,48,72 hr after start of fludarabine infusion)

Interventionliters/hour (Mean)
Combination Arm (FluCAM)9.46
Fludarabine Alone9.54

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Mean EuroQol Visual Analogue Scale (EQ-VAS) Scores to Measure Quality of Life at End of Treatment

"The EuroQol Visual Analogue Scale (EQ-VAS) was also used to capture the self-rating of current health status using a visual thermometer with the end points of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom." (NCT00086580)
Timeframe: up to month 6 (end of treatment)

Interventionunits on a scale (Mean)
Combination Arm (FluCAM)77.1
Fludarabine Alone75.7

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Mean EuroQol Visual Analogue Scale (EQ-VAS) Scores to Measure Quality of Life at Baseline

"The EuroQol Visual Analogue Scale (EQ-VAS) was also used to capture the self-rating of current health status using a visual thermometer with the end points of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom." (NCT00086580)
Timeframe: Day 0 (baseline)

Interventionunits on a scale (Mean)
Combination Arm (FluCAM)70.9
Fludarabine Alone70.2

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Area Under the Curve (AUC) of Fludarabine From (AUC 0-tau)

AUC (0-tau) is the area under the plasma concentration curve for fludarabine over the dosage interval (tau). (NCT00086580)
Timeframe: month 4 (cycle 4): first day of dosing (pre-dose, 0.5 hr end of infusion), second day of dosing (pre-dose, 0.5 hr end of infusion), third day of dosing (pre-dose, 0.25 hr, 0.5 hr end of infusion, 1,2,3,4,6,24,48,72 hr after start of fludarabine infusion)

Interventionng*h/mL (Mean)
Combination Arm (FluCAM)8203
Fludarabine Alone5669

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Mean EQ-5D™ Index Scores to Measure Quality of Life at Baseline

EQ-5D™ is a trademark of the EuroQol Group. EQ-5D™ is a standardized instrument for use as a measure of health outcome. The questionnaire asks about health status along 5 dimensions: mobility, self care, usual activities, pain/discomfort, and anxiety/depression, which are rated at three possible levels (no problems, some problems, extreme problems). The score ranges from best (+1) to worst (-0.59). (NCT00086580)
Timeframe: Day 0 (baseline)

Interventionunits on a scale (Mean)
Combination Arm (FluCAM)0.7959
Fludarabine Alone0.7822

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Maximum Plasma Concentration (Cmax) of Fludarabine

Cmax is the maximum plasma concentration of fludarabine observed. (NCT00086580)
Timeframe: month 4 (cycle 4): first day of dosing (pre-dose, 0.5 hr end of infusion), second day of dosing (pre-dose, 0.5 hr end of infusion), third day of dosing (pre-dose, 0.25 hr, 0.5 hr end of infusion, 1,2,3,4,6,24,48,72 hr after start of fludarabine infusion)

Interventionng/mL (Mean)
Combination Arm (FluCAM)4084
Fludarabine Alone1847

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Kaplan-Meier Estimates of Overall Survival Time for Participants With Rai Stage I-II

Overall survival was defined as the time in days from the date of randomization to the date of death due to any cause plus 1 day for all participants. Results are stated in months and include participants with Rai Stage I or II. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)NA
Fludarabine AloneNA

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Kaplan-Meier Estimates of Overall Survival Time

Overall survival was defined as the time in days from the date of randomization to the date of death due to any cause plus 1 day for all participants. Results are stated in months. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)NA
Fludarabine Alone52.93

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Kaplan-Meier Estimates for Time to Alternative Therapy

Time to alternative therapy was defined as the number of days from the date of randomization to the date of first alternative therapy for chronic lymphocytic leukemia (CLL) or death resulting from any cause. Participants who had not received alternative therapy as of the data cutoff date were censored at the last follow-up visit assessment date plus 1 day. Results are stated in months. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)25.43
Fludarabine Alone22.01

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Kaplan-Meier Estimates for Progression-free Survival (PFS) Based on Independent Response Review Panel (IRRP) for Participants With Rai Stage III-IV

Progression-free survival was defined as the number of days from the date of randomization to the date of first objective documentation of progressive disease (PD) as determined by the treatment-blinded IRRP, or death due to any cause. Results are expressed in months and include participants with Rai stage III or IV. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)20.53
Fludarabine Alone11.51

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Kaplan-Meier Estimates for Progression-free Survival (PFS) Based on Independent Response Review Panel (IRRP) Assessment

Progression-free survival was defined as the number of days from the date of randomization to the date of first objective documentation of progressive disease (PD) as determined by the treatment-blinded IRRP, or death due to any cause. Results are expressed in months. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)23.65
Fludarabine Alone16.48

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Kaplan-Meier Estimates for Duration of Response Assessed by the Independent Response Review Panel (IRRP)

Duration of response was analyzed for participants who achieved a complete response (CR) or partial response (PR) and was defined as the number of days from the first date of documented response to the date of progressive disease as determined by IRRP or death due to any cause. Results are stated in months. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)25.10
Fludarabine Alone19.14

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Kaplan Meier Estimates for Time to Disease Progression Assessed by the Independent Response Review Panel (IRRP)

Time to disease progression was defined as the number of days from the date of randomization to the date of first objective documentation of progressive disease as determined by IRRP. Results are stated in months. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)27.96
Fludarabine Alone18.68

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Kaplan-Meier Estimates of Overall Survival Time for Participants With Rai Stage III-IV

Overall survival was defined as the time in days from the date of randomization to the date of death due to any cause plus 1 day for all participants. Results are stated in months and include participants with Rai Stage III or IV. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)NA
Fludarabine Alone23.52

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Kaplan-Meier Estimates for Progression-free Survival (PFS) Based on Independent Response Review Panel (IRRP) for Participants With Rai Stage I-II

Progression-free survival was defined as the number of days from the date of randomization to the date of first objective documentation of progressive disease (PD) as determined by the treatment-blinded IRRP, or death due to any cause. Results are expressed in months and include participants with Rai stage I or II. (NCT00086580)
Timeframe: Up to 6 years

Interventionmonths (Median)
Combination Arm (FluCAM)23.75
Fludarabine Alone20.76

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Summary of Participants With Adverse Experiences (AEs)

Number of participants with adverse events (AEs). AEs were graded by the investigator using the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 and were assessed for relatedness to study treatment (4 point scale from 'not related' to 'definitely related'). Categories reported include participant counts for treatment-emergent AEs, AEs for infections, serious AEs, AEs causing discontinuation of study drug(s), and deaths. Related AEs for the combination arm can be related to either fludarabine or alemtuzumab. (NCT00086580)
Timeframe: Up to 6 years

,
Interventionparticipants (Number)
At least 1 treatment emergent AEAt least 1 related treatment emergent AEAt least 1 treatment-emergent infectionAt least 1 drug-related infectionAt least 1 serious AEAt least 1 related serious AEDiscontinuation of study drug due to AEDiscontinuation of study drug due to related AEDeathsPatients who died due to a related AEPatients who died within 30 days of the last dose
Combination Arm (FluCAM)1611596744544737321074
Fludarabine Alone1491255830412832241267

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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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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 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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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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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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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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Final Analysis: Time to New Chronic Lymphocytic Leukemia (CLL) Treatment

Time to new CCL treatment was defined as the time from randomization to the first day of new treatment for CCL or death. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1085.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1625.0

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Final Analysis: Time to Overall Survival Event

Overall survival (OS) was determined from the date of randomization to the date of death (OS event) irrespective of cause. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)2056.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)2167.0

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Final Analysis: Percentage of Participants With Complete Response

Complete response was defined as the disappearance of all signs of cancer in response to treatment. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionPercentage of participants (Number)
Fludarabine+Cyclophosphamide (FC)13.4
Fludarabine+Cyclophosphamide+Rituximab (FCR)25.0

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Progression-free Survival (PFS) as Assessed by the Independent Review Committee (IRC)

Progression-free survival as assessed by the IRC was defined as the time between randomization and the date of first documented disease progression, relapse after response, or death from any cause, whichever came first. Patients without a PFS event were censored at their last tumor assessment date. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)660
Fludarabine+Cyclophosphamide+Rituximab (FCR)813

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Number of Participants With Disease-free Survival (DFS) Events

Disease free survival was defined for all patients with a best overall response (BOR) of Complete Response (CR) and measured the time from first documented CR in a sequence of consecutive CRs until documented disease progression, relapse or death from any cause (DFS events). Patients without a DFS event at the time of the analysis (clinical data cut-off) were censored at their last tumor assessment date. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

,
Interventionparticipants (Number)
Patients with eventPatients without event
Fludarabine+Cyclophosphamide (FC)1026
Fludarabine+Cyclophosphamide+Rituximab (FCR)1948

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Number of Participants With Event-free Survival (EFS) Events

Event free survival was measured from the day of randomization to the date of first documented Progressive Disease (PD), relapse after response, start of a new treatment or death from any cause (EFS events). Patients without an EFS event were censored at their last tumor assessment date. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

,
Interventionparticipants (Number)
Patients with eventPatients without events
Fludarabine+Cyclophosphamide (FC)162114
Fludarabine+Cyclophosphamide+Rituximab (FCR)134142

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Number of Participants With Overall Survival (OS) Events

Overall survival was determined from the date of randomization to the date of death (OS event) irrespective of cause. Patients who had not died at the time of the final analysis (clinical data cut-off) were censored at the date of the last contact. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

,
Interventionparticipants (Number)
Patients with eventPatients without events
Fludarabine+Cyclophosphamide (FC)68208
Fludarabine+Cyclophosphamide+Rituximab (FCR)62214

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Number of Participants With Progression-free Survival (PFS) Events Assessed by the Independent Review Committee (IRC)

Progression-free survival as assessed by the IRC was defined as the time between randomization and the date of first documented disease progression, relapse after response, or death from any cause (PFS events), whichever came first. Patients without a PFS event were censored at their last tumor assessment date. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

,
Interventionparticipants (Number)
Patients with eventPatients without events
Fludarabine+Cyclophosphamide (FC)148128
Fludarabine+Cyclophosphamide+Rituximab (FCR)137139

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Final Analysis: Time to Disease-Free Survival Event

Time to disease-free survival (DFS) event was defined as the time from first documented response until the first documented DFS event: disease progression, relapse or death from any cause. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1285.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1803.0

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Overall Survival (OS)

Overall survival was determined from the date of randomization to the date of death irrespective of cause. Patients who had not died at the time of the final analysis (clinical data cut-off) were censored at the date of the last contact. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1580
Fludarabine+Cyclophosphamide+Rituximab (FCR)NA

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Disease-free Survival (DFS)

Disease free survival was defined for all patients with a best overall response (BOR) of Complete Response (CR) and measured the time from first documented CR in a sequence of consecutive CRs until documented disease progression, relapse or death from any cause. Patients without a DFS event at the time of the analysis (clinical data cut-off) were censored at their last tumor assessment date. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1285
Fludarabine+Cyclophosphamide+Rituximab (FCR)1204

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Final Analysis: Time to Progression-Free Survival Event

Time to progression-free survival (PFS) event was defined as the time between randomization and the date of first documented PFS event: disease progression, relapse or death by any cause, whichever came first. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)683.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)969.0

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Final Analysis: Duration of Response

Duration of response was defined as the time between the date of the earliest qualifying response and the date of disease progression or death due to any cause. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)869.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1333.0

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Final Analysis: Time to Event-Free Survival Event

Event free survival (EFS) was defined as the time from the day of randomization to the date of first EFS event: documented disease progression, relapse after response, start of a new treatment or death from any cause. (NCT00090051)
Timeframe: Median observation time was approximately 5 years

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)630.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)932.0

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Event-free Survival (EFS)

Event free survival was measured from the day of randomization to the date of first documented PD, relapse after response, start of a new treatment or death from any cause. Patients without an EFS event were censored at their last tumor assessment date. (NCT00090051)
Timeframe: Mean observation time at time of analysis was approximately 26 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)586
Fludarabine+Cyclophosphamide+Rituximab (FCR)874

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Duration of Response

(NCT00096018)
Timeframe: 4 weeks, every 3 months for 2 years, and then every 4 months for 2 years

Interventionmonths (Mean)
Phase I - Dose Escalation43.3
Phase II30.0

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Overall Responders (Complete and Partial Response)

Criteria for response were based on the Revised National Cancer Institute-sponsored Working Group Guidelines for response, which includes clinical, hematologic, and bone marrow features (Cheson, B.D., et al., National Cancer Institute-sponsored Working Group guidelines for chronic lymphocytic leukemia: revised guidelines for diagnosis and treatment. Blood. 1996;87:4990-97.) (NCT00096018)
Timeframe: 4 weeks, every 3 months for 2 years, and then every 4 months for 2 years

Interventionparticipants (Number)
Phase I - Dose Escalation8
Phase II18

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Clinical Tumor Regression

Tumor regression is defined as a complete response (CR) or partial response (PR) and was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is the disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. (NCT00096382)
Timeframe: Every 4-6 weeks for up to 1 year, and then every 6 months for up to 5 years.

,
InterventionParticipants (Number)
Complete ResponsePartial Response
TBI 200cGy + TIL +HD IL-2, No Prior IL-212
TBI 200cGy + TIL +HD IL-2, Prior IL-219

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Safety

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00096382)
Timeframe: 4 years

InterventionParticipants (Number)
TBI 200cGy + TIL +HD IL-2, Prior IL-223
TBI 200cGy + TIL +HD IL-2, No Prior IL-23

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Lymphoma Progression-free Survival

(NCT00096460)
Timeframe: Three years post-Hematopoietic Stem Cell Transplant (HSCT)

Interventionparticipants (Number)
Autologous Hematopoietic Stem Cell Transplant (HSCT)13
Allogeneic Hematopoietic Stem Cell Transplant (HSCT)6

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Number of Participants With a Complete Response After Treatment With Fludarabine & Rituximab Followed by Alemtuzumab

"A complete response, as defined by the National Cancer Institute Working Group (NCIWG):~- CR: no lymphadenopathy, hepatomegaly, splenomegaly or constitutional symptoms; normal complete blood count; confirmed by bone marrow (BM) aspirate & biopsy" (NCT00098670)
Timeframe: Duration of treatment (up to 13.5 months)

Interventionparticipants (Number)
Alemtuzumab Consolidation38

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Number of Participants With Severe Non-Hematologic Adverse Events During Treatment With Alemtuzumab

"The National Cancer Institute (NCI) Common Toxicity Criteria (CTC) Version 2.0 was used to evaluate toxicity. Severe Adverse events are defined as grade 3, 4 or 5, at least possibly related to treatment.~Grade 1: mild; Grade 2: moderate; Grade 3: Severe; Grade 4: Life Threatening; Grade 5: Death." (NCT00098670)
Timeframe: 6 weeks beginning at study week 36

Interventionparticipants (Number)
Alemtuzumab Consolidation23

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2 Year Survival

Percentage of participants who were alive at 2 years. The 2 year survival was estimated using the Kaplan Meier method. (NCT00098670)
Timeframe: 2 years from registration

Interventionpercentage of participants (Number)
Alemtuzumab Consolidation86

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2 Year Progression Free Survival

Percentage of patients who were alive and progression free at 2 years. The 2-year progression free survival was estimated using the Kaplan Meier method. (NCT00098670)
Timeframe: 2 years from registration

Interventionpercentage of participants (Number)
Alemtuzumab Consolidation72

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Number of Participants With a Complete or Partial Response After Induction Therapy With Fludarabine & Rituximab

"Response, as defined by the National Cancer Institute Working Group (NCIWG):~CR: no lymphadenopathy, hepatomegaly, splenomegaly or constitutional symptoms; normal complete blood count; confirmed by bone marrow (BM) aspirate & biopsy~PR: 50% decrease in peripheral blood lymphocytes, lymphadenopathy, liver/spleen size, presence/absence of constitutional symptoms; plus ≥1 of the following: ≥1500/μL polymorphonuclear leukocytes, >100,000/μL platelets, >11.0 g/dL hemoglobin or 50% improvement for these parameters without transfusions" (NCT00098670)
Timeframe: Up to 9 months

Interventionparticipants (Number)
Alemtuzumab Consolidation92

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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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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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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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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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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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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 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 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 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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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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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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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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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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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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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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Delayed Pneumonia Toxicity Rate

Delayed pneumonia toxicity rate is the proportion of patients who experienced significant pneumonia toxicity defined as nocardia or pneumocystis jiroveci after completing therapy. (NCT00117156)
Timeframe: Assessed after therapy completion incidentally or at a minimum every 6 months for 2 years and then annually up to 4 years.

Interventionproportion of patients (Number)
Fludarabine and Rituximab0.115

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Objective Response Rate

Objective response rate is defined as the proportion of patients who achieve complete remission (CR), complete remission/unconfirmed (CRu) or partial remission (PR) based on Cheson criteria (1999). (NCT00117156)
Timeframe: Assessed after three- and six-cycles of therapy.

Interventionproportion of patients (Number)
Fludarabine and Rituximab.85

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Delayed Bone Marrow Toxicity Rate

Delayed bone marrow toxicity rate is the proportion of patients who experienced significant bone marrow toxicity defined as aplastic anemia or myelodysplastic syndromes (MDS) after completing therapy. (NCT00117156)
Timeframe: Assessed after therapy completion incidentally or at a minimum every 6 months for 2 years and then annually up to 4 years.

Interventionproportion of patients (Number)
Fludarabine and Rituximab0.154

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3.1-Year Progression-Free Survival

3.1-year progression-free survival is the probability of patients remaining alive and progression-free at 3.1 years from study entry estimated using Kaplan-Meier methods. Disease progression was assessed per Cheson criteria (1999). (NCT00117156)
Timeframe: Assessed after 3- and 6-cycles of therapy, every 6 months for 2 years and then annually up to 4 years

Interventionprobability (Number)
Fludarabine and Rituximab0.795

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3.1-Year Overall Survival

3.1-year overall survival is the probability of patients remaining alive 3.1 years from study entry. (NCT00117156)
Timeframe: Assessed after 3- and 6-cycles of therapy, every 6 months for 2 years and then annually up to 4 years

Interventionprobability (Number)
Fludarabine and Rituximab0.874

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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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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 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 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 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 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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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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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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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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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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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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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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Response Rates

(NCT00119392)
Timeframe: Up to 8 years

InterventionParticipants (Count of Participants)
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant)25

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Overall Survival

OS rate at 1 year. (NCT00121186)
Timeframe: 1, 3, and 12 months after protocol treatment, then every 3 months for 1 year, every 6 months for year 2, then annually thereafter until 5 years after registration

Interventionparticipants (Number)
Nonmyeloablative Allogeneic Stem Cell Transplant1

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Progression-free Survival

PFS rate at 1 year. (NCT00121186)
Timeframe: 1, 3, and 12 months after protocol treatment, then every 3 months for 1 year, every 6 months for year 2, then annually thereafter until 5 years after registration

Interventionparticipants (Number)
Nonmyeloablative Allogeneic Stem Cell Transplant1

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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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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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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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Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation

The presence or absence of MRD before and after the bone marrow transplant (BMT) and its frequency distribution will be obtained for each time point separately. (NCT00145626)
Timeframe: Baseline before HSCT, 1 year post HSCT, and up to 5 years post HSCT

InterventionParticipants (Count of Participants)
Patient 172467502Patient 172467503Patient 172467501Patient 272467501Patient 272467502Patient 272467503Patient 372467501Patient 372467502Patient 372467503Patient 472467501Patient 472467502Patient 472467503
Data Not CollectedPositive MRDNegative MRD
Study Participants: 1 Year Post HSCT1
Study Participants: 5 Years Post HSCT1
Study Participants: 1 Year Post HSCT0
Study Participants: Before HSCT0
Study Participants: Before HSCT1
Study Participants: 5 Years Post HSCT0

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Kinetics of Lymphohematopoietic Reconstitution

The lymphohematopoietic reconstitution will be summarized using summary statistics and assessed in a longitudinal manner and analyzed accordingly. (NCT00145626)
Timeframe: From 0-3 months after HSCT through 4-5 years after HSCT

,,,,,,,
Interventioncells *10^3/µl (Median)
CD3 LymphocyteCD3 Gamma DeltaCD4 LymphocyteCD8 LymphocyteCD19 LymphocyteCD56 LymphocyteCD4/CD8 RatioAbsolute Lymphocyte Value
0-3 Months After HSCT0.220.000.130.010.260.362.600.88
1-2 Years After HSCT1.650.700.960.610.450.221.882.59
2-3 Years After HSCT2.880.241.561.050.560.321.383.76
3-4 Years After HSCT2.650.211.331.100.560.341.303.65
3-6 Months After HSCT0.570.040.420.090.480.335.531.29
4-5 Years After HSCT1.870.381.940.700.430.191.302.40
6-9 Months After HSCT1.150.080.920.290.610.203.361.95
9-12 Months After HSCT2.240.091.370.360.520.191.902.90

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Factors Affecting One-year Survival: Minimal Residual Disease (MRD)

Detection of leukemia blasts in bone marrow by flow cytometry (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
Negative for MRDPositive for MRD
Alive21
Expired10
Study Participants31

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Factors Affecting One-year Survival: Median Dose of NK Cells

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

InterventionNKcells X 10^6/kg (Median)
Alive40.2
Expired37.6
Study Participants38.9

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Factors Affecting One-year Survival: Donor Type

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
FatherMotherUncle
Alive250
Expired421
Study Participants671

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Factors Affecting One-year Survival: Disease Status at HSCT

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
Active DiseaseComplete Remission-1Complete Remission-2Progressive DiseaseRelapse
Alive06010
Expired12103
Study Participants18113

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One-year Survival

"The one-year survival of infants with high-risk hematologic malignancies who receive a haploidentical transplant procedure using a total body irradiation (TBI)-excluding conditioning regimen followed by an HLA-nonidentical family donor hematopoietic stem cell (HSC) graft depleted of T cells ex vivo using the CliniMACS CD34+ selection system, with a subsequent infusion of donor NK cells purified ex vivo using the CliniMACS CD3+ depletion and CD56+ enrichment system.~The Kaplan-Meier estimate for one-year survival is reported." (NCT00145626)
Timeframe: One year after transplant

Interventionpercentage of participants (Number)
Study Participants50

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Factors Affecting One-year Survival: Median Dose of CD34

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

InterventionCD34 X 10^6/kg (Median)
Alive35.2
Expired38.3
Study Participants37.8

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Factors Affecting One-year Survival: Median Age of Donor at HSCT

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

InterventionYears (Median)
Alive21.5
Expired27.2
Study Participants25.73

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Factors Affecting One-year Survival: Match N/6 HLA Loci

HLA typing determined the degree of match by looking at 6 different HLA loci. The results indicate the number of the 6 loci that matched for each participant. Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
3/6 HLA Loci4/6 HLA Loci
Alive61
Expired34
Study Participants95

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The Incidence of Grade II-IV Acute Graft vs. Host Disease.

Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion. (NCT00153985)
Timeframe: 3 years

Interventionparticipants (Number)
Transplant for Severe Hemoglobinopathies2

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Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.

Outcome was measured by ANC >500 for three consecutive days prior to day 30 after PBSC infusion, >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion. (NCT00153985)
Timeframe: 3 years

Interventionparticipants (Number)
Transplant for Severe Hemoglobinopathies2

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Immune Reconstitution - Mean Value (1 Year)

Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant. (NCT00167206)
Timeframe: 1 year post-transplant.

InterventionNumber of CD4 cells per microliter (Mean)
Intent-To-Treat860

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Number of Patients Who Exhibited Secondary Graft Failure

Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells. (NCT00167206)
Timeframe: Day 100 after hematopoietic cell transplant

InterventionParticipants (Number)
Intent-To-Treat1

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Number of Patients With Acute Graft Versus-Host Disease (aGVHD)

"Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack." (NCT00167206)
Timeframe: Day 100 after hematopoietic cell transplant

InterventionParticipants (Number)
Intent-To-Treat8

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Number of Patients With Chronic Graft Versus-Host Disease (GVHD)

"Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack." (NCT00167206)
Timeframe: 1 year after hematopoietic cell transplant

InterventionParticipants (Number)
Intent-To-Treat2

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Immune Reconstitution - Mean Value (2 Years)

Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant. (NCT00167206)
Timeframe: at 2 years after transplant

InterventionNumber of CD4 cells per microliter (Mean)
Intent-To-Treat1100

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Number of Patients Alive at 1 Year

Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant. (NCT00167206)
Timeframe: 1 year after transplant

InterventionParticipants (Number)
Intent-To-Treat11

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Number of Patients Alive at 2 Years

Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant. (NCT00167206)
Timeframe: 2 years after transplant

InterventionParticipants (Number)
Intent-To-Treat10

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Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment

Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10^9/Liter. (NCT00167206)
Timeframe: Day 42 after hematopoietic cell transplant

InterventionParticipants (Number)
Intent-To-Treat15

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Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment

"The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is perfect health and 0 is death." (NCT00176852)
Timeframe: 2 years

Interventionunits on a scale (Median)
RIC Bu/Flu (A) (Discontinued)100
MA Bu/Cy (B)100
RIC Cy/Flu/TBI (A2)100

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The Incidence of Chimerism at 6 Months

The number of patients whose blood and/or bone marrow contains > 10% donor cells. (NCT00176852)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)1
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)8

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The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)

The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)

The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. (NCT00176852)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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Disease Free Survival

Number of patients alive without disease 100 days after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)11

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Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment

"The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is perfect health and 0 is death." (NCT00176852)
Timeframe: 1 year

Interventionunits on a scale (Median)
RIC Bu/Flu (A) (Discontinued)97
MA Bu/Cy (B)100
RIC Cy/Flu/TBI (A2)100

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Overall Survival

Number of patients alive 1 year after transplant. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)12

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Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment

"The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is perfect health and 0 is death." (NCT00176852)
Timeframe: pre-transplant

Interventionunits on a scale (Median)
RIC Bu/Flu (A) (Discontinued)100
MA Bu/Cy (B)98
RIC Cy/Flu/TBI (A2)99

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The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)

The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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The Incidence of Chimerism at 1 Year

The number of patients whose blood and/or bone marrow contains > 10% donor cells. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)1
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)8

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Overall Survival

Number of patients alive 100 days after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)12

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The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)

The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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Disease Free Survival

Number of patients alive without disease 1 year after transplant. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)11

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The Incidence of Chimerism at 100 Days

The number of patients whose blood and/or bone marrow contains > 10% donor cells. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)1
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)11

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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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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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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 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 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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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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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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Number of Patients With Disease Recurrence

Number of patients who exhibited disease recurrence at 2 years. (NCT00176878)
Timeframe: 2 years

InterventionParticipants (Number)
Bone Marrow Failure Patients0

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Number of Patients With Grade 2-4 Acute Graft Versus Host Disease

Number of patients with Grade 2, 3 and 4 Acute (normally observed within the first 100 days) Graft Versus Host Disease. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of 1 to a high of 4. Patients with grade IV GVHD usually have a poor prognosis. Grade 2 = moderate, Grade 3 = severe, Grade 4 = life threatening. (NCT00176878)
Timeframe: 100 Days

InterventionParticipants (Number)
Bone Marrow Failure Patients5

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Number of Patients With Succcessful Engraftment After Transplantation

"Number of patients who received non-genotypic identical marrow or cord blood cells using a non-myeloablative preparative regimen and exhibited engraftment at Day 42." (NCT00176878)
Timeframe: 42 Days

InterventionParticipants (Number)
Bone Marrow Failure Patients10

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Number of Patients Alive at Three Years (Survival)

Number of subjects who survived 3 years post-transplant. (NCT00176878)
Timeframe: 3 years

InterventionParticipants (Number)
Bone Marrow Failure Patients6

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Number of Patients With Chronic Graft Versus Host Disease

Number of patients who exhibited chronic (normally occurs after 100 days) Graft Versus Host Disease at 2 years post transplant. Chronic graft-versus-host-disease, over its long-term course, can also cause damage to the connective tissue and exocrine glands. (NCT00176878)
Timeframe: 2 years

InterventionParticipants (Number)
Bone Marrow Failure Patients3

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Number of Patients Alive (Survival) at 2 Years

Calculated from day 1 of transplant to last contact. (NCT00176878)
Timeframe: 2 years

InterventionParticipants (Number)
Bone Marrow Failure Patients6

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Duration of Engraftment of Natural Killer (NK) Cells

NK cell engraftment defined as NK cell chimerism in recipients. (NCT00187096)
Timeframe: Measured at days 2, 7, 14, 21 and 28 after NK cell transplantation, and up to 189 days post transplant as clinically indicated

InterventionDays (Median)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine10

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Overall Survival

"Overall survival is defined as the time relapse from on study date to death with those alive at last follow up date censored. The Kaplan-Meier method was used to compute survival probability estimates and confidence interval was determined by binomial distribution (for no events or all events) or by log hazard method. The binomial interval is based on the number of patients at risk.~The confidence intervals for Arm 1 and Arm 2a were determined by binomial distribution.~The confidence interval for Arm 2b was determined by log hazard method." (NCT00187096)
Timeframe: Up to 2 years post NK cell transplantation

InterventionPercent probability (Number)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine100
Arm 2a: Conditioning Regimen: Cyclophosphamide/Fludarabine0
Arm 2b: Conditioning: Clofarabine/Etoposide/Cyclophosphamide45.0

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Number of Patients Experiencing Grade 3 or 4 Toxicities During Conditioning and up to 100 Days Post-transplant

Document the number of patients experiencing grade 3 or 4 toxicities during conditioning and up to 100 days post-transplant. Toxicities were identified using Common Toxicity Criteria V 3.0 criteria. (NCT00187096)
Timeframe: Beginning at on therapy through 100 days post-transplant

Interventionparticipants (Number)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine2
Arm 2a: Conditioning Regimen: Cyclophosphamide/Fludarabine3
Arm 2b: Conditioning: Clofarabine/Etoposide/Cyclophosphamide11

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Percent of Peak NK Cell Chimerism

The maximum percent of donor NK cell in recipients during a four-week period after NK cell infusion. (NCT00187096)
Timeframe: Days 2, 7, 14, 21 and 28 after NK cell transplantation

Interventionpercent of NK cells (Median)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine7

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Proportion of Patients Experiencing Grade 3 or 4 Toxicities During Conditioning and up to 100 Days Post-transplant

Document the proportion of patients experiencing grade 3 or 4 toxicities during conditioning and up to 100 days post-transplant. Toxicities were identified using Common Toxicity Criteria V 3.0 criteria. (NCT00187096)
Timeframe: Beginning at on therapy through 100 days post-transplant

Interventionproportion of patients (Number)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine0.20
Arm 2a: Conditioning Regimen: Cyclophosphamide/Fludarabine1.00
Arm 2b: Conditioning: Clofarabine/Etoposide/Cyclophosphamide0.917

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Relapse-free Survival

For Arm 1, the efficacy of NK cell transplantation will be reported as the proportion of participants who achieve complete or partial remission. Kaplan-Meier estimates of relapse-free survival and confidence interval was determined by binomial distribution because no events were observed. The binomial interval is based on the number of patients at risk. (NCT00187096)
Timeframe: Up to 2 years post NK cell transplantation

InterventionPercent probability (Number)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine100

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Number of KIR-mismatched NK Cells

Number of KIR-mismatched donor NK cells in recipients' blood at day 2 and day 14 post NK cell infusion. (NCT00187096)
Timeframe: Day 2 and day 14 post NK cell transplantation

Interventioncells/µl (Median)
Day 2Day 14
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine2105,800

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Number of Participants With Evidence of NK Cells Lysing a Target Cell Line (K562)

NK cells in recipient achieving ability to lyse target cell line (K562) within normal range established by donor NK cells. (NCT00187096)
Timeframe: Days 2, 7, 14, 21, and 28 after NK cell transplantation

Interventionparticipants (Number)
Lysed within normal rangeDid not Lyse within normal range
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine100

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Percent of Detectable Donor NK Cells at Day 28

The percent of detectable donor NK cells in recipients at 28 days after NK cell infusion. Three of 10 participants had detectable donor cells at week 4. The results report the percent of detectable cells in the 3 participants. (NCT00187096)
Timeframe: At 28 days

Interventionpercent of donor NK cells (Median)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine29

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Day That Maximum NK Cell Engraftment Was Reached

The time elapsed after transplantation in days until peak KIR-mismatched donor NK cell expansion was reached in recipients (NCT00187096)
Timeframe: Day 0 through Day 28 post NK cell transplantation

Interventionnumber of days (Median)
Arm 1: Conditioning Regimen: Cyclophosphamide/Fludarabine14

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Percentage of Participants With Overall Response at Different Observation Times

Participant had either complete response (CR) or partial response (PR) at different observation times (after 90 days; after 180 days; after 270 days). PR requires for at least 2 months: 50% decrease from Baseline in peripheral blood lymphocytes, lymphadenopathy, liver/spleen size, presence or absence of constitutional symptoms; plus ≥1 of the following: ≥1500/μL polymorphonuclear leukocytes, >100000/μL platelets, >11.0 g/dL hemoglobin, or 50% improvement from Baseline for these parameters without transfusions, nodular CR or persistent anemia/thrombocytopenia unrelated to disease. (NCT00206726)
Timeframe: from first date of confirmed response until relapse, or death, or study data cutoff date, whichever is earlier

Interventionpercentage of participants in response (Number)
After 90 daysAfter 180 daysAfter 270 days
Alemtuzumab Plus Fludarabine76.564.729.4

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Complete Response (CR)

Participants evaluated for therapeutic clinical response according to National Cancer Institute (NCI) response criteria, 28 days after 4 or 6 treatment cycles. Response confirmation involved bone marrow biopsy and aspirate performed 2 months after final treatment. CR requires for at least 2 months: no lymphadenopathy, hepatomegaly, splenomegaly or constitutional symptoms; normal complete blood count (CBC); confirmed by bone marrow aspirate and biopsy 2 months later with lymphocytes <30% of nucleated cells and procedure repeated in 4 weeks if hypocellular. (NCT00206726)
Timeframe: 28 days after last cycle with confirmation 2 months later

InterventionPercentage of participants with CR (Number)
Alemtuzumab Plus Fludarabine8.3

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Overall Survival (OS)

Percentage of participants alive 1 year after the first dose date, described as Kaplan-Meier estimate at 1 year (NCT00206726)
Timeframe: 1 year after start of treatment

InterventionPercentage of participants alive (Number)
Alemtuzumab Plus Fludarabine86.4

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Overall Response (OR)

Participant had either complete response (CR) or partial response (PR) at 28 days after last treatment cycle (date of OR) and at Months 2 follow-up. PR requires for at least 2 months: 50% decrease from Baseline in peripheral blood lymphocytes, lymphadenopathy, liver/spleen size, presence or absence of constitutional symptoms; plus ≥1 of the following: ≥1500/μL polymorphonuclear leukocytes, >100000/μL platelets, >11.0 g/dL hemoglobin, or 50% improvement from Baseline for these parameters without transfusions, nodular CR or persistent anemia/thrombocytopenia unrelated to disease. (NCT00206726)
Timeframe: 28 days after last cycle with confirmation 2 months later

InterventionPercentage of participants with CR or PR (Number)
Alemtuzumab Plus Fludarabine28.3

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Progression-free Survival (PFS)

Percentage of participants who survived progression-free at 1 year, described as Kaplan-Meier estimate at 1 year (NCT00206726)
Timeframe: 1 year after start of treatment

Interventionpercentage alive without progression (Number)
Alemtuzumab Plus Fludarabine48.8

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Number of Participants With Minimal Residual Disease (MRD)

Presence of MRD was assessed by laboratory testing of molecular responses in blood and bone marrow samples. (NCT00206726)
Timeframe: When CR is confirmed

Interventionparticipants (Number)
PositiveNegativeNot Assessed
Alemtuzumab Plus Fludarabine320

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Number of Patients's Who Had Complete Response and Partial Response to the Treatment of Fludarabine and Cyclophosphamide Followed by GM-CSF and Rituximab.

"Complete Response (CR): Disappearance of all clinical evidence of active tumor for a minimum of eight weeks and absence of any symptoms related to the tumor.~Partial Response (PR):50% decrease in the sum of the product diameters of all lesions that persist for at least four weeks. No lesion can increase in size and no new lesion can appear during this period.~Stable disease (SD):A tumor that is neither growing nor shrinking.No new tumors have developed" (NCT00208975)
Timeframe: 6 months

,
Interventionparticipants (Number)
Complete ResponsePartial ResponseStable Disease
Chronic Lymphocytic Leukemia421
Non Hodgkin Lymphoma520

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Number of Subjects Maintaining Partial Response (PR) or Complete Response (CR)

"Response criteria as per the NCI-WG Revised Guidelines for B-CLL~Complete remission:~No lymphadenopathy by physical exam No hepatomegaly or splenomegaly Absence of constitutional symptoms Polymorphonuclear leukocytes > 1,500/uL, Platelets > 100,000/uL, Hemoglobin > 11.0 g/dL Bone marrow aspirate and biopsy normocellular with < 30% lymphocytes Absent lymphoid nodules~Partial remission:~50% decrease in peripheral blood lymphocyte count from the pretreatment baseline value~50% reduction in lymphadenopathy and/or ≥ 50% reduction in the size of the liver and/or spleen AND one or more of the following Polymorphonuclear leukocytes > 1,500/uL or 50% improvement over baseline Platelets > 100,000/uL or 50% improvement over baseline Hemoglobin > 11.0 g/dL or 50% improvement over baseline" (NCT00230282)
Timeframe: 24 weeks

Interventionparticipants (Number)
Fludarabine and Cyclophosphamide, Followed by Alemtuzumab17

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Duration of Response

(NCT00230282)
Timeframe: 105 months

Interventionmonths (Median)
Fludarabine and Cyclophosphamide, Followed by Alemtuzumab38

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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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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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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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Number of Patients Experiencing Graft Failure

Graft versus Host Disease (GVHD) is a frequent complication of allogeneic bone marrow transplant in which the engrafted donor cells attacks the patient's organs and tissue. Acute GVHD (aGVHD) usually occurs during the first three months following an allogeneic BMT. Chronic GVHD (cGVHD) usually develops after the third month post-transplant. Patients may experience one, both or neither. (NCT00253513)
Timeframe: 42 days

Interventionparticipants (Number)
acute graft vs. host disease (aGVHD)chronic graft vs. host disease (cGVHD)
Treosulfan and Fludarabine Conditioning3631

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Number of Subjects Who Are Without Disease at One Year as Indicator of Disease Free Survival.

(NCT00253513)
Timeframe: One year

Interventionparticipants (Number)
Treosulfan and Fludarabine Conditioning58

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Incidence (Percent of Participants) With Nonrelapse Mortality (NRM) by Day 200 (Secondary Phase Only)

NRM (Non relapse mortality) - death not attributed to the primary cancer. (NCT00253513)
Timeframe: 200 days

Interventionpercent of participants (Number)
Treosulfan and Fludarabine Conditioning5

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Infective Event Rate

infective events=temperature >101 without symptoms or temp <101 with symptoms (NCT00254163)
Timeframe: 6 cycles of 28-day for FCR and 8 cycles of 21-day for PCR, or until PD, CR, or intolerable toxicity

Interventionpercentage of infective events (Number)
FCR Arm38
PCR Arm45

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Hematologic Recovery

defined as Hb >11g/dL and a platelet count >100 × 10^3/mm^3 (NCT00254163)
Timeframe: 2 months post-treatment

Interventionpercentage of participants (Number)
FCR Arm3.5
PCR Arm14.1

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Infection Rate

infection=febrile events requiring treatment (NCT00254163)
Timeframe: 6 cycles of 28-day for FCR and 8 cycles of 21-day for PCR, or until PD, CR, or intolerable toxicity

Interventionpercentage of participants (Number)
FCR Arm31.4
PCR Arm36.5

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Mean Absolute Neutrophil Count (ANC) at Post-treatment

mean Absolute Neutrophil Count (ANC) measured 2 months (8-10 weeks) following the last dose of study treatment (NCT00254163)
Timeframe: 2 months post-treatment

Intervention10^3 cells/mm^3 (Mean)
FCR Arm1.7
PCR Arm2.2

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Objective Remission Rate (ORR)

"Complete remission (CR) see Outcome Measure 6. Partial remission (PR) must exhibit criteria 1 and 2 as well as one or more of the remaining features for at least 2 months.~≥50% decrease in peripheral blood lymphocyte count from the pretreatment baseline value.~≥50% reduction in lymphadenopathy.~≥50% reduction in the size of the liver and/or spleen.~Polymorphonuclear leukocytes ≥ 1,500/mm^3 or 50% improvement over baseline.~Platelets >100,000/mm^3 or 50% improvement over baseline.~Hemoglobin >11.0 g/dL or 50% improvement over baseline without transfusions. Nodular partial remission (nPR) is defined as a CR with persistent bone marrow nodules; Objective Remission (OR) = CR + PR + nPR." (NCT00254163)
Timeframe: 6 cycles of 28-day for FCR and 8 cycles of 21-day for PCR, or until PD, CR, or intolerable toxicity

Interventionpercentage of participants (Number)
FCR Arm59.3
PCR Arm49.4

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Percentage of Patients Hospitalized

Percentage of patients who were hospitalized due to any reasons during the study period. (NCT00254163)
Timeframe: 6 cycles of 28-day for FCR and 8 cycles of 21-day for PCR, or until PD, CR, or intolerable toxicity

Interventionpercentage of participants (Number)
FCR Arm35
PCR Arm43.5

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Progression-free Survival (PFS) Rate at 1-year

PFS is measured from the date of randomization to the date of first documented disease progression or date of death, whichever comes first. If a patient neither progresses nor dies, this patient will be censored at last contact date (NCT00254163)
Timeframe: 12 months after registered.

Interventionprobability of Progression-free Survival (Number)
FCR Arm0.86
PCR Arm0.84

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Progression-free Survival (PFS) Rate at 2-year

PFS is measured from the date of randomization to the date of first documented disease progression or date of death, whichever comes first. If a patient neither progresses nor dies, this patient will be censored at last contact date. (NCT00254163)
Timeframe: 24 months after registered.

InterventionProbability of Progression-free Survival (Number)
FCR Arm0.72
PCR Arm0.63

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Complete Remission (CR)

"Definitions of response is evaluated using guidelines proposed by the National Cancer Institute-Sponsored Working Group for Chronic Lymphocytic Leukemia.~Complete remission (CR) requires all of the following for a period of at least 2 months:~Absence of lymphadenopathy by physical examination and appropriate radiographic techniques. Lymph nodes must be <1 cm.~No evidence of hepatomegaly or splenomegaly.~Absence of constitutional symptoms.~Normal CBC as exhibited by:~Polymorphonuclear leukocytes ≥ 1,500/mm^3~Platelets > 100,000/mm^3~Hemoglobin > 11.0 g/dL (untransfused)~Bone marrow aspirate and biopsy should be performed 2 months after clinical and laboratory results demonstrate that all of the requirements listed in 1-4 have been met to demonstrate that a CR has been achieved. The marrow sample must be at least normocellular for age, with less than 30% of the nucleated cells being lymphocytes.~Lymphoid nodules should be absent." (NCT00254163)
Timeframe: 6 cycles of 28-day for FCR and 8 cycles of 21-day for PCR, or until PD, CR, or intolerable toxicity

Interventionpercentage of participants (Number)
FCR Arm14.0
PCR Arm7.1

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Clinical Response Rate at 6 Months

Clinical Response Rate (combined morphological [NCI WG criteria] + flow cytometry criteria) at 6 months following treatment. Courses will be repeated every 28 to 42 days (+/- 7 days) depending on recovery of peripheral blood counts and toxicities for a maximum of 6 courses. Patients will be evaluated for response after 3 and 6 courses. Bone marrow biopsies will be performed at the end of Cycles 3 and 6 of chemotherapy. (NCT00254410)
Timeframe: End of Cycle 6

InterventionParticipants (Count of Participants)
FCM-R + Pegylated Filgrastim28

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Clinical Response Rate at 3 Months

Clinical Response Rate (combined morphological [NCI Working Group (WG) criteria] + flow cytometry criteria) at 3 months following treatment. Courses will be repeated every 28 to 42 days (+/- 7 days) depending on recovery of peripheral blood counts and toxicities for a maximum of 6 courses. Patients will be evaluated for response after 3 and 6 courses. Bone marrow biopsies will be performed at the end of Cycles 3 and 6 of chemotherapy. (NCT00254410)
Timeframe: End of cycle 3

InterventionParticipants (Count of Participants)
FCM-R + Pegylated Filgrastim29

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Molecular Response Rate at 3 Months

Molecular response rate (PCR for immunoglobulin heavy chain (IgH) rearrangements) at 3 months following treatment. Courses will be repeated every 28 to 42 days (+/- 7 days) depending on recovery of peripheral blood counts and toxicities for a maximum of 6 courses. Patients will be evaluated for response after 3 and 6 courses. Bone marrow biopsies will be performed at the end of Cycles 3 and 6 of chemotherapy. (NCT00254410)
Timeframe: End of cycle 3

InterventionParticipants (Count of Participants)
FCM-R + Pegylated Filgrastim17

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Molecular Response Rate at 6 Months

Molecular response rate (PCR for IgH rearrangements) at 6 months following treatment. Courses will be repeated every 28 to 42 days (+/- 7 days) depending on recovery of peripheral blood counts and toxicities for a maximum of 6 courses. Patients will be evaluated for response after 3 and 6 courses. Bone marrow biopsies will be performed at the end of Cycles 3 and 6 of chemotherapy. (NCT00254410)
Timeframe: End of 6 months

InterventionParticipants (Count of Participants)
FCM-R + Pegylated Filgrastim10

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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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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 Experiencing Major Infections

Number of participants experiencing Major Infections by the end of treatment (NCT00258427)
Timeframe: Day 1 through 1 year post-transplant

InterventionParticipants (Count of Participants)
Marrow Isolex3
USB Arm8
Marrow Clinimacs2
Sibling withoutCliniMACS1

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Number of Participants Experiencing Acute Graft-Versus-Host Disease

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00258427)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm1
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Acute Graft-Versus-Host Disease

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00258427)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host. (NCT00258427)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host. (NCT00258427)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Graft Failure

Graft failure is defined as absolute neutrophil count( ANC ) <5 x 10^8/L by day 30. (NCT00258427)
Timeframe: Day 30

InterventionParticipants (Count of Participants)
Marrow Isolex1
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Overall Survival

Overall Survival - Number of patients alive at 1 year post transplant (NCT00258427)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Marrow Isolex1
USB Arm4
Marrow Clinimacs1
Sibling withoutCliniMACS1

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Number of Participants Experiencing Relapse

Patients with leukemia will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. (NCT00258427)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm2
Marrow Clinimacs1
Sibling withoutCliniMACS0

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Median Time to Disease Relapse (Months)

Follow-up continued every 3 months after the allogeneic natural killer (NK) cell infusion, unless they were transplanted, relapsed or had progressive disease. Time in months to relapse of disease is calculcated from 1st day of treatment with NK cells. Relapse occurs when leukemia is detected in bone marrow or blood. (NCT00274846)
Timeframe: From 1st Day of treatment until death or receipt of bone marrow transplant.

InterventionMonths (Median)
Patients Achieving Complete Remission - Responders7.3

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Overall Survival Time of Patients With Complete Remission

Median number of months patients were alive after NK cell infusion. (NCT00274846)
Timeframe: From Day 1 of Treatment until death or patient received bone marrow transplant.

InterventionMonths (Median)
Patients Achieving Complete Remission - Responders13

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Number of Patients With Natural Killer (NK) Cell Expansion

Evaluation of expansion of donor allogeneic natural killer (NK) cells at day 14 following infusion (>100 donor-derived NK cells per uL of patient blood detectable at day +14). (NCT00274846)
Timeframe: Study Day 14

InterventionParticipants (Number)
Patients With Relapsed/Refractory AML - Evaluable Group2

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Number of Patients With Complete Remission

Clinical response is determined by achievement of a complete remission (CR) as judged by morphological criteria (< 1% blasts in bone marrow with neutrophil recovery). (NCT00274846)
Timeframe: Day 28-35

InterventionParticipant (Number)
Patients With Relapsed/Refractory AML - Evaluable Group2

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Survival

Survival (NCT00278512)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Autologous Stem Cell Transplant6
Allogeneic Stem Cell Transplant0

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Overall Survival Rate

The percentage of participants who are still alive. (NCT00280241)
Timeframe: Five years after starting rituximab, cyclophosphamide and fludarabine

Interventionpercentage of participants (Number)
FLUDARABINE, CYCLOSPHOSPHAMIDE AND RITUXIMAB85.5

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Tolerability of Rituximab, Cyclophosphamide and Fludarabine in Patients With Previously Untreated CLL/SLL

The number of patients who experience any grade 3-5 toxicity. (NCT00280241)
Timeframe: Duration of treatment on study

Interventionparticipants (Number)
FLUDARABINE, CYCLOSPHOSPHAMIDE AND RITUXIMAB42

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Duration of Response

The length of time for which the complete response is maintained. (NCT00280241)
Timeframe: From complete response to the time of progressive disease, death or last clinical examination

InterventionMonths (Median)
FLUDARABINE, CYCLOSPHOSPHAMIDE AND RITUXIMAB22.3

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Efficacy of Rituximab, Cyclophosphamide and Fludarabine in Patients With Previously Untreated CLL/SLL

The number of patients who experience a complete clinical response. (NCT00280241)
Timeframe: Three months after the sixth cycle (9 months)

Interventionparticipants (Number)
FLUDARABINE, CYCLOSPHOSPHAMIDE AND RITUXIMAB46

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Final Analysis: Time to Overall Survival Event

Overall survival (OS) was defined as the time between randomization and the date of death due to any cause. (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)2613.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)NA

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Final Analysis: Time to Progression-free Survival Event

Progression-free survival was defined as the time between randomization and the date of first documented disease progression, relapse or death by any cause, whichever came first. (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)998.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1703.0

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Event-free Survival (EFS)

Event-free survival (EFS) was defined as the time between randomization and the date of disease progression, relapse, start of new CLL treatment or death by any cause. (NCT00281918)
Timeframe: Median observation time at time of analysis was approximately 21 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)947.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1212.0

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Final Analysis: Duration of Response

Duration of response was defined as the time from the first documented Complete Response, Partial Response to disease progression or death by any cause. (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1102.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1718.0

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Final Analysis: Time to Disease-free Survival (DFS) Event in Participants With Complete Response (CR)

CR is defined by at least 8 weeks of: 1)Absence of lymphadenopathy 2)No hepatomegaly or splenomegaly 3)Absence of B-symptoms 4)Normal blood count 5)Bone marrow aspirate and biopsy 8 weeks after the clinical and laboratory results demonstrated that a CR was achieved. A marrow sample had to be normocellular for age with less than 30% lymphocytes. Lymphoid nodules had to be absent. If marrow was hypocellular,a repeat biopsy was taken 4 weeks later and samples were re-reviewed in conjunction with the prior pathology. DFS was calculated from time of CR to relapse or death (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1488.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1854.0

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Overall Survival (OS)

Overall survival (OS) was defined as the time between randomization and the date of death due to any cause. Median OS was not reached. (NCT00281918)
Timeframe: Median observation time at time of analysis was approximately 21 months

,
InterventionDays (Number)
Minimum number of days to an eventMaximum number of days to an event
Fludarabine+Cyclophosphamide (FC)51373
Fludarabine+Cyclophosphamide+Rituximab (FCR)41372

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Final Analysis: Percentage of Participants With Complete Response (CR) and Partial Response

CR is defined by at least 8 weeks of: 1)Absence of lymphadenopathy 2)No hepatomegaly or splenomegaly 3)Absence of B-symptoms 4)Normal blood count 5)Bone marrow aspirate and biopsy 8 weeks after the clinical and laboratory results demonstrated that a CR was achieved. A marrow sample had to be normocellular for age with less than 30% lymphocytes. Lymphoid nodules had to be absent. If marrow was hypocellular,a repeat biopsy was taken 4 weeks later and samples were re-reviewed in conjunction with the prior pathology. Partial response is defined as a decrease in the size of a tumor, or in the extent of cancer in the body, in response to treatment. (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionPercentage of participants (Number)
Fludarabine+Cyclophosphamide (FC)72.4
Fludarabine+Cyclophosphamide+Rituximab (FCR)85.8

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Final Analysis: Time to Event-free Survival Event

Event-free survival was defined as the time between randomization and the date of disease progression, relapse, start of new Chronic Lymphocytic Leukemia treatment or death by any cause. (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)951.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1666.0

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Final Analysis: Time to New Treatment for Chronic Lymphocytic Leukemia(CLL)

The time from randomization to the start of a new treatment. (NCT00281918)
Timeframe: Median observation time was approximately 66.4 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)1455.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)2082.0

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Disease-free Survival (DFS) of Patients With Confirmed Complete Response (CR).

CR is defined by at least 8 weeks of: 1)Absence of lymphadenopathy 2)No hepatomegaly or splenomegaly 3)Absence of B-symptoms 4)Normal blood count 5)Bone marrow aspirate and biopsy 8 weeks after the clinical and laboratory results demonstrated that a CR was achieved. A marrow sample had to be normocellular for age with less than 30% lymphocytes. Lymphoid nodules had to be absent. If marrow was hypocellular,a repeat biopsy was taken 4 weeks later and samples were re-reviewed in conjunction with the prior pathology. DFS was calculated from time of CR to relapse or death. Median DFS was not reached. (NCT00281918)
Timeframe: Median observation time at time of analysis was approximately 21 months

,
InterventionDays to an event (Number)
Minimum number of days to an eventMaximum number of days to an event
Fludarabine+Cyclophosphamide (FC)841164
Fludarabine+Cyclophosphamide+Rituximab (FCR)911226

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Progression-free Survival (PFS)

Progression-free survival (PFS) was defined as the time between randomization and the date of first documented disease progression, relapse or death by any cause, whichever came first. (NCT00281918)
Timeframe: Median observation time at time of analysis was approximately 21 months

InterventionDays (Median)
Fludarabine+Cyclophosphamide (FC)981.0
Fludarabine+Cyclophosphamide+Rituximab (FCR)1212.0

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Percentage of Participants With ≥90 Percent Donor-derived Hematopoeisis Around 100 Days Post Transplantation

The percentage of participants with ≥90 percent donor-derived hematopoeisis was assessed around day +100 using peripheral blood chimerism. (NCT00282282)
Timeframe: 100 days

Interventionpercentage of participants (Number)
Tacrolimus and Sirolimus78

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Incidence of Grade II-IV Acute GVHD (aGVHD) Developing by Day 100 Following Non-myeloablative PBSC Transplantation Using Tacrolimus and Sirolimus.

All participants received tacrolimus and sirolimus in this one arm study. There were no participants considered unevaluable for this measure (deceased prior to day 100). The total number of people who developed grade II-IV aGVHD before day 100 are reported here. (NCT00282282)
Timeframe: 100 days

Interventionparticipants (Number)
Tacrolimus and Sirolimus5

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Disease Response.

Disease response was assessed as 2 year progression-free survival. The median follow-up time was 1.84 years. The percentage of participants with who reached this timepoint with no disease progression are reported. (NCT00282282)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Tacrolimus and Sirolimus GVHD Prophylaxis48

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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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Median Progression Free Survival

Progression-free survival time is defined as time from registration/randomization to the date of progression or death from any cause. Median Progression Free Survival estimated by the method of Kaplan and Meier, and accompanied by 95% confidence interval. (NCT00290511)
Timeframe: up to 5 years

Interventionmonths (Median)
R-FIND + Zevalin84

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Overall Survival

OS is defined as the interval from first dose of study treatment to the date of death, irrespective of the cause of death; subjects still alive will be censored at the date of the last contact. Median Overall Survival was estimated by the method of Kaplan and Meier, and accompanied by 95% confidence interval. (NCT00290511)
Timeframe: up to 5 years

Interventionmonths (Number)
R-FIND + Zevalin143

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Percentage of Participants With Overall Survival Rate at 10 Years

OS is defined as the interval from first dose of study treatment to the date of death, irrespective of the cause of death; subjects still alive will be censored at the date of the last contact. Overall Survival Rate estimated by the method of Kaplan and Meier. (NCT00290511)
Timeframe: 10 years

Interventionpercentage of participants (Number)
R-FIND + Zevalin69

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Number of Participants With Time to Progression (TTP)

Regimen regarded as a success if median TTP can be prolonged to 36 months or greater. Time to Progression measured by the method of Kaplan and Meier, and accompanied by 95% confidence interval. Complete Response (CR) defined as those who achieve a normal state which includes no detectable evidence of disease on x-rays and Partial Response (PR) is defined as a 50% or more reduction in the sum of the products of the diameters of the 6 largest measurable lesions. No new sites of disease. av (NCT00290511)
Timeframe: baseline, 2 and 4 courses, approximately month 8, 9, and 12 months, then restaging every 4 months, then at 2 years every 4-6 months, then yearly until progressive disease or lost to follow up, average of 10 years

InterventionParticipants (Count of Participants)
Complete Response (CR)Partial Response (PR)
R-FIND + Zevalin424

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Tolerance and Efficacy of Maintenance Therapy With Rituximab

"To assess the tolerance and efficacy of maintenance therapy with rituximab. Participants proceeded to the rituximab maintenance program of 6 treatments comprising 1 every 2 months. CR: defined as those who achieve a normal state which includes no detectable evidence of disease on x-rays. CRu: defined as CR unconfirmed on the basis of minimal residual abnormalities on x-ray such as a residual mass <25% of original measurement, and or residual indeterminate bone marrow aggregates. PR: a 50% or more reduction in the sum of the products of the diameters of the 6 largest measurable lesions. No new sites of disease. Progressive disease (PD) is also defined by the appearance of new lymph nodes, of other new or worsening sites of disease, such as > 50% increase in the size of liver and/or spleen, or a > 50% increase in absolute number of circulating lymphocytes." (NCT00290511)
Timeframe: cycle 1 and every 2 weeks thereafter until completion of therapy, an average of 10 years

InterventionParticipants (Count of Participants)
Complete Response (CR)Progression Disease (PD)
R-FIND + Zevalin383

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Tolerance and Efficacy of Maintenance Therapy With Yttrium-90 Ibritumomab Tiuxetan (YIT)

"To assess the tolerance and efficacy of maintenance therapy with YIT. CR: defined as those who achieve a normal state which includes no detectable evidence of disease on x-rays. CRu: defined as CR unconfirmed on the basis of minimal residual abnormalities on x-ray such as a residual mass <25% of original measurement, and or residual indeterminate bone marrow aggregates. PR: a 50% or more reduction in the sum of the products of the diameters of the 6 largest measurable lesions. No new sites of disease. Progressive disease (PD) is also defined by the appearance of new lymph nodes, of other new or worsening sites of disease, such as > 50% increase in the size of liver and/or spleen, or a > 50% increase in absolute number of circulating lymphocytes." (NCT00290511)
Timeframe: cycle 1 and every 2 weeks thereafter until completion of therapy, an average of 10 years

InterventionParticipants (Count of Participants)
Complete Response (CR)Progressive Disease
R-FIND + Zevalin371

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Progression Free Survival at 10 Years

Progression-free survival time is defined as time from registration/randomization to the date of progression or death from any cause. PFS was estimated by the method of Kaplan and Meier, and accompanied by 95% confidence interval. (NCT00290511)
Timeframe: 10 years

Interventionmonths (Number)
R-FIND + Zevalin49

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Disease-free Survival With Correction of Disease at One Year Post Transplantation

"Patients deemed alive and well at follow-up timepoint later than 1-year post-transplantation" (NCT00301834)
Timeframe: 1 year post-transplantation

Interventionparticipants (Number)
Single Arm - Transplant Pre-conditioning Per Study Protocol22

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Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation

Peripheral blood chimerism studies were performed by quantitative real time polymerase chain reaction (qPCR) evaluation of differential short tandem repeat DNA sequences (NCT00301834)
Timeframe: 6 weeks post-transplant

Interventionparticipants (Number)
Single Arm - Transplant Pre-conditioning Per Study Protocol31

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Cytomegalovirus (CMV) Viral Infection and Disease Symptoms

polymerase chain reaction testing for presence of CMV weekly until at least day +100 then every 2 weeks until T-cell reconstitution as defined by cluster of differentiation 4 (CD4) > 200 cells/mm3. Median time to T-cell reconstitution was 6 months. (NCT00301834)
Timeframe: Up to one year post-transplant

,
Interventionparticipants (Number)
Positive CMV Viral Load AssaySymptomatic CMV disease
CMV Seronegative Participants10
CMV Seropositive Participants120

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Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation

(NCT00301834)
Timeframe: 1 year post-transplantation

Interventionparticipants (Number)
Grade 3-4 acute Graft-Versus-Host DiseaseGrade 3-4 mucositis
Single Arm216

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Treatment-related Mortality at 100 Days and 1 Year Post Transplantation

(NCT00301834)
Timeframe: 100 days and 1 year

Interventionparticipants (Number)
Transplantation-related mortality 0-100 daysTransplantation-related mortality 100-365 days
Single Arm - Transplant Pre-conditioning Per Study Protocol21

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Number of Patients With Graft Failure

Number of patients with graft failure defined as <500 donor neutrophils count by day 28 in the absence of residual or relapsed leukemia (NCT00303667)
Timeframe: Day 28

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema1
SCT w/Donor Natural Killer Cells - Extended Schema4

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Disease-free Survival at 1 Year

Number of patients alive without evidence of disease at 1 year after transplant (NCT00303667)
Timeframe: 1 Year

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema0
SCT w/Donor Natural Killer Cells - Extended Schema3

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Disease-free Survival at 6 Months

Number of patients alive without evidence of disease at 6 months after transplant (NCT00303667)
Timeframe: Month 6

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema0
SCT w/Donor Natural Killer Cells - Extended Schema4

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In Vivo Expansion of a Donor NK Cells NK Cell Product

Number of patients with in vivo expansion of donor NK cells. In vivo expansion of NK cell is defined as detection of >100 donor-derived NK cells per microliter of blood. (NCT00303667)
Timeframe: 12 - 14 days after NK cell infusion

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Extended Schema19

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Incidence of 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 (NCT00303667)
Timeframe: 1 Year

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema0
SCT w/Donor Natural Killer Cells - Extended Schema0

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Incidence of Grade III-IV Acute Graft Versus Host Disease

Grade III-IV acute graft versus host disease is a severe short term complication created by infusion of donor cells into a foreign host (NCT00303667)
Timeframe: Month 6

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema0
SCT w/Donor Natural Killer Cells - Extended Schema0

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Incidence of Post-transplant Lymphoproliferative Disorder (PTLD)

Post-transplant lymphoproliferative disorder (PTLD) is a virally-driven cancer of the lymphoid cells caused by immunosuppressive drugs taken after allogeneic stem cell transplantation to prevent or control graft versus host disease. (NCT00303667)
Timeframe: 1 Year

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema0
SCT w/Donor Natural Killer Cells - Extended Schema3

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Number of Patients With Disease Relapse

Disease relapse is the recurrence of leukemia in patients who had cleared their leukemia after treatment. Patients with persistent leukemia are not evaluable for relapse. (NCT00303667)
Timeframe: 1 Year

Interventionparticipants (Number)
SCT w/Donor Natural Killer Cells - Short Schema5
SCT w/Donor Natural Killer Cells - Extended Schema12

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Overall Survival

(NCT00303719)
Timeframe: 1 year

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

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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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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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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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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 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 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 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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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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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 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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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 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 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 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 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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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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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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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 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 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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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00314106)
Timeframe: 33 months

InterventionParticipants (Number)
TBI 1200 cGy + TIL +HD IL-2, Prior IL-218
TBI 1200 cGy + TIL +HD IL-2, no Prior IL-28

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Complete Response

"Determine if the combination of high dose aldesleukin, reinfused cells after lymphocyte depleting chemotherapy and 1200 cGy total body irradiation (TBI) is able to be associated with a modest fraction of patients with metastatic melanoma who can experience a complete response to therapy.~Complete response (CR) is a disappearance of all target lesions." (NCT00314106)
Timeframe: 33 months

InterventionParticipants (Number)
TBI 1200 cGy + TIL +HD IL-2, Prior IL-27
TBI 1200 cGy + TIL +HD IL-2, no Prior IL-23

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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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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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Non-relapse Mortality

(NCT00322101)
Timeframe: At 100 days

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)0
Arm II (Myeloablative Regimen)0

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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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Overall Survival

(NCT00322101)
Timeframe: At 2 years

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)6
Arm II (Myeloablative Regimen)6

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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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Chronic GVHD

Chronic GVHD is scored according to the BMT CTN MOP. The first day of chronic GVHD onset will be used to calculate cumulative incidence curves. (NCT00326417)
Timeframe: Day 365

Interventionpercentage of participants (Number)
Cyclophosphamide 100mg31.7
Cyclophosphamide 50mg22.5

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Overall Survival (OS)

OS is defined as alive at 1 year, the event is death from any cause. Patients alive at the time of last observation, for statistical purposes, will have a survival time which is censored. (NCT00326417)
Timeframe: Day 365

Interventionpercentage of participants (Number)
Cyclophosphamide 100mg80.5
Cyclophosphamide 50mg97.4

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Disease-free Survival (DFS)

DFS includes graft failure, regimen-related toxicity (RRT), and early death. Graft Failure is defined by lack of neutrophil engraftment (ANC less than 0.5 x 10^9/L for 3 consecutive days on different days). Major RRT is defined as severity of grade 4 in any organ system or grade 3 for pulmonary, cardiac, renal, oral mucosal or hepatic. Early death is defined as death prior to Day 100 post-transplant. (NCT00326417)
Timeframe: Day 100

,
Interventionparticipants (Number)
Graft FailureMajor RRTSurvivalAlive and engrafted
Cyclophosphamide 100mg693935
Cyclophosphamide 50mg343735

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Cumulative Incidence of Graft Failure

Primary and secondary graft failure are included, secondary graft failure is defined by initial neutrophil engraftment followed by subsequent decline in the ANC to less than 0.5 x 10^9/L for three consecutive measurements on different days, unresponsive to growth factor. (NCT00326417)
Timeframe: Day 365

Interventionpercentage of participants (Number)
Cyclophosphamide 100mg14.6
Cyclophosphamide 50mg11.7

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Acute Graft vs Host Disease (GVHD)

All GVHD grades 2-4 will be graded according to the BMT CTN Manual of Procedures (MOP) (NCT00326417)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Cyclophosphamide 100mg26.8
Cyclophosphamide 50mg23.7

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Safety

Here is the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. (NCT00328861)
Timeframe: 11/30/2006 - 7/31/2007

InterventionParticipants (Number)
NK Cells + IL-2: Melanoma7
NK Cells + IL-2: Renal Cell1

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Objective Response

Objective response (complete response (CR) or partial response (PR)) is measured by the Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Complete response (CR) is the disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. (NCT00328861)
Timeframe: very 4-6 weeks for up to 1 year, and then every 6 months for up to 5 years.

,
InterventionParticipants (Number)
Complete ResponsePartial Response
NK Cells + IL-2: Melanoma00
NK Cells + IL-2: Renal Cell00

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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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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 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 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 Experiencing Infections by Day 365

(NCT00352976)
Timeframe: by day 365

InterventionParticipants (Count of Participants)
Treatment With TBI56

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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 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 100 Days

(NCT00352976)
Timeframe: by 100 days

Interventionmg/dL (Mean)
Treatment With TBI71.1

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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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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 100

(NCT00352976)
Timeframe: by day 100

InterventionParticipants (Count of Participants)
Treatment With TBI53

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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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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 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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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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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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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 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 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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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 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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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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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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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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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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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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Severe Venous Occlusive Disease (VOD)/ Sinusoidal Obstructive Syndrome (SOS)

The number of subjects with severe VOD / SOS; severity staged according to criteria set forth by McDonald G.B., Hinds M.S., Fisher L.D., et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med. 1993;118:255-267. Assessed within the first 100 days post transplant. (NCT00361140)
Timeframe: 100 days

Interventionparticipants (Number)
AUC 60000
AUC 75001
AUC 90002

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Non-relapse Mortality

The number of participants dead due to causes unrelated to relapse within the first 100 days post transplant. (NCT00361140)
Timeframe: 100 days

Interventionparticipants (Number)
AUC 60003
AUC 75004
AUC 90002

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Overall Median Number of Days Patients Alive After Treatment

Calculated median number of days of survival (patients alive days after treatment). (NCT00376805)
Timeframe: First Day of Treatment Until Death

InterventionDays (Median)
NK Cells With or Without Total Body Irradiation124

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Number of Patients Who Had Expansion of Natural Killer Cells

Successful Natural Killer (NK) cell expansion is defined as detection of an absolute circulating donor-derived NK cell count of >100 cells/ul of whole blood 14 days after infusion with <5% donor T and B cells in mononuclear population (in metastatic breast cancer patients). (NCT00376805)
Timeframe: Day 14

InterventionParticipants (Number)
NK Cells With or Without Total Body Irradiation0

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Number of Patients by Disease Response

"Defined by the Response Evaluation Criteria in Solid Tumors (RECIST) criteria:~Complete Response (CR: Disappearance of all target lesions~Partial Response (PR): At least a 30% decrease in the sum of the longest diameter (LD) of target lesions~Stable Disease (SD): Neither sufficient shrinkage to qualify for PR or sufficient increase to qualify for PD~Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions of appearance of one or more new lesions~of clinical benefit (CB; stable disease for greater than 6 months." (NCT00376805)
Timeframe: 6 Months, 1 Year

InterventionParticipants (Number)
Stable DiseaseProgressive Disease
NK Cells With or Without Total Body Irradiation42

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Number of Patients Who Died While on Study

Number of patients who died within 100 days and after 100 days of natural killer (NK) treatment with or without total body irradiation. (NCT00376805)
Timeframe: Within 100 days, After 100 days

InterventionParticipants (Number)
Died within 100 daysDied after 100 days
NK Cells With or Without Total Body Irradiation15

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Survival and Non-relapse Mortality at Day +200 Using the Miltenyi Reagent System

Subjects with hematological malignancies receiving a myeloablative conditioning regimen of cyclophosphamide, fludarabine and total body irradiation followed by an infusion of stem cell product prepared using the Miltenyi CliniMacs system for CD34 selection and a delayed T cell depletion add back as donor lymphocyte infusion at day 90. The subjects receiving allogeneic stem cell transplantation will have stem cell product prepared using Miltenyi CliniMacs system to determine the overall survival and non-relapse mortality at day +200. (NCT00378534)
Timeframe: Day 200

Interventionparticipants (Number)
Survival at day 200Non-relapse mortality
T Cell Depletion Transplant Participants434

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Number of Participants Who Developed Limited Chronic GVHD

"Number of incidences of participants who developed Limited Chronic Graft vs Host Disease (GVHD).~Limited disease is characterized by localized skin involvement and/or evidence of hepatic dysfunction.~Limited disease is associated with a favorable outcome without systemic therapy, while extensive disease patients have an unfavorable outcome." (NCT00378534)
Timeframe: 36 months

InterventionParticipants (Count of Participants)
T Cell Depletion Transplant Participants8

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Number of Participants With Relapse of Disease

Number of participants with relapse of disease by day 200 (NCT00378534)
Timeframe: Day 200

InterventionParticipants (Count of Participants)
T Cell Depletion Transplant Participants17

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Number of Participants Who Develop Extensive GVHD

"Number of participants with extensive, Chronic Graft vs Host Disease (GVHD). Extensive chronic GVHD is defined as GVHD occurring after day 100 that did not meet the definition of limited chronic GVHD.~Extensive disease presents either with generalized skin involvement, or with localized skin involvement or hepatic dysfunction plus at least one of the following:~Liver histology showing chronic progressive hepatitis, bridging necrosis, or cirrhosis~Involvement of the eye (Schirmer's test with less than 5 mm wetting) (see Diagnosis and classification of Sjögren's syndrome)~Involvement of minor salivary glands or oral mucosa (as demonstrated on labial or mucosal biopsy specimen)~Involvement of any other target organ" (NCT00378534)
Timeframe: 36 months

InterventionParticipants (Count of Participants)
T Cell Depletion Transplant Participants18

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Number of Participants Who Developed Acute GVHD Grades I, II, III, IV

"Number of participants who developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV as defined by CIMBTR criteria for Organ Stages of Acute GVHD.~Grades are defined as:~Grade I: Skin = Maculopapular rash< 25% of body surface area (BSA); Liver = Total Bilirubin 2-3 mg/dL; Lower GI = stool output/day is 500-999 mL/day.~Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day.~Grade III: Skin = Rash on >50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea > 1500 mL/day.~Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin >15 mg/dL; Lower GI = Severe abdominal pain with or without ileus.~Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening." (NCT00378534)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Acute GVHD, Grade IAcute GVHD, Grade IIAcute GVHD, Grade IIIAcute GVHD, Grade IV
T Cell Depletion Transplant Participants141691

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Participant Overall Response Rate (ORR) at 6 Months Includes Complete Remissions, Partial Remission, or Nodule Partial Remissions.

Complete Remission:Normal exam/No symptoms; Absolute lymphocyte count (ALC)11 g/dL, absolute neutrophil count (ANC)>/=1.5x109/L, & platelet count>100x109/L. Bone marrow:<30% lymphocytes aspirate no biopsy evidence disease; Disappearance palpable lymph nodes/spleen/liver, no new lesions. Partial Remission:ALC reduced 50%,either Hb>11 g/dL or 50% improvement (imp.) in deviation, or ANC>/=1.5x109/L or 50% imp., or platelet>100x109/L or 50% imp. in deviation from normal. Reduced 50% palpable lymph nodes/spleen/liver no new lesions. Nodular Partial Response:ALC11 g/dL, ANC>/=1.5x109/L & platelet count>100x109/L; <30% lymphocytes - bone marrow biopsy aspirate + lymphoid nodules;No palpable lymph nodes/spleen/liver tumors without new lesions. Progressive Disease:50% increase (incr.) ALC>10x109/L twice; tumor lesion incr. 50% over entry or responder size at time max regression &/or appearance new malignant disease; Reappearance bone marrow disease. (NCT00381004)
Timeframe: Baseline to 6 Months

InterventionPercentage of Participants (Number)
FCR + Sargramostim100

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Number of Participants With Overall Response Includes Complete Remissions, Partial Remission, or Nodule Partial Remissions.

Complete Remission:Normal exam/No symptoms; Absolute lymphocyte count (ALC)11 g/dL, absolute neutrophil count (ANC)>/=1.5x109/L, & platelet count>100x109/L. Bone marrow:<30% lymphocytes aspirate no biopsy evidence disease; Disappearance palpable lymph nodes/spleen/liver, no new lesions. Partial Remission:ALC reduced 50%,either Hb>11 g/dL or 50% improvement (imp.) in deviation, or ANC>/=1.5x109/L or 50% imp., or platelet>100x109/L or 50% imp. in deviation from normal. Reduced 50% palpable lymph nodes/spleen/liver no new lesions. Nodular Partial Response:ALC11 g/dL, ANC>/=1.5x109/L & platelet count>100x109/L; <30% lymphocytes - bone marrow biopsy aspirate + lymphoid nodules;No palpable lymph nodes/spleen/liver tumors without new lesions. Progressive Disease:50% increase (incr.) ALC>10x109/L twice; tumor lesion incr. 50% over entry or responder size at time max regression &/or appearance new malignant disease; Reappearance bone marrow disease. (NCT00381004)
Timeframe: Baseline to 6 Months

InterventionParticipants (Number)
Complete Response (CR)Nodular Partial Response (nPR)Partial Response (PD)
FCR + Sargramostim4569

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Number of Participants Progression-free

Participants progression free as measured at six months following start of treatment. Criteria for Progressive Disease (PD): Peripheral blood: 50% increase in ALC with a level > 10 x 109/L on at least 2 occasions 2 weeks apart. Tumor: An increase of a lesion by 50% over the size present at entry on study or for patients who respond, the size at the time of maximum regression and/or the appearance of new areas of malignant disease. Reappearance of bone marrow disease. A deterioration in performance status or increasing symptoms do not constitute disease progression. (NCT00381004)
Timeframe: 6 months or until disease progression if earlier

Interventionparticipants (Number)
FCR + Sargramostim60

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Response Rate Including Complete Response, Partial Response, and Hematological Improvement Assessed by Blood Cell Counts, Number of Blasts in Bone Marrow, and Clinical Evaluation

Bone marrow aspiration and biopsies were performed prior to treatment, during week 3 of the first cycle, at the time of hematologic recovery from all cycles of therapy (defined as neutrophil count >500/mm3 and platelets >20,000/mm3 independently of transfusion), or at any time that leukemia regrowth was suspected. The overall response rate was defined as complete remission, partial remission, or hematologic improvement, lasting for ≥30 days. Given the different subsets of diseases, standardized response criteria were used for CMML (the Myelodysplastic Syndrome International Working Group criteria),33 CMML transforming to acute myeloid leukemia (standard AML response criteria) , accelerated MPN (Giles et al.), and transformation of MPN to secondary AML (Mascarenhas et al.). (NCT00381550)
Timeframe: Up to 4 years

Interventionparticipants (Number)
Arm I18

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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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Incidence of Chronic Graft-versus-host Disease (GVHD)

"Number of patients with chronic graft-versus-host disease at 1 year post transplant. Graft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. Grade I=mild, Grade 2=moderate, Grade 3=severe, Grade 4=life threatening. Chronic GVHD is an extension of acute GVHD." (NCT00392782)
Timeframe: 1 Year

InterventionParticipants (Number)
All Treated Patients4

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Incidence of Disease Relapse

Number of patients with disease at 1 year. (NCT00392782)
Timeframe: 1 Year

InterventionParticipants (Number)
All Treated Patients6

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Incidence of Disease-free Survival

Number of patients alive and without disease at 1 year after transplant. (NCT00392782)
Timeframe: 1 Year

InterventionParticipants (Number)
All Treated Patients18

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Incidence of Grade II-IV Acute Graft-vs-host Disease (GVHD)

"Number of patients with grade II-IV acute graft-versus-host disease at Day 100 post transplant. Graft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. Grade I=mild, Grade 2=moderate, Grade 3=severe, Grade 4=life threatening." (NCT00392782)
Timeframe: Day 100

InterventionParticipants (Number)
All Treated Patients6

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Incidence of Graft Failure

Number of patients with graft failure is defined by lack of neutrophil engraftment by 100 days after transplant in patients surviving a minimum of 14 days. (NCT00392782)
Timeframe: Day 100

InterventionParticipants (Number)
All Treated Patients1

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Overall Survival

Number of patients who were deceased at 1 year post transplant. (NCT00392782)
Timeframe: 1 Year

InterventionParticipants (Number)
All Treated Patients10

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Clinical Tumor Regression

"Response Evaluation Criteria In Solid Tumors (RECIST).~See the protocol Link module for full criteria if desired." (NCT00393029)
Timeframe: 1-11 months

InterventionParticipants (Number)
Metastatic Melanoma & Other Metastatic Cancers1

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In Vivo Survival of T-cell Receptor (TCR) Gene-engineered Cells in Participants

Survival of TCR gene-engineered cells is defined as >10% murine TCR positive cells. (NCT00393029)
Timeframe: 3-12 months

Interventionparticipants (Number)
Metastatic Melanoma2
Other Metastatic Cancers9

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The Number of Participants With Adverse Events

Here are the total number of participants with adverse events. Adverse events were described using the Common Terminology Criteria for Adverse Events (CTCAE) version 3. For the detailed list of adverse events see the adverse event module. (NCT00393029)
Timeframe: events related to all components of the treatment regimen were reported from the start of the non-myeloablative conditioning regiment through 30 days following treatment or resolution.

Interventionparticipants (Number)
Metastatic Melanoma2
Other Metastatic Cancers9

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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 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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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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AUC(0-inf) and AUC(0-672) After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)

AUC is defined as the area under the ofatumumab concentration-time curve as a measure of drug exposure. AUC(0-672) is AUC from start of infusion to 672 hours after start of infusion; AUC(0-inf) is AUC from start of infusion extrapolated to infinity. (NCT00410163)
Timeframe: Visit 2 (Week 0; up to 4 weeks after dose) and Visit 29 (Week 20; up to 9 months after dose)

,
InterventionMilligrams * hour per liter (mg.h/L) (Geometric Mean)
First infusion, AUC(0-inf), n=24, 26First infusion, AUC(0-672), n=24, 26Sixth infusion, AUC(0-inf), n=16, 16Sixth infusion, AUC(0-672), n=20, 19
Ofatumumab 1000 mg + FC19151915397577149019
Ofatumumab 500 mg + FC2453245214523674728

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Percent Change From Screening (Visit 1) in Complement (CH50) Levels at Visit 9 (Week 4)

Blood samples were drawn from participants at Visits 1 and 9 for analysis of complement (CH50) levels. Analysis of CH50 was done in batches, and CH50 levels were measured two hours after the end of study medication infusion. Percent change from Screening (Visit 1, Week -2) = (value at Visit 9 minus value at Visit 1 divided by value at Visit 1) * 100. (NCT00410163)
Timeframe: Visit 1 (Week -2) and Visit 9 (Week 4)

InterventionPercent change in complement levels (Median)
Ofatumumab 500 mg + FC0
Ofatumumab 1000 mg + FC0

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Progression-Free Survival

Progression-free survival (PFS) was defined as the time from randomization until the first radiologically or clinically documented evidence of progression or death due to any cause, if sooner. For participants who were lost to follow-up, PFS was censored at the date of the last attended visit at which the endpoint was assessed. The Kaplan-Meier method was used to estimate PFS. (NCT00410163)
Timeframe: From time of randomization to first documented evidence of disease progression or death due to any cause, whichever came first, assessed over 2 years

Interventionmonths (Median)
Ofatumumab 500 mg + FCNA
Ofatumumab 1000 mg + FC23.5

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Median Percent Change in CD5+CD19+ and CD5+CD20+ Cells in Peripheral Blood From Onset of Course 3 Throughout Follow-up (FU) Compared to Screening

Malignant B cells (CD5+CD19+ and CD5+CD20+) were measured in peripheral blood samples by flow cytometry. Percent change from Screening (Visit 1, Week -2) = (value at indicated visits minus value at Visit 1 divided by value at Visit 1) * 100. Visits 33 and 34 are measured in the number of months from the start of the last infusion. The start of the last infusion could have occurred up to Week 20. (NCT00410163)
Timeframe: Baseline Visit 2 (Week [Wk] 0); Visits 15 (Wk 8), 21 (Wk 12), 25 (Wk 16), 29 (Wk 20), 33 (Month [M] 1 after start of last infusion [LI]), 34 (M 3 after start of LI)

,
Interventionpercent change in cells (Median)
CD5+CD19+ cells, Visit 15 (Wk 8), n=25, 26CD5+CD19+ cells, Visit 21 (Wk 12), n=24, 24CD5+CD19+ cells, Visit 25 (Wk16), n=23, 21CD5+CD19+ cells, Visit 29 (Wk 20), n=22, 19CD5+CD19+ cells, Visit 33 (Wk 24), n=21, 24CD5+CD19+ cells, Visit 34 (Wk 32), n=20, 22CD5+CD20+ cells, Visit 15 (Wk 8), n=25, 26CD5+CD20+ cells, Visit 21 (Wk 12), n=24, 24CD5+CD20+ cells, Visit 25 (Wk16), n=23, 21CD5+CD20+ cells, Visit 29 (Wk 20), n=22, 19CD5+CD20+ cells, Visit 33 (Wk 24), n=21, 24CD5+CD20+ cells, Visit 34 (Wk 32), n=20, 22
Ofatumumab 1000 mg + FC-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00
Ofatumumab 500 mg + FC-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00

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Median Percent Change in Tumor Size From Baseline (Visit 2, Wk 0) at Visits 9, 21, 25, 29, 33, 34, 35, and 37

Tumor size was measured by physical examination of palpable abnormal lymph nodes. Percent change from Baseline (Visit 2, Week 0) = (value at indicated visits minus value at Visit 2 divided by value at Visit 2) * 100. Visits 33, 34, 35, 36, and 37 are measured in the number of months from the start of the last infusion. The start of the last infusion could have occurred up to Week 20. (NCT00410163)
Timeframe: Baseline Visit 2 (Week [Wk] 0); Visits 9 (Wk 4), 21 (Wk 12), 25 (Wk 16), 29 (Wk 20), 33 (Month [M] 1 after start of last infusion [LI]), 34 (M 3 after start of LI), 35 (M 6 after start of LI), 36 (M 9 after start of LI), and 37 (M 12 after start of L

,
Interventionpercent change in tumor size (Median)
Visit 9 (Week 4), n=29, 28Visit 21 (Week 12), n=24, 23Visit 25 (Week 16), n=23, 20Visit 29 (Week 20), n=22, 16Visit 33 (Month 1), n=21, 24Visit 34 (Month 3), n=22, 23Visit 35 (Month 6), n=19, 22Visit 36 (Month 9), n=20, 22Visit 37 (Month 12), n=20, 18
Ofatumumab 1000 mg + FC-70.90-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00
Ofatumumab 500 mg + FC-75.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00-100.00

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Number of Participants (Par.) Who Were Classified as Responders and Non-responders

Par. were evaluated by an IRC in accordance with NCI-WG 1996 guideline. Responders: CR, Nodular Partial Remission (nPR, same as CR, but persistent bone marrow nodules), and Partial Remission (PR, >=50% decrease in lymphocytes from pretreatment baseline (BL) value, >=50% reduction in lymphadenopathy, >=50% reduction of liver/spleen and neutrophils >= 1.5*10^9/L or platelets >100*10^9/L or hemoglobin >11 g/dL (or 50% improvement over BL for neutrophils, platelets, hemoglobin); non-responders: Stable Disease (SD, did not achieve CR/PR, and no PD), Progressive Disease (PD), or Not Evaluable (NE). (NCT00410163)
Timeframe: From start of treatment (Day 1 of Week 0) until 3 months after start of last infusion (up to Week 32)

,
Interventionparticipants (Number)
All respondersResponders, CRResponders, nPRResponders, PRAll Non-respondersNon-responders, SDNon-responders, PDNon-responders, NE
Ofatumumab 1000 mg + FC2215168251
Ofatumumab 500 mg + FC24101137322

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Vss After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)

Vss is defined as the volume of distribution at steady state of ofatumumab. (NCT00410163)
Timeframe: Visit 2 (Week 0; up to 4 weeks after dose) and Visit 29 (Week 20; up to 9 months after dose)

,
Interventionliters (Geometric Mean)
First infusion, Vss, n=24, 26Sixth infusion, Vss, n=16, 16
Ofatumumab 1000 mg + FC4.575.77
Ofatumumab 500 mg + FC3.885.15

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CL After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)

CL is the clearance of drug from plasma, which is defined as the volume of plasma from which the drug is cleared per unit time. (NCT00410163)
Timeframe: Visit 2 (Week 0; up to 4 weeks after dose) and Visit 29 (Week 20; up to 9 months after dose)

,
InterventionMilliliters per hour (mL/h) (Geometric Mean)
First infusion, CL, n=24, 26Sixth infusion, CL, n=20, 19
Ofatumumab 1000 mg + FC1576.7
Ofatumumab 500 mg + FC1226.7

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Duration of Response

The duration of response was defined as the time from the initial response (the first visit at which response was observed) to progression or death. For participants who were lost to follow-up, duration of response was censored at the date of the last attended visit at which the endpoint was assessed. (NCT00410163)
Timeframe: From time of initial response to disease progression or death, whichever came first, assessed over 2 years

Interventionmonths (Median)
Ofatumumab 500 mg + FCNA
Ofatumumab 1000 mg + FCNA

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t1/2 After the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)

t1/2 is defined as terminal half-life and is the time required for the amount of drug in the body to decrease by half. (NCT00410163)
Timeframe: Visit 2 (Week 0; up to 4 weeks after dose) and Visit 29 (Week 20; up to 9 months after dose)

,
Interventionhours (Geometric Mean)
First infusion, t1/2, n=24, 26Sixth infusion, t1/2, n=16, 16
Ofatumumab 1000 mg + FC18.8746
Ofatumumab 500 mg + FC19.4551

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Number of Participants With Positive Human Anti-human Anti Bodies (HAHA) at Visits 1, 21, 35, and 39

HAHA are indicators of immunogenicity to ofatumumab. Blood samples were drawn from participants at Visits 1, 21, 35, and 39 for analysis of HAHA. Analysis of HAHA was done in batches. (NCT00410163)
Timeframe: Visits 1 (Screening, Visit -2), 21 (Week 12), 35 (Month 6), and 39 (Month 18)

,
Interventionparticipants (Number)
Visit 1 (Week -2), n=31, 30Visit 21 (Week 12), n=24, 24Visit 35 (Month 6), n=19, 22Visit 39 (Month 18), n=14, 13
Ofatumumab 1000 mg + FC0000
Ofatumumab 500 mg + FC0000

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Number of Participants (Par.) With Complete Remission (CR), Measured From Start of Treatment Until 3 Months After Last Infusion

"Par. were evaluated for response by an Independent Endpoint Review Committee (IRC) in accordance with the National Cancer Institute-sponsored Working Group (NCI-WG) 1996 guideline. Par. with Complete Remission (CR) were classified as complete responders. As per NCI-WG, CR requires all of the following criteria for a period of >=2 months: absence of lymphadenopathy (all lymph nodes <1.0 centimeters), no hepatomegaly/splenomegaly, absence of constitutional symptoms, lymphocytes <=4.0*10^9/liter (L), neutrophil leukocytes >=1.5*10^9/L, platelets >100*10^9/L, and hemoglobin >11 grams/deciliter." (NCT00410163)
Timeframe: Start of treatment (Day 1 of Week 0) until 3 months after start of last infusion (up to Week 32)

Interventionparticipants (Number)
Ofatumumab 500 mg + FC10
Ofatumumab 1000 mg + FC15

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Number of Participants Classified as Responders Having CR Who Tested Negative for Minimal Residual Disease (MRD)

MRD refers to small number of leukemic cells that remain in the participant during treatment or after treatment when the participant has achieved CR. For all participants who achieved CR, the follow-up bone marrow sample was tested for malignant B cells (CD5+CD19+) to determine if there was any MRD. (NCT00410163)
Timeframe: From start of treatment (Day 1 of Week 0) until 3 months after start of last infusion (up to course 6 or Week 32)

Interventionparticipants (Number)
Ofatumumab 500 mg + FC2
Ofatumumab 1000 mg + FC6

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Number of Participants Who Experienced Any Adverse Event From First Treatment (Visit 2) to Visit 43 (Month 60)

An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. A list of AEs experienced in the study at a frequency threshold of 5% can be found in the AE section. (NCT00410163)
Timeframe: From first treatment (Visit 2) up to Visit 43 (Month 60)

Interventionparticipants (Number)
Ofatumumab 500 mg + FC31
Ofatumumab 1000 mg + FC30

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Ctrough and Cmax at the First Infusion (Visit 2, Week 0) and Sixth Infusion (Visit 29, Week 20)

Cmax is defined as the maximum concentration of drug in plasma samples. Ctrough is defined as the trough plasma concentration (measured concentration at the end of a dosing interval [taken directly before next administration]). No drug is present before the first infusion; therefore, there are no Ctrough results for the first infusion. (NCT00410163)
Timeframe: Visit 2 (Week 0; up to 4 weeks after dose) and Visit 29 (Week 20; up to 9 months after dose)

,
InterventionMilligrams per liter (mg/L) (Geometric Mean)
First infusion, Cmax, n=31, 29Sixth infusion, Cmax, n=22, 19Sixth infusion, Ctrough, n=22, 19
Ofatumumab 1000 mg + FC57.242762.2
Ofatumumab 500 mg + FC67.520119.9

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Number of Participants Who Reported Myelosuppression (Anemia, Leukopenia, Neutropenia, and Thrombocytopenia)

Myelosuppression is one of the expected AEs for FC treatment and is defined as the decrease in the ability of the bone marrow to produce blood cells. The number of participants with myelosuppression was assessed from laboratory measurements with Grades 3(severe)-4 (life-threatening/disabling) (1, mild; 2, moderate; 5, death) according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0. The CTCAE is issued by the National Cancer Institute (NCI) and is the standard classification used for the severity grading scale for AEs in cancer therapy clinical studies. (NCT00410163)
Timeframe: From first treatment (Visit 2) up to Visit 43 (Month 60)

,
Interventionparticipants (Number)
AnemiaLeukopeniaNeutropeniaThrombocytopenia
Ofatumumab 1000 mg + FC822268
Ofatumumab 500 mg + FC623294

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Number of Participants With Progression or Death

Disease progression is characterized by at least one of the following, per the Guidelines for the Diagnosis and Treatment of Chronic Lymphocytic Leukemia: a >=50% increase in the sum of the products of at least two lymph nodes on two consecutive determinations 2 weeks apart (at least one node must be >=2 centimeters); or the appearance of new palpable lymph nodes; or a >=50% increase in liver and/or spleen size (measurement below the costal margin); or the appearance of palpable hepatomegaly or splenomegaly not previously present; or a >=50% increase in the numbers of circulating lymphocytes to at least 5.0 * 10^9/Liter; or transformation to a more aggressive histology (e.g., Richter's syndrome or prolymphocytic leukemia with >55% prolymphocytes). For participants who were lost to follow-up, PFS was censored at the date of the last attended visit at which the endpoint was assessed. The Kaplan-Meier method was used to estimate PFS. (NCT00410163)
Timeframe: From time of randomization to first documented evidence of disease progression or death due to any cause, whichever came first, assessed over 2 years

InterventionParticipants (Number)
Ofatumumab 500 mg + FC3
Ofatumumab 1000 mg + FC7

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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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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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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 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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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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Immune Reconstruction/CD4+ Count at 3 Months

(NCT00425802)
Timeframe: 3 months

Interventioncells/microliters (Median)
Treatment253

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Time to Platelet Engraftment

(NCT00425802)
Timeframe: 1 year

Interventiondays (Median)
Treatment12

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Immune Reconstruction/CD4+ Count at 6 Months

(NCT00425802)
Timeframe: 6 months

Interventioncells/microliter (Median)
Treatment312

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Immune Reconstruction/CD4+ Count at 1 Year

(NCT00425802)
Timeframe: 1 year

Interventioncells/microliter (Median)
Treatment333

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Number of Patients With Engraftment Response

Engraftment defined as (1) the first of three consecutive days of an Absolute neutrophil count (ANC) >500/mL (b) the first of seven consecutive days of an unsupported platelet count 20,000. Patient needs to survive at least 28 days to be evaluable for engraftment. Chimerism studies need to demonstrate donor-derived hematopoiesis (>90%) (NCT00427336)
Timeframe: First 100 days post transplant.

InterventionParticipants (Number)
Fludarabine + Cyclophosphamide + ATG9

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Number of Participants With Engraftment

Engraftment defined as first of three (3) consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L; assessed from baseline to 100 days post-engraftment. (NCT00427557)
Timeframe: Baseline to 100 days post-engraftment

Interventionparticipants (Number)
Fludarabine + Melphalan + Umbilical Cord Blood Unit27

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Engraftment at 1 Year Post BMT.

Measurement of total PBMC chimerism (NCT00427661)
Timeframe: 1 year

Interventionparticipants (Number)
AHSC in Severe SCD6

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Development of GVHD Within 1 Year of BMT

GVHD is assessed by physical exam, bloodwork and biopsy. (NCT00427661)
Timeframe: 1 year

Interventionparticipants (Number)
AHSC in Severe SCD2

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Incidence of Grade 2-4 Acute GVHD.

(NCT00427661)
Timeframe: 100 days

Interventionparticipants with grade 2-4 AGVHD (Number)
Experimental1

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Number of Patients Who Achieve a CD4 Count > 200/Micro-liters

Determine the number of patients who achieve a CD4 count > 200/micro-liters by 60 days after transplant. (NCT00429416)
Timeframe: Through 60 Days Post Transplant

Interventionparticipants (Number)
LLME to Decrease GVHD Following HSC T13

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Incidence of Grade II-IV Acute Graft-Versus-Host-Disease (GVHD)

Determine the incidence of grade II-IV acute GVHD after administration of grafts when combined with Cyclosporine/Mycophenolate Mofetil for GVHD prophylaxis. GVHD assessments occur daily as an in patient and at each out patient visit. (NCT00429416)
Timeframe: Through 24 months post-treatment

Interventionparticipants (Number)
Developed grade II-IV GVHDDeveloped cGVHD (Chronic GVHD)
LLME to Decrease GVHD Following HSC T31

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Safety of CD34+ Stem Cell Infusions Followed by LLME as Measured by 100-Day Mortality

"Determine the safety of CD34+ stem cell infusions followed by the LLME treated CD34- fraction. This includes monitoring the patients for any side effects associated with the LLME treated cell infusion or any other unexpected adverse events.~This regimen will be gauged as to its safety using 100 day mortality as the measured endpoint. Deaths from all causes will be included." (NCT00429416)
Timeframe: Through 100 days post-transplant or death

Interventionparticipants (Number)
LLME to Decrease GVHD Following HSC T1

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Rate of Serious Infectious Complications

"Determine the rate of serious infectious complications. A serious infection will be defined as any requiring hospitalization or parenteral therapy.~CD4 counts will be measured monthly for the first 3 months after transplant." (NCT00429416)
Timeframe: Through 3 months post-transplant

Interventionparticipants (Number)
LLME to Decrease GVHD Following HSC T2

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Rate of Engraftment of Non-Myeloablative Transplants

Determine the engraftment rate of non-myeloablative transplants using CD34+ stem cells and LLME treated CD34- products. (NCT00429416)
Timeframe: Through 30 days post-transplant

Interventionparticipants (Number)
LLME to Decrease GVHD Following HSC T13

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Grade II-IV Toxicity

Non-hematopoietic toxicity within the first year of transplantation, acute Graft versus Host Disease (GVHD) above National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade I and chronic above Grade I are reported by participant incidence. Broad classification of adverse events (AE) categories based on anatomy and/or pathophysiology; within each category, AEs are listed accompanied by their descriptions of severity (Grade, Grade 1 least severe). (NCT00429572)
Timeframe: Up to one year.

InterventionParticipants (Number)
CardiacPulmonaryGastrointestinalRenalNeurologicalFever/Flu like symptomsInfectionGenitourinarySkin
Allogeneic Transplantation138022322

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Time to Progressive Disease

Progression-free was measured, by days, at time from transplantation to development to disease or death from any cause, which ever occurred first. (NCT00429572)
Timeframe: Transplant to Progression.

InterventionDays (Median)
Allogeneic Transplantation202

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Overall Survival

Survival duration was calculated from time of transplantation by number of days. (NCT00429572)
Timeframe: Transplant until death.

InterventionDays (Median)
Allogeneic Transplantation643

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Number of Participants With Tumor Response

Best response recorded from start of treatment until disease progression/recurrence using World Health Organization (WHO) criteria of Complete Response: disappearance of all disease/symptoms > 4 weeks; Partial response, > 50% reduction in sum of products of diameters of each measurable lesion for more than 4 weeks; Stable Disease, no change in tumor size; and Progressive Disease, appearance of new lesions or > 25% increase in sum of products of diameters of any measurable lesions. (NCT00429572)
Timeframe: Baseline to measured progressive disease (post study follow-up period 24 months starting from the date of the last drug administration). Data collected every 4 months.

Interventionparticipants (Number)
Complete ResponseStable DiseasePartial ResponseProgressive Disease
Allogeneic Transplantation5913

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Number of Participants With Acute or Chronic GVHD And Response to Therapy

"Participants diagnosed with Graft versus Host Disease (GVHD) post transplant were divided into either acute (aGVHD), normally observed within the first 100 days post-transplant; and chronic GVHD (cGVHD) cases, normally occur after 100 days, then evaluated and scored according to standard criteria from Consensus conference on acute GVHD grading, Bone Marrow Transplant 1995; 15: 825-828, noted is type of case and whether responds to therapy." (NCT00429572)
Timeframe: Transplant to 1 year post transplant

Interventionparticipants (Number)
aGVHDaGVHD responded to therapycGVHDcGVHD responded to therapy
Allogeneic Transplantation971414

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Overall Response Rate (ORR) to Fludarabine, Cyclophosphamide, Rituximab, and Bevacizumab Therapy in Previously Treated CLL

ORR defined as CR and PR response where response criteria for Complete response (CR) - Nodes: None; Liver/Spleen Size: Not palpable; Symptoms: None; polymorphonuclear leukocytes (PMN): >1,500/μl; Platelets >100,000/μl; Hemoglobin (untransfused): >11,0 g/dl; Lymphocytes: <4,000/μl; Bone Marrow aspirate: <30% lymphocytes; Biopsy: No lymphocyte infiltrate; and for Partial Response (PR), Nodes: >/= 50% decrease; Liver/Spleen Size: >/= 50% decrease; Symptoms: None; Platelets >100,000/μl or > 50% improvement from baseline; Hemoglobin (untransfused): >11.0 g/dl or >50% improvement from baseline; Lymphocytes: >50% decrease; Biopsy: <30% lymphocytes with residual disease on biopsy for nodular PR (NPR). (NCT00448019)
Timeframe: Response assessed after three 4-week courses and after six courses, up to 24 weeks

Interventionpercentage of participants (Number)
FCR + Bevacizumab79

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Progression Free Survival (PFS) Rate

Progression free survival (PFS) was defined as the time from the start of treatment to progression, which included treatment failure, relapse, or death. 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 (NCT00448019)
Timeframe: Baseline up to 5 years

InterventionMonths (Median)
FCR + Bevacizumab13.5

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Number of Participants With Complete or Partial Response to Fludarabine, Cyclophosphamide, Rituximab, and Bevacizumab Therapy in Previously Treated Chronic Lymphocytic Leukemia (CLL)

Response criteria for Complete response (CR) - Nodes: None; Liver/Spleen Size: Not palpable; Symptoms: None; polymorphonuclear leukocytes (PMN): >1,500/μl; Platelets >100,000/μl; Hemoglobin (untransfused): >11,0 g/dl; Lymphocytes: <4,000/μl; Bone Marrow aspirate: <30% lymphocytes; Biopsy: No lymphocyte infiltrate; and for Partial Response (PR), Nodes: >/= 50% decrease; Liver/Spleen Size: >/= 50% decrease; Symptoms: None; Platelets >100,000/μl or > 50% improvement from baseline; Hemoglobin (untransfused): >11.0 g/dl or >50% improvement from baseline; Lymphocytes: >50% decrease; Biopsy: <30% lymphocytes with residual disease on biopsy for nodular PR (NPR). (NCT00448019)
Timeframe: Response assessed after three 4-week courses and after six courses, up to 24 weeks

Interventionparticipants (Number)
Complete Response (CR)Partial Response (PR)Nodular Partial Response (NPR)
FCR + Bevacizumab13248

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Complete or Mixed Donor Chimerism at 30, 60, and 90 Days Post-transplant

"Complete chimerism is defined as 100% donor cells detected, suggesting complete hematopoietic replacement. Mixed donor chimerism means host cells are detected in particular cells like lymphocytes. Five to 90% donor cells set the criteria for mixed chimerism (MC).~Chimerism was not tabulated on day 30." (NCT00448201)
Timeframe: Days 30, 60, and 90

,
Interventionpercentage of patients (Number)
Complete DonorMixed Donor
Day 608218
Day 908713

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Graft-vs-host Disease at 6 Months Post-transplant

"Graft-vs-host disease (GVHD) can be mild, moderate or severe depending on the differences in tissue type between patient and donor. Its symptoms can include:~Rashes, which include burning and redness, that erupt on the palms or soles and may spread to the trunk and eventually to the entire body~Blistering, causing the exposed skin surface to flake off in severe cases~Nausea, vomiting, abdominal cramps, diarrhea and loss of appetite, which can indicate that the gastrointestinal (digestive) tract is affected~Jaundice, or a yellowing of the skin, which can indicate liver damage~Excessive dryness of the mouth and throat, leading to ulcers~Dryness of the lungs, vagina and other surfaces~Acute GVHD - Can occur soon after the transplanted cells begin to appear in the recipient. Acute GVHD ranges from mild, moderate or severe, and can be life-threatening if its effects are not controlled.~Extensive chronic GVHD - Usually occurs at about three months post-transplant." (NCT00448201)
Timeframe: 6 Months

Interventionpercentage of participants (Number)
Acute GVHDExtensive Chronic GVHD
All Trial Participants1330

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5-year Disease-free Survival

The length of time post-transplant that the patient survives without any signs or symptoms of that cancer. (NCT00448201)
Timeframe: Year 5

Interventionpercentage of participants (Number)
All Trial Participants31

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Incidence of DNA Chimerism in Patients Between One Month Post Transplant

Deoxyribonucleic acid (DNA) chimerism is a measure identifying the genetic profiles of the transplant recipient and of the donor and then evaluating the extent of mixture in the recipient's blood, bone marrow, or other tissue. (NCT00448357)
Timeframe: 30 days post transplant

InterventionParticipants (Count of Participants)
Whole blood chimerism-AnyWhole blood chimerism->=95% donorT Cell chimerism-AnyT Cell chimerism>=95% donor
Experimental: GVHD Prophylaxis49474630

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Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912)

Relapse is defined as new or increased sites of disease or positive one marrow after a complete response (CR). The RFS was calculated as the percentage of patients who were alive and without relapse at 3 years (NCT00448357)
Timeframe: Three years post-transplant

Interventionpercentage of participants (Number)
Low Busulfan AUC Tertile22
Intermediate Busulfan AUC Tertile39
High Busulfan AUC Tertile43

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Overall Survival

Percentage of participants alive at 3 years post transplant (NCT00448357)
Timeframe: Three years post-transplant

Interventionpercentage of participants (Number)
Low Busulfan AUC Tertile (5078)28
Intermediate Busulfan AUC Tertile (6372)39
High Busulfan AUC Tertile (7605)55

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Number of Participants With Dose Limiting Toxicities (DLTs)

Dose limiting toxicity will be defined as any irreversible grade 3 or any grade 4 non-hematologic toxicity that is related to busulfan infusion and not graft vs host disease or late infection after recovery from the initial period of myelosuppression. The maximum tolerated dose (MTD) is defined as the dose with probability of dose limiting toxicity (DLT) of 0.25. The dose of continuous infusion IV busulfan based on blood levels derived from a test dose in conjunction with fludarabine and alemtuzumab plus tacrolimus for GVHD prophylaxis. (NCT00448357)
Timeframe: first 6 weeks or 42 days following stem cell infusion

InterventionDLTs (Number)
Dose Level 11
Dose Level 21
Dose Level 31
Dose Level 42
Dose Level 52

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Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen

Test doses of busulfan were administered and plasma levels were measured to determine a targeted AUC dosing estimate. The capacity is reported as the precision with which these test dose goals predicted the actual 90-hour mean AUC levels.Dose targeting precision was estimated by root mean squared error. (NCT00448357)
Timeframe: Day -15 to Day -11

Interventionpercentage of error (Number)
Dose Level 111.7
Dose Level 24.9
Dose Level 310.2
Dose Level 411.1
Dose Level 515.9

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Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant

"GVHD can be mild, moderate or severe depending on the differences in tissue type between patient and donor. GVHD can be acute or chronic. Its symptoms can include:~Rashes, which include burning and redness, that erupt on the palms or soles and may spread to the trunk and eventually to the entire body~Blistering, causing the exposed skin surface to flake off in severe cases~Nausea, vomiting, abdominal cramps, diarrhea and loss of appetite, which can indicate that the gastrointestinal (digestive) tract is affected~Jaundice, or a yellowing of the skin, which can indicate liver damage~Excessive dryness of the mouth and throat, leading to ulcers~Dryness of the lungs, vagina and other surfaces" (NCT00448357)
Timeframe: 100 days post transplant

,,
InterventionParticipants (Count of Participants)
Acute GVHD grade >=IIAcute GVHD grades III and IVChronic GVHD; intermediate/severe
High Busulfan AUC Tertile1132
Intermediate Busulfan AUC Tertile1046
Low Busulfan AUC Tertile724

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Risk for Graft Failure

Count of participant that had graft failure. (NCT00450983)
Timeframe: Engraftment documented day +20

InterventionParticipants (Count of Participants)
Treatment0

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Risk for Mortality From Infection Before Day 180

Count of participant deaths from infection up to day 180. (NCT00450983)
Timeframe: Up to day 180

InterventionParticipants (Count of Participants)
Treatment0

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Risk of Developing Grades III-IV Acute Graft-vs-host Disease (GVHD)

Count of participants with acute GVHD grades III-IV. (NCT00450983)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
Treatment0

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Risk for Life-threatening Infections

Count of participants with life-threatening infections (NCT00450983)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
Treatment1

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Number of Participants With a Complete Response or Partial Response

According to International Workshop Response Criteria for Non-Hodgkin's Lymphomas: Complete remission (CR) defined as > 30% lymphocytes in the bone marrow, recovery of blood counts and no clinical symptoms; and Partial remission (PR) defined as > 50% decrease of clinical symptoms from baseline and recovery from blood counts. (NCT00452374)
Timeframe: Evaluation every 3 cycles of treatment (28 days per cycle), approximately 90 days

InterventionParticipants (Number)
Complete response (CR)Partial response (PR)
Oxaliplatin, Fludarabine, Cytarabine + Rituximab1228

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Maximum Tolerated Dose (MTD) Oxaliplatin

MTD defined as dose level at which 2/3 or 2/6 participants experience Dose Limiting Toxicity (DLT), where DLTs are any oxaliplatin-related ≥Grade 3 non-hematological toxicity involving a major organ system (brain, heart, kidney, liver, lung) in the National Cancer Institute (NCI) Version 3.0 toxicity scale. (NCT00452374)
Timeframe: From treatment onset to end of each cycle of treatment (every 21 days)

Interventionmg/m^2 (Number)
Oxaliplatin, Fludarabine, Cytarabine + Rituximab25

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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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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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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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Overall Survival

Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive. (NCT00455312)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Patients With DC12
Patients With SAA19

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Overall Survival

Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive. (NCT00455312)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Patients With DC14
Patients With SAA19

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Incidence of Grade 3-4 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. (NCT00455312)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Patients With DC0
Patients With SAA0

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Incidence of Chronic GVHD

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. (NCT00455312)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Patients With DC1
Patients With SAA2

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Incidence of Chronic GVHD

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. (NCT00455312)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Patients With DC0
Patients With SAA0

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Incidence of Grade 2-4 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. (NCT00455312)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Patients With DC1
Patients With SAA0

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Incidence of Late Secondary Malignancies

Defined as patients who have a secondary malignancy (cancer) occurring. (NCT00455312)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Patients With DC0
Patients With SAA4

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Neutrophil Engraftment

Defined as an absolute neutrophil count (ANC) >5 x 10^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation. (NCT00455312)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Patients With DC15
Patients With SAA20

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Incidence of Pulmonary Complications

Defined as patients who exhibit a pulmonary (lung) adverse event. (NCT00455312)
Timeframe: 6 Months

InterventionParticipants (Count of Participants)
Patients With DC3
Patients With SAA3

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Length of Survival

(NCT00462332)
Timeframe: At 2 years and a half from study entry

Interventionyears (Mean)
High Risk Patientes1.57
Low Risk Patients1.1

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Number of Patients With Complete Response

"Normal clinical or X-ray examination (lymph nodes, liver, spleen)~No symptoms~Lymphocytes higher or equal to 4.0 per 10^9/L~Neutrophils lower or equal to 1.5 per 10^9/L~Platelets >100 per 10^9/L~Hb >11.0 g/dL~Bone marrow lymphs according to age, lymphocytes <30%, no nodules." (NCT00462332)
Timeframe: At 2 years from study entry

Interventionparticipants (Number)
Low Risk Patients14
High Risk Patients3

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3 Year Progression Free Survival

"PFS defined as length of time either due to disease recurrence or death, from the time of stem cell infusion (Bone marrow or PBPC) to 3 years.~Response Criteria is measured by the bone marrow aspirate to determine it has leukemic blast. Bone marrow blast less than 5% is considered to be a complete response." (NCT00469144)
Timeframe: 3 years

InterventionDays (Median)
Fixed-Dose Busulfan + Fludarabine42
Adjusted Dose Busulfan + Fludarabine56

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Maximum Total Tolerated Dose (MTD) of Daily Combination Fludarabine 30 mg/m^2 and Cytarabine 500 mg/m^2 Among 3 Dose Levels (Dose Level 1: 2 Days, Dose Level 2: 3 Days or Dose Level 3: 4 Days)

Maximum dose levels for Phase I determined among three possible dose levels of Fludarabine and Cytarabine in combination with fixed doses of Oxaliplatin and Rituximab. Fludarabine and Cytarabine Dose Level 1: Days 2-3 (2 Days); Dose Level 2: Days 2-4 (3 Days); and Dose 3: Days 2-5 (4 Days). MTD is dose level at which less than 2/3 or 2/6 participants experience dose limiting toxicities (DLTs). The number of days of fludarabine and cytarabine administration increased simultaneously. Participants received a subsequent cycle of treatment with 1 additional day of fludarabine and cytarabine treatment no less than 4 weeks from the initiation of the previous cycle if no drug-related grade 3 or 4 non-hematologic life-threatening adverse events, and drug-related non-hematologic toxicity resolved to baseline or < grade 2. A maximum of 6 cycles were administered. (NCT00472849)
Timeframe: Up to 36 weeks (6 cycles each 4-6 weeks)

Interventionmg/m^2 (Number)
Fludarabine MTD (Total 3 Day Dose)Cytarabine MTD (Total 3 Day Dose)
OFAR (Phase I)901500

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Overall Response: Number of Participants With Complete Remission, Nodular Partial Remission, and Partial Remission

Overall Response includes Complete remission (CR), nodular partial remission (nPR), and partial remission (PR) in high-risk, previously untreated participants with Chronic Lymphocytic Leukemia treated with CFAR using National Cancer Institute - Working Group response criteria. CR defined as zero nodes, Liver/spleen not palpable, zero symptoms, polymorphonuclear leukocyte (PMN)>1,500/uL, Platelets >100,000uL, Hemoglobin (untransfused) >11.0g/dL, Lymphocytes <4,000/uL and Bone Marrow Aspirate biopsy <30% lymphocytes with no lymphocyte infiltrate; PR defined as nodes >/= 50% decrease,Liver/spleen >/= 50% decrease, symptoms not applicable, PMN >1,500/uL or >50% improvement from baseline, Platelets 100,000uL or >/=50% decrease improvement from baseline, Hemoglobin (untransfused) >11.0g/dL or >50% improvement from baseline, Lymphocytes >50% decrease and Bone Marrow Aspirate biopsy Not Applicable for PR; with nPR defined same as PR but with <30% lymphocytes with residual disease on biopsy. (NCT00472849)
Timeframe: Up to 36 weeks (6 cycles each 4-6 weeks)

Interventionparticipants (Number)
Complete RemissionPartial RemissionNodular Partial Remission
OFAR MTD (Phase II)3279

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Rate of Non-relapse Mortality at 100 Days Post-transplant

To evaluate the safety of Fludarabine/Busulfan as conditioned regimen for allogeneic stem cell transplantation in patients with myelofibrosis/myelodysplastic syndrome at 100 days post-transplant (NCT00475020)
Timeframe: Non-relapse mortality at 100 days post-transplant

InterventionParticipants (Count of Participants)
InfectionsOrgan FailuresSudden death: likely due to ischemic cardiac event
Participants With Myelofibrosis and Myelodysplastic Syndrome121

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Efficacy of This Therapy 3 Years Post-transplant

Efficacy Assessed as Number of Participants with Overall Survival, Leukemia Progression, Primary Graft Failure and Complete Hematological Response. Primary graft failure is defined as failure to achieve an ANC >/= 0.5 x 10 (9)/L for 3 consecutive days and evidence of donor chimerism by Day +28. Complete hematological response is defined by hemoglobin >/= 120 g/L; or achievement of transfusion independence, with stable Hb > 110 g/L, for RBC transfusion-dependent participants; Spleen not palpable; platelet count 150 x 10 (9)/L; White blood cell 4 x 10 (9)/L to 10 x 10(9)/L. (NCT00475020)
Timeframe: Up to 3 years post-transplant

InterventionParticipants (Count of Participants)
Overall survival at 3 yearsLeukemia progressionPrimary graft failuresComplete hematological remission
Participants With Myelofibrosis and Myelodysplastic Syndrome2527044

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Number of Patients With Acute Graft-Versus-Host Disease (GVHD)

Number of patients with GVHD. Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00478244)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Evaluable Patients1

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Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)

Number of patients with cGVHD; a severe long-term complication created by infusion of donor cells into a foreign host. (NCT00478244)
Timeframe: Day 365 Post Transplant

Interventionparticipants (Number)
Evaluable Patients0

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Number of Patients With Detectable Collagen Type VII

Number of patients with epidermolysis bullosa who had collagen type VII. Type VII collagen defects cause recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disorder often accompanied by epidermal cancers. (NCT00478244)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Evaluable Patients5

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Number of Patients With Donor Derived Cells in Skin

Number of patients who had donor skin chimerism - donor cells in the patient's epidermis (a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease). (NCT00478244)
Timeframe: Day 90 Post Transplant

Interventionparticipants (Number)
Evaluable Patients6

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Number of Patients With Neutrophil Engraftment

Number of patients with an absolute neutrophil count >5 x 10^8 cells/liter for 3 consecutive days. (NCT00478244)
Timeframe: Day 42 Post Transplant

Interventionparticipants (Number)
Evaluable Patients6

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Number of Patients With Platelet Engraftment

Number of patients with a platelet count >5 x 10^10 cells/liter for 3 consecutive measurements. (NCT00478244)
Timeframe: Day 180 Post Transplant

Interventionparticipants (Number)
Evaluable Patients5

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Number of Patients With Resistance to Blister Formation

Resistance to Blister Formation demonstrated by response to negative pressure. (NCT00478244)
Timeframe: Month 1 through Month 24 Inclusive

Interventionparticipants (Number)
Evaluable Patients2

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Number of Patients With >70% Donor Chimerism

Number of patients with donor chimerism - percentage of donor cells in the patient via the peripheral blood or bone marrow. (NCT00478244)
Timeframe: Days 21, 100, 180, 365 and 730 Post Transplant

Interventionparticipants (Number)
Day 21Day 100Day 180Day 365Day 730
Evaluable Patients65555

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Overall Survival

Survival is defined as the number of patients that were alive post transplant. (NCT00478244)
Timeframe: 1 year and 2 years Post Transplant

Interventionparticipants (Number)
1 Year Post Transplant2 Years Post Transplant
Evaluable Patients55

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Number of Participants With Objective Response

Objective response: Complete Response/Remission (CR) defined as a bone marrow with 5% or fewer blasts and peripheral blood count with an absolute neutrophil count of 10^9/Liters or more and platelet count of 100*10^9/Liters or more; Complete Response with Platelets/remission without platelet recovery (CRp) defined as a complete response except for a platelet less than 100*10^9/Liters and transfusion independent; and Partial Response/Remission defined as peripheral blood count recovery as for CR with decrease in marrow blasts >/= 50% and not more than 6-25% abnormal cells in the marrow. (NCT00480987)
Timeframe: After 2 months

InterventionParticipants (Number)
Complete responseComplete response with plateletsPartial response
Oxaliplatin + Cytarabine + Fludarabine320

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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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Number of Participants in Complete Molecular Remission at 1 Year

Participants at 1 year in molecular remission, post transplant, post imatinib mesylate and donor lymphocyte infusion (DLI). Molecular remission is a complete remission with no evidence of disease in the blood cells and/or bone marrow using sensitive polymerase chain reaction (PCR) tests (this test is most commonly used in clinical trials). (NCT00499889)
Timeframe: Baseline to 1 year

Interventionparticipants (Number)
Mesylate, Busulfan, Fludarabine + Antithymocyte Globulin21

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Participants' With mCR Response to Post Transplant DLI

Number of participants with response of molecular complete remission (mCR) to DLI as treatment for residual disease after transplant. Molecular remission is a complete remission with no evidence of disease in the blood cells and/or bone marrow using sensitive polymerase chain reaction (PCR) tests. (NCT00499889)
Timeframe: 1 year

InterventionParticipants (Number)
Mesylate, Busulfan, Fludarabine + Antithymocyte Globulin4

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Participants' With mCR Response to Post Transplant Imatinib Mesylate Therapy

Number of participants with response of molecular complete remission (mCR) to Imatinib Mesylate therapy as treatment for residual disease after transplant. Molecular remission is a complete remission with no evidence of disease in the blood cells and/or bone marrow using sensitive polymerase chain reaction (PCR) tests. (NCT00499889)
Timeframe: 1 Year

InterventionParticipants (Number)
Mesylate, Busulfan, Fludarabine + Antithymocyte Globulin10

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Number of Participants With Successful Engraftment

Successful Engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L. Failure to engraft by day +30 considered primary engraftment failure. Study period one week prior to transplant through post Day 28. (NCT00502905)
Timeframe: Study period one week prior to transplant through post Day 28

Interventionparticipants (Number)
Busulfan + Fludarabine192

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Number of Participants With Successful Engraftment at Day 100

(NCT00505895)
Timeframe: Day 100

Interventionparticipants (Number)
Fludarabine + Melphalan + Stem Cell Infusion27
Fludarabine + Lower-Dose Melphalan + Stem Cell Infusion23

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Acute Grade II-IV Graft Versus Host Disease (GVHD)

Effects of Rituximab as measured by percentage of participants with Acute and Chronic Graft Versus Host Disease (GVHD) incidences after allogeneic transplantation. GVHD occurring anytime after day 90 post transplant was considered chronic GVHD; otherwise it was considered acute GVHD. Acute GVHD status defined as GVHD with maximum grade ≥2. Clinical grading of Acute GVHD (Thomas et al., New England Journal of Medicine (NEJM), 229:895, 1975): Grade 1 to 4. (NCT00505895)
Timeframe: GVHD grading weekly during first 100 days; Annual examinations for nine year study period

,
Interventionpercentage of participants (Number)
Acute GVHDChronic GVHD
Fludarabine + Lower-Dose Melphalan + Stem Cell Infusion2148
Fludarabine + Melphalan + Stem Cell Infusion2229

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Participant Progression Free Survival at 2 Years

Progression-free survival defined as the number of participants without evidence of progression or death after 2 years from stem cell transplant. (NCT00505921)
Timeframe: 2 years

Interventionparticipants (Number)
Campath-1H15

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Participant's Response According to Physician's Global Assessment of Clinical Condition (PGA)

Response defined by Physician's Global Assessment of Clinical Condition (PGA) where Complete Response (CR) is No evidence of disease; 100% improvement; Partial Response (PR), one of following: 1) Very significant clearance ( > 90% to < 100%); only traces of disease remains; 2) Significant improvement ( > 75% to < 90%); some evidence of disease remain; 3) Intermediate between slight and marked improvement; ( > 50% to < 75%); 4) Some improvement ( > 25% to < 50%); however, significant evidence of disease remains; Stable Disease (SD): Disease has not changed from baseline condition (+<25%); Progressive Disease (PD): Disease is worse than at baseline evaluation by > 25% or more. Response confirmed by a second assessment at least 4 weeks following it. Assessments at baseline, pre and post transplant (100 days) then till disease progression or year one. (NCT00506129)
Timeframe: Response assessed pre-transplant and 100 days post transplant with follow up at 1 year.

Interventionparticipants (Number)
Complete Response (CR) Converted Post TransplantCR Prior to TransplantPartial Response (PR)Stable Disease (SD)Progressive Disease (PD)
Fludarabine + Melphalan With PBPC1960016

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Average Overall Survival (OS) Length

OS was defined as the time from transplantation to the date of death from any cause or last follow up, measured in days. (NCT00506129)
Timeframe: Baseline to disease progression, followed up to 5 years post transplant

InterventionDays (Mean)
Fludarabine + Melphalan With PBPC1207

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Maximum Tolerated Dose (MTD)

"Continual reassessment method (four times a day) used to determine an MTD, with a target toxicity probability of 20%, where toxicity is defined as grade 3 or 4 conventional toxicity [National Cancer Institute Common Toxicity Criteria (NCI-CTC)]. Participant evaluation in a cohort with each modality is 30 days." (NCT00506857)
Timeframe: 1 month

Interventionmg/kg (Number)
Busulfan + Fludarabine11.2

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Number of Participants With Graft Versus Host Disease (GVHD)

Tacrolimus and Methotrexate used for acute graft versus host disease (aGVHD) prophylaxis, clinical grading AGVHD criteria (Days 1-100): Grade 1: + to ++ skin rash; no gut involvement; no decrease in clinical performance status; Grade 2: + to +++ skin rash; + gut involvement and/or + liver involvement; mild decrease in performance status; Grade 3: ++ to +++ skin rash; ++ to +++ gut involvement and/or ++ to ++++ liver involvement; marked decrease in performance status; Grade 4: Similar to Grade 3 with ++ to ++++ organ involvement and extreme decrease in performance status. (NCT00506857)
Timeframe: 5 years

InterventionParticipants (Number)
Grade 2Grade 3-4
Tacrolimus + Methotrexate188

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Toxicity

Here is the number of participants with adverse events. For a detailed listing of adverse events, see the adverse event module. (NCT00509288)
Timeframe: 57 months

InterventionParticipants (Number)
Anti-MART-1 F5 TCR PBL + HD IL-221
Anti-MART-1 F5 TCR TIL + HD IL-23

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Clinical Tumor Regression.

Tumor regression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT00509288)
Timeframe: 7/5/07-4/23/09

,
InterventionParticipants (Number)
Complete ResponsePartial ResponseProgressive DiseaseStable Disease
Anti-MART-1 F5 TCR PBL + HD IL-206150
Anti-MART-1 F5 TCR TIL + HD IL-20020

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Clinical Tumor Regression.

Clinical tumor regression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is a disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease (SD)is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT00509496)
Timeframe: 20 months

,
InterventionParticipants (Number)
Complete ResponsePartial ResponseProgressive DiseaseStable Disease
Anti-gp100:154-162 TCR PBL + HD IL-212160
Anti-gp100:154-162 TCR TIL + HD IL-20110

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Toxicity

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT00509496)
Timeframe: 6 years

InterventionParticipants (Number)
Anti-gp100:154-162 TCR PBL + HD IL-219
Anti-gp100:154-162 TCR TIL + HD IL-22

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Number of Participants With Neuropathy, Any Grade

Before each drug dose, the patient will be evaluated for possible toxicities that may have occurred after the previous dose(s). Toxicities are to be assessed according to the NCI Common Toxicity Criteria (CTC). (NCT00510887)
Timeframe: up to 1 year

Interventionparticipants (Number)
VR-FND6

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Percentage of Subjects Experiencing Progression Free Survival

Progression free survival is measured from treatment to progression or death, whichever comes first. Progressive disease is measured as: 50% or greater increase from nadir in the sum of the products (SPD) of any previously identified abnormal node and the appearance of any new lesions during or at the end of treatment. (NCT00510887)
Timeframe: up to 2 years

Interventionpercentage of participants (Number)
VR-FND17

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Percentage of Subjects Experiencing Overall Survival

Overall survival is from the day of enrollment to date of death from any cause. (NCT00510887)
Timeframe: up to 2 years

Interventionpercentage of participants (Number)
VR-FND27

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Number of Participants With a Grade 3-4 Hematologic Toxicity.

Before each drug dose, the patient will be evaluated for possible toxicities that may have occurred after the previous dose(s). Toxicities are to be assessed according to the NCI Common Toxicity Criteria (CTC). (NCT00510887)
Timeframe: up to 1 year

Interventionparticipants (Number)
VR-FND7

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Duration of Response

Duration of response is measured from time of treatment to time of disease progression (NCT00510887)
Timeframe: up to 4 years

Interventionmonths (Mean)
VR-FND16.47143

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Complete and Partial Response

"Complete Response: Complete disappearance of all detectable clinical and radiographic evidence of disease, disappearance of all disease-related symptoms and normalization of biochemical abnormalities (eg. LDH) definitely assignable to follicular lymphoma.~Partial Response requires the following:~greater than or equal to 50% decrease in the SPD of the 6 largest dominant nodes of nodal masses.~No increase in size of other nodes, liver, or spleen.~Splenic and hepatic nodes must regress by at least 50% in sum of the products (SPD).~Bone marrow assessment in irrelevant for determination of Partial Response since it is not measurable disease; however, if positive the type of cell should be reported.~No new lesions." (NCT00510887)
Timeframe: 1 year

Interventionpercentage of participants (Number)
VR-FND64

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Number of Participant With Adverse Events (AE)

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. (NCT00513474)
Timeframe: Up to 71 months

InterventionParticipants (Count of Participants)
Rasburicase Group21
Control Group21

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Percentage of Participants With Grades II to IV Acute Graft-Versus-Host Disease (aGVHD)

"aGVHD severity was determined using International Bone Marrow Transplant Registry (IBMTR) scale stage and grade of the skin, liver and gut. Stage 1: Skin=maculopapular rash <25% of body surface; Liver=Bilirubin 2-3 mg/dL and Gut=500-999 mL diarrhea/day or peristent nausea with histologic evidence of GvHD. Stage 2: Skin=maculopapular rash 25-50% of body surface; Liver=Bilirubin 3.1-6 mg/dL and Gut=1000-1499 mL diarrhea/day. Stage 3: Skin=maculopapular rash >50% of body surface; Liver=Bilirubin 6.1-15 mg/dL and Gut=≥1500 mL diarrhea/day. Stage 4: Skin=generalized erythroderma with bulla formation; Liver=Bilirubin >15 mg/dL and Gut=severe abdominal pain.~Grade 1: Stage 1-2 rash; no liver or gut involvement. Grade II: Stage 3 rash, or stage 1 liver involvement, or stage 1 gut involvement. Grade III: None to stage 3 skin rash with stage 2-3 liver, or stage 2-4 gut involvement. Grade IV: Stage 4 skin rash, or stage 4 liver involvement." (NCT00513474)
Timeframe: Up to 71 months

Interventionpercentage of participants (Number)
Rasburicase Group24
Control Group57

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Uric Acid Levels

Blood was collected and analyzed at a laboratory for serum uric acid levels reported in milligrams(mg)/deciliter(dL). Data is presented for those participants who experienced Grade II to IV aGVHD and those participants who did not experience Grade II to IV aGVHD at pre-transplant and post-transplant. (NCT00513474)
Timeframe: Pre-transplant Day -7 to Day -1 and Post-transplant Day 0 to Day 6

,
Interventionmg/dL (Mean)
Day -7Day -6Day -5Day -4Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6
Control Group4.1573.4192.9672.5792.3581.8671.712.1632.6712.7782.8052.7582.5792.653
Rasburicase Group0.10.0750.0860.10.0670.0810.4380.9381.6242.0762.2712.5482.5952.705

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Clinical Response

Clinical response is defined as complete response (CR)- a disappearance of all target lesions, partial response (PR) - at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progression (PD)- at least a 20% increase in the sum of the LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease (SD) - neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT00513604)
Timeframe: every 1-3 months until disease progression. Total length of time -8/7/2007 to 9/27/2012

,,,,
InterventionParticipants (Number)
Complete ResponsePartial ResponseProgressionStable DiseaseNot evaluable - cell product did not growNot evaluable-toxicities re:disease/deathNot evaluable - Patient died of sepsis
Cohort 1 - NMA, TIL, Aldesleukin13200200
Cohort 2 - NMA, CD4+ TIL, Aldesleukin318160020
Cohort 3 - NMA, Total Body Irradiation37120101
Cohort 4 - NMA, Young TIL, Aldesleukin210211000
Cohort 5 - NMA, CD4+TIL, HD Aldesleukin34244000

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Toxicity

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT00513604)
Timeframe: 5 years

InterventionParticipants (Number)
Cohort 1 - NMA, TIL, Aldesleukin24
Cohort 2 - NMA, CD4+ TIL, Aldesleukin39
Cohort 3 - NMA, Total Body Irradiation23
Cohort 4 - NMA, Young TIL, Aldesleukin34
Cohort 5 - NMA, CD4+TIL, HD Aldesleukin35

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Overall Survival (OS) for High Risk Patients

Kaplan-Meier analysis was conducted to estimate the distribution of time from randomization to death from any cause. Estimates were not stratified. Patients who did not experience this primary outcome had their survival times censored at their last follow-up. (NCT00513747)
Timeframe: Up to 72 months

Interventionmonths (Median)
Arm A: High Risk Early InterventionNA
Arm B: High Risk Observation + Later TreatmentNA

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Disease-Free Survival in High Risk Patients

"Kaplan-Meier analysis was conducted to estimate disease free survival defined as:>~Arm A: Time from randomization until Second Treatment (first relapse) or death whichever comes first. Events were defined as the start of second treatment or death. Patients who had not experienced one of these defined events of interest were censored at their last known follow-up.>~Arm B: Time from randomization until First Treatment (first relapse) or death whichever comes first. Events were defined as the start of first treatment or death. Patients who had not experienced one of these defined events of interest were censored at their last known follow-up." (NCT00513747)
Timeframe: Up to 72 months

Interventionmonths (Median)
Arm A: High Risk Early Intervention62.7
Arm B: High Risk Observation + Later Treatment39.2

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Time to Second Treatment in High Risk Patients

Kaplan-Meier analysis was conducted to estimate the distribution of time from randomization to second treatment or death whichever comes first. Events were defined as the start of second treatment or death. Patients who had not experienced one of these defined events of interest were censored at their last known follow-up. (NCT00513747)
Timeframe: Up to 72 months

Interventionmonths (Median)
Arm A: High Risk Early Intervention62.7
Arm B: High Risk Observation + Later Treatment56.3

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Time to First Treatment Survival in Low Risk Patients

"Time to First Treatment Survival in low risk patients (registration to first treatment or death)>~• Events were defined as the start of first treatment or death from any cause. Patients who didn't receive their first treatment were censored at their last known follow-up." (NCT00513747)
Timeframe: Up to 72 months

Interventionmonths (Median)
Arm C: Low Risk Observation + Later Treatment58.1

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Overall Survival in Low Risk Patients

"Overall survival in low risk patients (registration to first treatment or death)>~• Events were defined as death from any cause. Low risk Patients who were alive were censored at their last known follow-up." (NCT00513747)
Timeframe: Up to 72 months

Interventionmonths (Median)
Arm C: Low Risk Observation + Later Treatment58.1

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Percentage of Participants With Grade II-IV Acute Graft Versus Host Disease (GVHD)

Acute GVHD is assessed by the 1994 Consensus Conference on Acute GVHD Grading criteria. See Przepiorka D, Weisdorf D, Martin P, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995; 15:825-8., for grading criteria. (NCT00520130)
Timeframe: 6 months

Interventionpercentage of participants (Number)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm42
B - Cyclosporine (AC) Arm38

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Days to Engraftment of Platelets

Platelet recovery: designated by the first of 7 days where the platelet count remains above 20,000/mm(3) without transfusion support (NCT00520130)
Timeframe: 2 years

InterventionDays (Median)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm19
B - Cyclosporine (AC Arm)14

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Overall Survival

Time between the first day of transplant to the day of death. (NCT00520130)
Timeframe: Patients were followed for an average of up to 5 years.

InterventionMonths (Median)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm41.7
B - Cyclosporine (AC) Arm18.8

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Percentage of Participants With Grade III-IV Acute Graft Versus Host Disease (GVHD)

Acute GVHD is assessed by the 1994 Consensus Conference on acute GVHD Grading criteria. See Przepiorka D, Weisdorf D, Martin P, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995; 15:825-8., for grading criteria. (NCT00520130)
Timeframe: 6 months

Interventionpercentage of participants (Number)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm13
B - Cyclosporine (AC) Arm21

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Days to Engraftment of Neutrophils

Days to engraftment is defined as neutrophil recovery: designated by the first of 3 consecutive days with an absolute neutrophil count (ANC) above 500/mm(3). (NCT00520130)
Timeframe: 2 years

InterventionDays to neutrophil engraftment (Median)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm11
B - Cyclosporine (AC Arm)9

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Toxicities

Here are the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT00520130)
Timeframe: 103 months and 22 days

Interventionparticipants (Number)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm43
B - Cyclosporine (AC) Arm42

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Changes in CD8 T Cell Receptor Vbeta Repertoire

Ribonucleic acid (RNA) was extracted from sorted CD4 and cluster of differentiation 8 (CD8) T cells and analyzed for Vbeta repertoire by nested polymerase chain reaction (PCR) analysis using Vbeta family specific primers and a labeled constant region primer (spectratyping). The receptor repertoire diversity was calculated from spectratyping data by creating a normal standard for repertoire diversity from healthy normal controls and assessing the divergence of individual patient's T cell receptor repertoire from these standard normal donor values. In this Vbeta repertoire divergence index, lower numbers are consistent with a more normal highly diverse repertoire, and high numbers represent a highly skewed, oligoclonal repertoire. The assay is described in Memon SA et al, J Immunol Methods, 2012, 375: 84-92. The repertoire diversity of the CD4 and CD8 T cells of the donor infusion is shown for comparison. (NCT00520130)
Timeframe: Donor at time of collection and recipient at 1, 3, 6 and 12 months post transplant

,
InterventionDivergence index (Median)
1 month3 months6 mo (TMS=7; AC= 9)12 mo (TMS=7; AC= 9)Donor CD8 cells (TMS=8; AC= 8)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm6254625547
B - Cyclosporine (AC) Arm7881848943

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Immune Reconstitution of Cluster of Differentiation 4 (CD4) T Cell Populations

Cluster of Differentiation 3 (CD3)+CD4+ and CD3+Cluster of Differentiation 8 (CD8)+ T cells within the lymphocyte population were determined by flow cytometry. The absolute numbers of cells/µl were calculated from the absolute lymphocyte count. (NCT00520130)
Timeframe: 2 weeks, and 1, 3, 6, 12 and 24 months post transplant

,
InterventionCells/µl (Median)
2 weeks1 month3 months6 months12 months24 months
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm171285297387447451
B - Cyclosporine (AC) Arm02161121132373

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Immune Reconstitution of Cluster of Differentiation 8 (CD8) T Cell Populations

Cluster of differentiation 3 (CD3)+cluster of differentiation 4 (CD4)+ and CD3+CD8+ T cells within the lymphocyte population were determined by flow cytometry. The absolute numbers of cells/µl were calculated from the absolute lymphocyte count. (NCT00520130)
Timeframe: 2 weeks, 1, 3, 6, 12 and 24 months post transplant

,
InterventionCells/µl (Median)
2 weeks1 month3 months6 months12 months24 months
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm72204334429485434
B - Cyclosporine (AC) Arm1654158243502

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Immune Reconstitution of Normal Killer (NK) Cells

Cluster of differentiation 3 (CD3) - cluster of differentiation 56 (CD56) + Natural Killer (NK) cells within the lymphocyte population were determined by flow cytometry. The absolute numbers of cells/µl were calculated from the absolute lymphocyte count. (NCT00520130)
Timeframe: 2 weeks, and 1, 3, 6, 12, and 24 months post transplant

,
InterventionCells/µl (Median)
2 weeks1 month3 months6 months12 months24 months
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm123270134136134133
B - Cyclosporine (AC) Arm1531124202150307

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Percentage of Participants With Chronic Graft Versus Host Disease (cGVHD)

Chronic GVHD is assessed by the 2005 Chronic GVHD Consensus Project. First the individual organ scoring is done, and then based on that the Global score is determined (mild-moderate-severe). See Citation: Filipovich AH, Weisdorf D, Pavletic S, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (NCT00520130)
Timeframe: 2 years post transplant

,
Interventionpercentage of participants (Number)
Moderate or Severe cGVHDSevere cGVHD
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm5028
B - Cyclosporine (AC) Arm125

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Recovery of Naïve Cluster of Differentiation 4 (CD4) T Cells

The percentage of C-C motif chemokine receptor 7 (CCR7)+CD45RA+ naïve T cells within the CD4 T cell populations was determined by flow cytometry. (NCT00520130)
Timeframe: Recipient recovery at 6, 12 and 24 months post transplant

,
Intervention% of naive (CCR7+CD45RA+) CD4 Cells (Median)
6 mo (TMS=28; AC= 28)12 mo (TMS=25; AC= 21)24 mo (TMS=18; AC= 13)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm242220
B - Cyclosporine (AC) Arm1725

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Recovery of Naïve Cluster of Differentiation 8 (CD8) T Cells

The percentage of CCR7+CD45RA+ naïve T cells within the CD4 and CD8 T cell populations was determined by flow cytometry. (NCT00520130)
Timeframe: Recipient recovery at 6, 12 and 24 months post transplant

,
Intervention% of naive (CCR7+CD45RA+) CD8 Cells (Median)
6 mo (TMS=28; AC= 28)12 mo (TMS=25; AC= 21)24 mo (TMS=18; AC= 13)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm201716
B - Cyclosporine (AC) Arm366

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Days to Engraftment of Lymphocytes

Lymphocyte recovery: designated by the first of 3 consecutive days with absolute lymphocyte count (ALC) above 500/mm(3). (NCT00520130)
Timeframe: 2 years

InterventionDays (Median)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm16
B - Cyclosporine (AC Arm)76

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Changes in Cluster of Differentiation 4 (CD4) T Cell Receptor Vbeta Repertoire

Ribonucleic acid (RNA) was extracted from sorted CD4 and cluster of differentiation 8 (CD8) T cells and analyzed for Vbeta repertoire by nested polymerase chain reaction (PCR) analysis using Vbeta family specific primers and a labeled constant region primer (spectratyping). The receptor repertoire diversity was calculated from spectratyping data by creating a normal standard for repertoire diversity from healthy normal controls and assessing the divergence of individual patient's T cell receptor repertoire from these standard normal donor values. In this Vbeta repertoire divergence index, lower numbers are consistent with a more normal highly diverse repertoire, and high numbers represent a highly skewed, oligoclonal repertoire. The assay is described in Memon SA et al, J Immunol Methods, 2012, 375: 84-92. The repertoire diversity of the CD4 and CD8 T cells of the donor infusion is shown for comparison. (NCT00520130)
Timeframe: Donor at time of collection and recipient at 1, 3, 6 and 12 months post transplant

,
InterventionDivergence index (Median)
1 month3 months6 mo (TMS=7; AC= 9)12 mo (TMS=7; AC=9)Donor CD4 cells (TMS=8; AC=8)
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm4831353023
B - Cyclosporine (AC) Arm9066756722

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Overall Participant Response

Overall Response: Complete remission (CR), nodular partial remission (nPR), and partial remission (PR) rates (overall response) in high-risk, previously untreated patients with CLL treated with CFAR. National Cancer Institute - Working Group (NCI-WG) response criteria. CR defined as zero nodes, Liver/spleen not palpable, zero symptoms, polymorphonuclear leukocyte (PMN)>1,500/uL, Platelets >100,000uL, Hemoglobin (untransfused) >11.0g/dL, Lymphocytes <4,000/uL and Bone Marrow Aspirate biopsy <30% lymphocytes with no lymphocyte infiltrate; PR defined as nodes >/= 50% decrease,Liver/spleen >/= 50% decrease, symptoms not applicable, PMN >1,500/uL or >50% improvement from baseline, Platelets 100,000uL or >/=50% decrease improvement from baseline, Hemoglobin (untransfused) >11.0g/dL or >50% improvement from baseline, Lymphocytes >50% decrease and Bone Marrow Aspirate biopsy Not Applicable for PR; with nPR defined same as PR but with <30% lymphocytes with residual disease on biopsy. (NCT00525603)
Timeframe: Evaluated after 3 courses of 4 week therapy (12 weeks)

InterventionParticipants (Number)
Complete remission (CR)Nodular partial remission (nPR)Partial remission (PR)
CFAR44110

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Overall Survival at 100 Days Post Transplant (Number of Surviving Participants)

Overall Survival defined as the number of participants living at day 100 following non-myeloablative allogeneic stem cell transplantation using rituximab, cyclophosphamide, fludarabine as a preparative regimen for participants with advanced or recurrent mantle cell lymphoma. (NCT00525876)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
Matched Sibling Transplant16
Allo MUD & MM19

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Treatment Efficacy as Defined by Complete or Partial Remission

(NCT00526292)
Timeframe: 3 Months following treatment

Interventionparticipants (Number)
Complete RemissionProgression of Disease (POD)
HLA Haploidentical Natural Killer Cell Infusion15

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Complete Response Rate

An improvement in complete response to at least 60% following treatment, assessed using CT scans, clinical/lab examinations, and bone marrow aspirations, as defined by National Cancer Institute Working Group Response Criteria. (NCT00536341)
Timeframe: At 12 weeks during treatment and 2 months post-treatment until disease progression, projected 8 months

Interventionparticipants (Number)
Dose Level 14
Dose Level 29

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Overall Survival

Defined as the time from Day 1 of treatment administration to date of death from any cause, estimated using Kaplan-Meier methods. (NCT00536341)
Timeframe: Every 3 months until treatment discontinuation, expected average of 6 months and then every 6 months thereafter up to 5 years

Interventionmonths (Median)
All PatientsNA

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Number of Adverse Events as a Measure of Safety and Tolerability

Recorded from first treatment until 30 days after last treatment and assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (NCT00536341)
Timeframe: 63 months

,
Interventionparticipants (Number)
FatigueNeutropeniaAnemiaLeukopeniaThrombocytopeniaRashNauseaConstipationAnorexiaFeverHyperhidrosisArthralgiaEdema LimbsPruritusBack painHeadacheChillsInsomniaVomitingDysgeusiaAbdominal PainAllergic ReactionDiarrheaCoughDizzinessDyspneaHypotensionMyalgia
Dose Level 18787867343313031222124322012
Dose Level 24041343430272515121313141215111311111111978881098

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Progression-Free Survival

Measured from first treatment to disease progression and assessed using Kaplan-Meier methods. (NCT00536341)
Timeframe: Every 3 months during treatment until disease progression and every 6 months thereafter, up to 5 years

Interventionmonths (Median)
All Patients24.64

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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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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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Overall Survival on Day 180 Days Post-transplant

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 180 days after transplant and Kaplan-Meier survival analysis was used to generate the Overall Survival estimate at 180 days. (NCT00544466)
Timeframe: Up to 180 days post-transplant

InterventionPercent Probability (Number)
Treatment (Enzyme Inhibitor, Radiation Therapy, Transplant)81

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Number of Grade 3 and Above Toxicities of Helical Tomotherapy (HT) in Combination With Fludarabine and Melphalan Followed by Allogeneic Stem Cell Transplantation.

Toxicities (adverse events) were evaluated using the modified Bearman Scale and the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. (NCT00544466)
Timeframe: 100 days post treatment

Interventionevents (Number)
Treatment (Enzyme Inhibitor, Radiation Therapy, Transplant)793

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Percentage of Participants With Immunoglobulin Heavy Locus (IgH) Rearrangement

Percentages of participants with IgH rearrangement during the Induction Phase, Maintenance Phase, and Follow-Up were reported. (NCT00545714)
Timeframe: Post-Induction Phase: at 6 months; during Maintenance Phase: at Cycles 9, 12, 15, 18 (cycle length = 2 months); during Follow-Up: at Follow-Up Months 6, 12, 18, 24, 30, 36

InterventionPercentage of Participants (Number)
Post-IPMP (9 Cycles)MP (12 Cycles)MP (15 Cycles)MP (18 Cycles)6 Months FU12 Months FU18 Months FU24 Months FU30 Months FU36 Months FU
Rituximab + Fludarabine + Cyclophosphamide36.237.120.029.433.3100.037.950.033.30.045.8

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Overall Survival (OS)

OS was defined as time from treatment start to death of the participant. For all other participants, the last follow-up available was taken as the last control. If the participant had not completed the study, the date of the last visit available was considered. OS was estimated using Kaplan-Meier (KM) methodology. (NCT00545714)
Timeframe: Baseline up to death due to any cause (up to 92 months)

InterventionYears (Median)
Rituximab + Fludarabine + Cyclophosphamide7.51

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Duration of Response (DOR)

DOR: time from CR/PR to MRD/PD. PD: new ADP (1.5 cm), HSM, RS, other infiltrated organs or >/=50% increase size in those with PR; blood Lymph increase >/=50% with B Lymph >/=5000/mm^3; cytopenia due to CLL. Progression of (nonautoimmune) cytopenia: 2-g/dL decrease basal Hb, Hb <10 g/dL, >/=50% decrease basal Plt or <100,000/mm^3 at >/=3 months post-treatment was PD if clonal CLL cell infiltration on BM biopsy. CR: no ADP/VSM in PE; no general Sx; blood Lymph <4000/mm^3; Neut >1500/mm^3; Plt >100,000/mm^3; Hb >11 g/dL (no transfusion); normocellular BM with <30% Lymph; BM aspirate/biopsy with no lymphoid nodule infiltration. PR: >50% decrease blood Lymph; >50% decrease in total sum up to 6 ADPs or baseline ADP of LD, no new/enlargement of prior ADP; >50% decrease VSM; Neut >1500/mm^3 or >50% increase; Plt >100,000/mm^3 or >50% increase; Hb >11.0 g/dL or >50% increase (no transfusion). All CR criteria but persistent anemia or thrombocytopenia was PR. MRD: Lymph >0.01% of blood/BM WBCs. (NCT00545714)
Timeframe: From first CR or PR up to detectable MRD or disease occurrence/PD, whichever occurred first (up to 92 months)

InterventionYears (Median)
Rituximab + Fludarabine + CyclophosphamideNA

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Percentage of Participants With Positive and Negative Zeta-Chain-Associated Protein Kinase 70 (ZAP-70) Expression

Percentages of participants with positive and negative ZAP-70 expression during the Induction Phase, Maintenance Phase, and Follow-Up were reported. Positive ZAP-70 was defined as ZAP-70 expression by >/=20% of CLL cells. Negative ZAP-70 was defined as ZAP-70 expression by <20% of CLL cells. (NCT00545714)
Timeframe: Post-Induction Phase: at 6 months; during Maintenance Phase: at Cycles 9, 12, 15, 18 (cycle length = 2 months); during Follow-Up: at Follow-Up Months 6, 12, 18, 24, 30, 36

InterventionPercentage of Participants (Number)
Post-IP: PositivePost-IP: NegativeMP (9 Cycles): PositiveMP (9 Cycles): NegativeMP (12 Cycles): PositiveMP (12 Cycles): NegativeMP (15 Cycles): PositiveMP (15 Cycles): NegativeMP (18 Cycles): PositiveMP (18 Cycles): Negative6 Months FU: Positive6 Months FU: Negative12 Months FU: Positive12 Months FU: Negative18 Months FU: Positive18 Months FU: Negative24 Months FU: Positive24 Months FU: Negative30 Months FU: Positive30 Months FU: Negative36 Months FU: Positive36 Months FU: Negative
Rituximab + Fludarabine + Cyclophosphamide57.342.757.542.562.137.957.142.954.945.163.636.460.040.0100.00.059.340.7100.00.057.142.9

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Percentage of Participants With Genetic Abnormalities

Percentages of participants with genetic abnormalities (deletion 6q, deletion 11q22-q23, deletion p53, trisomy 12, and deletion 13q14) in the course of the disease during the Induction Phase and Maintenance Phase were reported. (NCT00545714)
Timeframe: Post-Induction Phase: at 6 months; during Maintenance Phase: at Cycles 9, 12, 15, 18 (cycle length = 2 months)

InterventionPercentage of Participants (Number)
Post-IP: Deletion 6qPost-IP: Deletion 11q22-q23Post-IP: Deletion p53Post-IP: Trisomy 12Post-IP: Deletion 13q14MP (9C): Deletion 6qMP (9C): Deletion 11q22-q23MP (9C): Deletion p53MP (9C): Trisomy 12MP (9C): Deletion 13q14MP (12C): Deletion 6qMP (12C): Deletion 11q22-q23MP (12C): Deletion p53MP (12C): Trisomy 12 (n= 33)MP (12C): Deletion 13q14MP (15C): Deletion 6qMP (15C): Deletion 11q22-q23MP (15C): Deletion p53MP (15C): Trisomy 12MP (15C): Deletion 13q14MP (18C): Deletion 6qMP (18C): Deletion 11q22-q23MP (18C): Deletion p53MP (18C): Trisomy 12MP (18C): Deletion 13q14
Rituximab + Fludarabine + Cyclophosphamide3.626.24.815.550.04.325.50.017.055.33.021.20.021.251.54.531.80.018.259.13.423.70.018.649.2

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Percentage of Participants With Cluster of Differentiation (CD) 38 Cells >/=30% in Peripheral Blood

Percentages of participants with CD38 expression by >/=30% of CLL cells during the Induction Phase, Maintenance Phase, and Follow-Up were reported. (NCT00545714)
Timeframe: Post-Induction Phase: at 6 months; during Maintenance Phase: at Cycles 9, 12, 15, 18 (cycle length = 2 months); during Follow-Up: at Follow-Up Months 6, 12, 18, 24, 30, 36

InterventionPercentage of Participants (Number)
Post-IPMP (9 Cycles)MP (12 Cycles)MP (15 Cycles)MP (18 Cycles)6 Months FU12 Months FU18 Months FU24 Months FU30 Months FU36 Months FU
Rituximab + Fludarabine + Cyclophosphamide47.644.445.547.647.466.745.7100.041.450.035.5

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Percentage of Participants With Clinical Response of CR or PR as Assessed by Multiparameter Flow Cytometry

CR was defined as no ADPs and VSMs in PE; no general Sx; Lymph in peripheral blood <4000/mm^3; normalization of peripheral blood parameters: Neut >1500/mm^3, Plt >100,000/mm^3, Hb >11 g/dL without transfusion; normocellular BM with <30% Lymph; BM aspirate/biopsy with no evidence of infiltration of lymphoid nodules. PR was defined as decrease >50% in Lymph in peripheral blood; reduction in ADPs >50% in total sum of up to 6 ADPs or in the baseline ADP of largest diameter (LD), no new ADP or enlargement of a prior ADP; >50% decrease in VSM; Neut >1500/mm^3 or >50% increase from Baseline; Plt >100,000/mm^3 or >50% increase from Baseline; Hb >11.0 g/dL or >50% increase from Baseline value without transfusion. Participants who met all CR criteria but had persistent anemia or thrombocytopenia were considered as PR. (NCT00545714)
Timeframe: Post-Induction Phase (IP): at 6 months; during Maintenance Phase (MP): at Cycles 9, 12, 15, 18 (cycle length = 2 months); during Follow-Up (FU): at Follow-Up Months 6, 12, 18, 24, 30, 36

InterventionPercentage of Participants (Number)
Post-IP: CRPost-IP: PRMP (9 Cycles): CRMP (9 Cycles): PRMP (12 Cycles): CRMP (12 Cycles): PRMP (15 Cycles): CRMP (15 Cycles): PRMP (18 Cycles): CRMP (18 Cycles): PR6 Months FU: CR6 Months FU: PR12 Months FU: CR12 Months FU: PR18 Months FU: CR18 Months FU: PR24 Months FU: CR24 Months FU: PR30 Months FU: CR30 Months FU: PR36 Months FU: CR36 Months FU: PR
Rituximab + Fludarabine + Cyclophosphamide75.013.189.46.487.96.190.94.588.18.583.30.094.40.0100.00.093.10.0100.00.0100.00.0

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Percentage of Participants With Clinical Response of CR or PR Among Participants With Negative Minimal Residual Disease (MRD) as Assessed by Multiparameter Flow Cytometry

CR: no ADPs and VSMs in PE; no general Sx; Lymph in peripheral blood <4000/mm^3; normalization of peripheral blood parameters: Neut >1500/mm^3, Plt >100,000/mm^3, Hb >11 g/dL without transfusion; normocellular BM with <30% Lymph; BM aspirate/biopsy with no evidence of infiltration of lymphoid nodules. PR: decrease >50% in Lymph in peripheral blood; reduction in ADPs >50% in total sum of up to 6 ADPs or in the baseline ADP of largest diameter (LD), no new ADP or enlargement of a prior ADP; >50% decrease in VSM; Neut >1500/mm^3 or >50% increase from Baseline; Plt >100,000/mm^3 or >50% increase from Baseline; Hb >11.0 g/dL or >50% increase from Baseline value without transfusion. Participants who met all CR criteria but had persistent anemia or thrombocytopenia were considered as PR. Negative MRD: Lymph <0.01% of all white blood cells (WBCs) in blood or BM after two consecutive measurements. Analysis performed only in blood during the Maintenance Phase and Follow-Up. (NCT00545714)
Timeframe: Post-Induction Phase: at 6 months; during Maintenance Phase: at Cycles 9, 12, 15, 18 (cycle length = 2 months); during Follow-Up: at Follow-Up Months 6, 12, 18, 24, 36

InterventionPercentage of Participants (Number)
Post-IP: Blood MRD NegativePost-IP: BM MRD NegativeMP (9 Cycles): Blood MRD NegativeMP (12 Cycles): Blood MRD NegativeMP (15 Cycles): Blood MRD NegativeMP (18 Cycles): Blood MRD Negative6 Months FU: Blood MRD Negative12 Months FU: Blood MRD Negative18 Months FU: Blood MRD Negative24 Months FU: Blood MRD Negative36 Months FU: Blood MRD Negative
Rituximab + Fludarabine + Cyclophosphamide100.0100.0100.0100.0100.0100.0100.0100.0100.0100.0100.0

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Treatment-Free Survival (TFS)

TFS was defined time from start of study treatment until participant received new chemotherapy/immunotherapy because of PD and to reduce the disease with palliative or curative intent. PD was defined as new ADPs (1.5 cm), HSM, RS, or other infiltrated organs; >/=50% increase in size of Baseline prior ADPs or HSM in participants with PR; Lymph increase >/=50% in peripheral blood with B Lymph >/=5000/mm^3; cytopenia attributable to CLL. Progression of any cytopenia (not related to autoimmune cytopenia) reported as a 2-g/dL decrease in basal Hb, Hb <10 g/dL, >/=50% decrease in basal Plt count, or count <100,000/mm^3 at >/=3 months post-treatment was defined as PD if BM biopsy confirmed infiltration of clonal CLL cells. (NCT00545714)
Timeframe: Baseline up to PD or death due to any cause, whichever occurred first (up to 92 months)

InterventionYears (Median)
Rituximab + Fludarabine + Cyclophosphamide4.13

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Progression-Free Survival (PFS)

PFS was defined as time from start of study treatment to PD or death, whichever occurred first. For other participants, last follow-up available was taken as last control. If participant did not complete study, date of last visit available was considered. PFS was estimated using KM methodology. PD was defined as new ADPs (1.5 cm), HSM, RS, or other infiltrated organs; >/=50% increase in size of Baseline prior ADPs or HSM in participants with PR; Lymph increase >/=50% in peripheral blood with B Lymph >/=5000/mm^3; cytopenia attributable to CLL. Progression of any cytopenia (not related to autoimmune cytopenia) reported as a 2-g/dL decrease in basal Hb, Hb <10 g/dL, >/=50% decrease in basal Plt count, or count <100,000/mm^3 at >/=3 months post-treatment was defined as PD if BM biopsy confirmed infiltration of clonal CLL cells. (NCT00545714)
Timeframe: Baseline up to PD or death due to any cause, whichever occurred first (up to 92 months)

InterventionYears (Median)
Rituximab + Fludarabine + Cyclophosphamide6.96

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Percentage of Participants With CR With Incomplete Bone Marrow Recovery (CRi)

Participants with CRi were those who met all CR criteria (including BM examinations) but had persistent anemia, thrombocytopenia, or neutropenia apparently unrelated to chronic lymphocytic leukemia (CLL) but related to drug toxicity. CR: no ADPs and VSMs in PE; no general Sx; Lymph in peripheral blood <4000/mm^3; normalization of peripheral blood parameters: Neut >1500/mm^3, Plt >100,000/mm^3, Hb >11 g/dL without transfusion; normocellular BM with <30% Lymph; BM aspirate/biopsy with no evidence of infiltration of lymphoid nodules. (NCT00545714)
Timeframe: Baseline up to progressive disease (PD) or death due to any cause, whichever occurred first (up to 92 months)

InterventionPercentage of Participants (Number)
Rituximab + Fludarabine + Cyclophosphamide7.1

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Percentage of Participants With CR Achieved After the Rituximab, Fludarabine, and Cyclophosphamide Regimen

CR was defined as no adenopathies (ADPs) and visceromegalies (VSMs) in physical examination (PE); no general symptoms (Sx); lymphocytes (Lymph) in peripheral blood less than (<) 4000 per cubic millimeter (mm^3); normalization of peripheral blood parameters: neutrophils (Neut) greater than (>) 1500/mm^3, platelets (Plt) >100,000/mm^3, hemoglobin (Hb) >11 grams per deciliter (g/dL) without transfusion; normocellular bone marrow (BM) with <30% Lymph; BM aspirate/biopsy with no evidence of infiltration of lymphoid nodules. (NCT00545714)
Timeframe: Month 9

InterventionPercentage of Participants (Number)
Rituximab + Fludarabine + Cyclophosphamide95.2

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Percentage of Participants Who Died

(NCT00545714)
Timeframe: Baseline up to death due to any cause (up to 92 months)

InterventionPercentage of Participants (Number)
Rituximab + Fludarabine + Cyclophosphamide23.2

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Percentage of Participants With PD or Death

PD was defined as new ADPs (1.5 centimeters [cm]), hepato-/splenomegaly (HSM), Richter syndrome (RS), or other infiltrated organs; greater than or equal to (>/=) 50% increase in size of Baseline prior ADPs or HSM in participants with PR; Lymph increase >/=50% in peripheral blood with B Lymph >/=5000/mm^3; cytopenia attributable to CLL. Progression of any cytopenia (not related to autoimmune cytopenia) reported as a 2-g/dL decrease in basal Hb, Hb <10 g/dL, >/=50% decrease in basal Plt count, or count <100,000/mm^3 at >/=3 months post-treatment was defined as PD if BM biopsy confirmed infiltration of clonal CLL cells. (NCT00545714)
Timeframe: Baseline up to PD or death due to any cause, whichever occurred first (up to 92 months)

InterventionPercentage of Participants (Number)
Rituximab + Fludarabine + Cyclophosphamide39.29

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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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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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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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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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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 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 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 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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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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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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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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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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Percentage of Participants With Objective Response (OR)

OR was based on assessment of complete response(CR),unconfirmed CR(Cru),partial response(PR) as per International Response Criteria for Non-Hodgkin's Lymphoma.Confirmed response=response persists on repeat imaging at least 4 weeks after initial response.CR=complete disappearance of all detectable clinical/radiographic evidence of disease,lymph nodes regressed to normal size [at least 1.5 centimeter(cm) or less],spleen regressed,not palpable on physical examination,bone marrow infiltrate cleared on repeat aspiration/biopsy.PR=at least 50 percent (%) decrease in sum of product diameters of 6 greatest dominant nodes,no increase in other nodes,liver/spleen,no new sites of disease. CRu=residual lymph node greater than 1.5 cm in transverse diameter that has regressed more than 75% in product diameter.Individual nodes previously confluent,regressed more than 75% in product diameters.Indeterminate bone marrow (increased number/size of lymph aggregates without cytologic/architectural atypia). (NCT00562965)
Timeframe: Baseline, every 6 to 12 weeks during treatment period, end of treatment (EOT) (42 days after last dose), every 12 weeks during follow-up for up to 1 year after the last dose of study drug

Interventionpercentage of participants (Number)
Rituximab + Inotuzumab Ozogamicin93.3
Control Regimens R-CVP + R-FND64.3

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Number of Participants With Grade 3 or 4 Treatment Emergent Adverse Events (TEAEs)

AE=any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. Grade 3 (Severe) events=unacceptable or intolerable events, significantly interrupting usual daily activity, require systemic drug therapy/other treatment. Grade 4 (Life-threatening) events caused participant to be in imminent danger of death. TEAE=between first dose of study drug and up to 42 days after last dose that were absent before treatment or that worsened relative to pretreatment state. (NCT00562965)
Timeframe: Baseline up to 42 days post-treatment

Interventionparticipants (Number)
Rituximab + Inotuzumab Ozogamicin12
Control Regimens R-CVP + R-FND13

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Overall Survival Probability at Months 6, 12 and 24

Overall survival was defined as the time from randomization to death due to any cause, censoring at the date of last contact or the end of the study. Participants who withdrew or lost to follow-up from study without having death documented were censored at the date of last contact. Kaplan-Meier estimates of the probability of survival at 6, 12 and 24 months was used to estimate the survival function. (NCT00562965)
Timeframe: Baseline up to Month 6, 12, 24

,
Interventionpercent chance of survival (Number)
Overall Survival: Baseline up to Month 6Overall Survival: Baseline up to Month 12Overall Survival: Baseline up to Month 24
Control Regimens R-CVP + R-FND92.383.967.1
Rituximab + Inotuzumab Ozogamicin100.086.786.7

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Number of Participants With Clinically Significant Change From Baseline in Laboratory Findings

Criteria for laboratory test abnormality: Blood Chemistry (alkaline phosphatase [greater than{>}5*upper limit of normal {ULN}, calcium [less than {<}1.75 millimole per liter {mmol/L}, creatinine [>3*ULN], glucose [>13.9 mmol/L], phosphorous [<0.6 mmol/L], potassium [<3 mmol/L], aspartate transaminase [>5.0*ULN], total bilirubin [>3*ULN]), Coagulation (international normalized ratio [>2*ULN]), Hematology (hemoglobin [<80 grams/Liter], lymphocytes [<0.5*10^9/L], absolute neutrophil count [<1*10^9/L], platelet count [<50*10^9/L], WBC [<2.0*10^9/L]). (NCT00562965)
Timeframe: Baseline up to 42 days post-treatment, disease follow up (every 12 weeks for a minimum of 1 year and up to 2 years)

,
Interventionparticipants (Number)
Baseline up to 42 days post-treatmentDisease follow up
Control Regimens R-CVP + R-FND127
Rituximab + Inotuzumab Ozogamicin118

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Number of Participants With Clinically Significant Change From Baseline in Vital Signs

Criteria for determining significant change from baseline in vital signs abnormalities: heart rate value of <40 beats per minute and value >150 beats per minute, systolic blood pressure (SBP) of <80 or >210 millimeter of mercury (mmHg), diastolic blood pressure (DBP) of <40 or >130 mmHg, temperature <32 or >40 degree centigrade, and body weight>=10% increase or decrease of body weight in kilogram (kg). (NCT00562965)
Timeframe: Baseline up to 42 days post-treatment, disease follow up (every 12 weeks for a minimum of 1 year and up to 2 years)

,
Interventionparticipants (Number)
Baseline up to 42 days post-treatmentDisease follow up
Control Regimens R-CVP + R-FND12
Rituximab + Inotuzumab Ozogamicin03

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Number of Participants With Clinically Significant Change From Baseline in QT Interval Findings

Assessments of QT interval was performed only in rituximab + inotuzumab ozogamicinin group. QT interval corrected using Fridericia's formula (QTcF) and Bazett's formula (QTcB) was analyzed as per common terminology criteria for adverse events (CTCAE) version 3.0, where Grade 1 = mild AE, Grade 2 = moderate AE, Grade 3 = severe AE, Grade 4 = life-threatening or disabling AE, Grade 5 = death related to AE. Number of participants with change in CTCAE grading at post-baseline time point compared to the baseline were presented. The post-baseline value was defined as the maximum grade after the first dose date on or before the end of treatment. (NCT00562965)
Timeframe: Baseline up to 42 days post-treatment

Interventionparticipants (Number)
QTcB: BL normal, post-BL normalQTcB: BL normal, post-BL Grade 1QTcB: BL normal, post-BL Grade 2QTcF: BL normal, post-BL normalQTcF: BL normal, post-BL Grade 1
Rituximab + Inotuzumab Ozogamicin42363

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Progression-Free Survival (PFS)

PFS was defined as the time from randomization to disease progression or death due to any cause, whichever occurred first, censored at the last tumor evaluation date. PFS calculated as (Months) = (first event date minus randomization date plus 1) divided by 30.44. (NCT00562965)
Timeframe: Baseline until disease progression or death or up to 1 year after last dose of study drug

Interventionmonths (Median)
Rituximab + Inotuzumab OzogamicinNA
Control Regimens R-CVP + R-FND16.4

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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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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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Response Outcomes

assessed according to the IWG Criteria (NCT00572897)
Timeframe: 180 days

,
Interventionparticipants (Number)
clinical complete responseclinical partial responseclinical improvementstable diseaseprogressive disease
Sibling Donor781120
Unrelated Donor61541

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The Primary Endpoint is Progression-free Survival.

Number of participants alive at 2 years who are progression-free (NCT00572897)
Timeframe: 2 years

Interventionparticipants (Number)
Sibling Donor24
Unrelated Donor11

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PLT

Patients with PLT ≥20 × 109/L (NCT00572897)
Timeframe: 2 years

,
Interventionparticipants (Number)
yesno
Sibling Donor284
Unrelated Donor2014

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Absolute Neutrophil Count (ANC)

Patients with ANC ≥0.5 × 10^9/L (NCT00572897)
Timeframe: 2 years

,
Interventionparticipants (Number)
yesno
Sibling Donor311
Unrelated Donor268

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Overall Survival

The number of patients alive at last follow-up. (NCT00572897)
Timeframe: 73 months

Interventionparticipants (Number)
Sibling Donor25
Unrelated Donor11

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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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Overall Survival at Two Years

Overall survival (OS) was measured from initial treatment to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. (NCT00577278)
Timeframe: From the initial treatment to the last disease assessment, up to two years

Interventionpercentage of survival probability (Number)
0.4 mCi 90Y-Ibritumomab Tiuxetan63

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Relapse/Progression Rate at Two Years

The primary endpoint was 2-year cumulative incidence of relapse/progression (RP), defined as time from alloHCT to disease recurrence or progression. Cumulative incidences for RP was generated in a competing-risk setting, given that death events were competing events. (NCT00577278)
Timeframe: From the initial treatment to the last disease assessment, up to two years

Interventionpercentage of cumulative incidence (Number)
0.4 mCi 90Y-Ibritumomab Tiuxetan20

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Progression-free Survival at Two Years

Progression-free survival (PFS) was measured from initial treatment to disease progression or death from any cause, whichever came first. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. (NCT00577278)
Timeframe: From the initial treatment to the last disease assessment, up to two years

Interventionpercentage of survival probability (Number)
0.4 mCi 90Y-Ibritumomab Tiuxetan61

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Number of Participants With Overall Survival (10 Years) by Treatment

Overall Survival is the time from date of treatment start until date of death due to any cause or last Follow-up within 10 years. (NCT00577993)
Timeframe: 10 Years

InterventionParticipants (Count of Participants)
Fludarabine,Mitoxantrone, and Dexamethasone (FND)-Rituximab59
Rituximab- Fludarabine,Mitoxantrone, and Dexamethasone (FND)58

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Number of Participants With Progression Free Survival (10 Years) by Treatment

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. (NCT00577993)
Timeframe: 10 years

InterventionParticipants (Count of Participants)
Fludarabine,Mitoxantrone, and Dexamethasone (FND)-Rituximab59
Rituximab- Fludarabine,Mitoxantrone, and Dexamethasone (FND)58

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Immune Reconstitution

Immune reconstitution: defined as absolute lymphocyte count (ALC) >1000x10e3/microL (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion8

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Hematopoietic Reconstitution

Hematopoietic: defined as transfusion independence. (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion7

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Event-free Survival

Event-free survival is calculated from the date of transplant to the date of graft failure, disease recurrence or death from any cause. (NCT00578292)
Timeframe: up to 2 years post transplant

Interventionprobability of event-free survival (Number)
Bone Marrow or Stem Cell Infusion0.7

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Engraftment Rate After Transplant

Engraftment is defined as an absolute neutrophil count (ANC) >500/microL x 3 days. (NCT00578292)
Timeframe: up to 30 days

Interventionproportion of participants (Number)
Bone Marrow or Stem Cell Infusion1

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Number of Participants With CHRONIC GVHD

Chronic GVHD is graded by NIH guidelines for chronic GVHD, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3 where 0 means no chronic GVHD, and 3 is the highest stage of chronic GVHD. (NCT00578292)
Timeframe: Assessed monthly from month 3 to month 12

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion1

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Number of Participants With Infectious Complications

All AEs and SAEs (including infections) will be collected for evaluation of infectious complications. (NCT00578292)
Timeframe: up to day 100

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion7

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Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)

Stable hematopoietic chimerism is defined as having 50-99 percent of donor cells. (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion3

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Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)

Transient mixed hematopoietic chimerism is defined as any mixed chimerism return to 100 percent donor. (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion0

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Percentage of Participants With Engraftment

To estimate the engraftment rate for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors. (NCT00578643)
Timeframe: 28 days post transplant

Interventionpercentage of participants (Number)
Allogeneic Unrelated Transplant100

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Number of Patients That Have Complete Donor Chimerism After Transplant.

To estimate the likelihood of complete donor chimerism for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors. (NCT00578643)
Timeframe: 120 days post transplant

Interventionparticipants (Number)
Allogeneic Unrelated Transplant13

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Number of Patients With Successful Donor Engraftment

Each patient will be classified as a success or failure. A success will be defined as engraftment of at least 35% of cells 100 days after transplant. (NCT00579111)
Timeframe: 100 days

Interventionparticipants (Number)
HLA-identical Sibling Transplant2
Unrelated Matched or Single Antigen Mismatched Transplant1

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Patients Alive at 1 Year

(NCT00579137)
Timeframe: 1 Year

Interventionparticipants (Number)
Single Group3

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Number of Patients With Grade III to IV Acute GVHD

(NCT00579137)
Timeframe: 100 days

Interventionparticipants (Number)
Single Group0

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Number of Patients With Grade III or IV Toxicity

(NCT00579137)
Timeframe: 100 days

Interventionparticipants (Number)
Single Group0

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Number of Patients With Donor Engraftment

(NCT00579137)
Timeframe: 100 Days

Interventionparticipants (Number)
Single Group1

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Number of Participants Experiencing Engraftment Donor Chimerism (EDC)

(NCT00582894)
Timeframe: At time of study termination

InterventionParticipants (Number)
Reduced Intensity Regimen17

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Number of Participants Relapse-Free

(NCT00582894)
Timeframe: 100 days post-transplant

InterventionParticipants (Number)
Reduced Intensity Regimen5

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Number of Participants Overall Survival as a Function of Time.

(NCT00582894)
Timeframe: 100 days post transplant

InterventionParticipants (Number)
Reduced Intensity Regimen11

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Death From GVHD

To establish the early transplant-related severe morbidity and mortality and 3-the incidence and severity of GvHD. (NCT00582933)
Timeframe: 2 years

Interventionparticipants (Number)
Transplant Patients4

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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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Overall Survival at Two Years Post HSCT

Patients were evaluated for survival (OS) throughout the study. Kaplan Meier estiamtes were calculated for overall survival using time from HSCT to death of any cause or for surviving patients last contact date. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients who died (Number)
All Patients65.6

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Occurrence of Thrombotic Microangiopathy

Participants were monitored throughout the trial (median of 28 months) for various infections/complications. This is the number of participants who developed TMA. (NCT00589563)
Timeframe: Median Follow Up: 28 Months (Range: 1-49 months)

Interventionparticipants (Number)
All Patients7

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Occurence of Sinusoidal Obstructive Syndrome (SOS)

Participants were monitored throughout the trial (median of 28 months) for various infections/complications. This is the number of participants who developed SOS. (NCT00589563)
Timeframe: Median Follow Up: 28 Months (Range: 1-49 Months)

Interventionparticipants (Number)
All Patients1

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Non-relapse Mortality at Two Years Post HSCT

Patients were evaluated for non-relapse mortality (NRM) throughout the study. Non-relapse mortality was considered any death not attributable to relapse or disease progression. The cumulative incidence of NRM was determined using competing risk analysis. Competing risks for NRM were death due to disease progression, relapse and nonengraftment. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients with a NRM (Number)
All Patients15.6

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Incidence of Disease Relapse/Progression at 2 Years Post HSCT

Patients were evaluated for relapse/progression post transplant throughout the study. The cumulative incidence of relapse/progression was determined using competing risk analysis. Competing risks for relapse were non-relapse mortality and nonengraftment. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients who relapsed (Number)
All Patients12.5

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Event Free Survival at Two Years Post HSCT

Patients were evaluated for event free survival (EFS) throughout the study. Events were defined as death, relapse, progression, or nonengraftment. Kaplan Meier estimates were calculated as time from HSCT to event. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients with an event (Number)
All Patients61.3

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Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease (GVHD) at Day 100

Patients were evaluated for the development of acute GVHD within the first 100 days post HSCT. The cumulative incidence of grade II-IV acute GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death and nonengraftment. (NCT00589563)
Timeframe: 100 Days Post Hematopoietic Stem Cell Transplant (HSCT)

InterventionPercentage of patients developing aGVHD (Number)
All Patients37.3

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Cumulative Incidence of Chronic GVHD

Patients were evaluated for the development of chronic GVHD from 101 days post HSCT to last contact or documented evidence of the disease. The cumulative incidence of chronic GVHD was determined using competing risk analysis. Competing risks for GVHD were death and nonengraftment. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients developing cGVHD (Number)
All Patients62.5

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Non-relapse Mortality at 100 Days Post HSCT

Patients were evaluated for non-relapse mortality (NRM) throughout the study. Non-relapse mortality was considered any death not attributable to relapse or disease progression. The cumulative incidence of NRM was determined using competing risk analysis. Competing risks for NRM were death due to disease progression, relapse and nonengraftment. (NCT00589563)
Timeframe: 100 day point estimate was provided

InterventionPercentage of patients with a NRM (Number)
All Patients9.4

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Severity of Chronic GVHD

All Patients were considered for the evaluation of chronic GVHD severity. (NCT00589563)
Timeframe: Patients were evaluated until they developed chronic GVHD, a median of 130 days post HSCT

Interventionparticipants (Number)
No Chronic GVHDYes- LimitedYes - ExtensiveNo- Inevaluable (graft failure/died
All Patients44177

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Severity of Acute GVHD

All patients were considered for the evaluation of the severity of acute GVHD. (NCT00589563)
Timeframe: 100 Days Post HSCT

Interventionparticipants (Number)
No Acute GVHDYes - Grade IYes- Grade IIYes- Grade IIIYes - Grade IVNo- Inevaluable (graft failures)
All Patients999104

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Occurence of Infections Including Cytomegalovirus and Epstein-Barr Virus Reactivation

Participants were monitored throughout the trial (median of 28 months) for various infections/complications. (NCT00589563)
Timeframe: Median Follow Up: 28 months (Range: 1-49 months)

Interventionparticipants (Number)
Neither CMV or EBVCMV reactivation onlyEBV onlyBoth CMV and EBV
All Patients16934

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Time to Platelet Count Recovery (Engraftment)

Platelet recovery is defined as the first date of three consecutive laboratory values ≥ 25 x 10^9 L obtained on different days. (NCT00589563)
Timeframe: Patients were evaluated until platelet recovery, a median of 14 days

InterventionDays (Median)
All Patients14

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Time to Absolute Neutrophil Count Recovery (Engraftment)

Absolute neutrophil count (ANC) recovery is defined as an ANC of ≥ 0.5 x 10^9/L (500/mm3) for three consecutive laboratory values obtained on different days (NCT00589563)
Timeframe: Patients were evaluated until neutrophil recovery, a median of 15 days post HSCT

InterventionDays (Median)
All Patients14.5

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Patients With Limited Chronic GVHD From Day 100 to 365

Number of patients with limited chronic GVHD from day 100 to 365 (NCT00590460)
Timeframe: 365 days

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant0

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Patients With Grade III - IV Acute GVHD

Number of patients with Grade III-IV acute GVHD (NCT00590460)
Timeframe: 100 days

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant0

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Patients With Grade II - IV Acute Graft Versus Host Disease (GVHD)

Number of patients with grade II - IV acute Graft versus Host Disease (GVHD) (NCT00590460)
Timeframe: 100 days

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant0

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Days to Absolute Neutrophil Count (ANC) of 500/mm3

Number of days to Absolute neutrophil count (ANC) of 500/mm3 (NCT00590460)
Timeframe: 30 Days

Interventiondays (Median)
Allogeneic Stem Cell Transplant15

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Patients With Extensive Chronic GVHD From Day 100 to 365

Number of patients with extensive chronic GVHD from day 100 to 365. (NCT00590460)
Timeframe: 365 days

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant0

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Number of Patients With Donor Engraftment

Number of patients with engraftment of at least 65% of donor cells 100 days after transplantation (NCT00590460)
Timeframe: 100 Days

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant2

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Number of Patients Alive at 1 Year Post Transplant

Number of patients alive at 1 year post allogeneic stem cell transplant (NCT00590460)
Timeframe: 1 year

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant5

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Number of Patients With Graft Failure

Graft failure is defined as engraftment of less than 65% of donor cells 100 days after transplantation. (NCT00590460)
Timeframe: 100 days

Interventionparticipants (Number)
Allogeneic Stem Cell Transplant3

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Days to Platelet Count of 20,000/mm3 Without Transfusions

Number of days to Platelet count of 20,000 / mm3 without transfusions (NCT00590460)
Timeframe: 30 Days

Interventiondays (Median)
Allogeneic Stem Cell Transplant16

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Time to Platelet Engraftment

Time to platelet engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of platelets is defined as the time from day 0 to the first of seven consecutive days after transplantation during which the platelet count is at least 20 x109/l without transfusion support. Only patients who achieved engraftment of platelets will be included in the analysis. The median and 95% confidence intervals will be provided. (NCT00593554)
Timeframe: Transplant (Day 0) up to 1 year

Interventiondays (Median)
Treatment Without Paliferim22.5
Treatment With Palifermin20

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Acute Graft vs. Host Disease (GvHD)

Number of unique patients who had acute Graft vs. Host Disease (GvHD) diagnosed while on the study. (NCT00593554)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Treatment Without Paliferim0
Treatment With Palifermin0

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Chronic Graft vs. Host Disease (GvHD)

Number of unique patients who had chronic Graft vs. Host Disease (GvHD) diagnosed while on the study. (NCT00593554)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Treatment Without Paliferim0
Treatment With Palifermin0

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Frequency of Infection

Number of unique patients with bacterial and/or viral infections reported. (NCT00593554)
Timeframe: Day 0 through 1 year post transplantation

InterventionParticipants (Count of Participants)
Treatment Without Paliferim2
Treatment With Palifermin5

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Time to Neutrophil Engraftment

Time to neutrophil engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of neutrophil is defined as the time from transplant until absolute neutrophil count (ANC) > 500 uL for 3 consecutive days. The median and 95% confidence intervals will be provided. (NCT00593554)
Timeframe: Transplant (Day 0) up to 1 year

Interventiondays (Median)
Treatment Without Paliferim15
Treatment With Palifermin14

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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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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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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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Incidence & Quality of Engraftment & Hematopoietic Reconstitution

Number of patients who engrafted (NCT00595127)
Timeframe: 8 years

Interventionparticipants (Number)
Cyclophosphamide and Fludarabine19

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Overall Response

To obtain a preliminary estimate of efficacy of double unit UCBT as measured by overall response. (NCT00597519)
Timeframe: 1 year

Interventionparticipants (Number)
Complete RemissionRelapse
Treatment243

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Time-to-progression in Patients With Del(11q22.3)

Time to progression (TTP) in del(11q22.3) participants was defined as the registration date to date of progression or death due to any cause, whichever occurs first. TTP was estimated using the Kaplan Meier method. (NCT00602459)
Timeframe: Up to 15 years

Interventionmonths (Median)
Arm C2, FCR in Del(11q22.3)35.5
Arm D, FCR+L in Del(11q22.3)44.6

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Time-to-progression in Patients Without Del(11q22.3)

Time to progression (TTP) was defined as the registration date to date of progression or death due to any cause, whichever occurs first. TTP was estimated using the Kaplan Meier method. Progressive disease (PD) required at least one of the following: >= 50% increase in the absolute number of lymphocytes, appearance of new palpable lymph nodes, >= 50% increase in the product of at least two lymphnodes, >= 50% increase in the enlargement of the liver and/or spleen. (NCT00602459)
Timeframe: Up to 15 years

Interventionmonths (Median)
Arm A, FR in Non-del(11q22.3)43.5
Arm B, FR+L in Non-del(11q22.3)66.0
Arm C1, FCR in Non-del(11q22.3)78.0

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Overall Response Rates in Patients With Del(11q22.3)

Percentage of del(11q22.3) participants with a complete response (CR) or partial response (PR). CR: no lymphadenopathy, hepatomegaly, splenomegaly or constitutional symptoms; normal complete blood count; confirmed by bone marrow (BM) aspirate & biopsy PR: 50% decrease in peripheral blood lymphocytes, lymphadenopathy, liver/spleen size, presence/absence of constitutional symptoms; plus >= 1 of the following: >= 1500/uL polymorphonuclear leukocytes, > 100,000/uL platelets, > 11.0 g/dL hemoglobin or 50% improvement for these parameters without transfusions. (NCT00602459)
Timeframe: Up to 15 years

Interventionpercentage of participants (Number)
Arm C (Rituximab, Fludarabine Phosphate, Cyclophosphamide)59
Arm D (Rituximab, Fludarabine, Cyclophosphamide, Lenalidomide)74

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PFS Rate of Patients With Del(11q22.3)

Proportion of del (11q22.3) participants who were alive and progression free at 2 years. (NCT00602459)
Timeframe: 2 years

Interventionproportion of participants (Number)
Arm C2, FCR in Del(11q22.3)0.56
Arm D, FCR+L in Del(11q22.3)0.65

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Overall Response Rate in Patients Without Del(11q22.3)

Percentage of non-del(11q22.3) participants with a complete response (CR) or partial response (PR). CR: no lymphadenopathy, hepatomegaly, splenomegaly or constitutional symptoms; normal complete blood count; confirmed by bone marrow (BM) aspirate & biopsy PR: 50% decrease in peripheral blood lymphocytes, lymphadenopathy, liver/spleen size, presence/absence of constitutional symptoms; plus >= 1 of the following: >= 1500/uL polymorphonuclear leukocytes, > 100,000/uL platelets, > 11.0 g/dL hemoglobin or 50% improvement for these parameters without transfusions. (NCT00602459)
Timeframe: Up to 15 years

Interventionpercentage of participants (Number)
Arm A (Rituximab, Fludarabine Phosphate)75
Arm B (Rituximab, Fludarabine Phosphate, Lenalidomide)69
Arm C (Rituximab, Fludarabine Phosphate, Cyclophosphamide)71

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2-Year Progression Free Survival (PFS) Rate

Proportion of participants who were alive and progression free at 2 years. (NCT00602459)
Timeframe: 2 years

Interventionproportion of participants (Number)
Arm A, FR in Non-del(11q22.3)0.64
Arm B, FR+L in Non-del(11q22.3)0.71
Arm C1, FCR in Non-del(11q22.3)0.74

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Quality and Timing of Immunologic Reconstitution: Concentration of ATG Levels

Analyses of ATG levels in order to assess the immune system recuperation (NCT00603954)
Timeframe: Day 0, Day 3 and Day 10

Interventionmg/L (Median)
Median ATG serum levels at day 0Median ATG serum levels at day 3Median ATG serum levels at day 10
TLI-ATG42.20.95

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Percentage of 4-year Progression Free Survival in the 2 Groups

(NCT00603954)
Timeframe: 4 year after HCT

Interventionpercentage of participants (Number)
Flu-TBI54
TLI-ATG37

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Percentage of 5-year Overall Survival in the 2 Groups

(NCT00603954)
Timeframe: 5 year after HCT

Interventionpercentage of participants (Number)
Flu-TBI53
TLI-ATG55

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Percentage of Participants With Chronic GVHD in the 2 Groups

Comparaison of the number of Participants with chronic GVHD in the 2 groups (NCT00603954)
Timeframe: 2 years after HCT

Interventionpercentage of participants (Number)
Flu-TBI40.8
TLI-ATG18.8

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Percentage of 5-year Progression Free Survival in the 2 Groups

(NCT00603954)
Timeframe: 5 year after HCT

Interventionpercentage of participants (Number)
Flu-TBI50
TLI-ATG37

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Number of Participants With Graft Rejection as Defined by Whole Blood and T Cell Chimerism

graft rejection are reported in outcome measure data table (defined as ≤ 5% donor chimerism in T cells, total white blood cells and/or total bone marrow cells). (NCT00603954)
Timeframe: 1 year after HCT

Interventionparticipants (Number)
Flu-TBI3
TLI-ATG4

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Percentage of 4-year Overall Survival in the 2 Groups

(NCT00603954)
Timeframe: 4 year after HCT

Interventionpercentage of participants (Number)
Flu-TBI53
TLI-ATG54

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Incidences of Bacterial, Fungal and Viral Infections in the 2 Groups.

(NCT00603954)
Timeframe: D100 after HCT

,
Interventionparticipants (Number)
Bacterial infectionFungal infectionCMV reactivation
Flu-TBI19315
TLI-ATG25721

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Percentage of Non Relapse Mortality in the 2 Groups

(NCT00603954)
Timeframe: 1 year after HCT

Interventionpercentage of participants (Number)
Flu-TBI24
TLI-ATG13

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Percentage of Participants With Grade II-IV Acute GVHD Between the 2 Groups

"Percentage of participants with aGVHD according grades:~Grade I: rash skin < 25 % area; bilirubin: 20-30 mg/ml; diarrhea: 500-1000 ml/day Grade II: rash skin 25-50 % area; bilirubin: 30-60 mg/ml; diarrhea: 10000-1500 ml/day Grade III:rash skin > 50 % area; bilirubin: 60-150 mg/ml; diarrhea: >1500 ml/day Grade IV: erythroderma; bilirubin: > 150 mg/ml; diarrhea: >2000 ml/day~Grade IV is the worst grade Patients given a second allogeneic HCT were censured for GVHD analyses." (NCT00603954)
Timeframe: 180 days after HCT

Interventionpercentage of participants with aGVHD (Number)
Flu-TBI12.2
TLI-ATG8.9

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Percentage of Relapse Rate in the 2 Groups

(NCT00603954)
Timeframe: 1 year after HCT

Interventionpercentage of Relapse (Number)
Flu-TBI22
TLI-ATG50

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Number of Participants With 100-day Non-relapse Mortality

Evaluate the safety (as determined by the day 100 non-relapse mortality) and feasibility of single or double umbilical cord blood (UCB)stem cell transplant (SCT) in adult or pediatric patients with hematologic malignancies receiving graft-versus-host disease (GVHD) prophylaxis with tacrolimus and mycophenolate mofetil (MMF). (NCT00608517)
Timeframe: 100 days

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning1
Adult Myeloablative Conditioning0
Reduced-intensity Conditioning0

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Number of Participants With Chronic Graft Versus Host Disease (GVHD)

As opposed to acute GVHD, which is characterized by rash, cholestasis, and enteritis, chronic GVHD is characterized by nausea, anorexia, ocular and oral sicca, and other organ involvement (NCT00608517)
Timeframe: 100 days

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning0
Adult Myeloablative Conditioning0
Reduced-intensity Conditioning0

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Number of Subjects With All-cause Mortality

Death from any cause at 100 days (NCT00608517)
Timeframe: at 100 days

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning1
Adult Myeloablative Conditioning0
Reduced-intensity Conditioning0

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Number of Participants With Sustained Donor Engraftment of Umbilical Cord Blood Stem Cells

Recovery of the neutrophil portion of white blood cells and showing complete donor cells. (NCT00608517)
Timeframe: 42 days

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning1
Adult Myeloablative Conditioning1

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Overall Survival

Overall survival at 1 year (NCT00608517)
Timeframe: 1 year

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning1
Adult Myeloablative Conditioning3
Reduced-intensity Conditioning1

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Number of Participants Who Relapsed at 1 Year

(NCT00608517)
Timeframe: 1 year

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning0
Adult Myeloablative Conditioning0
Reduced-intensity Conditioning0

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Number of Participants With Acute Graft-versus-host Disease (GVHD)

Participants who exhibit acute GVHD. (NCT00608517)
Timeframe: 100 days

Interventionparticipants (Number)
Pediatric Myeloablative Conditioning0
Adult Myeloablative Conditioning1
Reduced-intensity Conditioning0

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Number of Participants With Metastatic Melanoma Who Develop Clinical Tumor Regression (CR or PR)

Clinical tumor response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v.1.0 criteria. Complete response (CR) is a disappearance of all target lesions. Partial response (PR) is a 30% decrease in lesions taking as reference the baseline sum longest diameter (LD). For details about the RECIST criteria see the protocol link module. (NCT00610311)
Timeframe: 4-6 weeks after treatment and then monthly for approximately 3 to 4 months or until off study criteria are met

InterventionParticipants (Number)
Partial response (PR)Complete response (CR)
ALVAC Plus Anti-gp100:154-162 TCR PBL + HD IL-210

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. (NCT00610311)
Timeframe: 18.5 months

InterventionParticipants (Number)
ALVAC Plus Anti-gp100:154-162 TCR PBL + HD IL-23

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. (NCT00612222)
Timeframe: 15 months

InterventionParticipants (Number)
ALVAC Plus Anti-MART-1 F5 TCR PBL + HD IL-24

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Number of Participants With Metastatic Melanoma Who Develop Clinical Tumor Regression (CR or PR)

Clinical tumor response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v.1.0 criteria. Complete response (CR) is a disappearance of all target lesions. Partial response (PR) is a 30% decrease in lesions taking as reference the baseline sum longest diameter (LD). For details about the RECIST criteria see the protocol link module. (NCT00612222)
Timeframe: 4-6 weeks after treatment and then monthly for approximately 3 to 4 months or until off study criteria are met

InterventionParticipants (Number)
ALVAC Plus Anti-MART-1 F5 TCR PBL + HD IL-20

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The Percentage of Patients Free From Progression at 1 Year

"One of the secondary outcomes that will be measured is progression free survival at 1 Year.~Progressive Disease (PD) is defined as a >25% increase in serum monoclonal paraprotein, a >25% increase in 24-hour urinary light chain excretion, a >25% increase in plasma cells in bone marrow aspirate, an increase in the size or the development of new bone lesions/soft tissue plasmacytomas, or the development of hypercalcemia." (NCT00615589)
Timeframe: 1 Year

Interventionpercentage of patients (Number)
Flu-Bu440

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Non Relapse Mortality (NRM) at 1 Year and 3 yearsThe Percentage of Deaths Not Attributable to Disease Relapse or Progression

Non relapse mortality, defined as the percentage of deaths not attributable to disease relapse or progression at 1 year and at 3 years. (NCT00615589)
Timeframe: 3 years

Interventionpercentage of deaths (Number)
NRM at 1 YearNRM at 3 Years
Flu-Bu41929

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The Percentage of Patients Alive 1 Year Post Transplant

The primary objective is overall survival, one year from the time of transplant. (NCT00615589)
Timeframe: 1 Year

Interventionpercentage of patients (Number)
Flu-Bu461

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Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)

"Incidence of acute (Stage II-IV and Stage III-IV) and chronic GVHD (any stage) were analyzed.~Acute GVHD Grading:~Stage II - Skin, 25-50% BSA (Body Surface Area); Liver, 3.1-6mg/dl bilirubin; Gut, 1000-1500ml/day diarrhea Stage III - Skin, generalized erythroderma; Liver, 6.1-15mg/dl bilirubin; Gut, >1500ml/day diarrhea Stage IV - Skin, bullae; Liver, >15mg/dl bilirubin; Gut, pain +/- ileus" (NCT00615589)
Timeframe: 100 days, 2 years

Interventionpercentage of participants (Number)
Grade II-IV Acute GVHDGrade III-IV Acute GVHDChronic GVHD
Flu-Bu4482355

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Overall Survival

Count of patients alive at 1 and 3 years. Deaths from any cause are events. Surviving patients are censored at the date of last contact. (NCT00618540)
Timeframe: Year 1, Year 3

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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Platelet Engraftment

Incidence of platelet recovery and donor chimerism at Day 100. (NCT00618540)
Timeframe: Day 100

Interventionparticipants (Number)
Alemtuzumab0

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Incidence of Chronic GVHD

Occurrence of symptoms in any organ system fulfilling the criteria of limited or extensive chronic GvHD (Appendix III), among patients surviving > 90 days with evidence of engraftment. Patients without chronic GvHD will be censored at time of death or last follow-up. (NCT00618540)
Timeframe: Day 100 and Month 6

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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

The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the criteria of Grades II, III and/or IV acute GVHD are considered events (Appendix II). Patients without acute GvHD will be censored at the time of death or last follow-up. Patients that survive <21 days and listed as not evaluable will be excluded. Patients receiving a second transplant will be censored at the time of second transplant. (NCT00618540)
Timeframe: Day 100 and Month 6

Interventionparticipants (Number)
Alemtuzumab Conditioning1

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

The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the criteria of Grades II, III and/or IV acute GVHD are considered events (Appendix II). Patients without acute GvHD will be censored at the time of death or last follow-up. Patients that survive <21 days and listed as not evaluable will be excluded. Patients receiving a second transplant will be censored at the time of second transplant. (NCT00618540)
Timeframe: Day 100 and Month 6

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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Neutrophil Engraftment

Incidence of neutrophil recovery and donor chimerism at Day 100. (NCT00618540)
Timeframe: Day 100

Interventionparticipants (Number)
Alemtuzumab Conditioning1

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Disease-free Survival at 12 Months Post Transplantation

"This outcome is defined as survival with resolution of LCH at 12 months post transplant.~Unresolved disease for over 12 months post-transplant, progressive disease after this time period, recurrence of disease and death from any cause are considered events.~Those who survive with resolution of disease are censored at the date of last contact." (NCT00618540)
Timeframe: Year 1

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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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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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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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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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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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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Percentage of Participants Alive at 1 Year

One of the secondary objectives was to determine overall survival for patients > 55 years in age with AML undergoing full or reduced transplant with the best available donor. (NCT00623935)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Fludarabine Plus Busulfan58

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Percentage of Participants With Relapse Free Survival at 1 Year

The primary objective was to determine the 1 year relapse free survival rate (RFS) for individuals > 55 years in age with Acute myeloid leukemia (AML) in Complete Remission (CR) or Partial Remission (PR) who undergo a 7-8/8 HLA- matched unrelated donor transplant using a reduced intensity regimen. (NCT00623935)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Fludarabine Plus Busulfan56

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Number of Patients Whose Disease Progressed After Treatment

Includes patients (with non-Hodgkin leukemia or chronic lymphocytic leukemia) whose disease progressed after treatment. (NCT00625729)
Timeframe: 6 Months

InterventionParticipants (Number)
Responder Patients2

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Number of Patients Exhibiting Natural Killer Cell Expansion

Successful natural killer (NK) cell expansion will be defined as an absolute circulating donor-derived NK cell count of >100 cells/μl 14 days after infusion with <5% donor T and B cells in the mononuclear population. (NCT00625729)
Timeframe: Day 14

InterventionParticipants (Number)
Patients Treated With Natural Killer Cells0

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Number of Patients With Adequate Natural Killer Cells Infused

Incidence of donor products that met release criteria in accordance with FDA regulations (Lot Release Criteria for allogeneic, interleukin-2 (IL-2) activated natural killer (NK) cell products (BB-IND 8847) and the NK cell numbers infused (donor NK cell dose 1.5-8.0 x 10^7/kg). (NCT00625729)
Timeframe: Day 0

InterventionParticipants (Number)
Patients Treated With Natural Killer Cells6

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Number of Patients With Interleukin-15 Production and NK Cell Expansion

Correlation of interleukin-15 production at day 0 with natural killer (NK) cells expansion (NCT00625729)
Timeframe: Day 0

InterventionParticipants (Number)
Patients Treated With Natural Killer Cells0

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Number of Patients With Overall Survival

Number of patients alive at 6 months after treatment. (NCT00625729)
Timeframe: 6 Months

InterventionParticipants (Number)
Patients Treated With Natural Killer Cells3

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Number of Patients With Overall Response

Overall response (complete remission plus partial remission) rate at 3 months, as defined by International Working Group for non-Hodgkin lymphoma and NCI Working Group guidelines for chronic lymphocytic leukemia (NCT00625729)
Timeframe: 3 Months

InterventionParticipants (Number)
Patients Treated With Natural Killer Cells4

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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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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 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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Progression Free Survival

Progression is defined as at least one of the following: 1) ≥ 50% increase in the sum of the products of at least two lymph nodes one two consecutive determinations (at least one node must be ≥ 2 cm); appearance of new palpable lymph nodes, 2) ≥ 50% increase in the size of the liver and/or spleen; appearance of palpable hepatomegaly or splenomegaly, which was not previously present, 3) ≥ 50% increase in the absolute number of circulating lymphocytes to at least 5,000/µl or 4)Transformation to a more aggressive histology. (NCT00636155)
Timeframe: 5 years

Interventionmonths (Median)
All Patients5.3

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Overall Survival

(NCT00636155)
Timeframe: 5 years

Interventionmonths (Median)
All Patients10.6

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Number of Patients With an Overall Response (Complete Response + Partial Response)

Overall Response is the total number of participants with a Complete (CR) or Partial (PR) response. CR requires the absence of lymphadenopathy, hepatomegaly or splenomegaly and constitutional symptoms and a normal CBC; bone marrow must be at least normocellular for age, with less than 30% nucleated cells being lymphocytes with no lymphoid nodules. Partial Response: requires ≥ 50% decrease in one of the following: peripheral blood lymphocyte count, lymphadenopathy, enlargement of liver and/or spleen, or bone marrow involvement by CLL AND at least one hematologic parameter met for 2 months. (NCT00636155)
Timeframe: every 3 cycles

Interventionparticipants (Number)
All Patients9

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Number of Patients With In Vivo Expansion of Infused Allogeneic Natural Killer (NK) Cell Product

Detection of an absolute donor derived cell count of > or = 100 cells/mL after NK cell infusion. (NCT00652899)
Timeframe: Day 12-14

InterventionPatients (Number)
Ovarian/Fallopian Tube/Peritoneal Cancer Patients0

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Median Overall Survival Number of Days Patients Alive After Treatment

Median number of days patients alive from date of treatment to date of death or date of last follow-up if censored. (NCT00652899)
Timeframe: From first date on-study (treatment) to date of death

InterventionDays (Median)
Total Body Irradiation171.5
No Total Body Irradiation291

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Median Number of Days to Progression

Median number of days from first date of treatment to date of disease progression (appearance of new metastatic lesions or objective tumor progression). Defined by computated tomography (CT) imaging based on Response Evaluation Criteria In Solid Tumors (RECIST): Progressive Disease (PD) > or = 20% increase in sum of all target or any new lesions. (NCT00652899)
Timeframe: From date of first treatment to disease progression

InterventionDays (Median)
No Total Body Irradiation107
Total Body Irradiation90

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Number of Patients Per Disease Response

Response Evaluation Criteria in Solid Tumors (RECIST) criteria: Complete Response (CR)-Disappearance of all target lesions (TL); Partial Response (PR)-< or = 30% decrease in the sum of the longest diameter (LD) of TL, reference baseline sum LD; Stable Disease (SD)-Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, reference the smallest sum LD since the treatment started; Progressive Disease (PD)- < or = 20% increase in the sum of the LD of TL, reference the smallest sum LD recorded since treatment started or appearance of < or = 1 new lesion. (NCT00652899)
Timeframe: 1 Month After Natural Killer Cell Infusion (Day 30)

,
InterventionPatients (Number)
Complete ResponsePartial ResponseStable DiseaseProgressive Disease
No Total Body Irradiation0241
Total Body Irradiation0140

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Number of Participants With Serious and Non-Serious Adverse Events

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT00670748)
Timeframe: Date treatment consent signed to date off study, approximately, 66 months and 10 days

InterventionParticipants (Count of Participants)
#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC17
#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa16
#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC7
#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa5

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Clinical Response Per the Response Evaluation Criteria in Solid Tumors (RECIST)

Response was determined by the RECIST. Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT00670748)
Timeframe: Approximately 3 years

,,,
InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseProgressive DiseaseStable DiseaseNot Evaluable
#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC36701
#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa17701
#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC11401
#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa03200

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Number of Participants With In Vivo Survival of T-Cell Receptor (TCR)-Engineered Cells

Immunological monitoring using both tetramer analysis and staining for the T cell receptor (TCR). This will provide data to estimate the in vivo survival of lymphocytes derived from the infused cells. (NCT00670748)
Timeframe: 1 month post treatment

InterventionParticipants (Count of Participants)
#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC16
#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa16
#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC6
#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa4

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Median Disease-free Survival

"All patients were administered the following drugs;~Fludarabine 30mg/m2 intravenously daily at the same time over 30 min on days -7,-6,-5,-4, and -3~Melphalan 140mg/m2 IV on day -2~Stem cell infusion on day 0~Campath 20mg IV on day -7,-6,-5,-4, and -3" (NCT00683046)
Timeframe: Patients evaluated continuously with disease specific re-evaluation at day 30, 3 months, 6 months, 1 year, and as indicated thereafter up to 10 years

InterventionDays (Median)
T-cell Depleted Allogeneic Stem Cell Transplantation333

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Median Overall Survival

"All patients were administered the following drugs;~Fludarabine 30mg/m2 intravenously daily at the same time over 30 min on days -7,-6,-5,-4, and -3~Melphalan 140mg/m2 IV on day -2~Stem cell infusion on day 0~Campath 20mg IV on day -7,-6,-5,-4, and -3" (NCT00683046)
Timeframe: Patients evaluated continuously with disease specific re-evaluation at day 30, 3 months, 6 months, 1 year, and as indicated thereafter up to 10 years

InterventionDays (Median)
T-cell Depleted Allogeneic Stem Cell Transplantation547

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Severity of Acute Graft-versus-host Disease (GVHD)

(NCT00691015)
Timeframe: Within 100 days after donor peripheral blood stem cell transplantation (PBSCT) as assessed by Glucksberg criteria

Intervention% of participants with severe aGVHD (Number)
All Participants33.3

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Incidence of Acute Graft-versus-host Disease (GVHD)

(NCT00691015)
Timeframe: Within 100 days after donor peripheral blood stem cell transplantation (PBSCT) as assessed by Glucksberg criteria

Interventionpercentage of participants (Number)
All Participants44.7

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Time to Engraftment (i.e., Absolute Neutrophil Recovery [ANC > 500/mm³] )

(NCT00691015)
Timeframe: post transplant, up to 4 weeks

InterventionDays (Median)
All Participants11

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Incidence of Chronic GVHD.

(NCT00691015)
Timeframe: Within 2 years after PBSCT

Interventionpercentage of participants (Number)
All Participants44.68

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Incidence of Infections, Including Bacterial, Fungal, and Viral Infections (i.e., CMV and EBV Reactivation, Including Post-transplant Lymphoproliferative Disorders)

(NCT00691015)
Timeframe: Within 6 months after PBSCT

Interventionpercentage of participants (Number)
All Participants80.85

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Karnofsky Performance Status Performance Status

"100 - Normal; no complaints; no evidence of disease. 90 - Able to carry on normal activity; minor signs or symptoms of disease. 80 - Normal activity with effort; some signs or symptoms of disease. 70 - Cares for self; unable to carry on normal activity or to do active work. 60 - Requires occasional assistance, but is able to care for most of their personal needs.~50 - Requires considerable assistance and frequent medical care. 40 - Disabled; requires special care and assistance. 30 - Severely disabled; hospital admission is indicated although death not imminent.~20 - Very sick; hospital admission necessary; active supportive treatment necessary.~10 - Moribund; fatal processes progressing rapidly. 0 - Dead" (NCT00691015)
Timeframe: At 90 days after PBSCT

Interventionunits on a scale (Median)
All Participants80

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Overall Survival.

(NCT00691015)
Timeframe: At 2 years after PBSCT

Interventionpercentage of participants (Number)
All Participants57.4

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Clinical Response (Complete Response + Partial Response)

Clinical response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is disappearance of all lesions. Partial response is a 30% decrease in the sum of the longest diameter (LD) of target lesions. (NCT00704938)
Timeframe: 5 months

,
InterventionParticipants (Number)
Complete ResponsePartial Response
Anti-p53 TCR PBL + DC + IL-2: Melanoma/RCC00
Anti-p53 TCR PBL + DC + IL-2: Other Histology00

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For the detailed list of adverse events see the adverse events module. (NCT00704938)
Timeframe: 5 months

InterventionParticipants (Number)
Anti-p53 TCR PBL + DC + IL-2: Melanoma/RCC2
Anti-p53 TCR PBL + DC + IL-2: Other Histology1

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Composite Incidence of Acute and Chronic Graft Versus Host Disease

(NCT00714259)
Timeframe: Up to 100 days post transplant.

Interventionparticipants (Number)
Single Arm Trial2

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Number of Participants With Detectable Donor Chimerism at up to 100 Days Post Transplant

Measured by number of participants that have chimerism study results that show the number of donor cells and the number of recipient cells present in the blood after post-transplant lymphocyte infusions continue to be predominately either donor or recipient. (NCT00714259)
Timeframe: Post transplant up to 100 days post transplant

Interventionparticipant (Number)
Single Arm Trial1

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Progression Free Survival Post Transplant

Subjects surviving without disease progression 2 years after transplant as evidenced by no new disease showing on radiologic scans and / or bone marrow pathology. (NCT00714259)
Timeframe: 2 years post transplant

Interventionparticipants (Number)
Single Arm Trial2

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Progressive Free Survival Post Transplant

Subjects surviving without disease progression 180 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology. (NCT00714259)
Timeframe: 180 days post transplant

Interventionparticipants (Number)
Single Arm Trial4

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Progressive Free Survival Post Transplant

Subjects surviving without disease progression 365 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology. (NCT00714259)
Timeframe: 365 days post transplant

Interventionparticipants (Number)
Single Arm Trial4

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Progressive Free Survival Post Transplant

subjects surviving without disease progression at 100 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology. (NCT00714259)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
Single Arm Trial4

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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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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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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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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 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 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 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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Number of Participants With Graft Failure/Rejection

descriptive (NCT00723099)
Timeframe: By day 55

Interventionparticipants (Number)
Treatment (Chemotherapy, Transplant)3

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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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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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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 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 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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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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Median Time to ANC > 500

(NCT00723099)
Timeframe: By day 55

Interventiondays (Median)
Treatment (Chemotherapy, Transplant)18

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Number of Patients Reaching Disease-free Survival (DSF) Overall

Response will be assessed by clinical examination, peripheral blood, bone marrow aspirate and biopsy, radiographic evaluation. Response will be evaluated at three different levels: clinical, cytometric and molecular. (NCT00727415)
Timeframe: After 6 months from study entry (end of treatment)

Interventionpercentage of participants on DFS (Number)
Phase I-II Lenalidomide35.33

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Maximum Tolerated Dose of Lenalidomide (Phase I)

Maximum tolerated dose of lenalidomide given in combination with fludarabine. (NCT00727415)
Timeframe: The MTD of Lenalinomide will be evaluated during the two courses given with the escalated dose of Lenalinomide defined by the respective dose level.

Interventionnumber of patients without DLT (Number)
Dose level 1 - 5 mgDose level 2 - 10 mg
Phase I-II Lenalidomide61

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Number of Patients With Severe Infections

Severe infection requiring more than 2 weeks of antibiotic therapy. (NCT00727415)
Timeframe: At 24 months from study entry (end of follow-up)

Interventionparticipants with severe infecitons (Number)
Phase I-II Lenalidomide2

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Correlation Between Complete Response (CR) and Baseline Biologic Parameters (i.e., IgHV, CD38, Etc.).

(NCT00727415)
Timeframe: After 6 months from study entry (end of treatment).

Interventionpercentage of participants (Number)
CR according to IgHV mutatedCR according to CD19+/CD38+, <30%CR according to CD19+/CD38+, >30%CR according to deletion 11q and 17p, absent
Phase I-II Lenalidomide28.0033.3316.6731.82

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Toxicity as Assessed by NCI CTCAE v3.0

Data from all subjects who receive any study drug will be included in the safety analyses. (NCT00727415)
Timeframe: At 24 months from study entry (end of follow-up)

InterventionParticipants who died during the study (Number)
Phase I-II Lenalidomide14

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Overall Complete Response (CR) Rate (Phase II)

Response will be assessed by clinical examination, peripheral blood, bone marrow aspirate and biopsy, radiographic evaluation. Response will be evaluated at three different levels: clinical, cytometric and molecular. (NCT00727415)
Timeframe: After 6 months from study entry (end of treatment).

Interventionpercentage of patients in CR (Number)
Phase I-II Lenalidomide22.5

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Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients

Successful primary donor engraftment = neutrophill recovery within the first 45 days after transplant and partial/complete donor chimerism (>/= 10%) (NCT00739141)
Timeframe: up to 13 days from engraftment

Interventionparticipants (Number)
Haplo-dCBT with early haplo-derived myeloid bridgeHaplo-dCBT with transient haplo-derived bridge with second neutrophil nadirHaplo-dCBT with no bridgeRemaining dCBT recipient
Participants With Hematologic Malignancies17131343

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Progression Free Survival/PFS at 1 Year Post UCBT.

To obtain a preliminary estimate of progression free survival at 1 year post UCBT. 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 (NCT00739141)
Timeframe: 1 year post UCBT

Interventionpercentage of participants (Number)
Participants With Hematologic Malignancies84

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Percentage of Participants With Sustained CB-derived Platelet Engraftment

The day 100 cumulative incidence of sustained CB-derived platelet engraftment to >/= 20 x 10^9/L (NCT00739141)
Timeframe: 100 days

InterventionPercentage of participants (Number)
Participants With Hematologic Malignancies93

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Percentage of Participants With Sustained CB-derived Neutrophil Engraftment

The day 100 cumulative incidence of sustained CB-derived neutrophil engraftment. (NCT00739141)
Timeframe: 100 days

InterventionPercentage of participants (Number)
Participants With Hematologic Malignancies99

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Number of Participants With Successful Bone Marrow Engraftment

Rates of successful engraftment. (NCT00741455)
Timeframe: Within 30 days of bone marrow transplant

InterventionParticipants (Count of Participants)
Engrafted Engrafted 16-30 DaysEngrafted >30 Days
Study Treatment1151

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Number of Participants Who Achieve Complete Donor Chimerism

Complete donor chimerism (NCT00741455)
Timeframe: Post-transplant days +30, +60, +100, +180 and +365

InterventionParticipants (Count of Participants)
Complete Chimerism 30 Days Post TransplantComplete Chimerism 60 Days Post TransplantComplete Chimerism 100 Days Post TransplantComplete Chimerism 180 Days Post TransplantComplete Chimerism 365 Days Post TransplantChimerism UnknownDid not achieve complete donor chimerism
Study Treatment2233412

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Overall Survival Measured in Participants

Mortality rates in subjects after successful completion of a bone marrow transplant (NCT00741455)
Timeframe: Up to 15 Years Post-Transplant

InterventionParticipants (Count of Participants)
Survival < 1 year post-transplantSurvival 1 to < 5 years post-transplantSurvival 5 to < 10 years post-transplantSurvival 10+ years post-transplant
Study Treatment2807

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To Describe the Incidence of Grade 3-4 Organ Toxicity

(NCT00744692)
Timeframe: 2 years post transplant

Interventionparticipants (Number)
RIC Cord Blood Transplant0

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To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant

(NCT00744692)
Timeframe: 2 years post transplant

Interventionparticipants (Number)
RIC Cord Blood Transplant0

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To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure

(NCT00744692)
Timeframe: at least 2 years post transplant

Interventionpercentage of patients (Number)
RIC Cord Blood Transplant0

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To Evaluate the Pace of Immune Reconstitution.

Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request. (NCT00744692)
Timeframe: 1 year post transplant

Interventioncells/uL (Median)
RIC Cord Blood Transplant805

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To Describe the Pace of Neutrophil Recovery

Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions (NCT00744692)
Timeframe: 42 days post transplant

Interventiondays (Median)
RIC Cord Blood Transplant20

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Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.

Determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor cells at 180 days) following reduced intensity conditioning regimens in children < 21 years receiving cord blood transplant for non-malignant disorders. (NCT00744692)
Timeframe: 180 days post transplant

Intervention% of participants (Number)
RIC Cord Blood Transplant88

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To Describe the Pace of Platelet Recovery

Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions (NCT00744692)
Timeframe: 180 days post transplant

Interventiondays (Median)
RIC Cord Blood Transplant48

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To Determine the Overall Survival at day180 Post-transplant

To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis (NCT00744692)
Timeframe: 180 days

Interventionpercentage of participants (Number)
RIC Cord Blood Transplant81.8

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To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)

To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis (NCT00744692)
Timeframe: 100 days post transplant

Interventionpercentage of participants (Number)
RIC Cord Blood Transplant22.7

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Number of Participants With Complete Remission (CR)

Complete Response defined by NCI Working Group / International Working Group for CLL criteria as no evidence of disease on physical examination (no adenopathy or organomegaly) or microscopic examination of blood (ALC <4,000/L) and bone marrow (<30% lymphocytes, no lymphoid nodules), and recovery of hemoglobin, neutrophil, and platelet counts. (NCT00759798)
Timeframe: After 6 months

InterventionParticipants (Count of Participants)
Fludarabine, Cyclophosphamide, Rituximab185

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Number of Patients Who Achieved Donor Cell Engraftment

(NCT00775931)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Marrow Graft Transplant Conditioning5
Cord Blood Transplant Conditioning0

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Incidence of Grade II - IV Acute Graft-versus-host Disease

(NCT00775931)
Timeframe: by Day 100 after transplant

InterventionParticipants (Count of Participants)
Marrow Graft Transplant Conditioning2
Cord Blood Transplant Conditioning0

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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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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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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 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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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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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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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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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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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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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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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 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. (NCT00787761)
Timeframe: Day 30

Interventionparticipants (Number)
Transplant Recipients12

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T-cell and Myeloid Chimerism at Days 90 Post-transplantation (>90% Chimerism)

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. (NCT00787761)
Timeframe: Day 90

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan17

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Disease-free Survival (DFS) at 24 Months

Disease Free survival is measured by the amount of time a patient spends in a disease free state after being transplanted. (NCT00787761)
Timeframe: 24 months

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan13

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Number of Patients Experiencing Extensive Chronic Graft Versus Host Disease (GVHD)

Patients who had post-transplant complication (GVHD) as seen by clinical evidence including but not limited to skin rash, elevated liver function tests, nausea/vomiting/diarrhea. (NCT00787761)
Timeframe: 2 years

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan13

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Non-relapse Mortality (NRM) at Day 180 Post-transplantation

non-relapse mortality refers to the death of a patient for causes other than relapsed disease. (NCT00787761)
Timeframe: Day 180

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan0

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Number of Patients Who Experience Severe (Grade 3 or 4) Acute Graft-versus-host Disease

number of patients who experienced post-transplant complication (GVHD) as seen by clinical evidence including but not limited to skin rash, elevated liver function tests, nausea/vomiting/diarrhea. (NCT00787761)
Timeframe: Day 100

Interventionparticipants (Number)
Severe Graft Versus Host Disease2

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Overall Survival (OS) at 24 Months

Overall survival refers to the length of time a patient is alive after transplant regardless of whether they have progressive or relapsed disease. (NCT00787761)
Timeframe: 24 months

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan16

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T-cell and Myeloid Chimerism at Days 180 Post-transplantation (>90%)

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. (NCT00787761)
Timeframe: 180 days

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan19

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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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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 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 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 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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Complete Remission (CR) Rate of FCR3 in Treatment-naïve Participants With Chronic Lymphocytic Leukemia (CLL) at 6 Months

CR Rate is defined as number of all treated participants with CR as defined by 2008 IWCLL update of NCI-WG response criteria: Complete remission (CR), requiring absence of peripheral blood clonal lymphocytes by immunophenotyping, absence of lymphadenopathy, absence of hepatomegaly or splenomegaly, absence of constitutional symptoms and satisfactory blood counts; Complete remission with incomplete marrow recovery (CRi), defined as CR above, but without normal blood counts; Partial remission (PR), defined as ≥ 50% fall in lymphocyte count, ≥ 50% reduction in lymphadenopathy or ≥ 50% reduction in liver or spleen, together with improvement in peripheral blood counts; Progressive disease (PD): ≥ 50% rise in lymphocyte count to > 5 x109/L, ≥ 50% increase in lymphadenopathy, ≥ 50% increase in liver or spleen size, Richter's transformation, or new cytopenias due to CLL; Stable disease, defined as not meeting criteria for CR, CRi, PR or PD. (NCT00794820)
Timeframe: 6 months

InterventionPercentage of Participants (Number)
FCR-Multiple Dose Rituximab75

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Overall Survival (OS) Rate

OS was defined as the time from the initiation of treatment to last follow-up date or death. OS were calculated using Kaplan-Meier estimates. (NCT00794820)
Timeframe: 6 months to disease progression, period covered up to 12 years following treatment; Data cutoff for analysis was October 2014.

InterventionPercentage of Participants (Number)
FCR-Multiple Dose Rituximab58

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Remission Duration/Time to Progression (TTP)

TTP was defined as time from initiation of treatment to primary refractory disease or CLL progression. TTP was censored for therapy-related myelodysplastic syndrome (t-MDS) or acute myelogenous leukemia (t-AML) and death in remission. TTP calculated using Kaplan-Meier estimates. (NCT00794820)
Timeframe: 6 months to disease progression, period covered up to 12 years following treatment; Data cutoff for analysis was October 2014.

InterventionMonths (Median)
FCR-Multiple Dose Rituximab81

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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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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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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 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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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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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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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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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 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 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 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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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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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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2-year Progression-Free Survival

Progression-free survival (PFS) is defined as the interval between day of transplant and day of death or disease progression. (NCT00800839)
Timeframe: First 25-35 days post transplant and then every 3 months for a maximum of 2 years

Interventionpercentage of participants (Number)
Busulfan + Fludarabine + Cyclophosphamide26

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Cumulative Incidence of Grade II to IV Acute GVHD

Graft Versus Host Disease (GVHD) defined as grade 2 to 4 GVHD within first 100 days post transplant. Death or disease progression before diagnosis of GVHD were considered competing risks in the estimation of the incidence of acute GVHD. (NCT00800839)
Timeframe: 100 days post transplant

Interventionpercentage of incidence (Number)
Busulfan + Fludarabine + Cyclophosphamide53

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Rate of Engraftment

Engraftment defined as the evidence of donor derived cells (more than 5%) by bone marrow chimerism studies in the presence of neutrophil recovery by day 28 post stem cell (SC) infusion. Engraftment date is the first day of three (3) consecutive days that the ANC exceeds 0.5 x109/L. Delayed engraftment is defined as the evidence of engraftment beyond day 28 post SC infusion achieved after the administration of therapeutic (high dose) hematopoietic growth factors. (NCT00800839)
Timeframe: From engraftment to 60 days post transplant

Interventiondays (Median)
Busulfan + Fludarabine + Cyclophosphamide18

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Cumulative Incidence of Grade III to IV Acute GVHD

Graft Versus Host Disease (GVHD) defined as grade 3 to 4 GVHD within first 100 days post transplant. Death or disease progression before diagnosis of GVHD were considered competing risks in the estimation of the incidence of acute GVHD. (NCT00800839)
Timeframe: 100 days post transplant

Interventionpercentage of incidence (Number)
Busulfan + Fludarabine + Cyclophosphamide22

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2-year Overall Survival

Overall Survival (OS) is defined as the interval between day of transplant and day of death. (NCT00800839)
Timeframe: First 25-35 days post transplant and then every 3 months for a maximum of 2 years

Interventionpercentage of participants (Number)
Busulfan + Fludarabine + Cyclophosphamide33

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Time to Neutrophil Engraftment

Following MAC AutoSCT, the median time to neutrophil (PMN) recovery will be measured. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

Interventiondays (Mean)
Arm A - Family Donor16
Arm B - Unrelated Cord Blood or Adult24

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Time to Platelet Engraftment

Following MAC AutoSCT, the median time to platelet recovery will be measured. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

Interventiondays (Mean)
Arm A - Family Donor22
Arm B - Unrelated Cord Blood or Adult53

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Total Number of Subjects With a Disease Relapse or Progression Following MAC AutoSCT

Includes subjects with any measurable growth of disease in a previously affected site or detection of disease in a new site confirmed by biopsy. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

InterventionParticipants (Count of Participants)
Arm A - Family Donor1
Arm B - Unrelated Cord Blood or Adult3

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Total Number of Subjects With a Complete Response (CR) Following Myeloablative Conditioning (MAC) and Autologous Stem Cell Transplantation (AutoSCT)

Complete Response is defined as the complete resolution of B symptoms (i.e., weight loss, night sweats and fever) and normalization of all sites of disease on the basis of physical exam, bone marrow biopsy, and imaging studies. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

InterventionParticipants (Count of Participants)
Arm A - Family Donor4
Arm B - Unrelated Cord Blood or Adult12

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Total Number of Subjects With Grade II-IV Acute Graft-versus-Host-Disease (GVHD)

The criteria for grading is based on extent of organ involvement (i.e., Skin, Liver and Gut - rash on >50% of skin, bilirubin 2-3 mg/dl, diarrhea > 500 ml/day) with Grade II being better outcome and Grade IV being worse outcome. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

Interventionparticipants (Number)
Arm A - Family Donor0
Arm B - Unrelated Cord Blood or Adult7

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Total Number of Subjects With Partial Response or Stable Disease Following MAC AutoSCT

Total includes subjects with partial response and patients with stable disease, defined as <50% reduction in measurable disease or the uninterrupted persistence of B symptoms. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

InterventionParticipants (Count of Participants)
Arm A - Family Donor4
Arm B - Unrelated Cord Blood or Adult9

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To Determine the Optimal Regimen of Post-graft Immunosuppression With High-dose Cy Following Fludarabine, Busulfan, and Transplantation of Fully HLA-matched Bone Marrow That Leads to an Acceptable Incidence of Grades III/IV Acute GVHD

Percentage of participants with grade III-IV acute graft versus host disease (GVHD). GVHD is graded on a combination of skin symptoms (rash), gut symptoms (diarrhea), and liver symptoms (using a lab test called bilirubin). Grades range from I to IV, where I is the least severe and IV is the most severe. (NCT00809276)
Timeframe: 1 year

Interventionpercentage of participants (Number)
BuFlu Transplant15

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Number of Participants With Molecular Response

Molecular Remission defined as two consecutive bone marrow samples done one month apart with negative PCR( polymerase chain reaction) tor CML. (NCT00813124)
Timeframe: 12 month post BMT

InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseProgressive Disease
Azacytidine Maintenance After Allotx804

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Overall Survival at 1 Year

Evaluation of overall survival at 1 year (# of patients who are alive at 1 year post-transplant) (NCT00818961)
Timeframe: 1 year

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation26

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Number of Patients Experiencing Grade 2-4 Acute Graft-versus-host Disease Post-transplant

patients experiencing acute graft versus host disease post-transplant (NCT00818961)
Timeframe: patients were followed for 2 years

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation15

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Platelet Engraftment

The number of patients experiencing platelet engraftment post-transplant (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation35

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Number of Patients Experiencing Chronic Graft Versus Host Disease

(NCT00818961)
Timeframe: >100 days post-transplant

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation20

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Non-relapse Mortality at Day 100

patients are evaluable for their cause of death at Day 100 (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation1

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Complete Donor Chimerism

Complete donor chimerism (defined as >/= 95% donor cells in peripheral blood CD3+ and CD33+ was measured. (NCT00818961)
Timeframe: 2 years

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation26

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Non-relapse Mortality at 1 Year Post-transplant

Number of patients who died of non-relapse causes at one year. this is in clusive of all patients who were transplanted on study even though only 10 patients died at by 1 year time point. This outcome will be referenced in the donor chimerism outcome. Only 26/36 patients were eligible for this time point as that is all that were alive. (NCT00818961)
Timeframe: 1 year

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation4

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Neutrophil Recovery

The number of patients experiencing neutrophil recovery post transplant (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation35

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Number of Patients Requiring the Use of Donor Leukocyte Infusion (DLI) for Early Mixed T-cell Chimerism

DLI is used for patients with mixed chimerism following transplant (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation19

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Number of Patients Experiencing Veno-occlusive Disease (VOD) Post-transplant

Patients will be evaluated up to 4 years post transplant (NCT00818961)
Timeframe: 4 years

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation0

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Survival at Day 100

Survival at Day 100 (NCT00818961)
Timeframe: 100 day

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation35

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Engraftment Response Rate: Number of Transplanted Participants With Complete Chimerism at Day 30

Number of participants with complete chimerism at day 30 where defined as: Engraftment: first day of three (3) consecutive days that Absolute neutrophil count (ANC) exceeds 0.5 X 109/L. Subsequent chimerism studies must demonstrate the presence of donor derived cells. Graft Failure: failure to achieve an ANC >0.5 X 109/L for 3 consecutive days within 28 days after transplantation or a decline of ANC <0.5 x 109/L for three consecutive days after initial documented engraftment unless this is correlated with progression / recurrence of the underlying malignancy. (NCT00822770)
Timeframe: 30 Days post engraftment

InterventionParticipants (Count of Participants)
Overall Study: Plerixafor + G-CSF32

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Number of Participants Alive With no Disease Progression at Time of Allo Transplant

In Phase II portion of study, number of participants with treatment failure defined as either disease recurrence or death, measured from start of treatment to allo transplant at Day 0. (NCT00822770)
Timeframe: Baseline till transplant, Day -9 to Day 0, to 10 days

Interventionpercentage of participants (Number)
Participants inParticipants not in Complete Remission
Overall Study: Plerixafor + G-CSF1616

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Number of Participants With Grade 4 Dose Limiting Toxicity to Determine Maximum Tolerated Dose (MTD) Plerixafor

Phase I determination of MTD dose of Plerixafor in combination with a fixed dose of Filgrastim where dose limiting toxicity defined as any grade 4 non-hematologic toxicity observed within 28 days from Day 0 (day of transplant). (NCT00822770)
Timeframe: 28 day cycle (Plerixafor Day -7 to Day -4)

Interventionparticipants (Number)
0 mcg/kg daily80 mcg/kg daily160 mcg/kg daily240 mcg/kg daily
Phase I Plerixafor + G-CSF1474

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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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Overall Response Rate

The proportion of subjects who achieve CR, CRu, or partial response (PR) relative to the response evaluable population. Disease response and progression were evaluated according to the modified IWRC criteria by radiographic imaging and other procedures as necessary. (NCT00850499)
Timeframe: Up to 8 cycles (1 cycle is 35 days: 280 days)

Interventionparticipants (Number)
Velcade + Fludarabine3
Rituximab + Fludarabine6

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Complete Response Rate

The proportion of response-evaluable subjects who achieved a confirmed complete response (CR) or complete response unconfirmed (CRu). Disease response and progression were evaluated according to modified International Workshop Response Criteria (IWRC) criteria by radiographic imaging and other procedures as necessary. (NCT00850499)
Timeframe: Up to 8 cycles (1 cycle is 35 days: 280 days)

Interventionparticipants (Number)
Velcade + Fludarabine2
Rituximab + Fludarabine3

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Rate of Complete Donor Chimerism - Blood

"Rate of Complete Donor Chimerism - Blood~Summarized using standard descriptive statistics." (NCT00856388)
Timeframe: Day 30

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)89

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Median Time to Platelet Engraftment

"Median Time to Platelet Engraftment~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Day 100

InterventionDays (Median)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)17.0

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1 yr Extenstive Chronic GVHD

"1 yr Extensive Chronic GVHD~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Up to 4.5 years

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)60

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3 yr Overall Survival

3 yr Overall Survival estimated using the Kaplan-Meier method. (NCT00856388)
Timeframe: Up to 4.5 years

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)46.0

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Acute GVHD Grade III-IV

"Acute GVHD grade III-IV~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Up to day 100

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)27

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Day 100 TRM

Day 100 Treatment Related Mortality An exact 95% confidence interval will be provided. (NCT00856388)
Timeframe: First 100 days

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)8.44

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Median Time to ANC Engraftment

"Median Time to ANC Engraftment~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Days 30

InterventionDays (Median)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)16.5

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Rate of Complete Donor Chimerism - Blood

"Rate of Complete Donor Chimerism - Blood~Summarized using standard descriptive statistics." (NCT00856388)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)94

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Complete Response Rate

Response assessments were made per the NCI working group criteria for CLL (Hallek et al, Blood, 2008). Complete response rate is defined as an achievement of all of the following: Peripheral blood lymphocytes (evaluated by blood and differential count) below 4 × 109/L (4000/μL), absence of significant lymphadenopathy (lymph nodes must be < 1.5 cm), absence of splenomegaly and hepatomegaly, absence of constitutional symptoms, normal blood counts, and bone marrow sample must be at least normocellular for age, with less than 30% of nucleated cells being lymphocytes. Lymphoid nodules should be absent. (NCT00860457)
Timeframe: 3 years

Interventionpercentage of patients (Number)
Chemotherapy36

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Incidence of Chronic GVHD

(NCT00860574)
Timeframe: at 6 months

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)20

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Incidence of Grades II-IV Acute GVHD

(NCT00860574)
Timeframe: at 6 months

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)57

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Median Donor CD3 + T Lymphocyte Chimerism in Peripheral Blood

Donor chimerism was evaluated in peripheral blood T cells (NCT00860574)
Timeframe: Day 28 after HCT

Interventionpercentage of T cells (Median)
Treatment (Allogeneic Transplantation)100

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Non Relapse Mortality (NRM) Incidence

Cumulative incidence of NRM at 6 months. NRM includes all deaths without relapse or disease progression. (NCT00860574)
Timeframe: At 6 months

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)6

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Non Relapse Mortality Incidence

(NCT00860574)
Timeframe: 1 year after HCT

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)6

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Relapse-free Survival

(NCT00860574)
Timeframe: at 2 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)62

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Overall Survival (OS)

(NCT00860574)
Timeframe: at 2 years

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)71

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Relapse Incidence

(NCT00860574)
Timeframe: At 6 months

InterventionParticipants (Count of Participants)
Treatment (Allogeneic Transplantation)18

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Number of Patients With Successful Natural Killer Expansion

Successful in vivo donor NK cell expansion will be defined as an absolute circulating donor-derived NK cell count of >100 cells/μl. (NCT00871689)
Timeframe: Day 72 Post Transplant

Interventionparticipants (Number)
Patients Receiving Double Umbilical Cord Blood Transplant1

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Number of Patients With Grade III-IV Acute Graft-Versus-Host (GVHD) Disease

"Number of patients with Grade III-IV GVHD. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body.~Acute GVHD usually happens within the first 3 months after transplant." (NCT00871689)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Patients Receiving Double Umbilical Cord Blood Transplant0

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Number of Patients With Neutrophil Engraftment

Number of patient with absolute neutrophils >500*10^8/kg by 42 days post transplant. (NCT00871689)
Timeframe: Day 42

InterventionParticipants (Number)
Patients Receiving Double Umbilical Cord Blood Transplant1

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Incidence of Primary Graft Failure

Incidence of graft failure defined as an absolute neutrophil count of less than 500/uL and a bone marrow that is less than 5% cellular (marrow aplasia) on day 42. (NCT00871689)
Timeframe: Day 42

InterventionParticipants (Number)
Patients Receiving Double Umbilical Cord Blood Transplant1

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Median Overall Survival

Average number of days the patients were alive after receiving UCB transplantation. (NCT00871689)
Timeframe: Month 6

InterventionDays (Median)
Patients Receiving Double Umbilical Cord Blood Transplant98.5

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Number of Patients With Acute Graft-Versus-Host (GVHD) Disease

"Number of patients with any grade of GVHD. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body.~Acute GVHD usually happens within the first 3 months after transplant." (NCT00871689)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Patients Receiving Double Umbilical Cord Blood Transplant1

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Number of Patients With Complete Remission of Disease

Disease response will be measured by rate of leukemic clearance (clearance of blasts in blood at timepoint 0) and complete remission (less than 5% blasts and recovery of hematopoiesis). (NCT00871689)
Timeframe: Day 100

InterventionParticipants (Number)
Patients Receiving Double Umbilical Cord Blood Transplant1

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Maximum Change From Baseline in the EORTC QLQ-C30 Financial Problems Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Financial Problems Domain Scale was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-10.9
Investigators Choice-2.3

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Maximum Change From Baseline in the EORTC QLQ-C30 Fatigue Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-4.9
Investigators Choice-2.9

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Kaplan Meier Estimate of Time to Treatment Failure (TTF) as Assessed by the Investigator

Time to treatment failure was defined as the time from the first dose of study drug to discontinuation of treatment for any reason, including disease progression assessed by the investigator, treatment toxicity, or death. Participants who were on-treatment or completed the treatment according to the protocol were censored at the last date of drug intake. (NCT00875667)
Timeframe: From the date of the first treatment to the data cut-off date of 07 March 2014; median treatment duration was 24.3 weeks for the lenalidomide arm and 13.1 weeks for the investigators choice arm

Interventionweeks (Median)
Lenalidomide24.4
Investigators Choice17.9

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Kaplan Meier Estimate of Time to Treatment Failure as Assessed by the Investigator at the Final Analysis

Time to treatment failure was defined as the time from the first dose of study drug to discontinuation of treatment for any reason, including disease progression assessed by the investigator, treatment toxicity, or death. Participants who were on-treatment or completed the treatment according to the protocol were censored at the last date of drug intake. (NCT00875667)
Timeframe: From date of first dose of treatment to the study discontinuation date of 09 October 2018; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm

Interventionweeks (Median)
Lenalidomide24.4
Investigators Choice17.9

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Mean Change From Baseline in the EORTC QLQ-C30 Constipation

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Constipation Domain was scored between 0 and 100, with a high score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice8.6-0.81.30.00.00.8
Lenalidomide12.56.34.23.5-0.710.2

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Mean Change From Baseline in the EORTC QLQ-C30 Diarhoea

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Diarhoea Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice12.6-3.11.3-4.20.00.0
Lenalidomide15.7-3.5-4.22.4-2.11.6

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Mean Change From Baseline in the EORTC QLQ-C30 Dyspnoea Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnoea Domain was scored between 0 and 100, with a high score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice21.2-0.80.04.25.60.8
Lenalidomide26.5-1.6-1.4-2.91.46.0

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Mean Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Domain ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice78.51.31.3-3.11.4-1.5
Lenalidomide73.73.43.68.14.5-1.3

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Mean Change From Baseline in the EORTC QLQ-C30 Fatigue Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice39.22.13.4-6.9-7.42.6
Lenalidomide40.20.1-3.2-1.0-3.95.2

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Mean Change From Baseline in the EORTC QLQ-C30 Financial Problems Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Financial Problems Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice10.8-0.8-3.8-4.2-5.61.6
Lenalidomide19.5-7.0-7.0-2.9-9.2-4.3

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Mean Change From Baseline in the EORTC QLQ-C30 Global Health Status / QoL Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status / QoL Domain was scored between 0 and 100, with a higher score representing a higher quality of life. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice58.42.33.27.38.3-1.0
Lenalidomide59.0-3.4-0.71.04.3-5.8

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Mean Change From Baseline in the EORTC QLQ-C30 Insomnia Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Insomnia Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice25.7-4.7-6.4-16.7-16.70.8
Lenalidomide29.4-7.6-5.2-1.8-7.1-3.2

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Mean Change From Baseline in the EORTC QLQ-C30 Nausea / Vomiting Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Nausea and Vomiting Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice3.80.45.82.12.86.6
Lenalidomide4.92.52.65.3-0.70.5

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Mean Change From Baseline in the EORTC QLQ-C30 Pain Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Pain Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineCycle 3 Day 1Cycle 5 Day 1Cycle 7 Day 1Cycle 9 Day 1Treatment Discontinuation
Investigators Choice13.7-1.2-2.60.0-2.83.5
Lenalidomide22.6-2.2-0.23.2-3.24.6

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Maximum Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide6.9
Investigators Choice3.7

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Mean Change From Baseline in the EORTC QLQ-C30 Role Functioning Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Role Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice73.93.5-6.40.013.9-4.3
Lenalidomide71.5-4.81.40.31.8-9.1

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Mean Change From Baseline in the EORTC QLQ-C30 Social Functioning Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Social Functioning Domain ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice78.4-1.2-4.52.10.0-2.7
Lenalidomide74.9-1.01.6-1.54.3-5.1

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Number of Participants With Treatment Emergent Adverse Events

Adverse events were assessed using National Cancer Institute, Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 3: according to the following scale: Grade 1 = Mild Adverse Event (AE), Grade 2 = Moderate AE, Grade 3 = Severe and Undesirable AE, Grade 4 = Life-threatening or Disabling AE, and Grade 5 = Death; Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. after the first dose of study drug and within 28 days after the last dose. A Treatment Emergent Adverse event (TEAE) is defined as any AE occurring or worsening on or after the first dose of study drug and within 28 days after the last dose of study drug. (NCT00875667)
Timeframe: From the date of the first dose of study drug to 28 days after the last dose, up to the study discontinuation date of 09 October 2018; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigators choice arm

,
InterventionParticipants (Count of Participants)
Any TEAEAny TEAE Grade 3 AEAny TEAE Grade 4 AEAny TEAE Grade 5 AEAny TEAE Related to the IPAny Grade 3 AE Related to IPAny Grade 4 AE Related to IPAny Grade 5 AE Related to IPAny Serious Adverse Event (SAE)Any SAE Related to IPAny TEAE Leading to Stopping of IPAny Treatment Related AE Leading to Stopping IPTEAE Leading to Dose Reduction/InterruptionRelated AE Leading to Dose Reduct/InterruptionTEAE Leading to Dose ReductionRelated AE Leading to Dose ReductionTEAE Leading to Dose InterruptionRelated AE Leading to Dose Interruption
Investigators Choice694929251361902212147332913102825
Lenalidomide159126561514110646075383118114103726911098

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Maximum Change From Baseline in the EORTC QLQ-C30 Appetite Loss Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Appetite Loss Domain to Treatment Scale was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-4.8
Investigators Choice-4.1

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Maximum Change From Baseline in the EORTC QLQ-C30 Dyspnoea Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnoea Domain to Treatment Scale was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-7.3
Investigators Choice-5.8

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Maximum Change From Baseline in the EORTC QLQ-C30 Diarhoea Domain to Treatment Discontinuation Visit

"The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact).~The EORTC QLQ-C30 Diarhoea Scale was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement." (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and time of discontinuation from treatment visit.Up to final data cut-0ff date of 07 March 2014

Interventionunits on a scale (Least Squares Mean)
Lenalidomide-7.2
Investigators Choice-5.8

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Maximum Change From Baseline in the EORTC QLQ-C30 Cognitive Functioning Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Cognitive Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide3.2
Investigators Choice2.9

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Maximum Change From Baseline in the EORTC QLQ-C30 Pain Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Pain Scale was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and time of discontinuation from treatment visit.Up to final data cut-0ff date of 07 March 2014

Interventionunits on a scale (Mean)
Lenalidomide-5.8
Investigators Choice-3.5

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Maximum Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide3.4
Investigators Choice-1.8

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Maximum Change From Baseline in the EORTC QLQ-C30 Constipation Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Constipation Scale was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-0.3
Investigators Choice-3.5

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Kaplan Meier Estimate of Time to Progression According to the IRC Central Review

Time to progression (TTP) was defined as the time from randomization until objective tumor progression. Time to progression did not include deaths. Participants without progression at the time of analysis were censored at the last assessment date that the participant was known to be progression-free. Participants who received a new anti-lymphoma treatment without documented progression were censored at the last assessment date that the participant was known to be progression-free. (NCT00875667)
Timeframe: From date of randomization to the data cut-off date of 07 March 2014; median study duration was 70.7 weeks for the lenalidomide arm and 69.3 weeks for the investigators choice arm

InterventionWeeks (Median)
Lenalidomide39.3
Investigators Choice24.7

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Kaplan Meier Estimate of Time to First Response as Assessed by the Investigator at the Final Analysis

Time to first response was defined as the time from first dose of study drug to the date of the first response (having at least a PR). Participants with progression at the time of analysis were censored at the first assessment date that the participant was known to have progressed. Participants with SD at the time of analysis were censored at the last assessment date that the subject was known to be progression-free. (NCT00875667)
Timeframe: From date of randomization to the study discontinuation date of 09 October 2018; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm

InterventionWeeks (Median)
Lenalidomide23.9
Investigators Choice40.0

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Kaplan Meier Estimate of Time to First Response (TTFR) According to the IRC Central Review

Time to Response was defined as the time from first dose of study drug to the date of the first response (having at least a PR) and was calculated only for responding participants. ). Participants with progression at the time of analysis were censored at the first assessment date that the participant was known to have progressed. Participants with SD at the time of analysis were censored at the last assessment date that the participant was known to be progression-free. (NCT00875667)
Timeframe: From randomization of study drug to time of first documented PR or better response; up to data cut-off date of 07 March 2014; median treatment duration was 24.3 weeks for the lenalidomide arm and 13.1 weeks for the investigators choice arm

InterventionWeeks (Median)
Lenalidomide18.7
Investigators ChoiceNA

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Kaplan Meier Estimate for Progression Free Survival by Investigator's Assessment at the Final Analysis

Kaplan Meier estimates of PFS were defined as the time from randomization to the first observation of disease progression or death due to any cause, whichever was first. If a participant had not progressed or died, PFS was censored at the time of last completed assessment when the participant was known not to have progressed. For participants who received other anti-lymphoma therapy with no evidence of progression, PFS was censored at time of last tumor assessment with no evidence of progression prior to the start of new anti-lymphoma treatment. (NCT00875667)
Timeframe: From randomization to progression of disease or death; up to study discontinuation of 09 October 2018; overall median follow-up time was 285 weeks

Interventionweeks (Median)
Lenalidomide37.3
Investigators Choice23.6

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Kaplan Meier Estimate for Progression Free Survival (PFS) by Independent Review Committee (IRC) Central Review

PFS was defined as time of randomization to the first observation of disease progression or death due to any cause, whichever was first. If a participant had not progressed or died, PFS was censored at the time of last assessment when the participant was known not to have progressed. For participants who received other anti-lymphoma therapy with no evidence of progression, PFS was censored at time of last tumor assessment with no evidence of progression prior to the start of new anti-lymphoma treatment. (NCT00875667)
Timeframe: From randomization to progression of disease or death; up to data cut off date of 07 March 2014; overall median follow-up time was 93.9 weeks

Interventionweeks (Median)
Lenalidomide37.6
Investigators Choice22.7

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Kaplan Meier Estimate for Overall Survival as Assessed by the Investigator at the Final Analysis

Overall survival was defined as the time from randomization until death from any cause. Participants alive or lost to follow-up at the time of analysis were censored at the last date they were known to be alive. (NCT00875667)
Timeframe: From randomization to progression of disease or death; up to the study discontinuation date of 09 October 2018; overall median follow-up time was 285 weeks

Interventionweeks (Median)
Lenalidomide120.6
Investigators Choice91.7

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Kaplan Meier Estimate for Overall Survival (OS) According to the IRC Central Review

Overall survival was defined as the time from randomization until death from any cause. Participants alive or lost to follow-up at the time of analysis were censored at the last date they were known to be alive. (NCT00875667)
Timeframe: From date of randomization to the data cut-off date of 07 March 2014; overall median follow-up was 93.9 weeks

Interventionweeks (Median)
Lenalidomide121.0
Investigators Choice91.7

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Kaplan Meier Estimate for Duration of Response as Assessed by the Investigator at the Final Analysis

Duration of response was defined as the time from when the first response of CR, CRu, or PR was first achieved until documented tumor progression, or until the participant died from any cause, whichever occurred first. Participants who did not progress or die at the time of analysis were censored at the last assessment date that the participant was known to be progression-free. Participants who received a new treatment without documented progression were censored at the last assessment date that the participant was known to be progression-free. (NCT00875667)
Timeframe: From date of randomization to the study discontinuation date of 09 October 2018; median study duration was 103.9 weeks for lenalidomide and 87.0 weeks for the investigator choice arm

InterventionWeeks (Median)
Lenalidomide70.1
Investigators Choice91.7

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Kaplan Meier Estimate for Duration of Response (DOR) According to the IRC Central Review

Duration of response was defined as the time from when the first response of CR, CRu, or PR was first achieved until documented tumor progression, or until the participant died from any cause, whichever occurred first. Participants who did not progress or die at the time of analysis were censored at the last assessment date that the participant was known to be progression-free. Participants who received a new treatment without documented progression were censored at the last assessment date that the participant was known to be progression-free. (NCT00875667)
Timeframe: From date of randomization to the data cut-off date of 07 March 2014; median study duration was 70.7 weeks for the lenalidomide arm and 69.3 weeks for the investigators choice arm

InterventionWeeks (Median)
Lenalidomide69.6
Investigators Choice45.1

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Mean Change From Baseline in the EORTC QLQ-C30 Cognitive Functioning Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Cognitive Functioning Domain ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice83.6-2.31.34.25.6-2.3
Lenalidomide84.60.0-1.9-3.2-2.5-5.1

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Kaplan Meier Estimate of Time to Progression as Assessed by the Investigator at the Final Analysis

Time to progression (TTP) was defined as the time from randomization until objective tumor progression. Time to progression did not include deaths. Participants without progression at the time of analysis were censored at the last assessment date that the participant was known to be progression-free. Participants who received a new anti-lymphoma treatment without documented progression were censored at the last assessment date that the participant was known to be progression-free. (NCT00875667)
Timeframe: From date of randomization to the study discontinuation date of 09 October 2018; median study duration was 103.9 weeks for lenalidomide and 87.0 weeks for the investigator choice arm

InterventionWeeks (Median)
Lenalidomide39.3
Investigators Choice24.7

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Mean Change From Baseline in the EORTC QLQ-C30 Appetite Loss Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Appetite Loss Domain was scored between 0 and 100, with a higher score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice16.2-0.85.1-12.5-11.15.4
Lenalidomide18.12.51.9-2.3-4.34.8

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Percentage of Participants With a Complete Response, Unconfirmed Complete Response, Partial Response and Stable Disease at the Final Analysis

Tumor control rate was defined as the percentage of participants with a complete response (CR), unconfirmed complete response (CRu), partial response (PR) and stable disease (SD). Tumor Response was assessed by a modification of the International Lymphoma Workshop Response Criteria, IWRC, Cheson, 1999); CR is defined as the disappearance of all clinical and radiographic evidence of disease; CRu is defined as a CR, with a 1) residual lymph node mass >1.5 cm that has decreased by 75% in the sum of the product of the diameters (SPD). Individual nodes previously confluent decreased by more than 75% in the SPD compared with original mass; 2) indeterminate bone marrow; PR = is defined ≥50% decrease in 6 largest nodes or nodal masses. Stable disease (SD) is defined as less than a PR (see above) but is not progressive disease or relapsed disease. (NCT00875667)
Timeframe: From date of randomization to the discontinuation date of 09 October 2018; median treatment duration was 24.3 weeks for the lenalidomide arm and 13.1 weeks for the investigators choice arm

InterventionPercentage of participants (Number)
Lenalidomide70.0
Investigators Choice65.5

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Percentage of Participants With a Complete Response, Unconfirmed Complete Response, Partial Response and Stable Disease According to the IRC Central Review

Tumor control rate was defined as the percentage of participants with a complete response (CR), unconfirmed complete response (CRu), partial response (PR) and stable disease (SD). Tumor Response was assessed by a modification of the International Lymphoma Workshop Response Criteria, IWRC, Cheson, 1999); CR is defined as the disappearance of all clinical and radiographic evidence of disease; CRu is defined as a CR, with a 1) residual lymph node mass >1.5 cm that has decreased by 75% in the sum of the product of the diameters (SPD). Individual nodes previously confluent decreased by more than 75% in the SPD compared with original mass; 2) indeterminate bone marrow; PR = is defined ≥50% decrease in 6 largest nodes or nodal masses. Stable disease (SD) is defined as less than a PR (see above) but is not progressive disease or relapsed disease. (NCT00875667)
Timeframe: From date of randomization to the data cut-off date of 07 March 2014; median treatment duration was 24.3 weeks for the lenalidomide arm and 13.1 weeks for the investigators choice arm

InterventionPercentage of Participants (Number)
Lenalidomide69.4
Investigators Choice63.1

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Percentage of Participants Who Achieved an Overall Response as Assessed by the Investigator at the Final Analysis

Overall Response Rate (ORR) was defined as the percentage of participants whose best response was Complete Response, Complete Response unconfirmed or Partial Response. Participants who had discontinued before any response has been observed or changed to other anti-lymphoma treatments before response had been observed, were considered as non-responders. Tumor Response was assessed by a modification of the International Lymphoma Workshop Response Criteria, IWRC, Cheson, 1999; CR is defined as the disappearance of all clinical and radiographic evidence of disease; CRu is defined as a CR, with a 1) residual lymph node mass >1.5 cm that has decreased by 75% in the sum of the product of the diameters (SPD). Individual nodes previously confluent decreased by more than 75% in the SPD compared with original mass; 2) indeterminate bone marrow; PR = is defined ≥50% decrease in 6 largest nodes or nodal masses. (NCT00875667)
Timeframe: From date of randomization to the study discontinuation date of 09 October 2018; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm

InterventionPercentage of Participants (Number)
Lenalidomide45.9
Investigators Choice22.6

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Percentage of Participants Who Achieved an Overall Response According to the IRC Central Review

Overall Response Rate (ORR) was defined as the percentage of participants whose best response was Complete Response (CR), Complete Response unconfirmed (CRu) or Partial Response (PR). Participants who discontinued before any response had been observed or changed to other anti-lymphoma treatments before response had been observed, were considered as non-responders. Tumor Response was assessed by a modification of the International Lymphoma Workshop Response Criteria, IWRC, Cheson, 1999; CR is defined as the disappearance of all clinical and radiographic evidence of disease; CRu is defined as a CR, with a 1) residual lymph node mass >1.5 cm that has decreased by 75% in the sum of the product of the diameters (SPD). Individual nodes previously confluent decreased by more than 75% in the SPD compared with original mass; 2) indeterminate bone marrow; PR = is defined ≥50% decrease in 6 largest nodes or nodal masses. (NCT00875667)
Timeframe: From date of randomization to the data cut-off date of 07 March 2014; median treatment duration was 24.3 weeks for the lenalidomide arm and 13.1 weeks for the investigators choice arm

InterventionPercentage of Participants (Number)
Lenalidomide40.0
Investigators Choice10.7

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Maximum Change From Baseline in the EORTC QLQ-C30 Social Functioning Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Social Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide5.1
Investigators Choice3.8

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Maximum Change From Baseline in the EORTC QLQ-C30 Role Functioning Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Role Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide3.1
Investigators Choice5.0

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Maximum Change From Baseline in the EORTC QLQ-C30 Nausea and Vomiting Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Nausea and Vomiting Scale was scored between 0 and 100, with a high score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-2.3
Investigators Choice-0.6

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Maximum Change From Baseline in the EORTC QLQ-C30 Insomnia Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Insomnia Scale was scored between 0 and 100, with a high score representing worse symptomatic expression. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide-12.8
Investigators Choice-7.6

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Maximum Change From Baseline in the EORTC QLQ-C30 Global Health Status / QoL Domain to Treatment Discontinuation Visit

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status / QoL Domain to Treatment Scale was scored between 0 and 100, with a higher score representing a higher quality of life. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

Interventionunits on a scale (Mean)
Lenalidomide4.6
Investigators Choice5.6

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Mean Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

The EORTC-QLQ-C30 is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale ranges from 0 to 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00875667)
Timeframe: Baseline known as screening visit, after cycle 2 (C3D1), after Cycle 4 (C5D1), after Cycle 6, (C7D1), after Cycle 8, (C9D1) and at discontinuation; median treatment duration was 24.3 weeks for lenalidomide and 13.1 weeks for the investigator choice arm.

,
Interventionunits on a scale (Mean)
BaselineChange from Baseline to Cycle 3 Day 1Change from Baseline to Cycle 5 Day 1Change from Baseline to Cycle 7 Day 1Change from Baseline to Cycle 9 Day 1Change from Baseline to Treatment Discontinuation
Investigators Choice78.9-3.7-2.14.211.1-5.1
Lenalidomide71.8-0.51.62.42.8-5.6

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Percentage of Participants With GVHD (Graft Versus Host Disease)

Acute grade 2 to 4 Graft versus host disease( GVHD )for patients who were able to be analyzed by measuring the T cell counts for increased CD3+ before and after lenalidomide. (NCT00899431)
Timeframe: Up to 6 months after allotransplant

InterventionParticipants (Count of Participants)
Group 10
Group 23
Group 31

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Number of Participants Who Did Not Engraft After Receiving a CD45RA+ T Cell Depleted PBSC Transplant

Graft failure is defined as either a failure to reach an ANC of >500/uL for 3 consecutive days by day 28 post-transplant, or an irreversible decrease in ANC <100 after an established donor graft, unless there is a reasonable explanation such as a viral infection or drug effect that may be responsible for a reversible decrease in ANC. (NCT00914940)
Timeframe: Up to 5 years post transplant

InterventionParticipants (Count of Participants)
Overall Study Participants0

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Number of Participants Who Have Relapsed Within 5 Years of CD45RA+ T Cell Depleted PBSC Transplant

Relapse is defined by the presence of malignant cells in marrow, peripheral blood, or extramedullary sites by histopathology. Testing for recurrent malignancy in the blood and bone marrow performed by monitoring the CBC and bone marrow at Day 28, 58, 80, 360, and as needed for suspected relapse. (NCT00914940)
Timeframe: Up to 5 years post transplant

InterventionParticipants (Count of Participants)
Overall Study Participants10

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Number of Participants With Chronic GVHD

Chronic GVHD measured by meeting NIH criteria and treated with immune suppression (NCT00914940)
Timeframe: Up to 5 years post transplant

InterventionParticipants (Count of Participants)
Chronic GVHD that meets NIH criteriaNo documented chronic GVHD
Overall Study Participants335

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To Estimate the Rate of Conversion of Partial Response (PR) to Complete Response (CR) After Fludarabine, Cyclophosphamide and Rituximab (FCR) Plus Vorinostat

Responses were measured after completion of FCR+ vorinostat induction therapy (within 21 days of starting maintenance) and again after completion of maintenance therapy (24 months after the start of maintenance therapy). Maintenance therapy began within 3 months after completion of induction therapy with FCR+vorinostat. Criteria for response are specified by the National Cancer Institute (NCI) working group guidelines; in addition, patients were required to meet computed tomography (CT) criteria as described in the protocol. Response categories include Complete Response (CR), Partial Response (PR), Nodular Partial Response (nPR), Stable Disease (SD) or Progressive Disease (PD). (NCT00918723)
Timeframe: After completion of maintenance therapy (24 months after start of maintenance)

Interventionpercentage of participants (Number)
Conversion from PR to CRConversion from nPR (nodular PR) to CR
Treatment (Induction and Maintenance Chemotherapy)50100

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Maximum Tolerated Dose (MTD) of Vorinostat That Can be Combined With Fludarabine Phosphate, Cyclophosphamide and Rituximab (FCR) (Phase I)

"The MTD of vorinostat in combination with FCR will be defined as the dose level immediately below the dose level at which greater than or equal to 2 patients out of 6 of a cohort experience dose-limiting toxicity. Toxicities will be assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0.~Doses of vorinostat analyzed to reach the MTD were 200 mg, 300 mg and 400 mg." (NCT00918723)
Timeframe: 28 days

Interventionmg (Number)
MTD for Vorinostat During Induction Therapy400
MTD for Vorinostat During Maintenance Therapy400

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Overall Survival

Overall survival (OS): The percentage of people in a study who are still alive at for a certain period of time after they started treatment. (NCT00918723)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Treatment (Induction and Maintenance Chemotherapy)90

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Percentage of Patients With Progression-free Survival at 2 Years

"Progression-free survival (PFS): The length of time during and after the treatment that a patient lives with the disease but it does not get worse.~Progressive disease is specified by the NCI (National Cancer Institute) working group guidelines and additional CT (computerized tomography) scan requirements:~Lymphadenopathy, > 50% increase in the sum of the products of at least two lymph nodes on two consecutive determinations; one lymph node must be at least 2 cm.~An increase in the liver or spleen size by 50% or more by CT scan or the de novo appearance of hepatomegaly or splenomegaly.~An increase in the number of blood lymphocytes by 50% or more with at least 5000 B lymphocytes per microliter.~Transformation to a more aggressive histology or occurrence of cytopenia (neutropenia, anemia, or thrombocytopenia) attributable to CLL." (NCT00918723)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Treatment (Induction and Maintenance Chemotherapy)87

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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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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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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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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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Toxicity Profile

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT00923195)
Timeframe: 32 months

InterventionParticipants (Number)
TBI 600cGy + PBL + HD IL-2+gp100:154-1622
TBI 600cGy+PBL+HD IL-2+MART-1:26-35(27L)2

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Complete Response Rates for Patients With Metastatic Melanoma

Complete response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Complete response is a disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progression is at least a 20% increase in the sum of the LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as reference the smallest sum LD. (NCT00923195)
Timeframe: Every 4-6 weeks after initial treatment regimen. If the patient has stable disease or tumor shrinkage, complete evaluations will be repeated every 1-3 months.

,
InterventionParticipants (Number)
Complete ResponsePartial ResponseProgressive DiseaseStable Disease
TBI 600cGy + PBL + HD IL-2+gp100:154-1620020
TBI 600cGy+PBL+HD IL-2+MART-1:26-35(27L)0020

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Overall Survival

Overall survival is defined as date of on-study to date of death from any cause or last follow up. (NCT00923364)
Timeframe: 2 years

Interventionmonths (Median)
Recipients Only76

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Incidence of Acute and Chronic Graft-versus-host Disease (GVHD)

Acute GVHD is assessed according to the 1994 Consensus Conference Grading Criteria. Chronic GVHD is assessed by the 2005 Chronic GVHD Consensus Project. GVHD can affect performance status and attack multiple organ systems such as the skin, liver, gut, mouth, eyes, joints, lung, etc. that can lead to a rash, diarrhea, metabolic changes, infection and/or death. The clinical grading of acute GVHD is grade 0 (none) to 4 (severe). Chronic GVHD is a delayed form of GVHD that may occur after day 100 post transplant. (NCT00923364)
Timeframe: 2 years

Interventionparticipants (Number)
AcuteChronic
Recipients Only23

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Number of Participants With Serious and Non-serious Adverse Events

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events v3.0. A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT00923364)
Timeframe: Date treatment consent signed to date off study, approximately, 91 months

InterventionParticipants (Count of Participants)
Recipients and Healthy Related Donors16

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Number of Participants With Disease Free Survival

Number of participants with documented evidence of disease progression following start of treatment. (NCT00923364)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Recipients Only8

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Days to Platelet Engraftment

Platelet engraftment is defined as a platelet count of >20 x 10(9) cells/L for 7 consecutive days without requiring a platelet transfusion. (NCT00923364)
Timeframe: 30 days

InterventionDays (Median)
Recipients Only30

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Days to Neutrophil Engraftment

Neutrophil engraftment is defined as a neutrophil count of >0.5 x 10(9) cells/L for 3 consecutive days. (NCT00923364)
Timeframe: 30 days

InterventionDays (Median)
Recipients Only12

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Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2)

Engraftment of donor cells was assessed using polymorphisms in regions known to contain short tandem repeats. Peripheral blood cluster of differentiation 14 (CD14+), cluster of differentiation 3 (CD3-)/cluster of differentiation 56 (CD56+), cluster of differentiation 19 (CD19+), and CD3+ subsets were isolated by flow cytometry and chimerism was assessed. (NCT00923364)
Timeframe: 2 years

Interventionpercentage of donor cells (Mean)
Donor CD14+ cells, %Donor CD19+ cells, %Donor CD3-/CD56+ cells, %Donor CD3+ cells, %
Recipients Only99.810099.897.6

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Number of Participiants With In-vivo Survival of Infused Cells

In-vivo survival of infused cells is determined by analysis of the sequence of the variable region of the T cell receptor or flow cytometry (FACS). (NCT00924001)
Timeframe: 44 days

InterventionParticipants (Number)
Metastatic Melanoma1

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Number of Participants With an Objective Clinical Tumor Regression Response According to RECIST Criteria

Response is determined by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is the disappearance of all target lesions, partial response (PR) is at least a 30% decrease in the target lesions, progression (PD) is at least a 20% increase in the target lesions or appearance of one or more new lesions, and stable disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. (NCT00924001)
Timeframe: 44 days

InterventionParticipants (Number)
Metastatic Melanoma0

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Number of Participants With Adverse Events

Here are the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00924001)
Timeframe: 44 days

InterventionParticipants (Number)
Metastatic Melanoma1

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Area Under the Plasma Concentration (AUC) - LMB2

AUC is a measure of the plasma concentration of drug over time. It is used to characterize drug absorption. Plasma levels were analyzed by cytotoxicity assay. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Interventionµg/-min/mL (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC161
All Other Patients144

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Duration of Response (Complete Response + Partial Response)

Duration of response is defined as a response lasting for at least 4 weeks but >8 weeks and is assessed by the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma. Complete remission (CR) is complete disappearance of all detectable clinical and radiographic evidence of disease and disappearance of all disease related symptoms if present before therapy and normalization of those biochemical abnormalities definitely assignable to the lymphoma. Partial response is reduction by ≥50% of leukemia cell count or ≥50% reduction in the size of all measurable lesions, and no increase in size of any measurable or evaluable lesion or appearance of new lesion. (NCT00924170)
Timeframe: 69 months

InterventionWeeks (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC69.6
All Other Patients0

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Half Life (t1/2) of LMB-2

Dilutions of patient plasma was tested by cytotoxicity assays to determine half life. Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Interventionmin (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC360
All Other Patients251

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Number of Participants With Dose Limiting Toxicity (DLT)

DLT is a grade II-IV LMB-2 or fludarabine and cyclophosphamide (FC)-related toxicity, except vascular leak syndrome, alopecia, grade II-III allergic reaction with asymptomatic bronchospasm or urticarial is considered DLT. Grade III aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, and fever are not considered DLT. Grade IV creatine phosphokinase associated with any other DLT or not resolving to NCT00924170)
Timeframe: 30 days after last dose of LMB2

InterventionParticipants (Count of Participants)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC0
All Other Patients0

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Number of Participants With Serious and Non-serious Adverse Events

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT00924170)
Timeframe: 7 years and 12 days

InterventionParticipants (Count of Participants)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC10
All Other Patients8

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Overall Survival (OS)

OS is the time between the first day of treatment to the day of disease progression. Progressive disease is assessed by the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma, and is the appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count. (NCT00924170)
Timeframe: 70 months

InterventionMonths (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC18.6
All Other Patients3.75

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Peak Level of LMB-2 in Adult T-Cell Lymphoma

The maximum analyte concentration in serum was reported using a cytotoxicity assay measuring the level of LMB-2 in the plasma and using purified LMB-2 as a standard curve. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Interventionng/mL (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC602
All Other Patients484

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Percentage of Patients Who Developed Neutralizing Antibodies After One or More Cycles of LMB-2

Blood was drawn prior to each cycle of LMB-2 to determine if the level, >75% neutralization of 1000ng/ml of LMB-2 of neutralizing antibodies is too high to give additional LMB-2. Analysis was performed by cytotoxicity assay. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Interventionpercentage of participants (Number)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC40
All Other Patients0

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Plasma Clearance (CL) of LMB-2

Dilutions of patient plasma was tested by cytotoxicity assays to determine plasma clearance of LMB-2.The CL is a quantitative measure of the rate at which a drug substance is removed from the body. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

InterventionmL/min (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC109
All Other Patients101

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Progression Free Survival (PFS)

PFS was determined by the Kaplan Meier method beginning at the on study date and continuing until progression or last follow-up without progression. Progressive disease is assessed by the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma, and is the appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count. (NCT00924170)
Timeframe: 70 months

InterventionMonths (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC11.6
All Other Patients1.05

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Volume of Distribution of LMB-2

Dilutions of patient plasma were tested by cytotoxicity assays to determine volume of distribution of LMB-2. Volume distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. This was measured during the first 24 hours after administration of the dose on cycle 2 day 1. (NCT00924170)
Timeframe: 24 hours

InterventionLiters (Median)
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC49.6
All Other Patients26.6

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Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2

Adverse events is assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT00924170)
Timeframe: 7 years and 12 days

,,
InterventionParticipants (Count of Participants)
NeutropeniaLeukopenia/lymphopeniaAnemiaTransaminasesThrombocytopeniaFever/chillsPneumonitisLow cluster of differentiation 4 (CD4) countHypotension/tachycardiaRectal hemorrhageBilirubinDysuriaBladder infectionHypoxiaProteinuriaSepsis*
3Fludarabine and Cyclophosphamide: 0 + 300mg/m^22200110100000000
Fludarabine and Cyclophosphamide: 20 + 200mg/m^21101000100000000
Fludarabine and Cyclophosphamide: 25 + 250mg/m^29984533121111111

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Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders

Tumor tissue, including lymph node or skin biopsies was examined and analysis performed by flow cytometry to determine the amount of cancer cells in the body by measuring proteins which fall off cancer cells and go into the blood. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Interventionpg/ml (Median)
Soluble CD25, lowest post-treatment (nadir) valueMedian sCD25 Nadir for respondersMedian sCD25 Nadir for non-respondersMedian sCD25 PRE-treatment for respondersMedian sCD25 PRE-treatment for non-responders
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC10941082707133189366419

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Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders

Tumor tissue, including lymph node or skin biopsies was examined and analysis performed by flow cytometry to determine the amount of cancer cells in the body by measuring proteins which fall off cancer cells and go into the blood. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Interventionpg/ml (Median)
Soluble CD25, lowest post-treatment (nadir) valueMedian sCD25 Nadir for non-respondersMedian sCD25 PRE-treatment for non-responders
All Other Patients345913459117079

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Percentage of Participants With a Minimally Durable Clinical Response Rate

Response is based on the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma and must last >8 weeks to meet the primary endpoint of the study. Complete remission (CR) is complete disappearance of all detectable clinical and radiographic evidence of disease and disappearance of all disease related symptoms if present before therapy and normalization of those biochemical abnormalities definitely assignable to the lymphoma. Partial response is reduction by ≥50% of leukemia cell count or ≥50% reduction in the size of all measurable lesions, and no increase in size of any measurable or evaluable lesion or appearance of new lesion. Stable disease is neither a response nor progressive disease. Progressive disease is appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count. (NCT00924170)
Timeframe: 8 weeks

,
Interventionpercentage of participants (Number)
Overall ResponseComplete ResponsePartial ResponseStable DiseaseProgressive DiseaseNot Evaluable
All Other Patients0002537.537.5
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC8060202000

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Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide

Adverse events is assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. Grade 3 (mild), Grade 4 (life threatening) and Grade 5 (death). (NCT00924170)
Timeframe: 7 years and 12 days

,,
InterventionParticipants (Count of Participants)
NeutropeniaNausea/vomiting/anorexiaFever/chillsLeukopenia/lymphopeniaTransaminasesHypotension/tachycardiaThrombocytopeniaAnemiaMyalgia/headacheHypoalbuminemiaHematuriaProteinuriaFatigue/dizzinessEdemaAbdominal painDiarrheaCreatine phosphokinase (CPK)MucositisHypomagnesemiaBilirubinAlkaline phosphatase/gamma-glutamyl transferasePneumonitisLow cluster of differentiation 4 (CD4) countLipaseBladder infectionDyspneaProthrombin timePruritis
Fludarabine and Cyclophosphamide: 20 + 200mg/m^22211311022101001000100100011
Fludarabine and Cyclophosphamide: 25 + 250mg/m^21010109888976574653433233132210
Fludarabine and Cyclophosphamide: 30 + 300mg/m^22212122121102000010000100001

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Post Treatment Effects of LMB-2 + Fludarabine and Cyclophosphamide (FC) on Normal B and T Cell Subsets by Flow Cytometry

Peripheral blood was obtained and analyzed by flow cytometry. (NCT00924170)
Timeframe: First 24 hours after the dose given on Cycle 2, day 1

,
InterventionCells/µL (Median)
Normal T-cellsNormal CD4+ cellsNormal CD8+ cellsNormal B-cells
All Other Patients23.518623.50.5
Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC2899.5288

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Number of Participants With Adverse Events

Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. (NCT00924287)
Timeframe: 12 days

InterventionParticipants (Number)
Metastatic Cancer1

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Number of Participants With In Vivo Survival of Transfused Cells

In-vivo survival of infused cells is determined by analysis of the sequence of the variable region of the T cell receptor or flow cytometry (FACS). (NCT00924287)
Timeframe: 12 days

InterventionParticipants (Number)
Metastatic Cancer1

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0).

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT00924326)
Timeframe: Date treatment consent signed to date off study, approximately 101 months and 17 days.

InterventionParticipants (Count of Participants)
1x10^9-1x10^10 Cells/kg + High-dose Interleukin-28
1x10^9-1x10^10 Cells/kg + High-dose Interleukin-2 Retreat1
0.5x10^7 Cells/kg2
2.5x10^6 Cells/kg5
1.0x10^6 Cells/kg6
1.0x10^6 Cells/kg (Reduced Chemo)7
2.0x10^6 Cells/kg (Reduced Chemo)10
6.0x10^6 Cells/kg (Reduced Chemo)1
2.0x10^6 Cells/kg (Moderate Chemo)2
2.0x10^6 Cells/kg (9-12 Days Culture)2

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Number of Participants With a Response Assessed by the Response Criteria for Malignant Lymphoma

Participants were assessed by the Response Criteria for Malignant Lymphoma. Complete Remission (CR) is complete disappearance of all detectable evidence of disease and disease-related symptoms if present before therapy. Partial Remission (PR) requires ≥50% decrease in sum of the product of the diameters (SPD) of up to 6 of the largest dominant nodes or nodal masses; no increase in size of nodes, liver or spleen and no new sites of disease. Progressive disease (PD) is defined by ≥50% increase from nadir in the sum of the products of at least two lymph nodes, or if a single node is involved at least a 50% increase in the product of the diameters of this one node; and appearance of a new lesion greater than 1.5 cm in any axis even if other lesions are decreasing in size. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. (NCT00924326)
Timeframe: Scans performed at 6 weeks, 12 weeks and every 3-6 months for approximately 2 years

,,,,,,,,,
InterventionParticipants (Count of Participants)
Complete RemissionPartial RemissionStable DiseaseProgressive DiseaseNot Evaluable
0.5x10^7 Cells/kg20000
1.0x10^6 Cells/kg32001
1.0x10^6 Cells/kg (Reduced Chemo)23020
1x10^9-1x10^10 Cells/kg + High-dose Interleukin-224101
1x10^9-1x10^10 Cells/kg + High-dose Interleukin-2 Retreat30000
2.0x10^6 Cells/kg (9-12 Days Culture)20000
2.0x10^6 Cells/kg (Moderate Chemo)20000
2.0x10^6 Cells/kg (Reduced Chemo)61120
2.5x10^6 Cells/kg30011
6.0x10^6 Cells/kg (Reduced Chemo)00010

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Maximum Tolerated Dose

Maximally tolerated area under the curve of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion. The number reported will be an Area Under the Curve (AUC) measure reported in µmol-min/L. (NCT00943319)
Timeframe: 5 years

Interventionmmol-min/L (Number)
Busulfan and Fludarabine6800

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Overall Survival

Overall Survival measured as median survival in days (NCT00943319)
Timeframe: 5 years

Interventiondays (Median)
Busulfan and Fludarabine161

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Disease Free Survival

Disease Free Survival measured by median survival time in days (NCT00943319)
Timeframe: 5 years

Interventiondays (Median)
Busulfan and Fludarabine172

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Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(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 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+).~Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al)~The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported" (NCT00943800)
Timeframe: Up to 2 years

Interventionpercentage of patients (Number)
Acute GVHD (grade II-IV)Chronic GVHD
Treatment Group16.13.4

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Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years

We reported overall survival at 2 years and 5 years after transplant (NCT00943800)
Timeframe: up to 5 years

Interventionpercentage of patients (Number)
Two-year overall survivalFive-year overall survival
Treatment Group43.732.9

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Percentage of Participants With Neutrophil Engraftment

Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection. (NCT00943800)
Timeframe: Transplant (Day 0) through Day +28

Interventionpercentage of patients (Number)
Treatment Group85.1

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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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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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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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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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Overall Survival (OS) Rate

Overall survival in participants with multiple myeloma treated with Bortezomib (Velcade®) containing conditioning regimen and autologous as well as allogeneic transplantation. (NCT00948922)
Timeframe: End of 2 year, post transplant follow-up

Interventionpercentage (Number)
A: Allogeneic Stem Cell Transplant79.2
B: Autologous Stem Cell Transplant89.2
BE: Group B Expansion97.3

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Progression Free Survival (PFS)

PFS: Number of participants, per treatment arm with progression free survival at time of analysis. Survival time will be measured from the date of transplant to the date of progression, death or the last follow-up, whichever comes first. Progressive Disease (PD): Increase of ≥ 25% from lowest response value in any one or more of the following: Serum M-component and/or; Urine M-component and/or; Only in patients without measurable serum and urine M-protein levels; the difference between involved and uninvolved FLC levels. The absolute increase must be > 10 mg/dL; Bone marrow plasma cell percentage; absolute percentage ≥ 10%; Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas; Development of hypercalcemia that can be attributed solely to the plasma cell proliferative disorder. (NCT00948922)
Timeframe: End of 2 year, post transplant follow-up

InterventionParticipants (Count of Participants)
A: Allogeneic Stem Cell Transplant17
B: Autologous Stem Cell Transplant25
BE: Group B Expansion19

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Percentage of Participants With Acute or Chronic Graft-versus-host Disease (GVHD) Following Transplant

Percentage of participants with Acute or Chronic GVHD following transplant (NCT00948922)
Timeframe: End of 2 year, post transplant follow-up

Interventionpercentage of participants (Number)
A: Allogenic Stem Cell Transplant34

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Number of Participants With Chronic GvHD

Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3. (NCT00950846)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant Treatment Plan1

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Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT00950846)
Timeframe: 1 year

Interventionprobability of overall survival (Number)
Umbilical Cord Blood Transplant Treatment Plan0.868

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Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT00950846)
Timeframe: 100 days

Interventionprobability of overall survival (Number)
Umbilical Cord Blood Transplant Treatment Plan0.947

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Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT00950846)
Timeframe: 3 years

Interventionprobability of overall survival (Number)
Umbilical Cord Blood Transplant Treatment Plan0.868

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Number of Participants With Donor Engraftment After Transplant.

To evaluate donor engraftment at 100 days, 6 and 12 months after transplant. (NCT00950846)
Timeframe: 100 days, 6 months and 12 months

InterventionParticipants (Count of Participants)
100 days6 months12 months
Umbilical Cord Blood Transplant Treatment Plan363333

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Incidence of Severe Grade III-IV Acute GvHD at Day 100.

Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD. (NCT00950846)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant Treatment Plan1

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Incidence of Grades III-IV Graft-vs-host Disease

Development of graft-versus-host disease by day 100. (NCT00963872)
Timeframe: 0 to 100 days

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose2
Complement Fragment A - Larger Cell Dose0

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Neutrophil Engraftment

Achieving 500 neutrophils/uL by day 42. (NCT00963872)
Timeframe: Day 42

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose22
Complement Fragment A - Larger Cell Dose5

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Non-Relapse Mortality

Deaths not due to relapse. (NCT00963872)
Timeframe: Day 180

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose3
Complement Fragment A - Larger Cell Dose0

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Non-relapse Mortality

Deaths not due to relapse. (NCT00963872)
Timeframe: Day 360

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose3
Complement Fragment A - Larger Cell Dose0

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Number of Patients With the Complement 3a (C3a) Unit Predominating

Efficacy of the pre-incubation of one of two umbilical cord blood (UCB) units with C3a as part of a unrelated donor double UCB nonmyeloablative transplantation in patients with high-risk hematological malignancies. (NCT00963872)
Timeframe: Day 180

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose9
Complement Fragment A - Larger Cell Dose5

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Overall Survival

Survival (alive) from transplantation to last follow-up. (NCT00963872)
Timeframe: Day 360

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose14
Complement Fragment A - Larger Cell Dose5

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Overall Survival at Day 720

Survival (alive) from transplantation to last follow-up at day 720. (NCT00963872)
Timeframe: 720 days

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose13
Complement Fragment A - Larger Cell Dose5

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Relapse of Disease

Patients who developed disease relapse after transplantation. (NCT00963872)
Timeframe: Day 360

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose10
Complement Fragment A - Larger Cell Dose0

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Relapse of Disease

Patients who developed disease relapse after transplantation. (NCT00963872)
Timeframe: Day 720

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose11
Complement Fragment A - Larger Cell Dose1

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Incidence of Grades II-IV Graft-vs-host Disease

Development of graft-versus-host disease through day 100. (NCT00963872)
Timeframe: Day 0 through Day 100

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose8
Complement Fragment A - Larger Cell Dose1

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Donor Chimerism in Blood

Percentage of donor DNA present in the peripheral blood (NCT00963872)
Timeframe: Day 60

Interventionpercentage of donor DNA (Mean)
Complement Fragment 3A - Small Cell Dose91
Complement Fragment A - Larger Cell Dose97

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Donor Chimerism in Blood

Percentage of donor DNA present in the peripheral blood (NCT00963872)
Timeframe: Day 28

Interventionpercentage of donor DNA (Mean)
Complement Fragment 3A - Small Cell Dose92
Complement Fragment A - Larger Cell Dose96

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Donor Chimerism

Percentage of donor DNA in the bone marrow. (NCT00963872)
Timeframe: Day 360

Interventionpercentage of donor DNA (Mean)
Complement Fragment 3A - Small Cell Dose93
Complement Fragment A - Larger Cell Dose100

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Donor Chimerism

Percentage of donor DNA in the bone marrow. (NCT00963872)
Timeframe: Day 180

Interventionpercentage of donor DNA (Mean)
Complement Fragment 3A - Small Cell Dose99
Complement Fragment A - Larger Cell Dose100

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Donor Chimerism

Percentage of donor DNA in the bone marrow. (NCT00963872)
Timeframe: Day 100

Interventionpercentage of donor DNA (Mean)
Complement Fragment 3A - Small Cell Dose90
Complement Fragment A - Larger Cell Dose94

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Disease Progression

Patients who developed disease progression after transplantation. (NCT00963872)
Timeframe: Day 720

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose0
Complement Fragment A - Larger Cell Dose0

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Disease Progression

Patients who developed disease progression after transplantation. (NCT00963872)
Timeframe: Day 360

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose0
Complement Fragment A - Larger Cell Dose0

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Chronic Graft-Versus-Host Disease

Patients who developed chronic graft-versus-host disease. (NCT00963872)
Timeframe: Day 360

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose0
Complement Fragment A - Larger Cell Dose0

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Bone Marrow Chimerism

Percentage of donor DNA in the bone marrow. (NCT00963872)
Timeframe: Day 21

Interventionpercentage of donor DNA (Mean)
Complement Fragment 3A - Small Cell Dose84
Complement Fragment A - Larger Cell Dose89

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Platelet Recovery

Number of patients with >20,000 platelets/uL by day 180 (NCT00963872)
Timeframe: Day 180

Interventionparticipants (Number)
Complement Fragment 3A - Small Cell Dose19
Complement Fragment A - Larger Cell Dose5

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The Incidence of Acute GvHD

(NCT00987480)
Timeframe: 100 days

Interventionpercentage of participants (Number)
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected6.7

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Overall Survival at 3 Years

Overall Survival is defined as time from date of transplant to event (death from any cause) or last follow-up. (NCT00987480)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected80

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Disease-free Survival at 3 Years

"Defined as time from date of transplant to relapse, graft rejection or graft failure, or death.~Primary non-engraftment is diagnosed when the participants fails to achieve an ANC >/= 500/ul at any time in the first 28 days post-transplant.~For participants with MDS or AML, relapse will be analyzed as to type and genetic origin of the MDS/leukemic cells. These will be defined by an increasing number of blasts in the marrow over 5% by the presence of circulating peripheral blasts, or by the presence of blasts in any extramedullary site. Cytogenetic analysis of the marrow and/or peripheral blood will also be obtained for the diagnosis of relapse." (NCT00987480)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected77.8

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Incidence of Disease Recurrence

(NCT01005576)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Conditioning Regimen1

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Median Time to ANC Engraftment

(NCT01005576)
Timeframe: 100 days

Interventiondays (Median)
Conditioning Regimen14

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Median Time to Platelet Engraftment

(NCT01005576)
Timeframe: 100 days

Interventiondays (Median)
Conditioning Regimen27.5

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Primary Objective: Event-free Survival at 1 Year.

(NCT01005576)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Conditioning Regimen17

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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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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 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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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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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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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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Engraftments

(NCT01010217)
Timeframe: Day 28

InterventionParticipants (Count of Participants)
Haplo Arm80
1AgMM Related/Unrelated Donors50
MUD Donor21
Second Transplant0
Myelofibrosis0

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Participants With a Complete Response

Complete Response (CR) was defined as: Neutrophil count ≥ 1.0 ×109/L, Platelet count ≥ 100 ×109/L, Bone marrow aspirate ≤5% blasts and No extramedullary leukemia. Response evaluation following Induction Therapy (Cycle 1) and every 2-3 cycles during Consolidation Therapy (Cycles 2 - 7) where Cycle is 4-6 weeks. (NCT01019317)
Timeframe: Minimally 6 weeks (Cycle 1) up to 1 year (7 cycles)

InterventionParticipants (Number)
Cytarabine + Fludarabine34

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2-year Progression-free Survival in Early Disease Participants

"Percentage of participants who were alive and progression free at 2 years for participants with early disease stage. The 2 year progression free survival, with 95% confidence interval, was estimated using the Kaplan Meier method.~A progression is defined as one of the following events:~>= 50% increase in the products of at least two lymph nodes on two consecutive determinations two weeks apart (at least one lymph node must be >= 2 cm); appearance of new palpable lymph nodes.~>= 50% increase in the size of the liver and/or spleen as determined by measurement below the respective costal margin; appearance of palpable hepatomegaly or splenomegaly, which was not previously present.~> 50% increase in peripheral blood lymphocytes with an absolute increase > 5000/μL.~Transformation to a more aggressive histology (i.e., Richter's syndrome or prolymphocytic leukemia with >= 56% prolymphocytes)." (NCT01027000)
Timeframe: 2 years post-registration

Interventionpercentage of participants (Median)
Treatment (Combination of Chemotherapy and Transplant)79.5

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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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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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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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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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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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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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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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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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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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Serious Infections

Serious infections are reported as the number of participants experienced serious infections. (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)6

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Overall Survival (OS), 1 Year

Assessed as subjects remaining alive 12 months after CD34+ cell infusion (ie, excludes death due to any cause) (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Number)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)2

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Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells

The maximum-tolerated dose (MTD) was to be determined based on the safety and feasibility observed for a pre-determined set of cellular dose level combinations of regulatory T-cells (T-reg) and conventional T-cells (T-con). (NCT01050764)
Timeframe: 30 days after HSCT infusion

Interventioncells/kg (Number)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)NA

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To Measure the Incidence and Severity of Acute and Chronic GvHD

Population of participants that received HSCT and T-reg plus T-con, and developed actue, chronic, or any graft vs host disease (GvHD) (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Acute GvHDChronic GvHDAny GvHD (actue + chronic)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)123

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Acute Graft-versus-Host-Disease (aGvHD)

The primary outcome was incidence of grade 3 or 4 acute graft-vs-host-disease (aGvHD), reported as the number of participants developing grade 3 or 4 aGvHD. (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)1

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Median Overall Survival (OS)

Reported as the median overall survival (OS) in months from infusion of the hematopoietic stem cells (HSCT) (NCT01050764)
Timeframe: 25 months

Interventionmonths (Median)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)3.7

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Number of Participants With an Overall Response

Overall (OR) is the total number of participants with any response: Complete remission (CR), is defined as > 30% lymphocytes in the bone marrow, recovery of blood counts and no clinical symptoms; Nodular partial remission (NPR), is the same as CR but with nodules; Partial remission (PR) is > 50% decrease of clinical symptoms from baseline and recovery from blood counts. (NCT01082939)
Timeframe: 6 cycles of treatment (28 days per cycle)

InterventionParticipants (Number)
Complete remission (CR)Nodular partial remission (NPR)Partial remission (PR)
CFAR23326

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Plasma Concentration of IDELA (Cohort 4)

(NCT01088048)
Timeframe: Predose at Week 0; predose, 0.5, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0 hours postdose at Week 4; predose, 1.5 hours postdose at Week 12; and predose, 1.5 hours postdose at Week 24

Interventionng/mL (Mean)
Pre-dose (Week 0)Pre-dose (Week 4)0.5 hr post-dose (Week 4)1.0 hr post-dose (Week 4)1.5 hr post-dose (Week 4)2.0 hr post-dose (Week 4)3.0 hr post-dose (Week 4)4.0 hr post-dose (Week 4)6.0 hr post-dose (Week 4)Pre-dose (Week 12)1.5 hr post-dose (Week 12)Pre-dose (Week 24)1.5 hr post-dose (Week 24)
Idelalisib 150 mg (Cohort 4)0.00.01119.5994.51758.2737.0605.0547.0524.0401.92018.2752.52251.1

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Plasma Concentration of IDELA (Cohort 6)

(NCT01088048)
Timeframe: Predose, 1.5 hours postdose at Weeks 0, 4, 12 and 24

Interventionng/mL (Mean)
Pre-dose (Week 0)1.5 hr post-dose (Week 0)Pre-dose (Week 4)1.5 hr post-dose (Week 4)Pre-dose (Week 12)1.5 hr post-dose (Week 12)Pre-dose (Week 24)1.5 hr post-dose (Week 24)
Idelalisib 150 mg (Cohort 6)0.01930.7530.81869.8677.91733.0346.81710.7

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Plasma Concentration of IDELA (Cohort 7)

(NCT01088048)
Timeframe: Predose, 1.5 hours postdose at Weeks 0, 5 and 13

Interventionng/mL (Mean)
Pre-dose (Week 0)1.5 hr post-dose (Week 0)Pre-dose (Week 5)1.5 hr post-dose (Week 5)Pre-dose (Week 13)1.5 hr post-dose (Week 13)
Idelalisib 150 mg (Cohort 7)NA1603.120.71621.3354.8592.7

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Plasma Concentration of IDELA (Cohort 1, Cohorts 2 and 3, Cohort 5)

(NCT01088048)
Timeframe: Predose, 0.5, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0 hours postdose at Week 0; predose, 1.5 hours postdose at Weeks 4, 12, and 24

,,
Interventionng/mL (Mean)
Pre-dose (Week 0)0.5 hr post-dose (Week 0)1.0 hr post-dose (Week 0)1.5 hr post-dose (Week 0)2.0 hr post-dose (Week 0)3.0 hr post-dose (Week 0)4.0 hr post-dose (Week 0)6.0 hr post-dose (Week 0)Pre-dose (Week 4)1.5 hr post-dose (Week 4)Pre-dose (Week 12)1.5 hr post-dose (Week 12)Pre-dose (Week 24)1.5 hr post-dose (Week 24)
Idelalisib 100 mg (Cohort 1)0.4437.61022.41434.01282.71001.0788.4523.7416.51297.2361.91309.5369.91061.6
Idelalisib 150 mg (Cohort 5)0.01380.01600.01564.71400.01170.0795.0517.0408.01877.9433.91426.8549.1885.6
Idelalisib 150 mg (Cohorts 2 and 3)68.81231.41789.32017.31732.81296.1910.0486.0364.11808.1351.41883.0419.71840.1

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Overall Survival

Overall Survival (OS) was defined as the interval from the start of study drug to death from any cause. (NCT01088048)
Timeframe: Up to 5 years

Interventionmonths (Median)
Idelalisib + RituximabNA
Idelalisib + BendamustineNA
Idelalisib + EverolimusNA
Idelalisib + BortezomibNA
Idelalisib + Rituximab + BendamustineNA
Idelalisib + OfatumumabNA
Idelalisib + FludarabineNA
Idelalisib + ChlorambucilNA
Idelalisib + Rituximab + ChlorambucilNA
Idelalisib + Rituximab + LenalidomideNA

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Progression-free Survival

"Progression free survival (PFS) was defined as the interval from the start of study drug to the earlier of the first documentation of disease progression or death from any cause.~The response definitions were based on the following standard criteria established for each indication:~CLL: International Workshop on chronic lymphocytic leukemia (IWCLL), 2008~iNHL & MCL: Cheson, 2007" (NCT01088048)
Timeframe: Up to 5 years

Interventionmonths (Median)
Idelalisib + RituximabNA
Idelalisib + BendamustineNA
Idelalisib + Everolimus4.3
Idelalisib + Bortezomib8.1
Idelalisib + Rituximab + BendamustineNA
Idelalisib + OfatumumabNA
Idelalisib + FludarabineNA
Idelalisib + ChlorambucilNA
Idelalisib + Rituximab + ChlorambucilNA
Idelalisib + Rituximab + LenalidomideNA

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Time to Response

Time to response (TTR) was defined as the interval from the start of study drug to the first documentation of CR or PR. (NCT01088048)
Timeframe: Up to 5 years

Interventionmonths (Median)
Idelalisib + Rituximab1.9
Idelalisib + Bendamustine1.9
Idelalisib + Everolimus1.9
Idelalisib + Bortezomib1.9
Idelalisib + Rituximab + Bendamustine1.9
Idelalisib + Ofatumumab1.9
Idelalisib + Fludarabine1.9
Idelalisib + Chlorambucil1.9
Idelalisib + Rituximab + Chlorambucil1.9
Idelalisib + Rituximab + Lenalidomide3.0

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Toxicity of Administration of IDELA

Percentage of participants experiencing toxicities of administration of IDELA were measured according to the Common Terminology Criteria for Adverse Events v4.02 (NCT01088048)
Timeframe: First dose date up to 5 years

Interventionpercentage of participants (Number)
Idelalisib + Rituximab100.0
Idelalisib + Bendamustine100.0
Idelalisib + Everolimus100.0
Idelalisib + Bortezomib83.33
Idelalisib + Rituximab + Bendamustine100.0
Idelalisib + Ofatumumab100.0
Idelalisib + Fludarabine100.0
Idelalisib + Chlorambucil100.0
Idelalisib + Rituximab + Chlorambucil100.0
Idelalisib + Rituximab + Lenalidomide100.0

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Duration of Exposure to IDELA

Duration of exposure to IDELA was summarized using descriptive statistics. (NCT01088048)
Timeframe: First dose date up to 12 months

Interventionmonths (Mean)
Idelalisib + Rituximab8.1
Idelalisib + Bendamustine7.6
Idelalisib + Everolimus4.2
Idelalisib + Bortezomib5.1
Idelalisib + Rituximab + Bendamustine8.0
Idelalisib + Ofatumumab8.3
Idelalisib + Fludarabine8.9
Idelalisib + Chlorambucil8.8
Idelalisib + Rituximab + Chlorambucil8.7
Idelalisib + Rituximab + Lenalidomide7.7

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Plasma Concentration of Lenalidomide

(NCT01088048)
Timeframe: Predose, 1.5 hours postdose at Week 1 and predose at Week 5

Interventionng/mL (Mean)
Pre-dose (Week 1)1.5 hr post-dose (Week 1)Pre-dose (Week 5)
Lenalidomide (Cohort 7a, 7b, 7c)NA51.6NA

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Sub-study: Plasma Concentration of IDELA (Cohorts 1-4)

(NCT01088048)
Timeframe: pre dose and 0.5, 1, 1.5, 2.0, 3.0, 4.0, and 6.0 hours post dose

,
Interventionng/mL (Mean)
Pre-dose0.5 hr post-dose1.0 hr post-dose1.5 hr post-dose2.0 hr post-dose3.0 hr post-dose4.0 hr post-dose6.0 hr post-dose
Idelalisib 100 mg (Cohort 1a, 1b)1.5437.61022.41264.71282.71001.0788.4523.7
Idelalisib 150 mg (Cohorts 2a, 2b, 3a, 3c, 3d, 3e, 3f, 3g, 4a, 4b)0.01222.11723.11599.21646.21238.5879.8489.3

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Plasma Concentration of Bendamustine

(NCT01088048)
Timeframe: Predose, 0.25, 0.5, 0.75, 1.0, 1.25, 1.5, 2.0, 3.0, 4.0, 5.0, 6.0 hours postdose at Week 0

Interventionng/mL (Mean)
Pre-dose (Week 0)0.25 hr post-dose (Week 0)0.5 hr post-dose (Week 0)0.75 hr post-dose (Week 0)1.0 hr post-dose (Week 0)1.25 hr post-dose (Week 0)1.5 hr post-dose (Week 0)2.0 hr post-dose (Week 0)3.0 hr post-dose (Week 0)4.0 hr post-dose (Week 0)5.0 hr post-dose (Week 0)6.0 hr post-dose (Week 0)
Bendamustine (Cohorts 1b, 2b, 3a, 3b, 3f, 3g, 4b, 5c)NA3484.14694.73433.22847.21916.41211.2514.0463.478.515.34.4

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Overall Response Rate

"Overall Response Rate (ORR) was defined as the percentage of participants achieving a complete response (CR) or partial response (PR).~The response definitions were based on the following standard criteria established for each indication:~CLL: International Workshop on chronic lymphocytic leukemia (IWCLL),2008~iNHL & MCL: Cheson, 2007" (NCT01088048)
Timeframe: Up to 5 years

Interventionpercentage of participants (Number)
Idelalisib + Rituximab78.4
Idelalisib + Bendamustine84.3
Idelalisib + Everolimus44.4
Idelalisib + Bortezomib61.1
Idelalisib + Rituximab + Bendamustine81.8
Idelalisib + Ofatumumab71.4
Idelalisib + Fludarabine91.7
Idelalisib + Chlorambucil66.7
Idelalisib + Rituximab + Chlorambucil93.3
Idelalisib + Rituximab + Lenalidomide71.4

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Plasma Concentration of Everolimus

(NCT01088048)
Timeframe: Predose, 1.5 hours postdose at Weeks 0 and 4

Interventionng/mL (Mean)
Pre-dose (Week 0)1.5 hr post-dose (Week 0)Pre-dose (Week 4)1.5 hr post-dose (Week 4)
Everolimus (Cohort 5a)NA93.03.056.3

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Duration of Response

Duration of response (DOR) was defined as the interval from the first documentation of CR or PR to the earlier of the first documentation of disease progression or death from any cause. (NCT01088048)
Timeframe: Up to 5 years

Interventionmonths (Median)
Idelalisib + RituximabNA
Idelalisib + BendamustineNA
Idelalisib + Everolimus5.6
Idelalisib + Bortezomib9.3
Idelalisib + Rituximab + BendamustineNA
Idelalisib + OfatumumabNA
Idelalisib + FludarabineNA
Idelalisib + ChlorambucilNA
Idelalisib + Rituximab + ChlorambucilNA
Idelalisib + Rituximab + LenalidomideNA

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Maximum Tolerated Dose (MTD) of Bendamustine Combined With Fixed-Dose Fludarabine and Rituximab (FBR)

MTD defined as highest dose level in which 6 participants have been treated with /= 3 non-hematologic toxicity. Hematologic toxicity grade >/= 3 that lasts longer than 42 days considered a DLT. Hematologic toxicity graded according to the 2008 IWCLL criteria for grading. Tumor lysis not considered a DLT. (NCT01096992)
Timeframe: After 4 week cycle

Interventionmg/m^2 (Number)
Phase 130

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Overall Response Rate of Bendamustine Combined With Fixed-Dose Fludarabine and Rituximab (FBR)

Overall Response is Complete response (CR) + Partial response (PR). Overall response evaluated by 2008 International Workshop on Chronic Lymphocytic Leukemia (IWCLL) 2008 for complete or partial response and progressive disease. Complete remission (CR), requiring absence of peripheral blood clonal lymphocytes by immunophenotyping, absence of lymphadenopathy, absence of hepatomegaly or splenomegaly, absence of constitutional symptoms and satisfactory blood counts; positive or negative minimal residual disease (MRD); Partial remission (PR), defined as ≥ 50% fall in lymphocyte count, ≥ 50% reduction in lymphadenopathy or ≥ 50% reduction in liver or spleen, together with improvement in peripheral blood counts; (NCT01096992)
Timeframe: Overall response assessed 2 months after 6th or last course if participants not able to receive all 6 intended courses of treatment.

InterventionParticipants (Count of Participants)
Phase 1 20 mg/m^25
Phase 1 30 mg/m^23
Phase 1 40 mg/m^25
Phase 1 50 mg/m^22
Phase 217

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Response Rate

Response includes Complete Response (CR), Partial Response (PR), and Stable Disease (SD) as defined by Response Evaluation Criteria In Solid Tumors Criteria (RECIST v.1.1) for target lesions and assessed by CT or MRI. Complete Response (CR), disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for Progressive Disease. (NCT01105650)
Timeframe: Month 3

Interventionparticipants (Number)
Arm 1: CsA1
Arm 2: CsA/Methylprednisolone (10mg)/6 Doses of Interleukin-22
Arm 3: CsA/Methylprednisolone (1mg)/6 Doses of Interleukin-24

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Overall Survival

Number of participants alive at 1 year. (NCT01105650)
Timeframe: 1 Year

Interventionparticipants (Number)
Arm 1: CsA0
Arm 2: CsA/Methylprednisolone (10mg)/6 Doses of Interleukin-21
Arm 3: CsA/Methylprednisolone (1mg)/6 Doses of Interleukin-23

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Time to Disease Progression

Time from study entry until progressive disease or data collection cutoff. (NCT01105650)
Timeframe: 1 Year

Interventiondays (Median)
Arm 1: CsA52
Arm 2: CsA/Methylprednisolone (10mg)/6 Doses of Interleukin-298
Arm 3: CsA/Methylprednisolone (1mg)/6 Doses of Interleukin-2100

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Number of Participants With Progressive Disease at One Year

(NCT01105650)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Arm 1: CsA2
Arm 2: CsA/Methylprednisolone (10mg)/6 Doses of Interleukin-21
Arm 3: CsA/Methylprednisolone (1mg)/6 Doses of Interleukin-25

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Percent of Patients With Successful Expansion of Natural Killer Cells After Infusion

The primary objective of this study was to estimate the incidence of in vivo expansion of natural killer (NK) cells 14 days after infusion of an allogeneic donor product enriched for NK progenitors. Successful in vivo donor NK cell expansion was defined by measuring an absolute circulating donor-derived NK cell count of >100 cells/ul in the patient's peripheral blood 14 days after infusion. (NCT01106950)
Timeframe: Day 14

InterventionPercentage of patients (Number)
Evaluable (Treated) Patients27

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Percent of Patients With Natural Killer Cell Expansion Versus KIR Genotype Versus Treg Depletion

Association between in vivo natural killer (NK) cell expansion and complete response without platelet recovery (CRp) with donor killer immunoglobulin-like (KIR) genotype and Treg depletion. In vivo donor NK cell expansion was correlated with regulatory T-cell (Treg) depletion as detected on flow cytometry. (NCT01106950)
Timeframe: Day 14

InterventionPercentage of patients (Number)
Evaluable (Treated) Patients (Expansion=No)43
Evaluable (Treated) Patients (Expansion=Yes)13

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Percent of Patients With Incidence of Relapse

Number of patients who have had a relapse(the return of disease after its apparent recovery/cessation) after obtaining a complete remission of their disease. (NCT01106950)
Timeframe: Month 6

InterventionPercentage of patients (Number)
Evaluable (Treated) Patients53

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Percent of Patients With Disease Free Survival

Number of patients alive and disease free at 6 months. 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. (NCT01106950)
Timeframe: Month 6

InterventionPercentage of patients (Number)
Evaluable (Treated) Patients33

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Percent of Patients With Complete Remission of Disease

Disease response was defined as complete remission (disease response) by morphologic criteria including <5% blasts in a moderately cellular or cellular marrow. Complete remission was also correlated with NK cell expansion in vivo, IL-15 levels and donor/recipient KIR B genotyping, and Treg depletion. (NCT01106950)
Timeframe: At least 4 weeks after last dose (28 days)

InterventionPercentage of patients (Number)
Evaluable (Treated) Patients53

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Survival and Disease-free Survival (DFS)

(NCT01119066)
Timeframe: 2 years post transplant

,,,
Intervention% of pts at 2 years (Number)
SurvivalDisease Free Survival
Busulfan, Melphalan and Fludarabine72.162.1
Clofarabine, Melphalan and Thiotepa63.859.6
Melphalan, Fludarabine and Thiotepa71.662.3
Total Body Irradiation, Thiotepa and Cyclophosphamide68.758.9

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Survival and Disease-free Survival (DFS)

(NCT01119066)
Timeframe: 1 year post transplant

,,,
Intervention% of pts at 1 year post transplant (Number)
SurvivalDisease free survival
Busulfan, Melphalan and Fludarabine83.873.7
Clofarabine, Melphalan and Thiotepa85.183.0
Melphalan, Fludarabine and Thiotepa81.872.7
Total Body Irradiation, Thiotepa and Cyclophosphamide80.868.3

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The Incidence of Durable Hematopoietic Engraftment for T-cell Depleted Transplants Fractionated by the CliniMACS System Administered After Each of the Four Disease Targeted Cytoreduction Regimens.

(NCT01119066)
Timeframe: 3 years

,,,
InterventionParticipants (Count of Participants)
EngraftedPrimary Graft FailureLate graft failureNot Evaluable Engraftment
Busulfan, Melphalan and Fludarabine205050
Clofarabine, Melphalan and Thiotepa45020
Melphalan, Fludarabine and Thiotepa10010
Total Body Irradiation, Thiotepa and Cyclophosphamide118011

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Survival and Disease-free Survival (DFS)

(NCT01119066)
Timeframe: at 6 months post transplant

,,,
Interventionpercentage of participants (Number)
SurvivalDisease Free Survival
Busulfan, Melphalan and Fludarabine92.489.0
Clofarabine, Melphalan and Thiotepa87.285.1
Melphalan, Fludarabine and Thiotepa90.990.9
Total Body Irradiation, Thiotepa and Cyclophosphamide93.382.5

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Number of Participants With Acute and Chronic GVHD Following T-cell Depleted, CD34+ Progenitor Cell Enriched Transplants Fractionated by the CliniMACS System.

Standard BMT-CTN and IBMTR systems clinical criteria as defined by Rowlings, et al will be used to establish and grade acute GvHD. Chronic GvHD will be diagnosed and graded according to the criteria of Sullivan (CIBMTR). (NCT01119066)
Timeframe: 3 years

,,,
Interventionparticipants (Number)
aGVHD II-IVcGVHD, LimitedcGVHD, Extensive
Busulfan, Melphalan and Fludarabine6041
Clofarabine, Melphalan and Thiotepa1011
Melphalan, Fludarabine and Thiotepa201
Total Body Irradiation, Thiotepa and Cyclophosphamide3428

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Proportion of Participants With Relapsed Multiple Myeloma With Progression-free (PFS)

(NCT01131169)
Timeframe: 2 years

InterventionProportion of participants PFS (Number)
Relapsed Multiple Myeloma0.31

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Proportion of Participants With Relapsed Multiple Myeloma Alive at 2 Years

(NCT01131169)
Timeframe: 2 years

InterventionProportion of pts alive at 2 years (Number)
Relapsed Multiple Myeloma0.54

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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 Participant With Adverse Event (AE)

An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with study treatment. (NCT01144403)
Timeframe: Up to 50 months (approximately)

Interventionparticipants (Number)
Rituximab8

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Progression-free Survival (PFS)

PFS is defined as the interval between the day of enrollment and the first documentation of progressive disease or death. Progression of disease is defined as at least a 20 percent (%) increase in the sum of longest diameter (LD) of target lesions, taking as reference the smallest sum LD recorded since the treatment started or the appearance of 1 or more new lesions. (NCT01144403)
Timeframe: From the time of enrollment until death due to any cause (up to 50 months [approximately])

Interventiondays (Median)
Rituximab653

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Overall Survival (OS)

Overall survival is defined as time from date of enrollment to the date of death, regardless of the cause of death. (NCT01144403)
Timeframe: From the time of enrollment until death due to any cause (up to 50 months [approximately])

Interventiondays (Median)
Rituximab927

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Overall Response Rate (ORR)

Overall Response Rate (ORR) was determined by tumor response according to International Workshop Group to Standardize Response Criteria for mantle cell lymphoma (MCL) criteria from confirmed evaluations of both target, radiographically evaluated, and non-target lesions. A responder is defined as a subject experiencing either a complete (CR)/ unconfirmed complete (Cru), or partial response (PR) by these criteria. As per criteria; CR = disappearance of all evidence of disease; CRu = the sum of the product of the diameters (SPD) of multiple nodes decreased by at least 75%; PR = regression of measurable disease and no new sites. (NCT01144403)
Timeframe: Up to 50 months (approximately)

Interventionpercentage of participants (Number)
Rituximab87.5

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Progression Free Survival Rate 2 Years After Initiation of Induction Therapy

"Death or disease progression defined by the 2008 IWCLL guideline as follows;~Greater than or equal to 50% increase in the SPD of at least 2 lymph nodes (at least one node must be greater than or equal to 2 cm); appearance of any new lymph nodes on physical examination or imaging~Greater than or equal to 50% increase in the size of the liver and/or spleen as determined by measurement below the respective costal margin or CT scan or appearance of palpable hepatomegaly or splenomegaly, which was not previously present~Greater than or equal to 50% increase in the absolute number of circulating lymphocytes to at least 5000/ul~Transformation to a more aggressive histology~Occurrence of any cytopenia attributable to CLL. After treatment: the progression of any cytopenia (unrelated to autoimmune cytopenia), as documented by a decrease of Hb levels by more than 20 g/L (2 g/dL) or to less than 100 g/L (10 g/dL), or by a decrease of platelet counts by more than 50% or to les" (NCT01145209)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
FO Arm14
FCO Arm9

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Participants With MRD Negativity at the Completion of Consolidation Immunotherapy Who Failed to Achieve MRD Negativity

Participants with MRD negativity at the completion of consolidation immunotherapy who failed to achieve MRD negativity following completion of induction chemoimmunotherapy (NCT01145209)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
FO Arm14
FCO Arm4

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Participants With Minimal Residual Disease (MRD) Negativity

Participants with minimal residual disease (MRD) negativity following the completion of induction chemoimmunotherapy. (NCT01145209)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
FO ARM4
FCO ARM6

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Participants With Complete Response Rates Following Induction Chemoimmunotherapy.

"Participants with complete response rates to induction chemoimmunotherapy.~Criteria for complete response (CR): CR requires all of the following:~Peripheral blood lymphocytes < 4000/uL~Absence of significant lymphadenopathy by physical examination and appropriate radiographic techniques (CT or MRI). All lymph nodes must have regressed to <=1.5cm in greatest diameter~Absence of hepatomegaly or splenomegaly by physical examination, or appropriate radiographic techniques. Spleen, if enlarged before therapy must have regressed in size and must not be palpable by physical exam.~Absence of constitutional symptoms~Normal CBC, defined as: - Polymorphonuclear cells ≥ 1,500/uL - Platelets > 100,000/uL (untransfused) - Hemoglobin > 11 g/dL (untransfused)~Bone marrow biopsy demonstrates normal cellularity for age, with less than 30% of nucleated cells being lymphocytes. Lymphoid nodules should be absent" (NCT01145209)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
FCO Arms6
FO Arm2

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Median Relationship of CD20 Expression With MRD Negativity Rate

Median relationship of biomarker, CD20 expression with MRD negativity clinical response rate. Flow cytometry was use to quantified surface CD20 on peripheral blood. (NCT01145209)
Timeframe: 2 years

Interventionsites per cell (Median)
Minimal Residual Disease (MRD) Positivity10247
Minimal Residual Disease (MRD) Negativity15131

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Progression-free Survival

"Progression-free survival rate (percentage) at 2 and 5 years is defined as the percentage of patients who are alive and progression free at 2 and 5 years from date of transplantation respectively.~AML progression is defined as:~Reappearance of leukemia blast cells in peripheral blood and > 5% blasts in marrow~If no circulating blasts, but the marrow contains 5-20% blasts, a repeat bone marrow >= 1 week later with > 5% blasts~Development of extramedullary leukemia MDS progression is defined as~For patients with <5% bone marrow blasts: ≥50% increase in blasts to >5% blasts~For patients with 5-10% bone marrow blasts: ≥50% increase to >10% blasts~Any of the following: Reappearance of prior documented characteristic cytogenetic abnormality or refractory cytopenias with unequivocal evidence of dysplasia on bone marrow biopsy/aspirate" (NCT01168219)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
PFS at 2 yearsPFS at 5 years
Treatment (Chemotherapy and Transplant)41.226.9

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Overall Survival (OS)

Overall survival rate (percentage) at 2 and 5 years is defined as the percentage of patients who are still alive 2 and 5 years after date of transplantation respectively. Estimated using the Kaplan-Meier product limit estimator. (NCT01168219)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
OS at 2 yearsOS at 5 years
Treatment (Chemotherapy and Transplant)45.731.2

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100-day Mortality

The number of death reported within the first 100 days after transplant. (NCT01168219)
Timeframe: Up to 100 days post-treatment

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy and Transplant)10

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Toxicities

Dasatinib may enhance the myelosuppression expected from fludarabine. This toxicity will be monitored with frequent CBC's. If after Day 21 of a cycle there is a grade 4 cytopenia, a dose reduction will occur in the next cycle of treatment, and that cycle cannot start until the ANC > 1,000 and the platelets > 25,000. There is also a risk for pleural effusions with dasatinib, but the risk will be low, since there is a break from dasatinib dosing on days 15-28 of each cycle. Nevertheless, if a grade 2 pleural effusion occurs, there will be a dose reduction in the next cycle of treatment. (NCT01173679)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Platelets72308625Neutropenia72308625Fatigue72308625Dyspnea72308625Pleural effusion72308625Bleeding72308625Fever alone72308625Infection72308625
345Did not have any
Dasatinib, Rituximab, Fludarabine4
Dasatinib, Rituximab, Fludarabine3
Dasatinib, Rituximab, Fludarabine1
Dasatinib, Rituximab, Fludarabine0
Dasatinib, Rituximab, Fludarabine9

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Progression-Free and Overall Survival

To describe the progression-free and overall surivial (NCT01173679)
Timeframe: 2 years

Interventionmonths (Median)
Progression-free survivalOverall survival
Dasatinib, Rituximab, Fludarabine8.7524

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Number of Patients With an Objective Response

The number of patients with a partial response (PR) or complete response (CR). For patients with non-hodgkin's lymphoma: CR - complete disappearance of all detectable clinical evidence of disease and disease-related symptoms. PR - at least a 50% decrease in sum of the product of the diameters of up to six of the largest dominant nodes or nodal masses. For patients with chronic lymphocytic leukemia: CR - disappearance of all palpable disease, normalization of the blood counts without transfusions, bone marrow aspirate lymphocyte percentage < 30%, and no evidence of disease on bone marrow biopsy. PR - 50% or more reduction in palpable disease as well as one or more of the remaining features: neutrophils >= 1.5 × 109/L or 50% improvement over baseline, platelets more than 100 × 109/L or 50% improvement over baseline, and hemoglobin more than 11.0 g/dL or 50% improvement over baseline without transfusions. (NCT01181258)
Timeframe: Month 2 Post Infusion

InterventionParticipants (Count of Participants)
Patients Receiving NK Cell Infusion4

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Time to Disease Progression

Cumulative incidence will be used to determine time to disease progression. (NCT01181258)
Timeframe: Day 1 through Month 12

Interventiondays (Median)
Patients Receiving NK Cell Infusion38

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Serious Adverse Events

Number of participants experiencing serious adverse events that occur during study. Adverse event collection for the purposes of this study will focus on targeted adverse events and unexpected adverse events at specific time points in relation to the NK cell infusion and post infusion IL2 injections. (NCT01181258)
Timeframe: Day 1 through Month 12

InterventionParticipants (Count of Participants)
Patients Receiving NK Cell Infusion15

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Patients With Expansion of NK Cells

Number of patients who experience in vivo expansion of allogeneic donor natural killer (NK) cells. (NCT01181258)
Timeframe: Day 14

InterventionParticipants (Count of Participants)
Patients Receiving NK Cell Infusion0

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Cumulative Incidence of Non-relapse Mortality

Non-relapse mortality is defined as participants who die from causes other than their underlying disease relapse, such as infection or graft versus host disease (NCT01181271)
Timeframe: 2-years after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant11.1

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Estimated Two Year Overall Survival Rate for All Participants

(NCT01181271)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant83

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Cumulative Incidence of Disease Relapse

(NCT01181271)
Timeframe: 2-years after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant17.2

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Estimated Two Year Overall Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants

(NCT01181271)
Timeframe: Two-years after Allogeneic Transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant89

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Estimated Two Year Progression Free Survival Rate for Participants Undergoing Only Autologous Transplant

(NCT01181271)
Timeframe: Two Years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant46

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Number of Days After Allogeneic Transplant Until Absolute Neutrophil Count Was Equal to or Greater Than 500/uL

(NCT01181271)
Timeframe: within 28 days after allogeneic transplant

Interventiondays (Median)
Autologous Then Allogeneic Transplant12

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Peripheral Blood All-cell Donor Chimerism

Successful donor stem cell engraftment is defined as when ≥ 80% of hematopoietic elements are donor-derived as determined by chimerism assays from peripheral blood at day 100 after non-myeloablative allogeneic stem cell transplantation. (NCT01181271)
Timeframe: 100 days post allogeneic transplant

Interventionpercentage of donor-derived elements (Median)
Autologous Then Allogeneic Transplant95

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Estimated Two Year Overall Survival Rate for Participants Undergoing Only Autologous Transplant

(NCT01181271)
Timeframe: two years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant69

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Estimated Two Year Progression Free Survival Rate for All Participants

(NCT01181271)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant64

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Estimated Two Year Progression Free Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants

(NCT01181271)
Timeframe: 2 years after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant72

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Cumulative Incidence of Grades II to IV Acute Graft Versus Host Disease (GVHD)

Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01181271)
Timeframe: within 200 days after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant13.8

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Cumulative Incidence of Extensive Chronic Graft-versus-host-disease

Extensive chronic graft versus-host-disease (GVHD) was defined as GVHD that required systemic immunosuppression. (NCT01181271)
Timeframe: 1-year after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant37.9

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Toxicity

To evaluate the toxicity profile of this regimen. Adverse event counts by grade are presented. (NCT01186458)
Timeframe: 6 months

Interventionnumber of adverse events (Number)
Total Adverse EventsGrade 1 Adverse EventsGrade 2 Adverse EventsGrade 3 Adverse EventsGrade 4 Adverse EventsGrade 5 Adverse Events
Fludarabine, Velcade and Rituximab1227735910

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Number of Patients Who Experienced Disease Relapse

Number of patients who relapsed who experienced disease relapse following Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia (NCT01187017)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia0

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Participant Survival Following Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia

Participant survival at 6 months following Fludarabine/ Cyclophosphamide in participants with Severe Aplastic Anemia (NCT01187017)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia1

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Response Rate at 6 Months

The primary objective is to assess hematologic response of Refractory Severe aplastic anemia (SAA) subjects to who have received fludarabine and cyclophosphamide. Subjects blood counts will be evaluated at 6 months to assess a hematologic response. The hematologic response will be defined as complete, partial or no response. (NCT01187017)
Timeframe: 6 months

Interventionparticipants (Number)
No ResponsePartial ResponseComplete Response
Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia100

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Number of Participants With Clonal Evolution

Number of participants with Severe Aplastic Anemia who received Fludarabine Plus Cyclophosphamide that experienced Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplasia or Acute Leukemia. (NCT01187017)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia0

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Rates of Acute and Chronic Graft Versus Host Disease (GVHD).

Count/Percentage rates of acute and chronic graft versus host disease (GVHD). (NCT01203020)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Acute GVHDChronic GVHD
Allogeneic Hematopoietic Progenitor Cell Transplant53

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Non-Relapse Mortality (NRM) Following RTC Transplantation at 1 Year.

Percentage of participants NRM following RTC transplantation at 1-year. (NCT01203020)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant17.1

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Non-Relapse Mortality (NRM) Following RTC Transplantation at 2 Years.

Percentage of participants NRM following RTC transplantation at 2-year. (NCT01203020)
Timeframe: At 2 years

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant24.6

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Relapse Rate (RR) Following Transplantation at 2-year.

Percentage of participants Relapse Rates (RR) following transplantation at 2-year. (NCT01203020)
Timeframe: At 2 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant39.7

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Relapse Rate (RR) Following Transplantation at 1-year.

Percentage of participants Relapse Rates (RR) following transplantation at 1-year. (NCT01203020)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant28.7

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Percentage of 1 Year Overall Survival (OS)

Percentage of participants--1 year overall survival (OS) following transplantation. (NCT01203020)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant63.6

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Percentage of 2-year Progression Free Survival (PFS)

Percentage of 2-year progression free survival (PFS) of patients with chemotherapy refractory Hodgkin's and non-Hodgkin's lymphoma (NHL) undergoing reduced-toxicity conditioning (RTC) with once daily intravenous Busulfex and fludarabine. (NCT01203020)
Timeframe: At 2 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant45.5

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Percentage of 2 Year Overall Survival (OS)

Percentage of participants-- 2 Year Overall Survival (OS) post transplant (NCT01203020)
Timeframe: At 2nd year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant59.1

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Percentage of 1-year Progression Free Survival (PFS)

Percentage of 1-year progression free survival (PFS) of patients with chemotherapy refractory Hodgkin's and non-Hodgkin's lymphoma (NHL) undergoing reduced-toxicity conditioning (RTC) with once daily intravenous Busulfex and fludarabine. (NCT01203020)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant59.1

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Number of Participants With a Response to Therapy

Response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment starts or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT01218867)
Timeframe: 5 years

,,,,,,,,,,
InterventionParticipants (Count of Participants)
Complete Response (CR)Partial Response (PR)Progressive Disease (PD)Stable Disease (SD)
Cohort 1 - 1x10(6) Cells (High Dose IL-2)0010
Cohort 10 - 1x10(10) Cells (Low Dose IL-2)0130
Cohort 11 - 3x10(10) Cells (Low Dose IL-2)0020
Cohort 2 - 3x10(6) Cells (High Dose IL-2)0010
Cohort 3 - 1x10(7) Cells (High Dose IL-2)0030
Cohort 4 - 3x10(7) Cells (High Dose IL-2)0010
Cohort 5 - 1x10(8) Cells (High Dose IL-2)0010
Cohort 6 - 3x10(8) Cells (High Dose IL-2)0010
Cohort 7 - 1x10(9) Cells (High Dose IL-2)0031
Cohort 8 - 1x10(9) Cells (Low Dose IL-2)0030
Cohort 9 - 3x10(9) Cells (Low Dose IL-2)0030

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Number of Participants With Serious and Non-Serious Adverse Events

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01218867)
Timeframe: Date treatment consent signed to date off study, approximately, 33 months and 25 days

InterventionParticipants (Count of Participants)
Cohort 1 - 1 x 10(6) Cells (High Dose IL-2)1
Cohort 2 - 3 x 10(6) Cells (High Dose IL-2)1
Cohort 3 - 1 x 10(7) Cells (High Dose IL-2)3
Cohort 4 - 3 x 10(7) Cells (High Dose IL-2)1
Cohort 5 - 1 x 10(8) Cells (High Dose IL-2)1
Cohort 6 - 3 x 10(8) Cells (High Dose IL-2)1
Cohort 7 - 1 x 10(9) Cells (High Dose IL-2)4
Cohort 8 - 1 x 10(9) Cells (Low Dose IL-2)3
Cohort 9 - 3 x 10(9) Cells (Low Dose IL-2)3
Cohort 10 - 1 x 10(10) Cells (Low Dose IL-2)3
Cohort 11 - 3 x 10(10) Cells (Low Dose IL-2)2

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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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Maximum Tolerated Dose (MTD)

The MTD was determined by evaluating dose limiting toxicities (DLT) of participants that received increasing doses of intravenous infusion of IL-12 gene transduced tumor infiltrating lymphocytes (TIL) (i.e., 1x10^6, 3x10^6, 3x10^7, 1x10^7, 3x10^7, 1x10^8, 3x10^8, 1x10^9, and 3x10^9) in cohorts 1-10. Maximum tolerated cell dose is the highest dose at which NCT01236573)
Timeframe: 4 years

InterventionCells (Number)
All Phase I Participants1,000,000,000

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT01236573)
Timeframe: 49 months and 20 days

Interventionparticipants (Number)
Group 1 - CD8 + TIL Expressing IL-12 1x10^6 (Phase 1)1
Group 2 - CD8 + TIL Expressing IL-12 3x10^6 (Phase 1)1
Group 3 - CD8 + TIL Expressing IL-12 1x10^7 (Phase 1)6
Group 4 - CD8 + TIL Expressing IL-12 3x10^7 (Phase 1)1
Group 5 - Bulk TIL Expressing IL-12 1x10^7 (Phase 1)1
Group 6 - Bulk TIL Expressing IL-12 3x10^7 (Phase 1)4
Group 7- Bulk TIL Expressing IL-12 1x10^8 (Phase 1)3
Group 8 - Bulk TIL Expressing IL-12 3x10^8 (Phase 1)3
Group 9 - Bulk TIL Expressing IL-12 1x10^9 (Phase 1)4
Group 10 - Bulk TIL Expressing IL12 3x10^9 (Phase 1)4
Group 11 - Bulk TIL Expressing MTD 1x10^9 (Phase 2)6

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Response (Complete Response (CR) + Partial Response (PR)) to Therapy

Response was determined by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0. Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline um LD. Progressive disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more lesions. (NCT01236573)
Timeframe: 4 years

,,,,,,,,,,
Interventionparticipants (Number)
Complete Response (CR)Partial Response (PR)Progressive Disease (PD)Not Assessed (NA)Not Evaluable (NE)
Group 1 - CD8 + TIL Expressing IL-12 1x10^6 (Phase 1)00100
Group 10 - Bulk TIL Expressing IL12 3x10^9 (Phase 1)13000
Group 11 - Bulk TIL Expressing MTD 1x10^9 (Phase 2)01311
Group 2 - CD8 + TIL Expressing IL-12 3x10^6 (Phase 1)00100
Group 3 - CD8 + TIL Expressing IL-12 1x10^7 (Phase 1)00600
Group 4 - CD8 + TIL Expressing IL-12 3x10^7 (Phase 1)00010
Group 5 - Bulk TIL Expressing IL-12 1x10^7 (Phase 1)00100
Group 6 - Bulk TIL Expressing IL-12 3x10^7 (Phase 1)00400
Group 7- Bulk TIL Expressing IL-12 1x10^8 (Phase 1)00210
Group 8 - Bulk TIL Expressing IL-12 3x10^8 (Phase 1)00210
Group 9 - Bulk TIL Expressing IL-12 1x10^9 (Phase 1)01210

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Number of Participants With Chronic GvHD

Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3. (NCT01247701)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant1

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Number of Participants With Platelet Engraftment

Achievement of untransfused platelet count > 20 x 10^9/L on three consecutive days (NCT01247701)
Timeframe: Day 180

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant13

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Number of Participants With Severe Acute GVHD Grade III-IV

Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD. (NCT01247701)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant2

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Number of Participants With Donor Engraftment After Transplant.

To evaluate donor engraftment at 100 days, 6, 9, 12, 24, and 36 months after transplant. (NCT01247701)
Timeframe: 100 days, 6, 9, 12, 24 and 36 months

InterventionParticipants (Count of Participants)
100 days6 months9 months12 months24 months36 months
Umbilical Cord Blood Transplant1166663

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Number of Participants With Relapse Rate After Transplant

To assess relapse rate at 1 and 3 years after transplant. Cumulative incidence of relapse was calculated from the date of umbilical cord blood transplant using the competing risk method as described in Gray(1988) with death prior to relapse as the competing risk. Participants still alive without a date of relapse were censored at the time of the last follow-up. (NCT01247701)
Timeframe: 1 and 3 years

Interventionpercentage of participants (Number)
1 year3 year
Umbilical Cord Blood Transplant53.353.3

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Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT01247701)
Timeframe: 100 days, 1 year, and 3 years

Interventionprobability of overall survival (Number)
100 days1-Year3-Year
Umbilical Cord Blood Transplant0.9230.9230.923

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Number of Participants With Neutrophil Engraftment

Achievement of absolute neutrophil count > 0.5 x 10^9/L on three consecutive days (NCT01247701)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant15

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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 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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Disease Free Survival (DFS)

Estimated using the Kaplan-Meier estimator. Proportions will be estimated using point as well as interval estimators. All interval estimators will be constructed using the finite sample size sampling distribution at the unadjusted two-sided level of 0.05. (NCT01256398)
Timeframe: 10 years

InterventionMonths (Median)
Treatment (Chemotherapy, Transplant)29.7

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Probability of Being BCR-ABL Negative in the Bone Marrow and Peripheral Blood at the Completion of the CNS Prophylaxis Course (Restricted to Those Patients Achieving a CR)

Proportions will be estimated based on the combined and individual cohorts. (NCT01256398)
Timeframe: 10 years

Interventionproportion of participants (Number)
Treatment (Chemotherapy, Transplant)0.667

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Disease Free Survival Defined From the Date of First Induction Complete Response (CR) to Relapse or Death Due to Any Cause

Estimated using the Kaplan-Meier estimator. Proportions will be estimated using point as well as interval estimators. All interval estimators will be constructed using the finite sample size sampling distribution at the unadjusted two-sided level of 0.05. (NCT01256398)
Timeframe: At 3 years after CR

Interventionpercentage of patients (Number)
Treatment (Chemotherapy, Transplant)52.6

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Overall Survival (OS)

Estimated using the Kaplan-Meier estimator. Proportions will be estimated using point as well as interval estimators. All interval estimators will be constructed using the finite sample size sampling distribution at the unadjusted two-sided level of 0.05. (NCT01256398)
Timeframe: 10 years

InterventionMonths (Median)
Treatment (Chemotherapy, Transplant)55.9

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Feasibility of Maintenance Therapy in This Patient Population (Restricted to Those Patients Achieving a CR). Feasibility Will be Defined as the Number of Deaths Ocuring.

Proportions will be estimated based on the combined and individual cohorts. (NCT01256398)
Timeframe: 10 years

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Transplant)5

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Response

Proportion of patients reaching a CR. A CR requires the following: an absolute neutrophil count (segs and bands) >1000/μL, no circulating blasts, platelets >100,000/μL; adequate bone marrow cellularity with trilineage hematopoiesis, and <5% marrow leukemia blast cells. All previous extramedullary manifestations of disease must be absent (e.g., lymphadenopathy, splenomegaly, skin or gum infiltration, testicular masses, or CNS involvement). (NCT01256398)
Timeframe: 10 years

Interventionproportion of participants (Number)
Treatment (Chemotherapy, Transplant).9846

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Quality of Life (QoL): Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Questionnaire

The FACIT-F questionnaire consists of 13 questions with a total score range of 0 to 52 with 0 indicating a better outcome and 52 indicating a worse outcome. (NCT01263704)
Timeframe: [Visit 1 (Screening, Week 0), at Visits 11 (Week 45) and 14 (Week 80) and at the end of the study (Month 42)]

Interventionscore on a scale (Mean)
Visit 1 (Screening, Week 0)Visit 11 (Week 45)Visit 14 (Week 80)End of the Study (Month 42)
Rituximab Plus Fludarabine and Cyclophosphamide36.139.440.247.0

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Hospitalization Days

(NCT01263704)
Timeframe: Up to 53 months

Interventiondays (Median)
Rituximab Plus Fludarabine and Cyclophosphamide8

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Overall Response Rate

Overall response rate was defined as the percentage of participants with a complete response (CR) or a partial response (PR) according to National Cancer Institute - Working Group [NCI-WG] guidelines. CR: no clonal B lymphocytes in peripheral blood, no significant lymphadenopathy, liver and spleen normal size, no disease symptoms, blood counts: absolute neutrophil count (ANC) >1,500/microliter (mcL), platelets > 100,000/mcL, hemoglobin > 11.0 grams/deciliter (g/dL), normocellular bone marrow. PR: >/= 50% decrease in clonal B lymphocyte count, >/= 50% reduction in lymphadenopathy, >/= 50% reduction of liver or spleen enlargement and ANC >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 11.0 g/dL OR >/= 50% increase in ANC, platelets or hemoglobin. (NCT01263704)
Timeframe: Up to 42 months

Interventionpercentage of participants (Number)
Rituximab Plus Fludarabine and Cyclophosphamide67.5

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Progression-free Survival (PFS)

PFS was defined as the interval from the first study drug treatment day to the first sign of disease progression according to NCI-WG guidelines. Progressive disease (PD): Any new lesion, any disease symptoms, >/=50% increase in lymphadenopathy, splenomegaly, hepatomegaly, >/= 50% increase in the number of circulating clonal B lymphocytes, decrease of hemoglobin levels by > 2.0 g/dL, >/= 50% decrease of platelet counts, increase of lymphocytes in bone marrow to more than 30% from normal. (NCT01263704)
Timeframe: Up to 53 months

Interventionmonths (Median)
Rituximab Plus Fludarabine and Cyclophosphamide36.1

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Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

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. Preexisting conditions which worsen during a study are also considered as adverse events. An SAE is any experience that suggests a significant hazard, contraindication, side effect, or precaution, and fulfills any of the following criteria: fatal (resulted in death), life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was medically significant or required intervention to prevent any of the other outcomes listed here. (NCT01263704)
Timeframe: Up to 53 months

Interventionpercentage of participants (Number)
AEsSAEs
Rituximab Plus Fludarabine and Cyclophosphamide97.645.2

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Event-free Survival

Event-free survival was defined as the time period from the first day of study treatment to occurrence of any of the following events: appearance of disease progression or relapse; prescription of a new treatment for disease relapse; death caused by B-cell chronic lymphocytic leukemia (B-CLL); or complications from B-CLL or therapy. Relapse was defined as disease progression in participants with complete or partial remission lasting at least 6 months after treatment completion. Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. Complete remission was defined as the disappearance of all signs of disease. Partial remission was defined as a reduction in tumor size by >50%. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
Rituximab + Fludarabine + Cyclophosphamide1567

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Duration of Response

Duration of Response was defined as the time period from the last day of study treatment to the day when disease progression occurred in participants who previously had complete or partial remission. Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. Complete remission was defined as the disappearance of all signs of disease. Partial remission was defined as a reduction in tumor size by >50%. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
Rituximab + Fludarabine + CyclophosphamideNA

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Percentage of Participants With Phenotypic Remission

Phenotypic remission was considered achieved if a participant had a negative test for minimal residual disease. A negative test for minimal residual disease was defined as tumor cells ≤0.01% of the total number of peripheral leukocytes. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide66.7

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Progression-free Survival

Progression-free survival was defined as the time period from the first day of study treatment to the day when disease progression occurred. Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
Rituximab + Fludarabine + CyclophosphamideNA

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Percentage of Participants With Partial Remission

Partial remission was defined as a reduction in tumor size by >50%. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide41.1

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Percentage of Participants With Disease Progression

Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide5.5

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Percentage of Participants With Complete Remission

Complete remission was defined as the disappearance of all signs of disease. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide49.3

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Percentage of Participants With Stable Disease

Stable disease was defined as not meeting the criteria for partial remission or disease progression (NCT01271010)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide4.1

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Overall Survival

Overall survival was defined as the time period from the first day of study treatment to participant death. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
Rituximab + Fludarabine + CyclophosphamideNA

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Percentage of Participants With Adverse Events (AEs) and Serious AEs

An AE was defined as any unfavorable medical occurrence in a participant receiving a study drug, regardless of relationship the study drug. An AE was considered serious if it met any of the following criteria: was fatal or life-threatening; required hospitalization or prolonged hospitalization; led to persistent or significant disability/incapacity; was a congenital anomaly/birth defect; was clinically significant and/or required an intervention to prevent any of the listed criteria. (NCT01271010)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
Non-serious AEsSerious AEs
Rituximab + Fludarabine + Cyclophosphamide78.6513.48

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Clinical Response

Clinical response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Complete response (CR) is a disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progression disease (PD) is at least a 20 % increase in the sum of the LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT01271907)
Timeframe: 7 months

InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseProgressive DiseaseStable Disease
Cohort 00120

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Safety of Drosophila Generated PBL Administered in Combination With a Lymphodepleting Preparative Regimen and Supportive Systemic Aldesleukin

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT01271907)
Timeframe: 7 months

InterventionParticipants (Count of Participants)
Cohort 03

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Toxicity Profile

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT01273181)
Timeframe: 2 years

InterventionParticipants (Number)
Phase I: Anti-MAGE A3/12 TCR PBL 5x10e93
Phase I:Anti-MAGE A3/12 TCR PBL 3x10e103
Phase II:Anti-MAGE A3/12 TCR PBL MTD+HD IL-2, Melanoma, RCC2
Phase II: Anti-MAGE A3/12 TCR PBL MTD +HD IL-2, Other Cancer1

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Clinical Tumor Regression (Complete Response (CR) + Partial Response (PR)) in Patients With Metastatic Cancer

Tumor regression response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is a disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. (NCT01273181)
Timeframe: 2 years

,,,
InterventionParticipants (Number)
Complete ResponsePartial Response
Anti-MAGE TCR PBL +HD IL-2, Other00
Anti-MAGE TCR PBL 5x10e10 +HD IL-202
Anti-MAGE TCR PBL 5x10e9 +HD IL-211
Anti-MAGE TCR PBL+HD IL-2, Mel, RCC00

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Duration of Response

Duration of Response was defined as the time period from the last day of study treatment to the day when disease progression occurred in participants who previously had complete or partial remission. Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. Complete remission was defined as the disappearance of all signs of disease. Partial remission was defined as a reduction in tumor size by >50%. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
FCR-liteNA
LR TherapyNA

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Event-free Survival

Event-free survival was defined as the time period from the first day of study treatment to occurrence of any of the following events: appearance of disease progression or relapse; prescription of a new treatment for disease relapse; death caused by B-cell chronic lymphocytic leukemia (B-CLL); or complications from B-CLL or therapy. Relapse was defined as disease progression in participants with complete or partial remission lasting at least 6 months after treatment completion. Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. Complete remission was defined as the disappearance of all signs of disease. Partial remission was defined as a reduction in tumor size by >50%. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
FCR-lite679
LR TherapyNA

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Overall Survival

Overall survival was defined as the time period from the first day of study treatment to participant death. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
FCR-liteNA
LR TherapyNA

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Percentage of Participants With Complete Remission

Complete remission was defined as the disappearance of all signs of disease. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
FCR-lite42.9
LR Therapy18.8

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Percentage of Participants With Disease Progression

Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
FCR-lite0
LR Therapy6.3

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Percentage of Participants With Partial Remission

Partial remission was defined as a reduction in tumor size by >50%. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
FCR-lite42.9
LR Therapy56.3

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Percentage of Participants With Phenotypic Remission

Phenotypic remission was considered achieved if a participant had a negative test for minimal residual disease. A negative test for minimal residual disease was defined as tumor cells ≤0.01% of the total number of peripheral leukocytes. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
FCR-lite25.0
LR Therapy30.0

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Percentage of Participants With Stable Disease

Stable disease was defined as not meeting the criteria for partial remission or disease progression (NCT01283386)
Timeframe: Up to approximately 5 years

Interventionpercentage of participants (Number)
FCR-lite14.3
LR Therapy18.8

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Progression-free Survival

Progression-free survival was defined as the time period from the first day of study treatment to the day when disease progression occurred. Disease progression was defined as an increase in lymphocytosis, or enlargement of the lymph nodes or spleen. (NCT01283386)
Timeframe: Up to approximately 5 years

Interventiondays (Median)
FCR-liteNA
LR TherapyNA

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Percentage of Participants With Adverse Events (AEs) and Serious AEs

An AE was defined as any unfavorable medical occurrence in a participant receiving a study drug, regardless of relationship the study drug. An AE was considered serious if it met any of the following criteria: was fatal or life-threatening; required hospitalization or prolonged hospitalization; led to persistent or significant disability/incapacity; was a congenital anomaly/birth defect; was clinically significant and/or required an intervention to prevent any of the listed criteria. (NCT01283386)
Timeframe: Up to approximately 5 years

,
Interventionpercentage of participants (Number)
Non-serious AEsSerious AEs
FCR-lite80.0020.00
LR Therapy56.2518.75

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Overall Survival

Time from date of treatment start until date of death due to any cause or last Follow-up. (NCT01289457)
Timeframe: up to 2 years

InterventionMonths (Median)
Group 1 CIA14.5
Group 2 FLAI15.1

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Response Rates of Clofarabine, Idarubicin, and Cytarabine (CIA) Versus Fludarabine, Idarubicin, and Cytarabine (FLAI)

NCI & Myelodysplastic syndromes (MDS) International Working Group (IWG) Definitions: Complete Response (CR): Neutrophil count ≥1.0 ×10^9/L, Platelet count ≥100 ×10^9/L, Bone marrow aspirate 10% after initial response. Response assessed Day 28 of every 2-3 cycles during treatment. (NCT01289457)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
Group 1 CIA107
Group 2 FLAI76

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Event-Free Survival (EFS) at 2 Years

Comparison of the event-free survival (EFS) between treatment CIA and FLAI, where an event is defined to be resistance to treatment, relapse (after response) or death, whichever occurred first. (NCT01289457)
Timeframe: Up to 2 years or until relapse/death

InterventionMonths (Median)
Group 1 CIA7.1
Group 2 FLAI8.4

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Maximum Tolerated Dose (MTD) of Clofarabine, Idarubicin, and Cytarabine

MTD is highest dose level in which <2 patients of 6 develop first cycle dose limiting toxicities (DLT). Toxicity defined as any treatment-related grade 3 or greater non-hematological toxicities. (NCT01289457)
Timeframe: 28 days

Interventionmg/m^2 (Number)
Clofarabine + Idarubicin + Cytarabine15

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Part 2: Percentage of Participants With Total B-Cell Depletion by Visit

Total B-cell depletion (normal B-cell plus Malignant B-cell depletion) was defined for each individual participant when the sum of CD5-/CD19+ (normal B-cells) and CD5+/CD19+ (malignant B-cells) cell counts decreased below 80 cells/μL. (NCT01292603)
Timeframe: Cycle 1 Pre-dose, 60 minutes post-dose, Days 2 and 3 in Cycle 2, day 1 in Cycles 3, 4, 5 and 6, Follow-up Days 28 and 56, and Follow-up Visits at Months 3, 6, 9, 12, 15, and 18 and Withdrawal Visit

,
Interventionpercentage of participants (Number)
Cycle 1 - Baseline (n=80,80))Cycle 2 - Pre-dose (n=71, 74)Cycle 2 - Post-dose (n=65, 66)Cycle 2 Day 2 (n=54, 59)Cycle 2 Day 3 (n=48, 54)Cycle 3 Day 1 (n=67,68)Cycle 4 Day 1 (n=71,69)Cycle 5 Day 1 (n=68,67)Cycle 6 Day 1 (n=71,64)FU 28 Day Visit (n=66,64)FU 56 Day Visit (n=63,67)FU 3 Month Visit (n=67,67)FU 6 Month Visit (n=60,69)FU 9 Month Visit (n=65,64)FU 12 Month Visit (n=60,61)FU 15 Month Visit (n=59,60)FU 18 Month Visit (n=57,58)FU 21 Month Visit (n=55,52)FU 24 Month Visit (n=56,51)Withdrawn/Termination (n=17,15)
Part 2 : Rituximab IV 500 mg/m^20.023.932.335.250.073.183.188.295.895.592.195.588.366.243.333.924.614.510.741.2
Part 2: Rituximab SC 1600 mg0.031.137.930.537.076.584.189.695.396.997.092.591.370.342.630.025.919.215.773.3

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Part 1: Total Cluster Differentiation19 Positive (CD19+) B-Cell Counts by Visit

CD 19 is a surface antigen (protein) present on B-lymphocytes. (NCT01292603)
Timeframe: Day 1 of Cycles 5 and 6 and Follow-up Days 28 and 56 and Follow-up Visits at Months 3, 6, 9, 12, 15,18, 21 and 24

,,,,
Interventioncells per microliter (cells/μL) (Median)
Cycle 5 Day 1 (n=15,13,19,1,2)Cycle 6 Day 1 (n=15,14,18,1,0)FU 28 Day Visit (n=15,11,19,1,0)FU 56 Day Visit (n=15,9,15,1,0)FU 3 Month Visit (n=16,8,18,0,0)FU 6 Month Visit (n=15,13,17,1,0)FU 9 Month Visit (n=15,13,15,1,0)FU 12 Month Visit (n=14,14,20,0,0)FU 15 Month Visit (n=14,11,15,0,0)FU 18 Month Visit (n=13,14,15,0,0)FU 21 Month Visit (n=13,12,16,0,0)FU 24 Month Visit(n=12, 4,16,0,0)
Part 1: No SC Dose Received2NANANANANANANANANANANA
Part 1: Rituximab SC 1000 mg8427714NA5174NANANANANA
Part 1: Rituximab SC 1400 Milligrams (mg)21213266126175175128238
Part 1: Rituximab SC 1600 mg3223111931418278110
Part 1: Rituximab SC 1870 mg0100122990106189149232

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Part 2: Time to Cmax (Tmax) of Rituximab at Cycle 6

Multiple blood samples were obtained at pre-dose, post-dose and on Days 2, 3, 8 ,15, 29 of Cycle 6 in Rituximab IV arm, and at pre-dose, and on Days 2, 3, 8 ,15, 29 of Cycle 6 in Rituximab SC arm and time to peak plasma concentration of rituximab was determined. (NCT01292603)
Timeframe: Rituximab IV arm: pre-dose, post-dose and on Days 2, 3, 8 ,15, 29 of Cycle 6; Rituximab SC arm: pre-dose, and on Days 2, 3, 8 ,15, 29 of Cycle 6

Interventiondays (Geometric Mean)
Part 2 : Rituximab IV 500 mg/m^20.22
Part 2: Rituximab SC 1600 mg3.14

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Part 1: Percentage of Participants and Nurses Recording a Preference For Either SC or IV Administration

In part 1 of the trial, upon completion of dosing in cycle 6, participants and their treating nurses were asked whether they have a preference of dosing route, IV vs SC (NCT01292603)
Timeframe: Days 4 to 5 in Cycle 6

,,
Interventionpercentage of participants or nurses (Number)
Participants who preferred SCParticipants who preferred IVNurses who preferred SCNurses who preferred IV
Part 1: Rituximab SC 1400 mg88.013.088.013.0
Part 1: Rituximab SC 1600 mg100.00.0100.00.0
Part 1: Rituximab SC 1870 mg91.09.091.09.0

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Part 1: Percentage of Participants With Anti-Rituximab Antibodies

Blood samples for the assessment of antibodies against rituximab (HACAs) were drawn pre-dose at Cycle 5 and Cycle 6 in Part 1 and at each follow up visit until 24 months after the last dose. (NCT01292603)
Timeframe: Predose at Cycles 5 and 6 and at each follow up visit until 24 months after the last dose

Interventionpercentage of participants (Number)
Pre-Dose Cycle 5: positive for HACAs (n=59)Pre-Dose Cycle 5: negative for HACAs (n=59)Post-Dose: positive for HACAs (n=61)Post-Dose: negative for HACAs (n=61)
Part 1: Rituximab SC0.0100.05.095.1

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Part 1: Percentage of Participants With Total B-Cell Depletion by Visit

Total B-cell depletion (normal B-cell plus Malignant B-cell depletion) was defined for each individual participant when the sum of CD5-/CD19+ (normal B-cells) and CD5+/CD19+ (malignant B-cells) cell counts decreased below 80 cells/μL. (NCT01292603)
Timeframe: Day 1 pre-dose of Cycles 5 and 6 and Follow-up Days 28 and 56 and Follow-up Visits at Months 3, 6, 9, 12, 15, 18, 21 and 24

,,,,
Interventionpercentage of participants (Number)
Cycle 5 Day 1 (n=15,1319,1,2)Cycle 6 Day 1(n= 15,14,18,1,0)FU 28 Day Visit (n=15,11,19,1,0)FU 56 Day Visit (n=15,9,15,1,0)FU 3 Month Visit (n=16,8,18,0,0)FU 6 Month Visit (n=15,13,17,1,0)FU 9 Month Visit (n=15,13,15,1,0)FU 12 Month Visit (n=14,14,20,0,0)FU 15 Month Visit (n=14,11,15,0,0)FU 18 Month Visit (n=13,14,15,0,0)FU 21 Month Visit (n=13,12,16,0,0)FU 24 Month Visit (n=12,14,16,0,0)
Part 1: No SC Dose Received100.0NANANANANANANANANANANA
Part 1: Rituximab SC 1400 mg93.393.393.393.393.886.753.328.635.730.830.825.0
Part 1: Rituximab SC 1600 mg92.3100.0100.0100.0100.092.384.685.754.550.050.021.4
Part 1: Rituximab SC 1870 mg100.0100.0100.0100.0100.094.160.045.040.026.718.818.8
Rituximab SC 1000 mg0.0100.0100.0100.0NA100.0100.0NANANANANA

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Part 2: Terminal Half-Life of Rituximab at Cycle 6

The terminal half-life (t1/2) of rituximab is defined as the time required for the plasma concentration of rituximab to reach half of its original concentration. (NCT01292603)
Timeframe: Rituximab IV arm: pre-dose, post-dose and on Days 2, 3, 8 ,15, 29 of Cycle 6; Rituximab SC arm: pre-dose, and on Days 2, 3, 8 ,15, 29 of Cycle 6

Interventiondays (Geometric Mean)
Part 2 : Rituximab IV 500 mg/m^230.09
Part 2: Rituximab SC 1600 mg30.71

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Part 1: Subcutaneous Rituximab Dose Resulting in Trough Concentration (Ctrough) Levels Non-Inferior to Intravenous Rituximab

Ctrough is defined as the trough or minimum serum concentration. Pharmacokinetic parameters for rituximab were assessed during Cycles 5 (IV rituximab) and 6 (SC rituximab). Rituximab pharmacokinetic (PK) data from Part 1 were integrated into a population PK model using parametric, nonlinear, mixed-effects modelling. Rituximab IV 500 mg/m^2 administered once every 4 weeks was compared to fixed doses of rituximab SC between 1400 mg and 1870 mg. The dose selection was performed on Ctrough concentrations at Cycle 5 (pre-dose Cycle 6). A test of the probability of success was applied to each of the 100 replicates, and the percentage of replicates with a positive test corresponded to the probability of success of the trial. (NCT01292603)
Timeframe: Pre-dose and post-dose (15 minutes to end of infusion) on Day 1 and on Days 2, 5, 11 and 15 of Cycle 5 and Pre-dose, Post-dose on Days 2, 3, 5,11, 15 and 29 of Cycle 6; Pre-dose was taken 2 hours prior rituximab dose

Interventionmg (Number)
Part 1: Rituximab SC1600

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Part 2: Physician/Nurse Opinion on Time Savings With Rituximab SC Compared With Rituximab IV

"Physicians and nurses who administered rituximab were asked to answer the following question: If used in routine practice, on average, how much staff time could be saved with each administration of rituximab SC as compared to rituximab IV? (Please do not consider the time needed for the first IV administration, consider only the subsequent ones). The number of nurses that responded for both the IV and SC arms was 70. The number of physicians that responded for the IV and SC arms were 78 and 81 respectively." (NCT01292603)
Timeframe: Days 4-5 in Cycle 6

,
Interventionpercentage of participants in the survey (Number)
Nurse: 4 or more hoursNurse: At least 3 hours but less than 4 hoursNurse: At least 2 hours but less than 3 hoursNurse: At least 1 hour but less than 2 hoursNurse: Less than 1 hourPhysician: 4 or more hoursPhysician: At least 3 hours but less than 4 hoursPhysician: At least 2 hours but less than 3 hoursPhysician: At least 1 hour but less than 2 hoursPhysician: Less than 1 hour
Part 2 : Rituximab IV 500 mg/m^221.023.026.017.013.021.018.024.022.010.0
Part 2: Rituximab SC 1600 mg21.029.023.011.016.022.021.026.019.07.0

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Part 2: Total CD19+ B-Cell Counts by Visit

CD 19 is a surface antigen (protein) present on B-lymphocytes. (NCT01292603)
Timeframe: Cycle 1 pre-dose, 60 minutes post-dose, Days 2 and 3 in Cycle 2, day 1 pre-dose in Cycles 3, 4, 5 and 6, Follow-up Days 28 and 56, and Follow-up Visits at Months 3, 6, 9, 12, 15, and 18 and Withdrawal Visit

,
Interventioncells/μL (Median)
Cycle 1 - Baseline (n=80,80)Cycle 2 - Pre-dose (n=71,74)Cycle 2 - Post-dose (n=65, 66)Cycle 2 Day 2 (n=54, 59)Cycle 2 Day 3 (n=48, 54)Cycle 3 Day 1 (n=67,68)Cycle 4 Day 1 (n=71,69)Cycle 5 Day 1 (n=68,67)Cycle 6 Day 1 (n=71,64)FU 28 Day Visit (n=66,64)FU 56 Day Visit (n=63,67)FU 3 Month Visit (n=67,67)FU 6 Month Visit (n=60,69)FU 9 Month Visit (n=65,64)FU 12 Month Visit (n=60,61)FU 15 Month Visit (n=59,60)FU 18 Month Visit (n=57,58)FU 21 Month Visit (n=55,52)FU 24 Month Visit (n=56,51)Withdrawn/Termination (n=17,15)
Part 2 : Rituximab IV 500 mg/m^268905338163154871242322233591135134171214150
Part 2: Rituximab SC 1600 mg5056525316826612584232223301041712232772566

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Part 2: Percentage of Participants With Anti-Rituximab Antibodies

In Part 2, samples for the HACA assay were collected at each treatment cycle prior to the administration of rituximab and at each follow-up visit until 24 months after the last dose of rituximab. (NCT01292603)
Timeframe: Day 0 of Cycle 1 and Day 1 of Cycles 1, 2, 3, 4, 5, and 6 and at each follow-up visit until 24 months after the last dose of rituximab.

,
Interventionpercentage of participants (Number)
Baseline pre Cycle 1: positive for HACAs (n=87,85)Baseline pre Cycle 1: negative for HACAs (n=87,85)Post-Baseline: positive for HACAs (n=89,85)Post-Baseline: negative for HACAs (n=89,85)
Part 2 : Rituximab IV 500 mg/m^20.0100.015.085.0
Part 2: Rituximab SC 1600 mg2.497.612.088.0

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Part 2: Physician/Nurse Opinion on Convenience of Rituximab SC Compared With Rituximab IV

"Physicians and nurses who administered rituximab were asked to answer the following question: Which formulation of rituximab (SC or IV) do you think is more convenient? with pre-specified responses as below. Percentage of participants with specified answers were reported. The number of nurses that responded for both the IV and SC arms was 70. The number of physicians that responded for the IV and SC arms were 78 and 81 respectively." (NCT01292603)
Timeframe: Days 4-5 in Cycle 6

,
Interventionpercentage of participants in the survey (Number)
Nurse: Rituximab SC is much more convenientNurse: Rituximab SC is a little more convenientNurse: Both formulations are equally convenientNurse: Rituximab IV is a little more convenientNurse: Rituximab IV is much more convenientPhysician: Rituximab SC is much more convenientPhysician: Rituximab SC a little more convenientPhysician: Both formulations equally convenientPhysician: Rituximab IV a little more convenientPhysician: Rituximab IV is much more convenient
Part 2 : Rituximab IV 500 mg/m^281.07.09.03.00.078.015.06.00.00.0
Part 2: Rituximab SC 1600 mg77.09.04.010.00.080.014.06.00.00.0

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Part 2: Maximum Observed Concentration (Cmax) of Rituximab at Cycle 6

Cmax was obtained directly from the measured concentration-time curves. The concentration-time curve is the result of time points of blood sampling and its measured concentration of rituximab in the blood samplings. (NCT01292603)
Timeframe: Rituximab IV arm: pre-dose, post-dose and on Days 2, 3, 8 ,15, 29 of Cycle 6; Rituximab SC arm: pre-dose, and on Days 2, 3, 8 ,15, 29 of Cycle 6

Interventionμg/mL (Geometric Mean)
Part 2 : Rituximab IV 500 mg/m^2279.78
Part 2: Rituximab SC 1600 mg202.16

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Part 2: Observed Area Under the Serum Concentration-Curve (AUC) of Rituximab at Cycle 6

AUC values were calculated by numerical integration using the linear trapezoidal rule. AUC levels were analyzed using the model below: Ln(AUC) = μ + τi + BlTLij + εij wherein, Ln is the natural log, μ denotes the overall mean effect, τi the effect in each treatment group, BlTLij the tumor load at baseline for each patient and εij a random error variable with normal distribution and mean 0. The treatment effect therein was based on a contrast statement in the model to calculate 90 % confidence intervals for ln(AUC SC)- ln(AUC IV). (NCT01292603)
Timeframe: Rituximab IV arm: pre-dose, post-dose and on Days 2, 3, 8 ,15, 29 of Cycle 6; Rituximab SC arm: pre-dose, and on Days 2, 3, 8 ,15, 29 of Cycle 6

Interventionμg*day/mL (Geometric Mean)
Part 2 : Rituximab IV 500 mg/m^23630.43
Part 2: Rituximab SC 1600 mg4088.78

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Part 2: Rituximab C Trough Levels at Cycle 5

Ctrough is defined as the trough or minimum serum concentration in a given cycle of treatment. The objective of Part 2 was to demonstrate the comparability of the observed Ctrough of rituximab SC 1600 mg and rituximab IV 500 mg/m2 at Cycle 5, as assessed by a non-inferiority test with a lower boundary of at least 0.8 for the 90% CI. (NCT01292603)
Timeframe: +/- 25hours around the 28th day post the 5th Cycle of Rituximab administration

Interventionμg/mL (Geometric Mean)
Part 2 : Rituximab IV 500 mg/m^261.50
Part 2: Rituximab SC 1600 mg97.53

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Number of Participants With Human Anti-Human Antibodies (HAHAs)

(NCT01300247)
Timeframe: Cycle 1 Day 1 (cycle length = 28 days) up to clinical data cutoff date 24 January 2013 (up to approximately 1.75 years)

Interventionparticipants (Number)
Obinutuzumab + Fludarabine + Cyclophosphamide0
Obinutuzumab + Bendamustine0

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Duration of Objective Response (DOR), Assessed by the Investigator According to IWCLL Guidelines

DOR for participants with OR: time from first CR, CRi or PR to disease progression (DP), relapse, or death, assessed by the investigator. DP: >=50% increase in lymphocytes to at least 5x10^9/L;new palpable lymph nodes (>15 millimeters [mm] in longest diameter) or any new extra-nodal lesion; >=50% increase in the longest diameter of any previous site of lymphadenopathy; >=50% increase in the enlargement of the liver and/or spleen; transformation to a more aggressive histology. CR:peripheral blood lymphocytes (PBL) <4x10^9/L; no lymphadenopathy; no hepatomegaly or splenomegaly (below relevant costal margin); no symptoms; bone marrow at least normocellular for age, with <30% of nucleated cells being lymphocytes. PR: >=50% decrease in PBL, >=50% reduction in lymphadenopathy, >=50% reduction of liver and/or spleen enlargement, either neutrophil, platelet, or hemoglobin (Hb) recovery. CRi: met CR criteria, lymphocyte infiltration <30%; may not meet Hb, platelet or neutrophil count recovery. (NCT01300247)
Timeframe: From first documented objective response up to disease progression or relapse or death, whichever occurred first (up to approximately 6 months)

Interventionpercentage of participants (Number)
Obinutuzumab + Fludarabine + CyclophosphamideNA
Obinutuzumab + Bendamustine100.00

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Percentage of Participants Who Had B-Cell Recovery

B-cell recovery was defined as CD19 >=0.07×10^9/L, where participants' CD19 were previously depleted. B-cell recovery was only considered possible when the participant had received the last dose of study treatment. (NCT01300247)
Timeframe: Follow-up at 6 months, 6-12 months and after 12 months up to end of study (up to approximately 4 years)

,
Interventionpercentage of participants (Number)
Follow-up at 6 monthsWithin 6-12 months of follow-upAfter 12 months follow-up
Obinutuzumab + Bendamustine0030.0
Obinutuzumab + Fludarabine + Cyclophosphamide09.542.9

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Percentage of Participants Who Had B-Cell Depletion

B-cell depletion was defined as cluster of differentiation 19 (CD19) <0.07×10^9/L and could occur only after at least one dose of study drug had been administered. (NCT01300247)
Timeframe: Up to the end of the treatment period, and follow-up at 6 months, 6-12 months and after 12 months up to end of study (up to approximately 4 years)

,
Interventionpercentage of participants (Number)
End of the treatment periodFollow-up at 6 monthsWithin 6-12 months of follow-upAfter 12 months follow-up
Obinutuzumab + Bendamustine100.0100.0100.070.0
Obinutuzumab + Fludarabine + Cyclophosphamide90.585.781.047.6

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Percentage of Participants With Objective Response, Assessed According to International Workshop on Chronic Lymphocytic Leukemia (IWCLL) Guidelines

Objective response was defined as a complete response (CR), CR with incomplete marrow recovery (CRi) or partial response (PR), as determined by investigator. CR:required peripheral blood lymphocytes <4x10^9/L; absence of lymphadenopathy; no hepatomegaly or splenomegaly by physical examination as determined by measurement below relevant costal margin; absence of disease/constitutional symptoms; bone marrow at least normocellular for age, with <30% of nucleated cells being lymphocytes. PR:Greater than equal to (>=) 50% decrease in peripheral blood lymphocyte count, >=50% reduction in lymphadenopathy, >=50% reduction of liver and/or spleen enlargement, either neutrophil, platelet, or hemoglobin (Hb) recovery. CRi:met all CR criteria including confirmed lymphocyte infiltration <30%; may not meet Hb, platelet or neutrophil count recovery. The 95% confidence interval (CI) was estimated by Clopper-Pearson method. The end of treatment response visit occurred 2-3 months after end of treatment. (NCT01300247)
Timeframe: Baseline up to relapse or progression or death from any cause, whichever occurred first up to end of treatment response visit (up to approximately 9 months)

Interventionpercentage of participants (Number)
Obinutuzumab + Fludarabine + Cyclophosphamide61.9
Obinutuzumab + Bendamustine90.0

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Percentage of Participants Who Were Alive and Progression Free

Progressive disease assessed using IWCLL: >=50% increase in the absolute number of circulating lymphocytes to at least 5x10^9/L; Appearance of new palpable lymph nodes (>15 millimeters [mm] in longest diameter) or any new extra-nodal lesion; >=50% increase in the longest diameter of any previous site of lymphadenopathy; >=50% increase in the enlargement of the liver and/or spleen; transformation to a more aggressive histology. (NCT01300247)
Timeframe: Baseline up to relapse or progression or death from any cause, whichever occurred first, up to end of study (up to approximately 4 years)

Interventionpercentage of participants (Number)
Obinutuzumab + Fludarabine + Cyclophosphamide90.5
Obinutuzumab + Bendamustine90.0

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Percentage of Participants Who Were Alive

(NCT01300247)
Timeframe: Baseline up to relapse or progression or death from any cause, whichever occurred first, up to end of study (up to approximately 4 years)

Interventionpercentage of participants (Number)
Obinutuzumab + Fludarabine + Cyclophosphamide95.2
Obinutuzumab + Bendamustine95.0

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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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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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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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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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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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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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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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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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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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Number of Participants With Side Effects After Ad/PNP-F-araAMP Treatment

Number of participants who had the most frequently observed undesirable effects after exposure to study drug (NCT01310179)
Timeframe: Entry through Study Day 56

Interventionparticipants (Number)
injection site symptomsfatiguefacial painnausea
Ad/PNP and Fludarabine Monophosphate12885

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Treatment Outcome and Percent Change in Tumor Volume

Measurement of tumor response to study drug, as measured by the percentage of change in tumor volume as measured by a physicial measurement using a ruler (NCT01310179)
Timeframe: Entry through Study Day 56

Interventionparticipants (Number)
Progression (>+20%)Stable (+20% to -30%)Partial Response (>-30%)
Ad/PNP and Fludarabine Monophosphate255

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Percentage of B Cells at One Year Post-transplant in Participants Who Did/Did Not Receive Leuprolide Following Bone Marrow Transplant (BMT)

B cell percentage is defined as the percentage of lymphocytes that are B cells. (NCT01338987)
Timeframe: after first Bone Marrow Transplant, approximately 12 months post-transplant

Interventionpercentage of cells (Median)
Males that received leuprolideMales that did not receive leuprolideFemales who all received leuprolide
Transplant Recipient22.121.914.3

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01338987)
Timeframe: Date treatment consent signed to date off study, approximately 79 months and 11 days.

InterventionParticipants (Count of Participants)
Males That Did Not Receive Leuprolide for 1st Transplant10
Males Randomized to Receive Leuprolide for 1st Transplant8
Females That Received Leuprolide for 1st Transplant20
Matched Related Donors for Transplant4
Recipients of 2nd Transplant2

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Time to Engraftment in First Transplant Recipients Only With Median Thoracic Spine Standardized Uptake Values (SUV) of 1.4 or Greater Than Those Patients With SUV's Less Than 1.4

18F-FLT imaging was performed serially on patients post transplant to identify the level of uptake of 18F-FLT at a day +5 to +12 scan and the day at which neutrophils recover to >500 (i.e., subclinical bone-marrow recovery within 5 days of Bone Marrow Transplantation (BMT infusion)). On each image for each patient, the region of interest was drawn within each thoracic medullary space (n=12), generating the SUV for each space. The mean of these was calculated for each scan. The analysis was the median of the means of the SUV of the thorax values of the day 5-12 scan (averaged the SUV of the thorax for each patient and then took the medians of these). (NCT01338987)
Timeframe: 18F FLT scan done between days +5 to +12 and then time from that scan to engraftment measured

InterventionDays (Median)
Patients with SUV 1.4 or greaterPatients with SUV less than 1.4
Transplant Recipient515

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Number of Participants With Primary Graft Failure

Primary graft failure is defined by lack of neutrophil engraftment. (NCT01339910)
Timeframe: 28 days post-transplant

InterventionParticipants (Count of Participants)
Myeloablative Conditioning Regimen (MAC)1
Reduced Intensity Conditioning (RIC)3

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Number of Participants With Donor Cell Engraftment

Donor cell engraftment will be assessed by donor-recipient chimerism assays. Full donor chimerism is defined as the presence of at least 95% donor cells as a proportion of the total population in the peripheral blood or bone marrow. Graft rejection is defined as the presence of no more than 5% donor cells as a proportion of the total population. Mixed chimerism is defined as the presence of between 5% and 95% donor cells. Mixed or full donor chimerism will be considered evidence of donor engraftment. (NCT01339910)
Timeframe: Days 28 and 100 and 18 months post-transplant

InterventionParticipants (Count of Participants)
Day 2872548419Day 2872548420Day 10072548420Day 1007254841918 Months7254842018 Months72548419
Mixed ChimerismFull Donor ChimerismGraft RejectionDeath Prior to AssessmentUnknown (relapsed or missing assay)
Myeloablative Conditioning Regimen (MAC)86
Reduced Intensity Conditioning (RIC)80
Myeloablative Conditioning Regimen (MAC)9
Reduced Intensity Conditioning (RIC)30
Myeloablative Conditioning Regimen (MAC)1
Myeloablative Conditioning Regimen (MAC)0
Reduced Intensity Conditioning (RIC)0
Myeloablative Conditioning Regimen (MAC)36
Reduced Intensity Conditioning (RIC)22
Myeloablative Conditioning Regimen (MAC)106
Reduced Intensity Conditioning (RIC)86
Myeloablative Conditioning Regimen (MAC)12
Myeloablative Conditioning Regimen (MAC)2
Myeloablative Conditioning Regimen (MAC)6
Reduced Intensity Conditioning (RIC)8
Myeloablative Conditioning Regimen (MAC)71
Reduced Intensity Conditioning (RIC)66
Myeloablative Conditioning Regimen (MAC)4
Reduced Intensity Conditioning (RIC)5
Reduced Intensity Conditioning (RIC)1
Myeloablative Conditioning Regimen (MAC)31
Reduced Intensity Conditioning (RIC)42
Myeloablative Conditioning Regimen (MAC)25
Reduced Intensity Conditioning (RIC)19

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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 3 consecutive measurements on different days. The first of the 3 days will be designated the day of neutrophil engraftment. Platelet engraftment is defined as achieving platelet counts greater than 20,000/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. (NCT01339910)
Timeframe: Days 28 and 60 post-transplant

,
Interventionpercentage (Number)
Neutrophil Engraftment at Day 28Platelet Engraftment at Day 60
Myeloablative Conditioning Regimen (MAC)98.595.5
Reduced Intensity Conditioning (RIC)97.896.2

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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" (NCT01339910)
Timeframe: Day 100 post-transplant

,
Interventionpercentage (Number)
Grade II-IV Acute GVHDGrade III-IV Acute GVHD
Myeloablative Conditioning Regimen (MAC)44.713.6
Reduced Intensity Conditioning (RIC)31.66.8

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Number of Participants With Secondary Graft Failure

Secondary graft failure is defined by initial neutrophil engraftment followed by subsequent decline in neutrophil counts to less than 500x10^6/liter that is unresponsive to growth factor therapy. (NCT01339910)
Timeframe: 18 months post-transplant

InterventionParticipants (Count of Participants)
Myeloablative Conditioning Regimen (MAC)1
Reduced Intensity Conditioning (RIC)4

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Percentage of Participants With Relapse-Free Survival (RFS)

Relapse-free survival is defined as survival without relapse of the primary disease. (NCT01339910)
Timeframe: 18 months post-randomization

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)67.8
Reduced Intensity Conditioning (RIC)47.3

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Percentage of Participants With Overall Survival (OS)

Overall survival is defined as survival of death from any cause. (NCT01339910)
Timeframe: 18 months post-randomization

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)77.5
Reduced Intensity Conditioning (RIC)67.7

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Percentage of Participants With Disease Relapse

Disease Relapse is defined as relapse of the primary disease. (NCT01339910)
Timeframe: 18 months post-randomization

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)13.5
Reduced Intensity Conditioning (RIC)48.3

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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. (NCT01339910)
Timeframe: 18 months post-transplant

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)64.0
Reduced Intensity Conditioning (RIC)47.6

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Time to Maximum Persistence of NY-ESO-1 Genetically Engineered T Cells

Time to maximum persistence is defined as date of maximum persistence for infusion visit minus date of first T cell infusion visit plus 1. Median and full range of time to maximum persistence are presented. (NCT01343043)
Timeframe: Up to 4.5 years

InterventionDays (Median)
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A8.0
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A8.0
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B8.0
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C9

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Duration of Overall Response

Duration of overall response (DOR) is defined as the time from first documented evidence of confirmed CR or confirmed PR until first documented date of disease progression or death due to any cause or surgical resection or start of prohibited medications. Duration of overall response was evaluated per RECIST v1.1. Median and full range of DOR are presented. (NCT01343043)
Timeframe: Up to 4.5 years

InterventionWeeks (Median)
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A31.0
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A8.6
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B32.1
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C16.4

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Number of Participants With at Least One Confirmed Positive Post-Baseline Anti-infused (NY-ESO-1 Genetically Engineered T) Cell Antibody Result

Serum samples were collected for the determination of anti-infused (NY-ESO-1 genetically engineered T) cell antibodies (ATA) using a validated electrochemiluminescent (ECL) immunoassay. The assay involved screening, confirmation and titration steps. If serum samples tested positive in the screening assay, they were considered 'potentially positive' and were further analyzed for the specificity using the confirmation assay. A participant was considered to have a confirmed positive ATA result if they have a positive screening assay result and a positive confirmation assay result. (NCT01343043)
Timeframe: Up to 4.5 years

InterventionParticipants (Count of Participants)
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A0
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A0
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B0
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C0

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Objective Response Rate (ORR)

ORR was calculated as the percentage of participants with a confirmed complete response (CR) or partial response (PR) relative to the total number of participants in the analysis population per response evaluation criteria in solid tumors (RECIST) version (v)1.1 as determined by the local investigators. (NCT01343043)
Timeframe: Up to 4.5 years

InterventionPercentage of Participants (Number)
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A50
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A30.8
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B20
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C26.7

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Best Overall Response

Best overall response is the best response recorded from the start of treatment (first T cell infusion) until disease progression/recurrence. Response categories from best to worst are confirmed CR, confirmed PR, stable disease (SD) and confirmed progressive disease (PD). Best overall response is a stable disease, if CR or PR are unconfirmed. Data for number of participants with CR, PR, SD and PD are presented. (NCT01343043)
Timeframe: Up to 4.5 years

,,,
InterventionParticipants (Count of Participants)
Complete responsePartial responseStable diseaseProgressive diseaseNot evaluable
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A15510
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A04711
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B01301
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C041010

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Overall Survival

Overall survival is defined as the interval between the date of the first T-cell infusion and date of death from any cause. Median and inter-quartile range (first quartile and third quartile) of overall survival are presented. (NCT01343043)
Timeframe: Up to 4.5 years

InterventionWeeks (Median)
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A80.7
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A43.1
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B86.4
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C105.3

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Number of Participants With Non-serious Adverse Events (Non-SAEs) and Serious Adverse Events (SAEs)

An adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. SAE is defined as any untoward medical occurrence that; results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect, other situations judged by physician, is associated with liver injury and impaired liver function. Number of participants who had non-SAEs and SAEs are presented. (NCT01343043)
Timeframe: Up to 5 years

,,,
InterventionParticipants (Count of Participants)
Non-SAESAE
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A159
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A137
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B54
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C156

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Progression Free Survival

Progression free survival is defined as the interval between the date of first T cell infusion and the earliest documented evidence of disease progression or death due to any cause or surgical resection or start of prohibited medications. Progression free survival was evaluated per RECIST v1.1. Median and inter-quartile range (first quartile and third quartile) of progression free survival are presented. (NCT01343043)
Timeframe: Up to 4.5 years

InterventionWeeks (Median)
Cohort 1: High NY-ESO-1 Expression Treated With Regimen A15.4
Cohort 2: Low NY-ESO-1 Expression Treated With Regimen A13.1
Cohort 3: High NY-ESO-1 Expression Treated With Regimen B8.6
Cohort 4: High NY-ESO-1 Expression Treated With Regimen C22.4

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To Compare the Relapse Rate at 1 Year of Patients With Myeloid Malignancies Receiving Each Treatment

(NCT01366612)
Timeframe: 1 year

InterventionPercent (Number)
Group 138.9
Group 218.8

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Number of Participants With Clinical Tumor Regression.

Clinical tumor regression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is a disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progression (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions.Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as reference the smallest sum LD. (NCT01369875)
Timeframe: up to approximately 8 months

,
InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseProgressionStable Disease
ECCE Young TIL0010
Standard Young TIL0010

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Number of Participants With Serious and Non-Serious Adverse Events

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01369875)
Timeframe: Date treatment consent signed to date off study, up to approximately 8 months.

InterventionParticipants (Count of Participants)
Standard Young TIL1
ECCE Young TIL1

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Phase 1: Maximum Tolerated Dose (MTD) of Intravenous Recombinant IL-15 as a Daily Intravenous Bolus for 10 Consecutive Days in Patients With Metastatic Melanoma Who Have Received a Lymphodepleting Chemotherapy and ACT TIL.

Intravenous recombinant IL-15 as a daily intravenous bolus for 10 consecutive days in patients with metastatic melanoma who have received a lymphodepleting chemotherapy and ACT TIL with dose escalation (i.e., dose level 1: 0.25 mcg, dose level 2: 0.50 mcg, dose level 3: 1 mcg, and dose level 4: 2 mcg) to further characterize the safety of the MTD prior to starting the phase 2 portion. (NCT01369888)
Timeframe: 2 years

Interventionmcg/kg/day (Number)
IL-15 Following Young TIL (0.25 mcg)NA
IL-15 Following Young TIL (0.50 mcg)NA

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT01369888)
Timeframe: 8 months, 9 days

Interventionparticipants (Number)
IL-15 Following Young TIL (0.25 mcg)1
IL-15 Following Young TIL (0.50 mcg)2

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Number of Participants With Molecular Complete Remission at 3 Month Post Transplant

Molecular Complete Remission is defined as participant alive and engrafted with molecular complete remission 100 days post transplant where molecular complete response is no BCR-ABL transcripts detected and engraftment is defined as the evidence of donor derived cells (more than 95%) by chimerism studies in the presence of neutrophil recovery by day 28 post stem cell infusion. (NCT01390402)
Timeframe: Baseline to up to 4 months post-transplant

Interventionparticipants (Number)
NK Infusion + Chemotherapy3

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Relapse-free Survival

The percentage of participants that have not died or had disease progression by two years. Relapse is defined by either morphological or cytogenetic evidence of the original malignancy consistent with pre-transplant features. (NCT01408563)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI49

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Rates of Grade II-IV and Grade III-IV Acute Graft Versus Host Disease (GVHD) at 100 Days

"Acute GVHD is assessed using Consensus Criteria:~Organ Classifications:~0: No rash due to GVHD; Bilirubin < 2 mg/dL; < 500 mL diarrhea/ day~1: Maculopapular rash < 25% of body surface; Bilirubin 2-3 mg/dL; 500 to 999 mL diarrhea/ day or persistent nausea with histologic evidence of GVHD in stomach/ duodenum~2: Maculopapular rash 25-50% of body surface; Bilirubin 3.1-6 mg/dL; 1,000 to 1,499 mL diarrhea/ day~3: Maculopapular rash > 50% of body surface; Bilirubin 6.1-15 mg/dL; 1,500 or more mL diarrhea/ day~4: Generalized erythroderma with bullous formation; Bilirubin > 15 mg/dL; Severe abdominal pain with or without ileus~Overall Clinical Grade:~0: No Stage 1-4 of any organ~I: Stage 1-2 rash and no liver or gut involvement~II: Stage 3 rash, or Stage 1 liver involvement, or Stage 1 gut involvement~III: None to Stage 3 skin rash with Stage 2-3 liver involvement, or Stage 2-4 gut involvement~IV: Stage 4 skin rash, or Stage 4 liver involvement" (NCT01408563)
Timeframe: 100 Days

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI16

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Rate of Post-transplant Lymphoma

The number of participants that were found to have lymphoma post-transplant. (NCT01408563)
Timeframe: 2.5 years

InterventionParticipants (Count of Participants)
Fludarabine/Melphalan/TBI0

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Overall Survival

The percentage of participants alive at two years (NCT01408563)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI55

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Median Time to Neutrophil Engraftment

The median number of days measured from the time of transplantation, until the first documented neutrophil engraftment. neutrophil engraftment is defined as the first of 3 consecutive days of absolute neutrophil count > 500 neutrophils per microliter of blood. (NCT01408563)
Timeframe: From the time of transplantation, until the time of neutrophil engraftment, median duration of 24 days

InterventionDays (Median)
Fludarabine/Melphalan/TBI24

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Number of Participants With Primary Graft Failure

Primary graft failure is defined as the failure to achieve an absolute neutrophil count (ANC) >500/ µL by day 42, in the absence of relapse. (NCT01408563)
Timeframe: From the time of transplantation until 42 days post transplantation

InterventionParticipants (Count of Participants)
Fludarabine/Melphalan/TBI8

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Median Time to Platelet Engraftment

The time to platelet engraftment is measured from the time of transplantation until the time of first documented platelet engraftment. Platelet engraftment is defined as a platelet count ≥ 20,000/µL for three consecutive measurements over three or more days. The first of the three days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 3 days or in the following 7 days after the day of engraftment, unless the platelet transfusion is being given specifically to achieve a platelet threshold to allow an elective invasive procedure, such as a central catheter removal. (NCT01408563)
Timeframe: From the time of transplantation, until the time of platelet engraftment, median duration of 52 days

InterventionDays (Median)
Fludarabine/Melphalan/TBI52

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Median Thrombopoietin Levels After Transplant

(NCT01408563)
Timeframe: 30 Days

Interventionpg/mL (Median)
Fludarabine/Melphalan/TBI2500

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Immune Reconstitution - Median CD4 Count at 12 Months

(NCT01408563)
Timeframe: 1 Year

Interventioncells/mm3 (Median)
Fludarabine/Melphalan/TBI362.4

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1 Year Relapse Rate

The percentage of participants that relapsed within 12 months. Relapse is defined by either morphological or cytogenetic evidence of the original malignancy consistent with pre-transplant features. (NCT01408563)
Timeframe: 1 year

Interventionpercentage (Number)
Fludarabine/Melphalan/TBI20

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The Rate of Chronic GVHD

Chronic Graft Versus Host Disease (GVHD) is assessed using the National Institutes of Health (NIH) consensus criteria. (NCT01408563)
Timeframe: From the time of transplantation until the time of chronic GVHD onset, up to 1 year

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI21

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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 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" (NCT01410344)
Timeframe: Day 100 Post-transplant

Interventionpercentage of participants (Number)
Grade II-IV Acute GVHDGrade III-IV Acute GVHD
Allogeneic Transplant41.211.8

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Percentage of Participants With Relapse/Progression

Relapse/Progression is defined as relapse or progression of the primary malignancy. (NCT01410344)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Allogeneic Transplant29.4

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Chimerism

Donor T-cell and myeloid chimerism will be described separately by conditioning regimen intensity (myeloablative or reduced intensity) according to proportions with mixed chimerism (5-95% donor cells out of all), full chimerism (>95% donor cells), or graft rejection (<5% donor cells). (NCT01410344)
Timeframe: Week 4, Day 100, and 6 months Post-transplant

InterventionParticipants (Count of Participants)
Week 472365409Week 472365410Day 10072365410Day 100723654096 Months723654096 Months72365410
Graft RejectionNo Assay ReportedFull ChimerismMixed ChimerismDead at Assessment
Reduced Intensity Allogeneic Transplant4
Myeloablative Allogeneic Transplant1
Myeloablative Allogeneic Transplant3
Myeloablative Allogeneic Transplant4
Reduced Intensity Allogeneic Transplant5
Myeloablative Allogeneic Transplant2
Reduced Intensity Allogeneic Transplant2
Myeloablative Allogeneic Transplant0
Reduced Intensity Allogeneic Transplant0

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Percentage of Participants Recovering Hematologic Function

Recovery of hematologic function is described by the time to neutrophil and platelet recovery. Time to neutrophil recovery will be the first of three consecutive days of > 500 neutrophils/μL following the expected nadir. Time to platelet engraftment will be described by the date when platelet count is > 20,000/μL for the first of three consecutive labs with no platelet transfusions 7 days prior. (NCT01410344)
Timeframe: Days 28 and 100 Post-transplant

Interventionpercentage of participants (Number)
Day 28 Neutrophil RecoveryDay 100 Platelet Recovery
Allogeneic Transplant100.094.1

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Percentage of Participants With Overall Survival

Overall survival is defined as the time from transplant to death from any cause. (NCT01410344)
Timeframe: Six months, 1 Year, and 2 Years Post-transplant

Interventionpercentage of participants (Number)
6 Months1 Year2 Years
Allogeneic Transplant82.458.850.2

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Percentage of Participants With Chronic Graft-Versus-Host Disease (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. (NCT01410344)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Allogeneic Transplant17.6

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Percentage of Participants With Non-Relapse Mortality

The events for non-relapse mortality are death due to any cause other than relapse of the underlying malignancy. (NCT01410344)
Timeframe: Day 100, 1 Year, and 2 Years Post-transplant

Interventionpercentage of participants (Number)
Day 1001 Year2 Years
Allogeneic Transplant0.011.818.3

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Chronic GVHD Requiring Systemic Immunosuppressive Treatment

Chronic GVHD will be defined by National Institutes of Health (NIH) criteria and requiring systemic treatment. A reduction in the cumulative incidence of GVHD from ~35% to ~15% at 1 year would represent a reasonable goal. A sample size of 42 patients provides 90% power to observe such a difference with one-side 5% type-1 error. (NCT01427881)
Timeframe: At 1 year after transplantation

Interventionpercent of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)16

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Grades II-IV and III-IV Acute GVHD

Grades II-IV and III-IV GVHD will be assessed with the use of cumulative incidence plots. (NCT01427881)
Timeframe: Through day +100 post-transplant

Interventionpercentage of patients (Number)
Grades II-IV GVHDGrades III-IV GVHD
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)770

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Persistent or Recurrent Malignancy After HCT

Recurrent or progressive malignancy will be assessed with the use of cumulative incidence plots. Recurrent malignancy will be defined by hematologic criteria. Recurrent malignancy will also be defined as any unplanned medical intervention designed to prevent progression of malignant disease in patients who have molecular, cytogenetic or flow-cytometric evidence of malignant cells after transplantation. (NCT01427881)
Timeframe: At 2 years

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)17

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Overall Survival

Overall survival will be evaluated as Kaplan-Meier estimates. (NCT01427881)
Timeframe: At 1 year post-transplant

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)75.6

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Hematologic Recovery

Descriptive statistics will be used to assess the median days of neutrophil and platelet recovery. The day of neutrophil recovery is defined as the first day of three consecutive lab values on different days, after the conditioning regimen-induced nadir of blood counts, that the absolute neutrophil count is > 500/uL. The day of platelet recovery is defined as the first day of three consecutive lab values on different days, after the conditioning regimen-induced nadir of blood counts, that the platelet count is >= 20,000/uL without platelet transfusion support in the seven days prior. (NCT01427881)
Timeframe: Up to day +100

Interventiondays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)1914

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Non-relapse Mortality

Defined as death in the absence of recurrent or progressive malignancy after HCT. Non-relapse morality will be assessed with the use of cumulative incidence plots. This secondary endpoint will be characterized and presented as a cumulative incidence. (NCT01427881)
Timeframe: At 2 years

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)14

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Graft Failure

Descriptive statistics will be used to assess the incidence of primary graft failure and secondary graft failure. Primary graft failure is defined as failure to achieve a sustained neutrophil count of >= 500/uL by >= 28 days post-transplant. Secondary graft failure is defined as the decline in neutrophil count to < 500/uL after achieving engraftment which is unrelated to infection or drug effect and is unresponsive to stimulation by growth factors. (NCT01427881)
Timeframe: By greater than or equal to 28 days post-transplant

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)2

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Donor Engraftment

Donor engraftment is defined as the count (percent) of patients with full donor chimerism. Full donor chimerism is defined as at least 95% donor CD3 cells in peripheral blood. (NCT01427881)
Timeframe: At day 28

InterventionParticipants (Count of Participants)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)6

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Disease-free Survival

Disease-free survival will be evaluated as Kaplan-Meier estimates. (NCT01427881)
Timeframe: At 1 year post-transplant

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)73.8

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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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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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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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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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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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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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Number of Participants With Treatment Failure

"Treatment failure will not occur until a minimum of 12 months after treatment at which time failure is defined as:~Increase of skin score (if > 14 on enrollment) by > 25% above enrollment value and must be documented on 2 occasions at least 6 months apart~Deterioration in percent predicted FVC by 10% below enrollment level, due to systemic sclerosis, and documented on 2 occasion at least 6 months apart" (NCT01445821)
Timeframe: up to and post 12 months of treatment

InterventionParticipants (Count of Participants)
Cyclophosphamide rATG/HSCT2
Cyclophosphamide rATG/Fludarabine/HSCT2

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Survival of Treatment

Survival of Hematopoietic Stem Cell Transplant. (NCT01445821)
Timeframe: up to 12 months post treatment

InterventionParticipants (Count of Participants)
Cyclophosphamide rATG/HSCT22
Cyclophosphamide rATG/Fludarabine/HSCT21

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Overall Response Rate

"Overall response rate: Defined as the composite endpoint of response to treatment which includes Complete Response (CR), Partial Response (PR), stable disease (SD) as defined in International Response Criteria.~International Myeloma Working Group Response Criteria for Multiple Myeloma:~CR: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and < 5% plasma cells in bone marrow PR: > 50% reduction of serum M-protein and reduction in 24 hours urinary M-protein by >90% or to < 200 mg/24 h SD: Not meeting criteria for CR, VGPR, PR, or progressive disease" (NCT01453101)
Timeframe: Up to 3 years

InterventionParticipants (Count of Participants)
Fludarabine, Melphalan, Bortezomib45

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Overall Survival (OS)

Overall survival (OS): Defined as time from the first dose of administration to death from any cause (NCT01453101)
Timeframe: Up to 3 years

Interventionpercentage (Number)
Fludarabine, Melphalan, Bortezomib42

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Progression Free Survival

The main primary endpoint of this study is two-year progression free survival. Patients are considered a failure with respect to PFS if they die or experience disease progression or relapse. The time to this event is the time from transplantation to relapse/progression, initiation of non-protocol anti-myeloma therapy, or death from any cause. Subjects alive without confirmed disease progression will be censored at the time of last disease evaluation. Deaths without progression are treated as failures no matter when they occur. (NCT01453101)
Timeframe: Subjects will be followed for progression-free survival for at least 36 months

InterventionMonths (Median)
Fludarabine, Melphalan, Bortezomib16.7

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Circulating Chimeric Antigen Receptor (CAR+) Cells in Peripheral Blood at 1 Month Post Treatment

CAR and vector presence were quantitated in peripheral blood mononuclear cell (PBMC) samples using established polymerase chain reaction (PCR) techniques (NCT01454596)
Timeframe: 1 month post transplant

InterventionK/µL (Median)
Group A (Steroids) - Cohort 1: 1x10(7)23
Group A (Steroids) - Cohort 2: 3x10(7)70
Group A (Steroids) - Cohort 3: 1x10(8)36
Group B (No Steroids) - Cohort 1: 1x10(7)67
Group B (No Steroids) - Cohort 2: 3x10(7)7
Group B (No Steroids) - Cohort 3: 1x10(8)43
Group B (No Steroids) - Cohort 4: 3x10(8)28
Group B (No Steroids) - Cohort 5: 1x10(9)25
Combined Steroids/no Steroids) - Cohort 6: 3x10(9)12
Combined Steroids/no Steroids) - Cohort 7: 1x10(10)67.5
Combined Steroids/no Steroids) - Cohort 8: 3-6x10(10)NA
Combined Steroids/no Steroids) - Cohort 9: 3x10(10)8

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Number of Patients With an Objective Response

Objective response was assessed by comparison with baseline dynamic contrast enhanced magnetic resonance imaging with perfusion using Neuro-oncology Working Group proposed guidelines. Complete Response is disappearance of all measurable and non-measurable disease for at least 4 weeks. Partial Response is >/= 50% decrease in lesions for at least 4 weeks. Stable Disease does not meet the criteria for complete response, partial response or progression and requires stable lesions compared with baseline. Progression is >/= 25% increase in lesions. (NCT01454596)
Timeframe: 4 weeks after cell infusion and monthly as feasible up to 12 months

InterventionParticipants (Count of Participants)
Group A (Steroids) - Cohort 1: 1x10(7)0
Group A (Steroids) - Cohort 2: 3x10(7)0
Group A (Steroids) - Cohort 3: 1x10(8)0
Group B (No Steroids) - Cohort 1: 1x10(7)0
Group B (No Steroids) - Cohort 2: 3x10(7)0
Group B (No Steroids) - Cohort 3: 1x10(8)0
Group B (No Steroids) - Cohort 4: 3x10(8)0
Group B (No Steroids) - Cohort 5: 1x10(9)0
Combined Steroids/no Steroids) - Cohort 6: 3x10(9)0
Combined Steroids/no Steroids) - Cohort 7: 1x10(10)0
Combined Steroids/no Steroids) - Cohort 8: 3-6x10(10)0
Combined Steroids/no Steroids) - Cohort 9: 3x10(10)0

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01454596)
Timeframe: 51 dys Grp A, Cohort 1; Cohort 2:68 dys; Cohort 3:40 dys; Grp B, Cohort 1:67 dys; Cohort 2:48 dys; Cohort 3:55 dys; Cohort 4: 46 dys; Cohort 5:147 dys; C. Ster/No Ster Grp, Cohort 6:12 mos, 26 dys; Cohort 7:11 mos, 18 dys; Cohort 8:7 dys; Cohort 9:70 dys.

InterventionParticipants (Count of Participants)
Group A (Steroids) - Cohort 1: 1x10(7)1
Group A (Steroids) - Cohort 2: 3x10(7)1
Group A (Steroids) - Cohort 3: 1x10(8)1
Group B (No Steroids) - Cohort 1: 1x10(7)1
Group B (No Steroids) - Cohort 2: 3x10(7)1
Group B (No Steroids) - Cohort 3: 1x10(8)1
Group B (No Steroids) - Cohort 4: 3x10(8)1
Group B (No Steroids) - Cohort 5: 1x10(9)3
Combined Steroids/no Steroids) - Cohort 6: 3x10(9)3
Combined Steroids/no Steroids) - Cohort 7: 1x10(10)3
Combined Steroids/no Steroids) - Cohort 8: 3-6x10(10)1
Combined Steroids/no Steroids) - Cohort 9: 3x10(10)1

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Progression Free Survival

Progression was assessed by the Response Assessment in Neuro-Oncology (RANO) criteria and is defined as the circumstance when the magnetic resonance imaging (MRI) scan is ranked -2 (definitely worse) or -3 (development of a new lesion). (NCT01454596)
Timeframe: Time from the date of registration to the date of first observation of progressive disease up to 6 months after end of treatment

Interventionmonths (Median)
Group A (Steroids) - Cohort 1: 1x10(7)1.1
Group A (Steroids) - Cohort 2: 3x10(7)1.1
Group A (Steroids) - Cohort 3: 1x10(8)1.3
Group B (No Steroids) - Cohort 1: 1x10(7)1.9
Group B (No Steroids) - Cohort 2: 3x10(7)2.0
Group B (No Steroids) - Cohort 3: 1x10(8)1.5
Group B (No Steroids) - Cohort 4: 3x10(8)1.2
Group B (No Steroids) - Cohort 5: 1x10(9)1.1
Combined Steroids/no Steroids) - Cohort 6: 3x10(9)2.7
Combined Steroids/no Steroids) - Cohort 7: 1x10(10)1.1
Combined Steroids/no Steroids) - Cohort 8: 3-6x10(10)0
Combined Steroids/no Steroids) - Cohort 9: 3x10(10)2.0

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Incidence of Graft Failure

Incidence of graft failure defined as an absolute neutrophil count of less than 500/uL and a bone marrow that is less than 5% cellular (marrow aplasia) (NCT01464359)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Incidence of Acute Graft-Versus-Host Disease

(NCT01464359)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Duration of Survival

(NCT01464359)
Timeframe: 6 months after Transplantation.

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia1

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Duration of Survival

(NCT01464359)
Timeframe: 2 years after Transplantation.

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Duration of Survival

(NCT01464359)
Timeframe: 1 year after Transplantation.

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Disease Free Survival

The primary endpoint is a disease free survival at 3 months in patients with chemotherapy refractory AML after a double T-cell depleted (TCD) umbilical cord blood (UCB) transplantation where one TCD unit is activated overnight in IL-2 followed by the administration of two courses of IL-2 three times a week for 6 doses beginning on day +3 and on day +60 to expand UCB-derived NK cells in vivo. (NCT01464359)
Timeframe: At 3 months

Interventionparticipants (Number)
Patients With Acute Myelogenous Leukemia1

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Clinical Disease Response

Defined as leukemia clearance and complete remission. Patients will be followed for disease response for 2 years from transplantation unless: consent is withdrawal, patient is unevaluable - if a patient is not evaluable, follow only untilthe resolution or stabilization of treatment related toxicity, new anti-cancer treatment is started, patient is discharged to hospice (terminal) care. (NCT01464359)
Timeframe: 2 Years from Transplantation

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia2

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Clinical Disease Response

Defined as leukemia clearance and complete remission. Patients will be followed for disease response for 1 year from transplantation unless: consent is withdrawal, patient is unevaluable - if a patient is not evaluable, follow only until the resolution or stabilization of treatment related toxicity, new anti-cancer treatment is started, patient is discharged to hospice (terminal) care. (NCT01464359)
Timeframe: 1 Year from Transplantation

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia2

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Objective Response in Patients With Metastatic Melanoma

Response is determined by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum of the LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT01468818)
Timeframe: Approximately 2 Years

Interventionparticipants (Number)
Not EvaluableProgressive DiseasePartial Response
Immunotherapy for Metastatic Melanoma1125

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Overall Survival (OS) Post Transplant at 1, 3 and 5 Years

Number of participants in the study who are alive and disease free at 1, 3 and 5 years post transplant. (NCT01471444)
Timeframe: Post transplant after 1, 3 and 5 years

,
InterventionParticipants (Count of Participants)
1 Year Post Transplant3 Year Post Transplant5 Year Post Transplant
Arm A (Flu+Bu)836964
Arm B (Flu+Clo+Bu)826963

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Number of Participants in the Study Who Are With no Grade 3 or 4 Acute Graft-versus-host Disease at Any Time During the First 100 Days Post Transplant.

Number of participants in the study who are with no Grade 3 or 4 acute graft-versus-host disease at any time during the first 100 days post transplant. (NCT01471444)
Timeframe: 100 days post transplant

InterventionParticipants (Count of Participants)
Arm A (Flu+Bu)125
Arm B (Flu+Clo+Bu)115

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Number of Participants With Non Relapse Mortality at 100 Day Post Transplant

Number of participants expired from complications other than relapsed disease at 100 day Post Transplant. (NCT01471444)
Timeframe: 100 day Post Transplant

InterventionParticipants (Count of Participants)
Arm A (Flu+Bu)3
Arm B (Flu+Clo+Bu)6

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Progression-Free Survival (PFS)

Number of events with progression free survival. (Progression is defined as more than 5% blast in the peripheral blood or bone marrow biopsy.) or expired from treatment related mortality post transplant. (NCT01471444)
Timeframe: From day of transplant to disease of progression or death of any cause, whichever came first, assessed up to 5 years

InterventionNumber of events (Number)
Arm A (Flu+Bu)69
Arm B (Flu+Clo+Bu)61

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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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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For the detailed list of adverse events, see the adverse event module. (NCT01495572)
Timeframe: 10 months

Interventionparticipants (Number)
Peripheral Blood Lymphocytes (PBL)5

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Clinical Tumor Response

Complete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10mm. Partial response (PR) is at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum diameters. Progressive disease (PD) is at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study) or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum diameters while on study. (NCT01495572)
Timeframe: One year

Interventionparticipants (Number)
Complete ResponsePartial ResponseProgressive DiseaseStable Disease
Peripheral Blood Lymphocytes (PBL)0050

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Number of Participants With Engraftment.

Median time to ANC engraftment and platelet engraftment in both groups as well as the transfusion requirements measured within 30 days after transplant. (NCT01499147)
Timeframe: Up to 30 days post-transplant

Interventionparticipants (Number)
Fludarabine/Busulfan + ATG18
Fludarabine/Melphalan + ATG12

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Number of Participants With Moderate to Severe (Grade 2-4) Acute Graft Versus Host Disease (GVHD).

Acute GVHD grade 2-4 was assessed in patients in the FluBU and FluMel groups up to 100 days after transplant. (NCT01499147)
Timeframe: Up to 100 days post-transplant (acute GVHD).

Interventionparticipants (Number)
Fludarabine/Busulfan + ATG2
Fludarabine/Melphalan +ATG1

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Time to ANC and Platelet Engraftment

Days to ANC or platelet engraftment (NCT01499147)
Timeframe: Up to 30 days post-transplant

Interventiondays to ANC and platelet engraftment (Median)
Fludarabine/Busulfan + ATG15
Fludarabine/Melphalan + ATG12

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Overall Survival (OS)

Overall Survival is defined as the interval between day of transplant and day of death. (NCT01518153)
Timeframe: Every 3 months until day of death

Interventiondays (Median)
Stem Cell Transplant + Donor Lymphocyte Infusion246

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Success Rate

Success rate defined as alive, engrafted without grade 3 or 4 GvHD or relapse at day 100 post allogeneic stem cell transplantation followed by donor lymphocyte infusion (DLI). (NCT01518153)
Timeframe: 100 days

Interventionparticipants (Number)
Low Dose Donor T-Cells1
High Dose Donor T-Cells3

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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 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 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 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 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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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 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 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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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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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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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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Duration of Remission (DoR) for Participants Who Achieved CR/CRi (Per Investigator Assessment)

DoR was defined as time from date of first response in responders (CR/CRi per Investigator assessment) to date of PFS event (i.e. death, progressive disease [objective progression, relapse from CR/CRi or treatment discontinuation due to global deterioration of health status] or starting new induction therapy or post-therapy stem cell transplant [SCT] without achieving CR/CRi). Responders without PFS events were censored at the last valid disease assessment including follow-up. (NCT01564784)
Timeframe: Up to 2 years from randomization

InterventionMonths (Median)
Inotuzumab Ozogamicin5.4
Defined Investigator's Choice of Chemotherapy3.5

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Maximum Observed Inotuzumab Ozogamicin Serum Concentration (Cmax) and Pre-Dose Inotuzumab Ozogamicin Serum Concentration (Ctrough) Following Single and Multiple Dosing

Blood samples were collected and analyzed for inotuzumab ozogamicin serum concentrations using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS/MS) method with a lower limit of quantification of 1.0 nanograms per milliliter (ng/mL). Cmax was the maximum observed concentration occurring between 0-8 hours post-dose. Ctrough was the concentration prior to subsequent dose (pre-dose) occurring after 8 hours. n = number of observations (non-missing concentrations). (NCT01564784)
Timeframe: Days 1, 4, 8, and 15 of Cycle 1, Days 1 and 8 of Cycle 2 and Day 1 of Cycle 4

Interventionng/mL (Mean)
Cmax (Cycle 1 Day 1, 1 hour post-dose) (n=128)Ctrough (Cycle 4 Day 1, pre-dose) (n=46)Cmax (Cycle 4 Day 1, 1 hour post-dose) (n=37)
Inotuzumab Ozogamicin21157.9308

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Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, Core 30 (EORTC QLQ-C30) Score

This questionnaire comprised 30 questions within which are 9 multi-item scales & 6 single-item measures. There are 5 functional scales; physical, role, cognitive, emotional & social, 3 symptom scales; fatigue, pain, & nausea & vomiting, & a global health status/quality of life (QoL) scale. There are 5 single item measures assessing additional symptoms commonly reported by cancer patients (loss of appetite, insomnia, constipation, diarrhea, & dyspnea) & a single item concerning perceived financial impact of the disease. Most questions used a 4 point scale (1='not at all' to 4='very much'); 2 questions used a 7-point scale (1='very poor' to 7='excellent'). Scores were averaged & transformed to a scale ranging from 0 to 100; a higher score indicates a better level of functioning or greater degree of symptoms. (NCT01564784)
Timeframe: Day 1 of each cycle prior to dosing and EoT

,
InterventionScore on a scale (Mean)
Physical Functioning C2D1Physical Functioning C3D1Physical Functioning C4D1Physical Functioning C5D1Physical Functioning C6D1Physical Functioning EoTRole Functioning C2D1Role Functioning C3D1Role Functioning C4D1Role Functioning C5D1Role Functioning C6D1Role Functioning EoTEmotional Functioning C2D1Emotional Functioning C3D1Emotional Functioning C4D1Emotional Functioning C5D1Emotional Functioning C6D1Emotional Functioning EoTCognitive Functioning C2D1Cognitive Functioning C3D1Cognitive Functioning C4D1Cognitive Functioning C5D1Cognitive Functioning C6D1Cognitive Functioning EoTSocial Functioning C2D1Social Functioning C3D1Social Functioning C4D1Social Functioning C5D1Social Functioning C6D1Social Functioning EoTGlobal Health Status C2D1Global Health Status C3D1Global Health Status C4D1Global Health Status C5D1Global Health Status C6D1Global Health Status EoTDyspnoea C2D1Dyspnoea C3D1Dyspnoea C4D1Dyspnoea C5D1Dyspnoea C6D1Dyspnoea EoTInsomnia C2D1Insomnia C3D1Insomnia C4D1Insomnia C5D1Insomnia C6D1Insomnia EoTAppetite Loss C2D1Appetite Loss C3D1Appetite Loss C4D1Appetite Loss C5D1Appetite Loss C6D1Appetite Loss EoTConstipation C2D1Constipation C3D1Constipation C4D1Constipation C5D1Constipation C6D1Constipation EoTDiarrhoea C2D1Diarrhoea C3D1Diarrhoea C4D1Diarrhoea C5D1Diarrhoea C6D1Diarrhoea EoTFinancial Difficulties C2D1Financial Difficulties C3D1Financial Difficulties C4D1Financial Difficulties C5D1Financial Difficulties C6D1Financial Difficulties EoTFatigue C2D1Fatigue C3D1Fatigue C4D1Fatigue C5D1Fatigue C6D1Fatigue EotNausea and Vomiting C2D1Nausea and Vomiting C3D1Nausea and Vomiting C4D1Nausea and Vomiting C5D1Nausea and Vomiting C6D1Nausea and Vomiting EoTPain C2D1Pain C3D1Pain C4D1Pain C5D1Pain C6D1Pain EoT
Defined Investigator's Choice of Chemotherapy0.32-13.330.00NANA-8.17-1.59-11.110.00NANA-12.374.76-19.440.00NANA4.350.00-5.5616.67NANA0.54-2.38-27.780.00NANA-2.150.40-16.678.33NANA-0.40-7.9411.110.00NANA0.541.590.000.00NANA0.003.170.000.00NANA11.830.000.000.00NANA-0.54-1.59-11.110.00NANA3.760.000.000.00NANA2.195.8222.22-11.11NANA5.73-0.790.00-16.67NANA3.49-7.14-5.56-33.33NANA-7.26
Inotuzumab Ozogamicin0.483.155.3311.527.22-3.385.4511.8116.6712.8816.671.325.217.415.696.822.78-0.914.055.794.580.76-8.330.834.205.5610.4212.8816.671.823.989.3811.258.710.690.00-5.41-7.41-12.50-3.03-2.78-2.31-2.40-9.26-7.50-4.55-11.11-2.31-4.20-8.33-11.67-6.062.78-1.32-1.210.47-2.500.005.562.64-3.30-5.09-0.83-9.52-2.782.31-1.800.47-1.67-3.03-5.560.33-4.10-8.33-9.17-7.320.00-0.330.15-4.63-3.332.270.00-0.17-8.86-8.56-4.170.76-2.78-1.98

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Change From Baseline in EQ-5D VAS

"The EQ-5D self-report questionnaire is a standardized measure of health status developed by the EuroQoL Group. It consists of the EQ-5D descriptive system and a visual analogue scale (VAS), EQ-VAS. The EQ-5D descriptive system measures a participants' health state on 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 3 levels, reflecting no problems, some problems, and extreme problems. The EQ-VAS records the respondent's self-rated health on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicate a better health state." (NCT01564784)
Timeframe: Day 1 of each cycle prior to dosing and EoT

,
InterventionScore on a scale (Mean)
Cycle 2, Day 1Cycle 3, Day 1Cycle 4, Day 1Cycle 5, Day 1Cycle 6, Day 1EoT
Defined Investigator's Choice of Chemotherapy5.9019.6744.00NANA-0.52
Inotuzumab Ozogamicin5.818.137.137.6215.094.62

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Progression-Free Survival (PFS)

PFS was defined as time from date of randomization to earliest date of the following events: death, progressive disease (objective progression, relapse from CR/CRi or treatment discontinuation due to global deterioration of health status) and starting new induction therapy or post-therapy SCT without achieving CR/CRi. Participants without a PFS event at time of analysis were censored at the last valid disease assessment. In addition, participants with documentation of an event after an unacceptably long interval (>28 weeks if there was post-baseline disease assessment, or >12 weeks if there was no post-baseline assessment) since the previous disease assessment were censored at the time of the previous assessment (date of randomization if no post-baseline assessment). Post-study treatment follow-up disease assessments was included. Kaplan-Meier method used and 2-sided 95% confidence interval (CI) calculated based on the Brookmeyer and Crowley method. (NCT01564784)
Timeframe: Up to 2 years from randomization

InterventionMonths (Median)
Inotuzumab Ozogamicin5.0
Defined Investigator's Choice of Chemotherapy1.7

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Percentage of Participants With Veno-Occlusive Liver Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS) Following Post Study HSCT

VOD/SOS was defined as the occurrence of 2 out of the following 3 clinical criteria: 1) total serum bilirubin level >34 micromoles per liter (μmol/L) (>2.0 milligrams per deciliter [mg/dL]), 2) an increase in liver size from baseline or development of right upper quadrant pain of liver origin and 3) sudden weight gain >2.5% (eg, within a 72 hour period) because of fluid accumulation in the weeks following infusion of study drug or chemotherapy, or HSCT conditioning/preparative therapy, or development of ascites not present at baseline following such exposures AND the absence of other explanations for these signs and symptoms, OR development of bilirubin elevation, weight gain, or hepatomegaly plus histologic abnormalities on liver biopsy demonstrating hepatocyte necrosis in zone 3 of the liver acinus, sinusoidal fibrosis, and centrilobular hemorrhage, with or without fibrosis of the terminal hepatic venules. (NCT01564784)
Timeframe: Up to 2 years from randomization

InterventionPercentage of Participants (Number)
Inotuzumab Ozogamicin22.8
Defined Investigator's Choice of Chemotherapy8.6

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Percentage of Participants With Hematologic Remission (Complete Remission [CR]/Complete Remission With Incomplete Hematologic Recovery [CRi]) as Assessed by the Endpoint Adjudication Committee (EAC)

CR was the disappearance of leukemia indicated by less than (<) 5 percent (%) marrow blasts & absence of peripheral blood leukemic blasts, with recovery of hematopoiesis defined by absolute neutrophil count (ANC) greater than or equal to (≥)1000 per microliter (/μL) & platelets ≥100,000/μL. C1 extramedullary disease status (i.e. complete disappearance of measurable & non-measurable extramedullary disease with the following exceptions: for participants with at least 1 measurable lesion, all nodal masses greater than (>) 1.5 centimeters (cm) in greatest transverse diameter (GTD) at baseline must have regressed to less than or equal to (≤) 1.5 cm in GTD; all nodal masses ≥1 cm & ≤1.5 cm in GTD at baseline must have regressed to <1 cm GTD or reduced by 75% in sum of products of greatest diameters, no new lesions, spleen & other previously enlarged organs must have regressed in size & must not be palpable) was required. CRi was defined as CR except ANC <1000/μL &/or platelets <100,000/μL. (NCT01564784)
Timeframe: Screening, Day 16 to 28 of Cycles 1, 2 and 3, then every 1 to 2 cycles (or as clinically indicated) up to approximately 4 weeks (end of treatment [EoT]) from the last dose

InterventionPercentage of Participants (Number)
Inotuzumab Ozogamicin80.7
Defined Investigator's Choice of Chemotherapy29.4

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Change From Baseline in EuroQol 5 Dimension Health Questionnaire (EQ-5D) Index Score

"The EQ-5D self-report questionnaire is a standardized measure of health status developed by the EuroQoL Group. It consists of the EQ-5D descriptive system and a visual analogue scale (VAS), EQ-VAS. The EQ-5D descriptive system measures a participants' health state on 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 3 levels, reflecting no problems, some problems, and extreme problems. The EQ-VAS records the respondent's self-rated health on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicate a better health state. EQ-5D summary index is obtained with a formula that weights each level of the dimensions. The index-based score is interpreted along a continuum of 0 (death) to 1 (perfect health)." (NCT01564784)
Timeframe: Day 1 of each cycle prior to dosing and EoT

,
InterventionScore on a scale (Mean)
Cycle 2, Day 1Cycle 3, Day 1Cycle 4, Day 1Cycle 5, Day 1Cycle 6, Day 1EoT
Defined Investigator's Choice of Chemotherapy0.02-0.080.00NANA-0.04
Inotuzumab Ozogamicin0.000.010.040.040.03-0.01

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Overall Survival (OS)

OS was defined as the time from randomization to date of death due to any cause. Participants last known to be alive were censored at date of last contact. (NCT01564784)
Timeframe: Up to 5 years after randomization or 2 years from randomization of the last participant, whichever occurs first.

InterventionMonths (Median)
Inotuzumab Ozogamicin7.7
Defined Investigator's Choice of Chemotherapy6.2

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Percentage of Participants Who Had a Hematopoietic Stem-Cell Transplant (HSCT)

HSCT rate was defined as the percentage of participants who underwent SCT following treatment with inotuzumab ozogamicin or Investigator's choice of chemotherapy. (NCT01564784)
Timeframe: Up to 19 weeks from last dose

InterventionPercentage of Participants (Number)
Inotuzumab Ozogamicin42.7
Defined Investigator's Choice of Chemotherapy11.1

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Cytogenetic Status (Based on Local Laboratory Analysis) of Participants With CR/CRi (Per EAC Assessment)

Karyotyping was required locally, at screening and at least once during the study in participants who had abnormal cytogenetics at baseline and who achieved CR/CRi. Data presented below are for participants who achieved CR/CRi per EAC and had abnormal karyotype at screening. (NCT01564784)
Timeframe: Up to approximately 4 weeks (EoT) from last dose of study drug

InterventionPercentage of Participants (Number)
Inotuzumab Ozogamicin3.7
Defined Investigator's Choice of Chemotherapy18.2

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Percentage of Participants Achieving MRD Negativity (Based on Central Laboratory Analysis) in Participants Achieving a CR/CRi (Per EAC Assessment)

MRD analysis was performed at least once in participants with prior assessment of CR or CRi. Bone marrow aspirates, collected at screening and during the study, were sent to the central laboratory and analyzed using multiparametric flow cytometry. The antibody combinations were designed to maximize discrimination between normal and abnormal cells of B-cell lineage and similar maturational stage and included antibodies detecting cluster of differentiation (CD) 9, CD10, CD13, CD19, CD20, CD33, CD34, CD38, CD45, CD58, CD66c, and CD123. A peripheral blood sample was provided if a participant had an inadequate bone marrow aspirate at screening. MRD negativity was considered to have been achieved if the lowest value of MRD from the first date of CR/CRi to EoT was <1 × 10^-4 blasts/nucleated cells. (NCT01564784)
Timeframe: Up to approximately 4 weeks (EoT) from last dose of study drug

InterventionPercentage of Participants (Number)
Inotuzumab Ozogamicin78.4
Defined Investigator's Choice of Chemotherapy28.1

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Graft Failure

Primary graft failure occurs when a transplant recipient does not achieve donor chimerism following a bone marrow transplant. Secondary graft failure occurs when graft fails after donor chimerism had initially occurred. (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Primary graft failureSecondary graft failure
Bone Marrow Transplant Recipients01

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Chronic Graft Versus Host Disease (GVHD)

Chronic GVHD was graded according to the National Institutes of Health (NIH) 2014 Consensus Criteria Diagnosis and scoring the severity of chronic GVHD is determined by evaluating symptoms of the skin, nails, hair, mouth, eyes, genitalia, gastrointestinal tract, liver, lungs, muscles, fascia and joints, immune function as well as other symptoms such as ascites and neuropathy. Chronic GVHD is graded as mild, moderate or severe based on the number of organ sites impacted and the severity of symptoms. (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Mild Chronic GVHDModerate Chronic GVHDSevere Chronic GVHD
Bone Marrow Transplant Recipients321

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Time to Neutrophil and Platelet Engraftment

Time to neutrophil engraftment is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of at least 500/µL after conditioning. Time to Platelet engraftment is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL, without receiving a platelet transfusion in the previous 7 days. (NCT01565616)
Timeframe: 1 year after transplant

InterventionDays (Median)
Neutrophil engraftmentPlatelet
Bone Marrow Transplant Recipients1721

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Event -Free Survival Rate

Event-free survival is defined as stable donor erythropoiesis with no new clinical evidence of sickle cell disease. Primary or late graft rejection, disease recurrence, and death are considered events for this endpoint. (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Bone Marrow Transplant Recipients19

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Acute Graft Versus Host Disease (GVHD)

"Acute GVHD was graded according to the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus criteria. Clinical manifestations of acute GVHD include skin, liver, and gastrointestinal symptoms. Grading of acute GVHD is determined by size of maculopapular rash, bilirubin and stool output. Acute GVHD grades range from 0 to 4 with 0 indicating no GVHD and 4 representing the most severe grade.~Grade II is defined as a maculopapular rash over 25-50% of body surface area (BSA), bilirubin of 3.1 to 6 mg/dL, and stool output of 1000-1500 mL/d (for adults).~Grade III is defined as a maculopapular rash over more than 50% of BSA, bilirubin of 6.1 to 15 mg/dL, and stool output of greater than 1500 mL/d (for adults).~Grade IV is defined as generalized erythroderma with bullous formation, bilirubin greater than 15 mg/dL, and severe abdominal pain with or without ileus." (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Grade II Acute GVHDGrade III Acute GVHDGrade IV Acute GVHD
Bone Marrow Transplant Recipients310

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Overall Survival

Overall survival is defined as survival with or without sickle cell disease after hematopoietic cell transplantation (HCT). (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Bone Marrow Transplant Recipients20

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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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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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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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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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Overall Survival

Number of participants that were diseased free and alive 3 years post-transplant. (NCT01572662)
Timeframe: Up to 3 years post-transplant

InterventionParticipants (Count of Participants)
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l16
Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.47

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Overall Survival

Number of participants that are disease free and alive one year post transplant. (NCT01572662)
Timeframe: Up to 1 year post-transplant

InterventionParticipants (Count of Participants)
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l29
Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.93

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Non-Relapse Mortality Rate (NRM)

Number of participants expired within the first 100 days after transplant not due to relapsed disease. (NCT01572662)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l2
Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.8

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01583686)
Timeframe: Date treatment consent signed to date off study, approximately 6 months and 17 days for Group A01, 16 months and 13 days for Group A02, 13 months and 3 days for Group A03, 10 months and 16 days for Group A04, and 11 months and 26 days for Group A05.

InterventionParticipants (Count of Participants)
Arm A01 Anti-mesothelin CAR PBL 1x10^6 + IL-23
Arm A02 Anti-mesothelin CAR PBL 3x10^6 + IL-23
Arm A03 Anti-mesothelin CAR PBL 1x10^7 + IL-23
Arm A04 Anti-mesothelin CAR PBL 3x10^7 + IL-23
Arm A05 Anti-mesothelin CAR PBL 1x10^8 + IL-23

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Number of Patients With Serious and Non-serious Adverse Events

Here is the number of serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0). A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. A non-serious adverse event is any untoward medical occurrence. (NCT01585428)
Timeframe: 51 months and 18 days

InterventionParticipants (Count of Participants)
Cervical18
NonCervical11

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Engraftment Rate of Patients With Non-malignant Diseases (Stratum A)

Engraftment will be defined as the development of an Absolute Neutrophil Count (ANC) >500 for 3 consecutive days plus donor CD14/15 cells >70%. The engraftment rate in the population used for historical control is 40%. If 3 patients in Stratum A experience graft rejection, this stratum will close early for failing to achieve superior engraftment compared to standard-of-care. (NCT01596699)
Timeframe: Participants will have engraftment blood studies starting approximately Day 30 post hematopoietic stem cell transplant and then monthly until stable. Average study participation is approximately 5 years.

Interventionpercentage of participants (Number)
Stratum A: Patients With Non-Malignancies66.67

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Mixed-donor Chimerism Rate of Patients With High-risk Myeloid Malignancies (Stratum B)

Full-donor chimerism will be defined by as ≥99% donor cells by Short Tandem Repeat (STR) analysis in all cell lines (CD3, CD14/15, and CD19) in peripheral blood. The historic control for Stratum B was determined using the 20 patients who were transplanted from 2005 - 2010 with Busulfan (BU)-based regimens and who retrospectively would have been eligible for the current trial. Of these 20 patients, at 100 days post-HCT, only 8 (40%) patients had full-donor chimerism. If 5 patients in Stratum B experienced mixed-donor chimerism at Day 100, we will close this stratum early for failing to achieve superior donor cell engraftment compared to standard-of-care. (NCT01596699)
Timeframe: Participants will have peripheral blood chimerism assessed at Day 100 post hematopoietic stem cell transplant and then monthly until stable.

Interventionpercentage of participants (Number)
Stratum B: Patients With Myeloid Malignancies66.67

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Maintenance of T Cell Receptor (TCR) Expression of Transduced T Cells (Arm I) i.e. Persistence

(NCT01640301)
Timeframe: Up to 28 days post intervention per patient

InterventionParticipants (Count of Participants)
Group 1 (Avelumab and MHC Class I Up-regulation)12

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Maintenance of Function of Transduced T Cells (Arm I)

(NCT01640301)
Timeframe: Up to 28 days post intervention per patient

InterventionParticipants (Count of Participants)
Group 1 (Avelumab and MHC Class I Up-regulation)9

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Incidence of Relapse After T Cell Therapy (Arm II)

(NCT01640301)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group 2 (Avelumab, MHC Class I Up-regulation, T Cells)9

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Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm I)

(NCT01640301)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group 1 (Avelumab and MHC Class I Up-regulation)6

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Anti-leukemic Potential Efficacy, in Terms of Duration of Response (Arm II)

"Response is assessed throughout the 1 year post treatment timeframe. Morphologic criteria for response:~Complete Response (CR) = Bone Marrow blasts <5%; no blasts with Auer rods; no extramedullary disease. Transfusion independent.~Platelets ≥ 100,000/μl Absolute neutrophil count >1000/μl (CRi: incomplete hematologic recovery CRp: incomplete platelet recovery)~Partial Response (PR) = Decrease of at least 50% in the percentage of blasts to 5-25% in the bone marrow and the normalization of blood counts as for CR. (PRi and PRp if incomplete hematologic or platelet recovery)~Stable Disease (SD) = Failure to achieve at least PR, but no evidence of progression for >4 weeks." (NCT01640301)
Timeframe: Up to 1 year

Interventiondays (Median)
Group 2 (Avelumab, MHC Class I Up-regulation, T Cells)28

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Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm II)

(NCT01640301)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group 2 (Avelumab, MHC Class I Up-regulation, T Cells)8

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Count of Participants Who Experienced Grade III-IV Acute Graft Versus Host Disease (GVHD) (Arm II)

(NCT01640301)
Timeframe: Up to 1 year following infusion per patient

InterventionParticipants (Count of Participants)
Group 2 (Avelumab, MHC Class I Up-regulation, T Cells)0

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Count of Participants Who Experienced Grade III-IV Acute Graft-versus-host Disease (GVHD) (Arm I)

(NCT01640301)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group 1 (Avelumab and MHC Class I Up-regulation)1

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Disease-free Survival After T Cell Therapy

(NCT01640301)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Arm I (High-risk for Relapse After HCT)13
Arm II (Relapsed After HCT)7

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Number of Participants With Progression Free Survival (PFS)

Progression-free survival (PFS), defined as the time from study entry to disease progression, relapse or death due to any cause, whichever is earlier, will be summarized with the Kaplan-Meier curve. Confidence intervals for the median and survival rates at different time points will be constructed if needed and appropriate. This secondary endpoint will be reported descriptively. (NCT01659151)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
Combination Therapy7

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Percentage of Participants With Overall Response (OR)

Overall response (OR) is defined as the patient being alive at month 12, and tumor size evaluated at screening and at month 12 using the RECIST 1.1 criteria to be a complete response (CR) or partial response (PR). Evaluations will be made by CT scan approximately 12 months from the date of tumor harvest, and by clinical evaluation during the first 12 months (NCT01659151)
Timeframe: 12 Months

Interventionpercentage of participants (Number)
Combination Therapy38

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Percentage of Participant Drop Out Rate

"The percentage of participants who drop out due to progression between the time of harvest and TIL transfer (i.e., the drop-out rate)." (NCT01659151)
Timeframe: Up to 12 months

Interventionpercentage of participants (Number)
Combination Therapy6

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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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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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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 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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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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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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Occurrence of Dose Limiting Toxicity (DLT) Events

Occurrence of adverse events with dose limiting toxicity, per adverse event category. (NCT01701674)
Timeframe: 3 months

InterventionDLT events (Number)
Eye-related: UveitisGastrointestinal: Colitis
Experimental: Combination Therapy11

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Rate of Meeting Feasibility Requirements

Number of participants who were successfully treated with at least 2 doses of ipilimumab and received TIL. Feasibility is defined as the ability to deliver at least 50% (i.e., two out of four) of the planned doses of ipilimumab and successfully treat at least 60% (i.e., ≥ 6/10) of the patients with TIL. (NCT01701674)
Timeframe: 3 months

InterventionParticipants (Count of Participants)
Experimental: Combination Therapy12

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Progression Free Survival (PFS)

Progression-free survival (PFS) per RECIST V1.1, defined as the time from study entry to disease progression, relapse or death due to any cause, whichever is earlier, will be summarized with the Kaplan-Meier curve. Progressive Disease (PD): At least a 20% increase in the sum of longest diameter (LD) of target lesions taking as reference the smallest sum recorded since the treatment started or the appearance of one or more new lesions. (NCT01701674)
Timeframe: 42 months

Interventionmonths (Median)
Experimental: Combination Therapy7.4

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Overall Response Rate (ORR)

Overall Response: Complete Response (CR) + Partial Response (PR), per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.1 for target lesions and assessed by computed tomography (CT) scan. CR: Disappearance of all target lesions. PR: At least a 30% decrease in the sum of the longest diameter (LD) of target lesions.. (NCT01701674)
Timeframe: 12 weeks

Interventionpercentage of participants (Number)
Experimental: Combination Therapy38.5

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Progression Free Survival

(NCT01707004)
Timeframe: Up to 1 year

Interventiondays (Median)
Decitabine + Bone Marrow Transplant141

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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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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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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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Overall Response Rate

Analysis of the other Secondary Endpoints: The overall response rate will be estimated by the number of patients with complete and partial responses divided by the total number of evaluable patients. (NCT01723839)
Timeframe: 28 day cycle, up to 6 cycles

InterventionPercentage of Participants (Number)
FCR With Lenalidomide95

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Complete Response

Analysis of the Primary Endpoint: The complete responses will be estimated by the number of patients with CR divided by the total number of evaluable patients. (NCT01723839)
Timeframe: 28 day cycle, up to 4 cycles

InterventionPercentage of Participants (Number)
FCR With Lenalidomide45

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Number of Participants That Engrafted and the Number of Participants That Had Full Donor Chimerism at Day 60

To estimate the number of participants that had engraftment rates and the number of participants that had full donor chimerism at Day 60 in patients undergoing an HLA haploidentical stem cell transplant with post transplant high dose cyclophosphamide. (NCT01749293)
Timeframe: Up to Day 60 post-transplant.

InterventionParticipants (Count of Participants)
Haploidentical Transplant0

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Number of Participants That Had an Overall Survival Rate

To estimate the number of participants that had an overall survival (OS) rate (NCT01749293)
Timeframe: Up to one year post-transplant.

InterventionParticipants (Count of Participants)
Haploidentical Transplant1

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Number of Participants That Had an Event Free Survival Rate

To estimate the number of participants who had an event free survival rate (NCT01749293)
Timeframe: Up to 1 year post-transplant

InterventionParticipants (Count of Participants)
Haploidentical Transplant0

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18-Month Overall Survival

Assessment of overall survival at 18 months. Participants alive at last contact are censored at last contact. (NCT01773395)
Timeframe: 18 Month

InterventionPercentage of participants (Number)
GVAX63
Placebo59

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Percentage of Participants Experiencing Grade 3 or Higher Non-Hematologic or Grade 4 or Higher Hematologic Adverse Events

Percentage of participants experiencing grade 3 or higher non-hematologic or grade or higher hematologic adverse events evaluated by CTCAE v5.0. (NCT01773395)
Timeframe: 18 Months

InterventionPercentage of participants (Number)
GVAX3.33
Placebo0

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Percentage of Participants Experiencing Acute and Chronic Graft-Versus-Host Disease

"Percentage of participants with acute and chronic graft-versus-host disease (GVHD). The time frame for the acute incidence is 1 year and for chronic is 3 years.Grading of aGVHD was derived by consensus grading (Przepiorka 1995). The aGVHD algorithm calculates the grade based on the organ (skin, GI and liver) stage and etiology/biopsy reported. Skin staging: Stage 1. <25% rash; 2. 25-50%; 3. >50%; 4. generalized erythroderma with bullae. GI staging: Stage 1. Diarrhea>500ml/d or persistent nausea; 2. >1000ml/d; 3. >1500ml/d; 4. Large volume diarrhea and severe abdominal pain +- ileus. Liver staging: Stage 1. bilirubin 2-3mg/dl; 2. bili 3-6 mg/dl; 3. bili 6-15 mg/dl; 4. bili>15mg/dl. Grade 4 is the worst outcome.~Chronic GVHD is classified using the NIH Consensus Criteria. 8 organs will be scored on a 0-3 scale to reflect degree of cGVHD involvement 0 being none, 1 mild, 2 moderate 3 severe. Liver and pulmonary function test results will also be recorded. Severe is the worst outcome" (NCT01773395)
Timeframe: 1 and 3 years

,
InterventionPercentage of participants (Number)
Grade 2-4 aGVHD @1yrGrade 3-4 aGVHD @1yrChronic GVHD @3yrsMod-severe cGVHD @3yrs
GVAX34164723
Placebo1205933

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Percentage of Participants With Relapse and/or Non-Relapse Mortality

Percentage of participants with relapse and/or non-relapse mortality at 18 months from registration. (NCT01773395)
Timeframe: 18 Months

,
InterventionPercentage of participants (Number)
Non-relapse MortalityRelapse
GVAX1730
Placebo7.737

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18-Month Progression Free Survival

"Progression-Free Survival (PFS) at 18 months after randomization. Progression-free survival is defined as time from randomization to progression of disease or death whichever occurs first. Patients with relapse/progressive disease who re-enter remission after vaccination or upon withdrawal of immune suppression alone will not be considered as having disease progression. The primary analysis will be performed using the modified intention-to-treat (ITT) principle, i.e., all transplanted and eligible patients who receive at least one vaccination will be included in the analysis according to the treatment arm they are randomized to. Patients who have not progressed and are alive are censored at the date the patient is known to be progression-free.~Progression is defined by either morphological evidence of acute leukemia or MDS consistent with pre-transplant features~is defined by either morphological evidence of acute leukemia or MDS consistent with pre-transplant features" (NCT01773395)
Timeframe: 18 months

InterventionPercentage (Number)
GVAX53
Placebo55

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Count of Participants Who Experienced Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0

(NCT01807182)
Timeframe: Adverse events will be collected through 4 weeks after T cell infusion. Beginning 4 weeks after the T cell infusion, only study-related toxicities will be collected for up to 1 year following T cell infusion.

InterventionParticipants (Count of Participants)
Treatment (TIL, Combination Chemotherapy, Aldesleukin)10

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Number of Participants Who Experienced in Vivo Persistence of Adoptively Transferred T Cells Following TIL Infusion

Whole exome and RNA sequencing of tumor cells and normal tissue was performed to identify non synonymous missense mutations, which was then used to generate peptide pools to identify neoantigen-reactive T cells. T-Cell Receptor (TCR) sequencing of T cell clonotypes in blood at time of treatment, 10 months and 24 months was performed and used to assess the persistence of a tumor antigen specific clonotype infused in the TIL product. (NCT01807182)
Timeframe: Up to 24 months

InterventionParticipants (Count of Participants)
At time of treatment10 months post infusion24 months post infusion
Treatment (TIL, Combination Chemotherapy, Aldesleukin)111

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A Count of Participants With Biomarker Expression Above Threshold

Several biomarkers, CXCL13+ CD4 cells, CXCL13+ CD8+ cells, and regulatory cells were evaluated to assess correlation with clinical responses to TIL therapy. (NCT01807182)
Timeframe: Up to 24 weeks

InterventionParticipants (Count of Participants)
CXCL13+ as % of CD4 T Cell (40% Threshold)Treg as % of CD4 T Cell (40% Threshold)CXCL13+ as % of CD8 T Cell (40% Threshold)
Treatment (TIL, Combination Chemotherapy, Aldesleukin)236

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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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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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Percentage of Participants With Ocular Melanoma Treated With Young Tumor Infiltrating Lymphocytes (TIL) With or Without High Dose Aldesleukin With an Objective Response Rate of (Complete Response (CR) + Partial Response (PR))

Objective response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. (NCT01814046)
Timeframe: approximately 3 years

,
Interventionpercentage of participants (Number)
Complete Response (CR)Partial Response (PR)
Cells + High Dose Aldesleukin4.5431.81
Cells and no High Dose Aldesleukin00

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Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01814046)
Timeframe: 46 months and 12 days

InterventionParticipants (Count of Participants)
Cells + High Dose Aldesleukin22
Cells and no High Dose Aldesleukin2

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Count of Participants With Changes in Visual Symptoms

Visual symptoms (e.g., blurred) were evaluated and if changes had occurred from baseline, i.e. in visual acuity, an ophthalmologic consult was performed. (NCT01814046)
Timeframe: 6 weeks (+/- 2 weeks)

InterventionParticipants (Count of Participants)
Cells + High Dose Aldesleukin1
Cells and no High Dose Aldesleukin0

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Overall Survival

Participants that survived between day of transplant and day of death on different dose levels. (NCT01823198)
Timeframe: Up to 2 years

,,,,,
InterventionParticipants (Count of Participants)
Group A: NK cells from KIR mismatched haplo donorsGroup B: NK cells from KIR mismatched cord blood donorsGroup C: NK cells from matched related donors
Phase I: NK Cell Dose Level 1_10^6212
Phase I: NK Cell Dose Level 2_10^7010
Phase I: NK Cell Dose Level 3_ 3x10^7003
Phase I: NK Cell Dose Level 4_ 10^8012
Phase II: NK Cell Dose Level 3_3x10^7010
Phase II: NK Cell Dose Level 4_ 10^8072

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Number of Participants With Grade 3 Toxicities

Number of participants that had grade 3 toxicities up to day 42. (NCT01823198)
Timeframe: Up to day 42

,,,,,
InterventionParticipants (Count of Participants)
Group A: NK cells from KIR mismatched haplo donorsGroup B: NK cells from KIR mismatched cord blood donorsGroup C: NK cells from matched related donors
Phase I: NK Cell Dose Level 1_10^6212
Phase I: NK Cell Dose Level 2_10^7122
Phase I: NK Cell Dose Level 3_ 3x10^7012
Phase I: NK Cell Dose Level 4_ 10^8012
Phase II: NK Cell Dose Level 3_3x10^7010
Phase II: NK Cell Dose Level 4_ 10^80116

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Number of Participants Who Experienced Dose-limiting Toxicities (DLT)

Participants that experienced DLT related to the NK Cells post transplant at different dose levels. (NCT01823198)
Timeframe: Up to 42 days

,,,,,
InterventionParticipants (Count of Participants)
Group A: NK cells from KIR mismatched haplo donorsGroup B: NK cells from KIR mismatched cord blood donorsGroup C: NK cells from matched related donors
Phase I: NK Cell Dose Level 1_10^6222
Phase I: NK Cell Dose Level 2_10^7123
Phase I: NK Cell Dose Level 3_ 3x10^7022
Phase I: NK Cell Dose Level 4_ 10^8022
Phase II: NK Cell Dose Level 3_3x10^7010
Phase II: NK Cell Dose Level 4_ 10^80129

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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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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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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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Number of Participants Who Experienced a Dose Limiting Toxicity (DLT)

To evaluate the side effects of giving Azacytidine before and during chemotherapy using the standard drugs Fludarabine, Cytarabine, IT Cytarabine (AML patients) and IT methotrexate (ALL patients) (NCT01861002)
Timeframe: From Day 1 to Day 42 (Cycle 1)

,
InterventionParticipants (Count of Participants)
# of patients with DLT# of patients without DLT# of patients not evaluable
Dose 75 mg/m2/Day Azacytidine Diagnosed With ALL020
Dose 75 mg/m2/Day Azacytidine Diagnosed With AML0121

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To Assess the Proportions of GvHD Response Post-administration of AP1903.

To assess the proportions of GvHD complete response (CR), partial response (PR), mixed response, no response, and progression among surviving patients at Day 3, 7, 14, 28, and 56 post-administration of AP1903. (NCT01875237)
Timeframe: Day 3, 7, 14, 28, and 56 post-administration of AP1903

InterventionParticipants (Count of Participants)
Day 3 post-administration of AP190371985950Day 3 post-administration of AP190371985951Day 7 post-administration of AP190371985951Day 7 post-administration of AP190371985950Day 14 post-administration of AP190371985951Day 14 post-administration of AP190371985950Day 28 post-administration of AP190371985951Day 28 post-administration of AP190371985950Day 56 post-administration of AP190371985951Day 56 post-administration of AP190371985950
no responsecomplete responseprogressionpartial response
Transplat Plus DLI1
Transplat Plus DLI0
Transplant Only0

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To Assess the Proportion of Patients Developing Grade I-IV Acute GvHD

To assess the proportion of patients developing grade I-IV acute GvHD by Day 28, 56, and 180 post DLI. (NCT01875237)
Timeframe: Day 28, 56, and 180 post DLI.

,
InterventionParticipants (Count of Participants)
Day 28Day 56Day 180
Transplant Only000
Transplat Plus DLI110

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To Assess the Incidence of GvHD Treatment Failure Post-administration of AP1903.

To assess the incidence of GvHD treatment failure, defined as no response, progression, administration of additional therapy for GvHD, or mortality post-administration of AP1903. (NCT01875237)
Timeframe: Day 3, 7, 14, 28, and 56 post-administration of AP1903.

,
InterventionParticipants (Count of Participants)
Day 3 post-administration of AP1903.Day 7 post-administration of AP1903.Day 14 post-administration of AP1903.Day 28 post-administration of AP1903.Day 56 post-administration of AP1903.
Transplant Only00000
Transplat Plus DLI11111

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To Evaluate the Safety of Donor Lymphocyte Infusion Followed by Dimerizer Drug, AP1903 by Number of Participants With Adverse Events.

To evaluate the safety of the infusion of inducible caspase 9 (BPZ-1001) modified T-cells followed by dimerizer drug, AP1903. Safety evaluated by number of participants with Adverse events. (NCT01875237)
Timeframe: up to 3.5 years

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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To Assess Post Donor Lymphocyte Infusion (DLI) Chimerism

To assess the number of Participants with 100% donor chimerism at 6 months post donor lymphocyte infusion (DLI) (NCT01875237)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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To Assess the Incidence of Acute GvHD Flare After CR/PR Requiring Additional Agent for Systemic Therapy Before Day 56 Post-administration of AP1903.

"To assess participants with incidence of acute GvHD flare after CR/PR requiring additional agent (including 2.5 mg/kg/day of prednisone [or methylprednisolone equivalent of 2 mg/kg/day]) for systemic therapy before Day 56 post-administration of AP1903." (NCT01875237)
Timeframe: before Day 56 post AP1903

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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To Assess the Incidence of Epstein-Barr Virus -PTLD or EBV Reactivation Requiring Therapy Post DLI.

To assess the incidence of Epstein-Barr virus (EBV)-associated lymphoproliferative disorder or EBV reactivation requiring therapy post DLI. (NCT01875237)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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Number of Participants Assessed Post Donor Lymphocyte Infusion (DLI): Disease-free Survival & Non-relapse Mortality, Chimerism and GVHD.

Participants to assess at 6 months post donor lymphocyte infusion (DLI): disease-free survival & non-relapse mortality, chimerism and GVHD (NCT01875237)
Timeframe: 6 months

,
InterventionParticipants (Count of Participants)
disease-free survivaIchimerism post DLIGVHD post DLInon-relapse mortality
Transplant Only0000
Transplat Plus DLI1110

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Overall Survival

"Secondary endpoints:~Overall survival: The distribution of time to death from any cause will be estimated by Kaplan- Meier product limit function and plotted. The overall survival will be measured from the time of transplant to any death and patients will be followed for 2 years." (NCT01877837)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Patients With Sickle Cell Anemia23

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Number of Participants With Graft Failure

"Primary endpoint:~In each group, the Number of participants with Graft Failure at the 2 years endpoint will be estimated using the Kaplan Meier product limit estimator." (NCT01877837)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Patients With Sickle Cell Anemia3

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Number of Participants With Acute GVHD, Graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0

(NCT01894477)
Timeframe: Up to 84 days

InterventionParticipants (Count of Participants)
Arm A (Treosulfan, Fludarabine Phosphate)29
Arm B (Treosulfan, Fludarabine Phosphate, TBI)50

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Number of Participants That Did Not Progress Within 6 Months

Progression is defined as relapse (NCT01894477)
Timeframe: At 6 months post-transplant

InterventionParticipants (Count of Participants)
Arm A (Treosulfan, Fludarabine Phosphate)20
Arm B (Treosulfan, Fludarabine Phosphate, TBI)48

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Duration of Remission (DOR) (Phase I, Phase II, and Pediatric)

DOR is defined only for patients who achieve a complete remission (CR), complete remission with incomplete blood count recovery (CRi), marrow complete response (mCR), or partial remission (PR), and is measured from the first date of attaining CR or PR until the date of disease progression or death. (NCT01898793)
Timeframe: Up to 3 years

Interventiondays (Median)
Phase I Dose Level 1: 0.5 x 10^6/kg CIML NK Cells126.0
Phase I Dose Level 2: 1.0 x 10^6/kg CIML NK Cells22.0
Phase I Dose Level 3: 1.0 x 10^7/kg CIML NK Cells76.5
Phase II (IL-2): 1.0 x 10^7/kg CIML NK Cells30.0
Pediatric Cohort: 1.0 x 10^7/kg CIML NK Cells80.0

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Maximal Tolerated or Tested Dose (MT/TD) of CIML-NK Cells (Phase I)

Defined as the dose level immediately below the dose level at which 2 patients of a cohort (of 2 to 6 patients) experience dose-limiting toxicity (DLT) or the maximum dose if less than or equal to 1 patient suffers a DLT at the maximum dose. (NCT01898793)
Timeframe: 35 days

Interventionx 10^7 cells/kg (Number)
Phase I1.0

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Time to Progression (Phase I, Phase II, and Pediatric)

TTP is defined as the time from date of first dose of fludarabine until evidence of disease progression. (NCT01898793)
Timeframe: Up to 3 years

Interventiondays (Median)
Phase I Dose Level 1: 0.5 x 10^6/kg CIML NK Cells20.0
Phase I Dose Level 2: 1.0 x 10^6/kg CIML NK Cells36.0
Phase I Dose Level 3: 1.0 x 10^7/kg CIML NK Cells46.0
Phase II (IL-2): 1.0 x 10^7/kg CIML NK Cells33.0
Lead-in Cohort & Phase II (ALT-803): 1.0 x 10^7/kg CIML NK Cells34.0
Pediatric Cohort: 1.0 x 10^7/kg CIML NK Cells28.0

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Complete Remission Rate (CR/CRi) in Participants With Relapsed or Refractory AML Following CIML NK Therapy (Phase II)

"Complete remission rate (CR): Morphologically leukemia free state (i.e. bone marrow with <5% blasts by morphologic criteria and no blasts with Auer rods, no evidence of extramedullary leukemia) and absolute neutrophil count ≥1000 /μL and platelets ≥100,000 /μL. Patient must be independent of transfusions~Complete Remission with Incomplete Blood Count Recovery (CRi): All of the above criteria for CR must be met, except that absolute neutrophils <1000 /μL or platelets <100,000 /μL in the blood." (NCT01898793)
Timeframe: Up to 3 years

InterventionParticipants (Count of Participants)
Phase II (IL-2): 1.0 x 10^7/kg CIML NK Cells3
Lead-in Cohort & Phase II (ALT-803): 1.0 x 10^7/kg CIML NK Cells0

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Overall Survival (OS) (Phase I, Phase II, and Pediatric)

OS is defined from the date of first dose of fludarabine on this study until death. (NCT01898793)
Timeframe: Up to 3 years

Interventiondays (Median)
Phase I Dose Level 1: 0.5 x 10^6/kg CIML NK Cells39.00
Phase I Dose Level 2: 1.0 x 10^6/kg CIML NK Cells142.50
Phase I Dose Level 3: 1.0 x 10^7/kg CIML NK Cells49.00
Phase II (IL-2): 1.0 x 10^7/kg CIML NK Cells38.00
Lead-in Cohort & Phase II (ALT-803): 1.0 x 10^7/kg CIML NK Cells92.00
Pediatric Cohort: 1.0 x 10^7/kg CIML NK Cells147.00

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Disease Free Survival (DFS) (Phase I, Phase II, and Pediatric)

DFS is defined as the time from the day CR, mCR, or CRi is documented until disease progression or death. (NCT01898793)
Timeframe: Up to 3 years

Interventiondays (Median)
Phase I Dose Level 1: 0.5 x 10^6/kg CIML NK Cells126.00
Phase I Dose Level 2: 1.0 x 10^6/kg CIML NK Cells73.00
Phase I Dose Level 3: 1.0 x 10^7/kg CIML NK Cells56.00
Phase II (IL-2): 1.0 x 10^7/kg CIML NK Cells30.00
Pediatric Cohort: 1.0 x 10^7/kg CIML NK Cells60.00

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Number of Participants With Adverse Events of Special Interest (AESIs)

"The following AEs were defined as AESIs: AEs with the preferred term Tumour Lysis Syndrome (TLS), Infusion-Related Reactions (IRRs) defined as AEs that occurred during or within 24 hours of the completion of obinutuzumab infusion and were assessed as related to obinutuzumab by the Investigator, Infections defined as AEs from System Organ Class (SOC) Infections and infestations and AEs with the preferred term Neutropenia. Reported are number of participants with total AESIs, IRRs, Infections, Neutropenia and TLS." (NCT01905943)
Timeframe: Baseline up to time of primary completion (3 years)

InterventionParticipants (Count of Participants)
Total AESIsIRRsNeutropeniaInfectionsTLS
Obinutuzumab90563559952162

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Number of Participants With Adverse Events of Particular Interest (AEPIs)

"The following AEs were defined as AEPIs: AEs with the preferred term Progressive multifocal leukoencephalopathy (PML), hepatitis B reactivation defined as AEs with preferred term containing Hepatitis B or hepatitis acute, thrombocytopenia defined via Roche MedDRA basket subgroup haematopoietic thrombocytopenia, second malignancies defined as AEs from the SOC Neoplasms benign, malignant and unspecified starting 6 months after the first study drug intake, second malignancies based on standardised MedDRA queries (SMQ) starting 6 months after the first study drug intake based on the MedDRA SMQ Malignant or unspecified tumours, in which benign neoplasms are not included, Cardiac events including AEs from the SOC Cardiac disorders, and hemorrhagic events defined via Roche MedDRA basket subgroup Haemorrhagic events. Reported are number of participants with total AEPIs and each of the AEPI categories." (NCT01905943)
Timeframe: Baseline up to time of primary completion (3 years)

InterventionParticipants (Count of Participants)
Total AEPIsThrombocytopeniaCardiac eventsSecond malignanciesSecond malignancies (SMQ)Hemorrhagic eventsHepatitis B reactivationPML
Obinutuzumab46731410982756931

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Number of Participants With Adverse Events (AEs)

An adverse event 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 and AEs of Particular 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). (NCT01905943)
Timeframe: Baseline up to time of primary completion (3 years)

InterventionParticipants (Count of Participants)
AEsGrade 3-5 AEsSAEs
Obinutuzumab950780516

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Percentage of Participants With Overall Response (OR) at Final Response Assessment (FRA)

OR: percentage of participants with complete response (CR) or CR with incomplete marrow recovery (CRi), or partial response (PR), as determined by the investigator based on International Workshop on Chronic Lymphocytic Leukemia (IWCLL) tumor response criteria. CR: Peripheral blood lymphocytes 4,000/mcL, no significant lymphadenopathy, no hepatomegaly and splenomegaly, no disease symptoms, blood counts: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L and bone marrow normocellular for age. Cri: CR with persistent cytopenia. PR: >/= 50% decrease in peripheral blood lymphocyte count AND >/= 50% reduction in lymphadenopathy OR >/= 50% reduction of liver enlargement OR >/= 50% reduction of spleen PLUS one of the following: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L OR >/= 50% increase in neutrophils, platelets or hemoglobin. (NCT01905943)
Timeframe: 3 months after the last dose of study treatment (up to approximately 5 years)

Interventionpercentage of participants (Number)
G Mono: Previously Untreated Fit71.0
G Mono: Previously Untreated Unfit59.4
G Mono: Relapsed/Refractory41.5
G-Benda: Previously Untreated Fit83.9
G-Benda: Previously Untreated Unfit81.6
G-Benda: Relapsed/Refractory73.2
G-FC: Previously Untreated Fit90.0
G-FC: Previously Untreated Unfit84.6
G-FC: Relapsed/Refractory85.0
G-Clb: Previously Untreated Fit100
G-Clb: Previously Untreated Unfit82.1
G-Clb: Relapsed/Refractory56.5

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Percentage of Participants With Best Overall Response (BOR)

BOR was defined as the percentage of participants with the best response obtained throughout the trial with CR, CRi, or PR, as determined by the investigator based on IWCLL tumor response criteria. CR: Peripheral blood lymphocytes 4,000/mcL, no significant lymphadenopathy, no hepatomegaly and splenomegaly, no disease symptoms, blood counts: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L and bone marrow normocellular for age. Cri: CR with persistent cytopenia. PR: >/= 50% decrease in peripheral blood lymphocyte count AND >/= 50% reduction in lymphadenopathy OR >/= 50% reduction of liver enlargement OR >/= 50% reduction of spleen PLUS one of the following: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L OR >/= 50% increase in neutrophils, platelets or hemoglobin. (NCT01905943)
Timeframe: Baseline, Day 85, end of treatment or early termination, and follow-up, assessed up to disease progression or death, whichever occurs first (up to approximately 5 years)

Interventionpercentage of participants (Number)
G Mono: Previously Untreated Fit83.9
G Mono: Previously Untreated Unfit71.9
G Mono: Relapsed/Refractory60.0
G-Benda: Previously Untreated Fit91.7
G-Benda: Previously Untreated Unfit93.9
G-Benda: Relapsed/Refractory86.8
G-FC: Previously Untreated Fit97.1
G-FC: Previously Untreated Unfit84.6
G-FC: Relapsed/Refractory97.5
G-Clb: Previously Untreated Fit100
G-Clb: Previously Untreated Unfit94.0
G-Clb: Relapsed/Refractory84.8

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Median Time to Response (TTR)

Kaplan Meier estimate of median TTR was defined as the time at which half of the participants reached CR or PR based on IWCLL tumor response criteria. CR: Peripheral blood lymphocytes 4,000/mcL, no significant lymphadenopathy, no hepatomegaly and splenomegaly, no disease symptoms, blood counts: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L and bone marrow normocellular for age. PR: >/= 50% decrease in peripheral blood lymphocyte count AND >/= 50% reduction in lymphadenopathy OR >/= 50% reduction of liver enlargement OR >/= 50% reduction of spleen PLUS one of the following: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L OR >/= 50% increase in neutrophils, platelets or hemoglobin. (NCT01905943)
Timeframe: Baseline, Day 85, end of treatment or early termination, and follow-up, assessed up to disease progression or death, whichever occurs first (up to approximately 5 years)

Interventionmonths (Median)
G Mono: Previously Untreated Fit3.6
G Mono: Previously Untreated Unfit3.6
G Mono: Relapsed/Refractory3.9
G-Benda: Previously Untreated Fit3.5
G-Benda: Previously Untreated Unfit3.5
G-Benda: Relapsed/Refractory3.7
G-FC: Previously Untreated Fit3.6
G-FC: Previously Untreated Unfit4.1
G-FC: Relapsed/Refractory3.6
G-Clb: Previously Untreated Fit3.3
G-Clb: Previously Untreated Unfit3.6
G-Clb: Relapsed/Refractory3.7

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Median Time to Progression-Free Survival (PFS)

Kaplan Meier estimate of the median PFS was defined as the time at which half of the participants have progressed (progressive disease [PD]) based on IWCLL tumor response criteria or died from any cause, whichever occurred first. PD: at least one of the following: >/= 50% increase in the absolute number of circulating lymphocytes to at least 5,000/mcL, appearance of new palpable lymph nodes, >/= 50% increase in the longest diameter of any previous site of clinically significant lymphadenopathy, >/= 50% increase in the enlargement of the liver and/or spleen, transformation to more aggressive histology, progression of any cytopenia, decrease of hemoglobin levels by more than 20 g/L or to less than 100 g/L, decrease of platelet counts by more than 50% or to less than 100,000 /mcL, decrease of neutrophil counts by more than 50% or to less than 1,000/mcL. (NCT01905943)
Timeframe: Baseline, Day 85, end of treatment or early termination, and follow-up, assessed up to disease progression or death, whichever occurs first (up to approximately 5 years)

Interventionmonths (Median)
G Mono: Previously Untreated Fit43.0
G Mono: Previously Untreated Unfit21.2
G Mono: Relapsed/Refractory17.6
G-Benda: Previously Untreated Fit58.0
G-Benda: Previously Untreated UnfitNA
G-Benda: Relapsed/Refractory28.6
G-FC: Previously Untreated FitNA
G-FC: Previously Untreated UnfitNA
G-FC: Relapsed/Refractory24.8
G-Clb: Previously Untreated Fit31.3
G-Clb: Previously Untreated Unfit31.8
G-Clb: Relapsed/Refractory14.1

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Median Time to Overall Survival (OS)

Kaplan Meier estimate of median OS was defined as the time at which half of the participants had died, regardless of the cause of death. (NCT01905943)
Timeframe: Baseline until death (Approximately up to 5 years)

Interventionmonths (Median)
G Mono: Previously Untreated FitNA
G Mono: Previously Untreated UnfitNA
G Mono: Relapsed/RefractoryNA
G-Benda: Previously Untreated FitNA
G-Benda: Previously Untreated UnfitNA
G-Benda: Relapsed/RefractoryNA
G-FC: Previously Untreated FitNA
G-FC: Previously Untreated UnfitNA
G-FC: Relapsed/RefractoryNA
G-Clb: Previously Untreated FitNA
G-Clb: Previously Untreated UnfitNA
G-Clb: Relapsed/RefractoryNA

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Median Time to Duration of Response (DoR)

Kaplan Meier estimate of median DoR was defined as the time at which half of the responding (PR or CR) participants had progressed (PD) or died from any cause, whichever occurred first. PR: >/= 50% decrease in peripheral blood lymphocyte count AND >/= 50% reduction in lymphadenopathy OR >/= 50% reduction of liver enlargement OR >/= 50% reduction of spleen PLUS one of the following: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L OR >/= 50% increase in neutrophils, platelets or hemoglobin. CR: Peripheral blood lymphocytes 4,000/mcL, no significant lymphadenopathy, no hepatomegaly and splenomegaly, no disease symptoms, blood counts: neutrophils >1,500/mcL, platelets > 100,000/mcL, hemoglobin > 110 g/L and bone marrow normocellular for age. PD: as defined in the description for Event-Free Survival outcome measure. (NCT01905943)
Timeframe: Baseline, Day 85, end of treatment or early termination, and follow-up, assessed up to disease progression or death, whichever occurs first (up to approximately 5 years)

Interventionmonths (Median)
G Mono: Previously Untreated Fit40.1
G Mono: Previously Untreated Unfit20.1
G Mono: Relapsed/Refractory15.0
G-Benda: Previously Untreated Fit55.0
G-Benda: Previously Untreated Unfit49.3
G-Benda: Relapsed/Refractory25.5
G-FC: Previously Untreated FitNA
G-FC: Previously Untreated UnfitNA
G-FC: Relapsed/Refractory21.2
G-Clb: Previously Untreated Fit28.1
G-Clb: Previously Untreated Unfit28.1
G-Clb: Relapsed/Refractory12.3

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Median Time to New Anti-Leukemia Therapy (TTNT)

Kaplan Meier estimate of median TTNT was defined as the time at which half of the participants have initiated a new anti-leukemic therapy. (NCT01905943)
Timeframe: Baseline until end of study (up to approximately 5 years)

Interventionmonths (Median)
G Mono: Previously Untreated FitNA
G Mono: Previously Untreated UnfitNA
G Mono: Relapsed/Refractory22.5
G-Benda: Previously Untreated FitNA
G-Benda: Previously Untreated UnfitNA
G-Benda: Relapsed/Refractory38.3
G-FC: Previously Untreated FitNA
G-FC: Previously Untreated UnfitNA
G-FC: Relapsed/Refractory32.6
G-Clb: Previously Untreated FitNA
G-Clb: Previously Untreated Unfit53.7
G-Clb: Relapsed/Refractory20.4

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Percentage of Participants With Minimal Residual Disease (MRD)-Negativity as Assessed by Flow Cytometry

MRD-negativity was defined as the presence of less than 1 chronic lymphocytic leukemia (CLL) cell per 10,000 leukocytes in blood and bone marrow as assessed by flow cytometry 3 months after last dose of study treatment (i.e. at final response assessment [FRA] visit). (NCT01905943)
Timeframe: 3 months after the last dose of study treatment (up to approximately 5 years)

,,,,,,,,,,
Interventionpercentage of participants (Number)
BloodBone Marrow
G Mono: Previously Untreated Fit8.34.2
G Mono: Previously Untreated Unfit23.13.8
G Mono: Relapsed/Refractory4.12.0
G-Benda: Previously Untreated Fit63.131.5
G-Benda: Previously Untreated Unfit65.327.2
G-Benda: Relapsed/Refractory39.814.9
G-Clb: Previously Untreated Unfit9.45.7
G-Clb: Relapsed/Refractory6.33.1
G-FC: Previously Untreated Fit72.040.0
G-FC: Previously Untreated Unfit58.341.7
G-FC: Relapsed/Refractory51.524.2

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Median Time to Event-Free Survival (EFS)

Kaplan Meier estimate of median EFS is the time at which half of the participants have progressed as assessed by investigator based on IWCLL tumor response criteria, or have initiated a non-protocol-specified anti-leukemia therapy or died, whichever occurs first. PD: at least 1 of the following: >/= 50% increase in absolute number of circulating lymphocytes to at least 5,000/mcL, appearance of new palpable lymph nodes, >/= 50% increase in longest diameter of any previous site of clinically significant lymphadenopathy, >/= 50% increase in enlargement of liver and/or spleen, transformation to more aggressive histology, progression of any cytopenia, decrease of hemoglobin levels by more than 20 g/L or to less than 100 g/L, decrease of platelet counts by more than 50% or to less than 100,000 /mcL, decrease of neutrophil counts by more than 50% or to less than 1,000/mcL. (NCT01905943)
Timeframe: Baseline, Day 85, end of treatment or early termination, and follow-up, assessed up to disease progression or death, whichever occurs first (up to approximately 5 years)

Interventionmonths (Median)
G Mono: Previously Untreated Fit35.2
G Mono: Previously Untreated Unfit17.9
G Mono: Relapsed/Refractory14.0
G-Benda: Previously Untreated Fit58.0
G-Benda: Previously Untreated Unfit52.9
G-Benda: Relapsed/Refractory25.1
G-FC: Previously Untreated FitNA
G-FC: Previously Untreated UnfitNA
G-FC: Relapsed/Refractory24.2
G-Clb: Previously Untreated Fit31.3
G-Clb: Previously Untreated Unfit31.8
G-Clb: Relapsed/Refractory13.7

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Percentage of Participants With a Response

Percentage of patients who have a clinical response (complete response or partial response) to treatment (objective tumor regression). Response was determined entirely by radiographic imaging using the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0 to compare target lesions in centimeters. Complete Response is defined as disappearance of all target lesions. Partial Response is defined as at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. (NCT01967823)
Timeframe: 6 and 12 weeks after cell infusion, then every 3 months x3, then every 6 months x 2, then per principal investigator (PI) discretion, up to five years or disease progression.

Interventionpercentage of participants (Number)
Complete ResponsePartial Response
Lymphodepleting Conditioning Foll/by Infusion of Anti-NY ESO1 Murine TCR-Gene Engineered Lymphocytes1050

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Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells

T Cell Receptor (TCR) and vector presence will be quantitated in peripheral blood mononuclear cells (PBMC) samples using established polymerase chain reaction (PCR) techniques. (NCT01967823)
Timeframe: 3 and 6 months, and 1 year post cell administration

InterventionPercentage of Tcells (Number)
10100031010006
T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells at ≥ 1 Year0.870.87

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Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells

T Cell Receptor (TCR) and vector presence will be quantitated in peripheral blood mononuclear cells (PBMC) samples using established polymerase chain reaction (PCR) techniques. (NCT01967823)
Timeframe: 3 and 6 months, and 1 year post cell administration

InterventionPercentage of Tcells (Number)
101000310100061010008101000910100101010011
T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells at 6 (± 2) Months7.157.150.2818.40.3246.2

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Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells

T Cell Receptor (TCR) and vector presence will be quantitated in peripheral blood mononuclear cells (PBMC) samples using established polymerase chain reaction (PCR) techniques. (NCT01967823)
Timeframe: 3 and 6 months, and 1 year post cell administration

InterventionPercentage of Tcells (Number)
10100011010002101000310100061010007101000810100091010010
T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells at 3 (± 1) Months1.9340.916.116.14416.931.80.37

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Percentage of T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells

T Cell Receptor (TCR) and vector presence will be quantitated in peripheral blood mononuclear cells (PBMC) samples using established polymerase chain reaction (PCR) techniques. (NCT01967823)
Timeframe: 3 and 6 months, and 1 year post cell administration

InterventionPercentage of Tcells (Number)
101000110100021010003101000610100071010008101000910100101010011
T Cell Receptor (TCR) in Cluster of Differentiation 3 (CD3) + Cells on Day of Infusion.818376.487.38086.78268.478.2

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Number of Subjects Disease-free Survival

Percentage of patients without relapse of disease at 2 years (NCT01991457)
Timeframe: 2 years post-transplant

Interventionpercentage of participants (Number)
Treatment63.2

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Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01993719)
Timeframe: Date treatment consent signed to date off study, approximately 102 months and 9 days, 92 months and 19 days, 86 months and 9 days, 99 months and 16 days, and 86 months and 26 days for each group respectively.

InterventionParticipants (Count of Participants)
Arm 1N -Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin7
Arm 1P - Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin9
Arm 2/Foll By Arm 1P-Low Dose Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin3
Arm 2- Low Dose Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin9
Arm 1P/Foll By Arm-1P/R-Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin2

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Overall Response Rate (ORR)

Overall response is the best response recorded from the start of treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.0). Progression is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Date of cells until time of disease progression, up to approximately 67.2 months.

Interventionpercentage of participants (Number)
Retreat
Arm 1P/Foll By Arm-1P/R-Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin50

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Overall Response Rate (ORR)

Overall response is the best response recorded from the start of treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.0). Progression is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Date of cells until time of disease progression, up to approximately 67.2 months.

,
Interventionpercentage of participants (Number)
Initial Treatment
Arm 1N -Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin57.10
Arm 2/Foll By Arm 1P-Low Dose Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin16.70

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Progression-free Survival (PFS)

PFS is defined as the time to disease progression following the start of treatment, and time to death following the start of treatment. Progression was assessed by the Response Evaluation Criteria In Solid Tumors (RECIST) v1.0 and is defined as at least a 20% increase in the sum of the longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Date of cells until time of disease progression up to approximately 67.2 months.

InterventionMonths (Median)
Initial TreatmentRetreat
Arm 1P - Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin3.11.6

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Number of Participants Who Have a Clinical Response to Treatment (Objective Tumor Regression)

Clinical response to treatment was assessed by the Response Evaluation Criteria In Solid Tumors (RECIST v1.0). Complete Response (CR) is disappearance of all target lesions. Partial Response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive Disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT01993719)
Timeframe: 4 weeks after cell infusion, then every 3 months x 3 and then every 6 months for 5 years, then per Principal Investigator (PI) discretion up to 5 years or disease progression

,,,
InterventionParticipants (Count of Participants)
First Treatment - Complete ResponseFirst Treatment - Partial ResponseFirst Treatment - Progressive DiseaseFirst Treatment - Stable DiseaseSecond Treatment - Complete ResponseSecond Treatment - Partial ResponseSecond Treatment - Progressive DiseaseSecond Treatment - Stable Disease
Arm 1N -Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin2221NANANANA
Arm 1P - Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin02830111
Arm 1P/Foll By Arm-1P/R-Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg AldesleukinNANANANA0110
Arm 2/Foll By Arm 1P-Low Dose Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin0201NANANANA

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Overall Response Rate (ORR)

Overall response is the best response recorded from the start of treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.0). Progression is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Date of cells until time of disease progression, up to approximately 67.2 months.

Interventionpercentage of participants (Number)
Initial TreatmentRetreat
Arm 1P - Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin23.1033.30

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Progression-free Survival (PFS)

PFS is defined as the time to disease progression following the start of treatment, and time to death following the start of treatment. Progression was assessed by the Response Evaluation Criteria In Solid Tumors (RECIST) v1.0 and is defined as at least a 20% increase in the sum of the longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Date of cells until time of disease progression up to approximately 67.2 months.

,
InterventionMonths (Median)
Initial Treatment
Arm 1N -Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin7.9
Arm 2/Foll By Arm 1P-Low Dose Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin2.1

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Progression-free Survival (PFS)

PFS is defined as the time to disease progression following the start of treatment, and time to death following the start of treatment. Progression was assessed by the Response Evaluation Criteria In Solid Tumors (RECIST) v1.0 and is defined as at least a 20% increase in the sum of the longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Date of cells until time of disease progression up to approximately 67.2 months.

InterventionMonths (Median)
Retreat
Arm 1P/Foll By Arm-1P/R-Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin32.5

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Overall Progression Free Survival (PFS)

PFS is defined as the time to disease progression following the start of treatment, and time to death following the start of treatment. Progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0 and is defined as at least a 20% increase in the sum of the longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT01993719)
Timeframe: Time to progression and time to death, approximately up to 67.2 months.

InterventionMonths (Median)
All Participants in Arms 1N, 1P, Arm 2/Foll By Arm 1P, Arm 2, and Arm 1P/Foll By Arm 1P/R3

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Overall Survival

Overall survival is defined as the time from treatment start date until date of death, or date last known alive. (NCT01993719)
Timeframe: Date of cells until time to death, up until 90.1 months.

InterventionMonths (Median)
Arm 1P - Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin14.3
Arm 1N -Standard Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg AldesleukinNA
Arm 2- Low Dose Chemotherapy Preparative Regimen + TIL + 720,000 IU/kg Aldesleukin15.1

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Overall Survival

Overall survival is defined as the time from treatment start date until date of death, or date last known alive. (NCT01993719)
Timeframe: An average of 25.6 months.

InterventionMonths (Median)
All Participants in Arms 1N, 1P, Arm 2/Foll By Arm 1P, Arm 2, and Arm 1P/Foll By Arm 1P/R17.5

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Number of Successful Unrelated Cord Blood (UCB) Transplants

The number of patients who received successful UCB transplants as evidenced by absolute neutrophil recovery. (NCT02007863)
Timeframe: 2 Years

Interventionparticipants (Number)
Umbilical Cord Blood + Chemotherapy2

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Percentage of Participants With the Best Clinical Response by Visit (Clinical Assessment)

Best clinical response was determined according to the NCI clinical evaluation and through radiological assessment. CR, CRi, CRu, partial remission (PR), partial remission with toxicity associated (PRTox), progressive disease (PD), and stable disease (SD) were evaluated. Assessment of response was performed according to the NCI revised guidelines for the diagnosis and treatment of CLL with additional CT scan evaluation of lymphadenopathy during the treatment period (Radiological). Response assessment for interim (Week 12), end of induction (Week 24) and at Final Staging (4 weeks after last maintenance dose). Last observation carried forward (LOCF) method was used for missing data. Percentages are based on the number of nonmissing observations within each stratum. (NCT02013817)
Timeframe: Weeks 12 and 24 and at Final Staging (Week 4 after last maintenance dose)

Interventionpercentage of participants (Number)
CR, Week 12CRu, Week 12CRi, Week 12PR, Week 12PRTox, Week 12SD, Week 12PD, Week 12Not evaluable, Week 12CR, Week 24CRu, Week 24CRi, Week 24PR, Week 24PRTox, Week 24SD, Week 24PD, Week 24Not evaluable, Week 24CR, Final stagingCRu, Final stagingCRi, Final stagingPR, Final stagingPRTox, Final stagingSD, Final stagingPD, Final stagingNot evaluable, Final staging
Rituximab, Fludarabine, Cyclophosphamide41.916.316.311.62.30.00.011.630.211.637.29.30.00.00.011.660.57.011.67.00.00.02.311.6

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Percentage of Participants With the Best Clinical Response by Visit (Clinical + Radiological Assessment)

Best clinical response was determined according to the NCI clinical evaluation and through radiological assessment. CR, CRi, CRu, PR, PRTox, PD, and SD were evaluated. Assessment of response was performed according to the NCI revised guidelines for the diagnosis and treatment of CLL with additional CT scan evaluation of lymphadenopathy during the treatment period (Radiological). Response assessment for interim (Week 12), end of induction (Week 24) and at Final Staging (4 weeks after last maintenance dose). LOCF method was used for missing data. Percentages are based on the number of nonmissing observations within each stratum. (NCT02013817)
Timeframe: Weeks 12 and 24 and at Final Staging (Week 4 after last maintenance dose)

Interventionpercentage of participants (Number)
CR, Week 12CRu, Week 12CRi, Week 12PR, Week 12PRTox, Week 12SD, Week 124PD, Week 12Not evaluable, Week 12CR, Week 24CRu, Week 24CRi, Week 24PR, Week 24PRTox, Week 24SD, Week 24PD, Week 24Not evaluable, Week 24CR, Final stagingCRu, Final stagingCRi, Final stagingPR, Final stagingPRTox, Final stagingSD, Final stagingPD, Final stagingNot evaluable, Final staging
Rituximab, Fludarabine, Cyclophosphamide30.24.77.030.29.37.00.011.623.37.030.214.07.07.00.011.648.87.07.011.62.32.39.311.6

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Percentage of Participants With a Best Clinical Response of Clinical Remission (CR)

Best clinical response was determined according to the National Cancer Institute (NCI) Clinical and Clinical plus (+) Radiological evaluations by central response assessment. Assessment of response was performed according to the NCI revised guidelines for the diagnosis and treatment of chronic lymphocytic lymphoma (CLL) with additional computerized tomography (CT) scan evaluation of lymphadenopathy. Per NCI guidelines, CR requires all of the following criteria at least 2 months after the last treatment: no lymphadenopathy (Ly)/ hepatomegaly/ splenomegaly/constitutional symptoms; neutrophils greater than (>)1500 per microliter (/µL), platelets (PL) >100,000/µL, hemoglobin (Hb) >11.0 grams per deciliter (g/dL), lymphocytes (LC) (less than) <4000/µL, bone marrow (BM) sample must be normocellular for age, <30% LC. (NCT02013817)
Timeframe: Weeks 1, 5, 9, 12, 13, 17, 21 and 24

Interventionpercentage of participants (Number)
NCI ClinicalNCI Clinical (including CRu, CRi)NCI Clinical + RadiologicalNCI Clinical + Radiological (including CRu, CRi)
Rituximab, Fludarabine, Cyclophosphamide73.794.763.278.9

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Percentage of Participants With Adverse Events (AEs)

AEs were recorded from the date of first medication administration until 28 days after the last trial medication. (NCT02013817)
Timeframe: Day 1 of Cycles 1, 2, 3, 4, 5, and 6 to 28 days after the last trial medication.

Interventionpercentage of participants (Number)
Any AERelated AESevere AESAEPregnancy
Rituximab, Fludarabine, Cyclophosphamide10093.076.762.80.0

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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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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 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 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 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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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 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 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 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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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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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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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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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 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 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 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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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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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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Overall Survival (OS) Rate at 3 Years

Overall survival was defined as time from randomization to death from any cause or date last known alive. Overall survival rate at 3 years was estimated using the method of Kaplan-Meier. (NCT02048813)
Timeframe: Assessed every 3 months until progression; after progression, assessed every 3 months for first 2 years, every 6 months for years 3-5, up to 4 years and 8 months

InterventionProportion of participants (Number)
Arm A (Ibrutinib, Rituximab)0.988
Arm B (Rituximab, Fludarabine Phosphate, Cyclophosphamide)0.915

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Progression-free Survival (PFS) Rate at 3 Years

"PFS was defined as the time from randomization to CLL progression or death, whichever occurred first. Progression is characterized by any of the following:~≥ 50% increase from nadir since start of treatment (tx) in the sum of the products of at least 2 lymph nodes on 2 consecutive examinations 2 weeks apart~≥ 50% increase from nadir since start of tx in the size of liver and/or spleen~≥ 50% increase in the absolute number of circulating lymphocytes not due to tumor flare reaction. The absolute lymphocyte count must be ≥ 5x10^9/L to qualify as disease progression.~Transformation to a more aggressive histology (e.g. Richter's syndrome or prolymphocytic leukemia with > 55% prolymphocytes). For patients who achieve a complete response or nodular partial response, progression is defined as recurrence of circulating leukemia cell clone in the peripheral blood and an absolute lymphocyte count > 5x10^9/L and/or recurrence of palpable lymphadenopathy > 1.5 cm by physical exam." (NCT02048813)
Timeframe: Assessed every 3 months until progression up to 4 years and 8 months

InterventionProportion of participants (Number)
Arm A (Ibrutinib, Rituximab)0.894
Arm B (Rituximab, Fludarabine Phosphate, Cyclophosphamide)0.729

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT02062359)
Timeframe: 3 months

Interventionparticipants (Number)
All Participants2

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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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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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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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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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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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Maximum Tolerated Cell Dose (MTD) of Cluster of Differentiation 4 (CD4) Cells Transduced With an Anti-MAGE-A3-DP0401/0402 Restricted (MAGE-A3-DP4) T Cell Receptor and Aldesleukin

Highest dose at which less than or equal to 1 of 6 patients experienced a dose-limiting toxicity (DLT) (all grade 3 and greater toxicities with the exception of myelosuppression and grade 3 fever, for example) or the highest dose level studied if DLTs are not observed at any of the dose levels. (NCT02111850)
Timeframe: Before progression to next-higher dose level, at least two weeks

Interventioncells (Number)
All Participants on Phase 1100,000,000,000

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Number of Engineered T Cell Receptor (TCR) Cells That Survived at 4 Weeks

T cell receptor (TCR) and vector presence was quantitated in peripheral blood mononuclear cells (PBMC) samples using flow cytometry. It is a process by which cells are suspended in a liquid so they can be counted. (NCT02111850)
Timeframe: 4 weeks

Interventioncells/uL (Median)
Phase I Dose Escalation Arm 1, Dose Level 1 - 1 X 10^7 Cells + Interleukin-21.0792
Phase I Dose Escalation Arm 1, Dose Level 2 - 3 X 10^7 Cells + Interleukin-21.9656
Phase I Dose Escalation Arm 1 Dose Level 3 - 1 X 10^8 Cells + Interleukin-22.8676
Phase I Dose Escalation Arm 1, Dose Level 5 - 1 X 10^9 Cells + Interleukin-20.17248
Phase I Dose Escalation Arm 1, Dose Level 6 - 3 X 10^9 Cells + Interleukin-22.8
Phase I Dose Escalation Arm 1, Dose Level 7 - 1 X 10^10 Cells + Interleukin-20
Phase I Dose Escalation Arm 1, Dose Level 8 - 3 X 10^10 Cells + Interleukin-26.79
Phase I Dose Escalation Arm 1, Dose Level 9 - 1 X 10^11 Cells + Interleukin-231.9
Phase 2 Arm 2, Cohort 1- Maximum Tolerated Dose + Interleukin-2 Other84.86
Phase 2 Arm 2, Cohort 2 - Maximum Tolerated Dose + Interleukin-2 Melanoma24.95

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Number of Participants With Dose-limiting Toxicity (DLT)

A dose-limiting toxicity (DLT) is all grade 3 and greater toxicities with the exception of myelosuppression, aldesleukin expected toxicities, expected chemotherapy toxicities, immediate hypersensitivity reactions occurring within 2 hours of cell infusion, grade 3 fever, grade 3 metabolic laboratory abnormalities without significant clinical sequela that resolve within grade 2 within 7 days, and grade 3 autoimmunity that resolves to less than or equal to a grade 2 autoimmune toxicity within 10 days. (NCT02111850)
Timeframe: Before progression to next-higher dose level, approximately 2 weeks

InterventionParticipants (Count of Participants)
Phase 10

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0).

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02111850)
Timeframe: Date treatment consent signed to date off study, an average of 17 months

InterventionParticipants (Count of Participants)
Phase I Dose Escalation Arm 1, Dose Level 1 - 1 X 10^7 Cells + Interleukin-21
Phase I Dose Escalation Arm 1, Dose Level 2 - 3 X 10^7 Cells + Interleukin-21
Phase I Dose Escalation Arm 1 Dose Level 3 - 1 X 10^8 Cells + Interleukin-21
Phase I Dose Escalation Arm 1, Dose Level 4 - 3 X 10^8 Cells + Interleukin-21
Phase I Dose Escalation Arm 1, Dose Level 5 - 1 X 10^9 Cells + Interleukin-22
Phase I Dose Escalation Arm 1, Dose Level 6 - 3 X 10^9 Cells + Interleukin-21
Phase I Dose Escalation Arm 1, Dose Level 7 - 1 X 10^10 Cells + Interleukin-21
Phase I Dose Escalation Arm 1, Dose Level 8 - 3 X 10^10 Cells + Interleukin-21
Phase I Dose Escalation Arm 1, Dose Level 9 - 1 X 10^11 Cells + Interleukin-26
Phase 2 Arm 2, Cohort 1- Maximum Tolerated Dose + Interleukin-2 Other5
Phase 2 Arm 2, Cohort 2 - Maximum Tolerated Dose + Interleukin-2 Melanoma1

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Percentage of Participants Who Have a Clinical Response to Treatment (Objective Tumor Regression)

Percentage of participants who have a clinical response to treatment (objective tumor regression) measured by the Response Evaluation Criteria in Solid Tumors (RECIST)v1.0. Complete response is disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter of target lesions. Progression is at least a 20% increase in the sum of longest diameter of target lesions or the appearance of one or more new lesions. And stable disease is neither sufficient shrinkage to qualify for partial response nor sufficient increase to qualify for progressive disease. (NCT02111850)
Timeframe: 6 and 12 weeks after cell infusion, then every 3 months x3, then every 6 months x2 years, then per principal investigator discretion, approximately 6 years

,,,,,,,,,,
Interventionpercentage of participants (Number)
Complete ResponsePartial ResponseProgressionStable Disease
Phase 2 Arm 2, Cohort 1- Maximum Tolerated Dose + Interleukin-2 Other020800
Phase 2 Arm 2, Cohort 2 - Maximum Tolerated Dose + Interleukin-2 Melanoma001000
Phase I Dose Escalation Arm 1 Dose Level 3 - 1 X 10^8 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 1 - 1 X 10^7 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 2 - 3 X 10^7 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 4 - 3 X 10^8 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 5 - 1 X 10^9 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 6 - 3 X 10^9 Cells + Interleukin-2100000
Phase I Dose Escalation Arm 1, Dose Level 7 - 1 X 10^10 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 8 - 3 X 10^10 Cells + Interleukin-2001000
Phase I Dose Escalation Arm 1, Dose Level 9 - 1 X 10^11 Cells + Interleukin-2022.277.80

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Count of Participants With Serious and Non-Serious Adverse Events

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE) v3.0. A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02111863)
Timeframe: 2 years and 59 days

InterventionParticipants (Count of Participants)
Lymphocyte Depleting Prep Regimen6

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Overall Survival (OS) of Patients Receiving a Lymphocyte Depleting Preparative Regimen

OS is defined as the time between the first day of treatment to the day of death or date last known alive. (NCT02111863)
Timeframe: up to 3 years

Interventionmonths (Median)
Lymphocyte Depleting Prep Regimen12

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Objective Response Rate of Patients With Metastatic Melanoma

Objective response is defined as complete response + partial response and was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0. Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD. (NCT02111863)
Timeframe: approximately 2 years

Interventionpercentage of participants (Number)
Progressive DiseasePartial ResponseComplete ResponseStable Disease
Lymphocyte Depleting Prep Regimen83.3316.6700

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Number of Patients With Hematopoietic Engraftment

Engraftment is defined as absolute neutrophil count (ANC) ≥ 5 X 10^8/L for 3 consecutive measurements. (NCT02145039)
Timeframe: 42 days

InterventionParticipants (Count of Participants)
Haploidentical Stem Cell Transplant2

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2 Year Survival

Percentage of patients that survive 2 years post-transplant (NCT02145039)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Haploidentical Stem Cell Transplant0

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Number of Patients Experiencing Acute Graft-versus-host Disease by 100 Days

(NCT02145039)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Haploidentical Stem Cell Transplant1

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Number of Patients With Objective Tumor Regression

Objective tumor regression is defined as the number of participants with a complete or partial response per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0. Complete response is disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. (NCT02153905)
Timeframe: 6 and 12 weeks after cell infusion on up to 2 years

,
InterventionParticipants (Count of Participants)
Complete ResponsePartial Response
Anti-MAGE-A3 A1 TCR PBL 1x10^8 Cells + Interleukin-2 (IL-2)01
Anti-MAGE-A3 A1 TCR PBL 1x10^9 Cells + Interleukin-2 (IL-2)00

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Number of Participants With Dose-Limiting Toxicity (DLT)

DLT is defined as follows: Grade 3-5 allergic reactions related to the study cell infusion. Grade 3 and greater autoimmune reactions. Grades 3 and greater organ toxicity (cardiac, dermatologic, gastrointestinal, hepatic, pulmonary, renal/genitourinary, or neurologic) not pre-existing or due to the underlying malignancy and occurring within 30 days of study cell infusion and does not resolve within 72 hours. Treatment-related death within 8 weeks of the study cell infusion. (NCT02153905)
Timeframe: Within 30 days of study cell infusion

InterventionParticipants (Count of Participants)
Anti-MAGE-A3 A1 TCR PBL 1x10^9 Cells + Interleukin-2 (IL-2)1
Anti-MAGE-A3 A1 TCR PBL 1x10^8 Cells + Interleukin-2 (IL-2)0

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Number of Participants With Serious and Non-Serious Adverse Events

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02153905)
Timeframe: Date treatment consent signed to date off study, approximately 53 days for the Anti-MAGE-A3 A1 TCR PBL 1x10^9 Cells + Interleukin-2 (IL-2) Arm/Group, and 1 year and 4 months for the Anti-MAGE-A3 A1 TCR PBL 1x10^8 Cells + Interleukin-2 (IL-2) Arm/Group.

InterventionParticipants (Count of Participants)
Anti-MAGE-A3 A1 TCR PBL 1x10^9 Cells + Interleukin-2 (IL-2)1
Anti-MAGE-A3 A1 TCR PBL 1x10^8 Cells + Interleukin-2 (IL-2)2

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Number of Patients Who Had a Minimal Residual Disease (MRD) Negative Complete Response (CR) 2 Months After Chemotherapy

To determine the rate of minimal residual disease negative complete response (MRD negative CR) in the bone marrow at 2 months post last cycle of FCR, participants will have a bone marrow biopsy procedure 2 months after completing combination therapy (IPI-145+ FCR) in tandem with a chest,neck, abdomen and pelvic PET CT scan. A central read of the PET CT scan will confirm a radiographic complete response, and the bone marrow pathology and morphology assessments will confirm morphological CR in the bone marrow, while MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. This will include all patients treated and evaluable at maximum tolerated dose, and at the recommended phase II dose ( RP2D) (NCT02158091)
Timeframe: 2 months after completion of combination therapy of IPI-145 and FCR

InterventionPercentage of participants (Number)
Phase I MTD and Phase II RP2D: IPI 145 25 mg BID + FCR23

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Number of Patients Who Experienced a Dose Limiting Toxicity (DLT) During Phase I

To assess the safety of IPI145 in combination with FCR in previously untreated younger patients with CLL. DLT is based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0. DLT refers to toxicities experienced at any time during the study treatment, defined as Grade 3 or greater hematologic toxicity (except Grade 3 or Grade 4 neutropenia or thrombocytopenia that lasts less than or equal to 10 days off treatment), any Grade 3 or greater non-hematologic toxicity (except Grade 3 or greater nausea, vomiting, diarrhea, Grade 3 infusion reactions), Grade 3 asymptomatic laboratory abnormalities that improve to grade 2 or less within 3 days, Inability to receive day 1 therapy of Cycle 2 even after a three week treatment delay due to drug related toxicity from prior cycle, and any Grade 4 or greater elevation in AST ALT values (NCT02158091)
Timeframe: . Participants were assessed every week or more often as needed during Cycle 1, and every Day 1 Cycles 2 and onward-Dose-limiting toxicities (DLTs) occurring during the first cycle of treatment will be used in determining the Phase II MTD/RP2D

InterventionParticipants (Count of Participants)
Phase I Cohort 1: IPI-145 25mg Once Daily + FCR2
Phase I Cohort 2 (MTD): IPI-145 25mg Twice Daily + FCR1

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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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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 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 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 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 Best Response

Best response was assessed by the International Myeloma Working Group response criteria. Partial Remission (PR) is 50% or greater reduction of serum M-protein and 90% or greater reduction in 24-h urinary M-protein. Progressive Disease (PD) is increases of greater or equal to 25% from the lowest post-treatment (nadir) value in serum M-component or urine component or percentage of bone marrow plasma cells. Definite development of new bone lesions or new plasmacytoma. Very Good Partial Remission (VGPR) is serum and urine M-protein detectable by immunofixation but not on electrophoresis. Stringent Complete Remission (sCR) is normal free light chain ratio and absence of clonal cells in bone marrow by immunohistochemistry. Complete Remission is negative immunofixation on the serum and urine. Stable Disease (SD) is not meeting criteria for CR, VGPR, PR or PD. (NCT02215967)
Timeframe: From start of treatment up to 84 weeks

,,,
InterventionParticipants (Count of Participants)
Partial RemissionStable DiseaseVery Good Partial RemissionComplete RemissionStringent Complete RemissionProgressive Disease
0.3 x 10^6 CAR-BCMA T-cells Infused120000
1 x 10^6 CAR-BCMA T-cells Infused030000
3 x 10^6 CAR-BCMA T-cells Infused031000
9 x 10^6 CAR-BCMA T-cells Infused426121

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02215967)
Timeframe: Date treatment consent signed to date off study, approx. 3 mos and 7 days for DL 0.3 x 10^6 CAR + T cells, 4 mos and 4 days for 1.0 x 10^6 CAR + T cells, 9 mos and 13 days for 3.0 x 10^6 CAR + T cells, and 48 mos and 12 days for 9.0 x 10^6 CAR + T cells.

InterventionParticipants (Count of Participants)
0.3 x 10^6 CAR-BCMA T-cells Infused3
1 x 10^6 CAR-BCMA T-cells Infused3
3 x 10^6 CAR-BCMA T-cells Infused4
9 x 10^6 CAR-BCMA T-cells Infused16

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Number of Participants With Dose Limiting Toxicities

Dose limiting toxicities are defined as follows: Grade 3 toxicities possibly or probably related to either the anti-BCMA CAR T cells or the fludarabine and cyclophosphamide chemotherapy and lasting more than 7 days. Grade 4 toxicities possibly or probably related to the study interventions. (NCT02215967)
Timeframe: After the start of treatment and up to 60 days

,,,
InterventionParticipants (Count of Participants)
HypophosphatemiaConfusionDyspnea (intubated)Ejection fraction decreasedEncephalopathyHypoxiaAcute kidney injury (CVVH)Sepsis (Staph Aureus)Muscle weakness lower limbsMuscle weakness upper limbsPlatelet count decreasedNeutrophil count decreasedAcute kidney injuryHypotensionCPK increasedMusculoskeletal/connective tissue disorders-Rhabd.
0.3 x 10^6 CAR-BCMA T-cells Infused0000000000000000
1 x 10^6 CAR-BCMA T-cells Infused0000000000000000
3 x 10^6 CAR-BCMA T-cells Infused0000000000000000
9 x 10^6 CAR-BCMA T-cells Infused4344334433343443

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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 With Chronic GVHD at One Year

(NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant3

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

(NCT02224872)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Transplant14

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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 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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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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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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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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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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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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Participants Who Convert From Bone Marrow MRD Negativity to MRD Positivity in Participants Who Discontinue Ibrutinib

.MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008) (NCT02251548)
Timeframe: Response evaluated at 2 months post iFCR. Treatment up to 6 cycles (28 days each).

InterventionParticipants (Count of Participants)
FCR+Ibrutinib 420mg Daily0

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Rate of MRD Negative CR After 3 Cycles of iFCR

To determine the rate of minimal residual disease negative complete response (MRD negative CR) in the bone marrow after 3 cycles of ibrutinib + FCR. Participants will have a bone marrow biopsy procedure after completing 3 cycles in tandem with a chest, neck, abdomen and pelvic PET CT scan. A central read of the PET CT scan will confirm a radiographic complete response, and the bone marrow pathology and morphology assessments will confirm morphological CR in the bone marrow, while MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008) (NCT02251548)
Timeframe: After 3 cycles of iFCR for each patient completing 3 cycles

InterventionParticipants (Count of Participants)
FCR +420mg Ibrutinib Daily9

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Complete Response Rate (CRR)

CRR defined as the proportion of participants achieving complete response. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008). (NCT02251548)
Timeframe: Response evaluated at 2 months post iFCR. Treatment up to 6 cycles (28 days each).

Interventionproportion of participants (Number)
FCR+ 420 mg Ibrutinib Daily0.24

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Overall Response Rate

The objective response rate (ORR) was defined as the proportion of participants achieving CR+CRi+PR, Complete Response with incomplete count recovery (CRi) or partial response (PR) based on 2008 IW-CLL criteria criteria (Hallek et al., 2008). (NCT02251548)
Timeframe: Response evaluated at 2 months post iFCR. Treatment up to 6 cycles (28 days each).

Interventionproportion of participants (Number)
FCR+ 420 mg Ibrutinib Daily0.96

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Partial Response Rate (PRR)

PRR defined as the proportion of participants achieving partial response. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008). (NCT02251548)
Timeframe: Response evaluated at 2 months post iFCR. Treatment up to 6 cycles (28 days each).

Interventionproportion of participant (Number)
FCR+ 420 mg Ibrutinib Daily0.61

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1-year Combined Response With MRD From Bone Marrow

Combined response with MRD from bone marrow reported as the MRD-negative CR. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008) (NCT02251548)
Timeframe: at 1 year

InterventionParticipants (Count of Participants)
FCR+ 420 mg Ibrutinib Daily2

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Part I: Participants Who Achieve a Minimal Residual Disease (MRD) Negative Complete Response (CR) in the Bone Marrow at 2 Months Post FCR

To assess the number of participants who achieve an MRD negative complete response at the 2 months post last dose of FCR timepoint by 2008 IW-CLL criteria ( Hallek et. al). Participants will have a bone marrow biopsy procedure 2 months after completing combination therapy (Ibrutinib+ FCR) in tandem with a chest, neck, abdomen and pelvic PET CT scan. A central read of the PET CT scan will confirm a radiographic complete response, and the bone marrow pathology and morphology assessments will confirm morphological CR in the bone marrow, while MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. (NCT02251548)
Timeframe: 2 months after completing combination therapy

InterventionParticipants (Count of Participants)
FCR+ 420mg Ibrutinib Daily28

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Number of Participants Who Convert From Bone Marrow MRD Negative PR to Bone Marrow MRD Negative CR After 2 Years on Treatment

MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008)- Bone marrow biopsies will be performed at baseline, after cycle 3, 2 months post FCR, at 1 year and 2 years of ibrutinib maintenance, and as clinically indicated thereafter- Participants will have a bone marrow biopsy procedure 24 months after completing combination therapy (Ibrutinib+ FCR) in tandem with a chest, neck, abdomen and pelvic PET CT scan. A central read of the PET CT scan will confirm a radiographic complete response, and the bone marrow pathology and morphology assessments will confirm morphological CR in the bone marrow, while MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. (NCT02251548)
Timeframe: 2 years (24 months) from start of therapy

InterventionParticipants (Count of Participants)
FCR+ 420 mg Ibrutinib Daily9

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Number of Participants Who Convert From Bone Marrow MRD Negative PR to Bone Marrow MRD Negative CR After 1 Year of Ibrutinib Maintenance

To determine the rate of minimal residual disease negative complete response (MRD negative CR) in the bone marrow at 12 months from start of therapy, participants will have a bone marrow biopsy procedure at the 1 year timepoint after completing combination therapy (ibrutinib + FCR) in tandem with a chest, neck, abdomen and pelvic PET CT scan. A central read of the PET CT scan will confirm a radiographic complete response, and the bone marrow pathology and morphology assessments will confirm morphological CR in the bone marrow, while MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008) (NCT02251548)
Timeframe: 12 months ( 1year) after starting therapy

InterventionParticipants (Count of Participants)
FCR+ 420 mg Ibrutinib Daily2

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Median Overall Survival (OS)

Overall Survival (OS) based on the Kaplan-Meier method is defined as the time from study entry to death or censored at date last known alive. (NCT02251548)
Timeframe: Median follow-up is: 63.24 months (range: 6.83-95.8).

Interventionmonths (Median)
FCR+ 420 mg Ibrutinib DailyNA

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Median Progression-Free Survival (PFS)

Progression-free survival based on the Kaplan-Meier method is defined as the duration between randomization and documented disease progression (PD) or death, or is censored at time of last dsease assessment. (NCT02251548)
Timeframe: Disease will be evaluated through imaging cycle 3-6 day 1, and In long-term follow-up, after removal or until participant withdrawal, death, or removal from study. Median follow-up is: 63.24 months (range: 6.83-95.8).

Interventionmonths (Median)
FCR+ 420 mg Ibrutinib DailyNA

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Median Time to Bone Marrow MRD Negativity

MRD testing will be done by four-color flow cytometry on the bone marrow aspirate with a detection level of 10-4. Response and progression will be evaluated in this study using the 2008 IW-CLL criteria for CLL (Hallek et al., 2008)- (NCT02251548)
Timeframe: Bone marrow biopsies will be performed at baseline, after cycle 3, 2 months post FCR, at 1 year and 2 years of ibrutinib maintenance, and as clinically indicated thereafter

Interventionmonths (Median)
FCR+ 420 mg Ibrutinib Daily3

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Percentage of Participants Engrafted by Day 42 Post-transplant

To estimate engraftment by day +42 post-transplant in patients who receive CD45RA-depleted haploidentical donor progenitor cell transplantation following reduced intensity conditioning regimen that includes haploidentical NK cells. Engraftment is defined as the first of 3 consecutive tests performed on different days of an ANC ≥ 500/mm^3 with evidence of donor cell engraftment. (NCT02259348)
Timeframe: Day 42 post transplantation

Interventionpercentage of participants (Number)
Participants100

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Overall Survival (OS)

The Kaplan-Meier estimate of OS 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 transplant and all participants surviving at the time of analysis without events will be censored. The number of participants surviving to one-year post-transplantation is given. (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
Participants2

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Mean of Days to Absolute Neutrophil Count (ANC) Engraftment

ANC engraftment is defined as the first of 3 consecutive tests performed on different days of an ANC ≥ 500/mm^3 with evidence of donor cell engraftment. (NCT02259348)
Timeframe: Day 42 post transplantation

Interventiondays (Mean)
Participants10.3

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Incidence of Malignant Relapse

The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Death is the competing risk event. The number of participants with incidence of malignant relapse is given. Relapse was evaluated using standard WHO criteria for each disease. (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
Participants2

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Median Days to Absolute Neutrophil Count (ANC) Engraftment

ANC engraftment is defined as the first of 3 consecutive tests performed on different days of an ANC ≥ 500/mm^3 with evidence of donor cell engraftment. (NCT02259348)
Timeframe: Day 42 post transplantation

Interventiondays (Median)
Participants10

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Event-free Survival (EFS)

The Kaplan-Meier estimate of event-free survival (EFS) 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. (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
Participants2

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Incidence and Severity of Chronic GvHD

"The cumulative incidence of chronic GvHD will be estimated using Kalbfleisch-Prentice method. Death is the competing risk event. The severity of chronic GvHD will be described. Chronic GvHD was evaluated using NIH Consensus Global Severity Scoring. The number of participants with incidence by severity is given." (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
MildModerateSevere
Participants000

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Incidence and Severity of Acute GvHD

The cumulative incidence of acute GvHD will be estimated using Kalbfleisch-Prentice method. Death is the competing risk event. The severity of acute GvHD. The number of participants with incidence by grade is given. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe. (NCT02259348)
Timeframe: 100 days post transplantation

Interventionparticipants (Number)
Grade IGrade IIGrade IIIGrade IV
Participants0031

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Expression of Programmed Cell Death 1 (PD-1) by Circulating E6 T-Cell Receptor (TCR) T-Cells

Presence of PD-1 on circulating lymphocytes by flow cytometry one month after treatment. (NCT02280811)
Timeframe: one month after treatment

Intervention% PD-1 circulating lymphocytes (Mean)
HPV-16 E6 mTCR PBL 1x10^9 + HD IL-21
HPV-16 E6 mTCR PBL 1x10^10 + HD IL-22
HPV-16 E6 mTCR PBL 1x10^11 + HD IL-23
HPV-16 E6 mTCR PBL >1x10^11 up to 2x10^11 + HD IL-21
HPV-16 E6 mTCR PBL MTD + HD IL-22.2

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Maximum Tolerated Dose (MTD)

The MTD is the highest dose at which ≤1 of 6 patients experienced a dose limiting toxicity (DLT) or the highest dose level studied if DLTs are not observed at any of the dose levels. (NCT02280811)
Timeframe: participants were followed for the duration of hospital stay, an average of 3 weeks

Intervention# of cells x 10^11 (Number)
All Treated Subjects2

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Number of Participants With a Dose Limiting Toxicity (DLT)

A dose limiting toxicity is all Grade 3 and greater toxicities with the exception of myelosuppression, defined as lymphopenia, neutropenia, decreased hemoglobin, and thrombocytopenia, due to chemotherapy preparative regimen. Aldesleukin expected toxicities as defined in Appendix 2 and 3 of the protocol. Expected chemotherapy toxicities as defined in the pharmaceutical information section. Immediate hypersensitivity reactions (excluding symptomatic bronchospasm and grade 4 hypotension) occurring within 2 hours of cell infusion (related to cell infusion) that are reversible to a grade 2 or less within 24 hours of cell administration with standard therapy. Grade 3 fever. Events that are clearly related to the patient's disease. (NCT02280811)
Timeframe: 19 months and 7 days

InterventionParticipants (Count of Participants)
HPV-16 E6 mTCR PBL 1x10^9 + HD IL-20
HPV-16 E6 mTCR PBL 1x10^10 + HD IL-20
HPV-16 E6 mTCR PBL 1x10^11 + HD IL-20
HPV-16 E6 mTCR PBL >1x10^11 up to 2x10^11 + HD IL-20
HPV-16 E6 mTCR PBL MTD + HD IL-20

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Number of Participants With Serious and Non-serious Adverse Events

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v3.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02280811)
Timeframe: 19 months and 7 days

InterventionParticipants (Count of Participants)
HPV-16 E6 mTCR PBL 1x10^9 + HD IL-21
HPV-16 E6 mTCR PBL 1x10^10 + HD IL-22
HPV-16 E6 mTCR PBL 1x10^11 + HD IL-21
HPV-16 E6 mTCR PBL >1x10^11 up to 2x10^11 + HD IL-26
HPV-16 E6 mTCR PBL MTD + HD IL-22

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Objective Tumor Response Rate (Complete or Partial Response)

Objective tumor response rate is defined as the number of participants with a complete or partial response per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0. Complete response is disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. (NCT02280811)
Timeframe: 4 years

,,,,
Interventionparticipants (Number)
Complete Response (CR)Partial Response (PR)
HPV-16 E6 mTCR PBL >1x10^11 up to 2x10^11 + HD IL-202
HPV-16 E6 mTCR PBL 1x10^10 + HD IL-200
HPV-16 E6 mTCR PBL 1x10^11 + HD IL-200
HPV-16 E6 mTCR PBL 1x10^9 + HD IL-200
HPV-16 E6 mTCR PBL MTD + HD IL-200

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Percentage of Cluster of Differentiation 3 (CD3+) Cells That Are E6 T-Cell Receptor Memory of Circulating T-Cells in Responders and Non-responders

Detection of E6 TCR T cells in patients peripheral blood leukocytes (PBL)/apheresis samples by flow cytometry. (NCT02280811)
Timeframe: One month after treatment

,,,,
Interventionpercentage of cells (Mean)
RespondersNon-responders
HPV-16 E6 mTCR PBL >1x10^11 up to 2x10^11 + HD IL-23829.9
HPV-16 E6 mTCR PBL 1x10^10 + HD IL-2NA4.4
HPV-16 E6 mTCR PBL 1x10^11 + HD IL-2NA12.6
HPV-16 E6 mTCR PBL 1x10^9 + HD IL-2NA30.7
HPV-16 E6 mTCR PBL MTD + HD IL-2NA37.1

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Duration of Response

Duration of response is measured from the time measurement criteria are met for complete response or partial response (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.0. Complete response is disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. Progression is at least a 20% increase in the sum of target lesions, taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. (NCT02280811)
Timeframe: up to one year

Interventionmonths (Mean)
HPV-16 E6 mTCR PBL 1x10^9 + HD IL-2NA
HPV-16 E6 mTCR PBL 1x10^10 + HD IL-2NA
HPV-16 E6 mTCR PBL 1x10^11 + HD IL-2NA
HPV-16 E6 mTCR PBL >1x10^11 up to 2x10^11 + HD IL-21.5
HPV-16 E6 mTCR PBL MTD + HD IL-2NA

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To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD

Patient will have donor chimerism of greater than 20% and resolution of infection or autoimmunity at end of follow up (NCT02282904)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
Greater than 20% donor chimerismResolution of inflammation or infection
CGD Recipient77

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Phase 2 Pivotal Study (Cohorts 1 and 2): Best Overall Response Using IRRC Per Cheson 2007

The best overall response for each participant was based on the assessments of response (CR, PR, stable disease [SD], PD, and not done [ND]) made by the the IRRC using IWG 2007 criteria (Cheson et al, 2007). CR and PR as defined in outcome measure 2. PD defined by at least one of the following: ≥ 50% increase from nadir in the sum of the products of at least 2 lymph nodes, or at least a 50% increase in the product of the diameters of a single lymph node; appearance of a new lesion > 1.5 cm in any axis; ≥ 50% increase in size of splenic or hepatic nodules; ≥ 50% increase in the longest diameter of any single previously identified node > 1 cm in its short axis. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Percentage of participants with best overall response of CR, PR, SD, PD, and ND was reported. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff date of 27 January 2017 (maximum: 20 months)

,
Interventionpercentage of participants (Number)
CRPRSDPDND
Phase 2 (Pivotal Study): Cohort 149192183
Phase 2 (Pivotal Study): Cohort 258214413

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Pharmacokinetics: Peak Level of Anti-CD19 CAR T Cells in Blood

Peak was defined as the maximum number of CAR T cells measured post-infusion. (NCT02348216)
Timeframe: Enrollment up to Month 6

Interventioncells/µL (Median)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy58.512
Phase 2 (Pivotal Study): Cohort 131.512
Phase 2 (Pivotal Study): Cohort 258.633
Phase 2 (Safety Management Study): Cohort 353.670
Phase 2 (Safety Management Study): Cohort 452.91
Phase 2 (Safety Management Study): Cohort 526.63
Phase 2 (Safety Management Study): Cohort 664.38

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Phase 1 Study: Number of Participants Experiencing Adverse Events (AEs) Defined as Dose Limiting Toxicities (DLTs)

"DLT was defined as axicabtagene ciloleucel-related events with onset within first 30 days following infusion:~Grade (GR) 4 neutropenia lasting > 21 days and GR 4 thrombocytopenia lasting > 35 days from day of cell transfer;~Any axicabtagene ciloleucel-related AE requiring intubation;~All other GR 3 toxicities lasting > 3 days and all GR 4 toxicities, with exception of following conditions which were not considered DLTs: aphasia/dysphasia or confusion/cognitive disturbance which resolved to GR ≤ 1 within 2 weeks and to baseline within 4 weeks; fever GR 3; myelosuppression defined as lymphopenia, decreased hemoglobin, neutropenia and thrombocytopenia unless neutropenia and thrombocytopenia met DLT definition described above; immediate hypersensitivity reactions occurring within 2 hours of cell infusion that were reversible to a ≤ GR 2 within 24 hours of cell administration with standard therapy; hypogammaglobulinemia GR 3 or 4." (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel up to 30 days

InterventionParticipants (Count of Participants)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy1

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Phase 1 Study: ORR as Assessed by Investigator Per Revised IWG Response Criteria for Malignant Lymphoma

ORR was defined as the percentage of participants achieving either a CR or a PR, as assessed by the study investigators using revised IWG Response Criteria for Malignant Lymphoma (Cheson et al, 2007). CR: complete disappearance of all detectable clinical evidence of disease and disease-related symptoms; all lymph nodes and nodal masses must have regressed to normal size; spleen and/or liver must be normal size, not be palpable, and no nodules; bone marrow aspirate and biopsy must show no evidence of disease. PR: a ≥ 50% decrease in SPD of up to 6 of the largest dominant nodes or nodal masses; no increase in size of nodes, liver or spleen and no new sites of disease; multiple splenic and hepatic nodules (if present) must regress by ≥ 50% in the SPD; > 50% decrease in the greatest transverse diameter for single nodules. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff date of 27 January 2017 (maximum: 20 months)

Interventionpercentage of participants (Number)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy71

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Phase 2 Pivotal Study (Cohorts 1 and 2): Duration of Response (DOR) Using IRRC Per Cheson 2007

Among participants who experience an objective response, DOR was defined as the date of their first objective response (CR or PR which was subsequently confirmed) to PD per the revised IWG Response Criteria for Malignant Lymphoma or death regardless of cause. CR and PR as defined in outcome measure 2. PD was defined by at least one of the following: ≥ 50% increase from nadir in the sum of the products of at least 2 lymph nodes, or at least a 50% increase in the product of the diameters of a single lymph node; appearance of a new lesion > 1.5 cm in any axis; ≥ 50% increase in size of splenic or hepatic nodules; ≥ 50% increase in the longest diameter of any single previously identified node > 1 cm in its short axis. Kaplan-Meier (KM) estimates was used for analyses. (NCT02348216)
Timeframe: First objective response to the data cutoff date of 27 January 2017 (maximum: 20 months)

Interventionmonths (Median)
Phase 2 (Pivotal Study): Cohort 15.4
Phase 2 (Pivotal Study): Cohort 2NA

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Pharmacodynamics: Peak Level of Cytokine (Ferritin) in Serum (Phase 1 and Phase 2 Cohorts 1 and 2)

Peak was defined as the maximum post-baseline level of the cytokine. (NCT02348216)
Timeframe: Enrollment up to Week 4

Interventionpg/mL (Median)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy1973400.0
Phase 2 (Pivotal Study): Cohort 13681400.0
Phase 2 (Pivotal Study): Cohort 21979360.0

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Pharmacodynamics: Peak Level of Cytokine (CRP) in Serum

Peak was defined as the maximum post-baseline level of the cytokine. (NCT02348216)
Timeframe: Enrollment up to Week 4

Interventionmg/mL (Median)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy112.6
Phase 2 (Pivotal Study): Cohort 1215.7
Phase 2 (Pivotal Study): Cohort 2186.6
Phase 2 (Safety Management Study): Cohort 3137.8
Phase 2 (Safety Management Study): Cohort 4126.53
Phase 2 (Safety Management Study): Cohort 574.84
Phase 2 (Safety Management Study): Cohort 676.11

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Phase 2 Safety Management Study (Cohort 4): Percentage of Participants With Treatment-Emergent CRS and Neurologic Toxicities by Severity Grades

TEAE was defined as any AE with onset on or after the start of treatment. CRS events were graded by Lee et al 2014. Grade 1 : No life threatening symptoms and require symptomatic treatment only; Grade 2: Symptoms require and respond to moderate intervention; Grade 3: Symptoms require and respond to aggressive intervention; Grade 4: Life-threatening symptoms and requirements for ventilator support or CVVHD, and Grade 5: Death. Neurologic toxicities were graded by CTCAE version 4.03. Grade 1: Mild, asymptomatic or mild symptoms and intervention not indicated; Grade 2: Moderate and minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening, hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening and urgent intervention indicated; Grade 5: Death related to AE. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff of 06 May 2019 (maximum: 47.5 months)

Interventionpercentage of participants (Number)
Worst Grade 1 CRSWorst Grade 2 CRSWorst Grade 3 CRSWorst Grade 4 CRSWorst Grade 5 CRSWorst Grade ≥ 3 CRSWorst Grade 1 Neurologic ToxicitiesWorst Grade 2 Neurologic ToxicitiesWorst Grade 3 Neurologic ToxicitiesWorst Grade 4 Neurologic ToxicitiesWorst Grade 5 Neurologic ToxicitiesWorst Grade ≥ 3 Neurologic Toxicities
Phase 2 (Safety Management Study): Cohort 4325920023410170017

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Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 4 and Grade 3 or Higher Resulting From Decreased Parameter Value

Grading categories were determined by CTCAE version 4.03. Shifts to Grade 3 or higher has been reported for Phase 2 Cohort 3. For Phase 1 and all other cohorts of Phase 2, shifts to Grade 4 has been reported. (NCT02348216)
Timeframe: First infusion date to data cutoff 27 Jan 2017 (Phase 1,Cohorts 1,2), 26 Apr 2018, 6 May 2019, 10 Sep 2020, 16 Jun 2020 for Cohorts 3,4,5,6 respectively (maximum: 20 months for Phase 1,Cohorts 1,2; 35, 47.5, 64, 61 months for Cohorts 3,4,5,6 respectively)

,,,,,,
Interventionpercentage of participants (Number)
HemoglobinNeutrophilsPlateletsPotassiumCalciumMagnesiumSodiumAlanine AminotransferaseAspartate AminotransferaseBilirubinCreatinineUrateAlbuminLeukocytesDirect BilirubinGlucoseLymphocytesPhosphateAlkaline Phosphatase
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy01005700000000001000010000
Phase 2 (Pivotal Study): Cohort 1082350400000000840010000
Phase 2 (Pivotal Study): Cohort 20791701300000000750010000
Phase 2 (Safety Management Study): Cohort 3479768132401600000139703100420
Phase 2 (Safety Management Study): Cohort 409017270000000093007120
Phase 2 (Safety Management Study): Cohort 508638220200000088004640
Phase 2 (Safety Management Study): Cohort 607823030000000085009500

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Phase 2: Progression-Free Survival (PFS) as Assessed by Investigator Per Revised IWG Response Criteria for Malignant Lymphoma

PFS was defined as the time from the axicabtagene ciloleucel infusion date to the date of disease progression per the revised IWG Response Criteria for Malignant Lymphoma (Cheson et al, 2007) or death from any cause. Participants not meeting the criteria for progression by the analysis data cutoff date were censored at their last evaluable disease assessment date. Disease progression was defined by at least one of the following: ≥ 50% increase from nadir in the sum of the products of at least 2 lymph nodes, or at least a 50% increase in the product of the diameters of a single lymph node; appearance of a new lesion > 1.5 cm in any axis; ≥ 50% increase in size of splenic or hepatic nodules; ≥ 50% increase in the longest diameter of any single previously identified node > 1 cm in its short axis. KM estimates was used for analyses. (NCT02348216)
Timeframe: First infusion date to PD or death or data cutoff date 27 Jan 2017, 26 Apr 2018, 06 May 2019, 10 Sep 2020, and 16 Jun 2020 for Cohorts 1 and 2, 3, 4, 5, 6 respectively (maximum: 20, 35, 47.5, 64, and 61 months for Cohorts 1 and 2, 3, 4, 5, 6 respectively)

Interventionmonths (Median)
Phase 2 (Pivotal Study): Cohort 15.1
Phase 2 (Pivotal Study): Cohort 2NA
Phase 2 (Safety Management Study): Cohort 36.2
Phase 2 (Safety Management Study): Cohort 4NA
Phase 2 (Safety Management Study): Cohort 53.1
Phase 2 (Safety Management Study): Cohort 6NA

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Phase 2 Safety Management Study: Number of Participants With the European Quality of Life Five Dimension Five Level Scale (EQ-5D) Score

"EQ-5D is a self-reported questionnaire used for assessing the overall health status of a participant scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was divided into 5 levels of severity: No problem, Slight problems, Moderate problems, Severe problems, and Extreme problems (unable to perform). EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state)." (NCT02348216)
Timeframe: Baseline, Week 4, Month 3, and Month 6

InterventionParticipants (Count of Participants)
Baseline: Mobility72294096Baseline: Mobility72294104Baseline: Mobility72294105Baseline: Mobility72294106Week 4: Mobility72294105Week 4: Mobility72294106Week 4: Mobility72294104Week 4: Mobility72294096Month 3: Mobility72294105Month 3: Mobility72294106Month 3: Mobility72294096Month 3: Mobility72294104Month 6: Mobility72294096Month 6: Mobility72294104Month 6: Mobility72294105Month 6: Mobility72294106Baseline: Self-care72294096Baseline: Self-care72294106Baseline: Self-care72294104Baseline: Self-care72294105Week 4: Self-care72294104Week 4: Self-care72294105Week 4: Self-care72294096Week 4: Self-care72294106Month 3: Self-care72294104Month 3: Self-care72294106Month 3: Self-care72294105Month 3: Self-care72294096Month 6: Self-care72294096Month 6: Self-care72294106Month 6: Self-care72294104Month 6: Self-care72294105Baseline: Usual activities72294096Baseline: Usual activities72294106Baseline: Usual activities72294104Baseline: Usual activities72294105Week 4: Usual activities72294096Week 4: Usual activities72294105Week 4: Usual activities72294106Week 4: Usual activities72294104Month 3: Usual activities72294106Month 3: Usual activities72294096Month 3: Usual activities72294104Month 3: Usual activities72294105Month 6: Usual activities72294104Month 6: Usual activities72294105Month 6: Usual activities72294096Month 6: Usual activities72294106Baseline: Pain/Discomfort72294104Baseline: Pain/Discomfort72294105Baseline: Pain/Discomfort72294106Baseline: Pain/Discomfort72294096Week 4: Pain/Discomfort72294096Week 4: Pain/Discomfort72294105Week 4: Pain/Discomfort72294104Week 4: Pain/Discomfort72294106Month 3: Pain/Discomfort72294096Month 3: Pain/Discomfort72294105Month 3: Pain/Discomfort72294104Month 3: Pain/Discomfort72294106Month 6: Pain/Discomfort72294096Month 6: Pain/Discomfort72294104Month 6: Pain/Discomfort72294105Month 6: Pain/Discomfort72294106Baseline: Anxiety/Depression72294096Baseline: Anxiety/Depression72294105Baseline: Anxiety/Depression72294104Baseline: Anxiety/Depression72294106Week 4: Anxiety/Depression72294104Week 4: Anxiety/Depression72294096Week 4: Anxiety/Depression72294105Week 4: Anxiety/Depression72294106Month 3: Anxiety/Depression72294105Month 3: Anxiety/Depression72294096Month 3: Anxiety/Depression72294106Month 3: Anxiety/Depression72294104Month 6: Anxiety/Depression72294096Month 6: Anxiety/Depression72294104Month 6: Anxiety/Depression72294105Month 6: Anxiety/Depression72294106
No problemSlight problemSevere problemUnable to performModerate problem
Phase 2 (Safety Management Study): Cohort 330
Phase 2 (Safety Management Study): Cohort 425
Phase 2 (Safety Management Study): Cohort 533
Phase 2 (Safety Management Study): Cohort 626
Phase 2 (Safety Management Study): Cohort 49
Phase 2 (Safety Management Study): Cohort 45
Phase 2 (Safety Management Study): Cohort 57
Phase 2 (Safety Management Study): Cohort 316
Phase 2 (Safety Management Study): Cohort 421
Phase 2 (Safety Management Study): Cohort 523
Phase 2 (Safety Management Study): Cohort 620
Phase 2 (Safety Management Study): Cohort 311
Phase 2 (Safety Management Study): Cohort 59
Phase 2 (Safety Management Study): Cohort 65
Phase 2 (Safety Management Study): Cohort 34
Phase 2 (Safety Management Study): Cohort 54
Phase 2 (Safety Management Study): Cohort 30
Phase 2 (Safety Management Study): Cohort 31
Phase 2 (Safety Management Study): Cohort 314
Phase 2 (Safety Management Study): Cohort 420
Phase 2 (Safety Management Study): Cohort 622
Phase 2 (Safety Management Study): Cohort 42
Phase 2 (Safety Management Study): Cohort 310
Phase 2 (Safety Management Study): Cohort 511
Phase 2 (Safety Management Study): Cohort 615
Phase 2 (Safety Management Study): Cohort 337
Phase 2 (Safety Management Study): Cohort 438
Phase 2 (Safety Management Study): Cohort 544
Phase 2 (Safety Management Study): Cohort 632
Phase 2 (Safety Management Study): Cohort 41
Phase 2 (Safety Management Study): Cohort 53
Phase 2 (Safety Management Study): Cohort 325
Phase 2 (Safety Management Study): Cohort 433
Phase 2 (Safety Management Study): Cohort 531
Phase 2 (Safety Management Study): Cohort 624
Phase 2 (Safety Management Study): Cohort 35
Phase 2 (Safety Management Study): Cohort 56
Phase 2 (Safety Management Study): Cohort 63
Phase 2 (Safety Management Study): Cohort 60
Phase 2 (Safety Management Study): Cohort 319
Phase 2 (Safety Management Study): Cohort 429
Phase 2 (Safety Management Study): Cohort 532
Phase 2 (Safety Management Study): Cohort 625
Phase 2 (Safety Management Study): Cohort 317
Phase 2 (Safety Management Study): Cohort 67
Phase 2 (Safety Management Study): Cohort 322
Phase 2 (Safety Management Study): Cohort 422
Phase 2 (Safety Management Study): Cohort 524
Phase 2 (Safety Management Study): Cohort 616
Phase 2 (Safety Management Study): Cohort 46
Phase 2 (Safety Management Study): Cohort 62
Phase 2 (Safety Management Study): Cohort 36
Phase 2 (Safety Management Study): Cohort 412
Phase 2 (Safety Management Study): Cohort 612
Phase 2 (Safety Management Study): Cohort 313
Phase 2 (Safety Management Study): Cohort 411
Phase 2 (Safety Management Study): Cohort 513
Phase 2 (Safety Management Study): Cohort 611
Phase 2 (Safety Management Study): Cohort 58
Phase 2 (Safety Management Study): Cohort 64
Phase 2 (Safety Management Study): Cohort 61
Phase 2 (Safety Management Study): Cohort 32
Phase 2 (Safety Management Study): Cohort 38
Phase 2 (Safety Management Study): Cohort 416
Phase 2 (Safety Management Study): Cohort 519
Phase 2 (Safety Management Study): Cohort 415
Phase 2 (Safety Management Study): Cohort 47
Phase 2 (Safety Management Study): Cohort 52
Phase 2 (Safety Management Study): Cohort 51
Phase 2 (Safety Management Study): Cohort 50
Phase 2 (Safety Management Study): Cohort 318
Phase 2 (Safety Management Study): Cohort 417
Phase 2 (Safety Management Study): Cohort 516
Phase 2 (Safety Management Study): Cohort 614
Phase 2 (Safety Management Study): Cohort 312
Phase 2 (Safety Management Study): Cohort 520
Phase 2 (Safety Management Study): Cohort 617
Phase 2 (Safety Management Study): Cohort 510
Phase 2 (Safety Management Study): Cohort 69
Phase 2 (Safety Management Study): Cohort 37
Phase 2 (Safety Management Study): Cohort 39
Phase 2 (Safety Management Study): Cohort 410
Phase 2 (Safety Management Study): Cohort 414
Phase 2 (Safety Management Study): Cohort 68
Phase 2 (Safety Management Study): Cohort 423
Phase 2 (Safety Management Study): Cohort 518
Phase 2 (Safety Management Study): Cohort 621
Phase 2 (Safety Management Study): Cohort 413
Phase 2 (Safety Management Study): Cohort 517
Phase 2 (Safety Management Study): Cohort 43
Phase 2 (Safety Management Study): Cohort 40
Phase 2 (Safety Management Study): Cohort 521
Phase 2 (Safety Management Study): Cohort 623
Phase 2 (Safety Management Study): Cohort 315
Phase 2 (Safety Management Study): Cohort 512
Phase 2 (Safety Management Study): Cohort 55
Phase 2 (Safety Management Study): Cohort 419
Phase 2 (Safety Management Study): Cohort 619
Phase 2 (Safety Management Study): Cohort 48
Phase 2 (Safety Management Study): Cohort 44
Phase 2 (Safety Management Study): Cohort 618
Phase 2 (Safety Management Study): Cohort 33

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Phase 2 Pivotal Study (Cohorts 1 and 2): ORR Per Independent Radiological Review Committee (IRRC)

ORR was defined as the percentage of participants achieving either a CR or a PR, as assessed by the IRRC using revised IWG Response Criteria for Malignant Lymphoma (Cheson et al, 2007). CR: complete disappearance of all detectable clinical evidence of disease and disease-related symptoms; all lymph nodes and nodal masses must have regressed to normal size; spleen and/or liver must be normal size, not be palpable, and no nodules; bone marrow aspirate and biopsy must show no evidence of disease. PR: a ≥ 50% decrease in SPD of up to 6 of the largest dominant nodes or nodal masses; no increase in size of nodes, liver or spleen and no new sites of disease; multiple splenic and hepatic nodules (if present) must regress by ≥ 50% in the SPD; > 50% decrease in the greatest transverse diameter for single nodules. 95% CI was calculated by Clopper-Pearson method. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff date of 27 January 2017 (maximum: 20 months)

Interventionpercentage of participants (Number)
Phase 2 (Pivotal Study): Cohort 169
Phase 2 (Pivotal Study): Cohort 279

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Pharmacodynamics: Peak Level of Cytokine (Ferritin) in Serum (Phase 2 Cohort 3)

Peak was defined as the maximum post-baseline level of the cytokine. (NCT02348216)
Timeframe: Enrollment up to Week 4

Interventionng/mL (Median)
Phase 2 (Safety Management Study): Cohort 32440.2

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Phase 2 Pivotal Study (Cohorts 1 and 2): Overall Response Rate (ORR) as Assessed by Investigator Per Revised International Working Group (IWG) Response Criteria for Malignant Lymphoma

ORR was defined as the percentage of participants achieving either a complete response (CR) or a partial response (PR), as assessed by the study investigators using revised IWG Response Criteria for Malignant Lymphoma (Cheson et al, 2007). CR: complete disappearance of all detectable clinical evidence of disease and disease-related symptoms; all lymph nodes and nodal masses must have regressed to normal size; spleen and/or liver must be normal size, not be palpable, and no nodules; bone marrow aspirate and biopsy must show no evidence of disease. PR: a ≥ 50% decrease in sum of the product of the diameters (SPD) of up to 6 of the largest dominant nodes or nodal masses; no increase in size of nodes, liver or spleen and no new sites of disease; multiple splenic and hepatic nodules (if present) must regress by ≥ 50% in the SPD; > 50% decrease in the greatest transverse diameter for single nodules. 95% confidence interval (CI) was calculated by Clopper-Pearson method. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff date of 27 January 2017 (maximum: 20 months)

Interventionpercentage of participants (Number)
Phase 2 (Pivotal Study): Cohort 182
Phase 2 (Pivotal Study): Cohort 283

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Phase 2 Pivotal Study (Cohorts 1 and 2): PFS Using IRRC Per Cheson 2007

PFS was defined as the time from the axicabtagene ciloleucel infusion date to the date of disease progression per the revised IWG Response Criteria for Malignant Lymphoma (Cheson et al, 2007) or death from any cause. Participants not meeting the criteria for progression by the analysis data cutoff date were censored at their last evaluable disease assessment date. PD defined by at least one of the following: ≥ 50% increase from nadir in the sum of the products of at least 2 lymph nodes, or at least a 50% increase in the product of the diameters of a single lymph node; appearance of a new lesion > 1.5 cm in any axis; ≥ 50% increase in size of splenic or hepatic nodules; ≥ 50% increase in the longest diameter of any single previously identified node > 1 cm in its short axis. KM estimates was used for analyses. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the date of disease progression or death from any cause or the data cutoff date of 27 January 2017 (maximum: 20 months)

Interventionmonths (Median)
Phase 2 (Pivotal Study): Cohort 15.0
Phase 2 (Pivotal Study): Cohort 2NA

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Percentage of Participants With Positive Replication Competent Retrovirus (RCR)

RCR was analyzed in blood samples by central laboratory. Because axicabtagene ciloleucel comprised retroviral vector transduced T cells, the presence of RCR in the blood of treated participants was reported. (NCT02348216)
Timeframe: Day 0 (pre-infusion) to data cutoff 27 Jan 2017 (Phase 1,Cohorts 1,2), 26 Apr 2018, 6 May 2019, 10 Sep 2020, 16 Jun 2020 for Cohorts 3,4,5,6 respectively (maximum:20 months for Phase 1,Cohorts 1,2; 35, 47.5, 64, 61 months for Cohorts 3,4,5,6 respectively)

Interventionpercentage of participants (Number)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy0
Phase 2 (Pivotal Study): Cohort 10
Phase 2 (Pivotal Study): Cohort 20
Phase 2 (Safety Management Study): Cohort 30
Phase 2 (Safety Management Study): Cohort 40
Phase 2 (Safety Management Study): Cohort 50
Phase 2 (Safety Management Study): Cohort 60

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Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAEs)

An adverse event was defined as any untoward medical occurrence in a clinical trial participants. The event did not necessarily have a relationship with study treatment. Adverse events included worsening of a pre-existing medical condition. Worsening indicated that the pre-existing medical condition had increased in severity, frequency, and/or duration or had an association with a worse outcome. A pre-existing condition that had not worsened during the study or involved an intervention such as elective cosmetic surgery or a medical procedure while on study, was not considered an adverse event. TEAE was defined as any AE with onset on or after the start of treatment. (NCT02348216)
Timeframe: First infusion date to data cutoff 27 Jan 2017 (Phase 1,Cohorts 1,2), 26 Apr 2018, 6 May 2019, 10 Sep 2020, 16 Jun 2020 for Cohorts 3,4,5,6 respectively (maximum: 20 months for Phase 1,Cohorts 1,2; 35, 47.5, 64, 61 months for Cohorts 3,4,5,6 respectively)

Interventionpercentage of participants (Number)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy100
Phase 2 (Pivotal Study): Cohort 1100
Phase 2 (Pivotal Study): Cohort 2100
Phase 2 (Safety Management Study): Cohort 3100
Phase 2 (Safety Management Study): Cohort 4100
Phase 2 (Safety Management Study): Cohort 5100
Phase 2 (Safety Management Study): Cohort 6100

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Percentage of Participants With Anti-Axicabtagene Ciloleucel Antibodies

(NCT02348216)
Timeframe: First infusion date to data cutoff 27 Jan 2017 (Phase 1,Cohorts 1,2), 26 Apr 2018, 6 May 2019, 10 Sep 2020, 16 Jun 2020 for Cohorts 3,4,5,6 respectively (maximum: 20 months for Phase 1,Cohorts 1,2; 35, 47.5, 64, 61 months for Cohorts 3,4,5,6 respectively)

Interventionpercentage of participants (Number)
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy0
Phase 2 (Pivotal Study): Cohort 14
Phase 2 (Pivotal Study): Cohort 20
Phase 2 (Safety Management Study): Cohort 35
Phase 2 (Safety Management Study): Cohort 40
Phase 2 (Safety Management Study): Cohort 58
Phase 2 (Safety Management Study): Cohort 68

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Phase 2 Safety Management Study (Cohorts 3, 4, 5, and 6): ORR as Assessed by Investigator Per the Revised IWG Response Criteria for Malignant Lymphoma

ORR was defined as the percentage of participants achieving either a CR or a PR, as assessed by the study investigators using revised IWG Response Criteria for Malignant Lymphoma (Cheson et al, 2007). CR: complete disappearance of all detectable clinical evidence of disease and disease-related symptoms; all lymph nodes and nodal masses must have regressed to normal size; spleen and/or liver must be normal size, not be palpable, and no nodules; bone marrow aspirate and biopsy must show no evidence of disease. PR: a ≥ 50% decrease in SPD of up to 6 of the largest dominant nodes or nodal masses; no increase in size of nodes, liver or spleen and no new sites of disease; multiple splenic and hepatic nodules (if present) must regress by ≥ 50% in the SPD; > 50% decrease in the greatest transverse diameter for single nodules. 95% CI was calculated by Clopper-Pearson method. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff date of 26 Apr 2018, 06 May 2019, 10 Sep 2020, and 16 Jun 2020 for Cohorts 3, 4, 5, and 6 respectively (maximum: 35, 47.5, 64, and 61 months for Cohorts 3, 4, 5, and 6 respectively)

Interventionpercentage of participants (Number)
Phase 2 (Safety Management Study): Cohort 363
Phase 2 (Safety Management Study): Cohort 473
Phase 2 (Safety Management Study): Cohort 572
Phase 2 (Safety Management Study): Cohort 695

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Phase 2 Safety Management Study: EQ-5D Visual Analogue Scale (VAS) Score

EQ-5D is a self-reported questionnaire used for assessing the overall health status of a participant. The EQ-5D-VAS records the participant's self-rated health on a vertical visual analogue scale and is asked to make a global assessment of their current state of health with 0 indicating the worst health they can imagine and 100 indicating the best health they can imagine. (NCT02348216)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,,,
Interventionunits on a scale (Mean)
BaselineWeek 4Month 3Month 6
Phase 2 (Safety Management Study): Cohort 371.267.874.977.1
Phase 2 (Safety Management Study): Cohort 469.567.278.885.1
Phase 2 (Safety Management Study): Cohort 566.770.873.377.1
Phase 2 (Safety Management Study): Cohort 670.976.176.579.8

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Phase 2: Duration of Response (DOR) as Assessed by Investigator Per Revised IWG Response Criteria for Malignant Lymphoma

Among participants who experience an objective response (OR), DOR was defined as the date of their first objective response (CR or PR which was subsequently confirmed) to disease progression per the revised IWG Response Criteria for Malignant Lymphoma or death regardless of cause. CR and PR as defined in outcome measure 2. Disease progression (PD) was defined by at least one of the following: ≥ 50% increase from nadir in the sum of the products of at least 2 lymph nodes, or at least a 50% increase in the product of the diameters of a single lymph node; appearance of a new lesion > 1.5 cm in any axis; ≥ 50% increase in size of splenic or hepatic nodules; ≥ 50% increase in the longest diameter of any single previously identified node > 1 cm in its short axis. Kaplan-Meier (KM) estimates was used for analyses. (NCT02348216)
Timeframe: First OR to data cutoff date of 27 Jan 2017, 26 Apr 2018, 6 May 2019, 10 Sep 2010, and 16 Jun 2020 for Cohorts 1 and 2, 3, 4, 5, and 6 respectively (median duration: 5.3, 4.9, 11.1, 5.2, 11.4, and 5.8 months for Cohorts 1, 2, 3, 4, 5, and 6 respectively)

Interventionmonths (Median)
Phase 2 (Pivotal Study): Cohort 14.2
Phase 2 (Pivotal Study): Cohort 2NA
Phase 2 (Safety Management Study): Cohort 3NA
Phase 2 (Safety Management Study): Cohort 4NA
Phase 2 (Safety Management Study): Cohort 5NA
Phase 2 (Safety Management Study): Cohort 6NA

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Phase 2: Overall Survival (OS)

OS was defined as the time from axicabtagene ciloleucel infusion to the date of death. Participants who did not die by the analysis data cutoff date were censored at their last contact date. KM estimates was used for analyses. (NCT02348216)
Timeframe: First infusion date to the death or data cutoff date of 27 Jan 2017, 26 Apr 2018, 6 May 2019, 10 Sep 2020, and 16 Jun 2020 for Cohorts 1 and 2, 3, 4, 5, 6 respectively (maximum: 20, 35, 47.5, 64, and 61 months for Cohorts 1 and 2, 3, 4, 5, 6 respectively)

Interventionmonths (Median)
Phase 2 (Pivotal Study): Cohort 111.5
Phase 2 (Pivotal Study): Cohort 2NA
Phase 2 (Safety Management Study): Cohort 315.4
Phase 2 (Safety Management Study): Cohort 4NA
Phase 2 (Safety Management Study): Cohort 514.6
Phase 2 (Safety Management Study): Cohort 6NA

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Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 4 and Grade 3 or Higher Resulting From Increased Parameter Value

Grading categories were determined by CTCAE version 4.03. Shifts to Grade 3 or higher has been reported for Phase 2 Cohort 3. For Phase 1 and all other cohorts of Phase 2, shifts to Grade 4 has been reported. (NCT02348216)
Timeframe: First infusion date to data cutoff 27 Jan 2017 (Phase 1,Cohorts 1,2), 26 Apr 2018, 6 May 2019, 10 Sep 2020, 16 Jun 2020 for Cohorts 3,4,5,6 respectively (maximum: 20 months for Phase 1,Cohorts 1,2; 35, 47.5, 64, 61 months for Cohorts 3,4,5,6 respectively)

,,,,,,
Interventionpercentage of participants (Number)
HemoglobinNeutrophilsPlateletsPotassiumCalciumMagnesiumSodiumAlanine AminotransferaseAspartate AminotransferaseBilirubinCreatinineUrateAlbuminLeukocytesDirect BilirubinGlucoseLymphocytesPhosphateAlkaline Phosphatase
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy00000001400000000000
Phase 2 (Pivotal Study): Cohort 10000100101100000000
Phase 2 (Pivotal Study): Cohort 20000000000400000000
Phase 2 (Safety Management Study): Cohort 300033802116551300188005
Phase 2 (Safety Management Study): Cohort 40002200020020002000
Phase 2 (Safety Management Study): Cohort 50000000000000000000
Phase 2 (Safety Management Study): Cohort 60000050000330000000

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Phase 2 Safety Management Study (Cohort 3): Percentage of Participants With Treatment-Emergent Cytokine Release Syndrome (CRS) and Neurologic Toxicities by Severity Grades

TEAE was defined as any AE with onset on or after the start of treatment. CRS events were graded by Lee et al 2014. Grade 1 : No life threatening symptoms and require symptomatic treatment only; Grade 2: Symptoms require and respond to moderate intervention; Grade 3: Symptoms require and respond to aggressive intervention; Grade 4: Life-threatening symptoms and requirements for ventilator support or continuous venovenous hemodialysis (CVVHD), and Grade 5: Death. Neurologic toxicities were graded by Common Terminology Criteria for Adverse Events (CTCAE) version 4.03. Grade 1: Mild, asymptomatic or mild symptoms and intervention not indicated; Grade 2: Moderate and minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening, hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening and urgent intervention indicated; Grade 5: Death related to AE. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff of 26 April 2018 (maximum: 35 months)

Interventionpercentage of participants (Number)
Worst Grade 1 CRSWorst Grade 2 CRSWorst Grade 3 CRSWorst Grade 4 CRSWorst Grade 5 CRSWorst Grade ≥ 3 CRSWorst Grade 1 Neurologic ToxicitiesWorst Grade 2 Neurologic ToxicitiesWorst Grade 3 Neurologic ToxicitiesWorst Grade 4 Neurologic ToxicitiesWorst Grade 5 Neurologic ToxicitiesWorst Grade ≥ 3 Neurologic Toxicities
Phase 2 (Safety Management Study): Cohort 3345503032424343339

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Phase 2 Safety Management Study (Cohort 5): Percentage of Participants With Treatment-Emergent CRS and Neurologic Toxicities by Severity Grades

TEAE was defined as any AE with onset on or after the start of treatment. CRS events were graded by Lee et al 2014. Grade 1 : No life threatening symptoms and require symptomatic treatment only; Grade 2: Symptoms require and respond to moderate intervention; Grade 3: Symptoms require and respond to aggressive intervention; Grade 4: Life-threatening symptoms and requirements for ventilator support or CVVHD, and Grade 5: Death. Neurologic toxicities were graded by CTCAE version 4.03. Grade 1: Mild, asymptomatic or mild symptoms and intervention not indicated; Grade 2: Moderate and minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening, hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening and urgent intervention indicated; Grade 5: Death related to AE. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff of 10 September 2020 (maximum: 64 months)

Interventionpercentage of participants (Number)
Worst Grade 1 CRSWorst Grade 2 CRSWorst Grade 3 CRSWorst Grade 4 CRSWorst Grade 5 CRSWorst Grade ≥ 3 CRSWorst Grade 1 Neurologic ToxicitiesWorst Grade 2 Neurologic ToxicitiesWorst Grade 3 Neurologic ToxicitiesWorst Grade 4 Neurologic ToxicitiesWorst Grade 5 Neurologic ToxicitiesWorst Grade ≥ 3 Neurologic Toxicities
Phase 2 (Safety Management Study): Cohort 5384602022618102012

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Pharmacodynamics: Peak Level of Cytokines (Ferritin, ICAM-1, IL-2 R, Perforin, and VCAM-1) in Serum (Phase 2 Cohorts 4, 5, and 6)

Peak was defined as the maximum post-baseline level of the cytokine. Following key cytokines were measured: Ferritin, ICAM-1, IL-2 R, Perforin, and VCAM-1. (NCT02348216)
Timeframe: Enrollment up to Week 4

,,
Interventionng/mL (Median)
FerritinICAM-1IL-2 RPerforinVCAM-1
Phase 2 (Safety Management Study): Cohort 41086.36907.9710.7817.221255.32
Phase 2 (Safety Management Study): Cohort 51516.11636.747.8210.85854.63
Phase 2 (Safety Management Study): Cohort 6903.50654.816.4310.12836.04

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Phase 2 Safety Management Study (Cohort 6): Percentage of Participants With Treatment-Emergent CRS and Neurologic Toxicities by Severity Grades

TEAE was defined as any AE with onset on or after the start of treatment. CRS events were graded by Lee et al 2014. Grade 1 : No life threatening symptoms and require symptomatic treatment only; Grade 2: Symptoms require and respond to moderate intervention; Grade 3: Symptoms require and respond to aggressive intervention; Grade 4: Life-threatening symptoms and requirements for ventilator support or CVVHD, and Grade 5: Death. Neurologic toxicities were graded by CTCAE version 4.03. Grade 1: Mild, asymptomatic or mild symptoms and intervention not indicated; Grade 2: Moderate and minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening, hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening and urgent intervention indicated; Grade 5: Death related to AE. (NCT02348216)
Timeframe: First infusion date of axicabtagene ciloleucel to the data cutoff of 16 June 2020 (maximum: 61 months)

Interventionpercentage of participants (Number)
Worst Grade 1 CRSWorst Grade 2 CRSWorst Grade 3 CRSWorst Grade 4 CRSWorst Grade 5 CRSWorst Grade ≥ 3 CRSWorst Grade 1 Neurologic ToxicitiesWorst Grade 2 Neurologic ToxicitiesWorst Grade 3 Neurologic ToxicitiesWorst Grade 4 Neurologic ToxicitiesWorst Grade 5 Neurologic ToxicitiesWorst Grade ≥ 3 Neurologic Toxicities
Phase 2 (Safety Management Study): Cohort 635450000251885315

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Pharmacodynamics: Peak Level of Cytokines (IP-10, Granzyme B, IFN-gamma, IL-1 RA, IL-10, IL-15, IL-2, IL-6, IL-7, IL-8, TNF Alpha, and GM-CSF) in Serum (Phase 2 Cohorts 4, 5, and 6)

Peak was defined as the maximum post-baseline level of the cytokine. Following key cytokines were measured: IP-10, granzyme B, IFN-gamma, IL-1 RA, IL-2, IL-6, IL-7, IL-8, IL-10, IL-15, TNF alpha, and granulocyte-macrophage colony-stimulating factor (GM-CSF). (NCT02348216)
Timeframe: Enrollment up to Week 4

,,
Interventionpg/mL (Median)
IP-10Granzyme BIFN-gammaIL-1 RAIL-2IL-6IL-7IL-8IL-10IL-15TNF alphaGM-CSF
Phase 2 (Safety Management Study): Cohort 41549.7023.10334.501093.7011.20136.7033.1067.4019.6045.805.704.40
Phase 2 (Safety Management Study): Cohort 51746.1527.90314.90908.0011.8597.9529.8075.1014.4534.155.252.90
Phase 2 (Safety Management Study): Cohort 61560.0318.40207.951279.508.4047.2528.2552.5513.3037.204.801.90

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Pharmacodynamics: Peak Level of Cytokines in Serum (Phase 1 and Phase 2 Cohorts 1, 2, and 3)

Peak was defined as the maximum post-baseline level of the cytokine. Following key cytokines were measured: interferon-gamma induced protein 10 (IP-10), ferritin, granzyme B, intercellular adhesion molecule (ICAM-1), interferon-gamma (IFN-gamma), interleukin-1 receptor antagonist (IL-1RA), IL-2, interleukin-2 receptor alpha (IL-2 R alpha), IL-6, IL-7, IL-8, IL-10, IL-15, perforin, tumor necrosis factor alpha (TNF alpha), and vascular cell adhesion molecule- 1 (VCAM-1). (NCT02348216)
Timeframe: Enrollment up to Week 4

,,,
Interventionpg/mL (Median)
IP-10Granzyme BICAM-1IFN-gammaIL-1 RAIL-2IL-2 R alphaIL-6IL-7IL-8IL-10IL-15PerforinTNF alphaVCAM-1
Phase 1 Study: Axicabtagene Ciloleucel and Conditioning Chemotherapy2000.033.1792754.3792.02173.318.416872.7305.351.586.452.557.15389.010.51387033.6
Phase 2 (Pivotal Study): Cohort 12000.031.11322829.3493.82371.225.014383.789.438.9118.443.956.511309.58.61478356.8
Phase 2 (Pivotal Study): Cohort 22000.017.3989188.4364.91999.913.47817.344.644.177.218.847.68278.76.81058453.9
Phase 2 (Safety Management Study): Cohort 32000.044.11009966.41857.22160.520.012386.4921.838.8120.948.250.315411.910.91367940.7

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Number of Reported Adverse Events

Determine the safety of the administration of vemurafenib in combination with TIL therapy including lymphodepleting chemotherapy and interleukin-2 treatment by collecting adverse events according to CTCAE v. 4.0. From start of treatment until 24 weeks after T cell infusion. (NCT02354690)
Timeframe: 0-40 weeks

InterventionTreatment related adverse events (Number)
TotalGrade 1-2Grade 3-4
T Cell Therapy With Vemurafenib Pretreatment1248935

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Objective Response Rate

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1): Complete Response (CR), Disappearance of all target and non-target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR (NCT02354690)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
T Cell Therapy With Vemurafenib Pretreatment9

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Overall Survival

Overall survival (OS), defined as the time from the start of treatment to death, will be described with the Kaplan-Meier curve. (NCT02354690)
Timeframe: Up to 40 months

InterventionMonths (Median)
T Cell Therapy With Vemurafenib Pretreatment28.8

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Progression Free Survival

"Progression-free survival (PFS), defined as the time from start of treatment to disease progression, relapse or death due to any cause, whichever is earlier, will be described with the Kaplan-Meier curve.~Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), 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." (NCT02354690)
Timeframe: Up to 40 months

InterventionMonths (Median)
T Cell Therapy With Vemurafenib Pretreatment4.8

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Objective Response Rate

Clinical responses will be evaluated by RECIST 1.1 (Response Criteria In Solid Tumors Criteria version 1.1) and assessed by CT scan. Complete response (CR), disapperance of all lesions; Partial response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Objective response (OR) = CR + PR (NCT02379195)
Timeframe: Up to 36 months

InterventionParticipants (Count of Participants)
Arm A: TIL + IFNalpha2

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Number of Participants With Adverse Events/Serious Adverse Events

Determine the safety of the administration of peginterferon in combination with TIL therapy including lymphodepleting chemotherapy and Interleukin-2 by reporting adverse events according to CTCAE v. 4.0 (NCT02379195)
Timeframe: 0-24 weeks

InterventionParticipants (Count of Participants)
Adverse eventsTreatment-related adverse eventsSerious adverse events
Arm A: TIL + IFNalpha12124

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Progression Free Survival

Progression free survival (PFS), defined as the time from treatment initiation to disease progression, relapse or death due to any cause, which ever comes first, will be described with the Kaplan Meier method. (NCT02379195)
Timeframe: Up to 36 months

Interventionmonths (Median)
Arm A: TIL + IFNalpha2.8

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Overall Survival

Overall survival (OS), defined as time from treatment initiation to death, described using the Kaplan Meier method (NCT02379195)
Timeframe: Up to 36 months

Interventionmonths (Median)
Arm A: TIL + IFNalpha11.75

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Percentage of Participants With Complete Remission and Complete Remission With Partial Hematological Recovery (CR/CRh) in the Gilteritinib Arm

The CR/CRh rate was defined as the number of participants who achieved either CR or CRh at any of the postbaseline visits divided by the number of participants in the analysis population. (NCT02421939)
Timeframe: From randomization until the data cut-off date 04 Aug 2017, the 142 patients included in the primary analysis of CR/CRh rate were followed up at least 112 days

InterventionPercentage of participants (Number)
CR/CRh rateCR rateCRh rate
Gilteritinib28.219.09.2

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Percentage of Participants Who Achieved Transfusion Conversion and Maintenance

Transfusion conversion & maintenance rate was defined for gilteritinib arm. Participants were classified as transfusion independent if there were no RBC or platelet transfusions within 28 days prior to the first dose to 28 days after the first dose; otherwise they were classified as transfusion dependent at baseline. Participants were considered independent postbaseline if they had 1 consecutive 8 week period without any RBC or platelet transfusion from 29 days after the first dose until the last dose date. For participants who were on treatment ≤ 4 weeks or > 4 weeks but < 12 weeks and there was no RBC or platelet transfusion within postbaseline period, they were considered not evaluable; otherwise, they were considered postbaseline transfusion dependent. Transfusion conversion rate was defined for participants who had evaluable postbaseline transfusion status. Transfusion status (independent vs. dependent) at baseline and postbaseline was reported in a 2 by 2 contingency table. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median treatment duration for gilteritinib was (126.00 [4.0, 885.0]) days versus salvage chemotherapy 28.0 [5.0, 217.0] days)

InterventionPercentage of participants (Number)
Baseline Independent/ Post baseline IndependentBaseline Independent/Post baseline DependentBaseline Independent/Post baseline Not EvaluableBaseline Dependent/Post baseline IndependentBaseline Dependent/Post baseline DependentBaseline Dependent/Post baseline Not Evaluable
Gilteritinib59.224.516.334.555.89.6

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Percentage of Participants Who Underwent Hematopoietic Stem Cell Transplant

Transplantation rate is defined as the percentage of participants undergoing Hematopoietic stem cell transplant (HSCT) during the study period. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionPercentage of participants (Number)
Gilteritinib25.5
Salvage Chemotherapy15.3

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Change From Baseline in Brief Fatigue Inventory (BFI)

The Brief Fatigue Inventory (BFI) is a screening tool designed to assess the severity and impact of fatigue on daily functioning of participants with cancer during the 24 hours. There are 9 items on the scale. The first three questions ask participants to rate their fatigues on a scale from 0 (no fatigue) - 10 (as bad as you can imagine), with higher scores indicating worse outcome. The remaining six questions ask participants to rate how much fatigue has interfered with their daily activities on a scale from 0 (Does not interfere) to 10 (Completely interferes). A global fatigue score can be obtained by averaging all the items on the BFI. The global BFI score will be calculated only if at least 5 of the 9 items are answered. A higher BFI fatigue score indicates worse outcome. (NCT02421939)
Timeframe: Baseline and cycle 1, day 8 and cycle 2 day 1 (up to data cut off date of 17 Sep 2018)

,
InterventionUnits on a scale (Mean)
Cycle 1 day 8 (C1D8)Cycle 2 day 1 (C2D1)
Gilteritinib-0.40.0
Salvage Chemotherapy1.00.4

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Percentage of Participants With Composite Complete Remission (CRc Rate)

CRc rate was defined as the number of participants who achieved the best response of CRc (CR,complete remission with incomplete platelet recovery (CRp) or complete remission with incomplete hematologic recovery (CRi) divided by the number of participants in the analysis population. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionPercentage of participants (Number)
Gilteritinib54.3
Salvage Chemotherapy21.8

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Percentage of Participants With Complete Remission (CR) With Partial Hematological Recovery (CRh)

CRh rate was defined as the number of participants who achieved CRh at any of the postbaseline visits and did not have a best response of CR divided by the number of participants in the analysis population. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionPercentage of participants (Number)
Gilteritinib34.0
Salvage Chemotherapy15.3

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Percentage of Participants With Complete Remission (CR) Rate

The CR rate was defined as the number of participants who achieved the best response of CR divided by the number of participants in the analysis population. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, all participants included in the primary analysis of CR rate were followed up at least 6 months

InterventionPercentage of participants (Number)
Gilteritinib21.1
Salvage Chemotherapy10.5

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Duration of Remission

Duration of remission included duration of composite complete remission (CRc), duration of complete remission (CR)/ complete remission with partial hematologic recovery (CRh), duration of CRh, duration of CR and duration of response (CRc + partial remission (PR). The duration of response was defined as the time from the date of either first CRc or PR until the date of documented relapse (i.e., the date of first NR after CRc or PR) for participants who achieved CRc or PR (relapse date - first CRc or PR disease assessment date + 1). Participants who died without report of relapse were considered nonevents and censored at their last relapse-free disease assessment date (last relapse-free disease assessment date - first CRc or PR disease assessment date + 1). Other participants who did not relapse during the study were considered nonevents and censored at the last relapse-free assessment date. Duration of CR was only applicable to participants with best overall response of CR. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionMonths (Median)
Gilteritinib14.8
Salvage Chemotherapy1.8

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Duration of Overall Survival (OS)

Overall survival was defined as the time from the date of randomization until the date of death from any cause (death date - randomization date + 1). For a participant who was not known to have died by the end of study follow-up, OS was censored at the date of last contact (date of last contact - randomized date + 1). The date of last contact was the latest date that the participant was known to be alive by the cutoff date. The last contact date was derived for participants alive at the analysis cutoff date. Survival rate and 95% CI were estimated using the Kaplan-Meier method and the Greenwood formula. (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionMonths (Median)
Gilteritinib9.3
Salvage Chemotherapy5.6

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Duration of Leukemia-Free Survival (LFS)

The LFS was defined as the time from the date of first CRc until the date of documented relapse (excluding relapse from PR) or death for participants who achieved CRc (relapse date or death date - first CRc disease assessment date + 1). For a participant who was not known to have relapsed or died, LFS was censored on the date of last relapse-free disease assessment date (last relapse-free disease assessment date - first CRc disease assessment date + 1). For a participant who was not known to have relapsed or died, LFS was censored on the date of last relapse-free disease assessment date (last relapse-free disease assessment date - first CRc disease assessment date + 1). (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionMonths (Median)
Gilteritinib4.4
Salvage Chemotherapy6.7

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Duration of Event-Free Survival (EFS)

"EFS was defined as the time from the date of randomization until the date of documented relapse (excluding relapse after PR), treatment failure or death from any cause within 30 days after the last dose of study drug, whichever occurred first (earliest of [relapse date, treatment failure date, death date] - randomization date + 1). If a participant experienced relapse or death within 30 days after the last dose of study drug, the participant was defined as having an EFS event related to either relapse or death, and the event date was the date of relapse or death. For a participant who was not known to have had a relapse or treatment failure or death event, EFS was censored at the date of last relapse-free disease assessment (last relapse-free disease assessment date - randomization date + 1). Data was estimated based on Kaplan-Meier estimates." (NCT02421939)
Timeframe: From randomization until the data cut-off date of 17 Sep 2018, median time of follow-up for OS was 17.8 months

InterventionMonths (Median)
Gilteritinib2.8
Salvage Chemotherapy0.7

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Number of Participants With Adverse Events

"A treatment-emergent adverse event (TEAE) was defined as an AE observed after starting administration of the study drug (gilteritinib or salvage chemotherapy). If the AE occurred on day 1 and the onset check box was marked Onset after first dose of study drug or the onset check box was left blank, then the AE was considered treatment emergent. If the AE occurred on day 1 and the onset check box was marked Onset before first dose of study drug, then the AE was not considered treatment emergent. Majority of salvage chemotherapy participants finished the study by cycle 2 of treatment, the duration of exposure was longer in the gilteritinib arm compared with the salvage chemotherapy arm (126.00 [4.0, 885.0] days versus 28.0 [5.0, 217.0] days). The NCI-CTCAE is defined as National Cancer Institute-Common Terminology Criteria for Adverse Events." (NCT02421939)
Timeframe: From first dose of study drug up to 30 days after the last dose of study drug (median treatment duration for gilteritinib was (126.00 [4.0, 885.0]) days versus salvage chemotherapy 28.0 [5.0, 217.0] days)

,
InterventionParticipants (Count of Participants)
Drug-related TEAESerious TEAEDrug-related serious TEAETEAE leading to deathDrug-related TEAE leading to deathTEAE leading to withdrawal of treatmentDrug-related TEAE lead withdrawal of treatmentNCI-CTCAE Grade 3 or higher TEAEDrug-related Grade 3 or higher TEAEDeath
Gilteritinib2062058871105827236153170
Salvage Chemotherapy713416165135945781

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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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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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Percentage of Participants With Infections Post-randomization by Infection Type

All Grade 2 and 3 infections are reported according to the BMT CTN MOP (Manual of Procedures) where a higher grade corresponds to more severe symptoms. The number of participants with post-randomization infections in each treatment arm is described by severity and type of infection. (NCT02440464)
Timeframe: 2 years post-randomization

,
InterventionParticipants (Count of Participants)
Participants with InfectionsMaximum Severity: Grade 2Maximum Severity: Grade 3Participants with Bacterial InfectionParticipants with Viral InfectionParticipants with Fungal InfectionParticipants with Other Infection
Ixazomib Maintenance8623601
Placebo1110121110

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Percentage of Participants With Disease Progression

Disease progression was evaluated using the International Uniform Response Criteria. Participants in sCR/CR must meet at least one of the criteria for disease progression specified in the protocol for sCR/CR participants; those not in sCR/CR must meet at least one of the disease progression criteria specified in the protocol for those not in sCR/CR. The cumulative incidence of progression from randomization will be estimated for each treatment arm using the Aalen-Johansen estimator, with death in remission treated as a competing risk. Initiation of anti-myeloma therapy will be considered evidence of progression. Participants who were event-free at two years post-transplant are censored at that time. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance34.744.7
Placebo27.336.4

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Percentage of Participants With Overall Survival (OS)

Overall survival (OS) is defined as freedom from death from any cause. OS post-randomization is estimated for each arm using the Kaplan-Meier estimator and compared between arms using the log rank test. Participants who are alive at two years post-transplant are censored at that time. Confidence intervals for values of 100% were not calculated and are shown as 100%. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance100.094.7
Placebo90.986.4

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Percentage of Participants With Progression-Free Survival

The primary endpoint compares progression-free survival as a time to event endpoint from randomization between patients randomized to ixazomib and placebo maintenance in high risk multiple myeloma. Participants are considered a failure of the primary endpoint if they die or suffer from disease progression or if they initiate non-protocol anti-myeloma therapy. Disease progression was evaluated using the International Uniform Response Criteria. Participants must meet one of the criteria for disease progression specified in the protocol. The time to this event is the time from randomization to progression, death, or initiation of non-protocol anti myeloma therapy whichever comes first. The Kaplan-Meier estimator was used to estimate progression-free survival during the 2 year post-transplant follow-up period. Participants who were event-free at two years post-transplant are censored at that time. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance65.355.3
Placebo72.759.1

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Percentage of Participants With Toxicities Post-randomization by Toxicity Type

Toxicities are graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0. Higher grades correspond to worse symptoms with the minimum grade being a 0 and the maximum grade being a 5. Toxicities post-randomization are described for each treatment arm by the type of toxicity as well as peak overall grade. (NCT02440464)
Timeframe: 2 years post-randomization

,
InterventionParticipants (Count of Participants)
Maximum Toxicity Grade : 0 - 2Maximum Toxicity Grade : 3Maximum Toxicity Grade : 4Maximum Toxicity Grade : 5Abnormal Liver SymptomsDysgeusiaEncephalopathyGrade 3-5 Allergic ReactionGrade 3-5 AnaphylaxisGrade 3-5 Blood/Lymphatic ToxicityGrade 3-5 Cardiac ToxicityGrade 3-5 FatigueGrade 3-5 FeverGrade 3-5 GI ToxicityGrade 3-5 Hearing LossGrade 3-5 HemorrhageGrade 3-5 Hepatobiliary/PancreasGrade 3-5 HyperthyroidismGrade 3-5 HypothyroidismGrade 3-5 Metabolic ToxicityGrade 3-5 Musculoskeletal ToxicityGrade 3-5 Nervous System ToxicityGrade 3-5 Ocular ToxicityGrade 3-5 Renal ToxicityGrade 3-5 Respiratory ToxicityGrade 3-5 Skin/Subcutaneous Tissue ToxicityGrade 3-5 Vascular ToxicityHepatitisIntestinal ObstructionLiver FailureSevere Muscle Weakness/ParalysisSudden Vision LossTumor Lysis Syndrome
Ixazomib Maintenance8130031000563060010041400212010001
Placebo6141122010873070070163425853100100

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Percentage of Participants With Best Response to Treatment After Randomization

Response was assessed using the International Uniform Response Criteria. Best response is the best of all the disease response status at each assessment time point after randomization. The order from best to worst is: Stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), Stable Disease (SD), and Progressive Disease (PD). All disease classifications are relative to the patient's disease status prior to allogeneic transplant (i.e. study entry). This outcome was compared between treatment groups using all response data up to 2 years post-transplant. These response data were summarized separately for patients in sCR/CR at the time of randomization vs. those who were not in sCR/CR at the time of randomization. Within each group (in sCR/CR vs not in sCR/CR at randomization), best response to treatment was compared between treatment groups using a Fisher's Exact test instead of a chi-square test because of the small sample size. (NCT02440464)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
Participants in sCR/CR at Randomization72551676Participants in sCR/CR at Randomization72551677Participants Not in sCR/CR at Randomization72551677Participants Not in sCR/CR at Randomization72551676
Partial Response (PR)Stable Disease (SD)Progressive Disease (PD)Stringent Complete Response (sCR)Complete Response (CR)Very Good Partial Response (VGPR)
Ixazomib Maintenance6
Placebo5
Ixazomib Maintenance2
Ixazomib Maintenance0
Placebo0
Placebo1
Ixazomib Maintenance3
Placebo4
Placebo3
Ixazomib Maintenance4
Ixazomib Maintenance1

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Percentage of Participants With Response to Treatment

Response was assessed using the International Uniform Response Criteria. All disease classifications are relative to the patient's disease status prior to allogeneic transplant (i.e. study entry). Response to treatment after randomization (sCR, CR, VGPR, or PR) is summarized in each arm post-transplant at 18 months and 24 months post-transplant. These response data were summarized separately for patients in sCR/CR at the time of randomization vs. those who were not in sCR/CR at the time of randomization. (NCT02440464)
Timeframe: 18 months and 24 months post-transplant

InterventionParticipants (Count of Participants)
Response to Treatment at 18 Months Post-transplant for Participants in sCR/CR at Randomization72551676Response to Treatment at 18 Months Post-transplant for Participants in sCR/CR at Randomization72551677Response to Treatment at 18 Months Post-transplant for Participants Not in sCR/CR at Randomization72551676Response to Treatment at 18 Months Post-transplant for Participants Not in sCR/CR at Randomization72551677Response to Treatment at 24 Months Post-transplant for Participants in sCR/CR at Randomization72551676Response to Treatment at 24 Months Post-transplant for Participants in sCR/CR at Randomization72551677Response to Treatment at 24 Months Post-transplant for Participants Not in sCR/CR at Randomization72551677Response to Treatment at 24 Months Post-transplant for Participants Not in sCR/CR at Randomization72551676
Stringent Complete Response (sCR)Complete Response (CR)Very Good Partial Response (VGPR)Not EvaluablePartial Response (PR)Stable Disease (SD)Progressive Disease (PD)Died Before Evaluation
Ixazomib Maintenance3
Placebo3
Placebo2
Placebo0
Ixazomib Maintenance2
Placebo4
Ixazomib Maintenance4
Placebo1
Placebo6
Ixazomib Maintenance0
Ixazomib Maintenance1
Ixazomib Maintenance5

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Percentage of Participants With Infections Post-randomization by Time Point

All Grade 2 and 3 infections are reported according to the BMT CTN MOP (Manual of Procedures) where a higher grade corresponds to more severe symptoms. The cumulative incidence of severe, life-threatening, or fatal infections (Grade 3), treating death as a competing event, are estimated at 6, 12 and 18 months post randomization using Aalen-Johansen estimators for each treatment group. Comparison of cumulative incidence between the two treatment arms was done using a Z test at fixed time points. (NCT02440464)
Timeframe: 6, 12 and 18 months post-randomization

,
Interventionpercentage of participants (Number)
6 Months Post-randomization12 Months Post-randomization18 Months Post-randomization
Ixazomib Maintenance4.84.84.8
Placebo0.00.00.2

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Percentage of Participants With Acute GVHD (Grades III-IV)

Cumulative incidences of grade III-IV acute GVHD were determined using the Aalen-Johansen estimator. Death prior to acute GVHD is treated as the competing risk. Cumulative incidences are compared between treatment arms using Gray's test. Grading of acute GVHD was derived by consensus grading per BMT Clinical Trials Network (CTN) manual of procedures (MOP). The acute GVHD algorithm calculates the grade based on the organ (skin, GI and liver) stage and etiology/biopsy reported on the weekly GVHD form. Grade I aGVHD is defined as Skin stage of 1-2 and stage 0 for both GI and liver organs. Grade II aGVHD is stage 3 of skin, or stage 1 of GI, or stage 1 of liver. Grade III is stage 2-4 for GI, or stage 2-3 of liver. Grade IV is stage 4 of skin, or stage 4 of liver. Grade 4 is the worst outcome. (NCT02440464)
Timeframe: 100 days post-randomization

Interventionpercentage of participants (Number)
Ixazomib Maintenance9.5
Placebo0.0

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Medical Outcomes Study (MOS) - Short Form 36 (SF-36) Score

The MOS SF-36 instrument is a general assessment of health quality of life with eight components: Physical Functioning, Role Physical, Pain Index, General Health Perceptions, Vitality, Social Functioning, Role Emotional, and Mental Health Index. The scale is 0 to 100 where 0 is maximum disability and 100 is no disability, so the higher the score the more positive the outcome. The Physical Component Summary (PCS) and Mental Component Summary (MCS) were used as the outcome measures in summarizing the SF-36 data for this study. This self-reported questionnaire was completed at transplant, randomization, 6 months following the start of maintenance therapy (which corresponds to 6 months post-randomization), and 24 months post-transplant. Comparisons between treatment groups were performed prior to maintenance, 6 months post-randomization and at 24 months after transplant. Only English and Spanish speaking patients were eligible to participate in the quality of life component of this trial. (NCT02440464)
Timeframe: Randomization, 6 months post-randomization, 24 months post-transplant

,
Interventionscore on a scale (Median)
PCS: RandomizationPCS: 6 Months Post-randomizationPCS: 24 Months Post-transplantPCS: Change from Randomization to 6 Months Post-randomizationPCS: Change from Randomization to 24 Months Post-transplantMCS: RandomizationMCS: 6 Months Post-randomizationMCS: 24 Months Post-transplantMCS: Change from Randomization to 6 Months Post-randomizationMCS: Change from Randomization to 24 Months Post-transplant
Ixazomib Maintenance38.747.945.13.34.751.554.856.1-0.64.9
Placebo41.541.245.71.07.849.954.549.55.3-2.5

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Percentage of Participants With Chronic GVHD

Cumulative incidences of chronic GVHD were determined using the Aalen-Johansen estimator. Death prior to chronic GVHD is treated as the competing risk. Cumulative incidences are compared between treatment arms using Gray's test. Data of chronic GVHD were collected from providers and chart review according to the recommendations of the 2014 NIH Consensus Criteria. Eight organs are scored on a 0-3 scale to reflect degree of chronic GVHD involvement; 3 indicates the worst symptom. Liver and pulmonary function test results and use of systemic therapy for treatment of chronic GVHD are also recorded. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance68.668.6
Placebo63.663.6

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Functional Assessment of Cancer Therapy (FACT) - Bone Marrow Transplant (BMT) Total Score

The FACT-BMT version 4.0 instrument is comprised of the Functional Assessment of Cancer Therapy - General (FACT-G), which evaluates the health-related quality of life (HQL) of patients receiving treatment for cancer, and the BMT Concerns module, that addresses disease and treatment-related questions specific to bone marrow transplant. This scale goes from 0 to 196 where higher scores indicate better functioning. The FACT-BMT Total, which has all items in the FACT-G and BMT modules, was used as the outcome measure. This self-reported questionnaire was completed at transplant, randomization, 6 months following the start of maintenance therapy (which corresponds to 6 months post-randomization), and 24 months post-transplant. Comparisons between treatment groups were performed prior to maintenance, 6 months post-randomization and at 24 months after transplant. Only English and Spanish speaking patients were eligible to participate in the quality of life component of this trial. (NCT02440464)
Timeframe: Randomization, 6-months post-randomization, 24 months post-transplant

,
Interventionscore on a scale (Median)
Randomization6 Months Post-randomization24 Months Post-transplantChange from Randomization to 6 Months Post-randomizationChange from Randomization to 24 Months Post-transplant
Ixazomib Maintenance100.0121.4121.55.012.6
Placebo110.0114.0109.07.03.0

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Percentage of Participants With Toxicities Post-randomization by Time Point

Toxicities are graded using NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0. Higher grades correspond to worse symptoms with the minimum grade being a 0 and the maximum grade being a 5. The cumulative incidence of Grade ≥ 3 toxicity was estimated at 6, 12 and 18 months post randomization using Aalen-Johansen estimators for each treatment group. Death from a cause other than toxicity was treated as a competing risk. Comparison of cumulative incidence between the two treatment arms was done using a Z test at fixed time points. (NCT02440464)
Timeframe: 6, 12 and 18 months post-randomization

,
Interventionpercentage of participants (Number)
6 Months Post-randomization12 Months Post-randomization18 Months Post-randomization
Ixazomib Maintenance57.161.961.9
Placebo63.668.272.7

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Acute Graft-versus-Host Disease (GVHD)

Number of patients who develop acute graft-versus-host disease of any grade. (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine13

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Disease Free Survival at 1 Year Post-transplant

Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 1 year post-transplant. (NCT02497404)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine18

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Overall Survival at 6 Months Post-transplant

Number of participants alive at 6 months post-transplant (NCT02497404)
Timeframe: 6 months post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine35

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Disease Free Survival at 6 Months Post-transplant

Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 6 months post-transplant (NCT02497404)
Timeframe: 6 months post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine25

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Graft Failure

Number of patients who experience graft failure, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to <0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation. (NCT02497404)
Timeframe: 21 days post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine1

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High-Risk Extensive Chronic Graft-versus-Host-Disease

Number of patients who develop high-risk extensive chronic graft-versus-host disease. Extensive chronic GVHD is defined as generalized skin or multiple organ involvement. High risk chronic GVHD is defined as platelet count of less than 100k/microL (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine2

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Overall Survival at 1 Year Post-Transplant

Number of participants alive at 1 year post-transplant (NCT02497404)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine25

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Overall Survival at 2 Years Post-Transplant

Number of participants alive at 2 years post-transplant (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine15

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Disease Free Survival at 2 Years Post-transplant

Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 2 years post-transplant. (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine13

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Progression-Free Survival (PFS)

Treatment response was monitored throughout the study and assessed using standardized criteria. Disease progression was defined as the occurrence of at least one of the following: ≥50% increase in the longest diameter of at least two enlarged lymph nodes, increase in spleen and/or liver size by at least 2 cm from Baseline as determined by measurement below the costal margin, or ≥50% increase in the number of circulating lymphocytes. PFS was defined as the time from study inclusion until first event of disease progression or death and was estimated using Kaplan-Meier analysis. (NCT02533401)
Timeframe: Up to 5 years (from Baseline until disease progression or death, whichever occurred first)

Interventionmonths (Mean)
Rituximab + Fludarabine + Cyclophosphamide47.2

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Percentage of Participants With Death or Disease Progression

Treatment response was monitored throughout the study and assessed using standardized criteria. Disease progression was defined as the occurrence of at least one of the following: greater than or equal to (≥) 50 percent (%) increase in the longest diameter of at least two enlarged lymph nodes, increase in spleen and/or liver size by at least 2 centimeters (cm) from Baseline as determined by measurement below the costal margin, or ≥50% increase in the number of circulating lymphocytes. The percentage of participants with death or documented disease progression at any time during the study was calculated. (NCT02533401)
Timeframe: Up to 5 years (from Baseline until disease progression or death, whichever occurred first)

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide24

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Percentage of Participants Who Died

Participants were followed for survival throughout the study. The percentage of participants who died of any cause during the study was calculated. (NCT02533401)
Timeframe: Up to 5 years (from Baseline until death)

Interventionpercentage of participants (Number)
Rituximab + Fludarabine + Cyclophosphamide14

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Overall Survival (OS)

Participants were followed for survival throughout the study. OS was defined as the time from study inclusion until death from any cause and was estimated using Kaplan-Meier analysis (NCT02533401)
Timeframe: Up to 5 years (from Baseline until death)

Interventionmonths (Mean)
Rituximab + Fludarabine + Cyclophosphamide49.5

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Percentage of Participants With Complete Response (CR), Nodular Partial Response (nPR), or Partial Response (PR)

Treatment response was monitored throughout the study and assessed using standardized criteria. CR was defined as hemoglobin ≥11 grams per deciliter (g/dL), lymphocytes less than (<) 4000 cells per cubic millimeter (cells/mm^3), neutrophils greater than (>) 1500 cells/mm^3, platelets >100,000 cells/mm^3, bone marrow (BM) biopsy with <30% lymphocytes with no lymphocytic infiltrates, no evidence of lymphoid nodules on physical exam, and performance status of 0. PR was defined as >50% decrease in size of enlarged lymph nodes, hepatomegaly, and splenomegaly, with peripheral counts meeting the same criteria as CR or ≥50% improvement from pre-treatment values. Participants with lymphoid nodules on BM biopsy who otherwise met CR criteria were considered nPR. The percentage of participants with each level of best overall response was calculated. (NCT02533401)
Timeframe: Up to 4 years (assessed every 3 months during 6-month treatment period, every 2 months during 6-month safety follow-up, then every 3 months during 3-year safety follow-up)

Interventionpercentage of participants (Number)
CRnPRPR
Rituximab + Fludarabine + Cyclophosphamide71918

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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 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 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 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 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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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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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 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 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 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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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 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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Graft Rejection

Measurement of donor cells vs. recipient cells (NCT02581007)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Reduced-Intensity Mismatched Transplant3

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GVHD Incidence

The number of participants that developed graft-versus-host-disease before or at 100 days after transplant (NCT02581007)
Timeframe: 100 days

Interventionpercentage of patients (Number)
Grades II to IV acute GVHDGrades III to IV acute GVHDModerate chronic GVHDSevere chronic GVHD
Reduced-Intensity Mismatched Transplant2081612

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Overall Survival

Number of participants still alive 2 years after transplant (NCT02581007)
Timeframe: 2 years

Interventionpercentage of patients (Number)
1 yr post-transplant2 yr post-transplant
Reduced-Intensity Mismatched Transplant6856

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Relapse Incidence

Number of patients with disease reoccurrence at 1 and 2 years post-transplant (NCT02581007)
Timeframe: 2 years

Interventionpercentage of patients (Number)
1 yr post-transplant2 yr post-transplant
Reduced-Intensity Mismatched Transplant2436

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Number of Participants With Hematology Results by Maximum Grade Increase Post-Baseline

Blood samples were collected for the analysis of following hematology parameters: hemoglobin, lymphocytes, neutrophils, platelets and leukocytes. Laboratory parameters were graded according to National Cancer Institute-Common Toxicity Criteria for Adverse Events (NCI-CTCAE) version 4.03 where, Grade1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. Baseline is the most recent, non-missing value from a central laboratory within 7 days prior to the lymphodepleting chemotherapy. An increase in grade is defined as an increase in CTCAE grade relative to Baseline grade. Data for any grade increase at worst-case post-Baseline is presented (NCT02588612)
Timeframe: Up to 24 months

InterventionParticipants (Count of Participants)
Hemoglobin increasedHemoglobin (Anemia)Lymphocytes increasedLymphocytes decreasedNeutrophilsPlateletsLeukocytes (Leukocytosis)Leukocytes (Leukopenia)
Lete-cel03155505

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Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or is clinically significant or requires intervention to prevent one of the outcomes listed before. Number of participants with common (greater than or equal to [>=]5 percent[%]) non-serious AEs and SAEs are presented. (NCT02588612)
Timeframe: Up to 24 months

InterventionParticipants (Count of Participants)
Non SAEsSAEs
Lete-cel64

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Disease Control Rate (DCR)

DCR is defined as the percentage of participants with a stable disease (SD) or better as the BOR (Confirmed PR, confirmed CR, or SD >=12 weeks), as assessed by the investigator per RECIST v1.1. CI was calculated using the exact method. (NCT02588612)
Timeframe: Up to 24 months

InterventionPercentage of participants (Number)
Lete-cel20

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Duration of Response

Duration of response (DoR), defined as the interval of time in months from first documented evidence of PR or better to the time when disease progression is documented as assessed by RECIST v1.1 or death due to any cause among participants with a confirmed PR or CR as the BOR. (NCT02588612)
Timeframe: Up to 24 months

InterventionMonths (Number)
Lete-cel6.18

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Overall Response Rate (ORR)

ORR is defined as the percentage of participants with a confirmed Partial response (PR) or Complete response (CR) as the Best overall response (BOR), as assessed by the investigator per Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1. Confidence Interval (CI) was calculated using the exact method. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the Baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 millimeters. (NCT02588612)
Timeframe: Up to 24 Months

InterventionPercentage of participants (Number)
Lete-cel20

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Progression-Free Survival (PFS) by Investigator Assessment

Progression-free survival (PFS) is defined as the interval of time (in months) between the date of T-cell infusion and the earlier of the date of disease progression as assessed by the investigator and the date of death due to any cause. Progressive Disease (PD) is defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. PFS based on responses assessed by investigator per RECIST v1.1 is presented. Median and inter-quartile range (first quartile and third quartile) are presented. (NCT02588612)
Timeframe: Up to 24 months

InterventionMonths (Median)
Lete-cel1.81

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Number of Participants With Worst Case Post-Baseline Abnormal Electrocardiogram (ECG) Findings

12-lead ECGs were recorded in semi-supine position after 5 minutes rest using an ECG machine that automatically calculated the heart rate and measured PR, RR, QRS and QT duration corrected for heart rate by Fridericia's formula (QTcF) intervals. Data for number of participants with abnormal not clinically significant (NCS) and clinically significant (CS) ECG findings for worst case post-Baseline have been presented. Clinically significant abnormal laboratory findings are those which are not associated with the underlying disease, unless judged by the investigator to be more severe than expected for the participant's condition. (NCT02588612)
Timeframe: Up to 24 months

InterventionParticipants (Count of Participants)
Abnormal, NCSAbnormal, CS
Lete-cel21

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Time to Response

Time to response is defined as the interval of time (in months) between the date of T-cell infusion and the first documented evidence of response (PR or CR) in the subset of participants with a confirmed PR or CR as the BOR as assessed by the investigator per RECIST v1.1. (NCT02588612)
Timeframe: Up to 24 months

InterventionMonths (Number)
Lete-cel12.09

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Change From Baseline in Oxygen Saturation

Oxygen saturation measures the capacity of blood to transport oxygen to other parts of the body. Oxygen saturation was measured using a pulse oximeter. Baseline is the most recent, non-missing value within 7 days prior to initiating the lymphodepleting chemotherapy. Change from Baseline is the post-Baseline visit value minus Baseline value. (NCT02588612)
Timeframe: Baseline, Day 1 (pre-dose, 5, 15, and 30 minutes, 1, 1.5, 2, and 4 hours post dose), Days 2, 3, 4, 5, 8 and Week 2

InterventionPercentage of oxygen (Median)
Day 1, pre dose, n=5Day 1, 5 minutes post dose, n=4Day 1, 15 minutes post dose, n=4Day 1, 30 minutes post dose, n=4Day 1, 1 hour post dose, n=5Day 1, 1.5 hours post dose, n=2Day 1, 2 hours post dose, n=3Day 1, 4 hours post dose, n=4Day 2, n=4Day 3, n=5Day 4 , n=5Day 5, n=5Day 8, n=4Week 2, n=4
Lete-cel2.02.52.03.02.01.53.0-1.02.51.01.0-2.00.01.5

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Number of Participants With Any Grade Increase in Clinical Chemistry Parameters

Blood samples were collected for analysis of clinical chemistry parameters: glucose (Gl), albumin, alkaline phosphatase (ALP), alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin (Bil), creatinine (Creat), potassium (Pot), magnesium (Mg), phosphate (Ph), sodium (Sod) and calcium. Laboratory parameters were graded according to NCI-CTCAE version 4.03 where, Grade1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. An increase in grade is defined as an increase in CTCAE grade relative to Baseline grade. Data for any grade increase at worst-case post-Baseline is presented. (NCT02588612)
Timeframe: Up to 24 months

InterventionParticipants (Count of Participants)
Gl, HyperglycemiaGl, HypoglycemiaAlbuminALPALTASTBilCreatPot, HyperkalemiaPot, HypokalemiaMg, HypermagnesemiaMg, HypomagnesemiaPhSod, HypernatremiaSod, HyponatremiaCalcium, HypercalcemiaCalcium, Hypocalcemia
Lete-cel20223411210240113

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Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Self-Care Scale Score

"The European Quality of Life-5 Dimensions Health Questionnaire (EQ-5D) is a participant-answered questionnaire scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. For each dimension the participant is asked for a three-level assessment of their health on the current day: no problems (1), some problems (2), extreme problems (3). EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state). Positive numbers indicate improvement from baseline. The percentage of participants with each level of problem are reported." (NCT02601313)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,
Interventionpercentage of participants (Number)
Baseline: No problems washing or dressing,Baseline: Slight problems washing or dressingBaseline: Moderate problems washing or dressingBaseline: Severe problems washing or dressingBaseline: Unable to wash or dressWeek 4: No problems washing or dressing,Week 4: Slight problems washing or dressingWeek 4: Moderate problems washing or dressingWeek 4: Severe problems washing or dressingWeek 4: Unable to wash or dressMonth 3: No problems washing or dressing,Month 3: Slight problems washing or dressingMonth 3: Moderate problems washing or dressingMonth 3: Severe problems washing or dressingMonth 3: Unable to wash or dressMonth 6: No problems washing or dressing,Month 6: Slight problems washing or dressingMonth 6: Moderate problems washing or dressingMonth 6: Severe problems washing or dressingMonth 6: Unable to wash or dress
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel95320067174848311420933050
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel100000091900010000001000000

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Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Usual Activity Scale Score

"The European Quality of Life-5 Dimensions Health Questionnaire (EQ-5D) is a participant-answered questionnaire scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. For each dimension the participant is asked for a three-level assessment of their health on the current day: no problems (1), some problems (2), extreme problems (3). EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state). Positive numbers indicate improvement from baseline. The percentage of participants with each level of problem are reported." (NCT02601313)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,
Interventionpercentage of participants (Number)
Baseline: No problems doing usual activitiesBaseline: Slight problems doing usual activitiesBaseline: Moderate problems doing usual activitiesBaseline: Severe problems doing usual activitiesBaseline: Unable to do usual activitiesWeek 4: No problems doing usual activitiesWeek 4: Slight problems doing usual activitiesWeek 4: Moderate problems doing usual activitiesWeek 4: Severe problems doing usual activitiesWeek 4: Unable to do usual activitiesMonth 3: No problems doing usual activitiesMonth 3: Slight problems doing usual activitiesMonth 3: Moderate problems doing usual activitiesMonth 3: Severe problems doing usual activitiesMonth 3: Unable to do usual activitiesMonth 6: No problems doing usual activitiesMonth 6: Slight problems doing usual activitiesMonth 6: Moderate problems doing usual activitiesMonth 6: Severe problems doing usual activitiesMonth 6: Unable to do usual activities
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel82145004324226669167447317702
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel7517800365590064360007030000

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Percentage of Participants With Objective Response (OR) Per the Lugano Classification According to Independent Radiology Review Committee (IRRC) in Cohort 2

OR: CMR, CRR, PMR, PRR. CMR: score 1(no uptake above background) / 2(uptake ≤ mediastinum) / 3(uptake > mediastinum but ≤ liver) with/without a residual mass on positron emission tomography 5-point scale; no new lesions. CRR: target nodes/nodal masses regressed to ≤ 1.5 cm in LDi; no extralymphatic sites of disease;absent non-measured lesion NMLs; organ enlargement regress to normal; no new sites; bone marrow normal by morphology. PMR: score 4(uptake moderately > liver) /5 (uptake markedly > liver, new lesions) with reduced uptake compared with baseline and residual mass; no new lesions; responding disease at interim/residual disease at EOT. PRR: ≥ 50% decrease in SPD of up to 6 target measurable nodes and extra-nodal sites; absent/normal, regressed, but no increase of NMLs; spleen regressed by > 50% in length beyond normal. (NCT02601313)
Timeframe: Up to 2 years

Interventionpercentage of participants (Number)
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel93

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Change Over Time in EQ-5D Visual Analogue Scale (VAS) Score

EQ-5D is a standardized participant completed questionnaire that measures health-related quality of life and translates that score into an index value or utility score. EQ-5D-consists of two components: a health state profile and an optional visual analogue scale (VAS). The EQ5D-VAS records the participant's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. EQ-5D-VAS: range 0 to 100. A higher score indicates better self-reported health status. A positive change indicates an improvement. (NCT02601313)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,
Interventionscores on the scale (Mean)
BaselineWeek 4Month 3Month 6
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel82.074.580.184.8
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel82.871.486.489.9

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Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Pain / Discomfort Activity Scale Score

"The European Quality of Life-5 Dimensions Health Questionnaire (EQ-5D) is a participant-answered questionnaire scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. For each dimension the participant is asked for a three-level assessment of their health on the current day: no problems (1), some problems (2), extreme problems (3). EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state). Positive numbers indicate improvement from baseline. The percentage of participants with each level of problem are reported." (NCT02601313)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,
Interventionpercentage of participants (Number)
Baseline: No pain or discomfortBaseline: Slight pain or discomfortBaseline: Moderate pain or discomfortBaseline: Severe pain or discomfortBaseline: Extreme pain or discomfortWeek 4: No pain or discomfortWeek 4: Slight pain or discomfortWeek 4: Moderate pain or discomfortWeek 4: Severe pain or discomfortWeek 4: Extreme pain or discomfortMonth 3: No pain or discomfortMonth 3: Slight pain or discomfortMonth 3: Moderate pain or discomfortMonth 3: Severe pain or discomfortMonth 3: Extreme pain or discomfortMonth 6: No pain or discomfortMonth 6: Slight pain or discomfortMonth 6: Moderate pain or discomfortMonth 6: Severe pain or discomfortMonth 6: Extreme pain or discomfort
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel6622930631919006016184267211020
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel758170064181800642790070102000

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Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Mobility Scale Score

"The European Quality of Life-5 Dimensions Health Questionnaire (EQ-5D) is a participant-answered questionnaire scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. For each dimension the participant is asked for a three-level assessment of their health on the current day: no problems (1), some problems (2), extreme problems (3). EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state). Positive numbers indicate improvement from baseline. The percentage of participants with each level of problem are reported." (NCT02601313)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,
Interventionpercentage of participants (Number)
Baseline: No problems walkingBaseline: Slight problems walkingBaseline: Moderate problems walkingBaseline: Severe problems walkingBaseline: Unable to walkWeek 4: No problems walkingWeek 4: Slight problems walkingWeek 4: Moderate problems walkingWeek 4: Severe problems walkingWeek 4: Unable to walkMonth 3: No problems walkingMonth 3: Slight problems walkingMonth 3: Moderate problems walkingMonth 3: Severe problems walkingMonth 3: Unable to walkMonth 6: No problems walkingMonth 6: Slight problems walkingMonth 6: Moderate problems walkingMonth 6: Severe problems walkingMonth 6: Unable to walk
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel8511300493368469197427513850
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel831700073918009190009010000

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Change Over Time in European Quality of Life-5 Dimensions(EQ-5D) Anxiety / Depression Activity Scale Score

"The European Quality of Life-5 Dimensions Health Questionnaire (EQ-5D) is a participant-answered questionnaire scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. For each dimension the participant is asked for a three-level assessment of their health on the current day: no problems (1), some problems (2), extreme problems (3). EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state). Positive numbers indicate improvement from baseline. The Percentage of participants with each level of problem are reported." (NCT02601313)
Timeframe: Baseline, Week 4, Month 3, and Month 6

,
Interventionpercentage of participants (Number)
Baseline: Not anxious or depressedBaseline: Slight anxious or depressedBaseline: Moderate anxious or depressedBaseline: Severe anxious or depressedBaseline: Extreme anxious or depressedWeek 4: Not anxious or depressedWeek 4: Slight anxious or depressedWeek 4: Moderate anxious or depressedWeek 4: Severe anxious or depressedWeek 4: Extreme anxious or depressedMonth 3: Not anxious or depressedMonth 3: Slight anxious or depressedMonth 3: Moderate anxious or depressedMonth 3: Severe anxious or depressedMonth 3: Extreme anxious or depressedMonth 6: Not anxious or depressedMonth 6: Slight anxious or depressedMonth 6: Moderate anxious or depressedMonth 6: Severe anxious or depressedMonth 6: Extreme anxious or depressed
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel75205006726620692290062261200
Cohort 2: 0.5 x 10^8 Brexucabtagene Autoleucel673300073270009190009010000

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Percentage of Participants With Objective Response (OR) Per the Lugano Classification According to Independent Radiology Review Committee (IRRC) in Cohort 1

OR: complete metabolic response(CMR),complete radiological response(CRR),partial MR response(PMR),partial RR(PRR).CMR:score 1(no uptake above background)/2(uptake ≤ mediastinum)/3(uptake > mediastinum but ≤ liver)with/without a residual mass on positron emission tomography 5-point scale;no new lesions.CRR:target nodes/nodal masses regressed to ≤ 1.5 cm in longest transverse diameter of lesion(LDi);no extralymphatic sites of disease;absent non-measured lesion(NMLs);organ enlargement regress to normal;no new sites;bone marrow normal by morphology. PMR:score 4(uptake moderately > liver)/5(uptake markedly > liver, new lesions)with reduced uptake compared with baseline and residual mass;no new lesions;responding disease at interim/residual disease at end of treatment (EOT).PRR: ≥ 50% decrease in sum of the product of the diameters(SPD)of up to 6 target measurable nodes and extra-nodal sites;absent/normal, regressed, but no increase of NMLs;spleen regressed by > 50% in length beyond normal. (NCT02601313)
Timeframe: Up to 2 years

Interventionpercentage of participants (Number)
Cohort 1: 2 x 10^6 Brexucabtagene Autoleucel93

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Phase 2: Percentage of Participants Experiencing Treatment Emergent Adverse Events (TEAEs)

An AE was any untoward medical occurrence in a participant after brexucabtagene autoleucel infusion, which did not necessarily have a causal relationship with the treatment. An AE could therefore be any unfavorable and/or unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. TEAEs included all AEs with onset on or after initiation of the brexucabtagene autoleucel infusion. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg100

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Phase 2: Percentage of Participants With Anti-KTE-X19 Antibodies

(NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg7

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Phase 2: Change From Baseline Over Time in EQ-5D: Visual Analogue Scale (VAS)

EQ-5D is a self-reported questionnaire used for assessing the overall health status of a participant. The EQ-5D-VAS records the participant's self-rated health on a 20-cm vertical visual analogue scale and is asked to make a global assessment of their current state of health with 0 indicating the worst health they can imagine and 100 indicating the best health they can imagine. (NCT02614066)
Timeframe: Baseline, Day 28, Month 3, Month 6, Month 9, Month 12

Interventionscore on a scale (Mean)
BaselineChange at Day 28Change at Month 3Change at Month 6Change at Month 9Change at Month 12
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg68.24.19.210.62.216.1

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Phase 2: Number of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Value

Grading categories are determined by CTCAE version 4.03. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

InterventionParticipants (Count of Participants)
Hematology: HemoglobinHematology: LeukocytesHematology: PlateletsHematology: LymphocytesHematology: NeutrophilsChemistry: CalciumChemistry: AlbuminChemistry: PhosphateChemistry: MagnesiumChemistry: SodiumChemistry: PotassiumChemistry: GlucoseChemistry: Alanine AminotransferaseChemistry: Alkaline PhosphataseChemistry: Aspartate AminotransferaseChemistry: BilirubinChemistry: CreatinineChemistry: Direct BilirubinChemistry: Urate
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg42544652539527011700000000

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Phase 2: Number of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Value

Grading categories are determined by Common Terminology Criteria for Adverse Events (CTCAE) version 4.03. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

InterventionParticipants (Count of Participants)
Hematology: LymphocytesHematology: LeukocytesHematology: HemoglobinHematology: NeutrophilsHematology: PlateletsChemistry: CreatinineChemistry: GlucoseChemistry: Aspartate AminotransferaseChemistry: Alanine AminotransferaseChemistry: BilirubinChemistry: Alkaline PhosphataseChemistry: Direct BilirubinChemistry: UrateChemistry: SodiumChemistry: PotassiumChemistry: MagnesiumChemistry: CalciumChemistry: AlbuminChemistry: Phosphate
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg14000413141753811123000

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Phase 2: Number of Participants With 5-Level European Quality of Life-5 Dimensions (EQ-5D-5L): Health Utility Index Scale

"EQ-5D-5L is a self-reported questionnaire used for assessing the overall health status of a participant scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was divided into 5 levels of severity: No problem, Slight problems, Moderate problems, Severe problems, and Extreme problems or Unable to." (NCT02614066)
Timeframe: Baseline, Day 28, Month 3, Month 6, Month 9, Month 12

InterventionParticipants (Count of Participants)
Baseline: Mobility (No problem)Baseline: Mobility (Slight problem)Baseline: Mobility (Moderate problem)Baseline: Mobility (Severe problem)Baseline: Mobility (Unable to walk)Day 28: Mobility (No problem)Day 28: Mobility (Slight problem)Day 28: Mobility (Moderate problem)Day 28: Mobility (Severe problem)Day 28: Mobility (Unable to walk)Month 3: Mobility (No problem)Month 3: Mobility (Slight problem)Month 3: Mobility (Moderate problem)Month 3: Mobility (Severe problem)Month 3: Mobility (Unable to walk)Month 6: Mobility (No problem)Month 6: Mobility (Slight problem)Month 6: Mobility (Moderate problem)Month 6: Mobility (Severe problem)Month 6: Mobility (Unable to walk)Month 9: Mobility (No problem)Month 9: Mobility (Slight problem)Month 9: Mobility (Moderate problem)Month 9: Mobility (Severe problem)Month 9: Mobility (Unable to walk)Month 12: Mobility (No problem)Month 12: Mobility (Slight problem)Month 12: Mobility (Moderate problem)Month 12: Mobility (Severe problem)Month 12: Mobility (Unable to walk)Baseline: Self-care (No problem)Baseline: Self-care (Slight problem)Baseline: Self-care (Moderate problem)Baseline: Self-care (Severe problem)Baseline: Self-care (Unable to wash or dress)Day 28: Self-care (No problem)Day 28: Self-care (Slight problem)Day 28: Self-care (Moderate problem)Day 28: Self-care (Severe problem)Day 28: Self-care (Unable to wash or dress)Month 3: Self-care (No problem)Month 3: Self-care (Slight problem)Month 3: Self-care (Moderate problem)Month 3: Self-care (Severe problem)Month 3: Self-care (Unable to wash or dress)Month 6: Self-care (No problem)Month 6: Self-care (Slight problem)Month 6: Self-care (Moderate problem)Month 6: Self-care (Severe problem)Month 6: Self-care (Unable to wash or dress)Month 9: Self-care (No problem)Month 9: Self-care (Slight problem)Month 9: Self-care (Moderate problem)Month 9: Self-care (Severe problem)Month 9: Self-care (Unable to wash or dress)Month 12: Self-care (No problem)Month 12: Self-care (Slight problem)Month 12: Self-care (Moderate problem)Month 12: Self-care (Severe problem)Month 12: Self-care (Unable to wash or dress)Baseline: Usual activities (No problem)Baseline: Usual activities (Slight problem)Baseline: Usual activities (Moderate problem)Baseline: Usual activities (Severe problem)Baseline: Usual activities (Unable to do usual activities)Day 28: Usual activities (No problem)Day 28: Usual activities (Slight problem)Day 28: Usual activities (Moderate problem)Day 28: Usual activities (Severe problem)Day 28: Usual activities (Unable to do usual activities)Month 3: Usual activities (No problem)Month 3: Usual activities (Slight problem)Month 3: Usual activities (Moderate problem)Month 3: Usual activities (Severe problem)Month 3: Usual activities (Unable to do usual activities)Month 6: Usual activities (No problem)Month 6: Usual activities (Slight problem)Month 6: Usual activities (Moderate problem)Month 6: Usual activities (Severe problem)Month 6: Usual activities (Unable to do usual activities)Month 9: Usual activities (No problem)Month 9: Usual activities (Slight problem)Month 9: Usual activities (Moderate problem)Month 9: Usual activities (Severe problem)Month 9: Usual activities (Unable to do usual activities)Month 12: Usual activities (No problem)Month 12: Usual activities (Slight problem)Month 12: Usual activities (Moderate problem)Month 12: Usual activities (Severe problem)Month 12: Usual activities (Unable to do usual activities)Baseline: Pain/Discomfort (No problem)Baseline: Pain/Discomfort (Slight problem)Baseline: Pain/Discomfort (Moderate problem)Baseline: Pain/Discomfort (Severe problem)Baseline: Pain/Discomfort (Extreme Pain or discomfort)Day 28: Pain/Discomfort (No problem)Day 28: Pain/Discomfort (Slight problem)Day 28: Pain/Discomfort (Moderate problem)Day 28: Pain/Discomfort (Severe problem)Day 28: Pain/Discomfort (Extreme Pain or discomfort)Month 3: Pain/Discomfort (No problem)Month 3: Pain/Discomfort (Slight problem)Month 3: Pain/Discomfort (Moderate problem)Month 3: Pain/Discomfort (Severe problem)Month 3: Pain/Discomfort (Extreme Pain or discomfort)Month 6: Pain/Discomfort (No problem)Month 6: Pain/Discomfort (Slight problem)Month 6: Pain/Discomfort (Moderate problem)Month 6: Pain/Discomfort (Severe problem)Month 6: Pain/Discomfort (Extreme Pain or discomfort)Month 9: Pain/Discomfort (No problem)Month 9: Pain/Discomfort (Slight problem)Month 9: Pain/Discomfort (Moderate problem)Month 9: Pain/Discomfort (Severe problem)Month 9: Pain/Discomfort (Extreme Pain or discomfort)Month 12: Pain/Discomfort (No problem)Month 12: Pain/Discomfort (Slight problem)Month 12: Pain/Discomfort (Moderate problem)Month 12: Pain/Discomfort (Severe problem)Month 12: Pain/Discomfort (Extreme Pain or discomfort)Baseline: Anxiety/Depression (No problem)Baseline: Anxiety/Depression (Slight problem)Baseline: Anxiety/Depression (Moderate problem)Baseline: Anxiety/Depression (Severe problem)Baseline: Anxiety/Depression (Extreme Anxious or Depressed)Day 28: Anxiety/Depression (No problem)Day 28: Anxiety/Depression (Slight problem)Day 28: Anxiety/Depression (Moderate problem)Day 28: Anxiety/Depression (Severe problem)Day 28: Anxiety/Depression (Extreme Anxious or Depressed)Month 3: Anxiety/Depression (No problem)Month 3: Anxiety/Depression (Slight problem)Month 3: Anxiety/Depression (Moderate problem)Month 3: Anxiety/Depression (Severe problem)Month 3: Anxiety/Depression (Extreme Anxious or Depressed)Month 6: Anxiety/Depression (No problem)Month 6: Anxiety/Depression (Slight problem)Month 6: Anxiety/Depression (Moderate problem)Month 6: Anxiety/Depression (Severe problem)Month 6: Anxiety/Depression (Extreme Anxious or Depressed)Month 9: Anxiety/Depression (No problem)Month 9: Anxiety/Depression (Slight problem)Month 9: Anxiety/Depression (Moderate problem)Month 9: Anxiety/Depression (Severe problem)Month 9: Anxiety/Depression (Extreme Anxious or Depressed)Month 12: Anxiety/Depression (No problem)Month 12: Anxiety/Depression (Slight problem)Month 12: Anxiety/Depression (Moderate problem)Month 12: Anxiety/Depression (Severe problem)Month 12: Anxiety/Depression (Extreme Anxious or Depressed)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg39741019109131951011662018100111300044511031612223110023011090010140000241493117138311491101742209000111300023161200191490011960011572071200761003012720281130017630018430090100102200

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Phase 2: Relapse-free Survival (RFS)

RFS: time from brexucabtagene autoleucel infusion to date of disease relapse or death from any cause. Participants not meeting criteria for relapse by the analysis data cutoff date were censored at their last evaluable disease assessment date. Participants who had not achieved a CR or CRi at analysis data cutoff were evaluated as an RFS event at Day 0. CR and CRi are defined in Outcome Measures 4 and 5. Relapse is defined in Outcome Measure 6. KM estimates was used for analyses. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to relapse/death or data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionmonths (Median)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg11.6

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Phase 1: Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs)

"DLT is drug-related events with onset within first 28 days following infusion:~Grade (GR) 4 hematologic toxicity lasting more than 30 days (except lymphopenia) if not attributable to underlying disease~All drug-related GR 3 lasting for > 7 days or 4 non-hematologic toxicities regardless of duration (except: aphasia/dysphasia or confusion/cognitive disturbance which resolves to at least GR 1/baseline within 2 weeks or baseline within 4 weeks, fever GR 3/ 4, immediate hypersensitivity reactions within 2 hours of drug infusion that are reversible ≤ GR 2 within 24 hours, renal toxicity which requires dialysis for ≤ 7 days, intubation for airway protection if ≤ 7 days, tumor lysis syndrome, GR 3 liver function test elevation, provided there is resolution to ≤ GR 2 within 14 days, GR 4 transient serum hepatic enzyme abnormalities provided there is resolution to ≤ GR 3 within < 72 hours, hypogammaglobulinemia GR 3/ 4 and GR 3 nausea and/or anorexia)." (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel up to 28 days. Participants were evaluated in specified period but GR4 hematologic toxicity (specified in description) having onset in this period were further observed for 30 days for confirmation

Interventionpercentage of participants (Number)
Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg0

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Phase 2: Complete Remission With Incomplete Hematologic Recovery (CRi) Rate Per Independent Review

CRi: ≤ 5% blasts by morphology in BM; ANC ≥ 1000 and Plt < 100000 or ANC < 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses > 1.5 cm in GTD at baseline must have regressed to ≤ l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and > 1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. Percentage of participants with CRi was reported. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg14.5

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Phase 2: Duration of Remission (DOR) Per Independent Review

DOR was defined as the time from first CR or CRi to relapse or any death in the absence of documented relapse. CR and CRi are defined in Outcome Measures 4 and 5. Relapse: ≤ 5% blasts by morphology in BM; or circulating leukemia present in PB; or CNS EMD of CNS-2 (detectable CSF blast cells in a sample of CSF with < 5 white blood cells [WBCs] per mm^3 with neurological changes) or CNS-3 (detectable CSF blast cells in a sample of CSF with ≥ 5 WBCs per mm^3 with or without neurological changes); or progressive disease (PD): at least one of the following (≥ 50% increase from nadir in the sum of the products of at least two lymph nodes, or if a single node is involved at least a 50% increase in the product of the diameters of this one node; at least a 50% increase in the longest diameter of any single previously identified node more than 1 cm in its short axis; ≥ 50% increase in size of splenic, hepatic or any other non-nodal lesion). Kaplan-Meier (KM) estimates was used for analyses. (NCT02614066)
Timeframe: From first CR or CRi (Phase 2) to relapse/death or data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionmonths (Median)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg12.8

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Phase 2: Minimum Residual Disease (MRD) Negative Remission Rate

MRD was assessed utilizing multicolor flow cytometry to detect residual cancerous cells with a sensitivity of 10^-4. MRD negative remission was defined as MRD < 10^-4 threshold. Percentage of participants with MRD negative remission was reported. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg76

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Phase 2: Percentage of Participants With Allogeneic Stem Cell Transplant (Allo-SCT)

(NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg18

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Phase 2: MRD Negative Rate Among Complete Remission (CR) Participants

Percentage of participants with MRD negative remission among CR participants was reported. MRD was assessed utilizing multicolor flow cytometry to detect residual cancerous cells with a sensitivity of 10^-4. MRD negative remission was defined as MRD < 10^-4 threshold. CR: ≤5% blasts by morphology in BM; ANC ≥ 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses > 1.5 cm in GTD at baseline must have regressed to ≤ l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and > 1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg97

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Phase 2: Complete Remission (CR) Rate Per Independent Review

CR: ≤ 5% blasts by morphology in BM; ANC ≥ 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses > 1.5 cm in GTD at baseline must have regressed to ≤ l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and > 1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. Percentage of participants with CR was reported. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg56.4

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Phase 2: MRD Negative Rate Among Complete Remission With Incomplete Hematologic Recovery (CRi) Participants

Percentage of participants with MRD negative remission among CRi participants was reported. MRD was assessed utilizing multicolor flow cytometry to detect residual cancerous cells with a sensitivity of 10^-4. MRD negative remission was defined as MRD < 10^-4 threshold. CRi: ≤ 5% blasts by morphology in BM; ANC ≥ 1000 and Plt < 100000 or ANC < 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses >1.5 cm in GTD at baseline must have regressed to ≤l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and >1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg100

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Phase 2: OCR Rate (CR + CRi) Per Investigator Review

OCR rate: percentage of participants achieving CR+CRi. CR: ≤ 5% blasts by morphology in BM; ANC ≥1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses >1.5 cm in GTD at baseline must have regressed to ≤l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and >1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. CRi: all CR criteria except in PB ANC ≥1000 and Plt <100000 or ANC <1000 and Plt ≥100000. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg72.7

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Phase 2: Overall Complete Remission (OCR) Rate (Complete Remission [CR]+ Complete Remission With Incomplete Hematologic Recovery [CRi]) as Assessed Per Independent Review

OCR rate: percentage of participants achieving CR+CRi. CR: ≤5% blasts by morphology in bone marrow (BM); absolute neutrophil count (ANC) ≥1000 and platelets (Plt) ≥100000 in peripheral blood (PB); central nervous system extramedullary disease (CNS EMD) of CNS-1 (no detectable leukemia in cerebrospinal fluid [CSF]); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative positron emission tomography (PET) baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses >1.5 cm in greatest transverse diameter [GTD] at baseline must have regressed to ≤l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and >1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. CRi: all CR criteria except in PB ANC ≥1000 and Plt <100000 or ANC <1000 and Plt ≥100000. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionpercentage of participants (Number)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg70.9

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Phase 2: Overall Survival (OS)

OS was defined as the time from brexucabtagene autoleucel infusion to the date of death from any cause. Participants who had not died by the analysis data cutoff date were censored at their last contact date. KM estimates was used for analyses. (NCT02614066)
Timeframe: First infusion date of brexucabtagene autoleucel (Phase 2) to death or data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Interventionmonths (Median)
Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg18.2

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Incidence of Laboratory Abnormalities

Number (count) of participants that experienced a Grade 3 or higher laboratory toxicity (hematology and chemistry). Grades are defined using National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE), v4.03. Grade 1 = mild, no intervention needed; Grade 2 = moderate, minimal intervention needed; Grade 3 = severe or medically significant, hospitalization is required; Grade 4 = life-threatening, urgent intervention needed; Grade 5 = death related to adverse event. (NCT02614560)
Timeframe: Approximately 1 year

,
InterventionParticipants (Count of Participants)
Any chemistry testAmylase (iu/l)Alanine aminotransferase (iu/l)Aspartate aminotransferase (iu/l)Total bilirubin (mg/dl)Uric acid (mg/dl)Lipase (iu/l)Sodium (meq/l)Any hematology testHemoglobin (g/dl)Lymphocytes (x10^3/ul)Neutrophils (x10^3/ul)Platlets (x10^3/ul)White blood cell count (x10^3/ul)
Post-allo (After Stem Cell Transplant)30100011501445
Pre-allo (Before Stem Cell Transplant)43113100636666

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Rate of MRD Negativity

Rate of MRD (minimal residual disease) negativity at Day -1 (1 day prior to transplant) and Day 30 post-transplant (Part A only) (NCT02614560)
Timeframe: 30 days

InterventionPercentage of participants (Number)
Day -1 Rate of MRD NegativityDay 30 Rate of MRD Negativity
Pre-allo (Before Stem Cell Transplant)NA75

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Best Response of CR or CRi

Percentage of patients who achieved a best response of CRi (complete remission with incomplete blood count recovery) or CR (complete remission) (NCT02614560)
Timeframe: 9 weeks

InterventionParticipants (Count of Participants)
Pre-allo (Before Stem Cell Transplant)4

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Duration of Response

Defined as the time from the start of the first documented complete response (CR) or complete remission with incomplete blood count recovery (CRi) to the documentation of relapse or death due to any cause. (NCT02614560)
Timeframe: 9 weeks

Interventionweeks (Median)
Pre-allo (Before Stem Cell Transplant)6.57

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Overall Survival

Defined as the time from the day of alloSCT to the date of death due to any cause. (NCT02614560)
Timeframe: Approximately 96 weeks

Interventionweeks (Median)
Pre-allo (Before Stem Cell Transplant)11.07
Post-allo (After Stem Cell Transplant)NA

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Incidence of Adverse Events

AE: Adverse events; TEAE: Treatment-emergent adverse event. Grades are defined using National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE), v4.03. Grade 1 = mild, no intervention needed; Grade 2 = moderate, minimal intervention needed; Grade 3 = severe or medically significant, hospitalization is required; Grade 4 = life-threatening, urgent intervention needed; Grade 5 = death related to adverse event. An AE is considered serious if it was fatal, life threatening, required hospitalization, was disabling/incapacitating, resulted in a birth defect or congenital anomally, or was otherwise considered to be medically significant. (NCT02614560)
Timeframe: Approximately 1 year

,
InterventionParticipants (Count of Participants)
TEAEsAEs Related to Vadatuximab TalirineAEs Leading to Treatment DiscontinuationGrade 3 or Higher TEAEsSerious AEs
Post-allo (After Stem Cell Transplant)87162
Pre-allo (Before Stem Cell Transplant)66065

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1-year Survival Rate

1-year survival rate estimated using Kaplan-Meier methods The start date for overall survival is the day of alloSCT. (NCT02614560)
Timeframe: 12 months

Interventionpercentage of participants (Number)
Pre-allo (Before Stem Cell Transplant)0
Post-allo (After Stem Cell Transplant)75

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Clinical Response to Crenolanib With Standard Salvage Chemotherapy

To determine the response rate to crenolanib. Complete remission (CR) response criteria include a post-baseline bone marrow (BM) biopsy or aspiration % blasts <5%, absolute neutrophil count (ANC) >1×10^9/L and platelet count >100×10^9/L. CRi response included all CR criteria met, except participant did not have either platelet recovery or ANC recovery. CRh response included all CR criteria met, except subject only has partial platelet recovery and ANC recovery. Complete CR (CRc) response includes all subjects who achieve a CR, CRi and CRh. Partial Response (PR) response included a decrease of ≥50% in % blasts in the BM aspirate or biopsy from baseline but >5%. Morphologic Leukemia-Free State (MLFS) response included ≤5% in % blasts in the BM aspirate or biopsy. (NCT02626338)
Timeframe: 1 year

,,,
InterventionParticipants (Count of Participants)
Composite complete remission (CR+CRh+CRi)MLFSClinical benefit (CRc+PR+MLFS)
All Subjects737
Arm A: HAM Chemotherapy323
Arm B: FLAG-Ida Chemotherapy404
Arm C: MEC Chemotherapy010

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Liposome-encapsulated Cytarabine Time of Maximum Concentration

Median liposome-encapsulated cytarabine time of maximum observed plasma concentration will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

InterventionHOUR (Median)
Treatment (CPX-351 and FLAG)5

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Liposome-encapsulated Cytarabine Volume of Distribution

Geometric mean liposome-encapsulated cytarabine volume of distribution following IV infusion will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

InterventionMILLILITER (Geometric Mean)
Treatment (CPX-351 and FLAG)4158.0

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Liposome-encapsulated Daunorubicin Area Under the Curve

Geometric mean liposome-encapsulated daunorubicin area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

Intervention(NANOGRAM x HOUR) / MILLILITER (Geometric Mean)
Treatment (CPX-351 and FLAG)1288010.3

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Liposome-encapsulated Daunorubicin Clearance

Geometric mean liposome-encapsulated daunorubicin clearance following IV infusion will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

InterventionMILLILITER / HOUR (Geometric Mean)
Treatment (CPX-351 and FLAG)94.7

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Liposome-encapsulated Daunorubicin Volume of Distribution

Geometric mean liposome-encapsulated daunorubicin volume of distribution following IV infusion will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

InterventionMILLILITER (Geometric Mean)
Treatment (CPX-351 and FLAG)3827.7

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Number of Participants With a Dose-limiting Toxicity

Number of patients in the dose-finding phase with a dose-limiting toxicity, graded using the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. (NCT02642965)
Timeframe: 28 days

InterventionParticipants (Count of Participants)
Treatment (CPX-351 and FLAG)1

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Percentage of Responders (Complete Response or Complete Remission With Partial or Incomplete Platelet Recovery) After First Cycle of Therapy

Response (complete response or complete remission with partial or incomplete platelet recovery) after first cycle of therapy, where response is assessed using the revised Acute Myeloid Leukemia International Working Group Criteria. Percentage of responders is calculated by the total of number of patients with complete response or complete remission with partial or incomplete platelet recovery divided by the number of patients evaluable for response after the first cycle. 95% confidence interval is determined using a binomial exact method. (NCT02642965)
Timeframe: Up to 4 weeks

InterventionPercentage of responders (Number)
Treatment (CPX-351 and FLAG)75.68

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Percentage of Responders (Complete Response or Complete Remission With Partial Platelet Recovery) After up to 2 Cycles

Best response (complete response or complete remission with partial platelet recovery) after up to 2 cycles of therapy, where response is assessed using the revised Acute Myeloid Leukemia International Working Group Criteria. Percentage of responders is calculated using the methods of Jung and Kim. 90% confidence interval is determined using the methods of Koyama and Chen. (NCT02642965)
Timeframe: Up to 8 weeks

InterventionPercantage of best responders (Number)
Treatment (CPX-351 and FLAG)68.30

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Liposome-encapsulated Daunorubicin Time of Maximum Concentration

Median liposome-encapsulated daunorubicin time of maximum observed plasma concentration will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

InterventionHOUR (Median)
Treatment (CPX-351 and FLAG)2

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Liposome-encapsulated Cytarabine Area Under the Curve

Geometric mean liposome-encapsulated cytarabine area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

Intervention(NANOGRAM x HOUR) / MILLILITER (Geometric Mean)
Treatment (CPX-351 and FLAG)4418582.5

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Liposome-encapsulated Cytarabine Clearance

Geometric mean liposome-encapsulated cytarabine clearance following IV infusion will be determined for patients in the dose-finding phase. (NCT02642965)
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1

InterventionMILLILITER / HOUR (Geometric Mean)
Treatment (CPX-351 and FLAG)71.76

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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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Overall Survival (OS)

(NCT02652468)
Timeframe: Median follow up of 1689 days

Interventiondays (Median)
Peripheral Blood Stem Cell Transplant352

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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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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 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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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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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02659943)
Timeframe: Date treatment consent signed to date off study, approximately 49 months and 19 days.

InterventionParticipants (Count of Participants)
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only3
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells2
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells1
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only4
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells2
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only9

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Number of Participants With Evidence of Immunogenicity of the Chimeric Antigen Receptor (CAR) T-cell Product

Enzyme-linked spot (ELISPOT) assays were performed to look for anti-CAR T-cell responses. (NCT02659943)
Timeframe: 9 days to 6 weeks after CAR T-cell infusion

InterventionParticipants (Count of Participants)
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only0
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells0
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells0
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only0
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells0
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only0

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Number of Participants With a Dose-Limiting Toxicity (DLT)

Number of participants with DLT's defined as follows: Grade 3 toxicities possibly or probably related to toxicities possibly or probably related to either the anti-CD19 CAR T cells or the fludarabine and cyclophosphamide chemotherapy and lasting more than 7 days. Grade 4 toxicities possibly or probably related to the study interventions. (NCT02659943)
Timeframe: Within 60 days of Chimeric Antigen Receptor (CAR) T cells infusion

InterventionParticipants (Count of Participants)
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only1
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells0
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells0
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only2
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells0
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only1

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Number of Participants Who Had Anti-Lymphoma Activity

Depending on the type of disease, we use PET/CT imaging, tumor biopsies as well as bone marrow biopsies using immunohistochemistry and flow cytometry. (NCT02659943)
Timeframe: 14 days up to 5 years post cell infusion.

InterventionParticipants (Count of Participants)
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only3
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells2
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells1
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only2
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells1
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only6

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MTD

The maximum tolerated dose is the dose at which a maximum of 1 of 6 patients has a dose limiting toxicity (DLT- Grade 3 toxicities possibly or probably related to toxicities possibly or probably related to either the anti-CD19 CAR T cells or the fludarabine and cyclophosphamide chemotherapy and lasting more than 7 days . Grade 4 toxicities possibly or probably related to the study interventions.). (NCT02659943)
Timeframe: Within 60 days of Chimeric Antigen Receptor (CAR) T cells infusion

InterventionT-cells/kg (Number)
All ParticipantsNA

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Median Peak Chimeric Antigen Receptor (CAR) T Cells Level for Participants Treated

A quantitative polymerase chain reaction (PCR) assay or a flow cytometry assay will be used to quantitate Chimeric Antigen Receptor (CAR) + T cells. The absolute number of CAR+ peripheral blood mononuclear cells (PBMC) will be estimated by multiplying the percentage of CAR+ PBMC determined by PCR by the absolute number of lymphocytes plus monocytes per microliter of blood. (NCT02659943)
Timeframe: All post-infusion time-points up to at least 2 months after infusion, and CAR+ T cell analysis will continue until the CAR+ T cell level drops to undetectable levels unless a stable low level of CAR+ T cells is present at more than 3 years after infusion.

InterventionCAR+cell/microliter of blood (Median)
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only42
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells6
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells4
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only36
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells6
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only87

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Maximum Feasible Dose

Maximum feasible dose is the dose determined when the maximum tolerated dose (MTD) cannot be reached. (NCT02659943)
Timeframe: Within 60 days of Chimeric Antigen Receptor (CAR) T cells infusion

InterventionCAR+T cells/kg (Number)
All Participants0.000006

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Number of Participants With a Duration of Best Response in Months

Best Response defined as the first documentation of response was assessed by the Revised Response Criteria for Malignant Lymphoma, and Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification. (NCT02659943)
Timeframe: Response duration is the time from first documentation of response, which is one month after cell infusion in all participants, until progression, initiation of off-study treatment or the last documentation on ongoing response, approx. one month -5 years.

,,,,,
InterventionParticipants (Count of Participants)
Stable Disease - 1 MonthStable Disease - 1 Month (first of two treatments)Stable Disease - 1 Month After First Re-TreatmentStable Disease - 2 Months (first of three treatments)Stable Disease - 2 Months After Second Re-TreatmentPartial Remission - 1 Month After First Re-TreatmentPartial Remission - 2 monthsPartial Remission - 3 MonthsComplete Remission - 4 Months (first of two treatments)Progressive Disease - After first Re-treatmentComplete Remission - 40 Months (first of two treatments)Complete Remission - 5 Months After First Re-TreatmentComplete Remission - 6 MonthsComplete Remission - 6 Months (first of two treatments)Complete Remission - 29+ MonthsComplete Remission - 35+ MonthsComplete Remission - 45+ MonthsProgressive Disease
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only000000010000000020
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells010001001100000000
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells000000000011000000
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only200000000000100100
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells001111000000010000
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only100000200000001301

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Number of Participants Who Had a Second or Third Infusion of Chimeric Antigen Receptor (CAR)+ T Cells

Number of participants who had a second or third Infusion Chimeric Antigen Receptor (CAR)+ T cells. Participants were eligible for a subsequent CAR T-cell infusion if the response at one month after CAR T-cell infusion was partial remission (PR) or stable disease (SD). Participants could also receive a subsequent CAR T-cell infusion if the response was complete remission (CR) but the malignancy later relapsed. CR, PR, and SD was assessed by the Revised Response Criteria for Malignant Lymphoma, and Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification. Complete Remission is complete disappearance of all detectable clinical evidence of disease. Partial Remission is ≥50% decrease in sum of the product of the diameters (SPD) of up to 6 of the largest dominant nodes or nodal masses. Stable Disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease. (NCT02659943)
Timeframe: Participants could receive subsequent cell infusions any time 1 month after CAR T-cell infusion until 5 years after cell infusion.

,,,,,
InterventionParticipants (Count of Participants)
Second infusionThird infusion
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only00
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells20
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells10
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only00
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells11
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only00

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Number of Participants Who Had a Best Response of Complete Remission (CR), Partial Remission (PR), Stable Disease (SD), and Progressive Disease (PD)

Response was assessed by the Revised Response Criteria for Malignant Lymphoma, and Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification. Complete Remission is complete disappearance of all detectable clinical evidence of disease. Partial Remission is ≥50% decrease in sum of the product of the diameters (SPD) of up to 6 of the largest dominant nodes or nodal masses. Progressive Disease is ≥50% increase from nadir in the sum of the products of at least two lymph nodes, or if a single node is involved at least a 50% increase in the product of the diameters of this one node. Stable Disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease. (NCT02659943)
Timeframe: 30 days post Chimeric Antigen Receptor (CAR) T-cells up to 5 years

,,,,,
InterventionParticipants (Count of Participants)
Complete Remission After First Re-TreatmentComplete Remission After Second Re-TreatmentPartial Remission After Second Re-TreatmentStable Disease After First Re-TreatmentStable Disease After Second Re-TreatmentStable Disease After Third Re-TreatmentProgressive Disease After Second Re-Treatment
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only0000000
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells0010001
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells1000000
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only0000000
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells0011110
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only0000000

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Number of Participants That Had Any Grade 2, 3, 4 and 5 Adverse Events

Adverse Events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life-threatening or disabling, and Grade 5 is fatal. (NCT02659943)
Timeframe: Date treatment consent signed to date off study, approximately 49 months and 19 days.

,,,,,
InterventionAdverse events (Number)
< Grade 2Grade 2Grade 3Grade 4Grade 5
LEVEL 1 - Participants Who Received 0.66x10^6 CAR T Cells Only20010
LEVEL 1 Foll/by LEVEL 2-Participants Who Received - 0.66x10^6 CAR T Cells Foll/by 2x10^6 CAR T Cells20000
LEVEL 1 Foll/by LEVEL 3 - Participants Who Received 0.66x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells10000
LEVEL 2 - Participants Who Received 2x10^6 CAR T Cells Only31000
LEVEL 2 Followed by LEVEL 3-Participants Who Received 2x10^6 CAR T Cells Foll/by 6x10^6 CAR T Cells20000
LEVEL 3 - Participants Who Received 6x10^6 CAR T Cells Only62000

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Percentage of Enrolled Participants Who Actually Get Treated

Percentage of participants enrolled who received treatment with Chimeric Antigen Receptor (CAR) T cells, Fludarabine and cyclophosphamide. (NCT02659943)
Timeframe: 4-5 weeks after the first dose

Interventionpercentage of participants (Number)
All Participants76.9

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Highest Treatment Dose Administered on Study

Reporting outcome as the maximum durvalumab dose that we reached on the study. Patients were monitored for 28 days post infusion for dose limiting toxicities. The DLT rate was used to determine dose escalation. The study was terminated prior to reaching the maximum tolerated dose. (NCT02706405)
Timeframe: 28 days

Interventionmg (Number)
All Treated Patients750

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Rate of Complete Response (CR) by Investigator Assessment Using Lugano Criteria

This outcome is a count of participants who experienced a best response of complete response (CR) by investigator assessment using Lugano criteria. 1 patient in Group 1 Early Dose Level 2 could not be analyzed for the outcome because they expired before their first response assessment. (NCT02706405)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 21
Group I (JCAR014, Durvalumab) Late- Dose Level 11
Group I (JCAR014, Durvalumab) Late - Dose Level 22
Group II (Durvalumab, JCAR014) - Dose Level 10
Group II (Durvalumab, JCAR014) - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 32
Group II (Durvalumab, JCAR014) - Dose Level 42
Group II (Durvalumab, JCAR014) - Dose Level 51

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Progression Free Survival

Outcome will be reported as a count of those who did not progress and did not die while on study. (NCT02706405)
Timeframe: From date of first study treatment to progressive disease or death, assessed up to 1 year

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 22
Group I (JCAR014, Durvalumab) Late- Dose Level 11
Group I (JCAR014, Durvalumab) Late - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 10
Group II (Durvalumab, JCAR014) - Dose Level 20
Group II (Durvalumab, JCAR014) - Dose Level 32
Group II (Durvalumab, JCAR014) - Dose Level 42
Group II (Durvalumab, JCAR014) - Dose Level 50

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Overall Survival

Outcome will be reported as a count of participants who survived while on study. Survival was assessed up to 1 year. (NCT02706405)
Timeframe: From date of first study treatment to death, assessed up to 1 year

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 22
Group I (JCAR014, Durvalumab) Late- Dose Level 11
Group I (JCAR014, Durvalumab) Late - Dose Level 23
Group II (Durvalumab, JCAR014) - Dose Level 10
Group II (Durvalumab, JCAR014) - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 33
Group II (Durvalumab, JCAR014) - Dose Level 43
Group II (Durvalumab, JCAR014) - Dose Level 53

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Objective Response Rate by Investigator Assessment Using Lugano Criteria

ORR, defined as a count of participant with a best response of either complete response or partial response. 1 patient in Group 1 Early Dose Level 2 could not be analyzed for the outcome because they expired before their first response assessment. (NCT02706405)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 21
Group I (JCAR014, Durvalumab) Late- Dose Level 11
Group I (JCAR014, Durvalumab) Late - Dose Level 22
Group II (Durvalumab, JCAR014) - Dose Level 10
Group II (Durvalumab, JCAR014) - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 32
Group II (Durvalumab, JCAR014) - Dose Level 42
Group II (Durvalumab, JCAR014) - Dose Level 52

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Maximum JCAR014 Cmax in Blood by Quantitative Polymerase Chain Reaction (qPCR) Analysis

The number of copies of the CAR transgene/μg of DNA in the blood was determined by using quantitative polymerase chain reaction (qPCR) to detect the integrated Flap-EF1⍺ sequence, with a lower limit of detection of 10 transgene copies/μg of DNA. Excluding 1 patient in Group 2 Dose Level 5 who received an out-of-specification JCAR014 product. (NCT02706405)
Timeframe: Up to 12 months

InterventionCAR transgene copies/μg DNA (Mean)
Group I (JCAR014, Durvalumab) - Dose Level 137647.95
Group I (JCAR014, Durvalumab) - Dose Level 210043.68
Group II (Durvalumab, JCAR014) - Dose Level 13690.24
Group II (Durvalumab, JCAR014) - Dose Level 21949.22
Group II (Durvalumab, JCAR014) - Dose Level 38295.36
Group II (Durvalumab, JCAR014) - Dose Level 427841.31
Group II (Durvalumab, JCAR014) - Dose Level 526297.43

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Maximum JCAR014 Cmax by Flow Cytometry

Absolute CD4+ and CD8+ CAR-T cell counts were determined by multiplying the percentages of CD3+CD4+CD8-EGFRt+ and CD3+CD4-CD8+EGFRt+ events, respectively, in a viable CD45+ lymphocyte forward/side scatter gate by an absolute lymphocyte count performed on the same day. Excluding 1 patient in Group 2 Dose Level 5 who received an out-of-specification JCAR014 product. (NCT02706405)
Timeframe: Up to 12 months

InterventionCD3+ CAR-T cells/μL (Mean)
Group I (JCAR014, Durvalumab) - Dose Level 1201.21
Group I (JCAR014, Durvalumab) - Dose Level 213.71
Group II (Durvalumab, JCAR014) - Dose Level 18.27
Group II (Durvalumab, JCAR014) - Dose Level 23.01
Group II (Durvalumab, JCAR014) - Dose Level 313.15
Group II (Durvalumab, JCAR014) - Dose Level 418.13
Group II (Durvalumab, JCAR014) - Dose Level 57.53

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Duration of Response

Duration of response will be an average amount of days from first response assessment until progression or death for treated patients. Total number analyzed will be the patients that progressed or died, excluding those that did not progress or die. 1 patient in Group 1 Early Dose Level 2 could not be analyzed for the outcome because they expired before their first response assessment. (NCT02706405)
Timeframe: From first response to progressive disease or death, assessed up to 1 year

Interventiondays (Mean)
Group I (JCAR014, Durvalumab) Early - Dose Level 272.5
Group I (JCAR014, Durvalumab) Late- Dose Level 147.5
Group I (JCAR014, Durvalumab) Late - Dose Level 242
Group II (Durvalumab, JCAR014) - Dose Level 235
Group II (Durvalumab, JCAR014) - Dose Level 331
Group II (Durvalumab, JCAR014) - Dose Level 497.75
Group II (Durvalumab, JCAR014) - Dose Level 518.14

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Dose Limiting Toxicity (DLT) Rates

Will be summarized based on the dose limiting toxicity evaluable analysis set. The target toxicity rate for the maximum tolerated dose is 30%. Outcome will be reported as a count of patients in each arm that experienced a DLT. (NCT02706405)
Timeframe: 28 days post first infusion of Durvalumab (for participants in Group 1) or 28 days post infusion of JCAR (for participants in Group 2)

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 21
Group I (JCAR014, Durvalumab) Late- Dose Level 10
Group I (JCAR014, Durvalumab) Late - Dose Level 20
Group II (Durvalumab, JCAR014) - Dose Level 10
Group II (Durvalumab, JCAR014) - Dose Level 20
Group II (Durvalumab, JCAR014) - Dose Level 30
Group II (Durvalumab, JCAR014) - Dose Level 41
Group II (Durvalumab, JCAR014) - Dose Level 51

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Count of Participants Who Experienced Adverse Events

Toxicity graded using National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03. (NCT02706405)
Timeframe: 28 days post last infusion of Durvalumab, up to 1 year

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 25
Group I (JCAR014, Durvalumab) Late- Dose Level 13
Group I (JCAR014, Durvalumab) Late - Dose Level 23
Group II (Durvalumab, JCAR014) - Dose Level 11
Group II (Durvalumab, JCAR014) - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 33
Group II (Durvalumab, JCAR014) - Dose Level 46
Group II (Durvalumab, JCAR014) - Dose Level 57

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AUC of JCAR014 Cells by qPCR Analysis

The number of copies of the CAR transgene/μg of DNA in the blood was determined by using quantitative polymerase chain reaction (qPCR) to detect the integrated Flap-EF1⍺ sequence, with a lower limit of detection of 10 transgene copies/μg of DNA. Subjects had samples analyzed at approximately days 0, 3, 7, 10, 14, 21, and 28 after CAR-T cell infusion to generate the AUC from day 0 to 28.The areas under the curve of CAR T-cell counts by flow cytometry and qPCR between time points were calculated by using a trapezoidal rule computational algorithm. Excluding 1 patient in Group 2 Dose Level 5 who received an out-of-specification JCAR014 product. (NCT02706405)
Timeframe: Up to 28 days

InterventionCAR transgene copies/μg DNA x days (Mean)
Group I (JCAR014, Durvalumab) - Dose Level 1296188.95
Group I (JCAR014, Durvalumab) - Dose Level 2104088.38
Group II (Durvalumab, JCAR014) - Dose Level 118710.77
Group II (Durvalumab, JCAR014) - Dose Level 216721.68
Group II (Durvalumab, JCAR014) - Dose Level 363778.63
Group II (Durvalumab, JCAR014) - Dose Level 4353471.10
Group II (Durvalumab, JCAR014) - Dose Level 5389065.24

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Area Under the Curve (AUC) of JCAR014 by Flow Cytometry

Absolute CD4+ and CD8+ CAR-T cell counts were determined by multiplying the percentages of CD3+CD4+CD8-EGFRt+ and CD3+CD4-CD8+EGFRt+ events, respectively, in a viable CD45+ lymphocyte forward/side scatter gate by an absolute lymphocyte count performed on the same day. Subjects had samples analyzed at approximately days 0, 3, 7, 10, 14, 21, and 28 after CAR-T cell infusion to generate the AUC from day 0 to 28. The areas under the curve of CAR T-cell counts by flow cytometry and qPCR between time points were calculated by using a trapezoidal rule computational algorithm. Excluding 1 patient in Group 2 Dose Level 5 who received an out-of-specification JCAR014 product. (NCT02706405)
Timeframe: Up to 28 days

InterventionDays x CD3+ CAR-T cells/μL (Mean)
Group I (JCAR014, Durvalumab) - Dose Level 1844.77
Group I (JCAR014, Durvalumab) - Dose Level 2104.33
Group II (Durvalumab, JCAR014) - Dose Level 147.96
Group II (Durvalumab, JCAR014) - Dose Level 232.44
Group II (Durvalumab, JCAR014) - Dose Level 390.78
Group II (Durvalumab, JCAR014) - Dose Level 4160.94
Group II (Durvalumab, JCAR014) - Dose Level 571.97

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Time to Loss of JCAR014 Detection in Blood by qPCR Analysis

The number of copies of the CAR transgene/μg of DNA in the blood was determined by using quantitative polymerase chain reaction (qPCR) to detect the integrated Flap-EF1⍺ sequence, with a lower limit of detection of 10 transgene copies/μg of DNA. Excluding 1 patient in Group 2 Dose Level 5 who received an out-of-specification JCAR014 product. (NCT02706405)
Timeframe: Up to 12 months, +/- 30 days

Interventiondays (Mean)
Group I (JCAR014, Durvalumab) - Dose Level 1113
Group I (JCAR014, Durvalumab) - Dose Level 2191
Group II (Durvalumab, JCAR014) - Dose Level 128
Group II (Durvalumab, JCAR014) - Dose Level 3199
Group II (Durvalumab, JCAR014) - Dose Level 4183
Group II (Durvalumab, JCAR014) - Dose Level 564

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Rate of Partial Response (PR) by Investigator Assessment Using Lugano Criteria

This outcome is a count of participants who experienced a best response of partial response (PR) by investigator assessment using Lugano criteria. 1 patient in Group 1 Early Dose Level 2 could not be analyzed for the outcome because they expired before their first response assessment. (NCT02706405)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Group I (JCAR014, Durvalumab) Early - Dose Level 21
Group I (JCAR014, Durvalumab) Late- Dose Level 10
Group I (JCAR014, Durvalumab) Late - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 10
Group II (Durvalumab, JCAR014) - Dose Level 21
Group II (Durvalumab, JCAR014) - Dose Level 30
Group II (Durvalumab, JCAR014) - Dose Level 40
Group II (Durvalumab, JCAR014) - Dose Level 51

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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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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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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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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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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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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 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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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 - 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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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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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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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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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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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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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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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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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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Veno-occlusive Disease (VOD) Rate

"VOD is diagnosed by the presence of ≥ 2 of the following with no other identifiable cause for liver disease:~Jaundice (direct bilirubin ≥ 2 mg/dL or > 34 μmol/L)~Hepatomegaly with right upper quadrant pain~Ascites and/or weight gain (> 5% over baseline)" (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient0

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Rate of Disease Recurrence

Disease recurrence is defined as the return of sickle erythropoiesis (HbS level > 70%) and the absence of donor cell representation. (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient1

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Primary Graft Rejection Rate

Primary graft rejection is defined as the absence of donor cells assessed by peripheral blood chimerism assays on day 42. Primary graft rejection can be accompanied by pancytopenia and marrow aplasia or by autologous hematopoietic reconstitution without aplasia. (NCT02757885)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Bone Marrow Recipient1

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Overall Survival Rate

Overall survival is defined as survival with or without sickle cell disease (SCD) after hematopoietic cell transplantation (HCT). (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient6

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Late Graft Rejection Rate

The absence of donor hematopoietic cells in peripheral blood beyond day 42 up to 1 year in a patient who had initial evidence of hematopoietic recovery with > 20% donor cells will be considered a late graft rejection. (NCT02757885)
Timeframe: Day 42 Post transplant up to 1 year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient0

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Infection Rate

Significant infections will be recorded including but not limited to bacterial or fungal sepsis, CMV reactivation with/without clinical disease, adenovirus infection, EBV PTLD, other significant viral reactivations or community-acquired viral infections and invasive mold infections. (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient5

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Frequency of Stroke

An overt stroke is defined as a focal neurologic event and neurologic deficit lasting > 24 hours with neuroimaging changes. (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient0

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Frequency of Idiopathic Pneumonia Syndrome (IPS)

"IPS is diagnosed by evidence of widespread alveolar injury:~Radiographic evidence of bilateral, multi-lobar infiltrates (by chest x-ray or CT scan); AND~Evidence of abnormal respiratory physiology based upon oxygen saturation (SpO2) < 93% on room air or the need for supplemental oxygen to maintain oxygen saturation ≥ 93%; AND~Absence of active lower respiratory tract infection" (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient0

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Rate of Central Nervous System (CNS) Toxicity

CNS toxicity will be defined as seizures, CNS hemorrhage, or PRES. PRES is defined as an increased diffusion coefficient in areas of T2 hyperintensity on diffusion-weighted imaging in the context of clinical symptoms or physical findings including headache, seizures, visual disturbances, and altered level of consciousness. (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient2

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Event-free Survival (EFS) Rate

Event-free Survival (EFS) is defined as the survival with stable donor erythropoiesis with no new clinical evidence of sickle cell disease (SCD). Primary or late graft rejection with disease recurrence or death will count as events for this endpoint. (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient5

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Cumulative Incidence of Neutrophil Engraftment and Platelet Engraftment.

"Cumulative incidence of Neutrophil Engraftment and Platelet Engraftment. Neutrophil engraftment is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of ≥ 500/µL after conditioning.~Platelet engraftment is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL and did not receive a platelet transfusion in the previous 7 days." (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient5

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Chimerism Rate Following Hematopoietic Cell Transplantation for Sickle Cell Disease

Genomic DNA extracted from peripheral blood will be analyzed for variable number of tandem repeats (VNTR) to detect donor engraftment in myeloid and lymphoid fractions. (NCT02757885)
Timeframe: Up to One Year

InterventionParticipants (Count of Participants)
Bone Marrow Recipient5

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Assessment of Tumour Response From Baseline (RECIST)

Assessment of best tumour response from baseline according to Response Evaluation Criteria in Solid Tumours (RECIST) v1.1. (NCT02761915)
Timeframe: Day 28, 2 months and 4 months

,,,,
InterventionParticipants (Count of Participants)
Best Response (RECIST) stable disease (SD)Best Response (RECIST) progressive disease (PD)
Dose Level 121
Dose Level 201
Dose Level 310
Dose Level 411
Dose Level 512

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Safety and Tolerability of 1RG-CART Therapy

Number of serious adverse events, non-serious adverse events and adverse events that are related to fludarabine, cyclophosphamide or 1RG-CART. (NCT02761915)
Timeframe: From the time of informed consent to leukapheresis until withdrawal from the trial or start of other anti-cancer therapy (a median time period of 132 days [range 68 to 301 days])

,,,,
InterventionEvents (Number)
SAEsNSAEsFludarabine Related AEsCyclophosphamide Related AEs1RG-CART Related AEs
Dose Level 1031NANA4
Dose Level 2013NA82
Dose Level 3015770
Dose Level 4575181825
Dose Level 5786252733

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1RG-CART Counts in the Peripheral Blood

Number of patients with 1RG-CART levels in peripheral blood above the limit of quantification for the assay (10 cells/µL) by flow cytometry. (NCT02761915)
Timeframe: From Day 0 until end of trial (median 38.5 days, range 20 to 233 days)

InterventionParticipants (Count of Participants)
Dose Level 10
Dose Level 20
Dose Level 30
Dose Level 40
Dose Level 51

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To Evaluate Anti-tumour Activity (Overall Survival)

Overall survival. (NCT02761915)
Timeframe: Up to 2 years

InterventionDays (Median)
Dose Level 1170
Dose Level 2261
Dose Level 3113
Dose Level 460
Dose Level 5NA

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To Evaluate Anti-tumour Activity (Progression Free Survival)

Progression free survival (progression by RECIST criteria). (NCT02761915)
Timeframe: Up to 2 years

InterventionDays (Median)
Dose Level 1122
Dose Level 234
Dose Level 3113
Dose Level 439.5
Dose Level 527

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Assessment of Tumour Response From Baseline (irRC)

Assessment of best tumour response from baseline according to Immune Related Response Criteria (irRC). (NCT02761915)
Timeframe: Day 28, 2 months and 4 months

,,,,
InterventionParticipants (Count of Participants)
Best Response (irRC) stable disease (irSD)Best Response (irRC) progressive disease (irPD)Best Response (irRC) not evaluable (NE)
Dose Level 1210
Dose Level 2010
Dose Level 3100
Dose Level 4101
Dose Level 5111

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Number of Patients With 2-year Relapse Risk

Hypothesis is that following lymphodepleting chemotherapy and pembrolizumab, the 2-year relapse risk will decrease to ≤35% (NCT02771197)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Lymphodepletion Plus Pembrolizumab10

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Number of Participants With Toxicity Graded According to NCI-CTCAE Version 4.0

Toxicity graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 4.0. (NCT02774291)
Timeframe: Up to 15 years

InterventionParticipants (Count of Participants)
Treatment (mTCR, Aldesleukin)2

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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 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 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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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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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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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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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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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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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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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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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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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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Phase 1: Number of Participants Experiencing Dose-Limiting Toxicities (DLTs)

A DLT was defined as the following KTE-C19-related or ATZ-related events with an onset from immediately after the first ATZ infusion through 21 days after the first ATZ infusion: Grade 4 hematologic toxicity lasting > 30 days (except lymphopenia or B-cell aplasia); All KTE-C19- or ATZ-related Grade 3 non-hematologic toxicities lasting for > 7 days and all KTE-C19- or ATZ-related Grade 4 non-hematologic toxicities regardless of duration. (NCT02926833)
Timeframe: Baseline up to 21 days

InterventionParticipants (Count of Participants)
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)0
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)0
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)1

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Phase 1 and 2: Atezolizumab Levels in Blood

(NCT02926833)
Timeframe: Days 1, 14, 21, 22, 35, 42, 43, 56, 63, 64, 77, 84, 154, and 174

Interventionng/mL (Mean)
Day 1Day 22Day 43Day 64
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)37300073900138000159000

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Phase 1 and 2: Atezolizumab Levels in Blood

(NCT02926833)
Timeframe: Days 1, 14, 21, 22, 35, 42, 43, 56, 63, 64, 77, 84, 154, and 174

Interventionng/mL (Mean)
Day 14Day 35Day 56Day 77
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)43500084200176000240000

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Phase 1 and 2: Atezolizumab Levels in Blood

(NCT02926833)
Timeframe: Days 1, 14, 21, 22, 35, 42, 43, 56, 63, 64, 77, 84, 154, and 174

Interventionng/mL (Mean)
Day 1Day 22Day 43Day 64Day 154
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)32300011000013600019900066800

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Phase 1 and 2: Atezolizumab Levels in Blood

(NCT02926833)
Timeframe: Days 1, 14, 21, 22, 35, 42, 43, 56, 63, 64, 77, 84, 154, and 174

Interventionng/mL (Mean)
Day 21Day 42Day 63Day 84Day 174
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)4030009880017500020900091000

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Phase 1 and 2: Peak Serum Levels of C-X-C Motif Chemokine 10 (CXCL10), Interferon-Gamma (IFN-γ), Interleukin-1 Receptor Antagonist (IL-1RA), Interleukin (IL)-2, IL-6, IL-8, IL-15, and Tumor Necrosis Factor-Alpha (TNF-α) in Blood

(NCT02926833)
Timeframe: Baseline (pre-conditioning chemotherapy); Day 0 (pre-KTE-C19 infusion); Days 1, 4, 7, and 14, and 22 , optional Day 28 (Phase 2) or Day 35 (Phase 1 Cohort 3), optional Day 49, optional Day 69, and Day 94 post-KTE-C19 infusion

Interventionpg/mL (Median)
CXCL 10IFN-γIL-1RAIL-2IL-6IL-8IL-15TNF-α
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZ2000.00587.802801.2017.55121.55180.6548.958.20

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Phase 1 and 2: Percentage of Participants With Hematology Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Values

(NCT02926833)
Timeframe: Enrollment up to 30 days after completion of the final dose of atezolizumab, or 3 months after the KTE-C19 infusion, whichever was longer (up to approximately 2.5 years)

,,,
Interventionpercentage of participants (Number)
Shift to Grade 3 hemoglobinShift to Grade 3 leukocytesShift to Grade 4 leukocytesShift to Grade 4 lymphocytesShift to Grade 3 neutrophilsShift to Grade 4 neutrophilsShift to Grade 3 plateletsShift to Grade 4 platelets
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)100010010001003333
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)6701001003367067
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)83336710017831717
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)5918778614731832

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Phase 1 and 2: Percentage of Participants With Serum Chemistry Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Values

(NCT02926833)
Timeframe: Enrollment up to 30 days after completion of the final dose of atezolizumab, or 3 months after the KTE-C19 infusion, whichever was longer (up to approximately 2.5 years)

,,,
Interventionpercentage of participants (Number)
Shift to Grade 3 albuminShift to Grade 3 calciumShift to Grade 4 calciumShift to Grade 3 phosphateShift to Grade 4 phosphateShift to Grade 3 potassiumShift to Grade 3 sodiumShift to Grade 4 glucose
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)333306700670
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)000670000
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)00033001717
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)9518363614140

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Phase 1 and 2: Percentage of Participants With Serum Chemistry Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Values

ALT = alanine aminotransferase; ALP = alkaline phosphatase; AST = aspartate aminotransferase. (NCT02926833)
Timeframe: Enrollment up to 30 days after completion of the final dose of atezolizumab, or 3 months after the KTE-C19 infusion, whichever was longer (up to approximately 2.5 years)

,,,
Interventionpercentage of participants (Number)
Shift to Grade 3 ALTShift to Grade 4 ALTShift to Grade 3 ALPShift to Grade 3 ASTShift to Grade 4 ASTShift to Grade 3 bilirubinShift to Grade 4 calciumShift to Grade 3 creatinineShift to Grade 4 creatinineShift to Grade 3 direct bilirubinShift to Grade 3 glucoseShift to Grade 4 glucose
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)0000000003300
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)0000000003300
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)0000000001700
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)5514959595141814

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Phase 1 and 2: Complete Response Rate (CRR)

CRR was assessed based on the International Working Group Revised Response Criteria for Malignant Lymphoma (Cheson, 2007), and was defined as the percentage of participants achieving a complete response (CR) as assessed by the study investigators. CR was defined as the complete resolution of all disease-related radiological abnormalities and the disappearance of all signs and symptoms related to the disease. (NCT02926833)
Timeframe: Month 6

Interventionpercentage of participants (Number)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZ46

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Phase 1 and 2: Duration of Response (DOR)

DOR for participants who experienced an objective response was defined as the date of their first confirmed objective response (CR or PR) to disease progression per the revised International Working Group Response Criteria for Malignant Lymphoma (Cheson, 2007) or death regardless of cause. CR was defined as the complete resolution of all disease-related radiological abnormalities and the disappearance of all signs and symptoms related to the disease. PR was defined at least a 50% decrease in SPD of up to six of the largest dominant nodes or nodal masses. Disease progression was defined as any new lesion or increase by ≥ 50% of previously involved sites from nadir. The Kaplan-Meier approach was used to estimate DOR. (NCT02926833)
Timeframe: From the date of first confirmed objective response (CR or PR) to disease progression or death regardless of cause (up to approximately 2.5 years)

Interventionmonths (Median)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZNA

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Phase 1 and 2: Objective Response Rate (ORR)

ORR was assessed based on the International Working Group Revised Response Criteria for Malignant Lymphoma (Cheson, 2007), and was defined as the percentage of participants achieving a CR or partial response (PR) as assessed by the study investigators. CR was defined as the complete resolution of all disease-related radiological abnormalities and the disappearance of all signs and symptoms related to the disease. PR was defined at least a 50% decrease in sum of the product of the diameters (SPD) of up to six of the largest dominant nodes or nodal masses. Participants who did not meet the criteria for objective response by the analysis cutoff date were considered non-responders. (NCT02926833)
Timeframe: From enrollment until first occurrence of CR or PR (up to approximately 2.5 years)

Interventionpercentage of participants (Number)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZ75

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Phase 1 and 2: Overall Survival (OS)

OS was defined as the time from KTE-C19 infusion to the date of death from any cause. The Kaplan-Meier approach was used to estimate OS. (NCT02926833)
Timeframe: From the date of first KTE-C19 infusion to the date of death regardless of cause (up to approximately 2.5 years)

Interventionmonths (Median)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZNA

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Phase 1 and 2: Peak Anti-CD19 CAR T-Cell (KTE-C19) Level (Maximum Observed Plasma Concentration) in Blood

(NCT02926833)
Timeframe: Pre-infusion (Baseline); Post-infusion: Days 7 (Phase 2), 14, 22 (Phase 2), 28 (Phase 2; optional), 35 (Phase 1, Cohort 3; optional), 43, 49 (optional), 64, 69 (optional), 94; long-term follow-up: every 3 months from Month 6 to Month 18, and Month 24

Interventioncells/μL (Mean)
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)86.87
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)167.88
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)60.71
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)60.22

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Phase 1 and 2: Peak Serum Levels of C-Reactive Protein (CRP) in Blood

(NCT02926833)
Timeframe: Baseline (pre-conditioning chemotherapy); Day 0 (pre-KTE-C19 infusion); Days 1, 4, 7, and 14, and 22 , optional Day 28 (Phase 2) or Day 35 (Phase 1 Cohort 3), optional Day 49, optional Day 69, and Day 94 post-KTE-C19 infusion

Interventionmg/L (Median)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZ174.03

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Phase 1 and 2: Peak Serum Levels of Ferritin in Blood

(NCT02926833)
Timeframe: Baseline (pre-conditioning chemotherapy); Day 0 (pre-KTE-C19 infusion); Days 1, 4, 7, and 14, and 22 , optional Day 28 (Phase 2) or Day 35 (Phase 1 Cohort 3), optional Day 49, optional Day 69, and Day 94 post-KTE-C19 infusion

Interventionμg/mL (Median)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZ1.54

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Phase 1 and 2: Peak Serum Levels of Interleukin-2 Receptor Alpha (IL-2Rα) in Blood

(NCT02926833)
Timeframe: Baseline (pre-conditioning chemotherapy); Day 0 (pre-KTE-C19 infusion); Days 1, 4, 7, and 14, and 22 , optional Day 28 (Phase 2) or Day 35 (Phase 1 Cohort 3), optional Day 49, optional Day 69, and Day 94 post-KTE-C19 infusion

Interventionng/mL (Median)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZ17.25

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Phase 1 and 2: Percentage of Participants Experiencing Adverse Events (AEs)

(NCT02926833)
Timeframe: Enrollment up to 30 days after completion of the final dose of atezolizumab, or 3 months after the KTE-C19 infusion, whichever was longer (up to approximately 2.5 years)

Interventionpercentage of participants (Number)
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)100
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)100
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)100
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)100

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Phase 1 and 2: Percentage of Participants With Anti-Atezolizumab Antibodies

(NCT02926833)
Timeframe: Baseline up to approximately 2.5 years

Interventionpercentage of participants (Number)
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)0
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)0
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)0
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)0

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Phase 1 and 2: Percentage of Participants With Anti-KTE-C19 Antibodies

(NCT02926833)
Timeframe: Baseline up to approximately 2.5 years

Interventionpercentage of participants (Number)
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)0
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)0
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)0
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)0

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Phase 1 and 2: Percentage of Participants With Hematology Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Values

(NCT02926833)
Timeframe: Enrollment up to 30 days after completion of the final dose of atezolizumab, or 3 months after the KTE-C19 infusion, whichever was longer (up to approximately 2.5 years)

Interventionpercentage of participants (Number)
Phase 1 Cohort 1: KTE-C19 + ATZ (21 Days After KTE-C19)0
Phase 1 Cohort 2: KTE-C19 + ATZ (14 Days After KTE-C19)0
Phase 1 Cohort 3: KTE-C19 + ATZ (1 Day After KTE-C19)0
Phase 2: KTE-C19 + ATZ (1 Day After KTE-C19)0

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Phase 1 and 2: Progression-Free Survival (PFS)

PFS was defined as the time from the KTE-C19 infusion date to the date of disease progression per the revised International Working Group Response Criteria for Malignant Lymphoma (Cheson, 2007) or death from any cause. Disease progression was defined as any new lesion or increase by ≥ 50% of previously involved sites from nadir. The Kaplan-Meier approach was used to estimate PFS. (NCT02926833)
Timeframe: From the date of first KTE-C19 infusion to disease progression or death regardless of cause (up to approximately 2.5 years )

Interventionmonths (Median)
Phase 1 (Cohort 3) + Phase 2: KTE-C19 + ATZNA

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Overall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Independent Reviewer

Overall response rate (ORR) defined as the percentage of participants with a confirmed complete response (CR) or confirmed partial response (PR) via independent reviewer assessment per RECIST v1.1 relative to the total number of participants in the analysis population. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 millimeters (mm). Confidence intervals (CI) were calculated using the exact (Clopper-Pearson) method. (NCT02992743)
Timeframe: Up to 24 months

InterventionPercentage of participants (Number)
Reduced Lymphodepletion Dose Plus GSK337779420
Standard Lymphodepletion Dose Plus GSK337779440

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Area Under the Time Curve From Zero to Time 28 Days AUC(0-28) of GSK3377794

Area under the cell expansion-time curve from first T-cell infusion to Day 28. Blood samples were collected to measure AUC (0-28 days). (NCT02992743)
Timeframe: Up to 28 days

InterventionDays*copies per microgram genomic DNA (Geometric Mean)
Reduced Lymphodepletion Dose Plus GSK3377794727135.6
Standard Lymphodepletion Dose Plus GSK33777941142798.27

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Change From Baseline in ECG Mean Heart Rate

Single 12-lead ECG was to be obtained at designated time points during the study using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. Baseline is the most recent, non-missing value within 7 days prior to the lymphodepleting chemotherapy. Change from baseline was to be calculated by subtracting post-dose value from baseline value. (NCT02992743)
Timeframe: Baseline, Day 1 (Pre-dose), Day 4 and Day 8

,
InterventionBeats per minute (beats/minute) (Mean)
BASELINEDAY 1DAY 4DAY 8
Reduced Lymphodepletion Dose Plus GSK337779473.01.620.15.4
Standard Lymphodepletion Dose Plus GSK337779478.04.838.99.1

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Progression Free Survival (PFS) Assessed by Investigator

Progression free survival was defined as the interval between the date of T cell infusion and the earliest date of disease progression or death due to any cause. Progressive disease (PD) is defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. PFS based on responses assessed by investigator per RECIST v1.1 is presented. Kaplan-Meier Median and 95% confidence intervals are presented. Confidence intervals were calculated using the Brookmeyer-Crowley method. (NCT02992743)
Timeframe: Up to 24 months

InterventionMonths (Median)
Reduced Lymphodepletion Dose Plus GSK33777945.36
Standard Lymphodepletion Dose Plus GSK33777948.74

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Time to Cmax (Tmax)

Tmax was defined as time to peak cell expansion during the interventional phase. Blood samples were collected to measure Tmax. (NCT02992743)
Timeframe: Day 2 to Day 15

InterventionDays (Median)
Reduced Lymphodepletion Dose Plus GSK33777942
Standard Lymphodepletion Dose Plus GSK33777944

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Time to Response (TTR) Assessed by Independent Reviewer

Time to response was defined as the interval of time between the date of T-cell infusion and the first documented evidence of the confirmed response (PR or CR), in the subset of participants with a confirmed PR or CR as their best confirmed overall response. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 mm. (NCT02992743)
Timeframe: Up to 24 months

InterventionMonths (Median)
Reduced Lymphodepletion Dose Plus GSK33777942.366
Standard Lymphodepletion Dose Plus GSK33777941.889

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Duration of Response (DOR) Assessed by Independent Reviewer

Duration of response was defined as the interval between the initial date of confirmed response (PR/CR) and the date of progressive disease or death among participants with a confirmed response per RECIST 1.1. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 mm. Kaplan-Meier Median and 95% confidence intervals are presented. Confidence intervals were calculated using the Brookmeyer-Crowley method. (NCT02992743)
Timeframe: Up to 24 months

InterventionMonths (Median)
Reduced Lymphodepletion Dose Plus GSK3377794NA
Standard Lymphodepletion Dose Plus GSK33777947.16

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Number of Participants With Insertional Oncogenesis

Peripheral blood mononuclear cells (PBMC) samples were collected for monitoring insertional oncogenesis by PCR for gene modified cells in the blood. DNA from participant identified with >1% PBMC at >=1 year post T-cell infusion was sent for integration site analysis. Integration site analysis was used to assess the possibility of insertional oncogenesis. Participants with insertional oncogenesis were participants with any clones representing >20% of the total. (NCT02992743)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Reduced Lymphodepletion Dose Plus GSK33777940
Standard Lymphodepletion Dose Plus GSK33777940

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Duration of Response (DOR) Assessed by Investigator

Duration of response was defined as the interval between the initial date of confirmed response (PR/CR) and the date of progressive disease or death among participants with a confirmed response per RECIST 1.1. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 millimeters. Kaplan-Meier Median and 95% confidence intervals are presented. Confidence intervals were calculated using the Brookmeyer-Crowley method. (NCT02992743)
Timeframe: Up to 24 months

InterventionMonths (Median)
Reduced Lymphodepletion Dose Plus GSK33777945.31
Standard Lymphodepletion Dose Plus GSK33777947.47

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Maximum Transgene Expansion (Cmax) of GSK3377794

Cmax was defined as peak cell expansion during the interventional phase. Blood samples were collected to measure Cmax. (NCT02992743)
Timeframe: Day 2 to Day 15

InterventionCopies per microgram genomic DNA (Geometric Mean)
Reduced Lymphodepletion Dose Plus GSK337779466991.71
Standard Lymphodepletion Dose Plus GSK3377794108485.91

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Progression Free Survival (PFS) Assessed by Independent Reviewer

Progression free survival was defined as the interval between the date of T cell infusion and the earliest date of disease progression or death due to any cause. Progressive disease (PD) is defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. PFS based on responses assessed by independent reviewer per RECIST v1.1 is presented. Kaplan-Meier Median and 95% confidence intervals are presented. Confidence intervals were calculated using the Brookmeyer-Crowley method. (NCT02992743)
Timeframe: Up to 24 months

InterventionMonths (Median)
Reduced Lymphodepletion Dose Plus GSK33777944.67
Standard Lymphodepletion Dose Plus GSK33777949.03

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Time to Response (TTR) Assessed by Investigator

Time to response was defined as the interval of time between the date of T-cell infusion and the first documented evidence of the confirmed response (PR or CR), in the subset of participants with a confirmed PR or CR as their best confirmed overall response. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 mm. (NCT02992743)
Timeframe: Up to 24 months

InterventionMonths (Median)
Reduced Lymphodepletion Dose Plus GSK33777941.856
Standard Lymphodepletion Dose Plus GSK33777941.938

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Best Overall Response (BOR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Independent Reviewer Assessment

The Best Overall Response (BOR) with confirmation for a participant is defined as the best confirmed response (Confirmed Complete Response [CR] > Confirmed Partial Response [PR] > Stable Disease [SD] > Progressive Disease [PD] > Not Evaluable [NE]) from first T cell infusion until disease progression or initiation of new anti-cancer therapy, whichever is earlier, as assessed by independent reviewer per RECIST v1.1 criteria. (NCT02992743)
Timeframe: Up to 24 months

,
InterventionParticipants (Count of Participants)
Complete Response (CR)Partial Response (PR)Stable Disease (SD)Progressive Disease (PD)Not Evaluable (NE)
Reduced Lymphodepletion Dose Plus GSK337779402800
Standard Lymphodepletion Dose Plus GSK337779404303

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Best Overall Response (BOR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Investigator Assessment

The Best Overall Response (BOR) with confirmation for a participant is defined as the best confirmed response (Confirmed Complete Response [CR] > Confirmed Partial Response [PR] > Stable Disease [SD] > Progressive Disease [PD] > Not Evaluable [NE]) from first T cell infusion until disease progression or initiation of new anti-cancer therapy, whichever is earlier, as assessed by the investigator per RECIST v1.1 Criteria. (NCT02992743)
Timeframe: Up to 24 months

,
InterventionParticipants (Count of Participants)
Complete Response (CR)Partial Response (PR)Stable Disease (SD)Progressive Disease (PD)Not Evaluable (NE)
Reduced Lymphodepletion Dose Plus GSK337779402800
Standard Lymphodepletion Dose Plus GSK337779404510

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Change From Baseline in Electrocardiogram (ECG) Parameters- PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval

Triplicate 12-Lead ECGs were collected at baseline visit and single ECGs at other timepoints. At each time point during the study ECG was taken using an ECG machine that automatically calculates the heart rate and measured the PR interval, QRS duration, QTcB, QTcF intervals, RR interval and uncorrected QT interval. Baseline is the most recent, non-missing value within 7 days prior to the lymphodepleting chemotherapy. Change from baseline was to be calculated by subtracting post-dose value from baseline value. (NCT02992743)
Timeframe: Baseline, Day 1, Day 4 and Day 8

,
InterventionMilliseconds (msec) (Mean)
PR Interval, BASELINEPR Interval, DAY 1PR Interval, DAY 4PR Interval, DAY 8QRS Duration, BASELINEQRS Duration, DAY 1QRS Duration, DAY 4QRS Duration, DAY 8QT Interval, BASELINEQT Interval, DAY 1QT Interval, DAY 4QT Interval, DAY 8QTcB Interval, BASELINEQTcB Interval, DAY 1QTcB Interval, DAY 4QTcB Interval, DAY 8QTcF Interval, BASELINEQTcF Interval, DAY 1QTcF Interval, DAY 4QTcF Interval, DAY 8RR Interval, BASELINERR Interval, DAY 1RR Interval, DAY 4RR Interval, DAY 8
Reduced Lymphodepletion Dose Plus GSK3377794152.9-2.3-10.7-8.191.22.82.3-2.7401.1-6.4-49.4-16.742728101429.9-10.1-25.3-7.5842.6-15.8-162.5-71.6
Standard Lymphodepletion Dose Plus GSK3377794142-2.2-4186.10.90.8-1389.3-8.1-72.5-4.4423.336.3847425.31.33.316.2807.65.3-188-138.5

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Number of Participants With Adverse Event of Special Interest (AESI)

An AESI may be of scientific and medical concern related to the treatment, monitored, and rapidly communicated by investigator to sponsor. The AESI included events of Cytokine release syndrome (CRS), Haematopoietic cytopenias (including pancytopenia and aplastic anaemia), Graft versus host disease (GvHD), Immune Effector-Cell Associated Neurotoxicity Syndrome (ICANS), and Guillain-Barre syndrome. (NCT02992743)
Timeframe: Up to 24 months

,
InterventionParticipants (Count of Participants)
Participants with any AESICytokine release syndromeHaematopoietic cytopenias (including pancytopenia and aplastic anaemia)
Reduced Lymphodepletion Dose Plus GSK337779410610
Standard Lymphodepletion Dose Plus GSK3377794101010

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Number of Participants With Any Grade Increase in Clinical Chemistry Results at Worst-case Post-baseline

Blood samples were collected for the analysis clinical chemistry parameters: glucose, albumin, alkaline phosphatase, alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, creatinine, potassium, magnesium, sodium, phosphate, calcium. Laboratory parameters were graded according to National Cancer Institute-Common Toxicity Criteria for Adverse Events (NCI-CTCAE) version 4.0, where Grade1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. Baseline is the most recent, non-missing value from a central laboratory within 7 days prior to the lymphodepleting chemotherapy. An increase in grade is defined as an increase in CTCAE grade relative to baseline grade. Data of number of participants with any grade increase at worst-case post-baseline (maximum grade increase post-baseline) is presented. (NCT02992743)
Timeframe: Up to 24 months

,
InterventionParticipants (Count of Participants)
Glucose HyperglycemiaGlucose HypoglycemiaAlbuminAlkaline PhosphataseALTASTBilirubinCreatininePotassium HyperkalemiaPotassium HypokalemiaMagnesium HypermagnesemiaMagnesium HypomagnesemiaPhosphateSodium HypernatremiaSodium HyponatremiaCalcium Corrected for Albumin HypercalcemiaCalcium Corrected for Albumin Hypocalcemia
Reduced Lymphodepletion Dose Plus GSK337779451947834155292821
Standard Lymphodepletion Dose Plus GSK3377794849678221926101917

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Number of Participants With Any Grade Increase in Hematology Results at Worst-case Post-baseline

Blood samples were collected for the analysis of following hematology parameters: hemoglobin, lymphocytes, neutrophils, platelets, leukocytes. Laboratory parameters were graded according to National Cancer Institute-Common Toxicity Criteria for Adverse Events (NCI-CTCAE) version 4.0, where Grade1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. Baseline is the most recent, non-missing value from a central laboratory within 7 days prior to the lymphodepleting chemotherapy. An increase in grade is defined as an increase in CTCAE grade relative to baseline grade. Data of number of participants with any grade increase at worst-case post-baseline (maximum grade increase post-baseline) is presented. (NCT02992743)
Timeframe: Up to 24 months

,
InterventionParticipants (Count of Participants)
Hemoglobin AnemiaLymphocyte count decreasedNeutrophilsPlateletsLeukocytes
Reduced Lymphodepletion Dose Plus GSK337779491010910
Standard Lymphodepletion Dose Plus GSK33777941010101010

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Number of Participants With Serious Adverse Events (SAEs) and Non-SAEs

An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. SAE is defined as any untoward medical occurrence that, at any dose can result in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth or is medically significant or requires intervention to prevent one or the outcomes listed above. (NCT02992743)
Timeframe: Up to 24 months

,
InterventionParticipants (Count of Participants)
SAEsNon-SAEs
Reduced Lymphodepletion Dose Plus GSK3377794513
Standard Lymphodepletion Dose Plus GSK3377794710

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Number of Participants With Positive Anti-drug Antibodies (ADAs)

Serum samples were collected to analyze for the presence of ADAs using validated immunoassays. (NCT02992743)
Timeframe: Up to 24 Months

InterventionParticipants (Count of Participants)
Reduced Lymphodepletion Dose Plus GSK33777940
Standard Lymphodepletion Dose Plus GSK33777940

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Number of Participants With Replication Competent Lentivirus (RCL)

RCL was monitored using a polymerase chain reaction (PCR)-based assay that detects and measures copies of the gene coding for the vector's envelope protein, namely vesicular stomatitis virus G protein (VSV-G). (NCT02992743)
Timeframe: Day 1 (pre-infusion), and at Week 12, Week 24, and 1 year post-infusion

InterventionParticipants (Count of Participants)
Reduced Lymphodepletion Dose Plus GSK33777940
Standard Lymphodepletion Dose Plus GSK33777940

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Overall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Investigator Assessment

Overall response rate (ORR) defined as the percentage of participants with a confirmed complete response (CR) or confirmed partial response (PR) via investigator assessment per RECIST (Response Evaluation Criteria In Solid Tumors Criteria) v1.1 relative to the total number of participants in the analysis population. Partial response is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 millimeters (mm). Confidence intervals (CI) were calculated using the exact (Clopper-Pearson) method. (NCT02992743)
Timeframe: Up to 24 months

InterventionPercentage of participants (Number)
Reduced Lymphodepletion Dose Plus GSK337779420
Standard Lymphodepletion Dose Plus GSK337779440

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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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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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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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Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells

Peak percentage and the percentage 1 month after infusion of peripheral blood mononuclear cells (PBMC) that expressed the Chimeric Antigen Receptor (CAR) T-cells assessed by polymerase chain reaction (PCR). (NCT03049449)
Timeframe: From time of infusion until 1 month after infusion

Interventionpeak percentage (Number)
Participant #5 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #6 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #7 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #5 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #6 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #7 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion
Dose Escalation Cohort 1 Dose Level 2 - 1.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg5.6920263940.0661686650.8755795530.0365825560.0106072290.002843526

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Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells

Peak percentage and the percentage 1 month after infusion of peripheral blood mononuclear cells (PBMC) that expressed the Chimeric Antigen Receptor (CAR) T-cells assessed by polymerase chain reaction (PCR). (NCT03049449)
Timeframe: From time of infusion until 1 month after infusion

Interventionpeak percentage (Number)
Participant #9 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant 10 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #17 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #9 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion#10 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #17 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion
Dose Escalation Cohort 2 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg10.4257844361.1738701259.7018439540.1459374910.0063824660.09701347

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Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells

Peak percentage and the percentage 1 month after infusion of peripheral blood mononuclear cells (PBMC) that expressed the Chimeric Antigen Receptor (CAR) T-cells assessed by polymerase chain reaction (PCR). (NCT03049449)
Timeframe: From time of infusion until 1 month after infusion

Interventionpeak percentage (Number)
Participant #1 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #2 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #4 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #1 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #2 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #4 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion
Dose Escalation Cohort 1 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg2.5059949981.0035133970.257878064NANANA

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Number of Participants With a Dose Limiting Toxicity (DLT)

A DLT is defined as toxicities occurring within 30 days of CAR T-cell infusion. DLT's are Grade 3 or Grade 4 toxicities possibly, probably, or definitely related to either the anti-CD30 CAR T cells or the fludarabine and cyclophosphamide chemotherapy conditioning regimen and lasting more than 7 days. A DLT will only be considered in participants who have received CAR T-cell infusions. (NCT03049449)
Timeframe: 30 days within CAR T-cell infusion

,,,,,
InterventionParticipants (Count of Participants)
Grade 3 possibly relatedGrade 3 probably relatedGrade 3 definitely relatedGrade 4 possibly relatedGrade 4 probably relatedGrade 4 definitely related
Dose Escalation Cohort 1 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg000000
Dose Escalation Cohort 1 Dose Level 2 - 1.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg000000
Dose Escalation Cohort 1 Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg000000
Dose Escalation Cohort 1 Dose Level 4 - 9.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg000110
Dose Escalation Cohort 2 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg000000
Dose Escalation Cohort 2 Dose Level 2 - 3.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg000000

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Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT03049449)
Timeframe: Date treatment consent signed to date off study, approximately 11 months and 26 days, 7 months and 10 days, 39 months and 7 days, 17 months and 3 days, 6 months and 15 days, and 1 month and 7 days for each group respectively.

InterventionParticipants (Count of Participants)
Dose Escalation Cohort 1 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg3
Dose Escalation Cohort 1 Dose Level 2 - 1.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg3
Dose Escalation Cohort 1 Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg7
Dose Escalation Cohort 1 Dose Level 4 - 9.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg5
Dose Escalation Cohort 2 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg3
Dose Escalation Cohort 2 Dose Level 2 - 3.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg1

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Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells

Peak percentage and the percentage 1 month after infusion of peripheral blood mononuclear cells (PBMC) that expressed the Chimeric Antigen Receptor (CAR) T-cells assessed by polymerase chain reaction (PCR). (NCT03049449)
Timeframe: From time of infusion until 1 month after infusion

Interventionpeak percentage (Number)
Participant #19 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipants #19 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion
Dose Escalation Cohort 2 Dose Level 2 - 1.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kgNANA

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Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells

Peak percentage and the percentage 1 month after infusion of peripheral blood mononuclear cells (PBMC) that expressed the Chimeric Antigen Receptor (CAR) T-cells assessed by polymerase chain reaction (PCR). (NCT03049449)
Timeframe: From time of infusion until 1 month after infusion

Interventionpeak percentage (Number)
Participant #11 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #12 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #13 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant 14 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #15 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #18 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #26 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #11 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #12 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #13 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #14 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #15 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #18 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #26 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion
Dose Escalation Cohort 1 Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg25.065559512.0617235820.9518137933.77019985221.64168096.19335317725.56426658NA2.061723582NA2.3618840691.5843437016.193353177NA

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Peak Percentage and Persistence at 1 Month of Peripheral Blood Mononuclear Cells (PBMC) That Expressed the Chimeric Antigen Receptor (CAR) T-cells

Peak percentage and the percentage 1 month after infusion of peripheral blood mononuclear cells (PBMC) that expressed the Chimeric Antigen Receptor (CAR) T-cells assessed by polymerase chain reaction (PCR). (NCT03049449)
Timeframe: From time of infusion until 1 month after infusion

Interventionpeak percentage (Number)
Participant #20 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #21 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #22 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #24 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #25 - Peak percentage of CAR+ cells among peripheral blood mononuclear cellsParticipant #20 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #21 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #22 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #24 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusionParticipant #25 - %CAR+ cells among peripheral blood mononuclear cells 1 month after infusion
Dose Escalation Cohort 1 Dose Level 4 - 9.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg3.48418573551.3035579829.4075210322.4579037755.047606571.1104807278.5130335111.658734750.28927614518.33580648

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Number of Participants With Complete Response (CR), Partial Response (PR), Stable Disease (SD), or Progression as Their Best Response

Response was measured using the Cheson et al. Revised Response Criteria for Malignant Lymphoma, Journal of Clinical Oncology 2007 and Recommendations for Initial Evaluation, Staging, and Response Assessment of Non-Hodgkin Lymphoma: The Lugano Classification Journal of Clinical Oncology, 2014). Complete Response (CR) is complete disappearance of all detectable clinical evidence of disease and disease-related symptoms if present before therapy. Partial Response (PR) is ≥ 50% decrease in sum of the product of the diameters (SPD) of up to 6 of the largest dominant nodes or nodal masses. Progressive Disease (PD) ≥ 50% increase from nadir in the sum of the products of at least two lymph nodes, or appearance of a new lesion greater than 1.5 cm in any axis even if other lesions are decreasing in size. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. (NCT03049449)
Timeframe: Responses will be assessed as long as the patient is on-study at the following time-points: 1, 2, 3, 4, 6, 9, and 12 months after CAR T-cell infusion. Then responses were assessed every 6 months up until 3 years after CAR T-cell infusion.

,,,,,
InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseStable DiseaseProgressive Disease
Dose Escalation Cohort 1 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg0210
Dose Escalation Cohort 1 Dose Level 2 - 1.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg1020
Dose Escalation Cohort 1 Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg0340
Dose Escalation Cohort 1 Dose Level 4 - 9.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg0221
Dose Escalation Cohort 2 Dose Level 1 - 0.3x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kg0120
Dose Escalation Cohort 2 Dose Level 2 - 1.0x10^6 Chimeric Antigen Receptor (CAR) T Cells Per kgNANANANA

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Maximum Tolerated Dose of Anti-Tumor Necrosis Factor (TNF) Receptor Superfamily Member 8 (CD30) Chimeric Antigen Receptor (CAR) in Participants With Advanced CD30-expressing Lymphomas

Maximum tolerated dose is defined as the dose at which a maximum of 1 of 6 participants has a dose-limiting toxicity (DLT). A DLT is defined as toxicities occurring within 30 days of CAR T-cell infusion. DLT's are Grade 3 or Grade 4 toxicities possibly, probably, or definitely related to either the anti-CD30 CAR T cells or the fludarabine and cyclophosphamide chemotherapy conditioning regimen and lasting more than 7 days. A DLT will only be considered in participants who have received CAR T-cell infusions. (NCT03049449)
Timeframe: 4-5 weeks after first dose

Interventionmillion CAR T-cells/kg (Number)
All Participants3

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The Overall Response (OR) Rate (Complete Response + Incomplete Count Recovery + Partial Response)

Complete response or complete response with incomplete count recovery or partial response, of selinexor in combination with fludarabine and cytarabine for patients with relapsed or refractory AML in the phase II portion of the study (NCT03071276)
Timeframe: Between days 28 and 35 of cycle 1 (cycle length is 42-56 days)

Interventionpercentage of participants (Number)
Interventions: Selinexor, Fludarabine, and Cytarabine53.6

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Complete Response

Complete response, of selinexor in combination with fludarabine and cytarabine for patients with relapsed or refractory AML in the phase II portion of the study (NCT03071276)
Timeframe: Between days 28 and 35 of cycle 1 (cycle length is 42-56 days)

Interventionpercentage of participants (Number)
Interventions: Selinexor, Fludarabine, and Cytarabine46.4

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Complete Response or Complete Response With Incomplete Count Recovery

Complete response or complete response with incomplete count recovery, of selinexor in combination with fludarabine and cytarabine for patients with relapsed or refractory AML in the phase II portion of the study (NCT03071276)
Timeframe: Between days 28 and 35 of cycle 1 (cycle length is 42-56 days)

Interventionpercentage of participants (Number)
Interventions: Selinexor, Fludarabine, and Cytarabine50.0

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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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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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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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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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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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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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Maximum Persistence (Cmax) of GSK3377794

Blood samples were collected to measure persistence of infused GSK3377794 using polymerase chain reaction of a vector specific sequence in deoxyribonucleic acid (DNA) extracted from peripheral blood mononuclear cell (PBMC). (NCT03168438)
Timeframe: Up to 108 weeks

InterventionCopies per microgram genomic DNA (Geometric Mean)
GSK337779438509.67
GSK3377794+Pembrolizumab89640.56

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Duration of Response

Duration of response is defined as the interval between the initial date of the confirmed response (sCR, CR, VGPR, or PR) and the initial assesment date of confirmed progressive disease or death among participants with a confirmed response per IMWG (2016). (NCT03168438)
Timeframe: Up to 108 weeks

InterventionMonths (Median)
GSK33777942.1
GSK3377794+Pembrolizumab2.1

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Area Under the Plasma Concentration-time Curve From Zero to Day 28 (AUC[0-28])

Blood samples were collected to measure persistence of infused GSK3377794 using polymerase chain reaction of a vector specific sequence in DNA extracted from PBMC. (NCT03168438)
Timeframe: Up to Day 28

InterventionDays*Copies per microgram genomic DNA (Geometric Mean)
GSK3377794463989.35
GSK3377794+Pembrolizumab1498869.21

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Number of Participants With Treatment Limiting Toxicities-GSK3377794+Pembrolizumab Arm Only

The following toxicities were considered to be treatment limiting toxicities: any >=Grade 4 AE; Grade 3 non-infectious pneumonitis and any other Grade 3 AE (excluding pneumonitis), that did not improve to Grade 2 within 7 days after onset despite medical management and supportive care. Exceptions included the following: Grade 3 or 4 leukopenia, lymphopenia, neutropenia, or febrile neutropenia; Grade 3 or 4 thrombocytopenia not associated with significant bleeding; Grade 3 anemia; Grade 4 cytokine release syndrome (CRS) or toxicities related to CRS that resolved to Grade <=2 within 7 days; other Grade 3 laboratory abnormality determined to be not clinically significant by the Investigator; Grade 3 or 4 fever and chills; Grade 3 or 4 hypoalbuminemia or abnormal electrolytes that responded to supplementation/correction; AE related to the cancer or its progression. (NCT03168438)
Timeframe: Up to 3 weeks

InterventionParticipants (Count of Participants)
GSK3377794+Pembrolizumab0

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Number of Participants With Worst-case Results for Coagulation Parameters Relative to Normal Range Post-Baseline Relative to Baseline

Blood samples were collected for the assessment of following coagulation parameters: prothrombin time and partial thromboplastin time (PTT). A laboratory value that is outside the normal range is considered either high abnormal (value above the upper limit of the normal range) or low abnormal (value below the lower limit of the normal range). Participants were counted twice if the participant had values in 'Decreased to low' and 'Increased to high' during the post-Baseline period. Data for worst case post Baseline is presented. (NCT03168438)
Timeframe: Up to 108 weeks

InterventionParticipants (Count of Participants)
Prothrombin Time; decrease to lowProthrombin Time; To normal or no changeProthrombin Time; increase to highPTT; decrease to lowPTT; To normal or no changePTT; increase to high
GSK3377794001010

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Number of Participants With Worst-case Hematology Results by Any Grade Increase Post-Baseline Relative to Baseline

Blood samples were collected for the assessment of following hematology parameters: hemoglobin, lymphocytes, neutrophils, platelets and leukocytes. Laboratory parameters were graded according to NCI-CTCAE version 4.03 where, Grade1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. An increase in grade is defined as an increase in CTCAE grade relative to Baseline grade. Data for any grade increase at worst case post Baseline is presented. (NCT03168438)
Timeframe: Up to 108 weeks

,
InterventionParticipants (Count of Participants)
Hemoglobin (Anemia)Hemoglobin increasedLymphocyte count decreasedLymphocyte count increasedNeutrophil count decreasedPlatelet count decreasedLeukocyte count decreased
GSK33777942030333
GSK3377794+Pembrolizumab3030333

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Number of Participants With Worst-case Chemistry Results by Any Grade Increase Post-Baseline Relative to Baseline

Blood samples were collected for the assessment of following clinical chemistry parameters: glucose, albumin, alkaline phosphatase (ALP), alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, creatinine, potassium, magnesium, phosphate, sodium and calcium. Laboratory parameters were graded according to National Cancer Institute-Common Toxicity Criteria for Adverse Events (NCI-CTCAE) version 4.03 where, Grade1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. An increase in grade is defined as an increase in CTCAE grade relative to Baseline grade. Data for any grade increase at worst case post Baseline is presented. (NCT03168438)
Timeframe: Up to 108 weeks

,
InterventionParticipants (Count of Participants)
Glucose (Hyperglycemia)Glucose (Hypoglycemia)Albumin (Hypoalbuminemia)ALP increasedALT increasedAST increasedBilirubin increasedCreatinine increasedPotassium (Hyperkalemia)Potassium (Hypokalemia)Magnesium (Hypermagnesemia)Magnesium (Hypomagnesemia)Phosphate (Hypophosphatemia)Sodium (Hypernatremia)Sodium (Hyponatremia)Calcium (Hypercalcemia)Calcium (Hypocalcemia)
GSK337779420310001010220212
GSK3377794+Pembrolizumab20211113020120103

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Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE is any untoward medical occurrence in a participant or clinical investigation participant who received a study treatment and the event need not necessarily have a causal relationship with study treatment. An SAE is any AE that results in death; is life-threatening; requires hospitalization or prolongation of existing hospitalization; results in a persistent or significant disability; is a congenital anomaly/birth defect; is clinically significant or requires intervention to prevent one of the outcomes listed before. (NCT03168438)
Timeframe: Up to 108 weeks

,
InterventionParticipants (Count of Participants)
Any AEAny SAE
GSK337779432
GSK3377794+Pembrolizumab32

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Time to Response

Time to response is defined as the time interval (in months) from T-cell infusion to initial date of documented confirmed response (PR or better) in the subset of participants who showed a confirmed BOR of PR or better by Investigator assessment per IMWG (2016). (NCT03168438)
Timeframe: Up to 108 weeks

InterventionMonths (Median)
GSK33777940.7
GSK3377794+Pembrolizumab0.7

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Time to Maximum Persistence

Blood samples were collected to measure persistence of infused GSK3377794 using polymerase chain reaction of a vector specific sequence in DNA extracted from PBMC. (NCT03168438)
Timeframe: Up to 108 weeks

InterventionDays (Median)
GSK33777948.0
GSK3377794+Pembrolizumab8.0

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Progression-free Survival

Progression Free Survival is defined as the interval between the date of T-cell infusion and the initial assessment date of confirmed progressive disease as assessed by the investigator per IMWG (2016) or date of death. Progressive disease is defined as an increase of 25% from lowest confirmed response value in 1 or more of the following criteria: Serum M-protein with absolute increase of >=0.5 grams per deciliter (g/dL); Serum M-protein increase >=1 g/dL if the lowest M-component was >=5 g/dL; Urinary M-protein (absolute increase must be >=200 mg per 24 hours). (NCT03168438)
Timeframe: Up to 108 weeks

InterventionMonths (Median)
GSK33777942.79
GSK3377794+Pembrolizumab2.78

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Overall Response Rate

Overall response rate is defined as the percentage of participants with a best overall response (BOR) of confirmed stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR) per International Myeloma Working Group (IMWG) Response Criteria (2016); where, PR: >=50% reduction of serum M-Protein and reduction in 24-hour urinary M-protein by >=90% or to <200 milligrams (mg) per 24 hours; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein plus urine M-protein level <100 mg per 24 hours; CR: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and <= 5% plasma cells in bone marrow; or sCR: CR as defined by normal free light chain (FLC) ratio and absence of clonal cells by immunohistochemistry. Confidence intervals were calculated using the exact (Clopper-Pearson) method. (NCT03168438)
Timeframe: Up to 108 weeks

InterventionPercentage of participants (Number)
GSK337779433.3
GSK3377794+Pembrolizumab66.7

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Number of Participants With Worst-case Post Baseline Abnormal Electrocardiogram (ECG) Findings

ECG was recorded using an ECG machine that automatically calculated the heart rate and measured PR, RR, QRS and QT duration corrected for heart rate by Fridericia's formula (QTcF) intervals. Data for number of participants with abnormal clinically significant ECG findings for worst case post-Baseline has been presented. Clinically significant abnormal laboratory findings are those which are not associated with the underlying disease, unless judged by the investigator to be more severe than expected for the participant's condition. (NCT03168438)
Timeframe: Up to 108 weeks

InterventionParticipants (Count of Participants)
GSK33777940
GSK3377794+Pembrolizumab1

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Number of Participants With Objective Response

Participants displaying objective response associated with the treatment regimen per Response Evaluation Criteria in Solid Tumors (RECIST v.1.1). The best overall response is the best response recorded from the start of the treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started). The patient's best response assignment will depend on the achievement of both measurement and confirmation criteria. (NCT03215810)
Timeframe: Up to 40 months

InterventionParticipants (Count of Participants)
ConfirmedUnconfirmed
TIL+ Nivolumab33

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Rate of Dose Limiting Toxicity (DLT)

Investigators plan to demonstrate that treatment with nivolumab in patients undergoing TIL therapy is safe with a continuous Pocock-type stopping boundary for serious toxicity of < 17%, with safety reported based upon Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 criteria. DLT defined as: any grade ≥3 immune-related adverse event definitely attributable to nivolumab. DLT related to adoptive cell therapy will be defined as a non-hematologic grade 4 or higher adverse event that is immediately life-threatening occurring upon or after the start of therapy that is immediately life-threatening and not related to non-small cell lung cancer or other pre-existing condition. Safety: Toxicity will be assessed within 4 weeks of the adoptive TIL transfer. The accrual will be halted if excessive numbers of participants with toxicity are seen. For example, if there are 5 or more out of 10 participants (full follow-up) with toxicity, the trial will be stopped. (NCT03215810)
Timeframe: Up to 40 months

Intervention% participants w severe toxicity (Number)
TIL+ Nivolumab17

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Progression Free Survival

Progression free survival (PFS): Time from TIL infusion to disease progression, relapse or death due to any cause, which ever comes first, will be used as an event. (NCT03287674)
Timeframe: Up to 12 months after TIL infusion

InterventionDays (Median)
TIL Treated Patients93

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Overall Survival

Overall Survival (OS), defined as time from TIL infusion to death (NCT03287674)
Timeframe: Up to 3 years after TIL infusion

InterventionDays (Median)
TIL Treated Patients247

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Number of Participants With Reported Adverse Events by Type

Determine the safety of TIL therapy in combination with checkpoint inhibitors for patients with ovarian-, fallopian tube or primary peritoneal cancer by reporting adverse events according to CTCAE v. 4.0. (NCT03287674)
Timeframe: Up to 12 months

InterventionParticipants (Count of Participants)
Performance status dropFatigueNauseaVomitingDiarrheaHyponatremiaInfectionNeutropeniaTrombocytopeniaAnemiaAgammaglobulinemiaDyspneaFeverColitisDry skin
TIL Treated Patients331113266611311

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Number of Participants With Minimal Residual Disease (MRD) Response

After bone marrow transplant, bone marrow was collected every 3-6 months (as clinically indicated per treating investigator) for up to one year after bone marrow transplant. Bone marrow was evaluated by a clinical pathologist for any evidence of multiple myeloma (MM) or myelofibrosis (MF). Evidence of MM or MF in the bone marrow is referred to as minimal residual disease (MRD). A participant with evidence of MRD is MRD-positive. A participant with no evidence of MRD is MRD-negative, which is considered an MRD response. This outcome reports the number participants with MRD responses any time between bone marrow transplant up to one year of follow-up. (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
All Particpants - Treatment5

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Disease Free Survival at One Year

Disease free survival is defined as an absence of disease relapse or progression up to one year following stem cell transplant. (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
All Particpants - Treatment3

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Incidence of Acute Graft Versus Host Disease (GVHD)

Cases of acute graft versus host disease (GVHD) will be diagnosed by the treating physician and will be reported as a count of participants with acute GVHD. (NCT03303950)
Timeframe: Up to day 365

InterventionParticipants (Count of Participants)
All Particpants - Treatment2

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Incidence of Chronic GVHD

Chronic GVHD will be assessed based on criteria established by the National Institutes of Health Consensus Development Project in 2005, and recently updated in 2014. This will be reported as a count of participants diagnosed with chronic GVHD (NCT03303950)
Timeframe: Up to day 365

InterventionParticipants (Count of Participants)
All Particpants - Treatment1

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Non-relapse Mortality (NRM) at Day 100

NRM is defined as death due to GVHD, infections, sepsis, organ (lung, liver, kidney) toxicity. Death from underlying disease (i.e. progression or relapse) is not considered NRM. (NCT03303950)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
All Participants - Treatment1

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Non-relapse Mortality (NRM) at Day 365

NRM is defined as death due to GVHD, infections, sepsis, organ (lung, liver, kidney) toxicity. Death from underlying disease (i.e. progression or relapse) is not considered NRM. (NCT03303950)
Timeframe: Up to day 365

InterventionParticipants (Count of Participants)
All Participants - Treatment2

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Overall Survival at One Year

Overall survival is defined as the number of participants remaining alive up to one year following stem cell transplant. (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
All Particpants - Treatment3

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Number of Participants With Different Clinical Responses

Clinical Responses were determined by disease-specific criteria taking into account multiple clinical and molecular markers. Multiple Myeloma (MM) response was determined using International Myeloma Working Group (IMWG) consensus criteria for response. Participants with MM had either Stringent Complete Response (sCR) or Very Good Partial Response (VGPR). Myelofibrosis (MF) response was determined using International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus criteria. Participants with MF had either Complete Response (CR) or Stable Disease (SD) (also referred to in the protocol as no response). (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Stringent Complete Response (sCR)Very Good Partial Response (VGPR)Complete Response (CR)Stable Disease (SD)
All Particpants - Treatment1121

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Overall Survival (OS)

Overall survival is defined as the time from the first dose date of study drug to the date of death from any cause. Analysis was done using KM estimate. Participants who have not died by the analysis data cutoff date were censored at their last date known to be alive or cutoff date, whichever is earlier. (NCT03318861)
Timeframe: From KITE-585 infusion to date of data cutoff (maximum: 17.6 months)

Interventionmonths (Median)
Dose Escalation: 3 x 10^7 KITE-585NA
Dose Escalation: 1 x 10^8 KITE-5855.1
Dose Escalation: 3 x 10^8 KITE-5856.9
Dose Escalation: 1 x 10^9 KITE-585NA
Dose Expansion (Renal Impairment): 3 x 10^7 KITE-58512.2

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Percentage of Participants Experiencing Clinically Significant Laboratory Abnormalities

Clinically significant laboratory abnormalities were defined as per investigator's discretion. (NCT03318861)
Timeframe: Enrollment through 24 months after treatment with KITE-585 or up to disease progression or initiation of another anti-cancer therapy, whichever occurs first (maximum: 2.9 months)

Interventionpercentage of participants (Number)
Dose Escalation: 3 x 10^7 KITE-5850
Dose Escalation: 1 x 10^8 KITE-5850
Dose Escalation: 3 x 10^8 KITE-5850
Dose Escalation: 1 x 10^9 KITE-5850
Dose Expansion (Renal Impairment): 3 x 10^7 KITE-5850

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Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs)

"A DLT is a KITE-585-related event with onset in the first 28 days following infusion. DLTs are defined by events and duration of events, including:~Any duration: Grade (GR) 4 cytokine release syndrome (CRS), KITE-585-related GR 5 adverse events (AE) and GR 4 nonhematologic AE with the exceptions of fever, nausea, hepatic toxicity that resolves to GR 3 or better in ≤ 72 hours, hypogammaglobulinemia, tumor lysis syndrome, acute renal toxicity requiring dialysis for ≤ 7 days, intubation for airway protection for ≤ 7 days and AE resolves to ≤ GR 1 within 2 weeks and baseline within 4 weeks~≥ 72 hours: GR 3 CRS and GR 3 nonhematologic AE with the exceptions of fever, nausea, hepatic toxicity that resolves to GR 2 or better in ≤ 14 days, hypogammaglobulinemia and tumor lysis syndrome~≥ 30 days: GR 4 hematologic AE with the exceptions of cytopenias attributable to ongoing or recurrent multiple myeloma" (NCT03318861)
Timeframe: From KITE-585 infusion until 28 days after KITE-585 infusion

Interventionpercentage of participants (Number)
Dose Escalation: 3 x 10^7 KITE-5850
Dose Escalation: 1 x 10^8 KITE-5850
Dose Escalation: 3 x 10^8 KITE-5850
Dose Escalation: 1 x 10^9 KITE-5850

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Percentage of Participants Experiencing Treatment-Emergent Adverse Events

(NCT03318861)
Timeframe: Enrollment through 24 months after treatment with KITE-585 or up to disease progression or initiation of another anti-cancer therapy, whichever occurs first (maximum: 2.9 months)

Interventionpercentage of participants (Number)
Dose Escalation: 3 x 10^7 KITE-585100.0
Dose Escalation: 1 x 10^8 KITE-585100.0
Dose Escalation: 3 x 10^8 KITE-585100.0
Dose Escalation: 1 x 10^9 KITE-585100.0
Dose Expansion (Renal Impairment): 3 x 10^7 KITE-585100.0

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Progression Free Survival (PFS) as Determined by Study Investigators According to the IMWG Consensus Panel 1

PFS: Interval from first study drug dose date to the earlier of first documentation of definitive progressive disease (PD) per IMWG Consensus Panel 1 Criteria or death from any cause. PD: an increase of 25% from the lowest response value in 1 of the following: Serum and urine M-protein (absolute increase ≥ 0.5 g/dL and ≥ 200 mg/24 hours, respectively); In participants without measurable serum and urine M-protein levels, the difference between involved and uninvolved FLC levels (absolute increase > 10 mg/dL); In participants without measurable serum and urine M-protein and without measurable disease by FLC levels, bone marrow PC percentage (absolute percentage ≥ 10%). Definite development of new bone lesions or STP or definite increase in the size of existing bone lesions or STPs; Development of hypercalcemia (corrected serum calcium >11.5 mg/dL) that can be attributed solely to PC proliferative disorder. Analysis was done using Kaplan-Meier (KM) estimate. (NCT03318861)
Timeframe: From KITE-585 infusion to the earlier date of data cutoff and first administration of other anti-cancer therapies including stem cell transplant (maximum: 17.6 months)

Interventionmonths (Median)
Dose Escalation: 3 x 10^7 KITE-5851.1
Dose Escalation: 1 x 10^8 KITE-5851.0
Dose Escalation: 3 x 10^8 KITE-5850.8
Dose Escalation: 1 x 10^9 KITE-5851.0
Dose Expansion (Renal Impairment): 3 x 10^7 KITE-585NA

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Duration of Response (DOR) as Determined by Study Investigators According to the IMWG Consensus Panel 1

DOR is defined for participants who experience an objective response and is defined as the time from the date of their first objective response (which is subsequently confirmed) to PD per IMWG Consensus Panel 1 Criteria or death from any cause, whichever is earlier. Objective response is defined in Outcome measure 2. (NCT03318861)
Timeframe: From first response to the earlier date of data cutoff and first administration of other anti-cancer therapies including stem cell transplant (maximum: 17.6 months)

Interventionmonths (Number)
Dose Escalation: 3 x 10^7 KITE-585NA

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Objective Response Rate (ORR) as Determined by Study Investigators According to the International Myeloma Working Group (IMWG) Consensus Panel 1 Criteria

ORR: Percentage of participants who achieved a stringent CR (sCR), complete response (CR), partial response (PR), or very good PR (VGPR), as determined by IMWG Consensus Panel 1 Criteria. sCR: CR+normal free light chain (FLC) ratio, no clonal cells in BM by immunohistochemistry or immunofluorescence; CR: negative immunofixation (IFX) on serum and urine, no soft tissue plasmacytomas (STP), <5% plasma cells in bone marrow (BM); PR: ≥50% decrease of serum M-protein + 24hr urinary M-protein decrease by ≥90% or <200 mg/24hr. If unmeasurable serum and urine M-protein; and serum-free light assay; requires ≥ 50% decrease in the difference between involved and uninvolved FLC levels / ≥ 50% reduction in plasma cells (PC), provided baseline BM PC percentage was ≥ 30%, respectively. If present at baseline, ≥ 50% reduction in the size of STP is also required; VGPR: serum and urine M-protein detected by IFX but not electrophoresis, >90% in serum M-protein+urine, M-protein level <100 mg/24hr. (NCT03318861)
Timeframe: From KITE-585 infusion to the earlier date of data cutoff and first administration of other anti-cancer therapies including stem cell transplant (maximum: 17.6 months)

Interventionpercentage of participants (Number)
Dose Escalation: 3 x 10^7 KITE-58533.3
Dose Escalation: 1 x 10^8 KITE-5850
Dose Escalation: 3 x 10^8 KITE-5850
Dose Escalation: 1 x 10^9 KITE-5850
Dose Expansion (Renal Impairment): 3 x 10^7 KITE-5850

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Count of Patients That Experienced Adverse Events

Toxicity graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 No patients received more than one CAR T cell infusion, so AE assessment period was only 28 days after first and only CAR T infusion for all patients. (NCT03338972)
Timeframe: Up to 28 days after CAR T-cell infusion

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 17
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 28
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 37
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 43

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Dose-limiting Toxicities (DLT) Rate

Observed DLT rates will be summarized based on the DLT-Evaluable analysis set. Outcome will be reported as count of participants in each arm who experienced a DLT. No patients received more than one CAR T cell infusion, so DLT assessment period was only 28 days after first and only CAR T infusion for all patients. (NCT03338972)
Timeframe: Up to 28 days after CAR T cell infusion

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 10
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 20
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 31
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 40

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Duration of Persistence of Adoptively Transferred BCMA CAR-T Cells

Persistence of CART cells is tested by qPCR in PBMC. (NCT03338972)
Timeframe: Assessed from Baseline up to a maximum of 537 days

InterventionDays (Median)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 1134
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 2110
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 3386
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 4236

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Number of Participants With Detectable BCMA CART Cell Migration to Primary Disease Site (Bone Marrow) at Day 28

(NCT03338972)
Timeframe: Baseline up to Day 28

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 15
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 27
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 35
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 42

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Objective Response Rate (ORR)

Number of patients with a best response of either complete response, stringent complete response, very good partial response or partial response, assessed using modified International Myeloma Working group response criteria. (NCT03338972)
Timeframe: Baseline up to 3 months after CART infusion

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 17
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 28
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 37
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 43

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Overall Survival (OS)

Outcome is reported as a count of participants who were alive at the 1 year post-infusion timepoint. (NCT03338972)
Timeframe: Assessed up to 1 year after CART infusion

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 13
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 25
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 37
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 43

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Progression-free Survival (PFS)

Outcome is reported as the count of participants who were alive at the 1 year post treatment mark and did not experience disease progression by the 1 year post treatment mark. (NCT03338972)
Timeframe: Assessed up to 1 year after CART infusion

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 13
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 25
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 35
Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 42

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Complete Remission (CR)

Complete disappearance of all clinical evidence of disease (NCT03504410)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
CPI-613 + HD Cytarabine and Mitoxantrone20
Control (HAM) and Control Sub-groups (MEC and FLAG)22

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Survival of Treatment

Survival of Hematopoietic Stem Cell Transplant during treatment and post treatment up to 1 year. (NCT03593902)
Timeframe: During Treatment and Post Treatment up to 1 year

InterventionParticipants (Count of Participants)
Hematopoietic Stem Cell Transplantation9

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Change in Skin Score by mRSS

Defined by at least a 25% improvement (decline) in skin score by modified Rodnan skin score (mRSS) if skin score is greater than 14 on enrollment. If skin score is less than 14 on enrollment, improvement is defined by at least a 5% improvement on mRSS. The modified Rodnan skin score (MRSS) is a measure for skin disease in scleroderma and is calculated by summation of skin thickness in 17 different body sites. The scale ranges from at total score of normal skin thickness (0) to severe thickness (51). (NCT03593902)
Timeframe: Pre Treatment and Post Treatment

Interventionunits on a scale (Mean)
Pre TreatmentPost Treatment
Hematopoietic Stem Cell Transplantation2516

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Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0).

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT03602612)
Timeframe: Date treatment consent signed to date off study, approximately 16 months/17 days, 4 months/4 days, 4 months/18 days, 42 months/8 days, 20 months/19 days, 29 months/28 days, and 30 months/26 days for each Arm/Group respectively.

InterventionParticipants (Count of Participants)
Arm 1 Dose Escalation (Esc) Dose Level 1 - 0.75x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg6
Arm 1 Dose Escalation Dose Level 2 - 1.5x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg3
Arm 1 Dose Escalation Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg2
Arm 1 Dose Escalation Dose Level 4 - 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg3
Arm 1 Dose Escalation Dose Level 5 - 12.0x1^06 Chimeric Antigen Receptor (CAR)+ T Cells Per kg3
Arm 2 Dose Expansion (Exp) 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg8
Arm 1 DoseEsc DL 3-3.0x10^6 CAR+T Cells Per kg Followed by Arm 2 DoseExp 6.0x10^6 CAR+T Cells Per kg1

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Number of Participants at Each Dose Level Who Experience a Dose-Limiting Toxicity (DLT)

A DLT is Grade 3 toxicities possibly or probably or definitely related to the anti-B cell maturation antigen (BCMA) - chimeric antigen receptor (CAR)-T cells and lasting more than 9 days. Grade 4 toxicities possibly or probably or definitely related to the anti-BCMA CAR T cells. Grade 3 is severe, and Grade 4 is life-threatening. (NCT03602612)
Timeframe: First 28 days of treatment

InterventionParticipants (Count of Participants)
Arm 1 Dose Escalation (Esc) Dose Level 1 - 0.75x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg1
Arm 1 Dose Escalation Dose Level 2 - 1.5x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg0
Arm 1 Dose Escalation Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg0
Arm 1 Dose Escalation Dose Level 4 - 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg0
Arm 1 Dose Escalation Dose Level 5 - 12.0x1^06 Chimeric Antigen Receptor (CAR)+ T Cells Per kg0
Arm 2 Dose Expansion (Exp) 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg0
Arm 1 DoseEsc DL 3-3.0x10^6 CAR+T Cells Per kg Followed by Arm 2 DoseExp 6.0x10^6 CAR+T Cells Per kg0

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Maximum Tolerated Dose (MTD) of Anti-B Cell Maturation Antigen (BCMA) - Chimeric Antigen Receptor (CAR)-T Cells

The MTD is the dose at which a maximum of 1 of 6 patients has a DLT. A DLT is Grade 3 toxicities possibly or probably or definitely related to the Anti-B cell maturation antigen (BCMA) - chimeric antigen receptor (CAR)-expressing T-Cells lasting more than 9 days. Grade 4 toxicities possibly or probably or definitely related to the anti-BCMA CAR T cells. Grade 3 is severe, and Grade 4 is life-threatening. (NCT03602612)
Timeframe: First 28 days of treatment

Interventionmillion CAR + T cells (Number)
All Arm 1 Dose Escalation Dose Participants6

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Objective Response Rate (ORR) Per Investigator Review Assessed by International Workshop on CLL (IWCLL) 2018 Criteria

ORR was defined as percentage of participants achieving either complete response (CR), complete response with incomplete hematopoetic recovery (CRi) or partial response (PR). Criteria for CR: no lymphadenopathy >1.5 cm or hepatomegaly/splenomegaly, lymphocytes <4000/microliters (μL), bone marrow sample must be normocellular with 30% lymphocytes and no B-lymphoid nodules, platelets ≥100,000/µL, hemoglobin ≥11 grams per deciliter (g/dL). CRi: All CR criteria were met except with platelet count <100,000/μL, hemoglobin <11 g/dL or neutrophil count <500/μL. PR: ≥1 of these: ≥50% decrease in lymphocytes, lymphadenopathy, size of liver and spleen, 50% decrease in bone marrow infiltrates; and ≥1 of these: platelets ≥100,000/µL or ≥50% increase from Baseline, hemoglobin ≥11 g/dL or ≥50% increase from Baseline. Participants who did not meet criteria were considered nonresponders. 95% confidence interval (CI) was calculated by Clopper-Pearson method. (NCT03624036)
Timeframe: First infusion date up to last follow up visit (maximum duration: 42 months)

Interventionpercentage of participants (Number)
First Stage Cohort 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg50
First Stage Cohort 2: 2 x 10^6 Anti-CD19 CAR T Cells/kg33
Second Stage Cohort 3: 1 x 10^6 Anti-CD19 CAR T Cells/kg100
Second Stage Cohort 4A: 1 x 10^6 Anti-CD19 CAR T Cells/kg0

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Peak Level of Anti-CD19 CAR T-Cells in Blood

Peak was defined as the maximum number of CAR T cells measured post-infusion. (NCT03624036)
Timeframe: First infusion date up to 3 months post-infusion (approximately 3 months)

Interventioncells/μL (Median)
First Stage Cohort 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg1.46
First Stage Cohort 2: 2 x 10^6 Anti-CD19 CAR T Cells/kg1.08
Second Stage Cohort 3: 1 x 10^6 Anti-CD19 CAR T Cells/kg42.18
Second Stage Cohort 4A: 1 x 10^6 Anti-CD19 CAR T Cells/kg1.00

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Percentage of Participants Experiencing Treatment Emergent Adverse Events (TEAEs)

An AE is defined as any untoward medical occurrence in a clinical trial participant that does not necessarily have a relationship with study treatment or worsening of a pre-existing medical condition. TEAEs were defined as AEs with onset on or after the initiation of brexucabtagene autoleucel infusion. (NCT03624036)
Timeframe: First infusion date up to last follow up visit (maximum duration: 42 months)

Interventionpercentage of participants (Number)
First Stage Cohort 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg100
First Stage Cohort 2: 2 x 10^6 Anti-CD19 CAR T Cells/kg100
Second Stage Cohort 3: 1 x 10^6 Anti-CD19 CAR T Cells/kg100
Second Stage Cohort 4A: 1 x 10^6 Anti-CD19 CAR T Cells/kg100

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Number of Participants Experiencing Dose Limiting Toxicities (DLTs)

DLTs refer to toxicities with onset experienced during the first 28 days of study treatment that have been judged to be clinically significant and related to study treatment. DLTs evaluated may include with some exceptions: All brexucabtagene autoleucel related Grade 3 non-hematologic toxicities lasting for more than 7 days, Grade 4 non-hematologic toxicities regardless of duration, and Grade 4 hematologic toxicity lasting more than 30 days if not attributable to underlying disease. (NCT03624036)
Timeframe: First infusion date of brexucabtagene autoleucel up to 28 days. Participants were evaluated in specified period but Grade 4 hematologic toxicity (specified in description) having onset in this period were further observed for 30 days for confirmation.

InterventionParticipants (Count of Participants)
First Stage Cohort 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg0
First Stage Cohort 2: 2 x 10^6 Anti-CD19 CAR T Cells/kg0
Second Stage Cohort 3: 1 x 10^6 Anti-CD19 CAR T Cells/kg1
Second Stage Cohort 4A: 1 x 10^6 Anti-CD19 CAR T Cells/kg0

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Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Value

Grading categories were determined by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionpercentage of participants (Number)
HemoglobinAlanine AminotransferaseAlkaline AminotransferaseAspartate AminotransferaseBilirubinCalciumCreatinineGlucoseMagnesiumSodiumUrate
Axicabtagene Ciloleucel38052055155018

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Percentage of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Value

Grading categories were determined by CTCAE version 5.0. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionpercentage of participants (Number)
HemoglobinLeukocytesLymphocytesNeutrophilsPlateletsAlbuminCalciumGlucoseMagnesiumPotassiumSodium
Axicabtagene Ciloleucel409375952531003523

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Duration of Response (DOR) Per the Lugano Classification

DOR is defined only for participants who experience an objective response after axicabtagene ciloleucel infusion and is the time from the first objective response to disease progression or death from any cause. Objective response is defined in outcome measure (OM) 2. (NCT03761056)
Timeframe: From the date of first confirmed objective response (CR or PR) to disease progression or death regardless of cause (up to approximately 26.2 months)

Interventionmonths (Median)
Axicabtagene CiloleucelNA

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Event-Free Survival (EFS)

EFS was defined as time from axicabtagene ciloleucel infusion date to earliest date of disease progression (Lugano classification), commencement of subsequent new anti-lymphoma therapy including stem cell transplant, or death from any cause. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionmonths (Median)
Axicabtagene CiloleucelNA

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Complete Response (CR) Rate Per the Lugano Classification as Determined by Study Investigators

Complete Response Rate (CRR): percentage of participants with CR [complete metabolic response (CMR); complete radiological response (CRR)]. CMR: positron emission tomography (PET) 5-point scale (5-PS) scores of 1 (no uptake above background), 2 (uptake ≤ mediastinum), 3 (uptake > mediastinum but ≤ liver) with/without a residual mass); no new lesions; and no evidence of fluorodeoxyglucose (FDG)-avid disease in bone marrow (BM). CRR: target nodes/nodal masses regressed to ≤ 1.5 cm in longest transverse diameter of lesion (LDi); no extralymphatic sites of disease; absent non-measured lesion (NMLs); organ enlargement regress to normal; no new sites; and bone marrow normal by morphology. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (maximum duration: 26.2 months)

Interventionpercentage of participants (Number)
Axicabtagene Ciloleucel78

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Objective Response Rate (ORR) Per the Lugano Classification as Determined by Study Investigators

ORR: percentage of participants with CR [CMR;CRR] or PR [partial metabolic response (PMR); partial radiologic response (PRR)].CMR: PET 5PS scores of 1 (no uptake above background, 2 (uptake ≤ mediastinum), 3 (uptake > mediastinum but ≤ liver) with/without a residual mass; no new lesions; no evidence of FDG-avid disease in BM. CRR:target nodes/nodal masses regressed to ≤ 1.5 cm in LDi;no extralymphatic sites of disease;absent NMLs;organ enlargement regress to normal;no new sites;bone marrow morphology normal. PMR:scores 4 (uptake moderately > liver),5 (uptake markedly > liver, new lesions) with reduced uptake compared with baseline and residual mass;no new lesions;responding disease at interim/residual disease at end of treatment (EOT).PRR: ≥ 50% decrease in sum of the product of perpendicular diameters (SPD) of up to 6 target measurable nodes and extra-nodal sites;absent/normal, regressed, but no increase of NMLs;spleen regressed by > 50% in length beyond normal;no new sites. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (maximum duration: 26.2 months)

Interventionpercentage of participants (Number)
Axicabtagene Ciloleucel89

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Time to Peak Serum Level of Granzyme B, Interferon-gamma (IFNg), Interleukin (IL)-2, IL-5, IL-6, IL-8, CRP, and Ferritin

Time to peak is defined as the number of days from axicabtagene ciloleucel infusion to the date when the cytokine first reached the maximum post-baseline level. (NCT03761056)
Timeframe: From enrollment up to Week 4

Interventiondays (Median)
Granzyme BIFNgIL-2IL-5IL-6IL-8CRPFerritin
Axicabtagene Ciloleucel84418848

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Overall Survival (OS)

OS is defined as the time from axicabtagene ciloleucel infusion to the date of death from any cause. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionmonths (Median)
Axicabtagene Ciloleucel24.5

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Peak Serum Level of Granzyme B, Interferon-gamma (IFNg), Interleukin (IL)-2, IL-5, IL-6, IL-8

Peak is defined as the maximum post-baseline level of cytokine from baseline to Week 4. (NCT03761056)
Timeframe: From enrollment up to Week 4

Interventionpg/mL (Median)
Granzyme BIFNgIL-2IL-5IL-6IL-8
Axicabtagene Ciloleucel28.5409.416.46.335.163.0

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Percentage of Participants With Treatment-Emergent Adverse Events (TEAE) and Treatment-Emergent Serious Adverse Events (SAE)

An AE was any untoward medical occurrence in a participant in a clinical trial participant, which did not necessarily have a causal relationship with the treatment. Treatment-emergent adverse events were defined as any adverse event with onset on or after the axicabtagene ciloleucel infusion. Serious adverse event was defined as an event that resulted in the following: death; life-threatening situation; in-patient hospitalization or prolongation of existing hospitalization; persistent or significant disability or incapacity; congenital anomaly or birth defect; and medically important event or reaction. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionpercentage of participants (Number)
TEAETreatment-Emergent SAE
Axicabtagene Ciloleucel10045

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Peak Serum Level of C-Reactive Protein (CRP)

Peak is defined as the maximum post-baseline level of cytokine from baseline to Week 4. (NCT03761056)
Timeframe: From enrollment up to Week 4

Interventionmg/L (Median)
Axicabtagene Ciloleucel208.4

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Peak Serum Level of Ferritin

Peak is defined as the maximum post-baseline level of cytokine from baseline to Week 4. (NCT03761056)
Timeframe: From enrollment up to Week 4

Interventionng/mL (Median)
Axicabtagene Ciloleucel749.1

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Pharmacokinetics: Peak Level of Anti-CD19 CAR T Cells in Blood

Peak was defined as the maximum number of CAR T cells in blood measured after infusion. (NCT03761056)
Timeframe: From enrollment up to Month 24

Interventioncells/µL (Median)
Axicabtagene Ciloleucel36.27

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Progression-Free Survival (PFS)

PFS is defined as the time from the axicabtagene ciloleucel infusion date to the date of disease progression per Lugano classification or death from any cause. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionmonths (Median)
Axicabtagene CiloleucelNA

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Relapse With Central Nervous Disease (CNS) Disease

Relapse with CNS disease was defined as the time from the axicabtagene ciloleucel infusion date to the earliest date of CNS involvement with lymphoma as determined by typical symptoms, cerebrospinal fluid (CSF) evaluation, and/or diagnostic imaging. (NCT03761056)
Timeframe: First infusion date of axicabtagene ciloleucel to data cut off date of 17 May 2021 (Up to approximately 26.2 months)

Interventionmonths (Median)
Axicabtagene Ciloleucel0

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Elimination Half-life of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS

Median (Range) of the elimination half-life of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level measured pre-dose, end of infusion, 4-6 hours, and 24 hours post-dose infusion on Days 1 and 5. (NCT03813147)
Timeframe: Up to 5 days

InterventionHour (Median)
Stratum 1 With 20 mg/m^24.7
PK With 20 mg/m^25.5

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Maximum Time to Concentration of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS

Median (Range) of the maximum time to concentration of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level measured pre-dose, end of infusion, 4-6 hours, and 24 hours post-dose infusion on Days 1 and 5. (NCT03813147)
Timeframe: Up to 5 days

InterventionHour (Median)
Stratum 1 With 20 mg/m^21.2
PK With 20 mg/m^21.1

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Total Plasma Clearance of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS

Median (Range) of the total plasma clearance of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level measured pre-dose, end of infusion, 4-6 hours, and 24 hours post-dose infusion on Days 1 and 5. (NCT03813147)
Timeframe: Up to 5 days

InterventionL/h/m^2 (Median)
Stratum 1 With 20 mg/m^220.1
PK With 20 mg/m^219.2

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Maximum Concentration of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS

Median (Range) of the maximum concentration of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level measured pre-dose, end of infusion, 4-6 hours, and 24 hours post-dose infusion on Days 1 and 5. (NCT03813147)
Timeframe: Up to 5 days

Interventionng/mL (Median)
Stratum 1 With 20 mg/m^2248.5
PK With 20 mg/m^2213

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Number of Participants With Adverse Events Attributable to MLN4924 (Pevonedistat)

Frequency of patients with at least one grade 3 adverse event at least possibly attributable to MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level. (NCT03813147)
Timeframe: Up to 70 days

InterventionParticipants (Count of Participants)
Stratum 1 With 20 mg/m^26
PK With 20 mg/m^26

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Number of Participants With Antitumor Activity of MLN4924 (Pevonedistat)

Frequency of participants with best overall response by dose level of PR or CR for MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction Per Response Evaluation Criteria for CR (M1 bone marrow (< 5% blasts) with no evidence of circulating blasts or extramedullary disease and with recovery of peripheral blood counts (ANC > 1000/uL and platelet count > 100,000/uL), CRp (M1 bone marrow (< 5% blasts) and no evidence of circulating blasts or extramedullary disease and with recovery of ANC > 1000/uL and platelet transfusion independence), CRi (M1 bone marrow (<5% blasts) and no evidence of circulating blasts or extramedullary disease and with ANC < 1000/uL or platelet count < 100,000/uL without platelet transfusion independence), or PR (M2 marrow status (> 5% or < 25% blasts cells) and at least 50% decrease in bone marrow blast percent from baseline. (NCT03813147)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Stratum 1 With 20 mg/m^21
PK With 20 mg/m^22

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Number of Participants With Dose Limiting Toxicities of MLN4924 (Pevonedistat)

Frequency of patients with dose limiting toxicity in the first cycle of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level among patients in the dose escalation cohort. (NCT03813147)
Timeframe: Up to 35 days

InterventionParticipants (Count of Participants)
Stratum 1 With 20 mg/m^21
PK With 20 mg/m^22

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Area Under the Plasma Concentration Versus Time Curve of MLN4924 (Pevonedistat) Added to the 3-drug Backbone of Azacitidine (Aza), Fludarabine, and Cytarabine Re-induction for Pediatric Patients With Recurrent/Refractory AML and MDS

Median (Range) of the area under the plasma concentration versus time curve of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine, and cytarabine re-induction for pediatric patients with recurrent/refractory AML and MDS by dose level measured pre-dose, end of infusion, 4-6 hours, and 24 hours post-dose infusion on Days 1 and 5. (NCT03813147)
Timeframe: Up to 5 days

Interventionhr*ng/mL (Median)
Stratum 1 With 20 mg/m^21004.9
PK With 20 mg/m^21047.4

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AML: AUC of Isatuximab

Plasma samples were collected at specified timepoints to determine the AUC of isatuximab. (NCT03860844)
Timeframe: From Week 0 to Week 1, Week 0 to Week 3, and Week 0 to Week 8

Interventionmg*h/L (Mean)
Week 0 to Week 1Week 0 to Week 3Week 0 to Week 8
Acute Myeloid Leukemia (AML)28592130862291962

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Percentage of Participants With Complete Response (CR) Rate

The complete response rate (CR + CRi [complete response with incomplete peripheral recovery]) was defined as the percentage of participants achieving complete response (CR + CRi) assessed by the investigator per National Comprehensive Cancer Network (NCCN) guidelines version 1.2018 criteria. CR was defined as <5% blasts in a bone marrow aspirate (BMA) with spicules; no circulating blasts (ALL)/no blasts with Auer rods (AML) or extramedullary disease, no lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/central nervous system involvement (ALL), trilineage hematopoiesis (ALL); Absolute neutrophil count (ANC) >=1000/microliter (mcL); platelets >100000/mcL; red blood cell transfusion independence. If the physician documented transfusion dependency related to study treatment and not to the participant's underlying disease, CRi was reported. CRi met the same criteria as for CR, except neutrophils and/or platelets recovery (ANC <1000/mcL or platelets <100000/mcL). (NCT03860844)
Timeframe: From enrollment until the primary analysis completion date of 12 Sep 2022; the median duration of exposure was approximately 7 weeks

Interventionpercentage of participants (Number)
B-cell Acute Lymphoblastic Leukemia (B-ALL)52.0
T-cell Acute Lymphoblastic Leukemia (T-ALL)45.5
Acute Myeloid Leukemia (AML)60.9

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Overall Response Rate (ORR)

ORR:Percentage of participants with CR/CRi or partial response (PR) for blood and bone marrow disease based on NCCN guideline. CR: <5% blasts in BMA with spicules; no circulating blasts (ALL)/no blasts with Auer rods (AML) or extramedullary disease, no lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/central nervous system involvement (ALL), trilineage hematopoiesis (ALL);ANC >=1000/mcL; platelets >100000/mcL; RBC transfusion independence. If the physician documented transfusion dependency related to study treatment;not to participant's underlying disease, CRi was reported. CRi met the same criteria as CR, except neutrophils and/or platelets recovery (ANC <1000/mcL or platelets <100000/mcL). PR: >50% decrease in the sum of the product of the greatest perpendicular diameters of the mediastinal enlargement. For participants with a previous positive positron emission tomography (PET) scan, a post-treatment PET was to be positive in at least 1 previously involved site. (NCT03860844)
Timeframe: From enrollment until the primary analysis completion date of 12 Sep 2022; the median duration of exposure was approximately 7 weeks

Interventionpercentage of participants (Number)
B-cell Acute Lymphoblastic Leukemia (B-ALL)52.0
T-cell Acute Lymphoblastic Leukemia (T-ALL)54.5
Acute Myeloid Leukemia (AML)65.2

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Number of Participants With Infusion Reactions (IRs)

An IR was an AE related to isatuximab typically with onset within 24 hours from the start of the isatuximab infusion and was reported by the investigator. (NCT03860844)
Timeframe: From the time of the first treatment administration (Day 1) up to 30 days after the last treatment (maximum duration of exposure of 13.1 weeks for B-ALL cohort, 10.7 weeks for T-ALL cohort and 7.1 weeks for AML cohort)

InterventionParticipants (Count of Participants)
B-cell Acute Lymphoblastic Leukemia (B-ALL)9
T-cell Acute Lymphoblastic Leukemia (T-ALL)5
Acute Myeloid Leukemia (AML)15

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CD38 Receptor Occupancy

Blood samples were collected to assess CD38 receptor occupancy as a pharmacodynamics marker. It was assessed across complete responders and non-complete responders. Multicolor flow cytometry assay was validated for CD38 receptor occupancy (CD38RO) quantification, based on the use of two murine monoclonal antibodies (MAbs), one competing with SAR650984 to determine the number of free CD38 receptors (MAb1) and one recognizing a different binding epitope on CD38 to measure the total number of receptors (MAb2) at the cell surface of the cancer cells. Cells were tagged with either MAb1 (Tube #1) or MAb2 (Tube #2). The percentage RO was calculated using the following equation: % CD38RO = [(sABC MAb2 - sABC MAb1)/sABC MAb2] X 100. (NCT03860844)
Timeframe: Pre-dose on Day 15

Interventionpercent receptor occupancy (Mean)
Blood plasma cells: Non CR/CRi;Blood NK cells: CR/CRiBlood NK cells: Non CR/CRi
B-cell Acute Lymphoblastic Leukemia (B-ALL)44.055.661.3

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Cluster of Differentiation (CD)38 Receptor Density

"Blood samples were collected to assess CD38 receptor density as a predictive biomarker. It was assessed across complete responders and non-complete responders. The Antibody Binding Capacity (ABC) was calculated using the following equation: ABC = 10^(Logarithm(Mean Fluorescence Intensity)*a+b) where a was the slope and b was the Y-intercept of the calibration curve equation. Specific and absolute quantitative values (specific antibody-binding capacity [sABC]) of binding of the selected antibodies were calculated after subtraction of the negative isotypic immunoglobulin G (IgG) control." (NCT03860844)
Timeframe: Pre-dose on Day 1

,,
InterventionsABC (Mean)
Blood blast cells: CR/CRiBlood blast cells: Non CR/CRi;Blood immune cells (Natural Killer [NK] cells): CR/CRiBlood immune cells (NK cells): Non CR/CRi
Acute Myeloid Leukemia (AML)19502.09815.011220.322530.0
B-cell Acute Lymphoblastic Leukemia (B-ALL)20345.631080.013506.216650.0
T-cell Acute Lymphoblastic Leukemia (T-ALL)12780.022952.022639.033859.0

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Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

An AE was defined as any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. SAEs were any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as an AE which occurred after the first dose of study treatment administration until the last dose plus 30 days, or until the start of hematological recovery period or a new anti-leukemia/lymphoma therapy, whichever occurred first. (NCT03860844)
Timeframe: From the time of the first treatment administration (Day 1) up to 30 days after the last treatment (maximum duration of exposure of 13.1 weeks for B-ALL cohort, 10.7 weeks for T-ALL cohort and 7.1 weeks for AML cohort)

,,
InterventionParticipants (Count of Participants)
Any TEAEAny TESAE
Acute Myeloid Leukemia (AML)2616
B-cell Acute Lymphoblastic Leukemia (B-ALL)2719
T-cell Acute Lymphoblastic Leukemia (T-ALL)1312

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CD38 Receptor Occupancy

Blood samples were collected to assess CD38 receptor occupancy as a pharmacodynamics marker. It was assessed across complete responders and non-complete responders. Multicolor flow cytometry assay was validated for CD38 receptor occupancy (CD38RO) quantification, based on the use of two murine monoclonal antibodies (MAbs), one competing with SAR650984 to determine the number of free CD38 receptors (MAb1) and one recognizing a different binding epitope on CD38 to measure the total number of receptors (MAb2) at the cell surface of the cancer cells. Cells were tagged with either MAb1 (Tube #1) or MAb2 (Tube #2). The percentage RO was calculated using the following equation: % CD38RO = [(sABC MAb2 - sABC MAb1)/sABC MAb2] X 100. (NCT03860844)
Timeframe: Pre-dose on Day 15

Interventionpercent receptor occupancy (Mean)
Blood plasma cells: CR/CRiBlood plasma cells: Non CR/CRi;Blood NK cells: CR/CRiBlood NK cells: Non CR/CRi
T-cell Acute Lymphoblastic Leukemia (T-ALL)40.555.066.770.0

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B-ALL and T-ALL: Concentrations at the End of Infusion (Ceoi) of Isatuximab

Ceoi is the plasma concentration observed at the end of intravenous infusion of isatuximab. (NCT03860844)
Timeframe: At end of infusion on Cycle 1 Days 1 and 29

,
Interventionmcg/mL (Mean)
Cycle 1: Day 1Cycle 1: Day 29
B-cell Acute Lymphoblastic Leukemia (B-ALL)452835
T-cell Acute Lymphoblastic Leukemia (T-ALL)259745

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B-ALL and T-ALL: Area Under the Concentration Time Curve (AUC) of Isatuximab

Plasma samples were collected at specified timepoints to determine the AUC of isatuximab. (NCT03860844)
Timeframe: From Week 0 to Week 1, Week 0 to Week 5, and Week 0 to Week 10

,
Interventionmg*hour (h)/Liter (L) (Mean)
Week 0 to Week 1Week 0 to Week 5Week 0 to Week 10
B-cell Acute Lymphoblastic Leukemia (B-ALL)31703299071582686
T-cell Acute Lymphoblastic Leukemia (T-ALL)29057289167540375

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AML: Ceoi of Isatuximab

Ceoi is the plasma concentration observed at the end of intravenous infusion of isatuximab. (NCT03860844)
Timeframe: At end of infusion on Cycle 1 Days 1 and 15

Interventionmcg/mL (Mean)
Cycle 1: Day 1Cycle 1: Day 15
Acute Myeloid Leukemia (AML)363562

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Number of Participants Experiencing a Dose-limiting Toxicity (DLT)

Defined by any grade 3 or NCI CTCAE toxicities and modified CRS grading as applicable. (NCT03873805)
Timeframe: Up to 28 days post treatment

InterventionParticipants (Count of Participants)
Dose Level 10
Dose Level 22
Dose Level 30

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Grade 3 Toxicity Profile

Grade 3 toxicity profile as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v)5 and modified Cytokine Release Syndrome (CRS) grading as applicable post chimeric antigen receptor (CAR) T cell infusion. (NCT03873805)
Timeframe: Up to 32 months

,,
InterventionParticipants (Count of Participants)
Anemia : YesAnemia : NoLymphocyte count decreased : YesLymphocyte count decreased : NoFatigue : YesFatigue : NoPain : YesPain : NoCystitis noninfective : YesCystitis noninfective : NoHematuria : YesHematuria : NoRash maculo-papular : YesRash maculo-papular : No
Dose Level 121120303030303
Dose Level 224062415241515
Dose Level 305140505050505

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Phase 1A: Percentage of Participants Experiencing Adverse Events Defined as Dose-Limiting Toxicities (DLTs)

A DLT is defined as protocol-defined KITE-439 related Grade 3 events with onset within the first 21 days following KITE-439 infusion and which do not resolve to ≤Grade 2 events within 48 hours, ≥Grade 4 events with onset within the first 21 days following KITE-439 infusion, regardless of duration. (NCT03912831)
Timeframe: First infusion date of KITE-439 up to 21 days

Interventionpercentage of participants (Number)
Phase 1A: 1 x 10^6 KITE-439 (Cohort 1)0
Phase 1A: 3 x 10^6 KITE-439 (Cohort 2)0
Phase 1A: 1 x 10^7 KITE-439 (Cohort 3)0
Phase 1A: 3 x 10^7 KITE-439 (Cohort 4)0
Phase 1A: 1 x 10^8 KITE-439 (Cohort 5)0
Phase 1A: 1 x 10^8 KITE-439 (Cohort 6)0

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Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT03958656)
Timeframe: Date treatment consent signed to date off study, approximately 3 months and 27 days for Level 1, 3 months and 18 days for Level 2, 5 months and 23 days for Level 3, and 1 month and 29 days for Level 4.

InterventionParticipants (Count of Participants)
LEVEL 1 - 0.66x10^6 Per Kilogram (kg)3
LEVEL 2 - 2.0x10^6 Per kg3
LEVEL 3 - 6.0x10^6 Per kg3
LEVEL 4 - 12.0x10^6 Per kg1

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Number of Participants That Had Any Grade ≤2, and 3, 4 and 5 Adverse Events Following Administration of T Cells Expressing Anti- Signaling Lymphocytic Activation Molecule F7 (SLAMF7) Chimeric Antigen Receptor (CAR)

Adverse Events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life-threatening or disabling, and Grade 5 is fatal. (NCT03958656)
Timeframe: Date treatment consent signed to date off study, approximately 3 months and 27 days for Level 1, 3 months and 18 days for Level 2, 5 months and 23 days for Level 3, and 1 month and 29 days for Level 4.

,,,
InterventionParticipants (Count of Participants)
< Grade 2Grade 2Grade 3Grade 4Grade 5
LEVEL 1 - 0.66x10^6 Per Kilogram (kg)03330
LEVEL 2 - 2.0x10^6 Per kg03330
LEVEL 3 - 6.0x10^6 Per kg13330
LEVEL 4 - 12.0x10^6 Per kg01110

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Number of Participants With a Response

Response was assessed by the International Uniform Response Criteria for Multiple myeloma 2016 updated version. Complete Remission (CR) is negative immunofixation on the serum and urine, and disappearance of any soft tissue plasmacytomas. Very Good Partial Remission (VGPR) is serum and urine M-protein detectable by immunofixation but not on electrophoresis, or 90% or greater reduction in serum M-protein plus urine M-protein level <100mg per 24 h. Partial Remission (PR) is 50% or greater reduction of serum M-protein and 90% or greater reduction in 24-h urinary M-protein (or to less than 200mg per 24 h). Progressive Disease (PD) is serum M-component (minimum absolute increase of 0.5g/dL), or urine M-component (minimum absolute increase of 200mg/24h). Stable Disease (SD) is not meeting criteria for CR, VGPR, PR or progressive disease. (NCT03958656)
Timeframe: At two and five weeks for stable disease and partial remission, respectively, and up to 5 months and 23 days for progressive disease

,,,
InterventionParticipants (Count of Participants)
Partial Remission at 5 WeeksStable Disease at 2 WeeksProgressive Disease
LEVEL 1 - 0.66x10^6 Per Kilogram (kg)021
LEVEL 2 - 2.0x10^6 Per kg111
LEVEL 3 - 6.0x10^6 Per kg021
LEVEL 4 - 12.0x10^6 Per kg010

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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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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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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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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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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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Objective Response Rate

Objective response rate (ORR) is a measure of clinical activity as response in NHL by the revised Lugano Classification (Cheson et al, 2016) or a response in CLL/SLL by the International Workshop on Chronic Lymphocytic Leukemia 2018 guidelines. (NCT04030195)
Timeframe: 1 year

,,
InterventionParticipants (Count of Participants)
RespondersComplete ResponsePartial ResponseNon-responders
Dose Level 1 of PBCAR20A CAR T Cells2116
Dose Level 2 of PBCAR20A CAR T Cells1012
Dose Level 3 of PBCAR20A CAR T Cells3034

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Maximum Tolerated Dose (MTD)

The maximum tolerated dose (MTD) is the dose level at which fewer than 33% of patients experience a dose limiting toxicity (DLT) using a 3+3 strategy. (NCT04030195)
Timeframe: Day 1 to Day 28

Intervention10^6 CAR T cells (Number)
PBCAR20A CAR T CellsNA

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Number of Participants With Dose-Limiting Toxicities

Dose-limiting toxicities (DLT) are certain Grade 3 and Grade 4 toxic reactions as defined by the protocol and CTCAE v5.0. (NCT04030195)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Dose Level 1 of PBCAR20A CAR T Cells1
Dose Level 2 of PBCAR20A CAR T Cells0
Dose Level 3 of PBCAR20A CAR T Cells0

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Progression-free Survival (PFS)

Progression-free survival is defined as the duration (days) from Day 0 to disease progression or death. (NCT04030195)
Timeframe: 1 year

InterventionDays (Median)
Dose Level 1 of PBCAR20A CAR T Cells29.5
Dose Level 2 of PBCAR20A CAR T Cells29.0
Dose Level 3 of PBCAR20A CAR T Cells29.0

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Last Time-Point at Which Chimeric Antigen Receptors (CAR) T Cells Were Detected in the Blood

Chimeric Antigen Receptors (CAR) T cell persistence was measured in the blood by quantitative polymerase chain reaction (PCR). CAR T cells that are detected in the participant's blood that persist for a significant length of time is a positive finding. (NCT04160195)
Timeframe: 119 days after CAR T-cell infusion

InterventionDays (Number)
1/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Dose Escalation119

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Maximum Tolerated Dose (MTD) of Chimeric Antigen Receptors (CAR) T Cells

The maximum tolerated dose is the dose at which a maximum of 1 of 6 participants has a dose-limiting toxicity (DLT). A DLT are defined as toxicities that are possibly, probably, or definitely attributable to protocol interventions and occurring between the first protocol treatment through 28 days after the CAR T-cell infusion. (NCT04160195)
Timeframe: First protocol treatment through 28 days after the CAR T-cell infusion.

InterventionT cells (Number)
All ParticipantsNA

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT04160195)
Timeframe: Date treatment consent signed to date off study, approximately 7 months and 18 days.

InterventionParticipants (Count of Participants)
1/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Dose Escalation1
2/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Expansion Phase0

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Percentage of Peak Blood Chimeric Antigen Receptors (CAR) T Cells

We measured CAR T-cell persistence by detecting the CAR gene in peripheral blood mononuclear cells (PBMC) by polymerase chain reaction (PCR). (NCT04160195)
Timeframe: 119 days after CAR T-cell infusion

Interventionpercentage of PBMC (Number)
1/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Dose Escalation10.5

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Percentage of Peripheral Blood Mononuclear Cells (PBMC) of Chimeric Antigen Receptors (CAR) T Cells

Peak blood levels of Chimeric Antigen Receptors (CAR) T cells were measured by exact Wilcoxon rank sum test. (NCT04160195)
Timeframe: pretreatment and multiple days from day 1 to day 173 after infusion.

Interventionpercentage of PBMC (Number)
1/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Dose Escalation10.47

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Number of Participants Administered T Cells Expressing a Novel Fully- Human Anti-cluster of Differentiation 19 (CD19) and Anti-cluster of Differentiation 20 (CD20) Chimeric Antigen Receptors (CAR) Who Experienced a Dose-limiting Toxicity (DLT)

A DLT are defined as toxicities assessed by the Common Terminology Criteria for Adverse Events v5.0 that are possibly, probably, or definitely attributable to protocol interventions and occurring between the first protocol treatment through 28 days after the CAR T-cell infusion. (NCT04160195)
Timeframe: First protocol treatment through 28 days after the CAR T-cell infusion.

,
InterventionParticipants (Count of Participants)
Peripheral Motor Neuropathy Possibly RelatedGuillain-Barre Syndrome Possibly Related
1/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Dose Escalation11
2/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Expansion Phase00

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Number of Participants With Clinical Response

Response for lymphoma was assessed by the Revised Response Criteria for Malignant Lymphoma and The Lugano Classification. Complete Remission (CR) is complete disappearance of all detectable clinical evidence of disease. Partial Remission (PR) is ≥ 50% decrease in nodes or masses. Progressive Disease (PD) is Response ≥ 50% increase in a single node. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor PD. For participants with Chronic Lymphocytic Leukemia (CLL),response was assessed by the International Workshop on CLL. CR is no lymph nodes ≥ 1.5 cm on physical exam or relevant computed tomography. PR is a ≥ 50% decrease in peripheral B lymphocyte count from pre-treatment value. PD is a ≥ 50% increase in the greatest diameter of any lymph node that was enlarged pretreatment. And SD are participants who do not fulfill the criteria for CR, PR or PD. (NCT04160195)
Timeframe: Approximately 1 year 5 months

InterventionParticipants (Count of Participants)
Complete RemissionPartial RemissionProgressive DiseaseStable Disease
1/Conditioning Chemotherapy Plus Chimeric Antigen Receptors (CAR) T-cells Dose Escalation0100

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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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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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Progression Free Survival (PFS)

Kaplan-Meier curve will be generated for PFS. (NCT04339101)
Timeframe: From the date of stem cell infusion to the date of death, disease relapse/progression, or last follow-up, whichever occurs first, assessed at 1 year post transplant

Interventionpercentage of probability (Number)
Treatment (Itacitinib Adipate, Tacrolimus, Sirolimus)70

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Cumulative Incidence of Grade II-IV Acute GVHD

Acute GVHD will be graded and staged according to the Consensus Grading. (NCT04339101)
Timeframe: From day 0 (date of stem cell infusion) through 100 days post-transplant

Interventionpercentage of probability (Number)
Treatment (Itacitinib Adipate, Tacrolimus, Sirolimus)4

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Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year

GRFS is defined as time from the date of transplantation to the first time of observing following events: grade 3-4 acute graft versus host disease (GVHD), chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier curve will be generated for GRFS. (NCT04339101)
Timeframe: From the date of transplantation to the first time of observing following events: grade 3-4 acute graft versus host disease (GVHD), chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first, assessed at 1 year post transplant.

Interventionpercentage of probability (Number)
Treatment (Itacitinib Adipate, Tacrolimus, Sirolimus)56

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Proportion of Failure of the Haplo-Graft

Proportion of patients with a failed haplo-graft, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to <0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation (second nadir) (NCT04395222)
Timeframe: 21 days post-transplant

InterventionParticipants (Count of Participants)
ATG Group I2
ATG Group II5
ATG Group III1

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Percentage of Subjects With Successful Haplo-derived Neutrophil Engraftment

"This is defined as:~Achieve an absolute neutrophil count (ANC) of 500 cells/microL for three consecutive days with the first on or prior to Day +21 post-transplant, AND~Absence of a second nadir - a drop in the ANC to <300 cells/microL for five consecutive days - after initial neutrophil recovery." (NCT04395222)
Timeframe: 21 days post-transplant

Interventionpercentage of participants (Number)
ATG Group I80
ATG Group II50
ATG Group III0

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Participants That Displayed Transgenic T Cells in Tumor Tissue

Outcome will be reported as a count of participants that displayed transgenic T cells in their tumor tissue after treatment. This was assessed by WRPE staining and scRNA sequencing. Unfortunately, no transgenic cells were detected on the one treated patient's tumors. (NCT04639245)
Timeframe: 1 year post infusion

InterventionParticipants (Count of Participants)
Treatment (FH-MagIC TCR-T Cells, Atezolizumab)0

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Overall Survival

Outcome will be reported as a count of participants that were alive at the 1 year post infusion timepoint. (NCT04639245)
Timeframe: 1 year post infusion

InterventionParticipants (Count of Participants)
Treatment (FH-MagIC TCR-T Cells, Atezolizumab)0

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Peripheral Blood Concentration of Infused Transgenic T Cells Over Time

Results will be reported by sample for the single treated patient. Patient samples were tested using a WPRE assay showing transgenic T cells in blood. This was detected by qPCR. (NCT04639245)
Timeframe: 1 year post infusion

InterventionWPRE copies/Cell (Number)
BU712PRETXBU712I1D000BU712I1D001BU712I1D003BU712I1D007BU712I1D014BU712I1D021BU712I1D028BU712I1D056BU712I1D180BU712I2D03BU712I2D07BU712I2D14BU712I2D21BU712I2D28BU712I2D56BU712I2D84
Treatment (FH-MagIC TCR-T Cells, Atezolizumab)0.00.00.01010.03030.03790.03790.01110.00870.01070.00620.11670.07950.03530.02670.03330.00270.0070

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Progression-free Survival

Outcome will be reported as a count of participants that were alive as the 1 year post infusion timepoint and also had not experienced progression at that timepoint. (NCT04639245)
Timeframe: 1 year post infusion

InterventionParticipants (Count of Participants)
Treatment (FH-MagIC TCR-T Cells, Atezolizumab)0

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Objective Response Rates

Evaluated by immune-related RECIST criteria. Outcome will be reported as a count of participants that experienced a Complete Response or Partial Response per RECIST criteria. A complete response (CR) will be defined as total regression of all tumors, a Partial Response as 30% or greater decrease in the sum of the longest diameter of target lesions compared to baseline and Progressive Disease as 20% increase in the sum of the longest diameter of target lesions compared to the smallest prior diameter. If the disease does not fall in either category it will be considered Stable Disease (RECIST v1.1 criteria). (NCT04639245)
Timeframe: 1 year post infusion

InterventionParticipants (Count of Participants)
Treatment (FH-MagIC TCR-T Cells, Atezolizumab)0

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Time to Cmax (Tmax)

Tmax was defined as time to reach Cmax, determined directly from the persistence-time data. Blood samples were collected for PK analysis. (NCT05993299)
Timeframe: Day 1 to Day 14

InterventionDays (Median)
Letetresgene Autoleucel (Lete-cel)6.90

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Overall Response Rate (ORR)

ORR is defined as the percentage of participants with a confirmed complete response (CR) or confirmed partial response (PR) via investigator assessment per Response Evaluation Criteria in Solid Tumors Criteria (RECIST) version 1.1 relative to the total number of participants in the analysis population. CR is defined as disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 millimeters (mm). PR is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference, the baseline sum of the diameters (e.g., percent change from baseline). 95% CI is based on Clopper-Pearson exact confidence interval. (NCT05993299)
Timeframe: Up to approximately 36 months

Interventionpercentage of participants (Number)
Letetresgene Autoleucel (Lete-cel)80

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Maximum Transgene Expansion (Cmax)

Cmax was defined as maximum observed persistence, determined directly from the persistence-time data. Blood samples were collected for PK analysis. (NCT05993299)
Timeframe: Day 1 to Day 14

InterventionCopies per microgram genomic DNA (Geometric Mean)
Letetresgene Autoleucel (Lete-cel)119701.64

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Disease Control Rate (DCR)

DCR is defined as the percentage of participants with a confirmed CR, PR, or stable disease (SD) with a minimal 12 weeks (84 days ± 7 day window) duration relative to the total number of participants within the analysis population at the time of primary analysis as determined by local investigators per RECIST v1.1. CR is defined as disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<)10 millimeters (mm). PR is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference, the baseline sum of the diameters (e.g., percent change from baseline). SD is defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease. The disease progression (PD) is defined as the date of radiological disease progression based on imaging data per RECIST v1.1. 95% CI is based on Clopper-Pearson exact confidence interval. (NCT05993299)
Timeframe: Up to approximately 36 months

InterventionPercentage of Participants (Number)
Letetresgene Autoleucel (Lete-cel)80.0

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Area Under the Time Curve From Zero to Time 28 Days (AUC[0-28])

Area under the persistence-time curve from time zero to Day 28. Blood samples were collected for PK analysis. (NCT05993299)
Timeframe: Up to 28 days

InterventionDays*copies per microgram genomic DNA (Geometric Mean)
Letetresgene Autoleucel (Lete-cel)1911782.96

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